0001438533-23-000008 | 10-K | XBRL Viewer

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Cover Page - USD ($)	12 Months Ended
Cover Page - USD ($)	Dec. 31, 2022	Feb. 21, 2023	Jun. 30, 2022
Cover [Abstract]
Document Type	10-K
Document Annual Report	true
Document Period End Date	Dec. 31, 2022
Current Fiscal Year End Date	--12-31
Document Transition Report	false
Entity File Number	001-36257
Entity Registrant Name	TRAVERE THERAPEUTICS, INC.
Entity Incorporation, State or Country Code	DE
Entity Tax Identification Number	27-4842691
Entity Address, Address Line One	3611 Valley Centre Drive
Entity Address, Address Line Two	Suite 300
Entity Address, City or Town	San Diego
Entity Address, State or Province	CA
Entity Address, Postal Zip Code	92130
City Area Code	888
Local Phone Number	969-7879
Title of 12(b) Security	Common Stock, par value $0.0001 per share
Trading Symbol	TVTX
Security Exchange Name	NASDAQ
Entity Well-known Seasoned Issuer	Yes
Entity Voluntary Filers	No
Entity Current Reporting Status	Yes
Entity Interactive Data Current	Yes
Entity Filer Category	Large Accelerated Filer
Entity Small Business	false
Entity Emerging Growth Company	false
ICFR Auditor Attestation Flag	true
Entity Shell Company	false
Entity Public Float			$ 1,538,771,266
Entity Common Stock, Shares Outstanding		64,744,990
Documents Incorporated by Reference	DOCUMENTS INCORPORATED BY REFERENCE: Portions of the Proxy Statement for the registrant’s 2023 Annual Meeting of Stockholders, to be filed within 120 days after the conclusion of the registrant's fiscal year ended December 31, 2022, are incorporated by reference into Part III of this Annual Report on Form 10-K.
Entity Central Index Key	0001438533
Amendment Flag	false
Document Fiscal Year Focus	2022
Document Fiscal Period Focus	FY

Audit Information	12 Months Ended
Audit Information	Dec. 31, 2022
Audit Information [Abstract]
Auditor Firm ID	243
Auditor Name	BDO USA, LLP
Auditor Location	San Diego, California

CONSOLIDATED BALANCE SHEETS - USD ($) $ in Thousands	Dec. 31, 2022	Dec. 31, 2021
Current assets:
Cash and cash equivalents	$ 61,688	$ 165,753
Marketable debt securities, at fair value	388,557	387,129
Accounts receivable, net	16,646	15,914
Inventory, net	6,922	7,313
Prepaid expenses and other current assets	12,624	6,718
Total current assets	486,437	582,827
Property and equipment, net	9,049	11,106
Operating lease right of use assets	21,000	23,196
Intangible assets, net	145,038	148,435
Other assets	11,061	11,069
Total assets	672,585	776,633
Current liabilities:
Accounts payable	17,290	15,144
Accrued expenses	95,742	75,180
Deferred revenue, current portion	11,976	16,268
Business combination-related contingent consideration, current portion	7,000	7,400
Operating lease liabilities, current portion	4,433	3,908
Other current liabilities	5,722	6,188
Total current liabilities	142,163	124,088
Convertible debt	375,545	226,581
Deferred revenue, less current portion	10,931	20,379
Business combination-related contingent consideration, less current portion	64,200	59,700
Operating lease liabilities, less current portion	27,510	31,497
Other non-current liabilities	9,385	12,276
Total liabilities	629,734	474,521
Commitments and contingencies (See Note 11)
Stockholders' Equity:
Preferred stock $0.0001 par value; 20,000,000 shares authorized; no shares issued and outstanding as of December 31, 2022 and 2021	0	0
Common stock $0.0001 par value; 200,000,000 shares authorized; 64,290,570 and 62,491,498 issued and outstanding as of December 31, 2022 and 2021, respectively	6	6
Additional paid-in capital	1,059,975	1,068,634
Accumulated deficit	(1,014,223)	(765,966)
Accumulated other comprehensive loss	(2,907)	(562)
Total stockholders' equity	42,851	302,112
Total liabilities and stockholders' equity	$ 672,585	$ 776,633

CONSOLIDATED BALANCE SHEETS (Parenthetical) - $ / shares	Dec. 31, 2022	Dec. 31, 2021
Statement of Financial Position [Abstract]
Preferred stock, par value (in dollars per share)	$ 0.0001	$ 0.0001
Preferred stock, shares authorized (in shares)	20,000,000	20,000,000
Preferred stock, shares issued (in shares)	0	0
Preferred stock, shares outstanding (in shares)	0	0
Common stock, par value (in dollars per share)	$ 0.0001	$ 0.0001
Common stock, shares authorized (in shares)	200,000,000	200,000,000
Common stock, shares issued (in shares)	64,290,570	62,491,498
Common stock, shares outstanding (in shares)	64,290,570	62,491,498

CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS - USD ($) $ in Thousands	12 Months Ended
	Dec. 31, 2022	Dec. 31, 2021	Dec. 31, 2020
Total revenue	$ 212,018	$ 227,490	$ 198,321
Operating expenses:
Cost of goods sold	7,592	6,784	6,126
Research and development	235,780	210,328	131,773
Selling, general and administrative	220,206	149,883	135,799
Change in fair value of contingent consideration	15,006	22,260	3,655
Acquired IPR&D expense	0	0	97,131
Total operating expenses	478,584	389,255	374,484
Operating loss	(266,566)	(161,765)	(176,163)
Other income (expenses), net:
Interest income	6,276	1,993	5,003
Interest expense	(11,275)	(20,141)	(19,050)
Other income, net	974	231	1,420
Loss on extinguishment of debt	(7,578)	0	0
Total other expense, net	(11,603)	(17,917)	(12,627)
Loss before income tax (provision) benefit	(278,169)	(179,682)	(188,790)
Income tax (provision) benefit	(313)	(409)	19,359
Net loss	$ (278,482)	$ (180,091)	$ (169,431)
Basic net loss per common share (in dollars per share)	$ (4.37)	$ (3.01)	$ (3.56)
Diluted net loss per common share (in dollars per share)	$ (4.37)	$ (3.01)	$ (3.56)
Basic weighted average common shares outstanding (in shares)	63,758,515	59,832,287	47,539,631
Diluted weighted average common shares outstanding (in shares)	63,758,515	59,832,287	47,539,631
Comprehensive loss:
Net loss	$ (278,482)	$ (180,091)	$ (169,431)
Unrealized loss on defined benefit pension plan	(368)	0	0
Foreign currency translation gain (loss)	507	1,515	(1,452)
Unrealized loss on marketable debt securities	(2,484)	(1,175)	(176)
Comprehensive loss	(280,827)	(179,751)	(171,059)
Net product sales
Total revenue	200,528	210,776	198,321
License and collaboration revenue
Total revenue	$ 11,490	$ 16,714	$ 0

CONSOLIDATED STATEMENTS OF STOCKHOLDERS' EQUITY - USD ($) $ in Thousands	Total	Cumulative Effect, Period of Adoption, Adjustment	Underwritten Equity Offering	At-The-Market Offering	Common Stock	Common Stock Underwritten Equity Offering	Common Stock At-The-Market Offering	Additional Paid-in Capital	Additional Paid-in Capital Cumulative Effect, Period of Adoption, Adjustment	Additional Paid-in Capital Underwritten Equity Offering	Additional Paid-in Capital At-The-Market Offering	Accumulated Other Comprehensive Income (Loss)	Accumulated Deficit	Accumulated Deficit Cumulative Effect, Period of Adoption, Adjustment
Beginning balance (in shares) at Dec. 31, 2019					43,088,921
Beginning balance at Dec. 31, 2019	$ 221,196				$ 4			$ 636,910				$ 726	$ (416,444)
Increase (Decrease) in Stockholders' Equity [Roll Forward]
Share based compensation	22,733							22,733
Issuance of common shares under the equity incentive plan and proceeds from exercise (in shares)					649,075
Issuance of common stock under the equity incentive plan and proceeds from exercise	3,895							3,895
Employee stock purchase program stock purchase and expense (in shares)					167,629
Employee stock purchase program stock purchase and expense	2,928							2,928
Issuance of common stock, net of offering costs (in shares)						7,475,000	867,806
Issuance of common stock, net of offering costs			$ 108,692	$ 22,828		$ 1				$ 108,691	$ 22,828
Unrealized loss on defined benefit pension plan	0
Foreign currency translation adjustments	(1,452)											(1,452)
Unrealized loss on marketable debt securities	(176)											(176)
Net loss	(169,431)												(169,431)
Ending balance (in shares) at Dec. 31, 2020					52,248,431
Ending balance at Dec. 31, 2020	$ 211,213				$ 5			797,985				(902)	(585,875)
Accounting Standards Update [Extensible List]	Accounting Standards Update 2020-06 Cumulative Effect, Period of Adoption [Member]
Increase (Decrease) in Stockholders' Equity [Roll Forward]
Share based compensation	$ 29,862							29,862
Issuance of common shares under the equity incentive plan and proceeds from exercise (in shares)					1,302,966
Issuance of common stock under the equity incentive plan and proceeds from exercise	12,841							12,841
Employee stock purchase program stock purchase and expense (in shares)					166,961
Employee stock purchase program stock purchase and expense	3,012							3,012
Issuance of common stock, net of offering costs (in shares)						7,532,500	1,240,640
Issuance of common stock, net of offering costs			$ 189,279	35,656		$ 1				$ 189,278	35,656
Unrealized loss on defined benefit pension plan	0
Foreign currency translation adjustments	1,515											1,515
Unrealized loss on marketable debt securities	(1,175)											(1,175)
Net loss	$ (180,091)												(180,091)
Ending balance (in shares) at Dec. 31, 2021	62,491,498				62,491,498
Ending balance at Dec. 31, 2021	$ 302,112	$ (44,720)			$ 6			1,068,634	$ (74,945)			(562)	(765,966)	$ 30,225
Increase (Decrease) in Stockholders' Equity [Roll Forward]
Share based compensation	37,897							37,897
Issuance of common shares under the equity incentive plan and proceeds from exercise (in shares)					935,598
Issuance of common stock under the equity incentive plan and proceeds from exercise	4,585							4,585
Employee stock purchase program stock purchase and expense (in shares)					161,874
Employee stock purchase program stock purchase and expense	4,259							4,259
Issuance of common stock, net of offering costs (in shares)							701,600
Issuance of common stock, net of offering costs				$ 19,545							$ 19,545
Unrealized loss on defined benefit pension plan	(368)											(368)
Foreign currency translation adjustments	507											507
Unrealized loss on marketable debt securities	(2,484)											(2,484)
Net loss	$ (278,482)												(278,482)
Ending balance (in shares) at Dec. 31, 2022	64,290,570				64,290,570
Ending balance at Dec. 31, 2022	$ 42,851				$ 6			$ 1,059,975				$ (2,907)	$ (1,014,223)

CONSOLIDATED STATEMENTS OF STOCKHOLDERS' EQUITY (Parenthetical) - USD ($) $ in Millions	12 Months Ended
	Dec. 31, 2022	Dec. 31, 2021	Dec. 31, 2020
Underwritten Equity Offering
Equity issuance costs		$ 12.2	$ 7.2
At-The-Market Offering
Equity issuance costs	$ 0.6	$ 1.1	$ 0.7

CONSOLIDATED STATEMENTS OF CASH FLOWS - USD ($) $ in Thousands	12 Months Ended
	Dec. 31, 2022	Dec. 31, 2021	Dec. 31, 2020
Cash Flows From Operating Activities:
Net loss	$ (278,482)	$ (180,091)	$ (169,431)
Adjustments to reconcile net loss to net cash used in operating activities:
Depreciation and amortization	33,079	26,617	24,579
Share based compensation	39,177	30,766	23,614
Change in estimated fair value of contingent consideration	15,006	22,260	3,655
Payments from change in fair value of contingent consideration	(8,602)	(14,375)	(12,407)
Loss on extinguishment of debt	7,578	0	0
Loss on allowance for inventory	2,455	2,110	2,225
Amortization of debt discount and issuance costs	1,622	11,242	10,478
Acquired IPR&D expense	0	0	97,131
Other	972	6,700	3,455
Changes in operating assets and liabilities:
Accounts receivable	(682)	(59)	2,301
Inventory	(2,064)	(1,815)	(5,067)
Tax receivable	(374)	17,099	(15,746)
Prepaid expenses and other current and non-current assets	(4,908)	(1,438)	(4,314)
Change in lease assets and liabilities, net	(984)	5,577	(1,203)
Accounts payable	2,307	2,770	(6,036)
Accrued expenses	22,261	18,308	4,417
Deferred revenue, current and non-current	(12,929)	38,300	0
Other current and non-current liabilities	(1,723)	1,237	(394)
Net cash used in operating activities	(186,291)	(14,792)	(42,743)
Cash Flows From Investing Activities:
Proceeds from the sale/maturity of marketable debt securities	381,993	433,604	273,482
Purchase of marketable debt securities	(385,389)	(547,076)	(214,964)
Purchase of fixed assets	(191)	(5,101)	(6,767)
Purchase of intangible assets	(28,366)	(19,050)	(17,799)
Purchase of IPR&D, net of cash acquired	0	0	(95,279)
Other	(600)	0	0
Net cash used in investing activities	(32,553)	(137,623)	(61,327)
Cash Flows From Financing Activities:
Payment of guaranteed minimum royalty	(2,100)	(2,100)	(2,100)
Payment of business combination-related contingent consideration	(2,479)	(6,104)	(7,648)
Proceeds from issuance of 2029 convertible senior notes	316,250	0	0
Payment of debt issuance costs	(9,882)	0	0
Repurchase of 2025 convertible senior notes including premium	(211,324)	0	0
Proceeds from exercise of stock options	4,585	12,841	3,895
Proceeds from the issuances under the employee stock purchase program	2,978	2,112	2,046
Net cash provided by financing activities	117,573	231,683	127,713
Effect of exchange rate changes on cash	(2,794)	1,713	(1,307)
Net (decrease) increase in cash and cash equivalents	(104,065)	80,981	22,336
Cash and cash equivalents, beginning of year	165,753	84,772	62,436
Cash and cash equivalents, end of year	61,688	165,753	84,772
Supplemental Disclosure of Cash Flow Information:
Operating cash flows used for operating leases	6,020	2,205	2,537
Cash paid for interest	10,159	7,327	6,901
Cash (paid) refunded for income taxes	(996)	16,420	3,373
Non-cash investing and financing activities:
Accrued capital expenditures	0	100	1,900
Accrued royalty in excess of minimum payable to the sellers of Thiola	16,067	19,362	18,168
Underwritten Equity Offering
Cash Flows From Financing Activities:
Proceeds from issuance of common stock	0	189,278	108,692
At-The-Market Offering
Cash Flows From Financing Activities:
Proceeds from issuance of common stock	$ 19,545	$ 35,656	$ 22,828

DESCRIPTION OF BUSINESS	12 Months Ended
DESCRIPTION OF BUSINESS	Dec. 31, 2022
Organization, Consolidation and Presentation of Financial Statements [Abstract]
DESCRIPTION OF BUSINESS	DESCRIPTION OF BUSINESS Organization and Description of Business Travere Therapeutics, Inc. (“we”, “our”, “us”, “Travere” and the “Company”) refers to Travere Therapeutics, Inc., a Delaware corporation, as well as our direct and indirect subsidiaries. Travere is a fully integrated biopharmaceutical company headquartered in San Diego, California focused on identifying, developing and delivering life-changing therapies to people living with rare kidney, liver, and metabolic diseases. We regularly evaluate and, where appropriate, act on opportunities to expand our product pipeline through licenses and acquisitions of products in areas that will serve patients with serious unmet medical need and that we believe offer attractive growth characteristics. FILSPARI™ (sparsentan) On February 17, 2023, the FDA granted accelerated approval of FILSPARI™ (sparsentan) to reduce proteinuria in adults with primary IgAN at risk of rapid disease progression, generally at UPCR ≥1.5 gram/gram. FILSPARI, a once-daily, oral medication is designed to selectively target two critical pathways in the disease progression of IgAN (endothelin 1 and angiotensin-II). Clinical-Stage Programs: Sparsentan remains a novel investigational product candidate which has been granted Orphan Drug Designation for the treatment of focal segmental glomerulosclerosis (FSGS) in the U.S. and Europe. Evaluation of sparsentan is ongoing in two pivotal Phase 3 clinical studies in rare kidney diseases. Pegtibatinase (TVT-058) is a novel investigational human enzyme replacement candidate being evaluated for the treatment of classical homocystinuria (HCU). Pegtibatinase has been granted Rare Pediatric Disease, Fast Track and Breakthrough Therapy designations by the FDA, as well as orphan drug designation in the United States and European Union. Pegtibatinase is currently being evaluated in the Phase 1/2 COMPOSE Study to assess its safety, tolerability, pharmacokinetics, pharmacodynamics and clinical effects in patients with classical HCU. The Company acquired pegtibatinase as part of the November 2020 acquisition of Orphan Technologies Limited. Chenodal (chenodeoxycholic acid or CDCA) is a naturally occurring bile acid that is approved for the treatment of people with radiolucent stones in the gallbladder. In September 2022, we were granted Fast Track Designation by the FDA for the investigation of Chenodal for cerebrotendinous xanthomatosis (CTX). In January 2020, we randomized the first patients in our Phase 3 RESTORE Study to evaluate the effects of Chenodal in adult and pediatric patients with CTX, and the study enrollment remains open. The pivotal study is intended to support an NDA submission for marketing authorization of Chenodal for CTX in the United States. Preclinical Programs: The Company is a participant in a Cooperative Research and Development Agreement ("CRADA"), which forms a multi-stakeholder approach to pool resources with leading experts, and incorporate the patient perspective early in the therapeutic identification and development process. The Company is partnering with the National Institutes of Health’s National Center for Advancing Translational Sciences ("NCATS") and a leading patient advocacy organization, Alagille Syndrome Alliance, aimed at the identification of potential small molecule therapeutics for Alagille syndrome ("ALGS"). There are no treatment options currently approved for ALGS. Other Commercial Products: •Thiola® and Thiola EC® (tiopronin tablets) are approved in the United States for the prevention of cystine (kidney) stone formation in patients with severe homozygous cystinuria. •Cholbam® (cholic acid capsules) is approved in the United States for the treatment of bile acid synthesis disorders due to single enzyme defects and is further indicated for adjunctive treatment of patients with peroxisomal disorders. •Chenodal (chenodiol tablets) is approved in the United States for the treatment of patients suffering from gallstones in whom surgery poses an unacceptable health risk due to disease or advanced age.

SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES

12 Months Ended

Dec. 31, 2022

Accounting Policies [Abstract]

SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES

A summary of the significant accounting policies applied in the preparation of the accompanying consolidated financial statements follows:

Principles of Consolidation

The consolidated financial statements represent the consolidation of the accounts of the Company, its subsidiaries and variable interest entities for which the Company has been determined to be the primary beneficiary, in conformity with accounting principles generally accepted in the United States ("U.S. GAAP"). All intercompany accounts and transactions have been eliminated in consolidation. See Note 5 for further discussion of variable interest entities (“VIE”) that the Company consolidates.

Use of Estimates

In preparing financial statements in conformity with U.S. GAAP, management is required to make estimates and assumptions that affect the reported amounts of assets and liabilities and disclosure of contingent assets and liabilities at the date of the financial statements, as well as the reported amounts of expenses during the reporting period. Due to inherent uncertainty involved in making estimates, actual results reported in future periods may be affected by changes in these estimates. On an ongoing basis, the Company evaluates its estimates and assumptions. These estimates and assumptions include revenue recognition, forecasting probability-weighted cash flows based upon estimates of forecasted revenues, clinical and regulatory timelines and discount rates, valuing equity securities in share-based payments, estimating expenses of contracted research organizations, estimating reserves for inventory, estimating the useful lives of depreciable and amortizable assets, goodwill impairment, estimating the fair value of contingent consideration, estimating of valuation allowances and uncertain tax positions, and estimates associated with the assessment of impairment for long-lived assets.

Revenue Recognition

The Company recognizes revenue when its customer obtains control of promised goods or services, in an amount that reflects the consideration which the entity expects to receive in exchange for those goods or services. To determine revenue recognition for arrangements that an entity determines are within the scope of Accounting Standards Codification ("ASC") 606, Revenue from Contracts with Customers ("ASC 606"), the entity performs the following five steps: (i) identify the contract(s) with a customer; (ii) identify the performance obligations in the contract; (iii) determine the transaction price; (iv) allocate the transaction price to the performance obligations in the contract; and (v) recognize revenue when (or as) the entity satisfies a performance obligation. The Company only applies the five-step model to contracts when it is probable that the entity will collect substantially all the consideration it is entitled to in exchange for the goods or services it transfers to the customer. See Note 3 and Note 4 for further discussion.

Payments received under collaboration and licensing agreements may include non-refundable fees at the inception of the arrangements, milestone payments for specific achievements and royalties on the sale of products. At the inception of arrangements that include milestone payments, the Company uses judgement to evaluate whether the milestones are probable of being achieved and estimates the amount to include in the transaction price utilizing the most likely amount method. If it is probable that a significant revenue reversal will not occur, the estimated amount is included in the transaction price. Milestone payments that are not within the Company or the licensee’s control, such as regulatory approvals, are considered to be constrained due to a high degree of uncertainty and are not included in the transaction price until such uncertainty is resolved. At the end of each reporting period, the Company re-evaluates the probability of achievement of development milestones and any related constraint and adjusts the estimate of the overall transaction price, if necessary. The Company recognizes aggregate sales-based milestones and royalty payments from product sales of which the license is deemed to be the predominant item to which the royalties relate, at the later of when the related sales occur or when the performance obligation to which the sales-based milestone or royalty has been allocated has been satisfied.

The Company utilizes significant judgement to develop estimates of the stand-alone selling price for each distinct performance obligation based upon the relative stand-alone selling price. Variable consideration that relates specifically to the Company’s efforts to satisfy specific performance obligations is allocated entirely to those performance obligations. The stand-alone selling price for license-related performance obligations requires judgement in developing assumptions to project probability-weighted cash flows based upon estimates of forecasted revenues, clinical and regulatory timelines and discount rates. The stand-alone selling price for clinical development performance obligations is based on forecasted expected costs of satisfying a performance obligation plus an appropriate margin.

If the licenses to intellectual property are determined to be distinct from the other performance obligations identified in the arrangement and have stand-alone functionality, the Company recognizes revenues from non-refundable, upfront fees allocated to the license when the license is transferred to the licensee and the licensee is able to benefit from the license. For licenses that are not distinct from other promises, the Company applies judgment to assess the nature of the combined performance obligation to determine whether the combined performance obligation is satisfied over time or a point in time and, if over time, the appropriate method of measuring progress for purposes of recognizing revenue from non-refundable, upfront fees. The Company evaluates the measure of progress each reporting period and, if necessary, adjusts the related revenue recognition accordingly.

The selection of the method to measure progress towards completion requires judgment and is based on the nature of the products or services to be provided. Revenue is recorded proportionally as costs are incurred. The Company generally utilizes the cost-to-cost method of progress because it best measures the transfer of control to the customer which occurs as the Company incurs costs. Under the cost-to-cost measure of progress, the extent of progress towards completion is measured based on the ratio of costs incurred to date to the total estimated costs at completion of the performance obligation. The Company uses judgment to estimate the total costs expected to complete the clinical development performance obligations, which include subcontractor costs, labor, materials, other direct costs and an allocation of indirect costs. The Company evaluates these cost estimates and the progress each reporting period and adjusts the measure of progress, if necessary.

Research and Development Expenses

Research and development includes expenses related to sparsentan, pegtibatinase, and the Company's other pipeline programs. The Company expenses all research and development costs as they are incurred. The Company's research and development costs are composed of salaries and bonuses, benefits, share-based compensation, license fees, milestones under license agreements, costs paid to third-party contractors to perform research, conduct clinical trials, develop drug materials and delivery devices, manufacture drug product supplies to support clinical development, and associated overhead expenses and facilities costs. The Company charges direct internal and external program costs to the respective development programs. The Company also incurs indirect costs that are not allocated to specific programs because such costs benefit multiple development programs and allow us to increase our pharmaceutical development capabilities. These consist of internal shared resources related to the development and maintenance of systems and processes applicable to all of our programs.

Nonrefundable advance payments for goods and services to be received in the future for use in research and development activities are recorded as prepaid expenses. The prepaid amounts are expensed as the related goods are delivered or the services are performed, or when it is no longer expected that the goods will be delivered or the services rendered.

During the three months ended December 31, 2022, the Company recorded an out of period adjustment that decreased research and development expenses and related accrued expenses by $7.7 million, $1.5 million of which was previously recorded in fiscal year 2022 and $6.2 million that was originally recorded in fiscal year 2021. The adjustment was the result of a certain pre-launch inventory contract that was not properly evaluated for accounting implications at inception. The Company evaluated the impact of the adjustment and concluded it is not material, individually and in the aggregate, to the current or any prior period financial statements.

Clinical Trial Expenses

The Company records expenses in connection with its clinical trials under contracts with contract research organizations ("CROs") that support conducting and managing clinical trials, as well as contract manufacturing organizations ("CMOs") for the manufacture of drug product supplies to support clinical development. The financial terms and activities of these agreements vary from contract to contract and may result in uneven expense levels. Generally, these agreements set forth activities that drive the recording of expenses such as start-up, initiation activities, enrollment, treatment of patients, or the completion of other clinical trial activities, and in the case of CMOs, costs associated with the production of drug product supplied and the procurement of raw materials to be consumed in the manufacturing process.

Expenses related to clinical trials are accrued based on our estimates of the progress of services performed, including actual level of patient enrollment, completion of patient studies and progress of the clinical trials or the delivery of goods. Other incidental costs related to patient enrollment or treatment are accrued when reasonably certain. If the amounts we are obligated to pay under our clinical trial agreements are modified (for instance, as a result of changes in the clinical trial protocol or scope of work to be performed), the Company adjusts its estimates accordingly on a prospective basis. Revisions to the Company's contractual payment obligations are charged to expense in the period in which the facts that give rise to the revision become reasonably certain.

The Company currently has one Phase 1/2 clinical trial and three Phase 3 clinical trials in process that are in varying stages of activity, with ongoing non-clinical support trials. As such, clinical trial expenses will vary depending on all the factors set forth above and may fluctuate significantly from quarter to quarter.

Share-Based Compensation

The Company recognizes all employee share-based compensation as a cost within research and development expenses and selling, general, and administrative expenses. Equity-classified awards principally related to stock options, restricted stock units (“RSUs”) and performance stock units ("PSUs"), are measured at the grant date fair value of the award. The Company determines grant date fair value of stock option awards using the Black-Scholes option-pricing model. The fair value of RSUs and PSUs are determined using the closing price of the Company’s common stock on the grant date. For service based vesting grants, expense is recognized over the requisite service period based on the number of options or shares expected to ultimately vest. For PSUs, expense is recognized over the implicit service period, assuming vesting is probable. No expense is recognized for PSUs if it is not probable the vesting criteria will be satisfied. Forfeitures are accounted for as they occur.


	Expiration Term	Vesting Term
Stock Options	10 years	3 to 4 years
Restricted Stock Units	----	1 to 4 years

Earnings (Loss) Per Share

The Company calculates basic earnings per share by dividing net income/(loss) by the weighted average number of shares outstanding during the period. The diluted earnings/(loss) per share computation includes the effect, if any, of shares that would be issuable upon the exercise of outstanding stock options, derivative warrant liability, convertible debt and RSUs, reduced by the number of shares which are assumed to be purchased by the Company from the resulting proceeds at the average market price during the year, when such amounts are dilutive to the earnings per share calculation.

Cash and Cash Equivalents

The Company considers all highly liquid marketable securities with an original maturity of three months or less to be cash equivalents. Due to the short-term maturity of such investments, the carrying amounts are a reasonable estimate of fair value.

Concentration of Credit Risk

The Company maintains its cash and cash equivalents at insured financial institutions, the balances of which may, at times, exceed federally insured limits. Generally, these deposits may be redeemed upon demand, and the Company believes there is minimal risk of losses on such balances.

The Company monitors its investments with counterparties with the objective of minimizing concentrations of credit risk. The Company's investment policy is to invest only in institutions that meet high credit quality standards and established limits on the amount and time to maturity of investments with any individual counterparty. The policy also requires that investments are only entered into with corporate and financial institutions that meet high quality standards.

Marketable Debt Securities

The Company classified marketable debt securities held as “available-for-sale” and carries them at fair value. The Company classifies these investments as current assets, even if the maturity when acquired by the Company is greater than one year due to the ability to liquidate within the next 12 months. The amortized cost of marketable debt securities is adjusted for amortization of premiums and accretion of discounts to maturity. Such amortization and accretion, as well as interest and dividends, is included in interest income. Unrealized gains and losses on marketable debt securities are recorded as a separate component of stockholders’ equity as accumulated other comprehensive loss, unless an impairment is determined to be the result of credit-related factors or the Company intends to sell the security or it is more likely than not that the Company will be required to sell the security before recovery. Unrealized losses that are determined to be credit-related are recorded as an allowance against the amortized cost basis. Realized gains or losses on debt security transactions and declines in value that are determined to be the result of credit losses, if any, are reported in other income or expense in the Consolidated Statements of Operations and Comprehensive Loss. The cost of securities sold is based on the specific identification method. Marketable debt securities are maintained at one financial institution and are governed by the Company’s investment policy. See Note 6 for further discussion.

Accounts Receivables, Net

Trade accounts receivable are recorded net of reserves for prompt pay discounts and expected credit losses. Estimates for allowances for credit losses are determined based on existing contractual obligations, historical payment patterns and individual customer circumstances. The allowance for credit losses was zero at both December 31, 2022 and 2021, respectively. For the years ended December 31, 2022, 2021 and 2020, bad debt expense recorded in the Consolidated Statements of Operations and Comprehensive Loss was immaterial. The Company's evaluation and application of ASU No. 2016-13, Financial Instruments - Credit Losses for the current period included an assessment of our aged trade receivables balances and their underlying credit risk characteristics. Our evaluation of past events, current conditions, and reasonable and supportable forecasts about the future resulted in an expectation of immaterial credit losses.

Supplier Concentration Risk

The Company has no manufacturing capabilities and relies on third party manufacturers who are sole source suppliers for manufacturing of its products. The Company intends to rely on third-party manufacturers for the long-term commercial supply of FILSPARI and for its development stage product candidates, including sparsentan for the treatment of FSGS and pegtibatinase (TVT-058). The Company expects the manufacturers of each product or product candidate to, at least initially and potentially for a significant period of time, be single source suppliers to the Company.

Inventory, Related Reserves and Cost of Goods Sold

Inventory, which is recorded at the lower of cost or net realizable value, includes materials, labor, and other direct and indirect costs and is valued using the first-in, first-out method. The Company periodically analyzes its inventory levels to identify inventory that may expire prior to expected sale or has a cost basis in excess of its estimated realizable value, and writes down such inventory as appropriate. In addition, the Company's products are subject to strict quality control and monitoring which the Company’s manufacturers perform throughout their manufacturing process. The Company does not directly manufacture any product. The Company has single suppliers for products Chenodal and Thiola, and prospectively arranges for manufacture from contract service providers for its product Cholbam. The inventory reserve was $3.5 million and $4.1 million at December 31, 2022 and 2021, respectively.

Inventory, net of reserves, consisted of the following at December 31, 2022 and 2021 (in thousands):


	December 31, 2022		December 31, 2021
Raw materials	$	3,627	$	5,205
Finished goods	3,295		2,108
Total inventory	$	6,922	$	7,313

Cost of goods sold includes the cost of inventory sold, third party manufacturing and supply chain costs, product shipping and handling costs, and provisions for excess and obsolete inventory.

Capitalization of Inventory Costs

Prior to the regulatory approval of the Company's drug candidates, the Company incurs expenses for the manufacture of drug product supplies to support clinical development that could potentially be available to support the commercial launch of those drugs. The Company capitalizes inventory costs associated with its products after regulatory approval, when, based on management’s judgment, future commercialization is considered probable and the future economic benefit is expected to be realized. Until the date at which regulatory approval has been received, costs related to the production of inventory are recorded as research and development expenses as incurred. Any eventual sale of previously expensed ("zero-cost") inventories may impact future margins, for any periods in which those inventories are sold. As of December 31, 2022, we have recognized approximately $7.5 million in research and development expenses related to the production of active pharmaceutical ingredient that could potentially support the commercial launch of FILSPARI.

Segment Information

The Company currently operates in one operating segment focused on the development and commercialization of innovative therapies for people with serious and life-threatening rare diseases and medical conditions. The Company is not organized by market and is managed and operated as one business. A single management team reports to the chief operating decision maker who comprehensively manages the entire business. The Company does not operate any separate lines of business or separate business entities with respect to its products. Accordingly, the Company does not accumulate discrete financial information with respect to separate products, other than revenues, and does not have separately reportable segments.

Property and Equipment, net

Property and equipment are stated at cost, net of accumulated depreciation. Depreciation is computed using the straight-line method over the related estimated useful lives as presented in the table below. Significant additions and improvements are capitalized, while repairs and maintenance are charged to expense as incurred. Property and equipment purchased for specific research and development projects with no alternative use is expensed as incurred.

The major classifications of property and equipment, including their respective expected useful lives, consist of the following:


Computers and equipment			3 years
Furniture and fixtures			7 years
Leasehold improvements			Shorter of length of lease or life of the asset

Leases

The Company determines whether a contract is, or contains, a lease at inception. The Company classifies each of its leases as operating or financing considering factors such as the length of the lease term, the present value of the lease payments, the nature of the asset being leased, and the potential for ownership of the asset to transfer during the lease term. Leases with terms greater than one year are recognized on the Consolidated Balance Sheets as Right-of-use assets and Lease liabilities and are measured at the present value of the fixed payments due over the expected lease term minus the present value of any incentives, rebates or abatement expected to be received from the lessor. Options to extend a lease are typically excluded from the expected lease term as the exercise of the option is typically not reasonably certain. The interest rate implicit in lease contracts is typically not readily determinable. As such, the Company utilizes the appropriate incremental borrowing rate, which is the rate incurred to borrow on a collateralized basis an amount equal to the lease payments over a similar term and in a similar economic environment.

In addition to rent, the leases may require the Company to pay additional amounts for taxes, insurance, maintenance, and other expenses, which are generally referred to as non-lease components. The Company has elected to not separate lease and non-lease components. Only the fixed costs for lease components and their associated non-lease components are accounted for as a single lease component and recognized as part of the Right-of-use assets and Lease liabilities. The Company records expense to recognize fixed lease payments, including payment escalation, on a straight-line basis over the expected lease term. Costs determined to be variable and not based on an index or rate are not included in the measurement of the lease liability and are expensed as incurred.

The Company has made an accounting policy election to not recognize short-term leases, or leases that have a lease term of 12 months or less at commencement date, within its Consolidated Balance Sheets and to recognize those lease payments in the Consolidated Statements of Operations and Comprehensive Loss on a straight-line basis over the lease term.

Acquired IPR&D

The Company assesses whether IPR&D assets acquired from others in an asset acquisition have alternative future use (in research and development projects or otherwise) at the acquisition date. If such assets have alternative future use, they are capitalized and recognized as an intangible asset. If such assets do not have alternative use, nor is there an alternative indication that the Company plans to pursue, the upfront consideration transferred, including any contingent consideration that is probable and reasonably estimable, is charged to expense at the acquisition date.

The Company expensed the acquired IPR&D associated with the acquisition of Orphan Technologies in 2020, which included the developmental product candidate for the treatment of classical homocystinuria (HCU), pegtibatinase (TVT-058).

Intangible Assets, Net

The Company's intangible assets consist of licenses and purchased technology. Intangible assets with definite lives are amortized on a straight-line basis over their estimated useful lives and are reviewed periodically for impairment.

Intangible assets related to in-process research and development (IPR&D) projects are considered to be indefinite-lived until the completion or abandonment of the associated research and development efforts. During the period the assets are considered indefinite-lived, they will not be amortized but will be tested for impairment. If and when development is complete, which generally occurs when regulatory approval to market a product is obtained, the associated assets are deemed finite-lived and are amortized over a period that best reflects the economic benefits provided by these assets.

Intangible Assets with Cost Accumulation Model

In 2014, the Company entered into a license agreement with Mission Pharmacal in which the Company obtained the exclusive right to license the trademark of Thiola. The acquisition of the Thiola license qualified as an asset acquisition under the principles of ASC 805, Business Combinations ("ASC 805") in effect at the time of acquisition. The license agreement requires the Company to make royalty payments based on net sales of Thiola. The liability for royalties in excess of the annual contractual minimum is recognized in the period in which the royalties become probable and estimable, which is typically in the period corresponding with the respective sales. The Company records an offsetting increase to the cost basis of the asset under the cost accumulation model. The additional cost basis is subsequently amortized over the remaining life of the license agreement.

Consistent with all prior periods since Thiola was acquired, the Company has not accrued any liability for future royalties in excess of the annual contractual minimum at December 31, 2022 as such royalties are not yet probable and estimable.

Variable Interest Entity

The Company reviews each investment and collaboration agreement to determine if it has a variable interest in the entity. In assessing whether the Company has a variable interest in the entity as a whole, the Company considers and makes judgements regarding the purpose and design of the

entity, the value of the licensed assets to the entity, the value of the entity’s total assets and the significant activities of the entity. If the Company has a variable interest in the entity as a whole, the Company assesses whether or not the Company is a primary beneficiary of that VIE, based on a number of factors, including: (i) which party has the power to direct the activities that most significantly affect the VIE’s economic performance, (ii) the parties’ contractual rights and responsibilities pursuant to the collaboration agreement, and (iii) which party has the obligation to absorb losses of or the right to receive benefits from the VIE that could be significant to the VIE. If the Company determines that it is the primary beneficiary of a VIE at the onset of the collaboration, the collaboration is treated as a business combination and the Company consolidates the financial statements of the VIE into the Company’s consolidated financial statements. On a quarterly basis, the Company evaluates whether it continues to be the primary beneficiary of the consolidated VIE. If the Company determines that it is no longer the primary beneficiary of a consolidated VIE, it deconsolidates the VIE in the period in which the determination is made.

Assets and liabilities recorded as a result of consolidating the financial results of the VIE into the Company’s consolidated balance sheet do not represent additional assets that could be used to satisfy claims against the Company’s general assets or liabilities for which creditors have recourse to the Company’s general assets.

Goodwill

Goodwill represents the excess of purchase price over fair value of net assets acquired in a business combination and is not amortized. Goodwill is subject to impairment testing at least annually or when a triggering event occurs that could indicate a potential impairment. The Company has one segment and one reporting unit and as such reviews goodwill for impairment at the consolidated level.

For the years ended December 31, 2022, 2021 and 2020 there were no impairments to goodwill.

Impairment of Long-Lived Assets

The Company's long-lived assets are primarily comprised of intangible assets, right-of-use assets, and property and equipment. The Company evaluates its finite-lived intangible assets, right-of-use assets, and property and equipment for impairment whenever events or changes in circumstances indicate the carrying value of an asset or group of assets may not be recoverable. If these circumstances exist, recoverability of assets to be held and used is measured by a comparison of the carrying amount of an asset group to future undiscounted net cash flows expected to be generated by the use and eventual disposition of the asset group. If such assets are considered to be impaired, the impairment to be recognized is measured by the amount by which the carrying amount of the assets exceeds the fair value of the assets.

In addition, indefinite-lived intangible assets, comprised of IPR&D, are reviewed for impairment annually and whenever events or changes in circumstances indicate that it is more likely than not that the asset is impaired by comparing the fair value to the carrying value of the asset. To determine the fair value of the asset, the Company used the multi-period excess earnings method of the income approach. The more significant assumptions inherent in the application of this method include: the amount and timing of projected future cash flows (including revenue, cost of sales, research and development costs, and sales and marketing expenses), and the discount rate selected to measure the risks inherent in the future cash flows.

There were no impairments related to finite-lived intangible assets in the years ended December 31, 2022, 2021 and 2020.

Contingent Consideration

The Company records contingent consideration resulting from a business combination at its fair value on the acquisition date. On a quarterly basis, the Company revalues these obligations and record increases or decreases from their fair value as an adjustment to the consolidated statement of operations. Changes to contingent consideration obligations can result from changes to discount rates, accretion of the liability due to the passage of time, changes in revenue forecasts and changes in our estimates of the likelihood or timing of achieving commercial revenue milestones.

Income Taxes

The Company follows ASC 740, Income Taxes, which requires recognition of deferred tax assets and liabilities for the expected future tax consequences of events that have been included in the financial statements or tax returns. Under this method, deferred tax assets and liabilities are based on the differences between the financial statement and tax basis of assets and liabilities using enacted tax rates in effect for the year in which the differences are expected to reverse. Deferred tax assets are reduced by a valuation allowance to the extent management concludes it is more likely than not that the asset will not be realized.

The standard addresses the determination of whether tax benefits claimed or expected to be claimed on a tax return should be recorded in the financial statements. Under ASC 740, the Company may recognize the tax benefit from an uncertain tax position only if it is more likely than not that the tax position will be sustained on examination by the tax authorities, based on the technical merits of the position. The tax benefits recognized in the financial statements from such a position should be measured based on the largest benefit that has a greater than fifty percent likelihood of being realized upon ultimate settlement. ASC 740 also provides guidance on de-recognition, classification, interest and penalties on income taxes,

accounting in interim periods and requires increased disclosures. The Company’s policy is to record estimated interest and penalty related to the underpayment of income taxes or unrecognized tax benefits as a component of its income tax provision.

Foreign Currency Translation

Functional and presentation currency

Items included in the financial statements of each entity comprising the Company are measured using the currency of the primary economic environment in which the entity operates (the functional currency).

Transactions and balances

Foreign currency transactions in each entity comprising the Company are remeasured into the functional currency of the entity using the exchange rates prevailing at the respective transaction dates. Foreign exchange gains and losses resulting from the settlement of such transactions and from the remeasurement at year-end exchange rates of monetary assets and liabilities denominated in foreign currencies are recognized within Other income (expense), net in the Consolidated Statements of Operations and Comprehensive Loss.

The aggregate gain arising from foreign exchange transactions included in Other income (expense), net for the years ended December 31, 2022, 2021 and 2020 was $1.4 million, $0.3 million, and $1.2 million, respectively.

The results and financial position of the Company that have a functional currency different from the US dollar are translated as follows:

a.assets and liabilities presented are translated at the closing exchange rate as of December 31, 2022 and 2021;

b.income and expenses for the statements of operations and comprehensive loss are translated at average exchange rates that are relevant for the respective periods for which the income and expenses occurred; and

c.significant transactions use the exchange rate on the date of the transaction.

All resulting exchange differences arising from such translations are recognized directly in comprehensive income and presented as a separate component of equity.

The following table summarizes the foreign currency translation adjustment included in accumulated other comprehensive loss for the year ended December 31, 2022, 2021 and 2020 (in thousands):


	Foreign Currency Translation Adjustments included in Accumulated Other Comprehensive Loss
	2022		2021		2020
Balance at January 1,	$	(92)	$	(1,607)	$	(155)
Foreign currency translation adjustments	507		1,515		(1,452)
Balance at December 31,	$	415	$	(92)	$	(1,607)

Reclassifications

Certain reclassifications have been made to the prior year financial statements in order to conform to the current year’s presentation, including reclassifying operating lease right of use assets and operating lease liabilities from other assets and other liabilities, respectively on the Consolidated Balance Sheets. These reclassifications did not have an impact on total assets or total liabilities of the Consolidated Balance Sheets.

Patents

The Company expenses external costs, such as filing fees and associated attorney fees, incurred to obtain issued patents and patent applications pending. The Company also expenses costs associated with maintaining and defending patents subsequent to their issuance in the period incurred.

Legal Contingencies

The Company may, from time to time, be involved in various claims and legal actions that arise in the ordinary course of business. The Company accrues for legal contingencies when it is determined probable that a liability has been incurred and the amount of the loss can be reasonably estimated. See Note 11 for further discussion.

Recently Adopted Accounting Pronouncements

In August 2020, the FASB issued ASU No. 2020-06, Accounting for Convertible Instruments and Contracts in an Entity's Own Equity. The ASU includes amendments to the guidance on convertible instruments and the derivative scope exception for contracts in an entity's own equity in Subtopic 815-40 and simplifies the accounting for convertible instruments which include beneficial conversion features or cash conversion features by removing certain separation models in Subtopic 470-20. Additionally, the ASU will require entities to use the "if-converted" method when calculating diluted earnings per share for convertible instruments. The ASU is effective for fiscal years beginning after December 15, 2021, including interim periods within those fiscal years. The adoption of the new standard impacted the Company's accounting for its Convertible Senior Notes Due 2025 (2025 Notes), discussed in Note 7, which were previously accounted for using the cash conversion model applied under ASC 470-20, Debt with Conversion and Other Options ("ASC 470-20"). The Company adopted ASU 2020-06 on January 1, 2022 using the modified retrospective method. The cumulative effect of the accounting change as of January 1, 2022 increased the carrying amount of the 2025 Notes by $44.7 million, reduced additional paid-in capital by $74.9 million, and reduced accumulated deficit by $30.2 million. The Company has not adjusted prior period comparative information and will continue to disclose prior period financial information in accordance with the previous accounting guidance.

Recently Issued Accounting Pronouncements

From time to time, new accounting pronouncements are issued by the Financial Accounting Standards Board ("FASB") or other standard setting bodies. Unless otherwise discussed, the Company believes that the impact of recently issued standards that are not yet effective will not have a material impact on its consolidated financial position or results of operations upon adoption.

REVENUE RECOGNITION

12 Months Ended

Dec. 31, 2022

Revenue from Contract with Customer [Abstract]

REVENUE RECOGNITION

Product Sales, Net

Product sales consist of bile acid products (Chenodal and Cholbam) and tiopronin products (Thiola and Thiola EC). The Company sells its products through direct-to-patient distributors worldwide, with the United States and Canada representing 97% and 2% of net product sales, respectively, and rest of world representing less than 1% of net product sales, based on the product shipment destination.

Revenues from product sales are recognized in satisfaction of a single performance obligation when the customer obtains control of the Company’s product, which occurs upon delivery to the customer. The Company receives payments from its product sales, primarily through third party payers, based on terms that generally are within 30 days of delivery of product to the patient. Contracts do not contain significant financing components based on the typical period of time between performance of services and collection of consideration.

Deductions from Revenue

Revenues from product sales are recorded at the net sales price, which includes provisions resulting from discounts, rebates and co-pay assistance that are offered to customers, health care providers, payers and other indirect customers relating to the Company’s sales of its products. These provisions are based on the estimates of the amounts earned or to be claimed on the related sales. These amounts are treated as variable consideration, estimated and recognized as a reduction of the transaction price at the time of the sale, using the most likely amount method, and are classified as a reduction of accounts receivable (if the amount is payable to a customer) or as a current liability (if the amount is payable to a party other than a customer). The Company includes these estimated amounts in the transaction price to the extent it is probable that a significant reversal of cumulative revenue recognized for such transactions will not occur. Where appropriate, these reserves take into consideration the Company’s historical experience, current contractual and statutory requirements and specific known market events and trends. Overall, these reserves reflect the Company’s best estimates of the amount of consideration to which it is entitled based on the terms of the contract. If actual results in the future vary from the Company’s provisions, the Company will adjust the estimate, which would affect net product revenue and earnings in the period such variances become known. For the years ended December 31, 2022, 2021 and 2020, the Company recorded a net product revenue increase of $0.8 million, $0.4 million, and $0.4 million, respectively, related to performance obligations satisfied in previous periods.

Government Rebates: The Company calculates the rebates that it will be obligated to provide to government programs and deducts these estimated amounts from its gross product sales at the time the revenues are recognized. Allowances for government rebates and discounts are established based on actual payer information, which is reasonably estimated at the time of delivery, and the government-mandated discounts applicable to government-funded programs. Rebate discounts are included in other current liabilities in the accompanying consolidated balance sheets.

Commercial Rebates: The Company calculates the rebates it incurs due to contracts with certain commercial payers and deducts these amounts from its gross product sales at the time the revenues are recognized. Allowances for commercial rebates are established based on actual payer information, which is reasonably estimated at the time of delivery. Rebate discounts are included in other current liabilities in the accompanying consolidated balance sheets.

Prompt Pay Discounts: The Company offers discounts to certain customers for prompt payments. The Company accrues for the calculated prompt pay discount based on the gross amount of each invoice for those customers at the time of sale.

Product Returns: Consistent with industry practice, the Company offers its customers a limited right to return product purchased directly from the Company, which is principally based upon the product’s expiration date. Generally, shipments are only made upon a patient prescription thus returns are minimal.

Co-pay Assistance: The Company offers a co-pay assistance program, which is intended to provide financial assistance to qualified commercially insured patients with prescription drug co-payments required by payers. The calculation of the accrual for co-pay assistance is based on an identification of claims and the cost per claim associated with product that has been recognized as revenue.

The following table summarizes net product sales for the year ended December 31, 2022, 2021 and 2020 (in thousands):


	Year Ended December 31,
	2022		2021		2020
Bile acid products	$	102,558	$	95,654	$	89,438
Tiopronin products	97,970		115,122		108,883
Total net product sales	$	200,528	$	210,776	$	198,321

- Definition

The entire disclosure of revenue from contract with customer to transfer good or service and to transfer nonfinancial asset. Includes, but is not limited to, disaggregation of revenue, credit loss recognized from contract with customer, judgment and change in judgment related to contract with customer, and asset recognized from cost incurred to obtain or fulfill contract with customer. Excludes insurance and lease contracts.

+ References

Reference 1: http://www.xbrl.org/2003/role/disclosureRef
-Publisher FASB
-Name Accounting Standards Codification
-Topic 606
-URI https://asc.fasb.org/topic&trid=49130388

Reference 2: http://www.xbrl.org/2003/role/disclosureRef
-Publisher FASB
-Name Accounting Standards Codification
-Topic 606
-SubTopic 10
-Section 50
-Paragraph 10
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Reference 3: http://www.xbrl.org/2003/role/disclosureRef
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Reference 5: http://www.xbrl.org/2003/role/disclosureRef
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Reference 6: http://www.xbrl.org/2003/role/disclosureRef
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Reference 7: http://www.xbrl.org/2003/role/disclosureRef
-Publisher FASB
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-SubTopic 10
-Section 50
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-Subparagraph (a)
-URI https://asc.fasb.org/extlink&oid=126920106&loc=SL49130554-203045

Reference 8: http://www.xbrl.org/2003/role/disclosureRef
-Publisher FASB
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-Section 50
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Reference 9: http://www.xbrl.org/2003/role/disclosureRef
-Publisher FASB
-Name Accounting Standards Codification
-Topic 606
-SubTopic 10
-Section 50
-Paragraph 15
-URI https://asc.fasb.org/extlink&oid=126920106&loc=SL49130558-203045

Reference 10: http://www.xbrl.org/2003/role/disclosureRef
-Publisher FASB
-Name Accounting Standards Codification
-Topic 606
-SubTopic 10
-Section 50
-Paragraph 12
-Subparagraph (c)
-URI https://asc.fasb.org/extlink&oid=126920106&loc=SL49130554-203045

+ Details

COLLABORATION AND LICENSE AGREEMENTS

12 Months Ended

Dec. 31, 2022

Organization, Consolidation and Presentation of Financial Statements [Abstract]

COLLABORATION AND LICENSE AGREEMENTS

On September 15, 2021, the Company entered into a license and collaboration agreement (“License Agreement”) with Vifor (International) Ltd. (“CSL Vifor”), pursuant to which the Company granted an exclusive license to CSL Vifor for the commercialization of sparsentan in Europe, Australia and New Zealand ("Licensed Territories"). CSL Vifor also has first right of negotiation to expand the licensed territories into Canada, China, Brazil and/ or Mexico. Under the terms of the License Agreement, the Company received an upfront payment of $55.0 million and will be eligible for up to $135.0 million in aggregate regulatory and market access related milestone payments and up to $655.0 million in aggregate sales-based milestone payments for a total potential value of up to $845.0 million. The Company is also entitled to receive tiered double-digit royalties of up to 40 percent of annual net sales of sparsentan in the Licensed Territories.

Under the License Agreement, CSL Vifor will be responsible for all commercialization activities in the Licensed Territories. The Company remains responsible for the worldwide clinical development of sparsentan through regulatory approval as defined and will retain all rights to sparsentan in the United States and rest of world outside of the Licensed Territories. Development costs for any post regulatory approval development activities, subject to approval by both parties, will be borne by the Company and CSL Vifor as defined, respectively. The License Agreement will remain in effect, unless terminated earlier, until the expiration of all royalty terms for sparsentan in the licensed territories. Each party has the right to terminate the License Agreement for the other party’s uncured material breach, insolvency or if the time required for performance under the License Agreement by the other party is extended due to a force majeure event that continues for six months or more.

The Company assessed the License Agreement and determined that it meets both criteria to be considered a collaborative agreement within the Scope of ASC 808, Collaborative Arrangements of active participation by both parties and exposures to significant risks and rewards dependent on the commercial success of the activities. Both parties participate on joint steering and other committees overseeing the collaboration activities. Also, both parties are exposed to significant risks and rewards based on the economic outcomes of regulatory approvals and commercialization of sparsentan.

The Company determined the transaction price under the License Agreement totaled $55.0 million, consisting of the fixed non-refundable upfront payment. The variable regulatory and access related milestones were excluded from the transaction price given the substantial uncertainty related to their achievement. Sales-based milestone payments and royalties on net sales were excluded from the transaction price and will be recognized at the later of when the related sales occur or when the performance obligation to which the sales-based milestone or royalty has been allocated have been satisfied.

The Company concluded that CSL Vifor represented a customer and applied relevant guidance from ASC 606 to evaluate the accounting under the License Agreement. In accordance with this guidance, the Company concluded that the promise to grant the license is distinct from the promise to provide clinical development services resulting in two performance obligations. As a result, the Company allocated $12.0 million of the transaction price, based on the performance obligations' relative standalone selling prices, to the license, which was recognized in full in 2021. The remaining $43.0 million of the transaction price was allocated to the clinical development activities and recorded as deferred revenue, which will be recognized over the development period based upon the ratio of costs incurred to date to the total estimated costs.

The Company recognized a total of $11.5 million in license and collaboration revenue for the year ended December 31, 2022, based upon the ratio of costs incurred to total estimated costs. The Company recognized a total of $16.7 million in license and collaboration revenue for the year ended

December 31, 2021, which consisted of $12.0 million from the license and $4.7 million from the clinical development activities, based upon the ratio of costs incurred to total estimated costs.

Deferred revenue related to the clinical development activities as of December 31, 2022 was $22.9 million. Of this amount, $12.0 million was classified as current as of December 31, 2022, based upon amount expected to be realized within the next year. The Company estimates that the remainder of the deferred revenue balance will be fully realized by mid-2025.

The following table sets forth a summary of changes in deferred revenue for the years ended December 31, 2022 and 2021 (in thousands):


	Deferred Revenue
	2022		2021
Balance at January 1,	$	36,647	$	—
New consideration resulting in deferred revenue	—		55,000
License revenue	—		(12,000)
Collaboration revenue	(11,490)		(4,714)
Foreign currency impact	(2,250)		(1,639)
Balance at December 31,	$	22,907	$	36,647

In February 2021, the Company entered into a limited co-promotion agreement with Albireo Pharma, Inc. ("Albireo"), whereby the Company's Cholbam dedicated sales representatives devoted a portion of their efforts to promoting Albireo's product, Bylvay (odevixibat), in the United States following the July 2021 launch of the product. The initial term of the arrangement was two years from the July 2021 launch of Bylvay, terminable at will by either party after one year following launch. In June 2022, the Company and Albireo mutually agreed to terminate the co-promotion agreement upon the one year anniversary of the launch, with such termination effective July 20, 2022. For the year ended December 31, 2022 and 2021, the Company recognized $1.7 million and $1.8 million, respectively, offset against selling, general, and administrative expenses.

ACQUISITIONS AND VARIABLE INTEREST ENTITIES	12 Months Ended
ACQUISITIONS AND VARIABLE INTEREST ENTITIES	Dec. 31, 2022
Business Combination and Asset Acquisition [Abstract]
ACQUISITIONS AND VARIABLE INTEREST ENTITIES	ACQUISITIONS AND VARIABLE INTEREST ENTITIES Acquisition of Orphan Technologies Limited In November 2020, the Company completed the acquisition of Orphan Technologies Limited (“Orphan”), including Orphan’s rare metabolic disorder drug pegtibatinase (TVT-058). The Company acquired Orphan by purchasing all of the outstanding shares. In exchange for the shares, the Company made an upfront cash payment at closing of $90.0 million plus closing adjustments, net liabilities assumed, and transaction expenses of $1.2 million, $1.8 million, and $4.2 million, respectively. Under the Agreement, the Company has also agreed to make contingent cash payments up to an aggregate of $427.0 million based on the achievement of certain development, regulatory and commercialization events as set forth in the Agreement, as well as additional tiered mid-single digit royalty payments based upon future net sales of any pegtibatinase products in the US and Europe, subject to certain reductions as set forth in the Agreement, and a contingent payment in the event a pediatric rare disease voucher for any pegtibatinase product is granted. Substantially all of the value of the assets acquired was concentrated within pegtibatinase, and as of the acquisition date, the Company did not anticipate any economic benefit to be derived from pegtibatinase other than the primary indication. Accordingly, the transaction was treated as an asset acquisition with amounts charged to expense for the acquired in-process research and development on the date of acquisition. The Company incurred $97.1 million in IPR&D expense related to the Orphan acquisition, which was recognized in 2020. In accordance with ASC 450, contingent cash payments will be accrued for when it is probable that a liability has been incurred and the amount can be reasonably estimated. As of December 31, 2022, no contingent cash payments have been accrued. Stock Purchase and Collaboration Agreement with PharmaKrysto On March 8, 2022, the Company entered into a Collaboration Agreement with PharmaKrysto Limited (“PharmaKrysto”), a privately held pre-clinical stage company related to PharmaKrysto's early-stage cystinuria discovery program, and concurrently therewith entered into a Stock Purchase Agreement with PharmaKrysto (together, the "Agreements"). Pursuant to the terms of the Agreements, the Company paid PharmaKrysto's shareholders $0.6 million in cash to purchase 5% of the outstanding common shares of PharmaKrysto and $0.4 million to PharmaKrysto as a one-time signing fee. Under the Collaboration Agreement, the Company will fund all research and development expenses for the pre-clinical activities associated with the cystinuria program, which are estimated to be approximately $5.0 million. The Agreements require the Company to purchase an additional 5% of the outstanding common shares for $1.0 million upon the occurrence of a specified pre-clinical milestone, and grant an option to the Company to purchase the remaining outstanding shares of PharmaKrysto for $5.0 million upon the occurrence of a subsequent pre-clinical milestone prior to expiration of the option on March 8, 2025. If the Company elects to exercise the option, it would be required to perform commercially reasonable clinical diligence obligations. In addition, it would be required to make cash milestone payments totaling up to an aggregate $16.0 million upon the achievement of certain development and regulatory milestones, plus tiered royalty payments of less than 4% on future net sales of a product, if approved. The Company has the right to terminate the Agreements and return the shares for a nominal price at any time upon 60 days’ notice, subject to survival of contingent obligations, if any. The Company determined that PharmaKrysto is a VIE because it lacks the resources to conduct the cystinuria clinical program and the limitation on the residual returns through the Company's option to purchase the remaining outstanding shares. The Company further concluded that it is the primary beneficiary of the VIE due to the Company's ultimate control over the research and development program, and its obligation, subject to continuation of the collaboration, to fund 100% of research and development costs of the program pursuant to the terms of the Collaboration Agreement. The upfront payments were expensed to research and development and other income (expense), net upon initial consolidation. The Company consolidated other current assets and accrued liabilities of $0.3 million as of December 31, 2022. The results of operations were not significant for the year ended December 31, 2022. The Company is not required to provide additional funding other than the contractually required amounts disclosed above. The creditors and beneficial holders of PharmaKrysto have no recourse to the general credit or assets of the Company.

MARKETABLE DEBT SECURITIES

12 Months Ended

Dec. 31, 2022

Investments, Debt and Equity Securities [Abstract]

MARKETABLE DEBT SECURITIES

The Company's marketable debt securities as of December 31, 2022 and 2021 were composed of available-for-sale commercial paper and corporate and government debt securities. The primary objective of the Company’s investment portfolio is to preserve capital and liquidity while enhancing overall returns. The Company’s investment policy limits interest-bearing security investments to certain types of instruments issued by institutions with primarily investment grade credit ratings and places restrictions on maturities and concentration by asset class and issuer.

Marketable debt securities consist of the following (in thousands):


	As of December 31,
	2022		2021
Marketable debt securities:
Commercial paper	$	123,647	$	127,379
Corporate debt securities	224,055		233,319
Securities of government-sponsored entities	40,855		26,431
Total marketable debt securities	$	388,557	$	387,129

The following is a summary of short-term marketable debt securities classified as available-for-sale as of December 31, 2022 (in thousands):


	Contractual Maturity (in years)	Amortized Cost		Unrealized Gains		Unrealized Losses		Aggregate Estimated Fair Value
Marketable debt securities:
Commercial paper	Less than 1	$	124,301	$	2	$	(656)	$	123,647
Corporate debt securities	Less than 1	155,841		—		(1,355)		154,486
Securities of government-sponsored entities	Less than 1	7,473		—		(80)		7,393
Total maturity less than 1 year		287,615		2		(2,091)		285,526
Corporate debt securities	1 to 2	70,195		33		(659)		69,569
Securities of government-sponsored entities	1 to 2	33,702		6		(246)		33,462
Total maturity 1 to 2 years		103,897		39		(905)		103,031
Total available-for-sale marketable debt securities		$	391,512	$	41	$	(2,996)	$	388,557

The following is a summary of short-term marketable debt securities classified as available-for-sale as of December 31, 2021 (in thousands):


	Contractual Maturity (in years)	Amortized Cost		Unrealized Gains		Unrealized Losses		Aggregate Estimated Fair Value
Marketable debt securities:
Commercial paper	Less than 1	$	127,435	$	—	$	(56)	$	127,379
Corporate debt securities	Less than 1	113,001		—		(98)		112,903
Securities of government-sponsored entities	Less than 1	21,909		—		(5)		21,904
Total maturity less than 1 year		262,345		—		(159)		262,186
Corporate debt securities	1 to 2	120,705		—		(289)		120,416
Securities of government-sponsored entities	1 to 2	4,549		—		(22)		4,527
Total maturity 1 to 2 years		125,254		—		(311)		124,943
Total available-for-sale marketable debt securities		$	387,599	$	—	$	(470)	$	387,129

During 2022 and 2021, the Company had less than $0.1 million in realized gains on marketable debt securities. The Company received proceeds from the sale or maturity of marketable debt securities of $382.0 million, $433.6 million and $273.5 million for 2022, 2021 and 2020, respectively. During 2022, 2021 and 2020, the Company had purchases of marketable debt securities of $385.4 million, $547.1 million, and $215.0 million, respectively. As of December 31, 2022 and December 31, 2021, the accrued interest receivable related to the Company's marketable debt securities was $1.9 million and $0.9 million, respectively, and was recorded in prepaid expenses and other current assets on the Consolidated Balance Sheets.

The Company reviews the available-for-sale marketable debt securities for declines in fair value below the cost basis each quarter. For any security whose fair value is below its amortized cost basis, the Company first evaluates whether it intends to sell the impaired security, or will otherwise be more likely than not required to sell the security before recovery. If either are true, the amortized cost basis of the security is written down to its fair value at the reporting date. If neither circumstance holds true, the Company assesses whether any portion of the unrealized loss is a result of a credit loss. Any amount deemed to be attributable to credit loss is recognized in the income statement, with the amount of the loss limited to the difference between fair value and amortized cost and recorded as an allowance for credit losses. The portion of the unrealized loss related to factors other than credit losses is recognized in other comprehensive income (loss).

The following is a summary of available-for-sale marketable debt securities in an unrealized loss position with no credit losses reported as of December 31, 2022 (in thousands):


	Less Than 12 Months				12 Months or Greater				Total
Description of Securities	Fair Value		Unrealized Losses		Fair Value		Unrealized Losses		Fair Value		Unrealized Losses
Commercial paper	$	117,853	$	656	$	—	$	—	$	117,853	$	656
Corporate debt securities	99,066		1,041		107,964		973		207,030		2,014
Securities of government-sponsored entities	31,402		263		4,456		63		35,858		326
Total	$	248,321	$	1,960	$	112,420	$	1,036	$	360,741	$	2,996

The following is a summary of available-for-sale marketable debt securities in an unrealized loss position with no credit losses reported as of December 31, 2021 (in thousands):


	Less Than 12 Months				12 Months or Greater				Total
Description of Securities	Fair Value		Unrealized Losses		Fair Value		Unrealized Losses		Fair Value		Unrealized Losses
Commercial paper	$	122,380	$	56	$	—	$	—	$	122,380	$	56
Corporate debt securities	231,879		387		—		—		231,879		387
Securities of government-sponsored entities	26,431		27		—		—		26,431		27
Total	$	380,690	$	470	$	—	$	—	$	380,690	$	470

As of December 31, 2022 and December 31, 2021, the amortized cost of the available-for-sale marketable debt securities in an unrealized loss position was $363.7 million and $381.2 million, respectively.

As of December 31, 2022 and December 31, 2021, the Company does not intend to sell these investments and it is not more likely than not that the Company will be required to sell the investments before recovery of their amortized cost basis. The increase in unrealized losses for the year ended December 31, 2022 was primarily due to increases in short-term interest rates. The Company does not believe the unrealized losses incurred during the period are due to credit-related factors. The credit ratings of the securities held remain of the highest quality. Moreover, the Company continues to receive payments of interest and principal as they become due, and our expectation is that those payments will continue to be received timely. Factors unknown to us at this time may cause actual results to differ and require adjustments to the Company’s estimates and assumptions in the future.

CONVERTIBLE NOTES PAYABLE

12 Months Ended

Dec. 31, 2022

Debt Disclosure [Abstract]

CONVERTIBLE NOTES PAYABLE

The composition of the Company’s convertible senior notes are as follows (in thousands):


	December 31, 2022		December 31, 2021
2.25% convertible senior notes due 2029	$	316,250	$	—
2.50% convertible senior notes due 2025	68,904		276,000
Unamortized debt discount	—		(46,045)
Unamortized debt issuance costs - 2.25% convertible senior notes due 2029	(8,750)		—
Unamortized debt issuance costs - 2.50% convertible senior notes due 2025	(859)		(3,374)
Total convertible senior notes, net of unamortized debt discount and debt issuance costs	$	375,545	$	226,581

Convertible Senior Notes Due 2029

On March 11, 2022, the Company completed a registered underwritten public offering of $316.3 million aggregate principal amount of 2.25% Convertible Senior Notes due 2029 (“2029 Notes”), which includes $41.3 million aggregate principal amount of 2029 Notes sold pursuant to the full exercise of the underwriters’ option to purchase additional 2029 Notes. The Company issued the 2029 Notes under an indenture, dated as of September 10, 2018, as supplemented by the second supplemental indenture, dated as of March 11, 2022 (collectively, the “2029 Indenture”). The 2029 Notes will mature on March 1, 2029, unless earlier repurchased, redeemed, or converted. The 2029 Notes are senior unsecured obligations of the Company and bear interest at an annual rate of 2.25%, payable semi-annually in arrears on March 1 and September 1 of each year, beginning on September 1, 2022.

The Company received net proceeds from the issuance of the 2029 Notes of $306.4 million, after deducting commissions and offering expenses of $9.9 million. At December 31, 2022, accrued interest on the 2029 Notes of $2.4 million is included in accrued expenses in the accompanying Consolidated Balance Sheets. The 2029 Notes comprise the Company’s senior, unsecured obligations and are (i) equal in right of payment with the Company’s existing and future senior, unsecured indebtedness; (ii) senior in right of payment to the Company’s existing and future indebtedness that is expressly subordinated to the 2029 Notes; (iii) effectively subordinated to the Company’s existing and future secured indebtedness, to the extent of the value of the collateral securing that indebtedness; and (iv) structurally subordinated to all existing and future indebtedness and other liabilities, including trade payables.

Holders may convert their 2029 Notes at their option only in the following circumstances: (1) during any calendar quarter commencing after the calendar quarter ending on June 30, 2022 (and only during such calendar quarter), if the last reported sale price per share of the Company’s common stock for each of at least 20 trading days, whether or not consecutive, during the period of 30 consecutive trading days ending on, and including, the last trading day of the immediately preceding calendar quarter exceeds 130% of the conversion price on the applicable trading day; (2) during the five consecutive business days immediately after any 10 consecutive trading day period (such 10 consecutive trading day period, the “measurement period”) if the trading price per $1,000 principal amount of 2029 Notes for each trading day of the measurement period was less than 98% of the product of the last reported sale price per share of the Company’s common stock on such trading day and the conversion rate on such trading day; (3) upon the occurrence of certain corporate events or distributions of the Company’s common stock; (4) if the Company calls the 2029 Notes for redemption; and (5) at any time from, and including, December 1, 2028 until the close of business on the scheduled trading day immediately before the maturity date. The Company will settle conversions by paying or delivering, as applicable, cash, shares of the Company’s common stock, or a combination of cash and shares of the Company’s common stock, at the Company’s election, based on the applicable conversion rate. The initial conversion rate for the 2029 Notes is 31.3740 shares of the Company’s common stock per $1,000 principal amount of 2029 Notes, which represents an initial conversion price of approximately $31.87 per share. If a “make-whole fundamental change” (as defined in the 2029 Indenture) occurs, then the Company will, in certain circumstances, increase the conversion rate for a specified period of time.

The 2029 Notes will be redeemable, in whole or in part at the Company’s option at any time, and from time to time, on or after March 2, 2026 and, in the case of any partial redemption, on or before the 40th scheduled trading day before the maturity date, at a cash redemption price equal to the principal amount of the 2029 Notes to be redeemed, plus accrued and unpaid interest, if any, to, but excluding, the redemption date but only if the last reported sale price per share of the Company’s common stock exceeds 130% of the conversion price on (1) each of at least 20 trading days, whether or not consecutive, during the 30 consecutive trading days ending on, and including, the trading day immediately before the date the

Company sends the related redemption notice; and (2) the trading day immediately before the date the Company sends such notice. However, the Company may not redeem less than all of the outstanding 2029 Notes unless at least $100.0 million aggregate principal amount of 2029 Notes are outstanding and not called for redemption as of the time the Company sends the related redemption notice. In addition, calling any 2029 Note for redemption will constitute a make-whole fundamental change with respect to that 2029 Note, in which case the conversion rate applicable to the conversion of that 2029 Note will be increased in certain circumstances if it is converted after it is called for redemption. If a fundamental change (as defined in the 2029 Indenture) occurs, then, except as described in the 2029 Indenture, holders may require the Company to repurchase their 2029 Notes at a cash repurchase price equal to the principal amount of the 2029 Notes to be repurchased, plus accrued and unpaid interest, if any, to, but excluding, the fundamental change repurchase date. In the event of conversion, holders would forgo all future interest payments, any unpaid accrued interest and the possibility of further stock price appreciation. Upon the receipt of conversion requests, the settlement of the 2029 Notes will be paid pursuant to the terms of the 2029 Indenture. In the event that all of the 2029 Notes are converted, the Company would be required to repay the principal amount and any conversion premium in any combination of cash and shares of its common stock at the Company’s option. In addition, calling the 2029 Notes for redemption will constitute a “make-whole fundamental change."

The Company incurred approximately $9.9 million of debt issuance costs relating to the issuance of the 2029 Notes, which were recorded as a reduction to the 2029 Notes on the Consolidated Balance Sheets. The debt issuance costs are being amortized and recognized as additional interest expense over the expected life of the 2029 Notes using the effective interest method. We determined the expected life of the debt is equal to the seven-year term of the 2029 Notes. The effective interest rate on the 2029 Notes is 2.74%.

Convertible Senior Notes Due 2025

On September 10, 2018, the Company completed a registered underwritten public offering of $276.0 million aggregate principal amount of 2.50% Convertible Senior Notes due 2025 ("2025 Notes"), and entered into a base indenture and supplemental indenture agreement ("2025 Indenture") with respect to the 2025 Notes. The 2025 Notes will mature on September 15, 2025, unless earlier repurchased, redeemed, or converted. The 2025 Notes are senior unsecured obligations of the Company and bear interest at an annual rate of 2.50%, payable semi-annually in arrears on March 15 and September 15 of each year, beginning on March 15, 2019.

The net proceeds from the issuance of the 2025 Notes were approximately $267.2 million, after deducting commissions and the offering expenses of $8.8 million payable by the Company. At December 31, 2022, accrued interest of $0.5 million is included in accrued expenses in the accompanying Consolidated Balance Sheets. The 2025 Notes comprise the Company’s senior, unsecured obligations and are (i) equal in right of payment with the Company’s existing and future senior, unsecured indebtedness; (ii) senior in right of payment to the Company’s existing and future indebtedness that is expressly subordinated to the 2025 Notes; (iii) effectively subordinated to the Company’s existing and future secured indebtedness, to the extent of the value of the collateral securing that indebtedness; and (iv) structurally subordinated to all existing and future indebtedness and other liabilities, including trade payables.

Holders may convert their 2025 Notes at their option only in the following circumstances: (1) during any calendar quarter commencing after the calendar quarter ending on December 31, 2018 (and only during such calendar quarter), if the last reported sale price per share of the Company’s common stock for each of at least 20 trading days, whether or not consecutive, during the period of 30 consecutive trading days ending on, and including, the last trading day of the immediately preceding calendar quarter exceeds 130% of the conversion price on the applicable trading day; (2) during the five consecutive business days immediately after any 10 consecutive trading day period (“measurement period”) if the trading price per $1,000 principal amount of 2025 Notes for each trading day of the measurement period was less than 98% of the product of the last reported sale price per share of the Company’s common stock on such trading day and the conversion rate on such trading day; (3) upon the occurrence of certain corporate events or distributions on the Company’s common stock; (4) if the Company calls the 2025 Notes for redemption; and (5) at any time from, and including, May 15, 2025 until the close of business on the scheduled trading day immediately before the maturity date. The Company will settle conversions by paying or delivering, as applicable, cash, shares of the Company’s common stock, or a combination of cash and shares of the Company’s common stock, at the Company’s election, based on the applicable conversion rate.

The initial conversion rate for the 2025 Notes is 25.7739 shares of the Company’s common stock per $1,000 principal amount of 2025 Notes, which represents an initial conversion price of approximately $38.80 per share. If a “make-whole fundamental change” (as defined in the 2025 Indenture) occurs, then the Company will, in certain circumstances, increase the conversion rate for a specified period of time.

The 2025 Notes will be redeemable, in whole or in part, at the Company’s option at any time, and from time to time, on or after September 15, 2022 and, in the case of any partial redemption, on or before the 40th scheduled trading day before the maturity date, at a cash redemption price equal to the principal amount of the 2025 Notes to be redeemed, plus accrued and unpaid interest, if any, to, but excluding, the redemption date, but only if the last reported sale price per share of the Company’s common stock exceeds 130% of the conversion price on each of at least 20 trading days during the 30 consecutive trading days ending on, and including, the trading day immediately before the date the Company sends the related redemption notice. If a fundamental change (as defined in the 2025 Indenture) occurs, then, subject to certain exceptions, holders may require the Company to repurchase their 2025 Notes at a cash repurchase price equal to the principal amount of the 2025 Notes to be repurchased, plus accrued and unpaid interest, if any, to, but excluding, the fundamental change repurchase date. In the event of conversion, holders would forgo all future interest payments, any unpaid accrued interest and the possibility of further stock price appreciation. Upon the receipt of conversion requests, the settlement of the 2025 Notes will be paid pursuant to the terms of the 2025 Indenture. In the event that all of the 2025 Notes are converted, the

Company would be required to repay the principal amount and any conversion premium in any combination of cash and shares of its common stock at the Company’s option. In addition, calling the 2025 Notes for redemption will constitute a “make-whole fundamental change."

The Company incurred approximately $8.8 million of debt issuance costs relating to the issuance of the 2025 Notes, which were recorded as a reduction to the 2025 Notes on the Consolidated Balance Sheets. The debt issuance costs are being amortized and recognized as additional interest expense over the expected life of the 2025 Notes using the effective interest method. The Company determined the expected life of the debt is equal to the seven-year term of the 2025 Notes. The effective interest rate on the 2025 Notes is 2.98%.

On March 11, 2022, the Company completed its repurchase of $207.1 million aggregate principal amount of 2025 Notes for cash, including accrued and unpaid interest, for a total of $213.8 million. This transaction involved a contemporaneous exchange of cash between the Company and holders of the 2025 Notes participating in the issuance of the 2029 Notes. Accordingly, we evaluated the transaction for modification or extinguishment accounting in accordance with ASC 470-50, Debt – Modifications and Extinguishments on a creditor-by creditor basis depending on whether the exchange was determined to have substantially different terms. The repurchase of the 2025 Notes and issuance of the 2029 Notes were deemed to have substantially different terms based on the present value of the cash flows or significant difference between the value of the conversion option immediately prior to and after the exchange. Therefore, the repurchase of the 2025 Notes was accounted for as a debt extinguishment. The Company recorded a $7.6 million loss on extinguishment of debt on its Consolidated Statements of Operations for the year ended December 31, 2022, which includes the write-off of related deferred financing costs of $3.4 million. After giving effect to the repurchase, the total remaining principal amount outstanding under the 2025 Notes as of December 31, 2022 was $68.9 million.

The 2025 and 2029 Notes are accounted for in accordance with ASC 470-20, Debt with conversion and Other Options (“ASC 470-20”) and ASC 815-40, Contracts in Entity’s Own Equity (“ASC 815-40”). Under ASC 815-40, to qualify for equity classification (or nonbifurcation, if embedded) the instrument (or embedded feature) must be both (1) indexed to the issuer’s stock and (2) meet the requirements of equity classification guidance. Based upon the Company’s analysis, it was determined that the 2025 Notes and the 2029 Notes do not contain embedded features requiring recognition as derivatives and bifurcation, and therefore are measured at amortized cost and recorded as liabilities on the Consolidated Balance Sheets.

The 2025 and 2029 Notes do not contain any financial or operating covenants or any restrictions on the payment of dividends, the issuance of other indebtedness or the issuance or repurchase of securities by the Company. There were no events of default for the 2025 Notes or 2029 Notes at December 31, 2022.

The 2025 and 2029 Notes are classified on the Company's Consolidated Balance Sheets at December 31, 2022 as long-tern convertible debt.

Interest Expense

The following table sets forth total interest expense recognized related to the 2025 and 2029 Notes (in thousands):


	Year Ended December 31,
	2022		2021		2020
Contractual interest expense	$	8,433	$	6,900	$	6,900
Amortization of debt discount	—		10,339		9,578
Amortization of debt issuance costs	1,622		903		900
Total interest expense for the 2025 and 2029 Notes	$	10,055	$	18,142	$	17,378

Total interest expense recognized for the years ended December 31, 2022, 2021 and 2020 was $11.3 million, $20.1 million and $19.1 million, respectively.

- Definition

The entire disclosure for information about short-term and long-term debt arrangements, which includes amounts of borrowings under each line of credit, note payable, commercial paper issue, bonds indenture, debenture issue, own-share lending arrangements and any other contractual agreement to repay funds, and about the underlying arrangements, rationale for a classification as long-term, including repayment terms, interest rates, collateral provided, restrictions on use of assets and activities, whether or not in compliance with debt covenants, and other matters important to users of the financial statements, such as the effects of refinancing and noncompliance with debt covenants.

+ References

Reference 1: http://fasb.org/us-gaap/role/ref/legacyRef
-Publisher FASB
-Name Accounting Standards Codification
-Topic 470
-URI https://asc.fasb.org/topic&trid=2208564

Reference 2: http://www.xbrl.org/2003/role/disclosureRef
-Publisher FASB
-Name Accounting Standards Codification
-Topic 470
-SubTopic 20
-Section 50
-Paragraph 1B
-Subparagraph (h)
-URI https://asc.fasb.org/extlink&oid=123466505&loc=SL123495323-112611

Reference 3: http://www.xbrl.org/2003/role/disclosureRef
-Publisher FASB
-Name Accounting Standards Codification
-Topic 470
-SubTopic 20
-Section 50
-Paragraph 1C
-Subparagraph (c)
-URI https://asc.fasb.org/extlink&oid=123466505&loc=SL123495334-112611

Reference 4: http://www.xbrl.org/2003/role/disclosureRef
-Publisher FASB
-Name Accounting Standards Codification
-Topic 470
-SubTopic 20
-Section 50
-Paragraph 1I
-Subparagraph (a)
-URI https://asc.fasb.org/extlink&oid=123466505&loc=SL123495371-112611

Reference 5: http://www.xbrl.org/2003/role/disclosureRef
-Publisher FASB
-Name Accounting Standards Codification
-Topic 470
-SubTopic 20
-Section 50
-Paragraph 1I
-Subparagraph (d)
-URI https://asc.fasb.org/extlink&oid=123466505&loc=SL123495371-112611

Reference 6: http://www.xbrl.org/2003/role/disclosureRef
-Publisher FASB
-Name Accounting Standards Codification
-Topic 235
-SubTopic 10
-Section S99
-Paragraph 1
-Subparagraph (SX 210.4-08(c))
-URI https://asc.fasb.org/extlink&oid=120395691&loc=d3e23780-122690

Reference 7: http://www.xbrl.org/2003/role/disclosureRef
-Publisher FASB
-Name Accounting Standards Codification
-Topic 470
-SubTopic 20
-Section 50
-Paragraph 1B
-Subparagraph (g)
-URI https://asc.fasb.org/extlink&oid=123466505&loc=SL123495323-112611

Reference 8: http://www.xbrl.org/2003/role/disclosureRef
-Publisher FASB
-Name Accounting Standards Codification
-Topic 470
-SubTopic 20
-Section 50
-Paragraph 1C
-Subparagraph (b)
-URI https://asc.fasb.org/extlink&oid=123466505&loc=SL123495334-112611

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-Publisher FASB
-Name Accounting Standards Codification
-Topic 470
-SubTopic 20
-Section 50
-Paragraph 1C
-Subparagraph (a)
-URI https://asc.fasb.org/extlink&oid=123466505&loc=SL123495334-112611

Reference 10: http://www.xbrl.org/2003/role/disclosureRef
-Publisher FASB
-Name Accounting Standards Codification
-Topic 470
-SubTopic 20
-Section 50
-Paragraph 1E
-Subparagraph (b)
-URI https://asc.fasb.org/extlink&oid=123466505&loc=SL123495348-112611

Reference 11: http://www.xbrl.org/2003/role/disclosureRef
-Publisher FASB
-Name Accounting Standards Codification
-Topic 470
-SubTopic 20
-Section 50
-Paragraph 1B
-URI https://asc.fasb.org/extlink&oid=123466505&loc=SL123495323-112611

Reference 12: http://www.xbrl.org/2003/role/disclosureRef
-Publisher FASB
-Name Accounting Standards Codification
-Topic 470
-SubTopic 20
-Section 50
-Paragraph 1I
-Subparagraph (b)
-URI https://asc.fasb.org/extlink&oid=123466505&loc=SL123495371-112611

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-Publisher FASB
-Name Accounting Standards Codification
-Topic 470
-SubTopic 20
-Section 50
-Paragraph 1B
-Subparagraph (i)
-URI https://asc.fasb.org/extlink&oid=123466505&loc=SL123495323-112611

+ Details

FAIR VALUE MEASUREMENTS

12 Months Ended

Dec. 31, 2022

Fair Value Disclosures [Abstract]

FAIR VALUE MEASUREMENTS

The Company utilizes a fair value hierarchy that prioritizes the inputs to valuation techniques used to measure fair value. The hierarchy gives the highest priority to unadjusted quoted prices in active markets for identical assets or liabilities (Level 1 measurements) and the lowest priority to unobservable inputs (Level 3 measurements). The three levels of the fair value hierarchy are described below:

Level 1 – Unadjusted quoted prices in active markets that are accessible at the measurement date for identical, unrestricted assets or liabilities;

Level 2 – Quoted prices in markets that are not active or financial instruments for which all significant inputs are observable, either directly or indirectly; and

Level 3 – Prices or valuations that require inputs that are both significant to the fair value measurement and unobservable.

The valuation techniques used to measure the fair value of the Company’s debt securities and all other financial instruments, all of which have counter-parties with high credit ratings, were valued based on quoted market prices or model driven valuations using significant inputs derived from or corroborated by observable market data. Based on the fair value hierarchy, the Company classified marketable debt securities within Level 2.

Financial instruments with carrying values approximating fair value include cash and cash equivalents, accounts receivable, and accounts payable, due to their short-term nature. As of December 31, 2022 and 2021, the fair value of the Company's 2.50% Convertible Senior Notes due 2025 was $62.9 million and $300.6 million, respectively. As of December 31, 2022, the fair value of the Company's 2.25% Convertible Senior Notes due 2029, which were issued in 2022, was $283.0 million. The fair values were estimated utilizing market quotations, and are considered Level 2.

The following table presents the Company’s asset and liabilities, measured and recognized at fair value on a recurring basis, classified under the appropriate level of the fair value hierarchy as of December 31, 2022 (in thousands):


	As of December 31, 2022		Fair Value Hierarchy at December 31, 2022
	Total carrying and estimated fair value		Quoted prices in active markets (Level 1)		Significant other observable inputs (Level 2)		Significant unobservable inputs (Level 3)
Assets:
Cash and Cash Equivalents	$	61,688	$	61,688	$	—	$	—
Marketable debt securities, available-for-sale	388,557		—		388,557		—
Total	$	450,245	$	61,688	$	388,557	$	—
Liabilities:
Business combination-related contingent consideration	$	71,200	$	—	$	—	$	71,200
Total	$	71,200	$	—	$	—	$	71,200

The following table presents the Company’s assets and liabilities that are measured and recognized at fair value on a recurring basis, classified under the appropriate level of the fair value hierarchy as of December 31, 2021 (in thousands):


	As of December 31, 2021		Fair Value Hierarchy at December 31, 2021
	Total carrying and estimated fair value		Quoted prices in active markets (Level 1)		Significant other observable inputs (Level 2)		Significant unobservable inputs (Level 3)
Assets:
Cash and Cash Equivalents	$	165,753	$	165,753	$	—	$	—
Marketable debt securities, available-for-sale	387,129		—		387,129		—
Total	$	552,882	$	165,753	$	387,129	$	—
Liabilities:
Business combination-related contingent consideration	$	67,100	$	—	$	—	$	67,100
Total	$	67,100	$	—	$	—	$	67,100

The Company acquired two businesses, related to the Cholbam and Chenodal products, whose purchase price included potential future payments that are contingent on the achievement of certain milestones and percentages of future net sales derived from the products acquired. The Company recorded contingent consideration liabilities at their fair value on the acquisition date and revalues them at the end of each reporting period. In estimating the fair value of the Company’s contingent consideration, the Company uses a Monte Carlo Simulation. The determination of the contingent consideration liabilities requires significant judgements including the reasonableness of estimates and assumptions included in the forecasts of future net sales, long-term growth rates and the discount rates applied to such forecasts. Changes in these estimates and assumptions could have a significant impact on the fair value of the contingent consideration liabilities.

Discount rates used to determine the fair value at December 31, 2022 and 2021 are as follows:


	Revenue Discount		Payment Discount
	Cholbam	Chenodal
December 31, 2022	7.75%	8.00%	8.10%
December 31, 2021	6.25%	7.25%	6.48%

Based on the fair value hierarchy, the Company classified the fair value of contingent consideration within Level 3 because valuation inputs are based on projected revenues discounted to a present value.

The following table sets forth a summary of changes in the estimated fair value of the Company's Level 3 business combination-related contingent consideration for the years ended December 31, 2022 and 2021 (in thousands):


	Fair Value Measurements of Acquisition-Related Contingent Consideration (Level 3)
	2022		2021
Balance at January 1,	$	67,100	$	65,100
Changes in the fair value of contingent consideration	15,006		22,260
Contractual payments	(8,066)		(17,444)
Contractual payments included in accrued liabilities at December 31,	(2,840)		(2,700)
Foreign currency impact	—		(116)
Balance at December 31,	$	71,200	$	67,100

- Definition

The entire disclosure for the fair value of financial instruments (as defined), including financial assets and financial liabilities (collectively, as defined), and the measurements of those instruments as well as disclosures related to the fair value of non-financial assets and liabilities. Such disclosures about the financial instruments, assets, and liabilities would include: (1) the fair value of the required items together with their carrying amounts (as appropriate); (2) for items for which it is not practicable to estimate fair value, disclosure would include: (a) information pertinent to estimating fair value (including, carrying amount, effective interest rate, and maturity, and (b) the reasons why it is not practicable to estimate fair value; (3) significant concentrations of credit risk including: (a) information about the activity, region, or economic characteristics identifying a concentration, (b) the maximum amount of loss the entity is exposed to based on the gross fair value of the related item, (c) policy for requiring collateral or other security and information as to accessing such collateral or security, and (d) the nature and brief description of such collateral or security; (4) quantitative information about market risks and how such risks are managed; (5) for items measured on both a recurring and nonrecurring basis information regarding the inputs used to develop the fair value measurement; and (6) for items presented in the financial statement for which fair value measurement is elected: (a) information necessary to understand the reasons for the election, (b) discussion of the effect of fair value changes on earnings, (c) a description of [similar groups] items for which the election is made and the relation thereof to the balance sheet, the aggregate carrying value of items included in the balance sheet that are not eligible for the election; (7) all other required (as defined) and desired information.

+ References + Details

INTANGIBLE ASSETS

12 Months Ended

Dec. 31, 2022

Goodwill and Intangible Assets Disclosure [Abstract]

INTANGIBLE ASSETS

Amortizable Intangible Assets

Ligand License Agreement

In 2012, the Company entered into an agreement with Ligand Pharmaceuticals, Inc. ("Ligand") for a worldwide sublicense to develop, manufacture and commercialize sparsentan (the “Ligand License Agreement”). The cost of the Ligand License Agreement, which is presented net of amortization in the accompanying Consolidated Balance Sheets in intangible assets, net, is being amortized to research and development on a straight-line basis through September 30, 2023. As consideration for the license, the Company is required to make substantial payments upon the achievement of certain milestones, totaling up to $114.1 million, including a $23.0 million milestone payment to Ligand (and Bristol-Myers Squibb Company ("BMS")) we plan to make in the first quarter of 2023, which was triggered upon the accelerated approval of FILSPARI in February 2023. Through December 31, 2022, the Company has capitalized $18.4 million for contractual milestone payments under the Ligand License Agreement. Should the Company commercialize sparsentan or any products containing related compounds, the Company will be obligated to pay to Ligand (and BMS) an escalating annual royalty between 15% and 17% of net sales of all such products. With the February 2023 approval of FILSPARI (sparsentan), the Company expects to begin incurring costs associated with these royalties in 2023.

Manchester Pharmaceuticals LLC

In 2014, the Company acquired intangible assets with finite lives related to the Chenodal product rights, trade names, and customer relationships with the values of $67.8 million, $0.2 million, and $0.4 million, respectively. The useful lives related to the acquired product rights, trade names, and customer relationships are expected to be approximately 16, 1 and, 10 years, respectively. Amortization of product rights, trade names and customer relationships are being recorded in selling, general and administrative expense over their respective lives.

Thiola License Agreement

The Company entered into a license agreement with Mission Pharmacal in 2014, in which the Company obtained an exclusive, royalty-bearing license to market, sell and commercialize Thiola (tiopronin) in the United States and Canada, and a non-exclusive license to use know-how relating to Thiola to the extent necessary to market Thiola. The initial term of the license is 10 years and will automatically renew thereafter for periods of one year.

The acquisition of the Thiola license qualified as an asset acquisition under the principles of ASC 805 in effect at the time of acquisition. The license agreement requires the Company to make royalty payments based on net sales of Thiola. The liability for royalties in excess of the annual contractual minimum is recognized in the period in which the royalties become probable and estimable, which is typically in the period corresponding with the respective sales. The Company records an offsetting increase to the cost basis of the asset under the cost accumulation model. The additional cost basis is subsequently amortized over the remaining life of the license agreement.

The Company paid Mission an up-front license fee of $3 million and during the term of the agreement will pay guaranteed minimum royalties during each calendar year the greater of $2 million or twenty percent (20%) of the Company’s net sales of Thiola.

In November 2017, the Company amended its agreement with Mission to extend the term of the current exclusive U.S. and Canada licensing agreement by an additional five years, to 2029. The royalty rate and guaranteed minimum payment were also extended through the new agreement term. Upon execution of the amendment, the Company capitalized an additional $5.9 million in intangible assets and recorded a guaranteed minimum liability for the same amount.

In November 2018, the Company amended its agreement with Mission to remove all territorial restrictions on our license. As consideration for the expanded territory the Company paid an up-front fee of $0.3 million and will pay guaranteed minimum royalties equaling the greater of $0.1 million or 20% of our Thiola net sales generated outside of the United States during each calendar year. Upon execution of the amendment, the Company capitalized an additional $1.0 million in intangible assets and recorded a guaranteed minimum liability of $0.7 million related to this amendment.

The present value of guaranteed minimum royalties payable using a discount rate ranging from approximately 7% to 11% based on the Company’s then borrowing rate is $11.1 million and $12.3 million as of December 31, 2022 and 2021, respectively. As of December 31, 2022, the guaranteed minimum royalty current and long-term liability was approximately $2.1 million and $9.0 million, respectively, and is recorded as Other Liability in the Consolidated Balance Sheet. As of December 31, 2021, the guaranteed minimum royalty current and long-term liability was approximately $2.1 million and $10.2 million, respectively, and is recorded as Other Liability in the Consolidated Balance Sheet. The Company has capitalized $139.4 million related to the Thiola intangible asset which consists of the up-front license fee, professional fees, present value of the guaranteed minimum royalties and any additional payments through December 31, 2022 in excess of minimum royalties. In 2022 the Company added $16.1 million to the intangible asset related to royalties in excess of the minimum. Consistent with all prior periods since Thiola was acquired, the Company has not accrued any liability for royalties in excess of the annual contractual minimum at December 31, 2022, as such royalties are not yet probable and estimable.

There are 6.4 years remaining in the term of the license agreement.

Cholbam Asset Purchase

On March 31, 2015, the Company completed its acquisition from Asklepion of all worldwide rights, titles and ownership of Cholbam, including all related contracts, data assets, intellectual property, regulatory assets and the PRV. The Company capitalized $75.9 million and $7.3 million for the U.S. and international economic interest, respectively.

Amortizable intangible assets as of December 31, 2022 (in thousands):


	Useful Life	Gross Carrying Amount		Accumulated Amortization		Net Book Value
Thiola license	15	$	139,383	$	(50,324)	$	89,059
Chenodal product rights	16	67,849		(37,175)		30,674
Economic interest - Cholbam revenue	10	75,900		(58,842)		17,058
Ligand license	11	19,400		(12,139)		7,261
Manchester customer relationships	10	403		(353)		50
Total amortizable intangible assets		$	302,935	$	(158,833)	$	144,102

Amortizable intangible assets as of December 31, 2021 (in thousands):


	Useful Life	Gross Carrying Amount		Accumulated Amortization		Net Book Value
Thiola license	15	$	123,316	$	(37,992)	$	85,324
Chenodal product rights	16	67,849		(32,938)		34,911
Economic interest - U.S. revenue Cholbam	10	75,900		(51,258)		24,642
Ligand license	11	7,900		(5,875)		2,025
Economic interest - International revenue Cholbam	10	7,982		(7,483)		499
Manchester customer relationships	10	403		(313)		90
Internal use software	5	207		(199)		8
Total amortizable intangible assets		$	283,557	$	(136,058)	$	147,499

The following table summarizes amortization expense for the year ended December 31, 2022, 2021 and 2020 (in thousands):


	2022		2021		2020
Research and development	$	6,264	$	1,158	$	1,162
Selling, general and administrative	24,700		23,788		21,275
Total amortization expense	$	30,964	$	24,946	$	22,437

As of December 31, 2022, amortization expense for the next five years and thereafter is expected to be as follows (in thousands):


2023	$	33,015
2024	25,793
2025	19,999
2026	18,129
2027	18,129
Thereafter	29,037
Total	$	144,102

Goodwill

As of December 31, 2022 and 2021, the Company had goodwill of $0.9 million.

ACCRUED EXPENSES

12 Months Ended

Dec. 31, 2022

Payables and Accruals [Abstract]

ACCRUED EXPENSES

Accrued expenses consist of the following at December 31, 2022 and 2021 (in thousands):


	2022		2021
Compensation related costs	$	35,267	$	25,998
Research and development	26,070		26,841
Sales discounts, rebates, and allowances	13,486		7,493
Selling, general and administrative	8,791		3,144
Accrued royalties	7,755		8,402
Miscellaneous accrued	4,373		3,302
Total accrued expenses	$	95,742	$	75,180

COMMITMENTS AND CONTINGENCIES	12 Months Ended
COMMITMENTS AND CONTINGENCIES	Dec. 31, 2022
Commitments and Contingencies Disclosure [Abstract]
COMMITMENTS AND CONTINGENCIES	COMMITMENTS AND CONTINGENCIES Commitments Certain of the Company's contractual arrangements with contract manufacturing organizations ("CMOs") require binding forecasts or commitments to purchase minimum amounts for the manufacture of drug product supply, which may be material to the Company's financial statements. As of December 31, 2022, we have a commitment to purchase $36.3 million in active pharmaceutical ingredient, to be delivered in 2023, which is planned to support the commercial launch of FILSPARI. Contingencies In October 2021, our Kolbam distributor in France notified us that the French authorities are asking for reimbursement for a portion of Kolbam sales in France during the periods from 2015-2020. During this period, the Company had aggregate revenues from sales of Kolbam in France of approximately $8.0 million. At this time, the Company is not able to estimate the potential liability that may be incurred, if any. Legal Proceedings From time to time in the normal course of business, the Company is subject to various legal matters such as threatened or pending claims or litigation. Although the results of claims and litigation cannot be predicted with certainty, the Company does not believe it is a party to any claim or litigation the outcome of which, if determined adversely to it, would individually or in the aggregate be reasonably expected to have a material adverse effect on its results of operations or financial condition.

STOCKHOLDERS’ EQUITY

12 Months Ended

Dec. 31, 2022

Equity [Abstract]

STOCKHOLDERS’ EQUITY

Common Stock

The Company is currently authorized to issue up to 200,000,000 shares of $0.0001 par value common stock. All issued shares of common stock are entitled to vote on a 1 share/1 vote basis.

Preferred Stock

The Company is currently authorized to issue up to 20,000,000 shares of $0.0001 par value preferred stock, of which 1,000 shares are designated Class "A" Preferred shares. Class A Preferred Shares are not entitled to interest, have certain liquidation preferences, special voting rights and other provisions. No preferred stock has been issued to date.

2015 Equity Incentive Plan

On June 8, 2015, the Company's stockholders approved the 2015 Equity Incentive Plan (the "2015 Plan"). The 2015 Plan is intended as the successor to and continuation of the Company’s 2014 Incentive Compensation Plan. Stockholders approved 1.4 million new shares to be issued under the 2015 Plan, in addition to 0.6 million unallocated shares remaining available for issuance under the 2014 Incentive Compensation Plan that were added to the 2015 Plan. Options issued under the 2015 Plan will generally expire ten years from the date of grant and vest over a three-year or four-year period.

On May 18, 2016, the Company's stockholders approved an amendment to the 2015 Plan (the "Amended 2015 Plan"). The amendment provides for an additional 1.6 million new shares to be issued under the Amended 2015 Plan, in addition to 0.7 million unallocated shares remaining available for issuance.

On May 17, 2017, the Company's stockholders approved an amendment to the Amended 2015 Plan. The amendment provides for an additional 1.8 million new shares to be issued under the Amended 2015 Plan.

2018 Equity Incentive Plan

On May 9, 2018, the Company's stockholders approved the 2018 Equity Incentive Plan (the "2018 Plan"). The 2018 Plan is intended as the successor to and continuation of the Amended 2015 Plan. Stockholders approved 1.8 million new shares to be issued under the 2018 Plan, in addition to 1.6 million unallocated shares remaining available for issuance under the Amended 2015 Plan that were added to the 2018 Plan. Options issued under the 2018 Plan will generally expire ten years from the date of grant and vest over a four-year period.

On May 9, 2019, the Company’s stockholders approved an amendment to the 2018 Plan that increased the number of authorized shares issuable under the 2018 Plan by 2.0 million shares.

On May 15, 2020, the Company's stockholders approved an amendment to the 2018 Plan that increased the number of authorized shares issuable under the 2018 Plan by 2.4 million shares.

On May 14, 2021, the Company’s stockholders approved an amendment to the 2018 Plan that increased the number of authorized shares issuable under the 2018 Plan by 3.2 million shares.

On May 11, 2022, the Company’s stockholders approved an amendment to the 2018 Plan that increased the number of authorized shares issuable under the 2018 Plan by 2.0 million shares.

2017 Employee Stock Purchase Plan

The 2017 Employee Stock Purchase Plan ("2017 ESPP") originated with 380,000 shares of common stock available for issuance. Beginning on January 1, 2018, and ending on (and including) January 1, 2026, the number of shares of common stock available for issuance under the 2017 ESPP may increase by an amount equal to the lesser of (i) one percent (1%) of the total number of shares of common stock outstanding on December 31st of the preceding calendar year or (ii) 300,000 shares of common stock.

Substantially all employees are eligible to participate and, through payroll deductions, can purchase shares on established dates semi-annually. The purchase price per share sold pursuant to the 2017 ESPP will be the lower of (i) 85% of the fair market value of common stock on the first day of the offering period or (ii) 85% of the fair market value on the purchase date. Each offering period will span up to six months. Purchases may be up to 15% of qualified compensation, with an annual limit of $25,000. The 2017 ESPP is intended to qualify as an “employee stock purchase plan” under Section 423 of the Internal Revenue Code.

As of December 31, 2022, there were approximately 1,880,000 shares authorized and 1,127,178 shares reserved for future issuance under the 2017 ESPP.

Stock Options

The fair values of stock option grants during the years ended December 31, 2022, 2021 and 2020 were calculated on the date of grant using the Black-Scholes option pricing model. Compensation expense is recognized over the period of service, generally the vesting period. During the year ended December 31, 2022, 1,746,270 stock options were granted by the Company. The following weighted average assumptions were used in the Black-Scholes options pricing model to estimate the fair value of stock options for the specified reporting periods:


	Year Ended December 31,
	2022		2021		2020
Risk free rate	1.7	%	0.6	%	1.5	%
Expected volatility	50	%	59	%	64	%
Expected life (in years)	6.4		6.4		6.3
Expected dividend yield	—		—		—

The risk-free interest rate was based on rates established by the Federal Reserve. The Company’s expected volatility was based on analysis of the Company’s historical volatility. The expected life of the Company’s options was determined using the Company's historical exercise activity. The dividend yield is based upon the fact that the Company has not historically paid dividends and does not expect to pay dividends in the foreseeable future.

The following table summarizes our stock option activity and related information for the year ended December 31, 2022:


		Weighted Average
	Shares Underlying Options	Exercise Price		Remaining Contractual Term (in years)	Aggregate Intrinsic Value (in thousands)
Outstanding at December 31, 2021	8,886,284	$	20.26	6.27	$	96,577
Granted	1,746,270	26.76		—	—
Forfeited and expired	(325,286)	24.63		—	—
Exercised	(374,846)	12.23		—	5,079
Outstanding at December 31, 2022	9,932,422	$	21.56	5.79	$	24,658
Vested and expected to vest at December 31, 2022	9,932,422	$	21.56	5.79	$	24,658

The aggregate intrinsic value of stock options exercised in the years ended December 31, 2022, 2021 and 2020 was $5.1 million, $7.5 million, and $1.8 million, respectively.

The following table summarizes our stock options exercisable at December 31, 2022, 2021 and 2020:


		Weighted Average
	Shares Underlying Options	Exercise Price		Remaining Contractual Term (in years)	Aggregate Intrinsic Value (in thousands)
Exercisable at December 31, 2020	5,488,207	$	19.37	5.36	$	46,626
Exercisable at December 31, 2021	6,011,349	$	19.34	5.16	$	71,063
Exercisable at December 31, 2022	6,999,669	$	20.22	4.62	$	21,806

The weighted average grant date fair value of options granted was $13.45, $14.00, and $9.54 during the years ended December 31, 2022, 2021 and 2020, respectively. The aggregate intrinsic value for outstanding options is calculated as the difference between the exercise price of the underlying awards and the closing price of the Company’s common stock of $21.03, $31.04 and $27.25 as of December 31, 2022, 2021 and 2020, respectively. Unrecognized compensation cost associated with unvested stock options amounts to $31.9 million as of December 31, 2022, which will be expensed over a weighted average remaining vesting period of 2.5 years.

In connection with the retirement of a Board Member, formerly the Chief Executive Officer, the Board of Directors approved a modification to extend the deadline to exercise each stock option held to the earlier of five years or the expiration of the 10-year term of the option. The modification resulted in incremental compensation cost of $3.2 million for the year ended December 31, 2022.

Restricted Stock Units

As of December 31, 2022, there was approximately $44.3 million of unrecognized compensation cost related to restricted stock units ("RSUs") granted. This amount is expected to be recognized over a weighted average period of 2.7 years.

The following table summarizes our restricted stock unit activity for the year ended December 31, 2022:


	Number of RSUs	Weighted Average Grant Date Fair Value
Unvested December 31, 2021	1,473,949	$	21.88
Granted	1,533,518	26.07
Vested	(543,252)	21.21
Forfeited/cancelled	(120,506)	24.35
Unvested December 31, 2022	2,343,709	$	24.65

The fair value of restricted stock units vested for the years ended December 31, 2022, 2021 and 2020 was $11.5 million, $7.4 million, and $5.3 million, respectively. The weighted average grant date fair value for stock awards granted during the years ended December 31, 2022, 2021 and 2020 was $21.21, $18.08, and $18.43, respectively.

Performance-based Stock Units

PSUs are subject to vest only if certain specified criteria are achieved. As of December 31, 2022, there was approximately $2.0 million of unrecognized compensation cost related to performance-based stock units ("PSUs") granted. This amount is expected to be recognized over a weighted average period of 1.1 years.

The following table summarizes our performance-based stock unit activity for the year ended December 31, 2022:


	Number of PSUs	Weighted Average Grant Date Fair Value
Unvested December 31, 2021	52,500	$	18.73
Granted	127,048	27.54
Vested	(17,500)	25.26
Forfeited/cancelled	(5,000)	15.46
Unvested December 31, 2022	157,048	$	25.24

The fair value of performance-based stock units vested for the years ended December 31, 2022, 2021 and 2020 was $0.4 million, $1.8 million, and $2.3 million, respectively. The weighted average grant date fair value for performance-based stock awards granted during the years ended December 31, 2022, 2021 and 2020 was $25.26, $15.46, and $21.58, respectively.

Share Based Compensation

Total non-cash stock-based compensation expense consisted of the following for the years ended December 31, 2022, 2021 and 2020 (in thousands):


	Year Ended December 31,
	2022		2021		2020
Selling, general and administrative expenses	$	25,319	$	18,134	$	14,247
Research and development expenses	13,858		12,632		9,367
Total stock-based compensation expense	$	39,177	$	30,766	$	23,614

- Definition

The entire disclosure for shareholders' equity comprised of portions attributable to the parent entity and noncontrolling interest, including other comprehensive income. Includes, but is not limited to, balances of common stock, preferred stock, additional paid-in capital, other capital and retained earnings, accumulated balance for each classification of other comprehensive income and amount of comprehensive income.

+ References

Reference 1: http://fasb.org/us-gaap/role/ref/legacyRef
-Publisher FASB
-Name Accounting Standards Codification
-Topic 505
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Reference 2: http://www.xbrl.org/2003/role/disclosureRef
-Publisher FASB
-Name Accounting Standards Codification
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Reference 3: http://www.xbrl.org/2003/role/disclosureRef
-Publisher FASB
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Reference 4: http://www.xbrl.org/2003/role/disclosureRef
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Reference 9: http://www.xbrl.org/2003/role/disclosureRef
-Publisher FASB
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Reference 10: http://www.xbrl.org/2003/role/disclosureRef
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-Publisher FASB
-Name Accounting Standards Codification
-Topic 505
-SubTopic 10
-Section 50
-Paragraph 14
-Subparagraph (a)
-URI https://asc.fasb.org/extlink&oid=126973232&loc=SL123496171-112644

Reference 12: http://www.xbrl.org/2003/role/disclosureRef
-Publisher FASB
-Name Accounting Standards Codification
-Topic 505
-SubTopic 10
-Section 50
-Paragraph 18
-Subparagraph (b)
-URI https://asc.fasb.org/extlink&oid=126973232&loc=SL123496189-112644

Reference 13: http://www.xbrl.org/2003/role/disclosureRef
-Publisher FASB
-Name Accounting Standards Codification
-Topic 505
-SubTopic 10
-Section 50
-Paragraph 16
-Subparagraph (b)
-URI https://asc.fasb.org/extlink&oid=126973232&loc=SL123496180-112644

Reference 14: http://www.xbrl.org/2003/role/disclosureRef
-Publisher FASB
-Name Accounting Standards Codification
-Topic 505
-SubTopic 10
-Section 50
-Paragraph 13
-Subparagraph (i)
-URI https://asc.fasb.org/extlink&oid=126973232&loc=SL123496158-112644

Reference 15: http://www.xbrl.org/2003/role/disclosureRef
-Publisher FASB
-Name Accounting Standards Codification
-Topic 235
-SubTopic 10
-Section S99
-Paragraph 1
-Subparagraph (SX 210.4-08(e)(1))
-URI https://asc.fasb.org/extlink&oid=120395691&loc=d3e23780-122690

Reference 16: http://www.xbrl.org/2003/role/disclosureRef
-Publisher FASB
-Name Accounting Standards Codification
-Topic 815
-SubTopic 40
-Section 50
-Paragraph 6
-Subparagraph (a)
-URI https://asc.fasb.org/extlink&oid=126731327&loc=SL126733271-114008

+ Details

NET LOSS PER COMMON SHARE

12 Months Ended

Dec. 31, 2022

Earnings Per Share [Abstract]

NET LOSS PER COMMON SHARE

Basic and diluted net loss per common share is calculated by dividing net loss applicable to common stockholders by the weighted-average number of common shares outstanding during the period. The Company’s potentially dilutive shares, which include outstanding stock options, restricted stock units, and shares issuable upon conversion of the 2025 Notes and 2029 Notes, are considered to be common stock equivalents and are not included in the calculation of diluted net loss per share because their effect is anti-dilutive.

Basic and diluted net loss per share is calculated as follows (net loss amounts are stated in thousands):


	For the year ended December 31,
	2022					2021					2020
	Shares	Net loss		EPS		Shares	Net loss		EPS		Shares	Net loss		EPS





Basic and diluted loss per share	63,758,515	$	(278,482)	$	(4.37)	59,832,287	$	(180,091)	$	(3.01)	47,539,631	$	(169,431)	$	(3.56)

For the years ended December 31, 2022, 2021 and 2020, the following shares were excluded because they were anti-dilutive:


	For the year ended December 31,
	2022	2021	2020
Convertible debt	10,869,000	7,113,402	7,113,402
Options	10,041,249	9,214,188	8,349,310
Restricted stock	2,175,100	1,569,470	1,368,096
Total anti-dilutive shares	23,085,349	17,897,060	16,830,808

INCOME TAXES

12 Months Ended

Dec. 31, 2022

Income Tax Disclosure [Abstract]

INCOME TAXES

For financial reporting purposes, net loss before income taxes includes the following components (in thousands):


	Year Ended December 31,
	2022		2021		2020
United States	$	(215,901)	$	(124,132)	$	(65,881)
Foreign*	(62,268)		(55,550)		(122,909)
Total	$	(278,169)	$	(179,682)	$	(188,790)

*Foreign losses in 2020 include charges relate to IPR&D. See Note 5 for further discussion.

The components of the provision (benefit) for income taxes, in the Consolidated Statements of Operations are as follows (in thousands):


	2022		2021		2020
Current
Federal	$	—	$	—	$	(19,436)
State	314		412		74
Foreign	(1)		(3)		3
	313		409		(19,359)
Deferred
Federal	—		—		—
State	—		—		—
	—		—		—
Total tax provision (benefit)	$	313	$	409	$	(19,359)

The following is a reconciliation of the statutory federal income tax rate to the Company’s effective tax rate expressed as a percentage of loss before income taxes:


	2022		2021		2020
Statutory rate - federal	(21.00)	%	(21.00)	%	(21.00)	%
State taxes, net of federal benefit	(3.46)	%	(2.71)	%	(1.57)	%
Foreign rate differential	0.96	%	3.28	%	6.65	%
IPR&D	—	%	—	%	6.50	%
Federal IRS Audit settlement	—	%	—	%	1.62	%
Nondeductible executive compensation	0.49	%	0.48	%	—	%
Excess tax benefits associated with share-based awards	(0.20)	%	0.19	%	—	%
Other permanent differences	0.20	%	0.15	%	0.09	%
Tax credits	(6.65)	%	(10.87)	%	(4.49)	%
Return to provision adjustments and other true-ups	(0.21)	%	2.42	%	4.88	%
CARES Act-Net operating loss carryback	—	%	—	%	(9.58)	%
Adoption of ASU 2020-06	(4.21)	%	—	%	—	%
Other	—	%	0.20	%	(0.10)	%
Change in valuation allowance	34.19	%	28.09	%	6.75	%
Income tax provision (benefit)	0.11	%	0.23	%	(10.25)	%

The significant components of the Company’s deferred tax assets and liabilities as of December 31, 2022 and 2021 are as follows (in thousands):


	2022		2021
Deferred Tax Assets:
Net operating loss	$	49,811	$	38,618
Research and development and other tax credits	91,052		69,213
Capitalized research and development	36,442		—
Contingent consideration	18,124		16,726
Other accrued expenses	13,247		8,678
Stock based compensation	22,936		18,003
Charitable contributions	4,781		3,427
Intangible assets greater than book	51,391		48,801
Interest expense limitation	4,346		2,802
Operating lease liabilities	8,018		8,825
Tax basis depreciation greater than book depreciation	105		13
Difference in basis of loan costs	15		—
	300,268		215,106
Deferred Tax Liabilities:
Operating lease right of use assets	(6,658)		(5,782)
Convertible debt	—		(11,020)
Prepaid assets	(206)		—
	(6,864)		(16,802)

Net deferred tax assets before valuation allowance	293,404		198,304
Valuation allowance	(293,404)		(198,304)
Total deferred tax assets	$	—	$	—

The Company has established a full valuation allowance against its U.S. federal, state, and foreign deferred tax assets due to the uncertainty surrounding the realization of such assets in future periods. The ultimate realization of deferred tax assets is dependent upon the generation of future taxable income during the periods in which temporary differences become deductible. Management considers the scheduled reversal of deferred liabilities and tax planning strategies in making this assessment and evaluates the recoverability of the deferred tax assets as of each reporting date. At such time as it is determined that it is more likely than not that deferred assets are realizable, the valuation allowance will be reduced accordingly and recorded as a tax benefit.

The Company has recorded a valuation allowance of $293.4 million as of December 31, 2022 to reflect the estimated amount of deferred tax assets that may not be realized. The Company increased its valuation allowance by $95.1 million and $50.5 million for the years ended December 31, 2022 and 2021, respectively.

As of December 31, 2022, the Company had available unused U.S. federal and state net operating loss (“NOL”) carryforwards of $90.2 million and $174.1 million, respectively, all of which are fully offset by a valuation allowance. Additionally, the Company had an interest expense limitation carryforward of $20.1 million that is fully offset by a valuation allowance. The federal NOL and interest expense limitation carryforward have an indefinite life. The state NOL carryforwards will begin to expire in 2023 unless previously utilized, except for $26.2 million of the state net operating losses that have an indefinite carryforward period. In addition, at December 31, 2022, the Company had federal orphan drug tax credit carryforwards of $82.0 million that begin to expire in 2033 unless utilized, federal research and development tax credit carryforwards of $5.4 million that begin to expire in 2033 unless utilized, state research and development tax credit carryforwards of $15.2 million, of which $1.4 million begin to expire in 2030 unless utilized and $13.8 million that have an indefinite carryforward period, and California Competes tax credit carryforwards of $2.0 million that begin to expire in 2024. Pursuant to Internal Revenue Code Sections 382 and 383, use of the Company’s federal net operating loss and credit carryforwards may be limited upon a cumulative change in ownership of more than 50% within a three-year period. The Company completed a study to analyze whether any ownership changes had occurred through December 31, 2021, and determined no ownership changes occurred. The Company is in the process of updating its analysis of owner shifts to determine whether an ownership change occurred since December 31, 2021.

As of December 31, 2022, the Company had Irish NOL carryforwards of $15.1 million which are fully offset by a valuation allowance and have an indefinite life. The Company also had Swiss NOL carryforwards of $148.2 million which are fully offset by a valuation allowance and begin to expire in 2023 as well as Federal Act on Tax Reform and AHV Financing (“TRAF”) cantonal tax benefits of $526.2 million which expire in 2029.

The Company accounts for uncertain tax benefits in accordance with the provisions of ASC 740-10 of the Accounting for Uncertainty in Income Taxes. As of December 31, 2022, the Company had $11.5 million in unrecognized tax benefits of which $9.9 million related to orphan drug and research and development tax credits and $1.6 million related to net operating loss carryforwards which were recorded as a reduction to the deferred tax assets with a corresponding reduction in the Company’s valuation allowance of $11.5 million. To the extent unrecognized tax benefits are recognized at a time when a valuation allowance does not exist, the recognition of the $11.5 million tax benefit would reduce the effective tax rate. The Company does not anticipate that the amount of unrecognized tax benefits as of December 31, 2022 will change materially within the following 12 months.

A reconciliation of the Company's unrecognized tax benefits for the years 2022 and 2021 is provided in the following table (in thousands):


	2022		2021
Balance as of January 1:	$	7,825	$	5,193
Increase in current period positions	2,056		2,255
Decrease due to settlements with tax authorities	(310)		—
Increase in prior period positions	1,919		377
Balance as of December 31:	$	11,490	$	7,825

The Company files income tax returns in the U.S. federal jurisdiction, various state and local, and foreign jurisdictions. With few exceptions, the Company’s income tax returns are open to examination by federal authorities for the years ended December 31, 2019, and forward, and for state and foreign tax authorities, for the years ended December 31, 2018, and forward.

The Company recognizes interest and penalties as a component of income tax expense. Interest and penalties for the years ended December 31, 2022 and 2021 were less than $0.1 million. The Company did not recognize any interest or penalties for the year ended December 31, 2020.

- Definition

The entire disclosure for income taxes. Disclosures may include net deferred tax liability or asset recognized in an enterprise's statement of financial position, net change during the year in the total valuation allowance, approximate tax effect of each type of temporary difference and carryforward that gives rise to a significant portion of deferred tax liabilities and deferred tax assets, utilization of a tax carryback, and tax uncertainties information.

+ References

Reference 1: http://www.xbrl.org/2003/role/disclosureRef
-Publisher FASB
-Name Accounting Standards Codification
-Topic 740
-SubTopic 10
-Section 50
-Paragraph 21
-URI https://asc.fasb.org/extlink&oid=121826272&loc=d3e32857-109319

Reference 2: http://www.xbrl.org/2003/role/disclosureRef
-Publisher FASB
-Name Accounting Standards Codification
-Topic 740
-SubTopic 10
-Section S99
-Paragraph 2
-Subparagraph (SAB Topic 11.C)
-URI https://asc.fasb.org/extlink&oid=122134291&loc=d3e330215-122817

Reference 3: http://www.xbrl.org/2003/role/disclosureRef
-Publisher FASB
-Name Accounting Standards Codification
-Topic 740
-SubTopic 10
-Section 50
-Paragraph 17
-Subparagraph (b)
-URI https://asc.fasb.org/extlink&oid=121826272&loc=d3e32809-109319

Reference 4: http://www.xbrl.org/2003/role/disclosureRef
-Publisher FASB
-Name Accounting Standards Codification
-Topic 740
-SubTopic 10
-Section S99
-Paragraph 1
-Subparagraph (SAB TOPIC 6.I.5.Q1)
-URI https://asc.fasb.org/extlink&oid=122134291&loc=d3e330036-122817

Reference 5: http://www.xbrl.org/2003/role/disclosureRef
-Publisher FASB
-Name Accounting Standards Codification
-Topic 740
-SubTopic 10
-Section 50
-Paragraph 14
-URI https://asc.fasb.org/extlink&oid=121826272&loc=d3e32705-109319

Reference 6: http://www.xbrl.org/2003/role/disclosureRef
-Publisher FASB
-Name Accounting Standards Codification
-Topic 740
-SubTopic 270
-Section 50
-Paragraph 1
-URI https://asc.fasb.org/extlink&oid=6424409&loc=d3e44925-109338

Reference 7: http://www.xbrl.org/2003/role/disclosureRef
-Publisher FASB
-Name Accounting Standards Codification
-Topic 740
-URI https://asc.fasb.org/topic&trid=2144680

Reference 8: http://www.xbrl.org/2003/role/disclosureRef
-Publisher FASB
-Name Accounting Standards Codification
-Topic 740
-SubTopic 30
-Section 50
-Paragraph 2
-Subparagraph (a)
-URI https://asc.fasb.org/extlink&oid=6424122&loc=d3e41874-109331

Reference 9: http://www.xbrl.org/2003/role/disclosureRef
-Publisher FASB
-Name Accounting Standards Codification
-Topic 235
-SubTopic 10
-Section S99
-Paragraph 1
-Subparagraph (SX 210.4-08(h)(2))
-URI https://asc.fasb.org/extlink&oid=120395691&loc=d3e23780-122690

+ Details

EQUITY OFFERINGS	12 Months Ended
EQUITY OFFERINGS	Dec. 31, 2022
Equity [Abstract]
EQUITY OFFERINGS	EQUITY OFFERINGS Underwritten Public Offering of Common Stock In February 2021, the Company sold an aggregate of 7.5 million shares of its common stock in an underwritten public offering, at a price of $26.75 per share. The net proceeds to the Company from the offering, after deducting the underwriting discounts and offering expenses, were $189.3 million. At-the-Market Equity Offering In February 2020, the Company entered into an Open Market Sale Agreement ("ATM Agreement") with Jefferies LLC, as agent (“Jefferies”), pursuant to which the Company may offer and sell, from time to time through Jefferies, shares of its common stock having an aggregate offering price of up to $100.0 million. Of the $100.0 million originally authorized for sale under the ATM Agreement, approximately $28.6 million were sold under the Company's prior registration statement on Form S-3 (Registration Statement No. 333-227182). An additional $51.9 million were sold under the Company's effective registration statement on Form S-3 (Registration Statement No. 333-259311), which included $20.1 million from the settlement of 701,600 shares under the ATM Agreement in the year ended December 31, 2022. As of December 31, 2022, an aggregate amount of $19.5 million remained eligible for sale under the ATM Agreement. Authorized Shares of Common Stock On May 14, 2021, in connection with the Company’s 2021 Annual Meeting of Stockholders, the Company’s stockholders approved, among other matters, a Certificate of Amendment (“Certificate of Amendment”) to the Company’s Certificate of Incorporation to increase the number of shares of common stock authorized for issuance thereunder from 100,000,000 to 200,000,000. Effective May 18, 2021, the Certificate of Amendment was filed with the Secretary of State of the State of Delaware.

RETIREMENT PLAN	12 Months Ended
RETIREMENT PLAN	Dec. 31, 2022
Retirement Benefits [Abstract]
RETIREMENT PLAN	RETIREMENT PLAN 401(k) Savings Plan The Company has a 401(k) defined contribution savings plan for the benefit of all eligible employees. Employer matching contributions were $2.1 million, $1.3 million, and $1.2 million for the years ended December 31, 2022, 2021 and 2020, respectively. Switzerland Defined Benefit Plan The Company maintains a defined benefit pension plan covering employees of its Swiss subsidiary, Travere Therapeutics Switzerland GmbH (the "Swiss Plan"). The Swiss Plan is a government-mandated retirement fund that provides employees with a minimum benefit. Employer and employee contributions are made to the Swiss Plan based on various percentages of participants' salaries and wages that vary according to the participants' age and other factors. As of December 31, 2022, the projected benefit obligations under the Swiss Plan were approximately $1.8 million, and plan assets were approximately $1.4 million. The funded status of the Swiss Plan is included in other long-term liabilities on the Company's Consolidated Balance Sheets.

PROPERTY AND EQUIPMENT

12 Months Ended

Dec. 31, 2022

Property, Plant and Equipment [Abstract]

PROPERTY AND EQUIPMENT

Property, plant and equipment, net consisted of the following (in thousands):


	December 31,
	2022		2021
Computers and equipment	$	2,049	$	1,950
Furniture and fixtures	2,938		2,783
Leasehold improvements	9,180		8,790
Construction-in-progress	77		631
	14,244		14,154
Less: Accumulated depreciation	(5,195)		(3,048)
Total property and equipment, net	$	9,049	$	11,106

The construction-in-process balance consists of costs related to the Company’s leasehold improvements at its facilities in San Diego, California.

Depreciation expense for the years ended December 31, 2022, 2021 and 2020 was $2.1 million, $1.7 million and $2.1 million, respectively.

LEASES

12 Months Ended

Dec. 31, 2022

Leases [Abstract]

LEASES

As of December 31, 2022, the Company had two operating leases, including one operating lease with Kilroy Realty, L.P. (the "Landlord") for office space located in San Diego, California, which was entered into in April 2019 and subsequently amended in May 2020. Coinciding with the Company's ability to direct the use of the office space, which occurred in phases over 2020, and utilizing a discount rate equal to the Company's estimated incremental borrowing rate, the Company established ROU assets totaling $34.6 million and lease liabilities totaling $34.5 million. The total ROU asset and lease liability at measurement were each offset by lease incentives associated with tenant improvement allowances totaling $7.9 million.

The initial term of the office lease ends in August 2028, and the Landlord has granted the Company an option to extend the term of the lease by a period of 5 years. At this time, it is not reasonably certain that the Company will extend the term of the lease and therefore the renewal period has been excluded from the aforementioned ROU asset and lease liability measurements. The measurement of the lease term occurs from the February 2021 occupancy date of the office space delivered in September 2020. The aggregate base rent due over the initial term of the lease is approximately $49.5 million.

The Company also has one operating lease with Esprit Investments Limited for office space located in Dublin, Ireland, which was entered into in October 2022. The initial term of the office lease ends in September 2027. The lease provides the option to extend the term of the lease by a period of 5 years, although it is not reasonably certain that the Company will elect this option and therefore the renewal period has been excluded from the initial lease measurement. The aggregate base rent due over the initial term of the lease is approximately $0.5 million. Utilizing a discount rate equal to the Company's borrowing rate, the Company established an ROU asset and corresponding lease liability of $0.4 million.

Following is a schedule of the future minimum rental commitments for our operating leases reconciled to the lease liability and ROU asset as of December 31, 2022 (in thousands):


	December 31, 2022
2023	$	6,314
2024	6,501
2025	6,673
2026	6,889
2027	7,064
Thereafter	4,781
Total undiscounted future minimum payments	38,222
Present value discount	(6,279)
Total lease liability	31,943
Unamortized lease incentives	(5,575)
Cash payments in excess of straight-line lease expense	(5,368)
Total ROU asset	$	21,000

The weighted-average remaining lease term and weighted-average discount rate of the Company's operating leases are as follows:


	December 31,
	2022		2021
Weighted-average remaining lease term in years	5.7		6.7
Weighted-average discount rate	6.48	%	6.47	%

For the year ended December 31, 2022, 2021 and 2020 the Company recorded $5.0 million, $4.9 million, and $2.0 million, respectively, in expense related to operating leases, including amortized tenant improvement allowances.

SUBSEQUENT EVENTS	12 Months Ended
SUBSEQUENT EVENTS	Dec. 31, 2022
Subsequent Events [Abstract]
SUBSEQUENT EVENTS	SUBSEQUENT EVENTS Ligand Milestone Payment Pursuant to the Ligand License Agreement, the Company is obligated to make a $23.0 million milestone payment to Ligand (and BMS) in the first quarter of 2023, as a result of the accelerated approval granted to FILSPARI (sparsentan). Upon payment, the $23.0 million will be capitalized as an intangible asset on the Consolidated Balance Sheets.

SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES (Policies)

12 Months Ended

Dec. 31, 2022

Accounting Policies [Abstract]

Principles of Consolidation

Principles of ConsolidationThe consolidated financial statements represent the consolidation of the accounts of the Company, its subsidiaries and variable interest entities for which the Company has been determined to be the primary beneficiary, in conformity with accounting principles generally accepted in the United States ("U.S. GAAP"). All intercompany accounts and transactions have been eliminated in consolidation.

Use of Estimates

Revenue Recognition and Deductions from Revenue

Revenue Recognition

Deductions from Revenue

Research and Development Expenses

Clinical Trial Expenses

Share-Based Compensation


	Expiration Term	Vesting Term
Stock Options	10 years	3 to 4 years
Restricted Stock Units	----	1 to 4 years

Earnings (Loss) Per Share

Cash and Cash Equivalents

Cash and Cash EquivalentsThe Company considers all highly liquid marketable securities with an original maturity of three months or less to be cash equivalents. Due to the short-term maturity of such investments, the carrying amounts are a reasonable estimate of fair value.

Concentration of Credit Risk

Marketable Debt Securities

Accounts Receivables, Net

Accounts Receivables, NetTrade accounts receivable are recorded net of reserves for prompt pay discounts and expected credit losses. Estimates for allowances for credit losses are determined based on existing contractual obligations, historical payment patterns and individual customer circumstances. The allowance for credit losses was zero at both December 31, 2022 and 2021, respectively. For the years ended December 31, 2022, 2021 and 2020, bad debt expense recorded in the Consolidated Statements of Operations and Comprehensive Loss was immaterial. The Company's evaluation and application of ASU No. 2016-13, Financial Instruments - Credit Losses for the current period included an assessment of our aged trade receivables balances and their underlying credit risk characteristics. Our evaluation of past events, current conditions, and reasonable and supportable forecasts about the future resulted in an expectation of immaterial credit losses.

Supplier Concentration Risk

Inventory, Related Reserves and Cost of Goods SoldInventory, which is recorded at the lower of cost or net realizable value, includes materials, labor, and other direct and indirect costs and is valued using the first-in, first-out method. The Company periodically analyzes its inventory levels to identify inventory that may expire prior to expected sale or has a cost basis in excess of its estimated realizable value, and writes down such inventory as appropriate. In addition, the Company's products are subject to strict quality control and monitoring which the Company’s manufacturers perform throughout their manufacturing process. The Company does not directly manufacture any product. The Company has single suppliers for products Chenodal and Thiola, and prospectively arranges for manufacture from contract service providers for its product Cholbam.

Capitalization of Inventory Costs

Segment Information

Property and Equipment, net

The major classifications of property and equipment, including their respective expected useful lives, consist of the following:


Computers and equipment			3 years
Furniture and fixtures			7 years
Leasehold improvements			Shorter of length of lease or life of the asset

Leases

Acquired IPR&D

Intangible Assets, Net and Intangible Assets with Cost Accumulation Model

Intangible Assets, Net

Intangible Assets with Cost Accumulation Model

Variable Interest Entity

Goodwill

GoodwillGoodwill represents the excess of purchase price over fair value of net assets acquired in a business combination and is not amortized. Goodwill is subject to impairment testing at least annually or when a triggering event occurs that could indicate a potential impairment. The Company has one segment and one reporting unit and as such reviews goodwill for impairment at the consolidated level.

Impairment of Long-Lived Assets

Contingent Consideration

Income Taxes

Foreign Currency Translation

Functional and presentation currency

Transactions and balances

The results and financial position of the Company that have a functional currency different from the US dollar are translated as follows:

a.assets and liabilities presented are translated at the closing exchange rate as of December 31, 2022 and 2021;

c.significant transactions use the exchange rate on the date of the transaction.

All resulting exchange differences arising from such translations are recognized directly in comprehensive income and presented as a separate component of equity.

Reclassifications

Patents

PatentsThe Company expenses external costs, such as filing fees and associated attorney fees, incurred to obtain issued patents and patent applications pending. The Company also expenses costs associated with maintaining and defending patents subsequent to their issuance in the period incurred.

Legal Contingencies

Recently Adopted and Issued Accounting Pronouncements

Recently Adopted Accounting Pronouncements

Recently Issued Accounting Pronouncements

Fair Value Measurements

Level 1 – Unadjusted quoted prices in active markets that are accessible at the measurement date for identical, unrestricted assets or liabilities;

Level 2 – Quoted prices in markets that are not active or financial instruments for which all significant inputs are observable, either directly or indirectly; and

Level 3 – Prices or valuations that require inputs that are both significant to the fair value measurement and unobservable.

SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES (Tables)

12 Months Ended

Dec. 31, 2022

Accounting Policies [Abstract]

Schedule of vesting award terms


	Expiration Term	Vesting Term
Stock Options	10 years	3 to 4 years
Restricted Stock Units	----	1 to 4 years

Schedule of inventory, net of reserve

Inventory, net of reserves, consisted of the following at December 31, 2022 and 2021 (in thousands):


	December 31, 2022		December 31, 2021
Raw materials	$	3,627	$	5,205
Finished goods	3,295		2,108
Total inventory	$	6,922	$	7,313

Schedule of major classifications of property, equipment and software, including their respective expected useful lives

The major classifications of property and equipment, including their respective expected useful lives, consist of the following:


Computers and equipment			3 years
Furniture and fixtures			7 years
Leasehold improvements			Shorter of length of lease or life of the asset

Property, plant and equipment, net consisted of the following (in thousands):


	December 31,
	2022		2021
Computers and equipment	$	2,049	$	1,950
Furniture and fixtures	2,938		2,783
Leasehold improvements	9,180		8,790
Construction-in-progress	77		631
	14,244		14,154
Less: Accumulated depreciation	(5,195)		(3,048)
Total property and equipment, net	$	9,049	$	11,106

Summary of foreign currency translation adjustment included in accumulated other comprehensive loss

The following table summarizes the foreign currency translation adjustment included in accumulated other comprehensive loss for the year ended December 31, 2022, 2021 and 2020 (in thousands):


	Foreign Currency Translation Adjustments included in Accumulated Other Comprehensive Loss
	2022		2021		2020
Balance at January 1,	$	(92)	$	(1,607)	$	(155)
Foreign currency translation adjustments	507		1,515		(1,452)
Balance at December 31,	$	415	$	(92)	$	(1,607)

REVENUE RECOGNITION (Tables)

12 Months Ended

Dec. 31, 2022

Revenue from Contract with Customer [Abstract]

Summary of net product revenue

The following table summarizes net product sales for the year ended December 31, 2022, 2021 and 2020 (in thousands):


	Year Ended December 31,
	2022		2021		2020
Bile acid products	$	102,558	$	95,654	$	89,438
Tiopronin products	97,970		115,122		108,883
Total net product sales	$	200,528	$	210,776	$	198,321

COLLABORATION AND LICENSE AGREEMENTS (Tables)

12 Months Ended

Dec. 31, 2022

Organization, Consolidation and Presentation of Financial Statements [Abstract]

Summary of changes in deferred revenue

The following table sets forth a summary of changes in deferred revenue for the years ended December 31, 2022 and 2021 (in thousands):


	Deferred Revenue
	2022		2021
Balance at January 1,	$	36,647	$	—
New consideration resulting in deferred revenue	—		55,000
License revenue	—		(12,000)
Collaboration revenue	(11,490)		(4,714)
Foreign currency impact	(2,250)		(1,639)
Balance at December 31,	$	22,907	$	36,647

MARKETABLE DEBT SECURITIES (Tables)

12 Months Ended

Dec. 31, 2022

Investments, Debt and Equity Securities [Abstract]

Schedule of marketable debt securities

Marketable debt securities consist of the following (in thousands):


	As of December 31,
	2022		2021
Marketable debt securities:
Commercial paper	$	123,647	$	127,379
Corporate debt securities	224,055		233,319
Securities of government-sponsored entities	40,855		26,431
Total marketable debt securities	$	388,557	$	387,129

Schedule of available for sale securities

The following is a summary of short-term marketable debt securities classified as available-for-sale as of December 31, 2022 (in thousands):


	Contractual Maturity (in years)	Amortized Cost		Unrealized Gains		Unrealized Losses		Aggregate Estimated Fair Value
Marketable debt securities:
Commercial paper	Less than 1	$	124,301	$	2	$	(656)	$	123,647
Corporate debt securities	Less than 1	155,841		—		(1,355)		154,486
Securities of government-sponsored entities	Less than 1	7,473		—		(80)		7,393
Total maturity less than 1 year		287,615		2		(2,091)		285,526
Corporate debt securities	1 to 2	70,195		33		(659)		69,569
Securities of government-sponsored entities	1 to 2	33,702		6		(246)		33,462
Total maturity 1 to 2 years		103,897		39		(905)		103,031
Total available-for-sale marketable debt securities		$	391,512	$	41	$	(2,996)	$	388,557

The following is a summary of short-term marketable debt securities classified as available-for-sale as of December 31, 2021 (in thousands):


	Contractual Maturity (in years)	Amortized Cost		Unrealized Gains		Unrealized Losses		Aggregate Estimated Fair Value
Marketable debt securities:
Commercial paper	Less than 1	$	127,435	$	—	$	(56)	$	127,379
Corporate debt securities	Less than 1	113,001		—		(98)		112,903
Securities of government-sponsored entities	Less than 1	21,909		—		(5)		21,904
Total maturity less than 1 year		262,345		—		(159)		262,186
Corporate debt securities	1 to 2	120,705		—		(289)		120,416
Securities of government-sponsored entities	1 to 2	4,549		—		(22)		4,527
Total maturity 1 to 2 years		125,254		—		(311)		124,943
Total available-for-sale marketable debt securities		$	387,599	$	—	$	(470)	$	387,129

Schedule of debt securities in unrealized loss position

The following is a summary of available-for-sale marketable debt securities in an unrealized loss position with no credit losses reported as of December 31, 2022 (in thousands):


	Less Than 12 Months				12 Months or Greater				Total
Description of Securities	Fair Value		Unrealized Losses		Fair Value		Unrealized Losses		Fair Value		Unrealized Losses
Commercial paper	$	117,853	$	656	$	—	$	—	$	117,853	$	656
Corporate debt securities	99,066		1,041		107,964		973		207,030		2,014
Securities of government-sponsored entities	31,402		263		4,456		63		35,858		326
Total	$	248,321	$	1,960	$	112,420	$	1,036	$	360,741	$	2,996

The following is a summary of available-for-sale marketable debt securities in an unrealized loss position with no credit losses reported as of December 31, 2021 (in thousands):


	Less Than 12 Months				12 Months or Greater				Total
Description of Securities	Fair Value		Unrealized Losses		Fair Value		Unrealized Losses		Fair Value		Unrealized Losses
Commercial paper	$	122,380	$	56	$	—	$	—	$	122,380	$	56
Corporate debt securities	231,879		387		—		—		231,879		387
Securities of government-sponsored entities	26,431		27		—		—		26,431		27
Total	$	380,690	$	470	$	—	$	—	$	380,690	$	470

CONVERTIBLE NOTES PAYABLE (Tables)

12 Months Ended

Dec. 31, 2022

Debt Disclosure [Abstract]

Schedule of long-term debt instruments

The composition of the Company’s convertible senior notes are as follows (in thousands):


	December 31, 2022		December 31, 2021
2.25% convertible senior notes due 2029	$	316,250	$	—
2.50% convertible senior notes due 2025	68,904		276,000
Unamortized debt discount	—		(46,045)
Unamortized debt issuance costs - 2.25% convertible senior notes due 2029	(8,750)		—
Unamortized debt issuance costs - 2.50% convertible senior notes due 2025	(859)		(3,374)
Total convertible senior notes, net of unamortized debt discount and debt issuance costs	$	375,545	$	226,581

The following table sets forth total interest expense recognized related to the 2025 and 2029 Notes (in thousands):


	Year Ended December 31,
	2022		2021		2020
Contractual interest expense	$	8,433	$	6,900	$	6,900
Amortization of debt discount	—		10,339		9,578
Amortization of debt issuance costs	1,622		903		900
Total interest expense for the 2025 and 2029 Notes	$	10,055	$	18,142	$	17,378

- Definition

Tabular disclosure of long-debt instruments or arrangements, including identification, terms, features, collateral requirements and other information necessary to a fair presentation. These are debt arrangements that originally required repayment more than twelve months after issuance or greater than the normal operating cycle of the entity, if longer.

+ References

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Reference 8: http://fasb.org/us-gaap/role/ref/legacyRef
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Reference 10: http://fasb.org/us-gaap/role/ref/legacyRef
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-Name Accounting Standards Codification
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-URI https://asc.fasb.org/extlink&oid=123599511&loc=d3e64711-112823

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+ Details

FAIR VALUE MEASUREMENTS (Tables)

12 Months Ended

Dec. 31, 2022

Fair Value Disclosures [Abstract]

Schedule of fair value on a recurring basis


	As of December 31, 2022		Fair Value Hierarchy at December 31, 2022
	Total carrying and estimated fair value		Quoted prices in active markets (Level 1)		Significant other observable inputs (Level 2)		Significant unobservable inputs (Level 3)
Assets:
Cash and Cash Equivalents	$	61,688	$	61,688	$	—	$	—
Marketable debt securities, available-for-sale	388,557		—		388,557		—
Total	$	450,245	$	61,688	$	388,557	$	—
Liabilities:
Business combination-related contingent consideration	$	71,200	$	—	$	—	$	71,200
Total	$	71,200	$	—	$	—	$	71,200


	As of December 31, 2021		Fair Value Hierarchy at December 31, 2021
	Total carrying and estimated fair value		Quoted prices in active markets (Level 1)		Significant other observable inputs (Level 2)		Significant unobservable inputs (Level 3)
Assets:
Cash and Cash Equivalents	$	165,753	$	165,753	$	—	$	—
Marketable debt securities, available-for-sale	387,129		—		387,129		—
Total	$	552,882	$	165,753	$	387,129	$	—
Liabilities:
Business combination-related contingent consideration	$	67,100	$	—	$	—	$	67,100
Total	$	67,100	$	—	$	—	$	67,100

Schedule of discounts rates used

Discount rates used to determine the fair value at December 31, 2022 and 2021 are as follows:


	Revenue Discount		Payment Discount
	Cholbam	Chenodal
December 31, 2022	7.75%	8.00%	8.10%
December 31, 2021	6.25%	7.25%	6.48%

Schedule of fair value measurements of acquisition-related contingent consideration


	Fair Value Measurements of Acquisition-Related Contingent Consideration (Level 3)
	2022		2021
Balance at January 1,	$	67,100	$	65,100
Changes in the fair value of contingent consideration	15,006		22,260
Contractual payments	(8,066)		(17,444)
Contractual payments included in accrued liabilities at December 31,	(2,840)		(2,700)
Foreign currency impact	—		(116)
Balance at December 31,	$	71,200	$	67,100

INTANGIBLE ASSETS (Tables)

12 Months Ended

Dec. 31, 2022

Goodwill and Intangible Assets Disclosure [Abstract]

Schedule of amortizable intangible assets

Amortizable intangible assets as of December 31, 2022 (in thousands):


	Useful Life	Gross Carrying Amount		Accumulated Amortization		Net Book Value
Thiola license	15	$	139,383	$	(50,324)	$	89,059
Chenodal product rights	16	67,849		(37,175)		30,674
Economic interest - Cholbam revenue	10	75,900		(58,842)		17,058
Ligand license	11	19,400		(12,139)		7,261
Manchester customer relationships	10	403		(353)		50
Total amortizable intangible assets		$	302,935	$	(158,833)	$	144,102

Amortizable intangible assets as of December 31, 2021 (in thousands):


	Useful Life	Gross Carrying Amount		Accumulated Amortization		Net Book Value
Thiola license	15	$	123,316	$	(37,992)	$	85,324
Chenodal product rights	16	67,849		(32,938)		34,911
Economic interest - U.S. revenue Cholbam	10	75,900		(51,258)		24,642
Ligand license	11	7,900		(5,875)		2,025
Economic interest - International revenue Cholbam	10	7,982		(7,483)		499
Manchester customer relationships	10	403		(313)		90
Internal use software	5	207		(199)		8
Total amortizable intangible assets		$	283,557	$	(136,058)	$	147,499

Schedule of amortization expense for the next five years

The following table summarizes amortization expense for the year ended December 31, 2022, 2021 and 2020 (in thousands):


	2022		2021		2020
Research and development	$	6,264	$	1,158	$	1,162
Selling, general and administrative	24,700		23,788		21,275
Total amortization expense	$	30,964	$	24,946	$	22,437

As of December 31, 2022, amortization expense for the next five years and thereafter is expected to be as follows (in thousands):


2023	$	33,015
2024	25,793
2025	19,999
2026	18,129
2027	18,129
Thereafter	29,037
Total	$	144,102

ACCRUED EXPENSES (Tables)

12 Months Ended

Dec. 31, 2022

Payables and Accruals [Abstract]

Schedule of accrued expenses

Accrued expenses consist of the following at December 31, 2022 and 2021 (in thousands):


	2022		2021
Compensation related costs	$	35,267	$	25,998
Research and development	26,070		26,841
Sales discounts, rebates, and allowances	13,486		7,493
Selling, general and administrative	8,791		3,144
Accrued royalties	7,755		8,402
Miscellaneous accrued	4,373		3,302
Total accrued expenses	$	95,742	$	75,180

STOCKHOLDERS’ EQUITY (Tables)

12 Months Ended

Dec. 31, 2022

Equity [Abstract]

Assumptions used in black-scholes options pricing model

The following weighted average assumptions were used in the Black-Scholes options pricing model to estimate the fair value of stock options for the specified reporting periods:


	Year Ended December 31,
	2022		2021		2020
Risk free rate	1.7	%	0.6	%	1.5	%
Expected volatility	50	%	59	%	64	%
Expected life (in years)	6.4		6.4		6.3
Expected dividend yield	—		—		—

Share-based compensation, stock options, activity

The following table summarizes our stock option activity and related information for the year ended December 31, 2022:


		Weighted Average
	Shares Underlying Options	Exercise Price		Remaining Contractual Term (in years)	Aggregate Intrinsic Value (in thousands)
Outstanding at December 31, 2021	8,886,284	$	20.26	6.27	$	96,577
Granted	1,746,270	26.76		—	—
Forfeited and expired	(325,286)	24.63		—	—
Exercised	(374,846)	12.23		—	5,079
Outstanding at December 31, 2022	9,932,422	$	21.56	5.79	$	24,658
Vested and expected to vest at December 31, 2022	9,932,422	$	21.56	5.79	$	24,658

The following table summarizes our stock options exercisable at December 31, 2022, 2021 and 2020:


		Weighted Average
	Shares Underlying Options	Exercise Price		Remaining Contractual Term (in years)	Aggregate Intrinsic Value (in thousands)
Exercisable at December 31, 2020	5,488,207	$	19.37	5.36	$	46,626
Exercisable at December 31, 2021	6,011,349	$	19.34	5.16	$	71,063
Exercisable at December 31, 2022	6,999,669	$	20.22	4.62	$	21,806

Schedule of unvested restricted shares

The following table summarizes our restricted stock unit activity for the year ended December 31, 2022:


	Number of RSUs	Weighted Average Grant Date Fair Value
Unvested December 31, 2021	1,473,949	$	21.88
Granted	1,533,518	26.07
Vested	(543,252)	21.21
Forfeited/cancelled	(120,506)	24.35
Unvested December 31, 2022	2,343,709	$	24.65

Share-based compensation, performance shares award nonvested activity

The following table summarizes our performance-based stock unit activity for the year ended December 31, 2022:


	Number of PSUs	Weighted Average Grant Date Fair Value
Unvested December 31, 2021	52,500	$	18.73
Granted	127,048	27.54
Vested	(17,500)	25.26
Forfeited/cancelled	(5,000)	15.46
Unvested December 31, 2022	157,048	$	25.24

Schedule of share based compensation expenses

Total non-cash stock-based compensation expense consisted of the following for the years ended December 31, 2022, 2021 and 2020 (in thousands):


	Year Ended December 31,
	2022		2021		2020
Selling, general and administrative expenses	$	25,319	$	18,134	$	14,247
Research and development expenses	13,858		12,632		9,367
Total stock-based compensation expense	$	39,177	$	30,766	$	23,614

NET LOSS PER COMMON SHARE (Tables)

12 Months Ended

Dec. 31, 2022

Earnings Per Share [Abstract]

Schedule of basic and diluted net loss per share

Basic and diluted net loss per share is calculated as follows (net loss amounts are stated in thousands):


	For the year ended December 31,
	2022					2021					2020
	Shares	Net loss		EPS		Shares	Net loss		EPS		Shares	Net loss		EPS





Basic and diluted loss per share	63,758,515	$	(278,482)	$	(4.37)	59,832,287	$	(180,091)	$	(3.01)	47,539,631	$	(169,431)	$	(3.56)

Schedule of common stock options, convertible debt and restricted stock units anti-dilutive

For the years ended December 31, 2022, 2021 and 2020, the following shares were excluded because they were anti-dilutive:


	For the year ended December 31,
	2022	2021	2020
Convertible debt	10,869,000	7,113,402	7,113,402
Options	10,041,249	9,214,188	8,349,310
Restricted stock	2,175,100	1,569,470	1,368,096
Total anti-dilutive shares	23,085,349	17,897,060	16,830,808

INCOME TAXES (Tables)

12 Months Ended

Dec. 31, 2022

Income Tax Disclosure [Abstract]

Schedule of components of net income before incomes taxes

For financial reporting purposes, net loss before income taxes includes the following components (in thousands):


	Year Ended December 31,
	2022		2021		2020
United States	$	(215,901)	$	(124,132)	$	(65,881)
Foreign*	(62,268)		(55,550)		(122,909)
Total	$	(278,169)	$	(179,682)	$	(188,790)

*Foreign losses in 2020 include charges relate to IPR&D. See Note 5 for further discussion.

Schedule of income tax provision (benefit)

The components of the provision (benefit) for income taxes, in the Consolidated Statements of Operations are as follows (in thousands):


	2022		2021		2020
Current
Federal	$	—	$	—	$	(19,436)
State	314		412		74
Foreign	(1)		(3)		3
	313		409		(19,359)
Deferred
Federal	—		—		—
State	—		—		—
	—		—		—
Total tax provision (benefit)	$	313	$	409	$	(19,359)

Schedule of reconciliation of the statutory federal income tax expense (benefit)

The following is a reconciliation of the statutory federal income tax rate to the Company’s effective tax rate expressed as a percentage of loss before income taxes:


	2022		2021		2020
Statutory rate - federal	(21.00)	%	(21.00)	%	(21.00)	%
State taxes, net of federal benefit	(3.46)	%	(2.71)	%	(1.57)	%
Foreign rate differential	0.96	%	3.28	%	6.65	%
IPR&D	—	%	—	%	6.50	%
Federal IRS Audit settlement	—	%	—	%	1.62	%
Nondeductible executive compensation	0.49	%	0.48	%	—	%
Excess tax benefits associated with share-based awards	(0.20)	%	0.19	%	—	%
Other permanent differences	0.20	%	0.15	%	0.09	%
Tax credits	(6.65)	%	(10.87)	%	(4.49)	%
Return to provision adjustments and other true-ups	(0.21)	%	2.42	%	4.88	%
CARES Act-Net operating loss carryback	—	%	—	%	(9.58)	%
Adoption of ASU 2020-06	(4.21)	%	—	%	—	%
Other	—	%	0.20	%	(0.10)	%
Change in valuation allowance	34.19	%	28.09	%	6.75	%
Income tax provision (benefit)	0.11	%	0.23	%	(10.25)	%

Schedule of deferred tax assets and liabilities

The significant components of the Company’s deferred tax assets and liabilities as of December 31, 2022 and 2021 are as follows (in thousands):


	2022		2021
Deferred Tax Assets:
Net operating loss	$	49,811	$	38,618
Research and development and other tax credits	91,052		69,213
Capitalized research and development	36,442		—
Contingent consideration	18,124		16,726
Other accrued expenses	13,247		8,678
Stock based compensation	22,936		18,003
Charitable contributions	4,781		3,427
Intangible assets greater than book	51,391		48,801
Interest expense limitation	4,346		2,802
Operating lease liabilities	8,018		8,825
Tax basis depreciation greater than book depreciation	105		13
Difference in basis of loan costs	15		—
	300,268		215,106
Deferred Tax Liabilities:
Operating lease right of use assets	(6,658)		(5,782)
Convertible debt	—		(11,020)
Prepaid assets	(206)		—
	(6,864)		(16,802)

Net deferred tax assets before valuation allowance	293,404		198,304
Valuation allowance	(293,404)		(198,304)
Total deferred tax assets	$	—	$	—

Schedule of unrecognized tax benefits

A reconciliation of the Company's unrecognized tax benefits for the years 2022 and 2021 is provided in the following table (in thousands):


	2022		2021
Balance as of January 1:	$	7,825	$	5,193
Increase in current period positions	2,056		2,255
Decrease due to settlements with tax authorities	(310)		—
Increase in prior period positions	1,919		377
Balance as of December 31:	$	11,490	$	7,825

PROPERTY AND EQUIPMENT (Tables)

12 Months Ended

Dec. 31, 2022

Property, Plant and Equipment [Abstract]

Summary of property, plant, and equipment

The major classifications of property and equipment, including their respective expected useful lives, consist of the following:


Computers and equipment			3 years
Furniture and fixtures			7 years
Leasehold improvements			Shorter of length of lease or life of the asset

Property, plant and equipment, net consisted of the following (in thousands):


	December 31,
	2022		2021
Computers and equipment	$	2,049	$	1,950
Furniture and fixtures	2,938		2,783
Leasehold improvements	9,180		8,790
Construction-in-progress	77		631
	14,244		14,154
Less: Accumulated depreciation	(5,195)		(3,048)
Total property and equipment, net	$	9,049	$	11,106

LEASES (Tables)

12 Months Ended

Dec. 31, 2022

Leases [Abstract]

Schedule of future minimum rent commitments

Following is a schedule of the future minimum rental commitments for our operating leases reconciled to the lease liability and ROU asset as of December 31, 2022 (in thousands):


	December 31, 2022
2023	$	6,314
2024	6,501
2025	6,673
2026	6,889
2027	7,064
Thereafter	4,781
Total undiscounted future minimum payments	38,222
Present value discount	(6,279)
Total lease liability	31,943
Unamortized lease incentives	(5,575)
Cash payments in excess of straight-line lease expense	(5,368)
Total ROU asset	$	21,000

Schedule of weighted-average remaining lease term and discount rate

The weighted-average remaining lease term and weighted-average discount rate of the Company's operating leases are as follows:


	December 31,
	2022		2021
Weighted-average remaining lease term in years	5.7		6.7
Weighted-average discount rate	6.48	%	6.47	%

SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES - Narrative (Details)	3 Months Ended	12 Months Ended
	Dec. 31, 2022 USD ($)	Dec. 31, 2022 USD ($) clinicalTrial segment reporting_unit	Dec. 31, 2021 USD ($)	Dec. 31, 2020 USD ($)	Jan. 01, 2022 USD ($)
New Accounting Pronouncements or Change in Accounting Principle [Line Items]
Decreased in research and development expenses and related accrued expenses	$ 7,700,000	$ 1,500,000	$ 6,200,000
Number of Phase1/2 clinical trials in process \| clinicalTrial		1
Number of Phase 3 clinical trials in process \| clinicalTrial		3
Allowance for doubtful accounts	0	$ 0	0
Inventory reserve	3,500,000	3,500,000	4,100,000
Research and development		$ 7,500,000
Number of segments \| segment		1
Number of reporting units \| reporting_unit		1
Goodwill, impairment		$ 0	0	$ 0
Impairment of intangible assets		0	0	0
Additional paid-in capital	(1,059,975,000)	(1,059,975,000)	(1,068,634,000)
Reduction in accumulated deficit	$ (1,014,223,000)	(1,014,223,000)	(765,966,000)
Foreign currency transaction gain		$ 1,400,000	$ 300,000	$ 1,200,000
Cumulative Effect, Period of Adoption, Adjustment \| Accounting Standards Update 2020-06
New Accounting Pronouncements or Change in Accounting Principle [Line Items]
Convertible notes payable					$ 44,700,000
Additional paid-in capital					74,900,000
Reduction in accumulated deficit					$ 30,200,000

SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES - Vesting Awards (Details)	12 Months Ended
	Dec. 31, 2022
Stock Options
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Expiration Term	10 years
Stock Options \| Minimum
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Vesting Term	3 years
Stock Options \| Maximum
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Vesting Term	4 years
Restricted Stock Units \| Minimum
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Vesting Term	1 year
Restricted Stock Units \| Maximum
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Vesting Term	4 years

SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES - Inventory, Net of Reserve (Details) - USD ($) $ in Thousands	Dec. 31, 2022	Dec. 31, 2021
Accounting Policies [Abstract]
Raw materials	$ 3,627	$ 5,205
Finished goods	3,295	2,108
Total inventory	$ 6,922	$ 7,313

SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES - Property and Equipment (Details)	12 Months Ended
	Dec. 31, 2022
Computers and equipment
Property, Plant and Equipment [Line Items]
Use life (in years)	3 years
Furniture and fixtures
Property, Plant and Equipment [Line Items]
Use life (in years)	7 years

SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES - Foreign Currency Translation Adjustment included in Accumulated Other Comprehensive Loss (Details) - USD ($) $ in Thousands	12 Months Ended
	Dec. 31, 2022	Dec. 31, 2021	Dec. 31, 2020
Foreign Currency Translation Adjustments included in Accumulated Other Comprehensive Loss
Beginning balance	$ 302,112	$ 211,213	$ 221,196
Ending balance	42,851	302,112	211,213
Accumulated Foreign Currency Adjustment Attributable to Parent
Foreign Currency Translation Adjustments included in Accumulated Other Comprehensive Loss
Beginning balance	(92)	(1,607)	(155)
Foreign currency translation adjustments	507	1,515	(1,452)
Ending balance	$ 415	$ (92)	$ (1,607)

- Definition

Total of all stockholders' equity (deficit) items, net of receivables from officers, directors, owners, and affiliates of the entity which are attributable to the parent. The amount of the economic entity's stockholders' equity attributable to the parent excludes the amount of stockholders' equity which is allocable to that ownership interest in subsidiary equity which is not attributable to the parent (noncontrolling interest, minority interest). This excludes temporary equity and is sometimes called permanent equity.

+ References

Reference 1: http://www.xbrl.org/2009/role/commonPracticeRef
-Publisher FASB
-Name Accounting Standards Codification
-Topic 235
-SubTopic 10
-Section S99
-Paragraph 1
-Subparagraph (SX 210.4-08(g)(1)(ii))
-URI https://asc.fasb.org/extlink&oid=120395691&loc=d3e23780-122690

Reference 2: http://www.xbrl.org/2003/role/exampleRef
-Publisher FASB
-Name Accounting Standards Codification
-Topic 852
-SubTopic 10
-Section 55
-Paragraph 10
-URI https://asc.fasb.org/extlink&oid=84165509&loc=d3e56426-112766

Reference 3: http://fasb.org/us-gaap/role/ref/legacyRef
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-Name Accounting Standards Codification
-Topic 310
-SubTopic 10
-Section S99
-Paragraph 2
-Subparagraph (SAB Topic 4.E)
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Reference 4: http://fasb.org/us-gaap/role/ref/legacyRef
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-Name Accounting Standards Codification
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-Subparagraph (SX 210.5-02(31))
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Reference 5: http://fasb.org/us-gaap/role/ref/legacyRef
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Reference 6: http://fasb.org/us-gaap/role/ref/legacyRef
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-SubTopic 10
-Section S99
-Paragraph 1
-Subparagraph (SX 210.5-02(30))
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Reference 7: http://www.xbrl.org/2009/role/commonPracticeRef
-Publisher FASB
-Name Accounting Standards Codification
-Topic 825
-SubTopic 10
-Section 50
-Paragraph 28
-Subparagraph (f)
-URI https://asc.fasb.org/extlink&oid=123596393&loc=d3e14064-108612

Reference 8: http://www.xbrl.org/2009/role/commonPracticeRef
-Publisher FASB
-Name Accounting Standards Codification
-Topic 323
-SubTopic 10
-Section 50
-Paragraph 3
-Subparagraph (c)
-URI https://asc.fasb.org/extlink&oid=114001798&loc=d3e33918-111571

+ Details

REVENUE RECOGNITION - Narrative (Details) - USD ($) $ in Millions	12 Months Ended
	Dec. 31, 2022	Dec. 31, 2021	Dec. 31, 2020
Disaggregation of Revenue [Line Items]
Revenue, performance obligation, description of timing	The Company receives payments from its product sales, primarily through third party payers, based on terms that generally are within 30 days of delivery of product to the patient.
Revenue increase in performance obligation satisfied in previous period	$ 0.8	$ 0.4	$ 0.4
Geographic Concentration Risk \| Revenue Benchmark \| United States
Disaggregation of Revenue [Line Items]
Concentration risk, percentage	97.00%
Geographic Concentration Risk \| Revenue Benchmark \| Canada
Disaggregation of Revenue [Line Items]
Concentration risk, percentage	2.00%
Geographic Concentration Risk \| Revenue Benchmark \| Rest of World
Disaggregation of Revenue [Line Items]
Concentration risk, percentage	1.00%

REVENUE RECOGNITION - Net Product Revenue (Details) - USD ($) $ in Thousands	12 Months Ended
	Dec. 31, 2022	Dec. 31, 2021	Dec. 31, 2020
Disaggregation of Revenue [Line Items]
Revenue	$ 212,018	$ 227,490	$ 198,321
Bile acid products
Disaggregation of Revenue [Line Items]
Revenue	102,558	95,654	89,438
Tiopronin products
Disaggregation of Revenue [Line Items]
Revenue	97,970	115,122	108,883
Net product sales
Disaggregation of Revenue [Line Items]
Revenue	$ 200,528	$ 210,776	$ 198,321

- Definition

Amount, excluding tax collected from customer, of revenue from satisfaction of performance obligation by transferring promised good or service to customer. Tax collected from customer is tax assessed by governmental authority that is both imposed on and concurrent with specific revenue-producing transaction, including, but not limited to, sales, use, value added and excise.

+ References

Reference 1: http://www.xbrl.org/2003/role/disclosureRef
-Publisher FASB
-Name Accounting Standards Codification
-Topic 280
-SubTopic 10
-Section 50
-Paragraph 30
-Subparagraph (a)
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Reference 2: http://www.xbrl.org/2003/role/disclosureRef
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-Name Accounting Standards Codification
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Reference 3: http://www.xbrl.org/2003/role/disclosureRef
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-Name Accounting Standards Codification
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Reference 4: http://www.xbrl.org/2003/role/disclosureRef
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-Name Accounting Standards Codification
-Topic 280
-SubTopic 10
-Section 50
-Paragraph 32
-Subparagraph (b)
-URI https://asc.fasb.org/extlink&oid=126901519&loc=d3e8933-108599

Reference 5: http://www.xbrl.org/2003/role/disclosureRef
-Publisher FASB
-Name Accounting Standards Codification
-Topic 280
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-Subparagraph (a)
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Reference 6: http://www.xbrl.org/2003/role/disclosureRef
-Publisher FASB
-Name Accounting Standards Codification
-Topic 606
-SubTopic 10
-Section 50
-Paragraph 4
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-URI https://asc.fasb.org/extlink&oid=126920106&loc=SL49130543-203045

Reference 7: http://www.xbrl.org/2003/role/disclosureRef
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-Name Accounting Standards Codification
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-Paragraph 22
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Reference 8: http://www.xbrl.org/2003/role/disclosureRef
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Reference 9: http://www.xbrl.org/2003/role/disclosureRef
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-Name Accounting Standards Codification
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-URI https://asc.fasb.org/extlink&oid=126901519&loc=d3e9054-108599

Reference 10: http://www.xbrl.org/2003/role/disclosureRef
-Publisher FASB
-Name Accounting Standards Codification
-Topic 606
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-URI https://asc.fasb.org/extlink&oid=126920106&loc=SL49130545-203045

Reference 11: http://www.xbrl.org/2003/role/disclosureRef
-Publisher FASB
-Name Accounting Standards Codification
-Topic 924
-SubTopic 10
-Section S99
-Paragraph 1
-Subparagraph (SAB Topic 11.L)
-URI https://asc.fasb.org/extlink&oid=6472922&loc=d3e499488-122856

+ Details

COLLABORATION AND LICENSE AGREEMENTS - Narrative (Details) $ in Thousands		1 Months Ended	4 Months Ended	12 Months Ended
	Sep. 15, 2021 USD ($) performance_obligation	Feb. 28, 2021	Dec. 31, 2021 USD ($)	Dec. 31, 2022 USD ($)	Dec. 31, 2021 USD ($)	Dec. 31, 2020 USD ($)
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items]
Revenue				$ 212,018	$ 227,490	$ 198,321
Deferred revenue			$ 36,647	22,907	36,647	0
Deferred revenue, current portion			16,268	11,976	16,268
License and collaboration revenue
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items]
Revenue				11,490	16,714	$ 0
Vifor Pharma \| Collaborative Arrangement
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items]
Upfront payment	$ 55,000
Maximum milestone payments	$ 845,000
Percentage of royalty on net sales receives	40.00%
Force majeure event period threshold	6 months
Revenue	$ 55,000
Number of performance obligations \| performance_obligation	2
Deferred revenue	$ 43,000			22,900
Deferred revenue, current portion				12,000
Vifor Pharma \| Collaborative Arrangement \| Sales-based Milestone Payments
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items]
Maximum milestone payments	655,000
Vifor Pharma \| Collaborative Arrangement \| Regulatory and Market Access Milestone
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items]
Maximum milestone payments	$ 135,000
Vifor Pharma \| Collaborative Arrangement \| License and collaboration revenue
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items]
Revenue			$ 12,000
Revenue recognized during the period				12,000
Vifor Pharma \| Collaborative Arrangement \| Clinical Development Activity
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items]
Revenue recognized during the period				4,700
Albireo Pharma, Inc.
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items]
Co-promotion agreement, term		2 years
Co-promotion agreement, period after which the agreement can be terminated		1 year
Co-promotion agreement, amount recognized				$ 1,700	$ 1,800

COLLABORATION AND LICENSE AGREEMENTS - Changes in Deferred Revenue (Details) - USD ($) $ in Thousands	12 Months Ended
	Dec. 31, 2022	Dec. 31, 2021
Contract with Customer, Liability [Roll Forward]
Balance at January 1,	$ 36,647	$ 0
New consideration resulting in deferred revenue	0	55,000
Foreign currency impact	(2,250)	(1,639)
Balance at December 31,	22,907	36,647
License revenue
Contract with Customer, Liability [Roll Forward]
Revenue recognized during the period	0	(12,000)
Collaboration revenue
Contract with Customer, Liability [Roll Forward]
Revenue recognized during the period	$ 11,490	$ 4,714

ACQUISITIONS AND VARIABLE INTEREST ENTITIES - Acquisition of Orphan Technologies Limited (Details) - USD ($) $ in Thousands	1 Months Ended	12 Months Ended
	Nov. 30, 2020	Dec. 31, 2022	Dec. 31, 2021	Dec. 31, 2020
Business Acquisition [Line Items]
Cash paid for acquisition		$ 0	$ 0	$ 95,279
Contingent consideration, liability		$ 71,200	$ 67,100
Orphan Technologies Limited
Business Acquisition [Line Items]
Cash paid for acquisition	$ 90,000
Closing price adjustments	1,200
Liabilities assumed in acquisition	1,800
Acquisition transaction costs	4,200			$ 97,100
Contingent consideration, liability	$ 427,000

ACQUISITIONS AND VARIABLE INTEREST ENTITIES - Stock Purchase and Collaboration Agreement with PharmaKrysto (Details) - USD ($) $ in Thousands	Mar. 08, 2022	Dec. 31, 2022	Dec. 31, 2021
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items]
Cash and cash equivalents		$ 61,688	$ 165,753
PharmaKrysto, LTD \| Collaborative Arrangement
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items]
Collaboration agreement payment	$ 400
Research and development expenses, estimated	5,000
Collaborative arrangement, option to purchase additional shares of VIE, amount	1,000
Collaborative arrangement, option to purchase remaining shares of VIE, amount	5,000
Milestone payments contingently due	$ 16,000
Royalty payments, percentage (less than)	4.00%
Agreement termination notice period		60 days
Percentage of research and development to be funded by the company	100.00%
PharmaKrysto, LTD
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items]
Payment to purchase interest in VIE	$ 600
Percentage ownership purchased	5.00%
PharmaKrysto, LTD \| Variable Interest Entity, Not Primary Beneficiary
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items]
Cash and cash equivalents		$ 300
Accrued liabilities		$ 300
PharmaKrysto, LTD \| Collaborative Arrangement
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items]
Additional ownership interest to be purchased upon achievement of certain milestones	5.00%

MARKETABLE DEBT SECURITIES - Marketable Securities (Details) - USD ($) $ in Thousands	Dec. 31, 2022	Dec. 31, 2021
Debt Securities, Available-for-sale [Line Items]
Total marketable debt securities	$ 388,557	$ 387,129
Commercial paper
Debt Securities, Available-for-sale [Line Items]
Total marketable debt securities	123,647	127,379
Corporate debt securities
Debt Securities, Available-for-sale [Line Items]
Total marketable debt securities	224,055	233,319
Securities of government-sponsored entities
Debt Securities, Available-for-sale [Line Items]
Total marketable debt securities	$ 40,855	$ 26,431

MARKETABLE DEBT SECURITIES - Available for Sale Securities (Details) - USD ($) $ in Thousands	Dec. 31, 2022	Dec. 31, 2021
Debt Securities, Available-for-sale [Line Items]
Marketable debt securities, available-for-sale, amortized cost basis, current	$ 287,615	$ 262,345
Marketable debt securities available for sale, unrealized gain, current	2	0
Marketable debt securities available for sale unrealized loss, current	(2,091)	(159)
Marketable debt securities, available-for-sale, current	285,526	262,186
Marketable debt securities, available-for-sale, amortized cost basis, noncurrent	103,897	125,254
Marketable debt securities, available for sale unrealized gain, noncurrent	39	0
Marketable debt securities available for sale unrealized loss, noncurrent	(905)	(311)
Marketable debt securities, available-for-sale, noncurrent	103,031	124,943
Marketable debt securities, available-for-sale, amortized cost	391,512	387,599
Marketable debt securities, available-for-sale, accumulated gross unrealized gain, before tax	41	0
Marketable debt securities, available-for-sale, accumulated gross unrealized loss, before tax	(2,996)	(470)
Marketable debt securities, available-for-sale	388,557	387,129
Commercial paper
Debt Securities, Available-for-sale [Line Items]
Marketable debt securities, available-for-sale, amortized cost basis, current	124,301	127,435
Marketable debt securities available for sale, unrealized gain, current	2	0
Marketable debt securities available for sale unrealized loss, current	(656)	(56)
Marketable debt securities, available-for-sale, current	123,647	127,379
Marketable debt securities, available-for-sale	123,647	127,379
Corporate debt securities
Debt Securities, Available-for-sale [Line Items]
Marketable debt securities, available-for-sale, amortized cost basis, current	155,841	113,001
Marketable debt securities available for sale, unrealized gain, current	0	0
Marketable debt securities available for sale unrealized loss, current	(1,355)	(98)
Marketable debt securities, available-for-sale, current	154,486	112,903
Marketable debt securities, available-for-sale, amortized cost basis, noncurrent	70,195	120,705
Marketable debt securities, available for sale unrealized gain, noncurrent	33	0
Marketable debt securities available for sale unrealized loss, noncurrent	(659)	(289)
Marketable debt securities, available-for-sale, noncurrent	69,569	120,416
Marketable debt securities, available-for-sale	224,055	233,319
Securities of government-sponsored entities
Debt Securities, Available-for-sale [Line Items]
Marketable debt securities, available-for-sale, amortized cost basis, current	7,473	21,909
Marketable debt securities available for sale, unrealized gain, current	0	0
Marketable debt securities available for sale unrealized loss, current	(80)	(5)
Marketable debt securities, available-for-sale, current	7,393	21,904
Marketable debt securities, available-for-sale, amortized cost basis, noncurrent	33,702	4,549
Marketable debt securities, available for sale unrealized gain, noncurrent	6	0
Marketable debt securities available for sale unrealized loss, noncurrent	(246)	(22)
Marketable debt securities, available-for-sale, noncurrent	33,462	4,527
Marketable debt securities, available-for-sale	$ 40,855	$ 26,431

MARKETABLE DEBT SECURITIES - Narrative (Details) - USD ($) $ in Thousands	12 Months Ended
	Dec. 31, 2022	Dec. 31, 2021	Dec. 31, 2020
Investments, Debt and Equity Securities [Abstract]
Debt securities, unrealized gains	$ 100	$ 100
Purchase of fixed assets	382,000	433,600	$ 273,500
Purchases of marketable debt securities	385,389	547,076	$ 214,964
Available-for-sale marketable debt securities in an unrealized loss position	363,700	381,200
Accrued interest receivable	$ 1,900	$ 900

MARKETABLE DEBT SECURITIES - Securities in an Unrealized Loss Position (Details) - USD ($) $ in Thousands	Dec. 31, 2022	Dec. 31, 2021
Debt Securities, Available-for-sale [Line Items]
Less than 12 months, fair value	$ 248,321	$ 380,690
Less than 12 months, unrealized losses	1,960	470
12 months or greater, fair value	112,420	0
12 months or greater, unrealized losses	1,036	0
Total, fair value	360,741	380,690
Total, unrealized losses	2,996	470
Commercial paper
Debt Securities, Available-for-sale [Line Items]
Less than 12 months, fair value	117,853	122,380
Less than 12 months, unrealized losses	656	56
12 months or greater, fair value	0	0
12 months or greater, unrealized losses	0	0
Total, fair value	117,853	122,380
Total, unrealized losses	656	56
Corporate debt securities
Debt Securities, Available-for-sale [Line Items]
Less than 12 months, fair value	99,066	231,879
Less than 12 months, unrealized losses	1,041	387
12 months or greater, fair value	107,964	0
12 months or greater, unrealized losses	973	0
Total, fair value	207,030	231,879
Total, unrealized losses	2,014	387
Securities of government-sponsored entities
Debt Securities, Available-for-sale [Line Items]
Less than 12 months, fair value	31,402	26,431
Less than 12 months, unrealized losses	263	27
12 months or greater, fair value	4,456	0
12 months or greater, unrealized losses	63	0
Total, fair value	35,858	26,431
Total, unrealized losses	$ 326	$ 27

CONVERTIBLE NOTES PAYABLE - Schedule of Carrying Amount of Debt (Details) - Senior Notes - USD ($) $ in Thousands	Dec. 31, 2022	Dec. 31, 2021
Debt Instrument [Line Items]
Unamortized debt discount	$ 0	$ (46,045)
Total convertible senior notes, net of unamortized debt discount and debt issuance costs	$ 375,545	226,581
Senior Notes Due 2029
Debt Instrument [Line Items]
Interest rate percentage	2.25%
Convertible senior notes	$ 316,250	0
Unamortized debt issuance costs	$ (8,750)	0
Senior Notes Due 2025
Debt Instrument [Line Items]
Interest rate percentage	2.50%
Convertible senior notes	$ 68,904	276,000
Unamortized debt issuance costs	$ (859)	$ (3,374)

- Definition

Amount, excluding unamortized premium (discount) and debt issuance cost, of long-term debt. Excludes lease obligation.

+ References + Details

CONVERTIBLE NOTES PAYABLE - Narrative (Details) $ / shares in Units, $ in Thousands			12 Months Ended
	Mar. 11, 2022 USD ($)	Sep. 10, 2018 USD ($)	Dec. 31, 2022 USD ($) day $ / shares	Dec. 31, 2021 USD ($)	Dec. 31, 2020 USD ($)
Debt Instrument [Line Items]
Debt instrument, repurchase amount Including accrued and unpaid interest	$ 213,800
Loss on extinguishment of debt			$ 7,578	$ 0	$ 0
Write off of deferred debt financing costs			3,400
Interest expense			$ 11,300	$ 20,100	$ 19,100
Senior Notes \| Senior Notes Due 2029
Debt Instrument [Line Items]
Long-term debt, excluding current maturities	316,300
Interest rate percentage			2.25%
Proceeds from issuance of debt	306,400
Debt issuance costs, net			$ 9,900
Accrued interest			$ 2,400
Debt instrument, convertible, threshold trading days \| day			20
Debt instrument, convertible, threshold consecutive trading days \| day			30
Debt instrument, convertible, threshold percentage of stock price trigger			130.00%
Debt instrument, convertible, conversion ratio			0.001374
Debt instrument, convertible, conversion price (in USD per share) \| $ / shares			$ 31.87
Notes payable			$ 100,000
Long-term debt, term			7 years
Debt instrument, interest rate, effective percentage			2.74%
Senior Notes \| Senior Notes Due 2029 \| Debt Conversion, Scenario One
Debt Instrument [Line Items]
Debt instrument, convertible, threshold trading days \| day			20
Debt instrument, convertible, threshold consecutive trading days \| day			30
Debt instrument, convertible, threshold percentage of stock price trigger			130.00%
Senior Notes \| Senior Notes Due 2029 \| Debt Conversion, Scenario Two
Debt Instrument [Line Items]
Debt instrument, convertible, threshold trading days \| day			5
Debt instrument, convertible, threshold consecutive trading days \| day			10
Debt instrument, convertible, threshold percentage of stock price trigger			98.00%
Senior Notes \| Senior Notes Due 2029, Issued Pursuant To Underwriters Option
Debt Instrument [Line Items]
Long-term debt, excluding current maturities	41,300
Senior Notes \| Senior Notes Due 2025
Debt Instrument [Line Items]
Long-term debt, excluding current maturities		$ 276,000
Interest rate percentage			2.50%
Proceeds from issuance of debt		267,200
Debt issuance costs, net		$ 8,800
Accrued interest			$ 500
Debt instrument, convertible, threshold trading days \| day			20
Debt instrument, convertible, threshold consecutive trading days \| day			30
Debt instrument, convertible, threshold percentage of stock price trigger			130.00%
Debt instrument, convertible, conversion ratio			0.0257739
Debt instrument, convertible, conversion price (in USD per share) \| $ / shares			$ 38.80
Long-term debt, term			7 years
Debt instrument, interest rate, effective percentage			2.98%
Debt instrument, repurchase amount	$ 207,100
Long-term debt, excluding current maturities, repaid if converted			$ 68,900
Senior Notes \| Senior Notes Due 2025 \| Debt Conversion, Scenario One
Debt Instrument [Line Items]
Debt instrument, convertible, threshold trading days \| day			20
Debt instrument, convertible, threshold consecutive trading days \| day			30
Debt instrument, convertible, threshold percentage of stock price trigger			130.00%
Senior Notes \| Senior Notes Due 2025 \| Debt Conversion, Scenario Two
Debt Instrument [Line Items]
Debt instrument, convertible, threshold trading days \| day			5
Debt instrument, convertible, threshold consecutive trading days \| day			10
Debt instrument, convertible, threshold percentage of stock price trigger			98.00%

CONVERTIBLE NOTES PAYABLE - Convertible Notes Payable (Details) - Senior Notes - Senior Notes Due 2025 - USD ($) $ in Thousands	12 Months Ended
	Dec. 31, 2022	Dec. 31, 2021	Dec. 31, 2020
Debt Instrument [Line Items]
Contractual interest expense	$ 8,433	$ 6,900	$ 6,900
Amortization of debt discount	0	10,339	9,578
Amortization of debt issuance costs	1,622	903	900
Total interest expense for the 2025 and 2029 Notes	$ 10,055	$ 18,142	$ 17,378

FAIR VALUE MEASUREMENTS - Narrative (Details) $ in Millions	12 Months Ended
FAIR VALUE MEASUREMENTS - Narrative (Details) $ in Millions	Dec. 31, 2022 USD ($) business	Dec. 31, 2021 USD ($)
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Number of businesses acquired \| business	2
Senior Notes Due 2025 \| Senior Notes
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Interest rate percentage	2.50%
Fair value of convertible debt	$ 62.9	$ 300.6
Senior Notes Due 2029 \| Senior Notes
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Interest rate percentage	2.25%
Fair value of convertible debt	$ 283.0

FAIR VALUE MEASUREMENTS - Assets and Liabilities Measured at Fair Value (Details) - USD ($) $ in Thousands	Dec. 31, 2022	Dec. 31, 2021	Dec. 31, 2020
Assets:
Cash and Cash Equivalents	$ 61,688	$ 165,753
Marketable debt securities, available-for-sale	388,557	387,129
Total	450,245	552,882
Liabilities:
Business combination-related contingent consideration	71,200	67,100
Total	71,200	67,100
Quoted prices in active markets (Level 1)
Assets:
Cash and Cash Equivalents	61,688	165,753
Marketable debt securities, available-for-sale	0	0
Total	61,688	165,753
Liabilities:
Business combination-related contingent consideration	0	0
Total	0	0
Significant other observable inputs (Level 2)
Assets:
Cash and Cash Equivalents	0	0
Marketable debt securities, available-for-sale	388,557	387,129
Total	388,557	387,129
Liabilities:
Business combination-related contingent consideration	0	0
Total	0	0
Significant unobservable inputs (Level 3)
Assets:
Cash and Cash Equivalents	0	0
Marketable debt securities, available-for-sale	0	0
Total	0	0
Liabilities:
Business combination-related contingent consideration	71,200	67,100	$ 65,100
Total	$ 71,200	$ 67,100

FAIR VALUE MEASUREMENTS - Discount Rates Used (Details)	Dec. 31, 2022	Dec. 31, 2021
Payment Discount
Fair Value Measurement Inputs and Valuation Techniques [Line Items]
Measurement input	0.0810	0.0648
Cholbam \| Revenue Discount
Fair Value Measurement Inputs and Valuation Techniques [Line Items]
Measurement input	0.0775	0.0625
Chenodal \| Revenue Discount
Fair Value Measurement Inputs and Valuation Techniques [Line Items]
Measurement input	0.0800	0.0725

FAIR VALUE MEASUREMENTS - Fair Value of Level 3 (Details) - USD ($) $ in Thousands	12 Months Ended
	Dec. 31, 2022	Dec. 31, 2021	Dec. 31, 2020
Fair Value, Liabilities Measured on Recurring Basis, Unobservable Input Reconciliation, Calculation [Roll Forward]
Beginning balance	$ 67,100
Changes in the fair value of contingent consideration	8,602	$ 14,375	$ 12,407
Ending balance	71,200	67,100
Significant unobservable inputs (Level 3)
Fair Value, Liabilities Measured on Recurring Basis, Unobservable Input Reconciliation, Calculation [Roll Forward]
Beginning balance	67,100	65,100
Changes in the fair value of contingent consideration	15,006	22,260
Contractual payments	(8,066)	(17,444)
Contractual payments included in accrued liabilities at December 31,	(2,840)	(2,700)
Foreign currency impact	0	(116)
Ending balance	$ 71,200	$ 67,100	$ 65,100

INTANGIBLE ASSETS - Narrative (Details) - USD ($) $ in Thousands	1 Months Ended		12 Months Ended
	Nov. 30, 2018	Nov. 30, 2017	Dec. 31, 2022	Dec. 31, 2021	Dec. 31, 2020	Dec. 31, 2014	Mar. 31, 2015
Finite-Lived Intangible Assets [Line Items]
Payments to date under terms of licensing agreement			$ 28,366	$ 19,050	$ 17,799
Finite-lived intangible asset			302,935	283,557
Amortization expense			30,964	24,946	$ 22,437
Goodwill			900	$ 900
Ligand License Agreement
Finite-Lived Intangible Assets [Line Items]
License agreement, milestone payments			114,100
License agreement, milestone payment, milestone one			23,000
Payments to date under terms of licensing agreement			$ 18,400
Ligand License Agreement \| Minimum
Finite-Lived Intangible Assets [Line Items]
Annual royalty percentage			15.00%
Ligand License Agreement \| Maximum
Finite-Lived Intangible Assets [Line Items]
Annual royalty percentage			17.00%
Product rights
Finite-Lived Intangible Assets [Line Items]
Assets useful life (in years)			16 years	16 years
Finite-lived intangible asset			$ 67,849	$ 67,849
Product rights \| Manchester Pharmaceuticals LLC
Finite-Lived Intangible Assets [Line Items]
Intangible assets with definite lives						$ 67,800
Assets useful life (in years)						16 years
Trade Name \| Manchester Pharmaceuticals LLC
Finite-Lived Intangible Assets [Line Items]
Intangible assets with definite lives						$ 200
Assets useful life (in years)						1 year
Customer relationships
Finite-Lived Intangible Assets [Line Items]
Assets useful life (in years)			10 years	10 years
Finite-lived intangible asset			$ 403	$ 403
Customer relationships \| Manchester Pharmaceuticals LLC
Finite-Lived Intangible Assets [Line Items]
Intangible assets with definite lives						$ 400
Assets useful life (in years)						10 years
Thiola License Agreement
Finite-Lived Intangible Assets [Line Items]
Assets useful life (in years)						10 years
Automatic renewal periods						1 year
Remaining weighed average period of amortization (in years)			6 years 4 months 24 days
Thiola License Agreement \| Mission Pharmacal Company
Finite-Lived Intangible Assets [Line Items]
Annual royalty percentage			20.00%
Mission an up-front license fee			$ 3,000
Guaranteed minimum royalties			2,000
Present value of guaranteed minimum royalties payable			11,100	12,300
Thiola License Agreement \| Mission Pharmacal Company \| Other Current Liabilities
Finite-Lived Intangible Assets [Line Items]
Guaranteed minimum royalties			2,100	2,100
Thiola License Agreement \| Mission Pharmacal Company \| Other Noncurrent Liabilities
Finite-Lived Intangible Assets [Line Items]
Guaranteed minimum royalties			$ 9,000	$ 10,200
Thiola License Agreement \| Mission Pharmacal Company \| Minimum
Finite-Lived Intangible Assets [Line Items]
Discount rate			7.00%
Thiola License Agreement \| Mission Pharmacal Company \| Maximum
Finite-Lived Intangible Assets [Line Items]
Discount rate			11.00%
Thiola
Finite-Lived Intangible Assets [Line Items]
Assets useful life (in years)			15 years	15 years
Licensing agreement, extension term		5 years
Finite-lived intangible asset		$ 5,900	$ 139,383	$ 123,316
Payments for the option to acquire business	$ 300
Payment of guaranteed minimum royalty	$ 100
Minimum royalty, percentage	20.00%
Amortization expense	$ 1,000
Guaranteed minimum liability	$ 700
Increase in intangible assets			$ 16,100
United States \| Asklepion Pharmaceuticals LLC
Finite-Lived Intangible Assets [Line Items]
Capitalized economic interest							$ 75,900
International \| Asklepion Pharmaceuticals LLC
Finite-Lived Intangible Assets [Line Items]
Capitalized economic interest							$ 7,300

INTANGIBLE ASSETS - Amortization of Intangible Assets (Details) - USD ($) $ in Thousands	12 Months Ended
	Dec. 31, 2022	Dec. 31, 2021	Nov. 30, 2017
Finite-Lived Intangible Assets [Line Items]
Gross Carrying Amount	$ 302,935	$ 283,557
Accumulated Amortization	(158,833)	(136,058)
Intangible assets finite-lived, net	$ 144,102	$ 147,499
Thiola license
Finite-Lived Intangible Assets [Line Items]
Useful Life	15 years	15 years
Gross Carrying Amount	$ 139,383	$ 123,316	$ 5,900
Accumulated Amortization	(50,324)	(37,992)
Intangible assets finite-lived, net	$ 89,059	$ 85,324
Chenodal product rights
Finite-Lived Intangible Assets [Line Items]
Useful Life	16 years	16 years
Gross Carrying Amount	$ 67,849	$ 67,849
Accumulated Amortization	(37,175)	(32,938)
Intangible assets finite-lived, net	$ 30,674	$ 34,911
Economic interest - Cholbam revenue
Finite-Lived Intangible Assets [Line Items]
Useful Life	10 years	10 years
Gross Carrying Amount	$ 75,900	$ 75,900
Accumulated Amortization	(58,842)	(51,258)
Intangible assets finite-lived, net	$ 17,058	$ 24,642
Ligand license
Finite-Lived Intangible Assets [Line Items]
Useful Life	11 years	11 years
Gross Carrying Amount	$ 19,400	$ 7,900
Accumulated Amortization	(12,139)	(5,875)
Intangible assets finite-lived, net	$ 7,261	$ 2,025
Economic interest - International revenue Cholbam
Finite-Lived Intangible Assets [Line Items]
Useful Life		10 years
Gross Carrying Amount		$ 7,982
Accumulated Amortization		(7,483)
Intangible assets finite-lived, net		$ 499
Manchester customer relationships
Finite-Lived Intangible Assets [Line Items]
Useful Life	10 years	10 years
Gross Carrying Amount	$ 403	$ 403
Accumulated Amortization	(353)	(313)
Intangible assets finite-lived, net	$ 50	$ 90
Internal use software
Finite-Lived Intangible Assets [Line Items]
Useful Life		5 years
Gross Carrying Amount		$ 207
Accumulated Amortization		(199)
Intangible assets finite-lived, net		$ 8

INTANGIBLE ASSETS - Amortization Expense (Details) - USD ($) $ in Thousands	12 Months Ended
	Dec. 31, 2022	Dec. 31, 2021	Dec. 31, 2020
Finite-Lived Intangible Assets [Line Items]
Amortization expense	$ 30,964	$ 24,946	$ 22,437
Research and development
Finite-Lived Intangible Assets [Line Items]
Amortization expense	6,264	1,158	1,162
Selling, general and administrative
Finite-Lived Intangible Assets [Line Items]
Amortization expense	$ 24,700	$ 23,788	$ 21,275

INTANGIBLE ASSETS - Amortization Expense Next Five Years (Details) - USD ($) $ in Thousands	Dec. 31, 2022	Dec. 31, 2021
Goodwill and Intangible Assets Disclosure [Abstract]
2023	$ 33,015
2024	25,793
2025	19,999
2026	18,129
2027	18,129
Thereafter	29,037
Intangible assets finite-lived, net	$ 144,102	$ 147,499

ACCRUED EXPENSES (Details) - USD ($) $ in Thousands	Dec. 31, 2022	Dec. 31, 2021
Payables and Accruals [Abstract]
Compensation related costs	$ 35,267	$ 25,998
Research and development	26,070	26,841
Sales discounts, rebates, and allowances	13,486	7,493
Selling, general and administrative	8,791	3,144
Accrued royalties	7,755	8,402
Miscellaneous accrued	4,373	3,302
Total accrued expenses	$ 95,742	$ 75,180

COMMITMENTS AND CONTINGENCIES (Details) - USD ($) $ in Thousands	1 Months Ended	12 Months Ended
	Oct. 31, 2021	Dec. 31, 2022	Dec. 31, 2021	Dec. 31, 2020
Other Commitments [Line Items]
Commitment to purchase		$ 36,300
Revenue		$ 212,018	$ 227,490	$ 198,321
France \| Kolbam
Other Commitments [Line Items]
Revenue	$ 8,000

- Definition

+ References

+ Details

STOCKHOLDERS’ EQUITY - Common Stock and Preferred Stock (Details)	12 Months Ended
	Dec. 31, 2022 vote $ / shares shares	Dec. 31, 2021 $ / shares shares	May 18, 2021 shares	May 17, 2021 shares
Class of Stock [Line Items]
Common stock, shares authorized (in shares)	200,000,000	200,000,000	200,000,000	100,000,000
Common stock, par value (in dollars per share) \| $ / shares	$ 0.0001	$ 0.0001
Number of votes per common share owned \| vote	1
Preferred stock, shares authorized (in shares)	20,000,000	20,000,000
Preferred stock, par value (in dollars per share) \| $ / shares	$ 0.0001	$ 0.0001
Preferred stock, shares issued (in shares)	0	0
Series A Preferred Stock
Class of Stock [Line Items]
Preferred stock, shares authorized (in shares)	1,000

STOCKHOLDERS’ EQUITY - 2015 Equity Incentive Plan (Details) - shares	12 Months Ended
	Dec. 31, 2022	Dec. 31, 2021	May 18, 2021	May 17, 2021	May 17, 2017	May 18, 2016	Jun. 08, 2015
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Common stock, shares authorized (in shares)	200,000,000	200,000,000	200,000,000	100,000,000
2015 Equity Incentive Plan
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Common stock, shares authorized (in shares)							1,400,000
Shares remaining available for issuance under the plan (in shares)							600,000
Expiration Term	10 years
2015 Equity Incentive Plan \| Minimum
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Vesting Term	3 years
2015 Equity Incentive Plan \| Maximum
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Vesting Term	4 years
2015 Equity Incentive Plan Amended
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Common stock, shares authorized (in shares)					1,800,000	1,600,000
Shares remaining available for issuance under the plan (in shares)						700,000

STOCKHOLDERS’ EQUITY - 2018 Equity Incentive Plan (Details) - shares	12 Months Ended
	Dec. 31, 2022	May 11, 2022	Dec. 31, 2021	May 18, 2021	May 17, 2021	May 14, 2021	May 15, 2020	May 09, 2019	May 09, 2018
Class of Stock [Line Items]
Common stock, shares authorized (in shares)	200,000,000		200,000,000	200,000,000	100,000,000
2018 Equity Incentive Plan
Class of Stock [Line Items]
Common stock, shares authorized (in shares)									1,800,000
Shares remaining available for issuance under the plan (in shares)									1,600,000
Expiration Term	10 years
Vesting Term	4 years
Increase in authorized shares issuable (in shares)		2,000,000				3,200,000	2,400,000	2,000,000

STOCKHOLDERS’ EQUITY - 2017 Employee Stock Purchase Plan (Details) - 2017 ESPP - USD ($)	12 Months Ended
	Dec. 31, 2022	Jan. 01, 2018
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Shares remaining available for issuance under the plan (in shares)	1,880,000	380,000
Potential increase in shares available for issuance, as a percent of total outstanding common stock	1.00%
Maximum number of additional shares authorized for issuance (in shares)	300,000
Purchase price of common stock, percent of fair market value	85.00%
Stock purchase offering period	6 months
Employee stock purchase plan, maximum compensation	15.00%
Employee stock purchase plan annual limit	$ 25,000
Shares reserved for future issuance (in shares)	1,127,178

STOCKHOLDERS’ EQUITY - Black Scholes Assumptions (Details) - Stock Options - shares	12 Months Ended
	Dec. 31, 2022	Dec. 31, 2021	Dec. 31, 2020
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Granted (in shares)	1,746,270
Risk free rate	1.70%	0.60%	1.50%
Expected volatility	50.00%	59.00%	64.00%
Expected life (in years)	6 years 4 months 24 days	6 years 4 months 24 days	6 years 3 months 18 days
Expected dividend yield	0.00%	0.00%	0.00%

STOCKHOLDERS’ EQUITY - Stock Option Activity (Details) - USD ($) $ / shares in Units, $ in Thousands	12 Months Ended
	Dec. 31, 2022	Dec. 31, 2021	Dec. 31, 2020
Share-based Compensation Arrangement by Share-based Payment Award, Options, Additional Disclosures [Abstract]
Exercised, aggregate intrinsic value	$ 5,079
Shares underlying options (in shares)	6,999,669	6,011,349	5,488,207
Exercise Price (in USD per share)	$ 20.22	$ 19.34	$ 19.37
Remaining contractual term, exercisable (in years)	4 years 7 months 13 days	5 years 1 month 28 days	5 years 4 months 9 days
Exercisable, aggregate intrinsic value	$ 21,806	$ 71,063	$ 46,626
Closing stock price of stock options outstanding and exercisable (in USD per share)	$ 21.03	$ 31.04	$ 27.25
Stock Options
Shares
Outstanding, beginning balance (in shares)	8,886,284
Granted (in shares)	1,746,270
Forfeited and expired (in shares)	(325,286)
Exercised (in shares)	(374,846)
Outstanding, ending balance (in shares)	9,932,422	8,886,284
Vested and expected to vest (in shares)	9,932,422
Weighted Average Exercise Price
Outstanding, beginning balance (in USD per share)	$ 20.26
Granted (in USD per share)	26.76
Forfeited and expired (in dollars per share)	24.63
Exercised (in USD per share)	12.23
Outstanding, ending balance (in USD per share)	21.56	$ 20.26
Vested and expected to vest (in USD per share)	$ 21.56
Share-based Compensation Arrangement by Share-based Payment Award, Options, Additional Disclosures [Abstract]
Remaining contractual term, outstanding (in years)	5 years 9 months 14 days	6 years 3 months 7 days
Vested and expected to vest (in years)	5 years 9 months 14 days
Outstanding, aggregate intrinsic value	$ 24,658	$ 96,577
Exercised, aggregate intrinsic value	5,100	$ 7,500	$ 1,800
Vested and expected to vest value	$ 24,658
Weighted average grant date fair value of options (in USD per share)	$ 13.45	$ 14.00	$ 9.54
Compensation expense not yet recognized, stock options	$ 31,900
Weighted average period for unrecognized costs (in years)	2 years 6 months
Expiration Term	10 years
Stock Options \| Chief Executive Officer
Share-based Compensation Arrangement by Share-based Payment Award, Options, Additional Disclosures [Abstract]
Expiration Term	10 years
Modification expense	$ 3,200

STOCKHOLDERS’ EQUITY - Restricted Stock (Details) - Restricted Stock Units - USD ($) $ / shares in Units, $ in Millions	12 Months Ended
	Dec. 31, 2022	Dec. 31, 2021	Dec. 31, 2020
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Unrecognized compensation cost related to restricted shares granted	$ 44.3
Weighted average period for unrecognized costs (in years)	2 years 8 months 12 days
Number of shares
Unvested beginning balance (in shares)	1,473,949
Granted (in shares)	1,533,518
Vested (in shares)	(543,252)
Forfeited/cancelled (in shares)	(120,506)
Unvested ending balance (in shares)	2,343,709	1,473,949
Weighted Average Grant Date Fair Value
Unvested beginning balance (in USD per share)	$ 21.88
Granted (in USD per share)	26.07
Vested (in USD per share)	21.21	$ 18.08	$ 18.43
Forfeited/cancelled (in USD per share)	24.35
Unvested ending balance (in USD per share)	$ 24.65	$ 21.88
Fair value of restricted stock units vested	$ 11.5	$ 7.4	$ 5.3

STOCKHOLDERS’ EQUITY - Performance-based Stock Options (Details) - Performance Shares - USD ($) $ / shares in Units, $ in Millions	12 Months Ended
	Dec. 31, 2022	Dec. 31, 2021	Dec. 31, 2020
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Unrecognized compensation cost related to restricted shares granted	$ 2.0
Weighted average period for unrecognized costs (in years)	1 year 1 month 6 days
Number of shares
Unvested beginning balance (in shares)	52,500
Granted (in shares)	127,048
Vested (in shares)	(17,500)
Forfeited/cancelled (in shares)	(5,000)
Unvested ending balance (in shares)	157,048	52,500
Weighted Average Grant Date Fair Value
Unvested beginning balance (in USD per share)	$ 18.73
Granted (in USD per share)	27.54
Vested (in USD per share)	25.26	$ 15.46	$ 21.58
Forfeited/cancelled (in USD per share)	15.46
Unvested ending balance (in USD per share)	$ 25.24	$ 18.73
Fair value of performance-based stock units vested	$ 0.4	$ 1.8	$ 2.3

STOCKHOLDERS’ EQUITY - Share Based Compensation (Details) - USD ($) $ in Thousands	12 Months Ended
	Dec. 31, 2022	Dec. 31, 2021	Dec. 31, 2020
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Total	$ 39,177	$ 30,766	$ 23,614
Selling, general and administrative
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Total	25,319	18,134	14,247
Research and development expenses
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Total	$ 13,858	$ 12,632	$ 9,367

NET LOSS PER COMMON SHARE - Basic and Diluted (Details) - USD ($) $ / shares in Units, $ in Thousands	12 Months Ended
	Dec. 31, 2022	Dec. 31, 2021	Dec. 31, 2020
Earnings Per Share [Abstract]
Basic shares outstanding (in shares)	63,758,515	59,832,287	47,539,631
Diluted shares outstanding (in shares)	63,758,515	59,832,287	47,539,631
Net loss (basic)	$ (278,482)	$ (180,091)	$ (169,431)
Net loss (diluted)	$ (278,482)	$ (180,091)	$ (169,431)
Basic (in dollars per share)	$ (4.37)	$ (3.01)	$ (3.56)
Diluted (in dollars per share)	$ (4.37)	$ (3.01)	$ (3.56)

- Definition

The amount of net income (loss) for the period per each share of common stock or unit outstanding during the reporting period.

+ References

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- Definition

The amount of net income (loss) for the period available to each share of common stock or common unit outstanding during the reporting period and to each share or unit that would have been outstanding assuming the issuance of common shares or units for all dilutive potential common shares or units outstanding during the reporting period.

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Amount, after deduction of tax, noncontrolling interests, dividends on preferred stock and participating securities; of income (loss) available to common shareholders.

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Amount, after deduction of tax, noncontrolling interests, dividends on preferred stock and participating securities, and addition from assumption of issuance of common shares for dilutive potential common shares; of income (loss) available to common shareholders.

+ References + Details

NET LOSS PER COMMON SHARE - Antidilutive Securities (Details) - shares	12 Months Ended
	Dec. 31, 2022	Dec. 31, 2021	Dec. 31, 2020
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items]
Anti-dilutive shares excluded from the calculation (in shares)	23,085,349	17,897,060	16,830,808
Convertible debt
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items]
Anti-dilutive shares excluded from the calculation (in shares)	10,869,000	7,113,402	7,113,402
Options
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items]
Anti-dilutive shares excluded from the calculation (in shares)	10,041,249	9,214,188	8,349,310
Restricted stock
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items]
Anti-dilutive shares excluded from the calculation (in shares)	2,175,100	1,569,470	1,368,096

INCOME TAXES - Components of Net Income Before Taxes (Details) - USD ($) $ in Thousands	12 Months Ended
	Dec. 31, 2022	Dec. 31, 2021	Dec. 31, 2020
Operating Loss Carryforwards [Line Items]
Loss before provision for income taxes	$ (278,169)	$ (179,682)	$ (188,790)
United States
Operating Loss Carryforwards [Line Items]
Loss before provision for income taxes	(215,901)	(124,132)	(65,881)
Foreign
Operating Loss Carryforwards [Line Items]
Loss before provision for income taxes	$ (62,268)	$ (55,550)	$ (122,909)

- Definition

Amount of income (loss) from continuing operations, including income (loss) from equity method investments, before deduction of income tax expense (benefit), and income (loss) attributable to noncontrolling interest.

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INCOME TAXES - Components of Income Tax Expense (Benefit) (Details) - USD ($) $ in Thousands	12 Months Ended
	Dec. 31, 2022	Dec. 31, 2021	Dec. 31, 2020
Current
Federal	$ 0	$ 0	$ (19,436)
State	314	412	74
Foreign	(1)	(3)	3
Total	313	409	(19,359)
Deferred
Federal	0	0	0
State	0	0	0
Total	0	0	0
Total tax provision (benefit)	$ 313	$ 409	$ (19,359)

INCOME TAXES - Reconciliation of Federal Income Tax Rate (Details)	12 Months Ended
	Dec. 31, 2022	Dec. 31, 2021	Dec. 31, 2020
Income Tax Disclosure [Abstract]
Statutory rate - federal	(21.00%)	(21.00%)	(21.00%)
State taxes, net of federal benefit	(3.46%)	(2.71%)	(1.57%)
Foreign rate differential	0.96%	3.28%	6.65%
IPR&D	0.00%	0.00%	6.50%
Federal IRS Audit settlement	0.00%	0.00%	1.62%
Nondeductible executive compensation	0.49%	0.48%	0.00%
Excess tax benefits associated with share-based awards	(0.20%)	0.19%	0.00%
Other permanent differences	0.20%	0.15%	0.09%
Tax credits	(6.65%)	(10.87%)	(4.49%)
Return to provision adjustments and other true-ups	(0.21%)	2.42%	4.88%
CARES Act-Net operating loss carryback	0.00%	0.00%	(9.58%)
Adoption of ASU 2020-06	(0.0421)	0	0
Other	0.00%	0.20%	(0.10%)
Change in valuation allowance	34.19%	28.09%	6.75%
Income tax provision (benefit)	0.11%	0.23%	(10.25%)

INCOME TAXES - Deferred Tax Assets and Liabilities (Details) - USD ($) $ in Thousands	Dec. 31, 2022	Dec. 31, 2021
Deferred Tax Assets:
Net operating loss	$ 49,811	$ 38,618
Research and development and other tax credits	91,052	69,213
Capitalized research and development	36,442	0
Contingent consideration	18,124	16,726
Other accrued expenses	13,247	8,678
Stock based compensation	22,936	18,003
Charitable contributions	4,781	3,427
Intangible assets greater than book	51,391	48,801
Interest expense limitation	4,346	2,802
Operating lease liabilities	8,018	8,825
Tax basis depreciation greater than book depreciation	105	13
Difference in basis of loan costs	15	0
Deferred tax assets	300,268	215,106
Deferred Tax Liabilities:
Operating lease right of use assets	(6,658)	(5,782)
Convertible debt	0	(11,020)
Prepaid assets	(206)	0
Deferred tax liabilities	(6,864)	(16,802)
Net deferred tax assets before valuation allowance	293,404	198,304
Valuation allowance	(293,404)	(198,304)
Total deferred tax assets	$ 0	$ 0

INCOME TAXES - Narrative (Details) - USD ($) $ in Thousands	12 Months Ended
INCOME TAXES - Narrative (Details) - USD ($) $ in Thousands	Dec. 31, 2022	Dec. 31, 2021	Dec. 31, 2020
Operating Loss Carryforwards [Line Items]
Valuation allowance	$ 293,404	$ 198,304
Increase (decrease) in valuation allowance	95,100	50,500
Unrecognized tax benefits	11,490	7,825	$ 5,193
Unrecognized tax benefits that would impact effective tax rate	11,500
Income tax examination, penalties and interest expense	100	$ 100
Unrecognized Tax Benefits
Operating Loss Carryforwards [Line Items]
Increase (decrease) in valuation allowance	(11,500)
U.S. federal
Operating Loss Carryforwards [Line Items]
Available unused NOL carryforwards	90,200
State and Local Jurisdiction
Operating Loss Carryforwards [Line Items]
Available unused NOL carryforwards	174,100
Operating loss carryforwards, indefinite carryforward period	26,200
Foreign \| Irish
Operating Loss Carryforwards [Line Items]
Available unused NOL carryforwards	15,100
Foreign \| Swiss
Operating Loss Carryforwards [Line Items]
Available unused NOL carryforwards	148,200
Tax credit carryforward	526,200
Interest Expense Limitation Carryforward
Operating Loss Carryforwards [Line Items]
Tax credit carryforward	20,100
Federal Orphan Drug Tax Credit
Operating Loss Carryforwards [Line Items]
Tax credit carryforward	82,000
Research Tax Credit Carryforward
Operating Loss Carryforwards [Line Items]
Unrecognized tax benefits	9,900
Research Tax Credit Carryforward \| U.S. federal
Operating Loss Carryforwards [Line Items]
Tax credit carryforward	5,400
Research Tax Credit Carryforward \| State and Local Jurisdiction
Operating Loss Carryforwards [Line Items]
Tax credit carryforward	15,200
Tax credit carryforward, finite carryforward period, amount	1,400
Tax credit carryforward, indefinite carryforward period, amount	13,800
Tax Competes Tax Credit Carryforward
Operating Loss Carryforwards [Line Items]
Tax credit carryforward	2,000
Deferred Tax Assets, Tax Credit Carryforwards
Operating Loss Carryforwards [Line Items]
Unrecognized tax benefits	$ 1,600

INCOME TAXES - Unrecognized Tax Benefits (Details) - USD ($) $ in Thousands	12 Months Ended
	Dec. 31, 2022	Dec. 31, 2021
Reconciliation of Unrecognized Tax Benefits, Excluding Amounts Pertaining to Examined Tax Returns [Roll Forward]
Beginning balance	$ 7,825	$ 5,193
Increase in current period positions	2,056	2,255
Decrease due to settlements with tax authorities	(310)	0
Increase in prior period positions	1,919	377
Ending balance	$ 11,490	$ 7,825

EQUITY OFFERINGS (Details) - USD ($) $ / shares in Units, $ in Millions	1 Months Ended	12 Months Ended
	Feb. 28, 2021	Dec. 31, 2022	Dec. 31, 2021	May 18, 2021	May 17, 2021	Feb. 29, 2020
Class of Stock [Line Items]
Common stock, shares authorized (in shares)		200,000,000	200,000,000	200,000,000	100,000,000
Underwritten Equity Offering
Class of Stock [Line Items]
Shares authorized in sale (in shares)	7,500,000
Sales price per share (in dollars per share)	$ 26.75
Proceeds from issuance of common stock	$ 189.3
At-The-Market Offering
Class of Stock [Line Items]
Shares authorized in sale (in shares)		701,600
Proceeds from issuance of common stock		$ 20.1
Aggregate offering amount authorized						$ 100.0
Remaining offering amount authorized		19.5
At-The-Market Offering Under Previous Registration Statement
Class of Stock [Line Items]
Amount sold to date		28.6
At-The-Market Offering Under Current Registration Statement
Class of Stock [Line Items]
Amount sold to date		$ 51.9

RETIREMENT PLAN (Details) - USD ($) $ in Millions	12 Months Ended
RETIREMENT PLAN (Details) - USD ($) $ in Millions	Dec. 31, 2022	Dec. 31, 2021	Dec. 31, 2020
Defined Benefit Plan, Plan Assets, Category [Line Items]
Defined contribution plan, employer contributions	$ 2.1	$ 1.3	$ 1.2
Switzerland Defined Benefit Plan
Defined Benefit Plan, Plan Assets, Category [Line Items]
Benefit obligation	1.8
Plan assets	$ 1.4

- Definition

Amount of asset segregated and restricted to provide benefit under defined benefit plan. Asset includes, but is not limited to, stock, bond, other investment, earning from investment, and contribution by employer and employee.

+ References + Details

PROPERTY AND EQUIPMENT (Details) - USD ($) $ in Thousands	12 Months Ended
PROPERTY AND EQUIPMENT (Details) - USD ($) $ in Thousands	Dec. 31, 2022	Dec. 31, 2021	Dec. 31, 2020
Property, Plant and Equipment [Line Items]
Property, plant and equipment, gross	$ 14,244	$ 14,154
Less: Accumulated depreciation	(5,195)	(3,048)
Total property and equipment, net	9,049	11,106
Depreciation expense	2,100	1,700	$ 2,100
Computers and equipment
Property, Plant and Equipment [Line Items]
Property, plant and equipment, gross	2,049	1,950
Furniture and fixtures
Property, Plant and Equipment [Line Items]
Property, plant and equipment, gross	2,938	2,783
Leasehold improvements
Property, Plant and Equipment [Line Items]
Property, plant and equipment, gross	9,180	8,790
Construction-in-progress
Property, Plant and Equipment [Line Items]
Property, plant and equipment, gross	$ 77	$ 631

LEASES - Additional Information (Details) $ in Thousands	12 Months Ended
LEASES - Additional Information (Details) $ in Thousands	Dec. 31, 2022 USD ($) lease	Dec. 31, 2021 USD ($)	Dec. 31, 2020 USD ($)
Lessee, Lease, Description [Line Items]
Operating lease right of use assets	$ 21,000	$ 23,196
Operating lease liability	31,943
Operating lease expense	$ 5,000	$ 4,900	$ 2,000
Office Lease 2020
Lessee, Lease, Description [Line Items]
Operating lease right of use assets			34,600
Operating lease liability			34,500
Tenant improvement allowance			$ 7,900
Operating lease extension term	5 years
Base rent due over the initial term of the lease	$ 49,500
Kilroy Realty, L.P.
Lessee, Lease, Description [Line Items]
Number of operating leases \| lease	2
Esprit Investments Limited
Lessee, Lease, Description [Line Items]
Number of operating leases \| lease	1
Operating lease right of use assets	$ 400
Operating lease liability	$ 400
Operating lease extension term	5 years
Base rent due over the initial term of the lease	$ 500

LEASES - Future Minimum Rent Commitments (Details) - USD ($) $ in Thousands	Dec. 31, 2022	Dec. 31, 2021
Leases [Abstract]
2023	$ 6,314
2024	6,501
2025	6,673
2026	6,889
2027	7,064
Thereafter	4,781
Total undiscounted future minimum payments	38,222
Present value discount	(6,279)
Total lease liability	31,943
Unamortized lease incentives	(5,575)
Cash payments in excess of straight-line lease expense	(5,368)
Total ROU asset	$ 21,000	$ 23,196

LEASES - Weighted-Average Remaining Lease Term and Discount Rate (Details)	Dec. 31, 2022	Dec. 31, 2021
Leases [Abstract]
Weighted-average remaining lease term in years	5 years 8 months 12 days	6 years 8 months 12 days
Weighted-average discount rate	6.48%	6.47%

SUBSEQUENT EVENTS (Details) $ in Millions	12 Months Ended
SUBSEQUENT EVENTS (Details) $ in Millions	Dec. 31, 2022 USD ($)
Ligand License Agreement
Subsequent Event [Line Items]
License agreement, milestone payment, milestone one	$ 23.0