Travere Therapeutics (TVTX) 10-Q 31 Oct 24 2024 Q3 Quarterly report Financial data

Cover

Cover - shares	9 Months Ended
	Sep. 30, 2024	Oct. 28, 2024
Cover [Abstract]
Document Type	10-Q
Document Quarterly Report	true
Document Period End Date	Sep. 30, 2024
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 Current Reporting Status	Yes
Entity Interactive Data Current	Yes
Entity Filer Category	Large Accelerated Filer
Entity Small Business	false
Entity Emerging Growth Company	false
Entity Shell Company	false
Entity Common Stock, Shares Outstanding		78,049,928
Entity Central Index Key	0001438533
Current Fiscal Year End Date	--12-31
Amendment Flag	false
Document Fiscal Year Focus	2024
Document Fiscal Period Focus	Q3

CONSOLIDATED BALANCE SHEETS

CONSOLIDATED BALANCE SHEETS - USD ($) $ in Thousands	Sep. 30, 2024	Dec. 31, 2023
Current assets:
Cash and cash equivalents	$ 36,409	$ 58,176
Marketable debt securities, at fair value	241,030	508,675
Accounts receivable, net	25,170	21,179
Inventory	6,356	9,410
Prepaid expenses and other current assets	15,775	19,335
Total current assets	324,740	616,775
Long-term inventory	36,522	31,494
Property and equipment, net	6,162	7,479
Operating lease right of use assets	15,662	18,061
Intangible assets, net	104,205	104,443
Other assets	17,119	10,661
Total assets	504,410	788,913
Current liabilities:
Accounts payable	23,195	41,675
Accrued expenses	83,916	118,991
Convertible debt, current portion	68,599	0
Deferred revenue, current portion	3,799	7,096
Operating lease liabilities, current portion	5,297	4,909
Other current liabilities	5,232	5,237
Total current liabilities	190,038	177,908
Convertible debt, less current portion	309,957	377,263
Operating lease liabilities, less current portion	18,581	22,612
Other non-current liabilities	16,288	10,320
Total liabilities	534,864	588,103
Commitments and Contingencies (See Note 13)
Stockholders' (Deficit) Equity:
Preferred stock $0.0001 par value; 20,000,000 shares authorized; no shares issued and outstanding as of September 30, 2024 and December 31, 2023	0	0
Common stock $0.0001 par value; 200,000,000 shares authorized; 77,909,042, and 75,367,117 issued and outstanding as of September 30, 2024 and December 31, 2023, respectively	8	7
Additional paid-in capital	1,357,457	1,327,881
Accumulated deficit	(1,386,903)	(1,125,622)
Accumulated other comprehensive loss	(1,016)	(1,456)
Total stockholders' (deficit) equity	(30,454)	200,810
Total liabilities and stockholders' (deficit) equity	$ 504,410	$ 788,913

CONSOLIDATED BALANCE SHEETS (Pa

CONSOLIDATED BALANCE SHEETS (Parenthetical) - $ / shares	Sep. 30, 2024	Dec. 31, 2023
Statement of Financial Position [Abstract]
Preferred stock, par value (in dollars per share)	$ 0.0001	$ 0.0001
Preferred stock, authorized (in shares)	20,000,000	20,000,000
Preferred stock, issued (in shares)	0	0
Preferred stock, outstanding (in shares)	0	0
Common stock, par value (in dollars per share)	$ 0.0001	$ 0.0001
Common stock, authorized (in shares)	200,000,000	200,000,000
Common stock, issued (in shares)	77,909,042	75,367,117
Common stock, outstanding (in shares)	77,909,042	75,367,117

CONSOLIDATED STATEMENTS OF OPER

CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE (LOSS) INCOME - USD ($) $ in Thousands	3 Months Ended		9 Months Ended
	Sep. 30, 2024	Sep. 30, 2023	Sep. 30, 2024	Sep. 30, 2023
Total revenue	$ 62,898	$ 37,095	$ 158,388	$ 100,179
Operating expenses:
Cost of goods sold	1,626	1,289	5,191	6,886
Research and development	51,679	60,590	155,429	185,244
Selling, general and administrative	65,619	67,801	194,618	201,954
In-process research and development	0	0	65,205	0
Restructuring	123	0	1,035	0
Total operating expenses	119,047	129,680	421,478	394,084
Operating loss	(56,149)	(92,585)	(263,090)	(293,905)
Other income, net:
Interest income	3,570	5,842	14,022	14,616
Interest expense	(2,777)	(2,821)	(8,365)	(8,513)
Other income (expense), net	520	335	(2,737)	220
Total other income, net	1,313	3,356	2,920	6,323
Loss from continuing operations before income tax provision	(54,836)	(89,229)	(260,170)	(287,582)
Income tax benefit (provision) on continuing operations	84	(12)	(192)	(155)
Loss from continuing operations, net of tax	(54,752)	(89,241)	(260,362)	(287,737)
(Loss) income from discontinued operations, net of tax	(59)	239,976	(919)	266,511
Net (loss) income	$ (54,811)	$ 150,735	$ (261,281)	$ (21,226)
Basic and diluted:
Net loss from continuing operations, basic (in dollars per share)	$ (0.70)	$ (1.17)	$ (3.36)	$ (3.91)
Net loss from continuing operations, diluted (in dollars per share)	(0.70)	(1.17)	(3.36)	(3.91)
Net income (loss) from discontinued operations, basic (in dollars per share)	0	3.14	(0.01)	3.62
Net income (loss) from discontinued operations, diluted (in dollars per share)	0	3.14	(0.01)	3.62
Net (loss) income per common share, basic (in dollars per share)	(0.70)	1.97	(3.37)	(0.29)
Net (loss) income per common share, diluted (in dollars per share)	$ (0.70)	$ 1.97	$ (3.37)	$ (0.29)
Weighted average common shares outstanding, basic (in shares)	77,779,379	76,305,603	77,473,161	73,523,620
Weighted average common shares outstanding, diluted (in shares)	77,779,379	76,305,603	77,473,161	73,523,620
Comprehensive (loss) income:
Net (loss) income	$ (54,811)	$ 150,735	$ (261,281)	$ (21,226)
Foreign currency translation (loss) gain	(803)	8	230	(728)
Unrealized gain (loss) on marketable debt securities	1,421	194	210	(18)
Comprehensive (loss) income	(54,193)	150,937	(260,841)	(21,972)
Net product sales
Total revenue	61,001	33,932	153,161	87,621
Operating expenses:
Cost of goods sold	1,626	1,289	5,059	3,842
License and collaboration revenue
Total revenue	1,897	3,163	5,227	12,558
Operating expenses:
Cost of goods sold	$ 0	$ 0	$ 132	$ 3,044

CONSOLIDATED STATEMENTS OF STOC

CONSOLIDATED STATEMENTS OF STOCKHOLDERS' (DEFICIT) EQUITY - USD ($) $ in Thousands	Total	Underwritten Equity Offering	Common Stock	Common Stock Underwritten Equity Offering	Additional Paid in Capital	Additional Paid in Capital Underwritten Equity Offering	Accumulated Other Comprehensive Loss	Accumulated Deficit
Beginning balance (in shares) at Dec. 31, 2022			64,290,570
Beginning balance at Dec. 31, 2022	$ 42,851		$ 6		$ 1,059,975		$ (2,907)	$ (1,014,223)
Increase (Decrease) in Stockholders' Equity [Roll Forward]
Share based compensation	36,273				36,273
Issuance of common stock under the equity incentive plan and proceeds from exercise (in shares)			960,657
Issuance of common stock under the equity incentive plan and proceeds from exercise	3,240				3,240
Employee stock purchase program purchase and expense (in shares)			156,540
Employee stock purchase program purchase and expense	3,545				3,545
Issuance of common stock, net of issuance costs (in shares)				9,703,750
Issuance of common stock, net of issuance costs		$ 191,199		$ 1		$ 191,198
Issuance of pre-funded common stock warrants, net of issuance costs	24,630				24,630
Foreign currency translation adjustments	(728)						(728)
Unrealized gain (loss) on marketable debt securities	(18)						(18)
Net (loss) income	(21,226)							(21,226)
Ending balance (in shares) at Sep. 30, 2023			75,111,517
Ending balance at Sep. 30, 2023	279,766		$ 7		1,318,861		(3,653)	(1,035,449)
Beginning balance (in shares) at Jun. 30, 2023			74,971,807
Beginning balance at Jun. 30, 2023	116,485		$ 7		1,306,517		(3,855)	(1,186,184)
Increase (Decrease) in Stockholders' Equity [Roll Forward]
Share based compensation	11,775				11,775
Issuance of common stock under the equity incentive plan and proceeds from exercise (in shares)			139,710
Issuance of common stock under the equity incentive plan and proceeds from exercise	151				151
Employee stock purchase program purchase and expense	418				418
Foreign currency translation adjustments	8						8
Unrealized gain (loss) on marketable debt securities	194						194
Net (loss) income	150,735							150,735
Ending balance (in shares) at Sep. 30, 2023			75,111,517
Ending balance at Sep. 30, 2023	279,766		$ 7		1,318,861		(3,653)	(1,035,449)
Beginning balance (in shares) at Dec. 31, 2023			75,367,117
Beginning balance at Dec. 31, 2023	200,810		$ 7		1,327,881		(1,456)	(1,125,622)
Increase (Decrease) in Stockholders' Equity [Roll Forward]
Share based compensation	26,763				26,763
Issuance of common stock under the equity incentive plan and proceeds from exercise (in shares)			1,052,425
Issuance of common stock under the equity incentive plan and proceeds from exercise	572		$ 1		571
Employee stock purchase program purchase and expense (in shares)			239,500
Employee stock purchase program purchase and expense	2,242				2,242
Exercise of pre-funded common stock warrants (in shares)			1,250,000
Foreign currency translation adjustments	230						230
Unrealized gain (loss) on marketable debt securities	210						210
Net (loss) income	(261,281)							(261,281)
Ending balance (in shares) at Sep. 30, 2024			77,909,042
Ending balance at Sep. 30, 2024	(30,454)		$ 8		1,357,457		(1,016)	(1,386,903)
Beginning balance (in shares) at Jun. 30, 2024			76,456,562
Beginning balance at Jun. 30, 2024	15,147		$ 8		1,348,865		(1,634)	(1,332,092)
Increase (Decrease) in Stockholders' Equity [Roll Forward]
Share based compensation	7,729				7,729
Issuance of common stock under the equity incentive plan and proceeds from exercise (in shares)			202,480
Issuance of common stock under the equity incentive plan and proceeds from exercise	571				571
Employee stock purchase program purchase and expense	$ 292				292
Exercise of pre-funded common stock warrants (in shares)	1,250,000		1,250,000
Foreign currency translation adjustments	$ (803)						(803)
Unrealized gain (loss) on marketable debt securities	1,421						1,421
Net (loss) income	(54,811)							(54,811)
Ending balance (in shares) at Sep. 30, 2024			77,909,042
Ending balance at Sep. 30, 2024	$ (30,454)		$ 8		$ 1,357,457		$ (1,016)	$ (1,386,903)

CONSOLIDATED STATEMENTS OF ST_2

CONSOLIDATED STATEMENTS OF STOCKHOLDERS' (DEFICIT) EQUITY (Parenthetical) $ in Millions	9 Months Ended
	Sep. 30, 2023 USD ($)
Pre-funded common stock warrants, net of issuance costs	$ 1.6
Underwritten Equity Offering
Issuance costs	$ 12.6

CONSOLIDATED STATEMENTS OF CASH

CONSOLIDATED STATEMENTS OF CASH FLOWS - USD ($) $ in Thousands	9 Months Ended
	Sep. 30, 2024	Sep. 30, 2023
Cash Flows From Operating Activities:
Net (loss) income	$ (261,281)	$ (21,226)
Net (loss) income from discontinued operations	(919)	266,511
Net loss from continuing operations	(260,362)	(287,737)
Adjustments to reconcile net loss to net cash used in operating activities:
Depreciation and amortization	31,464	29,042
Share based compensation	27,699	36,102
In-process research and development	65,205	0
Other	5,022	(5,815)
Changes in operating assets and liabilities:
Accounts receivable	(4,017)	(3,295)
Inventory	(3,486)	(17,027)
Prepaid expenses and other current and non-current assets	(4,903)	(4,402)
Accounts payable	(18,573)	7,468
Accrued expenses	(38,794)	(3,903)
Deferred revenue, current and non-current	(5,557)	(10,200)
Other current and non-current liabilities	4,909	(1,217)
Net cash used in operating activities - continuing operations	(201,393)	(260,984)
Net cash (used in) provided by operating activities - discontinued operations	(362)	51,436
Net cash used in operating activities	(201,755)	(209,548)
Cash Flows From Investing Activities:
Proceeds from the sale and maturity of marketable debt securities	271,285	284,575
Purchase of marketable debt securities	0	(381,294)
Purchase of fixed assets	(82)	(643)
Purchase of intangible assets	(26,569)	(36,086)
Payment of milestone	(65,000)	0
Net cash provided by (used in) investing activities - continuing operations	179,634	(133,448)
Net cash provided by investing activities - discontinued operations	0	206,174
Net cash provided by investing activities	179,634	72,726
Cash Flows From Financing Activities:
Payment of guaranteed minimum royalty	(1,575)	(1,575)
Proceeds from the issuance of pre-funded warrants, net of issuance costs	0	24,630
Proceeds from exercise of stock options	571	3,240
Proceeds from issuances under the employee stock purchase plan	1,307	2,258
Net cash provided by financing activities - continuing operations	303	219,751
Net cash used in financing activities - discontinued operations	0	(1,123)
Net cash provided by financing activities	303	218,628
Effect of exchange rate changes on cash	51	750
Net (decrease) increase in cash and cash equivalents	(21,767)	82,556
Cash and cash equivalents, beginning of year	58,176	61,688
Cash and cash equivalents, end of period	36,409	144,244
Underwritten Equity Offering
Cash Flows From Financing Activities:
Proceeds from the issuance of common stock, net of issuance costs	$ 0	$ 191,198

DESCRIPTION OF BUSINESS

DESCRIPTION OF BUSINESS	9 Months Ended
	Sep. 30, 2024
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 its 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 and metabolic diseases. The Company regularly evaluates and, where appropriate, acts on opportunities to expand its product pipeline through licenses and acquisitions of products in areas that will serve patients with serious unmet medical need and that the Company believes offer attractive growth characteristics. Discontinued Operations - Sale of Bile Acid Product Portfolio In July 2023, Travere entered into an Asset Purchase Agreement (the “Purchase Agreement”) with Mirum Pharmaceuticals, Inc. ("Mirum Pharmaceuticals" or “Mirum”), pursuant to which Mirum agreed to purchase from Travere substantially all of the assets primarily related to Travere’s business of development, manufacture (including synthesis, formulation, finishing or packaging) and commercialization of Chenodal and Cholbam (also known as Kolbam, and together with Chenodal, the “Products”), collectively, the "bile acid business". On August 31, 2023, the Company and Mirum consummated the transactions contemplated by the Purchase Agreement (the “Closing”). In connection with the Closing, Mirum paid Travere an upfront cash payment of $210.0 million. Pursuant to the Purchase Agreement, after the Closing, Travere is eligible to receive up to $235.0 million upon the achievement of certain milestones based on specified amounts of annual net sales (tiered from $125.0 million to $500.0 million) of the Products. The Company has reflected the bile acid business as a discontinued operation in the unaudited consolidated financial statements for all periods presented. See Note 18 for further discussion. Unless otherwise noted, amounts and disclosures throughout the Notes to the unaudited consolidated financial statements relate to the Company's continuing operations. Approved Products: FILSPARI® (sparsentan) On September 5, 2024, the FDA granted full approval of FILSPARI® (sparsentan) to slow kidney function decline in adults with primary Immunoglobulin A nephropathy (IgAN) who are at risk of disease progression. FILSPARI is the only oral, once-daily, non-immunosuppressive medication that directly targets glomerular injury in the kidney by blocking two critical pathways of IgAN disease progression (endothelin-1 and angiotensin II). FILSPARI had previously been granted accelerated approval in February 2023 based on the surrogate marker of proteinuria. Full approval is based on positive long-term confirmatory results from the PROTECT Study demonstrating that FILSPARI significantly slowed kidney function decline over two years compared to irbesartan. FILSPARI initially became commercially available in the U.S. in February 2023 under accelerated approval, and the Company is providing a comprehensive patient support program throughout the patient’s treatment journey. In September 2021, the Company entered into a license and collaboration agreement with Vifor (International) Ltd. ("CSL Vifor"). In April 2024, the Company and CSL Vifor announced that the European Commission has granted conditional marketing authorization (“CMA”) for FILSPARI (sparsentan) for the treatment of adults with primary IgAN with a urine protein excretion ≥1.0 g/day (or urine protein-to-creatinine ratio ≥0.75 g/g). The CMA is granted for all member states of the European Union, as well as in Iceland, Liechtenstein and Norway. The European Commission's decision follows the positive opinion from the Committee for Medicinal Products for Human Use (“CHMP”) in February 2024, based on results from the pivotal Phase 3 PROTECT Study of FILSPARI in IgAN. Under the terms of the License Agreement, the Company will be entitled to receive a regulatory milestone payment of $17.5 million upon receipt of full regulatory approval by the European Commission for IgAN, and an additional milestone payment upon achievement of market access initiatives in certain countries. FILSPARI became commercially available in Europe under the CMA in August 2024, with an initial launch in Germany and Austria. In October 2024, the Company and CSL Vifor announced that Swissmedic has granted temporary marketing authorization for FILSPARI for the treatment of adults with primary IgAN with a urine protein excretion ≥1.0 g/day (or urine protein-to-creatinine ratio ≥0.75 g/g). Thiola® and Thiola EC® (tiopronin tablets) 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. Clinical-Stage Programs: Sparsentan for the treatment of FSGS 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 the European Economic Area countries (the “EEA”). In December 2023, the Company announced that it had completed its planned Type C meeting with the FDA to discuss previously reported results from the Phase 3 DUPLEX Study of sparsentan in FSGS. The FDA acknowledged the high unmet need for approved therapies as well as the challenges in studying FSGS but indicated that the two-year results from the Phase 3 DUPLEX Study alone were not sufficient to support an sNDA submission. Together with CSL Vifor, the Company also plans to engage with the EMA to determine the potential for a subsequent variation to the CMA of sparsentan for the treatment of FSGS, subject to a review decision on the pending application for CMA of sparsentan in IgAN. The Company is conducting additional analyses of FSGS data and plans to engage with regulators to evaluate potential regulatory pathways for a sparsentan FSGS indication. Pegtibatinase Pegtibatinas e 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. In May 2023, the Company announced positive topline results from cohort 6 in the Phase 1/2 COMPOSE Study. In December 2023, the Company initiated the pivotal Phase 3 HARMONY Study to support the potential approval of pegtibatinase for the treatment of classical HCU. The HARMONY Study is a global, randomized, multi-center, double-blind, placebo-controlled Phase 3 clinical trial designed to evaluate the efficacy and safety of pegtibatinase as a novel treatment to reduce total homocysteine (tHcy) levels. In the beginning of 2024, the first patients were dosed in the HARMONY Study. In September 2024, the Company announced a voluntary pause of enrollment in the Phase 3 HARMONY Study. The voluntary enrollment pause enables the Company to work to address necessary process improvements in manufacturing scale-up to support commercial scale manufacturing as well as full enrollment in the HARMONY Study. Patients currently enrolled in pegtibatinase studies continue to receive study medication from small scale batches which are unaffected by the scale-up process. Currently enrolled patients will be able to continue on study medication as scheduled for the duration of the trials they are participating in. The voluntary enrollment pause was enacted following our determination that the desired drug substance profile was not achieved in the recent scale-up process. The Company expects to further evaluate the necessary commercial process improvements to enable the continuation of the Phase 3 program. The Company acquired pegtibatinase as part of the November 2020 acquisition of Orphan Technologies Limited. Preclinical Programs: The Company is party to a collaboration agreement with PharmaKrysto Limited and their early-stage cystinuria discovery program, whereby the Company is responsible for funding all research and development expenses for the pre-clinical activities associated with the cystinuria program.

BASIS OF PRESENTATION AND SIGNI

BASIS OF PRESENTATION AND SIGNIFICANT ACCOUNTING POLICIES	9 Months Ended
	Sep. 30, 2024
Accounting Policies [Abstract]
BASIS OF PRESENTATION AND SIGNIFICANT ACCOUNTING POLICIES	BASIS OF PRESENTATION AND SIGNIFICANT ACCOUNTING POLICIES The accompanying unaudited consolidated financial statements should be read in conjunction with the audited consolidated financial statements and notes thereto included in our Annual Report on Form 10-K for the year ended December 31, 2023, filed with the SEC on February 20, 2024. The accompanying consolidated financial statements have been prepared in accordance with accounting principles generally accepted in the United States (“GAAP”) for interim financial information, the instructions for Form 10-Q and the rules and regulations of the SEC. Accordingly, since they are interim statements, the accompanying consolidated financial statements do not include all of the information and notes required by GAAP for annual financial statements, but reflect all adjustments consisting of normal, recurring adjustments, that are necessary for a fair statement of the financial position, results of operations and cash flows for the interim periods presented. Interim results are not necessarily indicative of the results that may be expected for any future periods. The December 31, 2023 balance sheet information was derived from the audited financial statements as of that date. Certain reclassifications have been made to the prior period consolidated financial statements to conform to the current period presentation, including reclassifying the prior period revenues and expenses attributable to the bile acid business as net income from discontinued operations. These reclassifications did not have an impact on total assets or total liabilities in the Consolidated Balance Sheets or net loss in the Consolidated Statements of Operations. A summary of the significant accounting policies applied in the preparation of the accompanying consolidated financial statements follows: Principles of Consolidation The unaudited 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 6 for further discussion of variable interest entities (“VIE”) that the Company consolidates. 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 recognizes revenue from 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 judgment 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. Revenue from collaboration and licensing agreements may also include sales of inventory, at cost plus a margin, which is recorded in license and collaboration revenue. The Company utilizes significant judgment 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 judgment 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. Cost of goods sold 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. Cost of goods sold also includes the cost of goods sold under the Company's license and collaboration agreements, which currently consists of the sale of active pharmaceutical ingredients to the Company's collaboration partners, at cost or at cost plus a margin. The following table summarizes cost of goods sold for the three and nine months ended September 30, 2024 and 2023 ( in thousands ): Three Months Ended September 30, Nine Months Ended September 30, 2024 2023 2024 2023 Cost of goods sold - product sales $ 1,626 $ 1,289 $ 5,059 $ 3,842 Cost of goods sold - license and collaboration — — 132 3,044 Total cost of goods sold $ 1,626 $ 1,289 $ 5,191 $ 6,886 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. Prior to the February 2023 FDA accelerated approval of FILSPARI (sparsentan), the Company expensed the production of active pharmaceutical ingredients purchased to support the commercial launch of FILSPARI, in research and development expenses. For the three and nine months ended September 30, 2024 and 2023, sales of FILSPARI primarily consisted of zero-cost inventories. As of September 30, 2024, the Company had approximately $2.6 million of zero-cost inventory. The Company expects to continue to record zero cost of goods sold on the sale of previously expensed inventories through at least 2025. The Company began capitalizing inventory costs associated with FILSPARI following the February 2023 accelerated approval. 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, costs to develop drug materials and delivery devices, costs to 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. 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 two Phase 2 clinical trials and four 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. 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 ("Thiola Intangible"). The additional cost basis is subsequently amortized over the remaining useful life. In the second quarter of 2023, the Company reduced the estimated useful life of the Thiola Intangible to better reflect the pattern of projected future cash flows. The change in estimated useful life was accounted for as a change in accounting estimate with the remaining carrying amounts of the Thiola Intangible being amortized prospectively over the new useful life. 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 September 30, 2024 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 judgments 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. Equity Securities The Company applies the equity method of accounting for investments when it has significant influence, but no controlling interest in the investee. Judgment regarding the level of influence over each equity method investment includes key factors such as ownership interest, representation on the board of directors, participation in joint steering committees and material intercompany transactions. The Company evaluates any basis difference between the carrying value and fair value of the Company's proportionate share of the investee's net assets. Basis differences relating to in-process research and development (IPR&D) are expensed when the investee is not considered a business as defined in ASC 805, Business Combinations, due to substantially all of the estimated fair value of the gross assets being concentrated in a group of similar IPR&D assets with no alternative future use. For the nine months ending September 30, 2024, the Company recognized $3.4 million in other expense in the Company's consolidated statements of operations for these basis adjustments. The equity method investment's carrying value was reduced to zero as the Company's proportionate share of the basis difference exceeded the carrying value. See Note 6 for further discussion. Investments accounted for using the equity method may be reported on a lag of up to three months if the financial statements of the investee are not available in sufficient time for the Company to apply the equity method as of the current reporting date. Discontinued Operations Discontinued operations is presented when there is a disposal of a component or a group of components that in the Company's judgment represents a strategic shift that will have a major effect on the Company's operations and financial results. Results of operations directly related to discontinued operations are aggregated into a single line item in the Consolidated Statements of Operations for all periods presented. See Note 18 for further discussion. Restructuring Restructuring charges consist primarily of employee severance, one-time termination benefits related to the reduction of its workforce, and other costs. Liabilities for costs associated with a restructuring activity are recognized when the liability is incurred and are measured at fair value. One-time termination benefits are expensed at the date the entity notifies the employee, unless the employee must provide future service, in which case the benefits are expensed ratably over the service period. Termination benefits are calculated based on regional benefit practices and local statutory requirements. In December 2023, the Company initiated a restructuring plan that resulted in a reduction of its workforce, primarily impacting non-field-based employees. One-time termination benefits include severance, continuation of health insurance coverage, and other benefits for a specified period of time. The Company estimated that it will incur a total of $12.0 million to $14.0 million in non-recurring charges in connection with the restructuring, of which the Company has recognized a total of $12.4 million as of September 30, 2024, including $0.1 million and $1.0 million for the three and nine months ended September 30, 2024, respectively. As of September 30, 2024 and December 31, 2023, the Company had accruals related to the restructuring of $0.4 million and $11.4 million, respectively, which is included in accrued expenses in the Consolidated Balance Sheets. Cash payments totaling $12.1 million were made related to the restructuring during 2024. The Company expects that it will incur and pay all remaining restructuring costs during 2024. The following table sets forth a summary of changes in accrued restructuring costs for the nine months ended September 30, 2024 and 2023 ( in thousands ): Restructuring 2024 2023 Liability balance at January 1, $ 11,421 $ — Restructuring expenses 1,035 — Payments (12,132) — Foreign currency impact 34 — Liability balance at September 30, $ 358 $ — Recently Adopted Accounting Pronouncements In November 2023, the FASB issued ASU No. 2023-07, Improvements to Reportable Segment Disclosure. The FASB amended the guidance in ASC 280, Segment Reporting ("ASC 280"), to require a public entity to disclose significant segment expenses and other segment items on an annual and interim basis and to provide in interim periods all disclosures about a reportable segment's profit or loss and assets that are currently required annually. Public entities with a single reportable segment are required to provide the new disclosures and all the disclosures required under ASC 280. The guidance is applied retrospectively to all periods presented in financial statements, unless it is impracticable. This new guidance is effective for public business entities for annual periods beginning after December 15, 2023, and for interim periods beginning after December 15, 2024. The Company plans to adopt this new standard for its Annual Report on Form 10-K for the fiscal year ended December 31, 2024 . While this accounting standard will increase disclosures, it will not have a material impact on the Company's financial statements. 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. In December 2023, the FASB issued ASU No. 2023-09, Improvements to Income Tax Disclosures. This new standard does not change accounting for income taxes but requires new disclosures focusing on two areas, the effective rate reconciliation and taxes paid. This new standard is effective for public business entities for annual periods beginning after December 15, 2024. Early adoption is permitted. The Company expects to adopt this new standard beginning in fiscal 2025 when it becomes effective.

REVENUE RECOGNITION

REVENUE RECOGNITION	9 Months Ended
	Sep. 30, 2024
Revenue from Contract with Customer [Abstract]
REVENUE RECOGNITION	REVENUE RECOGNITION Product Sales, Net Product sales consist of FILSPARI and tiopronin products (Thiola and Thiola EC). The Company sells its products to specialty pharmacies and through direct-to-patient distributors worldwide, with the United States representing over 98% of net product sales. The Company sells FILSPARI to two direct-to-patient specialty pharmacies. The Company sells its tiopronin products to patients and pharmacies, with distribution facilitated through a single direct-to-patient distributor. Revenues from product sales are recognized in satisfaction of a single performance obligation when the customer obtains control of the Company’s product. For FILSPARI, sales are recognized upon delivery of the product to the specialty pharmacies. The Company receives payments from its FILSPARI sales based on terms that are generally 30 days from shipment of the product to the specialty pharmacy. For the Company's tiopronin products, product sales are recognized upon delivery to the patient. The Company receives payments from sales of its other products, 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, 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 nine months ended September 30, 2024 and 2023, adjustments to net product revenue related to performance obligations satisfied in previous periods were $0.5 million and $0.4 million, respectively. 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 an estimated allocation of payers and the government-mandated discounts applicable to government-funded programs. Rebate discounts are included in accrued expenses in the accompanying Consolidated Balance Sheets. Commercial Rebates: The Company calculates the rebates it incurs according to any 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 for applicable products. Rebate discounts are included in accrued expenses 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. Other Fees: The Company pays service fees to certain customers based on a contractually fixed percentage of the wholesale acquisition cost (WAC) and fees for data. Other fees are recorded as an offset to revenue based on contractual terms at the time revenue from the sale is recognized. 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. Historically, returns have been immaterial. 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 estimate of claims and the estimated cost per claim associated with product that has been recognized as revenue. The following table summarizes net product sales for the three and nine months ended September 30, 2024 and 2023 (in thousands): Three Months Ended September 30, Nine Months Ended September 30, 2024 2023 2024 2023 FILSPARI $ 35,619 $ 8,044 $ 82,578 $ 14,509 Tiopronin products 25,382 25,888 70,583 73,112 Total net product sales $ 61,001 $ 33,932 $ 153,161 $ 87,621

COLLABORATION AND LICENSE AGREE

COLLABORATION AND LICENSE AGREEMENTS	9 Months Ended
	Sep. 30, 2024
Organization, Consolidation and Presentation of Financial Statements [Abstract]
COLLABORATION AND LICENSE AGREEMENTS	COLLABORATION AND LICENSE AGREEMENTS License Agreement with CSL Vifor On September 15, 2021, the Company entered into a license and collaboration agreement (“CSL Vifor 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 ("CSL Vifor 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 CSL Vifor 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 up to 40 percent of annual net sales of sparsentan in the CSL Vifor Licensed Territories. Under the CSL Vifor License Agreement, CSL Vifor will be responsible for all commercialization activities in the CSL Vifor 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 CSL Vifor 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 CSL Vifor 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 CSL Vifor License Agreement for the other party’s uncured material breach, insolvency or if the time required for performance under the CSL Vifor 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 CSL Vifor License Agreement and determined that it meets both criteria to be considered a collaborative agreement within the Scope of ASC 808, Collaborative Arrangements ("ASC 808") 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 CSL Vifor 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 CSL Vifor 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. For the three and nine months ended September 30, 2024, the Company recognized $1.8 million and $5.1 million, respectively, in license and collaboration revenue for clinical development activities, based upon the ratio of costs incurred to total estimated costs. For the three and nine months ended September 30, 2023, the Company recognized $3.2 million and $9.3 million, respectively, in license and collaboration revenue for clinical development activities, based upon the ratio of costs incurred to total estimated costs. For the nine months ended September 30, 2023, the Company recognized an additional $3.3 million in license and collaboration revenue from the sale of active pharmaceutical ingredients to CSL Vifor at cost plus a margin. Deferred revenue related to the clinical development activities as of September 30, 2024 was $3.8 million, with the total amount classified as current based upon amounts expected to be realized within the next year. As of December 31, 2023, deferred revenue related to the clinical development activities was $8.9 million, of which $7.1 million was classified as current. The Company estimates that the remainder of the deferred revenue balance will be fully realized by mid-2025. For the three and nine months ended September 30, 2024, the Company recognized $0.1 million and $0.1 million, respectively, in license and collaboration revenue for royalties earned on net sales of FILSPARI in the CSL Vifor Licensed Territories following the August 2024 launch. Licensing Agreement with Renalys In January 2024, the license agreement (“Renalys License Agreement”) between the Company and Renalys Pharma, Inc. (“Renalys”) came into effect. Pursuant to the terms of the Renalys License Agreement, the Company granted an exclusive license to Renalys for the development and commercialization of sparsentan in Japan, South Korea, Taiwan and other specified Asian countries ("Renalys Licensed Territories"). Under the terms of the Renalys License Agreement, the Company received a non-refundable upfront payment and will be eligible to receive up to $120.0 million in aggregate development and sales-based milestones. The Company is also entitled to receive tiered double-digit to mid-20 percent royalties of annual net sales of sparsentan in the Renalys Licensed Territories. In addition, the Company received an option to purchase shares of common stock of Renalys (“Option Agreement”), which it exercised in January 2024. The Company has the option to purchase all equity securities of Renalys at any time prior to the top-line results of the Phase 3 trial in Japan (“Buyout Right”). Under the Renalys License Agreement, Renalys will be responsible for all development and commercialization activities in the Renalys Licensed Territories. The Renalys License Agreement will remain in effect, unless terminated earlier, until the expiration of all royalty terms for sparsentan in the Renalys Licensed Territories. Each party has the right to terminate the Renalys License Agreement for the other party’s uncured material breach or insolvency, or if the time required for performance under the Renalys License Agreement by the other party is extended due to a force majeure event that continues for nine months or more. Renalys may terminate the Renalys License Agreement for any reason upon prior written notice to the Company. The Company may terminate the Renalys License Agreement if Renalys abandons development in Japan or South Korea prior to first commercial sales of sparsentan in either Japan or South Korea. The Company assessed the Renalys 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 a joint steering committee overseeing the development and commercial 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 Renalys License Agreement totaled $8.3 million, consisting of the fixed non-refundable upfront payment, milestone payment and estimated fair value of the Option Agreement. The variable development-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 has been satisfied. The Company concluded that Renalys represents a customer and applied relevant guidance from ASC 606 to evaluate the accounting under the Renalys License Agreement. In accordance with this guidance, the Company concluded that the promise to grant the license is distinct, resulting in one performance obligation as the license has stand-alone functionally at contract inception. The Buyout Right precludes transferring control of the license to Renalys under ASC 606 and the Company’s option to repurchase the common stock at a price greater than the original license premium results in accounting for the Renalys License Agreement as a financing arrangement. The transaction price was recorded in other non-current liabilities, and will be recognized in revenue upon exercise or termination of the Buyout Right. See Note 6 for further discussion of VIE’s.

MARKETABLE DEBT SECURITIES

MARKETABLE DEBT SECURITIES	9 Months Ended
	Sep. 30, 2024
Investments, Debt and Equity Securities [Abstract]
MARKETABLE DEBT SECURITIES	MARKETABLE DEBT SECURITIES The Company's marketable debt securities as of September 30, 2024 and December 31, 2023 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 consisted of the following ( in thousands ): September 30, 2024 December 31, 2023 Marketable debt securities: Commercial paper $ — $ 34,458 Corporate debt securities 193,468 368,323 Securities of government sponsored entities 47,562 105,894 Total available-for-sale marketable debt securities $ 241,030 $ 508,675 The following is a summary of short-term marketable debt securities classified as available-for-sale as of September 30, 2024 ( in thousands ): Remaining Contractual Maturity Amortized Cost Unrealized Gains Unrealized Losses Aggregate Estimated Fair Value Marketable debt securities: Corporate debt securities Less than 1 $ 170,739 $ 663 $ (18) $ 171,384 Securities of government-sponsored entities Less than 1 47,523 50 (11) 47,562 Total maturity less than 1 year 218,262 713 (29) 218,946 Corporate debt securities 1 to 2 21,815 269 — 22,084 Total maturity 1 to 2 years 21,815 269 — 22,084 Total available-for-sale marketable debt securities $ 240,077 $ 982 $ (29) $ 241,030 The following is a summary of short-term marketable debt securities classified as available-for-sale as of December 31, 2023 ( in thousands ): Remaining Contractual Maturity Amortized Cost Unrealized Gains Unrealized Losses Aggregate Estimated Fair Value Marketable debt securities: Commercial paper Less than 1 $ 34,450 $ 25 $ (17) $ 34,458 Corporate debt securities Less than 1 133,463 29 (408) 133,084 Securities of government-sponsored entities Less than 1 81,334 36 (274) 81,096 Total maturity less than 1 year 249,247 90 (699) 248,638 Corporate debt securities 1 to 2 233,969 1,444 (174) 235,239 Securities of government-sponsored entities 1 to 2 24,718 106 (26) 24,798 Total maturity 1 to 2 years 258,687 1,550 (200) 260,037 Total available-for-sale securities $ 507,934 $ 1,640 $ (899) $ 508,675 For the three and nine months ended September 30, 2024 and 2023, realized gains and losses on marketable debt securities were immaterial. As of September 30, 2024 and December 31, 2023, the accrued interest receivable related to the Company's marketable debt securities was $2.5 million and $4.6 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 September 30, 2024 ( 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 Corporate debt securities $ — $ — $ 38,602 $ 18 $ 38,602 $ 18 Securities of government-sponsored entities — — 18,836 11 18,836 11 Total $ — $ — $ 57,438 $ 29 $ 57,438 $ 29 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, 2023 ( 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 $ 24,798 $ 17 $ — $ — $ 24,798 $ 17 Corporate debt securities 140,802 405 28,775 177 169,577 582 Securities of government-sponsored entities 61,933 217 12,540 83 74,473 300 Total $ 227,533 $ 639 $ 41,315 $ 260 $ 268,848 $ 899 As of September 30, 2024 and December 31, 2023, the amortized cost of the available-for-sale marketable debt securities in an unrealized loss position was $57.5 million and $269.7 million, respectively. As of September 30, 2024 and December 31, 2023, 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 decrease in unrealized losses for the nine months ended September 30, 2024 was primarily due to fluctuations 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.

VARIABLE INTEREST ENTITIES

VARIABLE INTEREST ENTITIES	9 Months Ended
	Sep. 30, 2024
Organization, Consolidation and Presentation of Financial Statements [Abstract]
VARIABLE INTEREST ENTITIES	VARIABLE INTEREST ENTITIES Stock Purchase and Collaboration Agreement with PharmaKrysto In March 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 all of 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 consolidated assets and liabilities as of September 30, 2024 and December 31, 2023 were immaterial. The results of operations were not significant for the three and nine months ended September 30, 2024 and 2023. 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. Licensing Agreement with Renalys In January 2024, the Renalys License Agreement between the Company and Renalys came into effect and the Company exercised its option to purchase shares of common stock of Renalys. The Company determined that Renalys is a VIE as they could require additional funding to support development and commercial activities. The Company has variable interests in Renalys, including an equity interest, Buyout Right and performance-related payments under the Renalys License Agreement that absorb variability from the performance of Renalys. In order to determine the primary beneficiary of Renalys, the Company evaluated its variable interest to identify if the Company had the power to direct the activities that most significantly impact the economic performance. Based upon the capital structure, governing documents and overall business operations, the Company determined that it is not the primary beneficiary as it does do not have the power to direct the activities that most significantly impact the economic performance of Renalys and does not have an obligation to absorb losses. As of September 30, 2024, the carrying amount of the liabilities related to the Company’s variable interests was $8.3 million, recorded in other non-current liabilities in the Company’s Consolidated Balance Sheets. The Company’s maximum exposure to loss as of September 30, 2024 is zero. The Company is not required to provide additional funding. The creditors have no recourse to the general credit or assets of the Company.

LEASES

LEASES	9 Months Ended
	Sep. 30, 2024
Leases [Abstract]
LEASES	LEASES As of September 30, 2024, 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 lease inception, it was 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. The Company 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 at lease inception, it was not reasonably certain that the Company would elect this option and therefore the renewal period was excluded from the initial lease measurement. Utilizing a discount rate equal to the Company's estimated incremental borrowing rate, the Company established an ROU asset and corresponding lease liability of $0.4 million. The following is a schedule of the future minimum rental commitments for the Company's operating leases reconciled to the lease liability and ROU asset as of September 30, 2024 ( in thousands ): September 30, 2024 2024 (remaining three months) $ 1,648 2025 6,673 2026 6,889 2027 7,064 2028 4,781 Total undiscounted future minimum payments 27,055 Present value discount (3,177) Total lease liability 23,878 Unamortized lease incentives (3,853) Cash payments in excess of straight-line lease expense (4,363) Total ROU asset $ 15,662 The weighted-average remaining lease term and weighted-average discount rate of the Company's operating leases are as follows: September 30, 2024 December 31, 2023 Weighted-average remaining lease term in years 3.9 4.7 Weighted-average discount rate 6.48 % 6.48 %

FAIR VALUE MEASUREMENTS

FAIR VALUE MEASUREMENTS	9 Months Ended
	Sep. 30, 2024
Fair Value Disclosures [Abstract]
FAIR VALUE MEASUREMENTS	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 September 30, 2024, the fair value of the Company's 2.5% Convertible Senior Notes due 2025 was $66.0 million and the fair value of the Company's 2.25% Convertible Senior Notes due 2029 was $272.8 million. As of December 31, 2023, the fair value of the Company's 2.5% Convertible Senior Notes due 2025 was $58.3 million and the fair value of the Company's 2.25% Convertible Senior Notes due 2029 was $212.1 million. The fair values were estimated utilizing market quotations and are considered Level 2. The following table presents the Company’s assets, measured and recognized at fair value on a recurring basis, classified under the appropriate level of the fair value hierarchy as of September 30, 2024 ( in thousands ): As of September 30, 2024 Total carrying and estimated fair value Quoted prices in active markets Significant other observable inputs (Level 2) Significant unobservable inputs (Level 3) Assets: Cash and cash equivalents $ 36,409 $ 36,409 $ — $ — Marketable debt securities, available-for-sale 241,030 — 241,030 — Total $ 277,439 $ 36,409 $ 241,030 $ — The following table presents the Company’s assets, measured and recognized at fair value on a recurring basis, classified under the appropriate level of the fair value hierarchy as of December 31, 2023 ( in thousands ): As of December 31, 2023 Total carrying and estimated fair value Quoted prices in active markets Significant other observable inputs (Level 2) Significant unobservable inputs (Level 3) Assets: Cash and cash equivalents $ 58,176 $ 58,176 $ — $ — Marketable debt securities, available-for-sale 508,675 — 508,675 — Total $ 566,851 $ 58,176 $ 508,675 $ —

INTANGIBLE ASSETS

INTANGIBLE ASSETS	9 Months Ended
	Sep. 30, 2024
Goodwill and Intangible Assets Disclosure [Abstract]
INTANGIBLE ASSETS	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”). 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. Through September 30, 2024 the Company has capitalized $47.2 million for contractual milestones achieved under the Ligand License Agreement, including $5.8 million for the nine months ended September 30, 2024. Pursuant to the Ligand License Agreement, the Company is obligated to pay to Ligand (and Bristol-Myers Squibb Company ("BMS")) an escalating royalty between 15% and 17% of net sales of sparsentan, with payments due quarterly. The Company began incurring costs associated with such royalties following the February 2023 approval of FILSPARI (sparsentan). For the three and nine months ended September 30, 2024, the Company capitalized $5.4 million and $12.4 million, respectively, to intangible assets for royalties owed on net sales of FILSPARI. The cost of the milestone payments and royalty payments are being amortized to selling, general and administration on a straight-line basis through April 30, 2033. The following table sets forth amortizable intangible assets as of September 30, 2024 and December 31, 2023 ( in thousands ): September 30, 2024 December 31, 2023 Finite-lived intangible assets $ 212,866 $ 183,037 Less: accumulated amortization (109,408) (79,347) Net carrying value $ 103,458 $ 103,690 As of September 30, 2024 and December 31, 2023, the Company had goodwill of $0.8 million. The following table summarizes amortization expense for the three and nine months ended September 30, 2024 and 2023 ( in thousands ): Three Months Ended September 30, Nine Months Ended September 30, 2024 2023 2024 2023 Research and development $ — $ 2,447 $ — $ 7,261 Selling, general and administrative 10,812 8,461 30,061 20,097 Total amortization expense $ 10,812 $ 10,908 $ 30,061 $ 27,358

CONVERTIBLE NOTES PAYABLE

CONVERTIBLE NOTES PAYABLE	9 Months Ended
	Sep. 30, 2024
Debt Disclosure [Abstract]
CONVERTIBLE NOTES PAYABLE	CONVERTIBLE NOTES PAYABLE The composition of the Company’s convertible senior notes are as follows ( in thousands ): September 30, 2024 December 31, 2023 2.25% convertible senior notes due 2029 $ 316,250 $ 316,250 2.50% convertible senior notes due 2025 68,904 68,904 Unamortized debt issuance costs - 2.25% convertible senior notes due 2029 (6,293) (7,348) Unamortized debt issuance costs - 2.50% convertible senior notes due 2025 (305) (543) Total convertible senior notes, net of unamortized debt discount and debt issuance costs $ 378,556 $ 377,263 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 September 30, 2024, accrued interest on the 2029 Notes of $0.6 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 Indentures, 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. The Company 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 (collectively, the "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 September 30, 2024, accrued interest of $0.1 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 nine months ended September 30, 2023, 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 September 30, 2024 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 September 30, 2024. The 2025 and 2029 Notes are classified on the Company's Consolidated Balance Sheets at September 30, 2024 as short-term and long-tern convertible debt, respectively. The following table sets forth total interest expense recognized related to the 2025 and 2029 Notes ( in thousands ): Three Months Ended September 30, Nine Months Ended September 30, 2024 2023 2024 2023 Contractual interest expense $ 2,210 $ 2,210 $ 6,629 $ 6,629 Amortization of debt issuance costs 432 430 1,293 1,288 Total interest expense for the 2025 and 2029 Notes $ 2,642 $ 2,640 $ 7,922 $ 7,917 Total interest expense recognized for the three and nine months ended September 30, 2024 was $2.8 million and $8.4 million, respectively. Total interest expense recognized for the three and nine months ended September 30, 2023 was $2.8 million and $8.5 million, respectively.

ACCRUED EXPENSES

ACCRUED EXPENSES	9 Months Ended
	Sep. 30, 2024
Payables and Accruals [Abstract]
ACCRUED EXPENSES	ACCRUED EXPENSES Accrued expenses at September 30, 2024 and December 31, 2023 consisted of the following ( in thousands ): September 30, 2024 December 31, 2023 Compensation related costs $ 28,295 $ 29,908 Research and development 17,065 26,006 Accrued royalties 10,168 6,991 Sales discounts, rebates, and allowances 9,920 13,730 Selling, general and administrative 7,607 7,190 French rebate accrual 6,292 5,377 Transition services accrual 1,239 12,282 Accrued restructuring costs 358 11,421 Miscellaneous accrued expenses 2,972 6,086 Total accrued expenses $ 83,916 $ 118,991

NET LOSS PER COMMON SHARE

NET LOSS PER COMMON SHARE	9 Months Ended
	Sep. 30, 2024
Earnings Per Share [Abstract]
NET LOSS PER COMMON SHARE	NET LOSS PER COMMON SHARE Basic and diluted net income (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. In accordance with ASC 260, Earnings per Share , if a company had a discontinued operation, the company uses income from continuing operations, adjusted for preferred dividend and similar adjustments, as its control number to determine whether potential common shares are dilutive. As discussed in Note 17, as part of its February 2023 underwritten public offering, the Company issued and sold pre-funded warrants to purchase 1.25 million shares of its common stock at a price to the public of $20.9999 per pre-funded warrant. The pre-funded warrants were immediately exercisable upon issuance, and were exercised in the third quarter of 2024, resulting in the issuance of 1.25 million shares of the Company's common stock. Due to the nominal exercise price of the pre-funded warrants and the lack of any contingencies to exercise, the shares underlying the pre-funded warrants have been included in the calculation of basic net loss per common share since the date the warrants were issued. 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 income (loss) per share is calculated as follows (net loss amounts are stated in thousands) : Three Months Ended September 30, 2024 2023 Shares Net Income (loss) EPS Shares Net Income (loss) EPS Continuing operations 77,779,379 $ (54,752) $ (0.70) 76,305,603 $ (89,241) $ (1.17) Discontinued operations 77,779,379 (59) — 76,305,603 239,976 3.14 Basic and diluted loss per share 77,779,379 $ (54,811) $ (0.70) 76,305,603 $ 150,735 $ 1.97 Nine Months Ended September 30, 2024 2023 Shares Net Income (loss) EPS Shares Net Income (loss) EPS Continuing operations 77,473,161 $ (260,362) $ (3.36) 73,523,620 $ (287,737) $ (3.91) Discontinued operations 77,473,161 (919) (0.01) 73,523,620 266,511 3.62 Basic and diluted loss per share 77,473,161 $ (261,281) $ (3.37) 73,523,620 $ (21,226) $ (0.29) The following common stock equivalents have been excluded because they were anti-dilutive: Three Months Ended September 30, Nine Months Ended September 30, 2024 2023 2024 2023 Convertible debt 11,697,953 11,697,952 11,697,953 11,697,952 Options 10,235,428 10,533,703 10,759,644 10,600,069 Restricted stock 3,809,142 3,504,350 3,828,261 3,482,829 Total anti-dilutive shares 25,742,523 25,736,005 26,285,858 25,780,850

COMMITMENTS AND CONTINGENCIES

COMMITMENTS AND CONTINGENCIES	9 Months Ended
	Sep. 30, 2024
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. Contingencies In November 2020, the Company completed the acquisition of Orphan Technologies Limited (“Orphan”), including Orphan’s rare metabolic disorder drug pegtibatinase. The Company acquired Orphan by purchasing all of the outstanding shares. 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 IPR&D expense on the date of acquisition. In accordance with ASC 450, Contingencies , contingent cash payments will be accrued for when it is probable that a liability has been incurred and the amount can be reasonably estimated. In March 2024, the Company recognized $65.2 million in IPR&D expense upon the achievement of a development milestone, which was paid during the second quarter of 2024 and recorded within investing activities in the Consolidated Statements of Cash Flows. 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 in which the outcome, 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.

SHARE-BASED COMPENSATION

SHARE-BASED COMPENSATION	9 Months Ended
	Sep. 30, 2024
Equity [Abstract]
SHARE-BASED COMPENSATION	SHARE-BASED COMPENSATION Stock Options The following table summarizes stock option activity during the nine months ended September 30, 2024: Shares Underlying Options Weighted Average Exercise Price Weighted Average Remaining Contractual Life (years) Aggregate Intrinsic Value (in thousands) Outstanding at December 31, 2023 10,211,353 $ 21.52 4.91 $ — Granted 1,524,300 8.63 — — Exercised (52,000) 10.99 — 159,600 Forfeited/canceled (1,920,196) 19.68 — — Outstanding at September 30, 2024 9,763,457 $ 19.92 5.50 $ 299,450 Vested and expected to vest at September 30, 2024 9,763,457 $ 19.92 5.50 $ 299,450 At September 30, 2024, unamortized stock compensation for stock options was $19.7 million, with a remaining weighted-average recognition period of 2.5 years. At September 30, 2024, outstanding options to purchase 7.2 million shares of common stock were exercisable with a weighted-average exercise price per share of $21.61. During the nine months ended September 30, 2024, the Company had forfeitures of 1.9 million shares due to a combination of employee turnover, the December 2023 reorganization and the August 2023 divestiture of the bile acid business. Restricted Stock Units Service Based Restricted Stock Units The following table summarizes the Company’s service based restricted stock unit activity during the nine months ended September 30, 2024: Number of Restricted Stock Units Weighted Average Grant Date Fair Value Unvested at December 31, 2023 2,874,046 $ 22.97 Granted 2,040,625 8.90 Vested (934,175) 23.11 Forfeited/canceled (372,070) 19.1 Unvested at September 30, 2024 3,608,426 $ 15.38 At September 30, 2024, unamortized stock compensation for service based restricted stock units was $41.4 million, with a remaining weighted-average recognition period of 2.5 years. Performance Based Restricted Stock Units The following table summarizes the Company’s performance based restricted stock unit activity during the nine months ended September 30, 2024: Number of Restricted Stock Units Weighted Average Grant Date Fair Value Unvested at December 31, 2023 175,458 $ 25.61 Granted 107,000 8.93 Vested — — Forfeited/canceled (66,250) 22.40 Unvested at September 30, 2024 216,208 $ 18.34 At September 30, 2024, unamortized stock compensation for performance based restricted stock units was $1.3 million, with a remaining weighted-average recognition period of 1.2 years. Share-Based Compensation Total share-based compensation presented in the Consolidated Statements of Stockholders' Equity includes both continuing operations and discontinued operations. The following table sets forth share-based compensation for continuing operations for the three and nine months ended September 30, 2024 and 2023 ( in thousands ): Three Months Ended September 30, Nine Months Ended September 30, 2024 2023 2024 2023 Research and development $ 3,321 $ 4,372 $ 10,752 $ 13,372 Selling, general and administrative 4,700 6,949 16,946 22,730 Total share-based compensation $ 8,021 $ 11,321 $ 27,698 $ 36,102

INVENTORY

INVENTORY	9 Months Ended
	Sep. 30, 2024
Inventory Disclosure [Abstract]
INVENTORY	INVENTORY Inventory consisted of the following at September 30, 2024 and December 31, 2023 ( in thousands ): September 30, 2024 December 31, 2023 Raw materials $ 33,129 $ 33,790 Work in process 5,560 4,727 Finished goods 4,189 2,387 Total inventory $ 42,878 $ 40,904 Classified as: Inventory $ 6,356 $ 9,410 Long-term inventory 36,522 31,494 Total inventory $ 42,878 $ 40,904 The balance classified as long-term inventory consists of raw materials and work in process for FILSPARI as of September 30, 2024 and December 31, 2023. The balance classified as long-term inventory as of September 30, 2024 also consists of raw materials and finished goods for tiopronin products. The Company maintains levels of these inventories beyond a one-year production plan to limit exposure to potential supply disruption. Such inventories are classified as long-term.

ACCOUNTS RECEIVABLE

ACCOUNTS RECEIVABLE	9 Months Ended
	Sep. 30, 2024
Receivables [Abstract]
ACCOUNTS RECEIVABLE	ACCOUNTS RECEIVABLE Accounts receivable, net of reserves for prompt pay discounts and expected credit losses, was $25.2 million and $21.2 million at September 30, 2024 and December 31, 2023, respectively. The total reserves for both periods were immaterial.

EQUITY OFFERINGS

EQUITY OFFERINGS	9 Months Ended
	Sep. 30, 2024
Equity [Abstract]
EQUITY OFFERINGS	EQUITY OFFERINGS Underwritten Public Offering of Common Stock In February 2023, the Company sold an aggregate of approximately 9.7 million shares of its common stock and pre-funded warrants to purchase 1.25 million shares of its common stock in an underwritten public offering, at a price to the public of $21.00 per share of common stock and $20.9999 per pre-funded warrant. The pre-funded warrants are exercisable immediately, subject to certain beneficial ownership limitations which can be modified by the respective holders with at least 61 days' notice, and are exercisable for one share of the Company's common stock. The exercise price of each pre-funded warrant is $0.0001 per share of common stock. The net proceeds to the Company from the offering, after deducting the underwriting discounts and offering expenses, were approximately $215.8 million. The pre-funded warrants were classified as a component of permanent stockholders' equity within additional paid-in capital and were recorded at the issuance date using a relative fair value allocation method. The pre-funded warrants are equity classified because they (i) are freestanding financial instruments that are legally detachable and separately exercisable from the equity instruments, (ii) are immediately exercisable, (iii) do not embody an obligation for the Company to repurchase its shares, (iv) permit the holders to receive a fixed number of shares of common stock upon exercise, (v) are indexed to the Company's common stock and (vi) meet the equity classification criteria. In addition, such pre-funded warrants do not provide any guarantee of value or return. The Company valued the pre-funded warrants at issuance, concluding that their sale price approximated their fair value, and allocated the aggregate net proceeds from the sale proportionately to the common stock and prefunded warrants, including approximately $24.6 million allocated to the pre-funded warrants and recorded as a component of additional paid-in capital. The pre-funded warrants were exercised in the third quarter of 2024, resulting in the issuance of 1.25 million shares of the Company's common stock. 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 No. 333-227182)(the "Prior Registration Statement"). An additional $51.9 million were sold under the Company's prior registration statement on Form S-3 (Registration Statement No. 333-259311), which included $20.1 million in the year ended December 31, 2022. The Company did not sell any shares under the ATM Agreement during the year ended December 31, 2023 or during the nine months ended September 30, 2024. The offering pursuant to the ATM Agreement terminated upon the expiration of the Prior Registration Statement on September 3, 2024.

DIVESTITURES

DIVESTITURES	9 Months Ended
	Sep. 30, 2024
Discontinued Operations and Disposal Groups [Abstract]
DIVESTITURES	DIVESTITURES Discontinued Operations Sale of Bile Acid Product Portfolio On August 31, 2023, the Company closed the sale of its bile acid business to Mirum Pharmaceuticals pursuant to the terms of the Purchase Agreement dated July 16, 2023 between the Company and Mirum. The assets sold consisted of substantially all of the assets primarily related to the Company’s business of development, manufacture (including synthesis, formulation, finishing or packaging) and commercialization of the products, Chenodal and Cholbam (also known as Kolbam). In connection with the Closing, the Company received an upfront cash payment of $210.0 million. Pursuant to the Purchase Agreement, after the Closing, the Company is eligible to receive up to $235.0 million upon the achievement of certain milestones based on specified amounts of annual net sales (tiered from $125.0 million to $500.0 million) of the Products. The Company will recognize the contingent consideration receivable in earnings when the target annual sales for the milestones are met and the contingency is resolved. The Company's sale of the bile acid business resulted in a gain, net of tax, of $226.0 million, which was recognized in 2023. The net gain consists of net consideration, including the upfront payment and the deduction of investment banker fees owed upon Closing, plus the derecognition of the carrying value of the net liabilities included in the transaction and the immaterial tax due on the sale. The Company and Mirum have also entered into a transition services agreement ("TSA") pursuant to which the Company has agreed to perform certain services for a period of time following the Closing, with respect to Mirum’s use and operation of the assets purchased in the Purchase Agreement. The TSA is designed to ensure and facilitate an orderly transfer of business operations, and the consideration to be received by the Company primarily consists of cost reimbursement. For the three and nine months ended September 30, 2024, the Company recognized less than $0.1 million and $0.5 million, respectively, under the TSA, included in continuing operations within other income (expense), net. The uncollected balance is included in accounts receivable of the Consolidated Balance Sheets. As part of the TSA, the Company is collecting certain receivables related to purchased assets for a period of time and remitting them to Mirum. The transition services accrual as of September 30, 2024 was $1.2 million, and is included in accrued expenses in the accompanying Consolidated Balance Sheets. TSA services provided by the Company are substantially complete as of September 30, 2024. The Company determined that the divestiture represents a strategic shift that will have a major effect on the Company's operations and financial results, and has therefore reflected the bile acid business as a discontinued operation for all periods presented. Results of discontinued operations are as follows ( in thousands ): Three Months Ended September 30, Nine Months Ended September 30, 2024 2023 2024 2023 Net product sales $ (103) $ 12,592 $ (554) $ 66,198 Total revenue (103) 12,592 (554) 66,198 Operating expenses: Cost of goods sold — 381 (10) 1,899 Research and development 13 1,491 247 6,161 Selling, general and administrative (57) 5,723 128 17,851 Change in fair value of contingent consideration — (9,069) — (473) Total operating expenses (44) (1,474) 365 25,438 Operating (loss) income (59) 14,066 (919) 40,760 Other income (expenses), net: Interest expense — (32) — (191) Gain on disposal of discontinued operations, net of tax — 225,942 — 225,942 Total other expense, net — 225,910 — 225,751 Net (loss) income from discontinued operations $ (59) $ 239,976 $ (919) $ 266,511 The Company held no assets or liabilities of discontinued operations as of September 30, 2024 and December 31, 2023.

Pay vs Performance Disclosure

Pay vs Performance Disclosure - USD ($) $ in Thousands	3 Months Ended		9 Months Ended
	Sep. 30, 2024	Sep. 30, 2023	Sep. 30, 2024	Sep. 30, 2023
Pay vs Performance Disclosure
Net (loss) income	$ (54,811)	$ 150,735	$ (261,281)	$ (21,226)

Insider Trading Arrangements

Insider Trading Arrangements	3 Months Ended	9 Months Ended
	Sep. 30, 2024 shares	Sep. 30, 2024 shares
Trading Arrangements, by Individual
Material Terms of Trading Arrangement		During the fiscal quarter ended September 30, 2024, our directors and/or officers (as defined in Rule 16a-1(f) under the Exchange Act) adopted, modified or terminated the “Rule 10b5-1 trading arrangements” or “non-Rule 10b5-1 trading arrangements,” as those terms are defined in Regulation S-K, Item 408, set forth below: Trading Arrangements Adopted: Name & Title Date Adopted Character of Trading Arrangement (1) Aggregate Number of Shares of Common Stock to be Sold Pursuant to Trading Arrangement Expiration Date (2) Roy D. Baynes, member of our Board of Directors August 16, 2024 Rule 10b5-1 Trading Arrangement Up to 26,000 shares (3) October 31, 2025 Sandra Calvin, SVP and Chief Accounting Officer September 9, 2024 Rule 10b5-1 Trading Arrangement Up to 86,187 shares (4) September 9, 2025 1 Each trading arrangement marked as a “Rule 10b5-1 Trading Arrangement” is intended to satisfy the affirmative defense of Rule 10b5-1(c) under the Exchange Act (the “Rule”). 2 Each trading arrangement permits transactions through and including the earlier to occur of (a) the completion of all sales and (b) the date listed in the table. Each trading arrangement marked as a “Rule 10b5-1 Trading Arrangement” only permits transactions upon expiration of the applicable mandatory cooling-off period under the Rule. 3 Consists of shares of underlying stock options expiring in 2026 and 2027. 4 Includes shares underlying stock options expiring in January 2025.
Non-Rule 10b5-1 Arrangement Adopted	false
Rule 10b5-1 Arrangement Terminated	false
Non-Rule 10b5-1 Arrangement Terminated	false
Roy D. Baynes [Member]
Trading Arrangements, by Individual
Name	Roy D. Baynes
Title	member of our Board of Directors
Rule 10b5-1 Arrangement Adopted	true
Adoption Date	August 16, 2024
Expiration Date	October 31, 2025
Arrangement Duration	441 days
Aggregate Available	26,000	26,000
Sandra Calvin [Member]
Trading Arrangements, by Individual
Name	Sandra Calvin
Title	SVP and Chief Accounting Officer
Rule 10b5-1 Arrangement Adopted	true
Adoption Date	September 9, 2024
Expiration Date	September 9, 2025
Arrangement Duration	365 days
Aggregate Available	86,187	86,187

BASIS OF PRESENTATION AND SIG_2

BASIS OF PRESENTATION AND SIGNIFICANT ACCCOUNTING POLICIES (Policies)	9 Months Ended
	Sep. 30, 2024
Accounting Policies [Abstract]
Principles of Consolidation	Principles of Consolidation The unaudited 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 6 for further discussion of variable interest entities (“VIE”) that the Company consolidates.
Revenue Recognition	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 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, 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 nine months ended September 30, 2024 and 2023, adjustments to net product revenue related to performance obligations satisfied in previous periods were $0.5 million and $0.4 million, respectively. 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 an estimated allocation of payers and the government-mandated discounts applicable to government-funded programs. Rebate discounts are included in accrued expenses in the accompanying Consolidated Balance Sheets. Commercial Rebates: The Company calculates the rebates it incurs according to any 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 for applicable products. Rebate discounts are included in accrued expenses 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. Other Fees: The Company pays service fees to certain customers based on a contractually fixed percentage of the wholesale acquisition cost (WAC) and fees for data. Other fees are recorded as an offset to revenue based on contractual terms at the time revenue from the sale is recognized. 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. Historically, returns have been immaterial. 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 estimate of claims and the estimated cost per claim associated with product that has been recognized as revenue.
Collaborative Arrangements	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 judgment 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. Revenue from collaboration and licensing agreements may also include sales of inventory, at cost plus a margin, which is recorded in license and collaboration revenue. The Company utilizes significant judgment 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 judgment 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.
Cost of goods sold	Cost of goods sold 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. Cost of goods sold also includes the cost of goods sold under the Company's license and collaboration agreements, which currently consists of the sale of active pharmaceutical ingredients to the Company's collaboration partners, at cost or at cost plus a margin.
Capitalization of Inventory Costs	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. Prior to the February 2023 FDA accelerated approval of FILSPARI (sparsentan), the Company expensed the production of active pharmaceutical ingredients purchased to support the commercial launch of FILSPARI, in research and development expenses. For the three and nine months ended September 30, 2024 and 2023, sales of FILSPARI primarily consisted of zero-cost inventories. As of September 30, 2024, the Company had approximately $2.6 million of zero-cost inventory. The Company expects to continue to record zero cost of goods sold on the sale of previously expensed inventories through at least 2025. The Company began capitalizing inventory costs associated with FILSPARI following the February 2023 accelerated approval.
Research and Development Expenses	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, costs to develop drug materials and delivery devices, costs to 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.
Clinical Trial Expenses	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 two Phase 2 clinical trials and four 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.
Intangible Assets with Cost Accumulation Model	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 ("Thiola Intangible"). The additional cost basis is subsequently amortized over the remaining useful life. In the second quarter of 2023, the Company reduced the estimated useful life of the Thiola Intangible to better reflect the pattern of projected future cash flows. The change in estimated useful life was accounted for as a change in accounting estimate with the remaining carrying amounts of the Thiola Intangible being amortized prospectively over the new useful life. 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 September 30, 2024 as such royalties are not yet probable and estimable.
Variable Interest Entity	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 judgments 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.
Equity Securities	Equity Securities The Company applies the equity method of accounting for investments when it has significant influence, but no controlling interest in the investee. Judgment regarding the level of influence over each equity method investment includes key factors such as ownership interest, representation on the board of directors, participation in joint steering committees and material intercompany transactions. The Company evaluates any basis difference between the carrying value and fair value of the Company's proportionate share of the investee's net assets. Basis differences relating to in-process research and development (IPR&D) are expensed when the investee is not considered a business as defined in ASC 805, Business Combinations, due to substantially all of the estimated fair value of the gross assets being concentrated in a group of similar IPR&D assets with no alternative future use. For the nine months ending September 30, 2024, the Company recognized $3.4 million in other expense in the Company's consolidated statements of operations for these basis adjustments. The equity method investment's carrying value was reduced to zero as the Company's proportionate share of the basis difference exceeded the carrying value. See Note 6 for further discussion. Investments accounted for using the equity method may be reported on a lag of up to three months if the financial statements of the investee are not available in sufficient time for the Company to apply the equity method as of the current reporting date.
Discontinued Operations	Discontinued Operations
Restructuring	Restructuring Restructuring charges consist primarily of employee severance, one-time termination benefits related to the reduction of its workforce, and other costs. Liabilities for costs associated with a restructuring activity are recognized when the liability is incurred and are measured at fair value. One-time termination benefits are expensed at the date the entity notifies the employee, unless the employee must provide future service, in which case the benefits are expensed ratably over the service period. Termination benefits are calculated based on regional benefit practices and local statutory requirements.
Recently Adopted and Issued Accounting Pronouncements	Recently Adopted Accounting Pronouncements In November 2023, the FASB issued ASU No. 2023-07, Improvements to Reportable Segment Disclosure. The FASB amended the guidance in ASC 280, Segment Reporting ("ASC 280"), to require a public entity to disclose significant segment expenses and other segment items on an annual and interim basis and to provide in interim periods all disclosures about a reportable segment's profit or loss and assets that are currently required annually. Public entities with a single reportable segment are required to provide the new disclosures and all the disclosures required under ASC 280. The guidance is applied retrospectively to all periods presented in financial statements, unless it is impracticable. This new guidance is effective for public business entities for annual periods beginning after December 15, 2023, and for interim periods beginning after December 15, 2024. The Company plans to adopt this new standard for its Annual Report on Form 10-K for the fiscal year ended December 31, 2024 . While this accounting standard will increase disclosures, it will not have a material impact on the Company's financial statements. 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. In December 2023, the FASB issued ASU No. 2023-09, Improvements to Income Tax Disclosures. This new standard does not change accounting for income taxes but requires new disclosures focusing on two areas, the effective rate reconciliation and taxes paid. This new standard is effective for public business entities for annual periods beginning after December 15, 2024. Early adoption is permitted. The Company expects to adopt this new standard beginning in fiscal 2025 when it becomes effective.
Net Loss Per Common Share	Basic and diluted net income (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. In accordance with ASC 260, Earnings per Share , if a company had a discontinued operation, the company uses income from continuing operations, adjusted for preferred dividend and similar adjustments, as its control number to determine whether potential common shares are dilutive. As discussed in Note 17, as part of its February 2023 underwritten public offering, the Company issued and sold pre-funded warrants to purchase 1.25 million shares of its common stock at a price to the public of $20.9999 per pre-funded warrant. The pre-funded warrants were immediately exercisable upon issuance, and were exercised in the third quarter of 2024, resulting in the issuance of 1.25 million shares of the Company's common stock. Due to the nominal exercise price of the pre-funded warrants and the lack of any contingencies to exercise, the shares underlying the pre-funded warrants have been included in the calculation of basic net loss per common share since the date the warrants were issued. 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.
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.

BASIS OF PRESENTATION AND SIG_3

BASIS OF PRESENTATION AND SIGNIFICANT ACCCOUNTING POLICIES (Tables)	9 Months Ended
	Sep. 30, 2024
Accounting Policies [Abstract]
Summary of Cost and Goods Sold	The following table summarizes cost of goods sold for the three and nine months ended September 30, 2024 and 2023 ( in thousands ): Three Months Ended September 30, Nine Months Ended September 30, 2024 2023 2024 2023 Cost of goods sold - product sales $ 1,626 $ 1,289 $ 5,059 $ 3,842 Cost of goods sold - license and collaboration — — 132 3,044 Total cost of goods sold $ 1,626 $ 1,289 $ 5,191 $ 6,886
Summary of Restructuring and Related Costs	The following table sets forth a summary of changes in accrued restructuring costs for the nine months ended September 30, 2024 and 2023 ( in thousands ): Restructuring 2024 2023 Liability balance at January 1, $ 11,421 $ — Restructuring expenses 1,035 — Payments (12,132) — Foreign currency impact 34 — Liability balance at September 30, $ 358 $ —

REVENUE RECOGNITION (Tables)

REVENUE RECOGNITION (Tables)	9 Months Ended
	Sep. 30, 2024
Revenue from Contract with Customer [Abstract]
Summary of Net Product Revenue	The following table summarizes net product sales for the three and nine months ended September 30, 2024 and 2023 (in thousands): Three Months Ended September 30, Nine Months Ended September 30, 2024 2023 2024 2023 FILSPARI $ 35,619 $ 8,044 $ 82,578 $ 14,509 Tiopronin products 25,382 25,888 70,583 73,112 Total net product sales $ 61,001 $ 33,932 $ 153,161 $ 87,621

MARKETABLE DEBT SECURITIES (Tab

MARKETABLE DEBT SECURITIES (Tables)	9 Months Ended
	Sep. 30, 2024
Investments, Debt and Equity Securities [Abstract]
Summary of Marketable Debt Securities	Marketable debt securities consisted of the following ( in thousands ): September 30, 2024 December 31, 2023 Marketable debt securities: Commercial paper $ — $ 34,458 Corporate debt securities 193,468 368,323 Securities of government sponsored entities 47,562 105,894 Total available-for-sale marketable debt securities $ 241,030 $ 508,675
Summary of Short-term Marketable Debt Securities Classified as Available-for-sale	The following is a summary of short-term marketable debt securities classified as available-for-sale as of September 30, 2024 ( in thousands ): Remaining Contractual Maturity Amortized Cost Unrealized Gains Unrealized Losses Aggregate Estimated Fair Value Marketable debt securities: Corporate debt securities Less than 1 $ 170,739 $ 663 $ (18) $ 171,384 Securities of government-sponsored entities Less than 1 47,523 50 (11) 47,562 Total maturity less than 1 year 218,262 713 (29) 218,946 Corporate debt securities 1 to 2 21,815 269 — 22,084 Total maturity 1 to 2 years 21,815 269 — 22,084 Total available-for-sale marketable debt securities $ 240,077 $ 982 $ (29) $ 241,030 The following is a summary of short-term marketable debt securities classified as available-for-sale as of December 31, 2023 ( in thousands ): Remaining Contractual Maturity Amortized Cost Unrealized Gains Unrealized Losses Aggregate Estimated Fair Value Marketable debt securities: Commercial paper Less than 1 $ 34,450 $ 25 $ (17) $ 34,458 Corporate debt securities Less than 1 133,463 29 (408) 133,084 Securities of government-sponsored entities Less than 1 81,334 36 (274) 81,096 Total maturity less than 1 year 249,247 90 (699) 248,638 Corporate debt securities 1 to 2 233,969 1,444 (174) 235,239 Securities of government-sponsored entities 1 to 2 24,718 106 (26) 24,798 Total maturity 1 to 2 years 258,687 1,550 (200) 260,037 Total available-for-sale securities $ 507,934 $ 1,640 $ (899) $ 508,675
Summary of Marketable Debt Securities in an 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 September 30, 2024 ( 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 Corporate debt securities $ — $ — $ 38,602 $ 18 $ 38,602 $ 18 Securities of government-sponsored entities — — 18,836 11 18,836 11 Total $ — $ — $ 57,438 $ 29 $ 57,438 $ 29 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, 2023 ( 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 $ 24,798 $ 17 $ — $ — $ 24,798 $ 17 Corporate debt securities 140,802 405 28,775 177 169,577 582 Securities of government-sponsored entities 61,933 217 12,540 83 74,473 300 Total $ 227,533 $ 639 $ 41,315 $ 260 $ 268,848 $ 899

LEASES (Tables)

LEASES (Tables)	9 Months Ended
	Sep. 30, 2024
Leases [Abstract]
Summary of Future Minimum Rental Commitments	The following is a schedule of the future minimum rental commitments for the Company's operating leases reconciled to the lease liability and ROU asset as of September 30, 2024 ( in thousands ): September 30, 2024 2024 (remaining three months) $ 1,648 2025 6,673 2026 6,889 2027 7,064 2028 4,781 Total undiscounted future minimum payments 27,055 Present value discount (3,177) Total lease liability 23,878 Unamortized lease incentives (3,853) Cash payments in excess of straight-line lease expense (4,363) Total ROU asset $ 15,662
Summary 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: September 30, 2024 December 31, 2023 Weighted-average remaining lease term in years 3.9 4.7 Weighted-average discount rate 6.48 % 6.48 %

FAIR VALUE MEASUREMENTS (Tables

FAIR VALUE MEASUREMENTS (Tables)	9 Months Ended
	Sep. 30, 2024
Fair Value Disclosures [Abstract]
Summary of Fair Value on a Recurring Basis	The following table presents the Company’s assets, measured and recognized at fair value on a recurring basis, classified under the appropriate level of the fair value hierarchy as of September 30, 2024 ( in thousands ): As of September 30, 2024 Total carrying and estimated fair value Quoted prices in active markets Significant other observable inputs (Level 2) Significant unobservable inputs (Level 3) Assets: Cash and cash equivalents $ 36,409 $ 36,409 $ — $ — Marketable debt securities, available-for-sale 241,030 — 241,030 — Total $ 277,439 $ 36,409 $ 241,030 $ — The following table presents the Company’s assets, measured and recognized at fair value on a recurring basis, classified under the appropriate level of the fair value hierarchy as of December 31, 2023 ( in thousands ): As of December 31, 2023 Total carrying and estimated fair value Quoted prices in active markets Significant other observable inputs (Level 2) Significant unobservable inputs (Level 3) Assets: Cash and cash equivalents $ 58,176 $ 58,176 $ — $ — Marketable debt securities, available-for-sale 508,675 — 508,675 — Total $ 566,851 $ 58,176 $ 508,675 $ —

INTANGIBLE ASSETS (Tables)

INTANGIBLE ASSETS (Tables)	9 Months Ended
	Sep. 30, 2024
Goodwill and Intangible Assets Disclosure [Abstract]
Summary of Finite-lived Amortizable Intangible Assets	The following table sets forth amortizable intangible assets as of September 30, 2024 and December 31, 2023 ( in thousands ): September 30, 2024 December 31, 2023 Finite-lived intangible assets $ 212,866 $ 183,037 Less: accumulated amortization (109,408) (79,347) Net carrying value $ 103,458 $ 103,690
Summary of Amortization Expense	The following table summarizes amortization expense for the three and nine months ended September 30, 2024 and 2023 ( in thousands ): Three Months Ended September 30, Nine Months Ended September 30, 2024 2023 2024 2023 Research and development $ — $ 2,447 $ — $ 7,261 Selling, general and administrative 10,812 8,461 30,061 20,097 Total amortization expense $ 10,812 $ 10,908 $ 30,061 $ 27,358

CONVERTIBLE NOTES PAYABLE (Tabl

CONVERTIBLE NOTES PAYABLE (Tables)	9 Months Ended
	Sep. 30, 2024
Debt Disclosure [Abstract]
Summary of Long-term Debt Instruments	The composition of the Company’s convertible senior notes are as follows ( in thousands ): September 30, 2024 December 31, 2023 2.25% convertible senior notes due 2029 $ 316,250 $ 316,250 2.50% convertible senior notes due 2025 68,904 68,904 Unamortized debt issuance costs - 2.25% convertible senior notes due 2029 (6,293) (7,348) Unamortized debt issuance costs - 2.50% convertible senior notes due 2025 (305) (543) Total convertible senior notes, net of unamortized debt discount and debt issuance costs $ 378,556 $ 377,263 The following table sets forth total interest expense recognized related to the 2025 and 2029 Notes ( in thousands ): Three Months Ended September 30, Nine Months Ended September 30, 2024 2023 2024 2023 Contractual interest expense $ 2,210 $ 2,210 $ 6,629 $ 6,629 Amortization of debt issuance costs 432 430 1,293 1,288 Total interest expense for the 2025 and 2029 Notes $ 2,642 $ 2,640 $ 7,922 $ 7,917

ACCRUED EXPENSES (Tables)

ACCRUED EXPENSES (Tables)	9 Months Ended
	Sep. 30, 2024
Payables and Accruals [Abstract]
Summary of Accrued Expenses	Accrued expenses at September 30, 2024 and December 31, 2023 consisted of the following ( in thousands ): September 30, 2024 December 31, 2023 Compensation related costs $ 28,295 $ 29,908 Research and development 17,065 26,006 Accrued royalties 10,168 6,991 Sales discounts, rebates, and allowances 9,920 13,730 Selling, general and administrative 7,607 7,190 French rebate accrual 6,292 5,377 Transition services accrual 1,239 12,282 Accrued restructuring costs 358 11,421 Miscellaneous accrued expenses 2,972 6,086 Total accrued expenses $ 83,916 $ 118,991

NET LOSS PER COMMON SHARE (Tabl

NET LOSS PER COMMON SHARE (Tables)	9 Months Ended
	Sep. 30, 2024
Earnings Per Share [Abstract]
Summary of Basic and Diluted Net Income (Loss) Per Share	Basic and diluted net income (loss) per share is calculated as follows (net loss amounts are stated in thousands) : Three Months Ended September 30, 2024 2023 Shares Net Income (loss) EPS Shares Net Income (loss) EPS Continuing operations 77,779,379 $ (54,752) $ (0.70) 76,305,603 $ (89,241) $ (1.17) Discontinued operations 77,779,379 (59) — 76,305,603 239,976 3.14 Basic and diluted loss per share 77,779,379 $ (54,811) $ (0.70) 76,305,603 $ 150,735 $ 1.97 Nine Months Ended September 30, 2024 2023 Shares Net Income (loss) EPS Shares Net Income (loss) EPS Continuing operations 77,473,161 $ (260,362) $ (3.36) 73,523,620 $ (287,737) $ (3.91) Discontinued operations 77,473,161 (919) (0.01) 73,523,620 266,511 3.62 Basic and diluted loss per share 77,473,161 $ (261,281) $ (3.37) 73,523,620 $ (21,226) $ (0.29)
Summary of Common Stock Options, Convertible Debt and Restricted Stock Units Anti-dilutive	The following common stock equivalents have been excluded because they were anti-dilutive: Three Months Ended September 30, Nine Months Ended September 30, 2024 2023 2024 2023 Convertible debt 11,697,953 11,697,952 11,697,953 11,697,952 Options 10,235,428 10,533,703 10,759,644 10,600,069 Restricted stock 3,809,142 3,504,350 3,828,261 3,482,829 Total anti-dilutive shares 25,742,523 25,736,005 26,285,858 25,780,850

SHARE-BASED COMPENSATION (Table

SHARE-BASED COMPENSATION (Tables)	9 Months Ended
	Sep. 30, 2024
Equity [Abstract]
Summary of Stock Option Issuances and Balances Outstanding	The following table summarizes stock option activity during the nine months ended September 30, 2024: Shares Underlying Options Weighted Average Exercise Price Weighted Average Remaining Contractual Life (years) Aggregate Intrinsic Value (in thousands) Outstanding at December 31, 2023 10,211,353 $ 21.52 4.91 $ — Granted 1,524,300 8.63 — — Exercised (52,000) 10.99 — 159,600 Forfeited/canceled (1,920,196) 19.68 — — Outstanding at September 30, 2024 9,763,457 $ 19.92 5.50 $ 299,450 Vested and expected to vest at September 30, 2024 9,763,457 $ 19.92 5.50 $ 299,450
Summary of Service Based Restricted Stock Activity	The following table summarizes the Company’s service based restricted stock unit activity during the nine months ended September 30, 2024: Number of Restricted Stock Units Weighted Average Grant Date Fair Value Unvested at December 31, 2023 2,874,046 $ 22.97 Granted 2,040,625 8.90 Vested (934,175) 23.11 Forfeited/canceled (372,070) 19.1 Unvested at September 30, 2024 3,608,426 $ 15.38
Summary of Performance Based Restricted Stock Activity	The following table summarizes the Company’s performance based restricted stock unit activity during the nine months ended September 30, 2024: Number of Restricted Stock Units Weighted Average Grant Date Fair Value Unvested at December 31, 2023 175,458 $ 25.61 Granted 107,000 8.93 Vested — — Forfeited/canceled (66,250) 22.40 Unvested at September 30, 2024 216,208 $ 18.34
Summary of Share Based Compensation Expenses	The following table sets forth share-based compensation for continuing operations for the three and nine months ended September 30, 2024 and 2023 ( in thousands ): Three Months Ended September 30, Nine Months Ended September 30, 2024 2023 2024 2023 Research and development $ 3,321 $ 4,372 $ 10,752 $ 13,372 Selling, general and administrative 4,700 6,949 16,946 22,730 Total share-based compensation $ 8,021 $ 11,321 $ 27,698 $ 36,102

INVENTORY (Tables)

INVENTORY (Tables)	9 Months Ended
	Sep. 30, 2024
Inventory Disclosure [Abstract]
Summary of Inventory	Inventory consisted of the following at September 30, 2024 and December 31, 2023 ( in thousands ): September 30, 2024 December 31, 2023 Raw materials $ 33,129 $ 33,790 Work in process 5,560 4,727 Finished goods 4,189 2,387 Total inventory $ 42,878 $ 40,904 Classified as: Inventory $ 6,356 $ 9,410 Long-term inventory 36,522 31,494 Total inventory $ 42,878 $ 40,904

DIVESTITURES (Tables)

DIVESTITURES (Tables)	9 Months Ended
	Sep. 30, 2024
Discontinued Operations and Disposal Groups [Abstract]
Summary Results, Assets and Liabilities of Discontinued Operations	Results of discontinued operations are as follows ( in thousands ): Three Months Ended September 30, Nine Months Ended September 30, 2024 2023 2024 2023 Net product sales $ (103) $ 12,592 $ (554) $ 66,198 Total revenue (103) 12,592 (554) 66,198 Operating expenses: Cost of goods sold — 381 (10) 1,899 Research and development 13 1,491 247 6,161 Selling, general and administrative (57) 5,723 128 17,851 Change in fair value of contingent consideration — (9,069) — (473) Total operating expenses (44) (1,474) 365 25,438 Operating (loss) income (59) 14,066 (919) 40,760 Other income (expenses), net: Interest expense — (32) — (191) Gain on disposal of discontinued operations, net of tax — 225,942 — 225,942 Total other expense, net — 225,910 — 225,751 Net (loss) income from discontinued operations $ (59) $ 239,976 $ (919) $ 266,511

DESCRIPTION OF BUSINESS (Detail

DESCRIPTION OF BUSINESS (Details) - USD ($) $ in Millions	Aug. 31, 2023	Apr. 30, 2024	Sep. 15, 2021
Vifor Pharma | Collaborative Arrangement
Income Statement, Balance Sheet and Additional Disclosures by Disposal Groups, Including Discontinued Operations [Line Items]
Maximum milestone payments from collaborator			$ 845
Regulatory Milestone | Vifor Pharma | Collaborative Arrangement
Income Statement, Balance Sheet and Additional Disclosures by Disposal Groups, Including Discontinued Operations [Line Items]
Maximum milestone payments from collaborator		$ 17.5
Disposed of by Sale | Bile Acid Product Portfolio
Income Statement, Balance Sheet and Additional Disclosures by Disposal Groups, Including Discontinued Operations [Line Items]
Proceeds from sale of business	$ 210
Consideration receivable on sale of business (up to)	235
Disposed of by Sale | Minimum | Bile Acid Product Portfolio
Income Statement, Balance Sheet and Additional Disclosures by Disposal Groups, Including Discontinued Operations [Line Items]
Milestone thresholds	125
Disposed of by Sale | Maximum | Bile Acid Product Portfolio
Income Statement, Balance Sheet and Additional Disclosures by Disposal Groups, Including Discontinued Operations [Line Items]
Milestone thresholds	$ 500

BASIS OF PRESENTATION AND SIG_4

BASIS OF PRESENTATION AND SIGNIFICANT ACCCOUNTING POLICIES - Summary of Cost and Goods Sold (Details) - USD ($) $ in Thousands	3 Months Ended		9 Months Ended
	Sep. 30, 2024	Sep. 30, 2023	Sep. 30, 2024	Sep. 30, 2023
New Accounting Pronouncements or Change in Accounting Principle [Line Items]
Total cost of goods sold	$ 1,626	$ 1,289	$ 5,191	$ 6,886
Cost of goods sold - product sales
New Accounting Pronouncements or Change in Accounting Principle [Line Items]
Total cost of goods sold	1,626	1,289	5,059	3,842
Cost of goods sold - license and collaboration
New Accounting Pronouncements or Change in Accounting Principle [Line Items]
Total cost of goods sold	$ 0	$ 0	$ 132	$ 3,044

BASIS OF PRESENTATION AND SIG_5

BASIS OF PRESENTATION AND SIGNIFICANT ACCCOUNTING POLICIES - Additional Information (Details)	1 Months Ended	3 Months Ended		9 Months Ended		10 Months Ended
	Mar. 31, 2024 USD ($)	Sep. 30, 2024 USD ($)	Sep. 30, 2023 USD ($)	Sep. 30, 2024 USD ($) clinicalTrial	Sep. 30, 2023 USD ($)	Sep. 30, 2024 USD ($)	Dec. 31, 2023 USD ($)
New Accounting Pronouncements or Change in Accounting Principle [Line Items]
Number of phase 2 clinical trials in process | clinicalTrial				2
Number of phase 3 clinical trials in process | clinicalTrial				4
In-process research and development	$ 65,200,000	$ 0	$ 0	$ 65,205,000	$ 0
Equity method investments		0		0		$ 0
Restructuring costs		100,000		1,000,000		12,400,000
Accrued restructuring costs		358,000		358,000		358,000	$ 11,421,000
Payments				12,132,000	$ 0
Minimum
New Accounting Pronouncements or Change in Accounting Principle [Line Items]
Restructuring and related cost, expected cost							12,000,000
Maximum
New Accounting Pronouncements or Change in Accounting Principle [Line Items]
Restructuring and related cost, expected cost							$ 14,000,000
Equity Method Investments
New Accounting Pronouncements or Change in Accounting Principle [Line Items]
In-process research and development				3,400,000
FILSPARI
New Accounting Pronouncements or Change in Accounting Principle [Line Items]
Zero cost inventory		$ 2,600,000		$ 2,600,000		$ 2,600,000

BASIS OF PRESENTATION AND SIG_6

BASIS OF PRESENTATION AND SIGNIFICANT ACCCOUNTING POLICIES - Summary of Restructuring and Related Costs (Details) - USD ($) $ in Thousands	3 Months Ended		9 Months Ended
	Sep. 30, 2024	Sep. 30, 2023	Sep. 30, 2024	Sep. 30, 2023
Restructuring Reserve [Roll Forward]
Liability balance at January 1,			$ 11,421	$ 0
Restructuring expenses	$ 123	$ 0	1,035	0
Payments			(12,132)	0
Foreign currency impact			34	0
Liability balance at September 30,	$ 358	$ 0	$ 358	$ 0

REVENUE RECOGNITION - Additiona

REVENUE RECOGNITION - Additional Information (Details) $ in Millions	9 Months Ended
	Sep. 30, 2024 USD ($) pharmacy	Sep. 30, 2023 USD ($)
Disaggregation of Revenue [Line Items]
Number of direct-to-patient pharmacies sold to | pharmacy	2
Revenue, performance obligation, description of timing	For FILSPARI, sales are recognized upon delivery of the product to the specialty pharmacies. The Company receives payments from its FILSPARI sales based on terms that are generally 30 days from shipment of the product to the specialty pharmacy. For the Company's tiopronin products, product sales are recognized upon delivery to the patient. The Company receives payments from sales of its other products, 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 obligations satisfied in previous period | $	$ 0.5	$ 0.4
Geographic Concentration Risk | Revenue Benchmark | United States
Disaggregation of Revenue [Line Items]
Concentration risk, percentage	98%

REVENUE RECOGNITION - Summary o

REVENUE RECOGNITION - Summary of Net Product Revenue (Details) - USD ($) $ in Thousands	3 Months Ended		9 Months Ended
	Sep. 30, 2024	Sep. 30, 2023	Sep. 30, 2024	Sep. 30, 2023
Disaggregation of Revenue [Line Items]
Total net product sales	$ 62,898	$ 37,095	$ 158,388	$ 100,179
Net product sales
Disaggregation of Revenue [Line Items]
Total net product sales	61,001	33,932	153,161	87,621
FILSPARI
Disaggregation of Revenue [Line Items]
Total net product sales	35,619	8,044	82,578	14,509
Tiopronin products
Disaggregation of Revenue [Line Items]
Total net product sales	$ 25,382	$ 25,888	$ 70,583	$ 73,112

COLLABORATION AND LICENSE AGR_2

COLLABORATION AND LICENSE AGREEMENTS (Details) $ in Thousands		1 Months Ended	3 Months Ended		9 Months Ended		12 Months Ended
	Sep. 15, 2021 USD ($) performance_obligation	Jan. 31, 2024 USD ($) performance_obligation	Sep. 30, 2024 USD ($)	Sep. 30, 2023 USD ($)	Sep. 30, 2024 USD ($)	Sep. 30, 2023 USD ($)	Dec. 31, 2021 USD ($)	Dec. 31, 2023 USD ($)
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items]
Revenue			$ 62,898	$ 37,095	$ 158,388	$ 100,179
Deferred revenue, current portion			3,799		3,799			$ 7,096
License and collaboration revenue
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items]
Revenue			1,897	3,163	5,227	12,558
Vifor Pharma | License and Collaboration - Clinical Development Activities
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items]
Revenue			1,800	$ 3,200	5,100	9,300
Vifor Pharma | License and Collaboration - Sale of Pharmaceutical Ingredients
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items]
Revenue						$ 3,300
Collaborative Arrangement | Vifor Pharma
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items]
Upfront payment	$ 55,000
Maximum milestone payments from collaborator	$ 845,000
Percentage of royalty on net sales receives up to	40%
Force majeure event period threshold	6 months
Revenue	$ 55,000
Number of performance obligations | performance_obligation	2
Deferred revenue	$ 43,000		3,800		3,800			8,900
Deferred revenue, current portion								$ 7,100
Revenue from royalties			$ 100		$ 100
Collaborative Arrangement | Vifor Pharma | Regulatory and Market Access Milestone
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items]
Maximum milestone payments from collaborator	135,000
Collaborative Arrangement | Vifor Pharma | Sales-based Milestone Payments
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items]
Maximum milestone payments from collaborator	$ 655,000
Collaborative Arrangement | Vifor Pharma | License and collaboration revenue
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items]
Revenue							$ 12,000
Collaborative Arrangement | Renalys Pharma Inc
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items]
Force majeure event period threshold		9 months
Revenue		$ 8,300
Number of performance obligations | performance_obligation		1
Collaborative Arrangement | Renalys Pharma Inc | Regulatory Development And Sales Based Milestone
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items]
Maximum milestone payments from collaborator		$ 120,000

MARKETABLE DEBT SECURITIES - Su

MARKETABLE DEBT SECURITIES - Summary of Marketable Debt Securities (Details) - USD ($) $ in Thousands	Sep. 30, 2024	Dec. 31, 2023
Debt Securities, Available-for-sale [Line Items]
Total available-for-sale marketable debt securities	$ 241,030	$ 508,675
Commercial paper
Debt Securities, Available-for-sale [Line Items]
Total available-for-sale marketable debt securities	0	34,458
Corporate debt securities
Debt Securities, Available-for-sale [Line Items]
Total available-for-sale marketable debt securities	193,468	368,323
Securities of government sponsored entities
Debt Securities, Available-for-sale [Line Items]
Total available-for-sale marketable debt securities	$ 47,562	$ 105,894

MARKETABLE DEBT SECURITIES - _2

MARKETABLE DEBT SECURITIES - Summary of Short-term Marketable Debt Securities Classified as Available-for-sale (Details) - USD ($) $ in Thousands	Sep. 30, 2024	Dec. 31, 2023
Debt Securities, Available-for-sale [Line Items]
Marketable debt securities, available-for-sale, amortized cost basis, current	$ 218,262	$ 249,247
Marketable debt securities, available for sale, unrealized gain, current	713	90
Marketable debt securities, available for sale, unrealized loss, current	(29)	(699)
Marketable debt securities, available-for-sale, current	218,946	248,638
Marketable debt securities, available-for-sale, amortized cost basis, noncurrent	21,815	258,687
Marketable debt securities, available for sale, unrealized gain, noncurrent	269	1,550
Marketable debt securities, available for sale, unrealized loss, noncurrent	0	(200)
Marketable debt securities, available-for-sale, noncurrent	22,084	260,037
Marketable debt securities, available-for-sale, amortized cost	240,077	507,934
Marketable debt securities, available for sale, unrealized gains	982	1,640
Marketable debt securities, available-for-sale, unrealized loss	(29)	(899)
Total available-for-sale marketable debt securities	241,030	508,675
Commercial paper
Debt Securities, Available-for-sale [Line Items]
Marketable debt securities, available-for-sale, amortized cost basis, current		34,450
Marketable debt securities, available for sale, unrealized gain, current		25
Marketable debt securities, available for sale, unrealized loss, current		(17)
Marketable debt securities, available-for-sale, current		34,458
Total available-for-sale marketable debt securities	0	34,458
Corporate debt securities
Debt Securities, Available-for-sale [Line Items]
Marketable debt securities, available-for-sale, amortized cost basis, current	170,739	133,463
Marketable debt securities, available for sale, unrealized gain, current	663	29
Marketable debt securities, available for sale, unrealized loss, current	(18)	(408)
Marketable debt securities, available-for-sale, current	171,384	133,084
Marketable debt securities, available-for-sale, amortized cost basis, noncurrent	21,815	233,969
Marketable debt securities, available for sale, unrealized gain, noncurrent	269	1,444
Marketable debt securities, available for sale, unrealized loss, noncurrent	0	(174)
Marketable debt securities, available-for-sale, noncurrent	22,084	235,239
Total available-for-sale marketable debt securities	193,468	368,323
Securities of government sponsored entities
Debt Securities, Available-for-sale [Line Items]
Marketable debt securities, available-for-sale, amortized cost basis, current	47,523	81,334
Marketable debt securities, available for sale, unrealized gain, current	50	36
Marketable debt securities, available for sale, unrealized loss, current	(11)	(274)
Marketable debt securities, available-for-sale, current	47,562	81,096
Marketable debt securities, available-for-sale, amortized cost basis, noncurrent		24,718
Marketable debt securities, available for sale, unrealized gain, noncurrent		106
Marketable debt securities, available for sale, unrealized loss, noncurrent		(26)
Marketable debt securities, available-for-sale, noncurrent		24,798
Total available-for-sale marketable debt securities	$ 47,562	$ 105,894

MARKETABLE DEBT SECURITIES - Ad

MARKETABLE DEBT SECURITIES - Additional Information (Details) - USD ($) $ in Millions	Sep. 30, 2024	Dec. 31, 2023
Investments, Debt and Equity Securities [Abstract]
Accrued interest receivable	$ 2.5	$ 4.6
Available-for-sale marketable debt securities in an unrealized loss position	$ 57.5	$ 269.7

MARKETABLE DEBT SECURITIES - _3

MARKETABLE DEBT SECURITIES - Summary of Marketable Debt Securities in an Unrealized Loss Position (Details) - USD ($) $ in Thousands	Sep. 30, 2024	Dec. 31, 2023
Debt Securities, Available-for-sale [Line Items]
Less than 12 months, fair value	$ 0	$ 227,533
Less than 12 months, unrealized losses	0	639
12 months or greater, fair value	57,438	41,315
12 months or greater, unrealized losses	29	260
Total, fair value	57,438	268,848
Total, unrealized losses	29	899
Commercial paper
Debt Securities, Available-for-sale [Line Items]
Less than 12 months, fair value		24,798
Less than 12 months, unrealized losses		17
12 months or greater, fair value		0
12 months or greater, unrealized losses		0
Total, fair value		24,798
Total, unrealized losses		17
Corporate debt securities
Debt Securities, Available-for-sale [Line Items]
Less than 12 months, fair value	0	140,802
Less than 12 months, unrealized losses	0	405
12 months or greater, fair value	38,602	28,775
12 months or greater, unrealized losses	18	177
Total, fair value	38,602	169,577
Total, unrealized losses	18	582
Securities of government-sponsored entities
Debt Securities, Available-for-sale [Line Items]
Less than 12 months, fair value	0	61,933
Less than 12 months, unrealized losses	0	217
12 months or greater, fair value	18,836	12,540
12 months or greater, unrealized losses	11	83
Total, fair value	18,836	74,473
Total, unrealized losses	$ 11	$ 300

VARIABLE INTEREST ENTITIES (Det

VARIABLE INTEREST ENTITIES (Details) - USD ($) $ in Thousands	1 Months Ended
	Mar. 31, 2022	Sep. 30, 2024	Dec. 31, 2023
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items]
Other non-current liabilities		$ 16,288	$ 10,320
Other assets		17,119	$ 10,661
Variable Interest Entity, Not Primary Beneficiary
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items]
Other non-current liabilities		8,300
Other assets		$ 0
Collaborative Arrangement | PharmaKrysto, LTD
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%
Agreement termination notice period	60 days
Percentage of research and development to be funded by the company	100%
PharmaKrysto, LTD
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items]
Payment to purchase interest in VIE	$ 600
Percentage ownership purchased	5%
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%

LEASES - Additional Information

LEASES - Additional Information (Details) $ in Thousands	3 Months Ended		9 Months Ended
	Sep. 30, 2024 USD ($) lease	Sep. 30, 2023 USD ($)	Sep. 30, 2024 USD ($) lease	Sep. 30, 2023 USD ($)	Dec. 31, 2023 USD ($)	Dec. 31, 2020 USD ($)
Lessee, Lease, Description [Line Items]
Operating lease right of use assets	$ 15,662		$ 15,662		$ 18,061
Lease liability	$ 23,878		$ 23,878
Operating lease extension term	5 years		5 years
Operating lease expense	$ 1,300	$ 1,200	$ 3,700	$ 3,700
Office Lease 2020
Lessee, Lease, Description [Line Items]
Operating lease right of use assets						$ 34,600
Lease liability						34,500
Lease incentive, tenant improvements						$ 7,900
Kilroy Realty, L.P.
Lessee, Lease, Description [Line Items]
Number of operating leases | lease	2		2
Esprit Investments Limited
Lessee, Lease, Description [Line Items]
Number of operating leases | lease	1		1
Operating lease right of use assets	$ 400		$ 400
Lease liability	$ 400		$ 400
Operating lease extension term	5 years		5 years

LEASES - Summary of Future Mini

LEASES - Summary of Future Minimum Rental Commitments (Details) - USD ($) $ in Thousands	Sep. 30, 2024	Dec. 31, 2023
Leases [Abstract]
2024 (remaining three months)	$ 1,648
2025	6,673
2026	6,889
2027	7,064
2028	4,781
Total undiscounted future minimum payments	27,055
Present value discount	(3,177)
Total lease liability	23,878
Unamortized lease incentives	(3,853)
Cash payments in excess of straight-line lease expense	(4,363)
Total ROU asset	$ 15,662	$ 18,061

LEASES - Summary of Weighted-av

LEASES - Summary of Weighted-average Remaining Lease Term and Discount Rate (Details)	Sep. 30, 2024	Dec. 31, 2023
Leases [Abstract]
Weighted-average remaining lease term in years	3 years 10 months 24 days	4 years 8 months 12 days
Weighted-average discount rate	6.48%	6.48%

FAIR VALUE MEASUREMENTS - Addit

FAIR VALUE MEASUREMENTS - Additional Information (Details) - Senior Notes - USD ($) $ in Millions	Sep. 30, 2024	Dec. 31, 2023
Senior Notes Due 2025
Business Acquisition [Line Items]
Interest rate percentage	2.50%	2.50%
Fair value of convertible debt	$ 66	$ 58.3
Senior Notes Due 2029
Business Acquisition [Line Items]
Interest rate percentage	2.25%	2.25%
Fair value of convertible debt	$ 272.8	$ 212.1

FAIR VALUE MEASUREMENTS - Summa

FAIR VALUE MEASUREMENTS - Summary of Fair Value on a Recurring Basis (Details) - USD ($) $ in Thousands	Sep. 30, 2024	Dec. 31, 2023
Assets:
Cash and cash equivalents	$ 36,409	$ 58,176
Marketable debt securities, available-for-sale	241,030	508,675
Total	277,439	566,851
Quoted prices in active markets (Level 1)
Assets:
Cash and cash equivalents	36,409	58,176
Marketable debt securities, available-for-sale	0	0
Total	36,409	58,176
Significant other observable inputs (Level 2)
Assets:
Cash and cash equivalents	0	0
Marketable debt securities, available-for-sale	241,030	508,675
Total	241,030	508,675
Significant unobservable inputs (Level 3)
Assets:
Cash and cash equivalents	0	0
Marketable debt securities, available-for-sale	0	0
Total	$ 0	$ 0

INTANGIBLE ASSETS - Additional

INTANGIBLE ASSETS - Additional Information (Details) - USD ($) $ in Thousands	3 Months Ended	9 Months Ended		153 Months Ended
	Sep. 30, 2024	Sep. 30, 2024	Sep. 30, 2023	Sep. 30, 2024	Dec. 31, 2023
Finite-Lived Intangible Assets [Line Items]
Payments to date under terms of agreement		$ 26,569	$ 36,086
Goodwill	$ 800	800		$ 800	$ 800
Ligand License Agreement
Finite-Lived Intangible Assets [Line Items]
Substantial payments payable upon achievement of milestones	114,100	114,100		114,100
Payments to date under terms of agreement		$ 5,800		$ 47,200
Ligand License Agreement | Minimum
Finite-Lived Intangible Assets [Line Items]
Annual royalty percentage		15%
Ligand License Agreement | Maximum
Finite-Lived Intangible Assets [Line Items]
Annual royalty percentage		17%
Royalty Agreements
Finite-Lived Intangible Assets [Line Items]
Payments to date under terms of agreement	$ 5,400	$ 12,400

INTANGIBLE ASSETS - Summary of

INTANGIBLE ASSETS - Summary of Finite-lived Amortizable Intangible Assets (Details) - USD ($) $ in Thousands	Sep. 30, 2024	Dec. 31, 2023
Goodwill and Intangible Assets Disclosure [Abstract]
Finite-lived intangible assets	$ 212,866	$ 183,037
Less: accumulated amortization	(109,408)	(79,347)
Net carrying value	$ 103,458	$ 103,690

INTANGIBLE ASSETS - Summary o_2

INTANGIBLE ASSETS - Summary of Amortization Expense (Details) - USD ($) $ in Thousands	3 Months Ended		9 Months Ended
	Sep. 30, 2024	Sep. 30, 2023	Sep. 30, 2024	Sep. 30, 2023
Finite-Lived Intangible Assets [Line Items]
Total amortization expense	$ 10,812	$ 10,908	$ 30,061	$ 27,358
Research and development
Finite-Lived Intangible Assets [Line Items]
Total amortization expense	0	2,447	0	7,261
Selling, general and administrative
Finite-Lived Intangible Assets [Line Items]
Total amortization expense	$ 10,812	$ 8,461	$ 30,061	$ 20,097

CONVERTIBLE NOTES PAYABLE - Sum

CONVERTIBLE NOTES PAYABLE - Summary of Long-term Debt Instruments (Details) - Senior Notes - USD ($) $ in Thousands	Sep. 30, 2024	Dec. 31, 2023
Debt Instrument [Line Items]
Total convertible senior notes, net of unamortized debt discount and debt issuance costs	$ 378,556	$ 377,263
Senior Notes Due 2029
Debt Instrument [Line Items]
Interest rate percentage	2.25%	2.25%
Convertible senior notes	$ 316,250	$ 316,250
Unamortized debt issuance costs	$ (6,293)	$ (7,348)
Senior Notes Due 2025
Debt Instrument [Line Items]
Interest rate percentage	2.50%	2.50%
Convertible senior notes	$ 68,904	$ 68,904
Unamortized debt issuance costs	$ (305)	$ (543)

CONVERTIBLE NOTES PAYABLE - Add

CONVERTIBLE NOTES PAYABLE - Additional Information (Details) $ / shares in Units, $ in Thousands			3 Months Ended		9 Months Ended
	Mar. 11, 2022 USD ($)	Sep. 10, 2018 USD ($)	Sep. 30, 2024 USD ($) $ / shares	Sep. 30, 2023 USD ($)	Sep. 30, 2024 USD ($) day $ / shares	Sep. 30, 2023 USD ($)	Dec. 31, 2023
Debt Instrument [Line Items]
Debt instrument, repurchase amount Including accrued and unpaid interest	$ 213,800
Loss on extinguishment of debt						$ 7,600
Write off of deferred debt financing costs						3,400
Interest expense recognized			$ 2,777	$ 2,821	$ 8,365	$ 8,513
Senior Notes | Senior Notes Due 2029
Debt Instrument [Line Items]
Long-term debt, excluding current maturities	316,300
Interest rate percentage			2.25%		2.25%		2.25%
Proceeds from issuance of debt	306,400
Debt issuance costs, net	9,900		$ 9,900		$ 9,900
Accrued interest			$ 600		$ 600
Debt instrument, convertible, threshold percentage of stock price trigger					130%
Conversion ratio					0.03137
Conversion price (in dollars per share) | $ / shares			$ 31.87		$ 31.87
Notes payable			$ 100,000		$ 100,000
Long-term debt, term			7 years		7 years
Effective interest percentage			2.74%		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%
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%
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%		2.50%		2.50%
Proceeds from issuance of debt		267,200
Debt issuance costs, net		$ 8,800
Accrued interest			$ 100		$ 100
Debt instrument, convertible, threshold percentage of stock price trigger					130%
Conversion ratio					0.02577
Conversion price (in dollars per share) | $ / shares			$ 38.80		$ 38.80
Long-term debt, term			7 years		7 years
Effective interest percentage			2.98%		2.98%
Debt instrument, repurchase amount	$ 207,100
Long-term debt, excluding current maturities, repaid if converted			$ 68,900		$ 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%
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%

CONVERTIBLE NOTES PAYABLE - S_2

CONVERTIBLE NOTES PAYABLE - Summary of Interest Expense (Details) - Senior Notes Due 2025 and Senior Notes Due 2029 - Senior Notes - USD ($) $ in Thousands	3 Months Ended		9 Months Ended
	Sep. 30, 2024	Sep. 30, 2023	Sep. 30, 2024	Sep. 30, 2023
Debt Instrument [Line Items]
Contractual interest expense	$ 2,210	$ 2,210	$ 6,629	$ 6,629
Amortization of debt issuance costs	432	430	1,293	1,288
Total interest expense for the 2025 and 2029 Notes	$ 2,642	$ 2,640	$ 7,922	$ 7,917

ACCRUED EXPENSES (Details)

ACCRUED EXPENSES (Details) - USD ($) $ in Thousands	Sep. 30, 2024	Dec. 31, 2023
Payables and Accruals [Abstract]
Compensation related costs	$ 28,295	$ 29,908
Research and development	17,065	26,006
Accrued royalties	10,168	6,991
Sales discounts, rebates, and allowances	9,920	13,730
Selling, general and administrative	7,607	7,190
French rebate accrual	6,292	5,377
Transition services accrual	1,239	12,282
Accrued restructuring costs	358	11,421
Miscellaneous accrued expenses	2,972	6,086
Total accrued expenses	$ 83,916	$ 118,991

NET LOSS PER COMMON SHARE - Add

NET LOSS PER COMMON SHARE - Additional Information (Details) - $ / shares shares in Thousands	3 Months Ended
	Sep. 30, 2024	Feb. 28, 2023
Subsidiary, Sale of Stock [Line Items]
Exercise of pre-funded common stock warrants (in shares)	1,250
Underwritten Equity Offering
Subsidiary, Sale of Stock [Line Items]
Sales price per share (in dollars per share)		$ 21
Underwritten Equity Offering | Pre Funded Warrant
Subsidiary, Sale of Stock [Line Items]
Number of securities called by warrants or rights (in shares)		1,250
Sales price per share (in dollars per share)		$ 20.9999

NET LOSS PER COMMON SHARE - Sum

NET LOSS PER COMMON SHARE - Summary of Basic and Diluted Net Income (Loss) Per Share (Details) - USD ($) $ / shares in Units, $ in Thousands	3 Months Ended		9 Months Ended
	Sep. 30, 2024	Sep. 30, 2023	Sep. 30, 2024	Sep. 30, 2023
Earnings Per Share [Abstract]
Continuing operations, basic (in shares)	77,779,379	76,305,603	77,473,161	73,523,620
Continuing operations, diluted (in shares)	77,779,379	76,305,603	77,473,161	73,523,620
Discontinued operations, basic (in shares)	77,779,379	76,305,603	77,473,161	73,523,620
Discontinued operations, diluted (in shares)	77,779,379	76,305,603	77,473,161	73,523,620
Weighted average common shares outstanding, basic (in shares)	77,779,379	76,305,603	77,473,161	73,523,620
Weighted average common shares outstanding, diluted (in shares)	77,779,379	76,305,603	77,473,161	73,523,620
Net Income (loss) from continuing operations	$ (54,752)	$ (89,241)	$ (260,362)	$ (287,737)
Net Income (loss) from discontinued operations, net of tax	(59)	239,976	(919)	266,511
Net Income (loss), basic	(54,811)	150,735	(261,281)	(21,226)
Net Income (loss), diluted	$ (54,811)	$ 150,735	$ (261,281)	$ (21,226)
EPS from continuing operations, basic (in dollars per share)	$ (0.70)	$ (1.17)	$ (3.36)	$ (3.91)
EPS from continuing operations, diluted (in dollars per share)	(0.70)	(1.17)	(3.36)	(3.91)
EPS from discontinued operations, basic (in dollars per share)	0	3.14	(0.01)	3.62
EPS from discontinued operations, diluted (in dollars per share)	0	3.14	(0.01)	3.62
Net (loss) income per common share, diluted (in dollars per share)	(0.70)	1.97	(3.37)	(0.29)
Net (loss) income per common share, basic (in dollars per share)	$ (0.70)	$ 1.97	$ (3.37)	$ (0.29)

NET LOSS PER COMMON SHARE - S_2

NET LOSS PER COMMON SHARE - Summary of Common Stock Options, Convertible Debt and Restricted Stock Units Anti-dilutive (Details) - shares	3 Months Ended		9 Months Ended
	Sep. 30, 2024	Sep. 30, 2023	Sep. 30, 2024	Sep. 30, 2023
Total anti-dilutive shares
Anti-dilutive shares excluded from the calculation (in shares)	25,742,523	25,736,005	26,285,858	25,780,850
Convertible debt
Total anti-dilutive shares
Anti-dilutive shares excluded from the calculation (in shares)	11,697,953	11,697,952	11,697,953	11,697,952
Options
Total anti-dilutive shares
Anti-dilutive shares excluded from the calculation (in shares)	10,235,428	10,533,703	10,759,644	10,600,069
Restricted stock
Total anti-dilutive shares
Anti-dilutive shares excluded from the calculation (in shares)	3,809,142	3,504,350	3,828,261	3,482,829

COMMITMENTS AND CONTINGENCIES (

COMMITMENTS AND CONTINGENCIES (Details) - USD ($) $ in Thousands	1 Months Ended	3 Months Ended		9 Months Ended
	Mar. 31, 2024	Sep. 30, 2024	Sep. 30, 2023	Sep. 30, 2024	Sep. 30, 2023	Nov. 30, 2020
Business Acquisition [Line Items]
In-process research and development	$ 65,200	$ 0	$ 0	$ 65,205	$ 0
Orphan Technologies Limited
Business Acquisition [Line Items]
Contingent consideration, liability						$ 427,000

SHARE-BASED COMPENSATION - Summ

SHARE-BASED COMPENSATION - Summary of Stock Option Issuances and Balances Outstanding (Details) $ / shares in Units, $ in Thousands	9 Months Ended	12 Months Ended
	Sep. 30, 2024 USD ($) $ / shares shares	Dec. 31, 2023 USD ($) $ / shares shares
Aggregate Intrinsic Value (in thousands)
Number of shares exercisable (in shares) | shares	7,200,000
Weighted average exercise price (in dollars per share) | $ / shares	$ 21.61
Stock Options
Shares Underlying Options
Beginning balance (in shares) | shares	10,211,353
Granted (in shares) | shares	1,524,300
Exercised (in shares) | shares	(52,000)
Forfeited/canceled (in shares) | shares	(1,920,196)
Ending balance (in shares) | shares	9,763,457	10,211,353
Vested and expected to vest (in shares) | shares	9,763,457
Weighted Average Exercise Price
Beginning balance (in dollars per share) | $ / shares	$ 21.52
Granted (in dollars per share) | $ / shares	8.63
Exercised (in dollars per share) | $ / shares	10.99
Forfeited/canceled (in dollars per share) | $ / shares	19.68
Ending balance (in dollars per share) | $ / shares	19.92	$ 21.52
Vested and expected to vest (in dollars per share) | $ / shares	$ 19.92
Weighted Average Remaining Contractual Life (years)
Weighted Average Remaining Contractual Life (years)	5 years 6 months	4 years 10 months 28 days
Vested and expected to vest	5 years 6 months
Aggregate Intrinsic Value (in thousands)
Aggregate intrinsic value | $	$ 299,450	$ 0
Exercised, aggregate intrinsic value | $	159,600
Aggregate intrinsic value, vested and expected to vest | $	299,450
Unamortized stock compensation expense | $	$ 19,700
Weighted-average recognition period (in years)	2 years 6 months

SHARE-BASED COMPENSATION - Su_2

SHARE-BASED COMPENSATION - Summary of Service Based Restricted Stock Activity (Details) - Restricted stock units $ / shares in Units, $ in Millions	9 Months Ended
	Sep. 30, 2024 USD ($) $ / shares shares
Number of Restricted Stock Units
Beginning balance (in shares) | shares	2,874,046
Granted (in shares) | shares	2,040,625
Vested (in shares) | shares	(934,175)
Forfeited/canceled (in shares) | shares	(372,070)
Ending balance (in shares) | shares	3,608,426
Weighted Average Grant Date Fair Value
Beginning balance (in dollars per share) | $ / shares	$ 22.97
Granted (in dollars per share) | $ / shares	8.90
Vested (in dollars per share) | $ / shares	23.11
Forfeited/canceled (in dollars per share) | $ / shares	19.1
Ending balance (in dollars per share) | $ / shares	$ 15.38
Unamortized stock compensation expense | $	$ 41.4
Weighted-average recognition period (in years)	2 years 6 months

SHARE-BASED COMPENSATION - Su_3

SHARE-BASED COMPENSATION - Summary of Performance Based Restricted Stock Activity (Details) - Performance Shares $ / shares in Units, $ in Millions	9 Months Ended
	Sep. 30, 2024 USD ($) $ / shares shares
Number of Restricted Stock Units
Beginning balance (in shares) | shares	175,458
Granted (in shares) | shares	107,000
Vested (in shares) | shares	0
Forfeited/canceled (in shares) | shares	(66,250)
Ending balance (in shares) | shares	216,208
Weighted Average Grant Date Fair Value
Beginning balance (in dollars per share) | $ / shares	$ 25.61
Granted (in dollars per share) | $ / shares	8.93
Vested (in dollars per share) | $ / shares	0
Forfeited/canceled (in dollars per share) | $ / shares	22.40
Ending balance (in dollars per share) | $ / shares	$ 18.34
Unamortized stock compensation expense | $	$ 1.3
Weighted-average recognition period (in years)	1 year 2 months 12 days

SHARE-BASED COMPENSATION - Su_4

SHARE-BASED COMPENSATION - Summary of Share Based Compensation Expenses (Details) - USD ($) $ in Thousands	3 Months Ended		9 Months Ended
	Sep. 30, 2024	Sep. 30, 2023	Sep. 30, 2024	Sep. 30, 2023
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Total share-based compensation	$ 8,021	$ 11,321	$ 27,698	$ 36,102
Research and development
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Total share-based compensation	3,321	4,372	10,752	13,372
Selling, general and administrative
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Total share-based compensation	$ 4,700	$ 6,949	$ 16,946	$ 22,730

INVENTORY (Details)

INVENTORY (Details) - USD ($) $ in Thousands	Sep. 30, 2024	Dec. 31, 2023
Inventory Disclosure [Abstract]
Raw materials	$ 33,129	$ 33,790
Work in process	5,560	4,727
Finished goods	4,189	2,387
Total inventory	42,878	40,904
Inventory	6,356	9,410
Long-term inventory	$ 36,522	$ 31,494

ACCOUNTS RECEIVABLE (Details)

ACCOUNTS RECEIVABLE (Details) - USD ($) $ in Thousands	Sep. 30, 2024	Dec. 31, 2023
Receivables [Abstract]
Accounts receivable, net	$ 25,170	$ 21,179

EQUITY OFFERINGS (Details)

EQUITY OFFERINGS (Details) - USD ($) $ / shares in Units, $ in Thousands	1 Months Ended	3 Months Ended	9 Months Ended		12 Months Ended
	Feb. 28, 2023	Sep. 30, 2024	Sep. 30, 2024	Sep. 30, 2023	Dec. 31, 2023	Dec. 31, 2022	Feb. 29, 2020
Class of Stock [Line Items]
Proceeds from the issuance of pre-funded warrants, net of issuance costs			$ 0	$ 24,630
Exercise of pre-funded common stock warrants (in shares)		1,250,000
Common Stock
Class of Stock [Line Items]
Exercise of pre-funded common stock warrants (in shares)		1,250,000	1,250,000
Underwritten Equity Offering
Class of Stock [Line Items]
Shares authorized in sale (in shares)	9,700,000
Sales price per share (in dollars per share)	$ 21
Proceeds from the issuance of common stock, net of issuance costs	$ 215,800
Underwritten Equity Offering | Common Stock
Class of Stock [Line Items]
Prefunded warrants, notice period for modifying limitations	61 days
Pre-funded warrants exercisable for share of common stock (in shares)	1
Underwritten Equity Offering | Pre Funded Warrant
Class of Stock [Line Items]
Number of securities called by warrants or rights (in shares)	1,250,000
Sales price per share (in dollars per share)	$ 20.9999
Exercise price of each pre-funded warrant (in dollars per share)	$ 0.0001
Proceeds from the issuance of pre-funded warrants, net of issuance costs	$ 24,600
At-The-Market Offering
Class of Stock [Line Items]
Shares authorized in sale (in shares)			0		0
Proceeds from the issuance of common stock, net of issuance costs						$ 20,100
Aggregate offering amount authorized							$ 100,000
At-The-Market Offering Under Previous Registration Statement
Class of Stock [Line Items]
Amount sold to date		$ 28,600	$ 28,600
At-The-Market Offering Under Current Registration Statement
Class of Stock [Line Items]
Amount sold to date		$ 51,900	$ 51,900

DIVESTITURES - Additional Infor

DIVESTITURES - Additional Information (Details) - USD ($) $ in Thousands		3 Months Ended		9 Months Ended
	Aug. 31, 2023	Sep. 30, 2024	Sep. 30, 2023	Sep. 30, 2024	Sep. 30, 2023
Income Statement, Balance Sheet and Additional Disclosures by Disposal Groups, Including Discontinued Operations [Line Items]
Transition services accruals, current		$ 1,200		$ 1,200
Disposed of by Sale | Bile Acid Product Portfolio
Income Statement, Balance Sheet and Additional Disclosures by Disposal Groups, Including Discontinued Operations [Line Items]
Proceeds from sale of business	$ 210,000
Consideration receivable on sale of business (up to)	235,000
Gain on disposal of discontinued operations, net of tax	226,000	0	$ 225,942	0	$ 225,942
Discontinued operation, intra-entity amounts, discontinued operation after disposal, expense		$ 100		$ 500
Disposed of by Sale | Bile Acid Product Portfolio | Minimum
Income Statement, Balance Sheet and Additional Disclosures by Disposal Groups, Including Discontinued Operations [Line Items]
Milestone thresholds	125,000
Disposed of by Sale | Bile Acid Product Portfolio | Maximum
Income Statement, Balance Sheet and Additional Disclosures by Disposal Groups, Including Discontinued Operations [Line Items]
Milestone thresholds	$ 500,000

DIVESTITURES - Summary Results,

DIVESTITURES - Summary Results, Assets and Liabilities of Discontinued Operations (Details) - USD ($) $ in Thousands		3 Months Ended		9 Months Ended
	Aug. 31, 2023	Sep. 30, 2024	Sep. 30, 2023	Sep. 30, 2024	Sep. 30, 2023
Income Statement, Balance Sheet and Additional Disclosures by Disposal Groups, Including Discontinued Operations [Line Items]
Net (loss) income from discontinued operations		$ (59)	$ 239,976	$ (919)	$ 266,511
Disposed of by Sale | Bile Acid Product Portfolio
Income Statement, Balance Sheet and Additional Disclosures by Disposal Groups, Including Discontinued Operations [Line Items]
Total revenue		(103)	12,592	(554)	66,198
Cost of goods sold		0	381		1,899
Cost of goods sold				(10)
Research and development		13	1,491	247	6,161
Selling, general and administrative		(57)
Selling, general and administrative			5,723	128	17,851
Change in fair value of contingent consideration		0	(9,069)	0	(473)
Total operating expenses		(44)	(1,474)	365	25,438
Operating (loss) income		(59)	14,066	(919)	40,760
Interest expense		0	(32)	0	(191)
Gain on disposal of discontinued operations, net of tax	$ 226,000	0	225,942	0	225,942
Total other expense, net		0	225,910	0	225,751
Net (loss) income from discontinued operations		(59)	239,976	(919)	266,511
Net product sales | Disposed of by Sale | Bile Acid Product Portfolio
Income Statement, Balance Sheet and Additional Disclosures by Disposal Groups, Including Discontinued Operations [Line Items]
Total revenue		$ (103)	$ 12,592	$ (554)	$ 66,198