Cover
Cover - shares | 3 Months Ended | |
Mar. 31, 2023 | May 02, 2023 | |
Cover [Abstract] | ||
Document Type | 10-Q | |
Document Quarterly Report | true | |
Document Period End Date | Mar. 31, 2023 | |
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 $0.0001 per share | |
Trading Symbol | TVTX | |
Security Exchange Name | NASDAQ | |
Entity Current Reporting Status | Yes | |
Entity Interactive Data Current | Yes | |
Entity Filer Category | Large Accelerated Filer | |
Entity Small Business | false | |
Entity Emerging Growth Company | false | |
Entity Shell Company | false | |
Entity Common Stock, Shares Outstanding | 74,607,901 | |
Entity Central Index Key | 0001438533 | |
Current Fiscal Year End Date | --12-31 | |
Amendment Flag | false | |
Document Fiscal Year Focus | 2023 | |
Document Fiscal Period Focus | Q1 |
CONSOLIDATED BALANCE SHEETS
CONSOLIDATED BALANCE SHEETS - USD ($) $ in Thousands | Mar. 31, 2023 | Dec. 31, 2022 |
Current assets: | ||
Cash and cash equivalents | $ 161,376 | $ 61,688 |
Marketable debt securities, at fair value | 400,137 | 388,557 |
Accounts receivable, net | 21,537 | 16,646 |
Inventory, net | 6,712 | 6,922 |
Prepaid expenses and other current assets | 15,142 | 12,624 |
Total current assets | 604,904 | 486,437 |
Property and equipment, net | 9,127 | 9,049 |
Operating lease right of use assets | 20,289 | 21,000 |
Intangible assets, net | 162,883 | 145,038 |
Other assets | 11,028 | 11,061 |
Total assets | 808,231 | 672,585 |
Current liabilities: | ||
Accounts payable | 16,727 | 17,290 |
Accrued expenses | 85,766 | 95,742 |
Deferred revenue, current portion | 10,975 | 11,976 |
Business combination-related contingent consideration, current portion | 6,900 | 7,000 |
Operating lease liabilities, current portion | 4,545 | 4,433 |
Other current liabilities | 5,732 | 5,722 |
Total current liabilities | 130,645 | 142,163 |
Convertible debt | 375,974 | 375,545 |
Deferred revenue, less current portion | 8,778 | 10,931 |
Business combination-related contingent consideration, less current portion | 68,300 | 64,200 |
Operating lease liabilities, less current portion | 26,326 | 27,510 |
Other non-current liabilities | 9,068 | 9,385 |
Total liabilities | 619,091 | 629,734 |
Commitments and Contingencies (See Note 13) | ||
Stockholders' Equity: | ||
Preferred stock $0.0001 par value; 20,000,000 shares authorized; no shares issued and outstanding as of March 31, 2023 and December 31, 2022 | 0 | 0 |
Common stock $0.0001 par value; 200,000,000 shares authorized; 74,586,806, and 64,290,570 issued and outstanding as of March 31, 2023 and December 31, 2022, respectively | 7 | 6 |
Additional paid-in capital | 1,291,863 | 1,059,975 |
Accumulated deficit | (1,100,554) | (1,014,223) |
Accumulated other comprehensive loss | (2,176) | (2,907) |
Total stockholders' equity | 189,140 | 42,851 |
Total liabilities and stockholders' equity | $ 808,231 | $ 672,585 |
CONSOLIDATED BALANCE SHEETS (Pa
CONSOLIDATED BALANCE SHEETS (Parenthetical) - $ / shares | Mar. 31, 2023 | Dec. 31, 2022 |
Statement of Financial Position [Abstract] | ||
Preferred stock, par value (in dollars per share) | $ 0.0001 | $ 0.0001 |
Preferred stock, shares authorized (in shares) | 20,000,000 | 20,000,000 |
Preferred stock, shares issued (in shares) | 0 | 0 |
Preferred stock, shares outstanding (in shares) | 0 | 0 |
Common stock par value (in dollars per share) | $ 0.0001 | $ 0.0001 |
Common stock shares authorized (in shares) | 200,000,000 | 200,000,000 |
Common stock shares issued (in shares) | 74,586,806 | 64,290,570 |
Common stock shares outstanding (in shares) | 74,586,806 | 64,290,570 |
CONSOLIDATED STATEMENTS OF OPER
CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS - USD ($) $ in Thousands | 3 Months Ended | |
Mar. 31, 2023 | Mar. 31, 2022 | |
Total revenue | $ 56,993 | $ 48,487 |
Operating expenses: | ||
Cost of goods sold | 5,125 | 2,138 |
Research and development | 59,913 | 56,611 |
Selling, general and administrative | 72,245 | 46,788 |
Change in fair value of contingent consideration | 6,756 | 9,080 |
Total operating expenses | 144,039 | 114,617 |
Operating loss | (87,046) | (66,130) |
Other income (expenses), net: | ||
Interest income | 3,646 | 278 |
Interest expense | (2,940) | (2,515) |
Other income, net | 87 | 26 |
Loss on extinguishment of debt | 0 | (7,578) |
Total other income (expense), net | 793 | (9,789) |
Loss before income tax provision | (86,253) | (75,919) |
Income tax provision | (78) | (52) |
Net loss | $ (86,331) | $ (75,971) |
Basic net loss per common share (in dollars per share) | $ (1.27) | $ (1.20) |
Diluted net loss per common share (in dollars per share) | $ (1.27) | $ (1.20) |
Basic weighted average common shares outstanding (in shares) | 68,174,099 | 63,132,841 |
Diluted weighted average common shares outstanding (in shares) | 68,174,099 | 63,132,841 |
Comprehensive loss: | ||
Net loss | $ (86,331) | $ (75,971) |
Foreign currency translation (loss) gain | (566) | 71 |
Unrealized gain (loss) on marketable debt securities | 1,297 | (1,204) |
Comprehensive loss | (85,600) | (77,104) |
Net product sales | ||
Total revenue | 50,283 | 46,443 |
Operating expenses: | ||
Cost of goods sold | 2,088 | 2,138 |
License and collaboration revenue | ||
Total revenue | 6,710 | 2,044 |
Operating expenses: | ||
Cost of goods sold | $ 3,037 | $ 0 |
CONSOLIDATED STATEMENTS OF STOC
CONSOLIDATED STATEMENTS OF STOCKHOLDERS' EQUITY - USD ($) $ in Thousands | Total | Underwritten Equity Offering | At-The-Market Offering | Cumulative Effect, Period of Adoption, Adjustment | Common Stock | Common Stock Underwritten Equity Offering | Common Stock At-The-Market Offering | Additional Paid in Capital | Additional Paid in Capital Underwritten Equity Offering | Additional Paid in Capital At-The-Market Offering | Additional Paid in Capital Cumulative Effect, Period of Adoption, Adjustment | Accumulated Other Comprehensive Loss | Accumulated Deficit | Accumulated Deficit Cumulative Effect, Period of Adoption, Adjustment |
Beginning balance (in shares) at Dec. 31, 2021 | 62,491,498 | |||||||||||||
Beginning balance at Dec. 31, 2021 | $ 302,112 | $ (44,720) | $ 6 | $ 1,068,634 | $ (74,945) | $ (562) | $ (765,966) | $ 30,225 | ||||||
Increase (Decrease) in Stockholders' Equity [Roll Forward] | ||||||||||||||
Share based compensation | 7,935 | 7,935 | ||||||||||||
Issuance of common stock under the equity incentive plan and proceeds from exercise (in shares) | 317,179 | |||||||||||||
Issuance of common stock under the equity incentive plan and proceeds from exercise | 122 | 122 | ||||||||||||
Employee stock purchase program purchase and expense | 251 | 251 | ||||||||||||
Issuance of common stock/equity offering, net of issuance costs (in shares) | 701,600 | |||||||||||||
Issuance of common stock/equity offering, net of issuance costs | $ 19,545 | $ 19,545 | ||||||||||||
Foreign currency translation adjustments | 71 | 71 | ||||||||||||
Unrealized gain (loss) on marketable debt securities | (1,204) | (1,204) | ||||||||||||
Net loss | (75,971) | (75,971) | ||||||||||||
Ending balance (in shares) at Mar. 31, 2022 | 63,510,277 | |||||||||||||
Ending balance at Mar. 31, 2022 | 208,141 | $ 6 | 1,021,542 | (1,695) | (811,712) | |||||||||
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 | 13,325 | 13,325 | ||||||||||||
Issuance of common stock under the equity incentive plan and proceeds from exercise (in shares) | 592,486 | |||||||||||||
Issuance of common stock under the equity incentive plan and proceeds from exercise | 2,296 | 2,296 | ||||||||||||
Employee stock purchase program purchase and expense | 439 | 439 | ||||||||||||
Issuance of common stock/equity offering, net of issuance costs (in shares) | 9,703,750 | |||||||||||||
Issuance of common stock/equity offering, net of issuance costs | $ 191,199 | $ 1 | $ 191,198 | |||||||||||
Issuance of pre-funded common stock warrants, net of issuance costs of $1.6 million | 24,630 | 24,630 | ||||||||||||
Foreign currency translation adjustments | (566) | (566) | ||||||||||||
Unrealized gain (loss) on marketable debt securities | 1,297 | 1,297 | ||||||||||||
Net loss | (86,331) | (86,331) | ||||||||||||
Ending balance (in shares) at Mar. 31, 2023 | 74,586,806 | |||||||||||||
Ending balance at Mar. 31, 2023 | $ 189,140 | $ 7 | $ 1,291,863 | $ (2,176) | $ (1,100,554) |
CONSOLIDATED STATEMENTS OF ST_2
CONSOLIDATED STATEMENTS OF STOCKHOLDERS' EQUITY (Parenthetical) - USD ($) $ in Millions | 3 Months Ended | |
Mar. 31, 2023 | Mar. 31, 2022 | |
Pre-funded Common Stock Warrants, Net of Issuance Costs | $ 1.6 | |
Underwritten Equity Offering | ||
Issuance costs | $ 12.6 | |
At-The-Market Offering | ||
Issuance costs | $ 0.6 |
CONSOLIDATED STATEMENTS OF CASH
CONSOLIDATED STATEMENTS OF CASH FLOWS - USD ($) $ in Thousands | 3 Months Ended | |
Mar. 31, 2023 | Mar. 31, 2022 | |
Cash Flows From Operating Activities: | ||
Net loss | $ (86,331) | $ (75,971) |
Adjustments to reconcile net loss to net cash used in operating activities: | ||
Depreciation and amortization | 9,545 | 7,092 |
Share based compensation | 13,764 | 8,186 |
Change in estimated fair value of contingent consideration | 6,756 | 9,080 |
Payments from change in fair value of contingent consideration | (2,289) | (2,075) |
Loss on extinguishment of debt | 0 | 7,578 |
Other | (488) | 2,112 |
Changes in operating assets and liabilities: | ||
Accounts receivable | (4,891) | 1,241 |
Inventory | (513) | (157) |
Prepaid expenses and other current and non-current assets | (2,350) | 648 |
Accounts payable | (627) | (4,606) |
Accrued expenses | (9,611) | (4,413) |
Deferred revenue, current and non-current | (3,844) | (2,146) |
Other current and non-current liabilities | (238) | (1,889) |
Net cash used in operating activities | (81,117) | (55,320) |
Cash Flows From Investing Activities: | ||
Proceeds from the sale/maturity of marketable debt securities | 94,118 | 64,715 |
Purchase of marketable debt securities | (103,190) | (26,283) |
Purchase of fixed assets | (630) | (131) |
Purchase of intangible assets | (27,337) | (5,136) |
Net cash (used in) provided by investing activities | (37,039) | 33,165 |
Cash Flows From Financing Activities: | ||
Payment of guaranteed minimum royalty | (525) | (525) |
Payment of business combination-related contingent consideration | (604) | (667) |
Proceeds from issuances of 2029 convertible senior notes | 0 | 316,250 |
Payment of debt issuance costs | 0 | (9,488) |
Repurchase of 2025 convertible senior notes including premium | 0 | (211,324) |
Proceeds from exercise of stock options | 2,296 | 122 |
Proceeds from the issuance of pre-funded warrants, net of issuance costs | 24,630 | 0 |
Net cash provided by financing activities | 216,996 | 113,913 |
Effect of exchange rate changes on cash | 848 | (939) |
Net increase in cash and cash equivalents | 99,688 | 90,819 |
Cash and cash equivalents, beginning of year | 61,688 | 165,753 |
Cash and cash equivalents, end of period | 161,376 | 256,572 |
Underwritten Equity Offering | ||
Cash Flows From Financing Activities: | ||
Proceeds from the issuance of common stock, net of issuance costs | 191,199 | 0 |
At-The-Market Offering | ||
Cash Flows From Financing Activities: | ||
Proceeds from the issuance of common stock, net of issuance costs | $ 0 | $ 19,545 |
DESCRIPTION OF BUSINESS
DESCRIPTION OF BUSINESS | 3 Months Ended |
Mar. 31, 2023 | |
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, liver, 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. FILSPARI™ (sparsentan) On February 17, 2023, the FDA granted accelerated approval of FILSPARI™ (sparsentan) to reduce proteinuria in adults with primary IgAN at risk of rapid disease progression, generally at UPCR ≥1.5 gram/gram. FILSPARI, a once-daily, oral medication is designed to selectively target two critical pathways (endothelin 1 and angiotensin-II) in the disease progression of IgAN. Clinical-Stage Programs: The continued approval of FILSPARI for IgAN may be contingent upon confirmation of a clinical benefit in the Company's ongoing Phase 3 clinical trial of sparsentan for the treatment of IgAN (the "PROTECT Study"), which is designed to demonstrate whether FILSPARI slows kidney function decline. Topline results from the two-year confirmatory endpoints in the PROTECT Study are expected in the fourth quarter of 2023 and are intended to support traditional approval of FILSPARI. Sparsentan remains a novel investigational product candidate which has been granted Orphan Drug Designation for the treatment of focal segmental glomerulosclerosis (FSGS) in the U.S. and Europe. The double-blind portion of the Phase 3 study of sparsentan for FSGS has recently concluded and, following release of the top-line data from the study which showed that the study did not meet its primary endpoint, the Company is conducting further analyses of the data and is preparing to engage with regulators to explore a potential path forward toward a potential regulatory submission in FSGS. Pegtibatinase ( TVT-058) is a novel investigational human enzyme replacement candidate being evaluated for the treatment of classical homocystinuria (HCU). Pegtibatinase has been granted Rare Pediatric Disease, Fast Track and Breakthrough Therapy designations by the FDA, as well as orphan drug designation in the United States and European Union. Pegtibatinase is currently being evaluated in the Phase 1/2 COMPOSE Study to assess its safety, tolerability, pharmacokinetics, pharmacodynamics and clinical effects in patients with classical HCU. The Company acquired pegtibatinase as part of the November 2020 acquisition of Orphan Technologies Limited. Chenodal (chenodeoxycholic acid or CDCA) is a naturally occurring bile acid that is approved for the treatment of people with radiolucent stones in the gallbladder. In September 2022, the Company was granted Fast Track Designation by the FDA for the investigation of Chenodal for cerebrotendinous xanthomatosis (CTX). In January 2020, the Company randomized the first patients in its Phase 3 RESTORE Study to evaluate the effects of Chenodal in adult and pediatric patients with CTX, and the study enrollment remains open. The pivotal study is intended to support an NDA submission for marketing authorization of Chenodal for CTX in the United States. Preclinical Programs: The Company is a participant in a Cooperative Research and Development Agreement ("CRADA"), which forms a multi-stakeholder approach to pool resources with leading experts, and incorporate the patient perspective early in the therapeutic identification and development process. The Company is partnering with the National Institutes of Health’s National Center for Advancing Translational Sciences ("NCATS") and a leading patient advocacy organization, Alagille Syndrome Alliance, aimed at the identification of potential small molecule therapeutics for Alagille syndrome ("ALGS"). There are no treatment options currently approved for ALGS. 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. Other Commercial Products: • Thiola ® and Thiola EC ® (tiopronin tablets) are approved in the United States for the prevention of cystine (kidney) stone formation in patients with severe homozygous cystinuria. • Cholbam ® (cholic acid capsules) is approved in the United States for the treatment of bile acid synthesis disorders due to single enzyme defects and is further indicated for adjunctive treatment of patients with peroxisomal disorders. |
BASIS OF PRESENTATION AND SIGNI
BASIS OF PRESENTATION AND SIGNIFICANT ACCOUNTING POLICIES | 3 Months Ended |
Mar. 31, 2023 | |
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, 2022, filed with the SEC on February 23, 2023. 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, 2022 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. 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 judgement to evaluate whether the milestones are probable of being achieved and estimates the amount to include in the transaction price utilizing the most likely amount method. If it is probable that a significant revenue reversal will not occur, the estimated amount is included in the transaction price. Milestone payments that are not within the Company or the licensee’s control, such as regulatory approvals, are considered to be constrained due to a high degree of uncertainty and are not included in the transaction price until such uncertainty is resolved. At the end of each reporting period, the Company re-evaluates the probability of achievement of development milestones and any related constraint and adjusts the estimate of the overall transaction price, if necessary. The Company recognizes aggregate sales-based milestones and royalty payments from product sales of which the license is deemed to be the predominant item to which the royalties relate, at the later of when the related sales occur or when the performance obligation to which the sales-based milestone or royalty has been allocated has been satisfied. Revenue from collaboration and licensing agreements may also include sales of inventory, at cost plus a margin, and is recorded in license and collaboration revenue. The Company utilizes significant judgement to develop estimates of the stand-alone selling price for each distinct performance obligation based upon the relative stand-alone selling price. Variable consideration that relates specifically to the Company’s efforts to satisfy specific performance obligations is allocated entirely to those performance obligations. The stand-alone selling price for license-related performance obligations requires judgement in developing assumptions to project probability-weighted cash flows based upon estimates of forecasted revenues, clinical and regulatory timelines and discount rates. The stand-alone selling price for clinical development performance obligations is based on forecasted expected costs of satisfying a performance obligation plus an appropriate margin. If the licenses to intellectual property are determined to be distinct from the other performance obligations identified in the arrangement and have stand-alone functionality, the Company recognizes revenues from non-refundable, upfront fees allocated to the license when the license is transferred to the licensee and the licensee is able to benefit from the license. For licenses that are not distinct from other promises, the Company applies judgment to assess the nature of the combined performance obligation to determine whether the combined performance obligation is satisfied over time or a point in time and, if over time, the appropriate method of measuring progress for purposes of recognizing revenue from non-refundable, upfront fees. The Company evaluates the measure of progress each reporting period and, if necessary, adjusts the related revenue recognition accordingly. The selection of the method to measure progress towards completion requires judgment and is based on the nature of the products or services to be provided. Revenue is recorded proportionally as costs are incurred. The Company generally utilizes the cost-to-cost method of progress because it best measures the transfer of control to the customer which occurs as the Company incurs costs. Under the cost-to-cost measure of progress, the extent of progress towards completion is measured based on the ratio of costs incurred to date to the total estimated costs at completion of the performance obligation. The Company uses judgment to estimate the total costs expected to complete the clinical development performance obligations, which include subcontractor costs, labor, materials, other direct costs and an allocation of indirect costs. The Company evaluates these cost estimates and the progress each reporting period and adjusts the measure of progress, if necessary. 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 ingredient to the Company's collaboration partner, at cost plus a margin. The following table summarizes cost of goods sold for the three months ended March 31, 2023 and 2022 ( in thousands ): Three Months Ended March 31, 2023 2022 Cost of goods sold - product sales $ 2,088 $ 2,138 Cost of goods sold - license and collaboration 3,037 — Total cost of goods sold $ 5,125 $ 2,138 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. Sales of FILSPARI for the three months ended March 31, 2023 primarily consisted of zero-cost inventories, which favorably impacted gross margin for related sales. Prior to the February 2023 FDA approval of FILSPARI (sparsentan), the Company recognized approximately $7.5 million in research and development expenses related to the production of active pharmaceutical ingredient to support the commercial launch of FILSPARI. The Company expects to continue to benefit from the sale of previously expensed inventories through at least 2024. Research and Development Expenses Research and development includes expenses related to sparsentan, pegtibatinase, and the Company's other pipeline programs. The Company expenses all research and development costs as they are incurred. The Company's research and development costs are composed of salaries and bonuses, benefits, share-based compensation, license fees, milestones under license agreements, costs paid to third-party contractors to perform research, conduct clinical trials, develop drug materials and delivery devices, manufacture drug product supplies to support clinical development, and associated overhead expenses and facilities costs. The Company charges direct internal and external program costs to the respective development programs. The Company also incurs indirect costs that are not allocated to specific programs because such costs benefit multiple development programs and allow us to increase our pharmaceutical development capabilities. These consist of internal shared resources related to the development and maintenance of systems and processes applicable to all of our programs. Nonrefundable advance payments for goods and services to be received in the future for use in research and development activities are recorded as prepaid expenses. The prepaid amounts are expensed as the related goods are delivered or the services are performed, or when it is no longer expected that the goods will be delivered or the services rendered. Clinical Trial Expenses The Company records expenses in connection with its clinical trials under contracts with contract research organizations ("CROs") that support conducting and managing clinical trials, as well as contract manufacturing organizations ("CMOs") for the manufacture of drug product supplies to support clinical development. The financial terms and activities of these agreements vary from contract to contract and may result in uneven expense levels. Generally, these agreements set forth activities that drive the recording of expenses such as start-up, initiation activities, enrollment, treatment of patients, or the completion of other clinical trial activities, and in the case of CMOs, costs associated with the production of drug product supplied and the procurement of raw materials to be consumed in the manufacturing process. Expenses related to clinical trials are accrued based on our estimates of the progress of services performed, including actual level of patient enrollment, completion of patient studies and progress of the clinical trials or the delivery of goods. Other incidental costs related to patient enrollment or treatment are accrued when reasonably certain. If the amounts we are obligated to pay under our clinical trial agreements are modified (for instance, as a result of changes in the clinical trial protocol or scope of work to be performed), the Company adjusts its estimates accordingly on a prospective basis. Revisions to the Company's contractual payment obligations are charged to expense in the period in which the facts that give rise to the revision become reasonably certain. The Company currently has one Phase 1/2 clinical trial and three Phase 3 clinical trials in process that are in varying stages of activity, with ongoing non-clinical support trials. As such, clinical trial expenses will vary depending on all the factors set forth above and may fluctuate significantly from quarter to quarter. Intangible Assets with Cost Accumulation Model In 2014, the Company entered into a license agreement with Mission Pharmacal in which the Company obtained the exclusive right to license the trademark of Thiola. The acquisition of the Thiola license qualified as an asset acquisition under the principles of ASC 805, Business Combinations ("ASC 805") in effect at the time of acquisition. The license agreement requires the Company to make royalty payments based on net sales of Thiola. The liability for royalties in excess of the annual contractual minimum is recognized in the period in which the royalties become probable and estimable, which is typically in the period corresponding with the respective sales. The Company records an offsetting increase to the cost basis of the asset under the cost accumulation model. The additional cost basis is subsequently amortized over the remaining life of the license agreement. Consistent with all prior periods since Thiola was acquired, the Company has not accrued any liability for future royalties in excess of the annual contractual minimum at March 31, 2023 as such royalties are not yet probable and estimable. Variable Interest Entity The Company reviews each investment and collaboration agreement to determine if it has a variable interest in the entity. In assessing whether the Company has a variable interest in the entity as a whole, the Company considers and makes judgements regarding the purpose and design of the entity, the value of the licensed assets to the entity, the value of the entity’s total assets and the significant activities of the entity. If the Company has a variable interest in the entity as a whole, the Company assesses whether or not the Company is a primary beneficiary of that VIE, based on a number of factors, including: (i) which party has the power to direct the activities that most significantly affect the VIE’s economic performance, (ii) the parties’ contractual rights and responsibilities pursuant to the collaboration agreement, and (iii) which party has the obligation to absorb losses of or the right to receive benefits from the VIE that could be significant to the VIE. If the Company determines that it is the primary beneficiary of a VIE at the onset of the collaboration, the collaboration is treated as a business combination and the Company consolidates the financial statements of the VIE into the Company’s consolidated financial statements. On a quarterly basis, the Company evaluates whether it continues to be the primary beneficiary of the consolidated VIE. If the Company determines that it is no longer the primary beneficiary of a consolidated VIE, it deconsolidates the VIE in the period in which the determination is made. Assets and liabilities recorded as a result of consolidating the financial results of the VIE into the Company’s consolidated balance sheet do not represent additional assets that could be used to satisfy claims against the Company’s general assets or liabilities for which creditors have recourse to the Company’s general assets. Recently Adopted Accounting Pronouncements In August 2020, the FASB issued ASU No. 2020-06, Accounting for Convertible Instruments and Contracts in an Entity's Own Equity. The ASU includes amendments to the guidance on convertible instruments and the derivative scope exception for contracts in an entity's own equity in Subtopic 815-40 and simplifies the accounting for convertible instruments which include beneficial conversion features or cash conversion features by removing certain separation models in Subtopic 470-20. Additionally, the ASU will require entities to use the "if-converted" method when calculating diluted earnings per share for convertible instruments. The ASU is effective for fiscal years beginning after December 15, 2021, including interim periods within those fiscal years. The adoption of the new standard impacted the Company's accounting for its Convertible Senior Notes Due 2025 (2025 Notes), discussed in Note 10, which were previously accounted for using the cash conversion model applied under ASC 470-20, Debt with Conversion and Other Options ("ASC 470-20"). The Company adopted ASU 2020-06 on January 1, 2022 using the modified retrospective method. The cumulative effect of the accounting change as of January 1, 2022 increased the carrying amount of the 2025 Notes by $44.7 million, reduced additional paid-in capital by $74.9 million, and reduced accumulated deficit by $30.2 million. Recently Issued Accounting Pronouncements From time to time, new accounting pronouncements are issued by the Financial Accounting Standards Board ("FASB") or other standard setting bodies. Unless otherwise discussed, the Company believes that the impact of recently issued standards that are not yet effective will not have a material impact on its consolidated financial position or results of operations upon adoption. |
REVENUE RECOGNITION
REVENUE RECOGNITION | 3 Months Ended |
Mar. 31, 2023 | |
Revenue from Contract with Customer [Abstract] | |
REVENUE RECOGNITION | REVENUE RECOGNITION Product Sales, Net Product sales consist of FILSPARI, bile acid products (Chenodal and Cholbam) 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 and Canada representing approximately 98% and 1% of net product sales, respectively, and rest of world representing less than 1% of net product sales, based on the product shipment destination. The Company sells FILSPARI to three direct-to-patient specialty pharmacies. The Company sells its other 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 other 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 three months ended March 31, 2023 and 2022, the Company recorded a net product revenue increase of $0.4 million and $0.1 million, respectively, related to performance obligations satisfied in previous periods. Government Rebates: The Company calculates the rebates that it will be obligated to provide to government programs and deducts these estimated amounts from its gross product sales at the time the revenues are recognized. Allowances for government rebates and discounts are established based on an estimated allocation of payers and the government-mandated discounts applicable to government-funded programs. Rebate discounts are included in other current liabilities in the accompanying consolidated balance sheets. Commercial Rebates: The Company calculates the rebates it incurs 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 other current liabilities in the accompanying consolidated balance sheets. Prompt Pay Discounts: The Company offers discounts to certain customers for prompt payments. The Company accrues for the calculated prompt pay discount based on the gross amount of each invoice for those customers at the time of sale. Product Returns: Consistent with industry practice, the Company offers its customers a limited right to return product purchased directly from the Company, which is principally based upon the product’s expiration date. 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 months ended March 31, 2023 and 2022 ( in thousands ): Three Months Ended March 31, 2023 2022 Bile acid products $ 26,105 $ 25,075 Tiopronin products 21,174 21,368 FILSPARI 3,004 — Total net product sales $ 50,283 $ 46,443 |
COLLABORATION AND LICENSE AGREE
COLLABORATION AND LICENSE AGREEMENTS | 3 Months Ended |
Mar. 31, 2023 | |
Organization, Consolidation and Presentation of Financial Statements [Abstract] | |
COLLABORATION AND LICENSE AGREEMENTS | COLLABORATION AND LICENSE AGREEMENTS On September 15, 2021, the Company entered into a license and collaboration agreement (“License Agreement”) with Vifor (International) Ltd. (“CSL Vifor”), pursuant to which the Company granted an exclusive license to CSL Vifor for the commercialization of sparsentan in Europe, Australia and New Zealand ("Licensed Territories"). CSL Vifor also has first right of negotiation to expand the licensed territories into Canada, China, Brazil and/ or Mexico. Under the terms of the License Agreement, the Company received an upfront payment of $55.0 million and will be eligible for up to $135.0 million in aggregate regulatory and market access related milestone payments and up to $655.0 million in aggregate sales-based milestone payments for a total potential value of up to $845.0 million. The Company is also entitled to receive tiered double-digit royalties of up to 40 percent of annual net sales of sparsentan in the Licensed Territories. Under the License Agreement, CSL Vifor will be responsible for all commercialization activities in the Licensed Territories. The Company remains responsible for the worldwide clinical development of sparsentan through regulatory approval as defined and will retain all rights to sparsentan in the United States and rest of world outside of the Licensed Territories. Development costs for any post regulatory approval development activities, subject to approval by both parties, will be borne by the Company and CSL Vifor as defined, respectively. The License Agreement will remain in effect, unless terminated earlier, until the expiration of all royalty terms for sparsentan in the licensed territories. Each party has the right to terminate the License Agreement for the other party’s uncured material breach, insolvency or if the time required for performance under the License Agreement by the other party is extended due to a force majeure event that continues for six months or more. The Company assessed the License Agreement and determined that it meets both criteria to be considered a collaborative agreement within the Scope of ASC 808, Collaborative Arrangements of active participation by both parties and exposures to significant risks and rewards dependent on the commercial success of the activities. Both parties participate on joint steering and other committees overseeing the collaboration activities. Also, both parties are exposed to significant risks and rewards based on the economic outcomes of regulatory approvals and commercialization of sparsentan. The Company determined the transaction price under the License Agreement totaled $55.0 million, consisting of the fixed non-refundable upfront payment. The variable regulatory and access related milestones were excluded from the transaction price given the substantial uncertainty related to their achievement. Sales-based milestone payments and royalties on net sales were excluded from the transaction price and will be recognized at the later of when the related sales occur or when the performance obligation to which the sales-based milestone or royalty has been allocated have been satisfied. The Company concluded that CSL Vifor represented a customer and applied relevant guidance from ASC 606 to evaluate the accounting under the License Agreement. In accordance with this guidance, the Company concluded that the promise to grant the license is distinct from the promise to provide clinical development services resulting in two performance obligations. As a result, the Company allocated $12.0 million of the transaction price, based on the performance obligations' relative standalone selling prices, to the license, which was recognized in full in 2021. The remaining $43.0 million of the transaction price was allocated to the clinical development activities and recorded as deferred revenue, which will be recognized over the development period based upon the ratio of costs incurred to date to the total estimated costs. For the three months ended March 31, 2023, the Company recognized $6.7 million in license and collaboration revenue, which consisted of $3.3 million from the sale of active pharmaceutical ingredient to CSL Vifor at cost plus a margin, and $3.4 million for clinical development activities, based upon the ratio of costs incurred to total estimated costs. For the three months ended March 31, 2022, the Company recognized $2.0 million in license and collaboration revenue for clinical development activities, based upon the ratio of costs incurred to total estimated costs. |
MARKETABLE DEBT SECURITIES
MARKETABLE DEBT SECURITIES | 3 Months Ended |
Mar. 31, 2023 | |
Investments, Debt and Equity Securities [Abstract] | |
MARKETABLE DEBT SECURITIES | MARKETABLE DEBT SECURITIES The Company's marketable debt securities as of March 31, 2023 and December 31, 2022 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 ): March 31, 2023 December 31, 2022 Marketable debt securities: Commercial paper $ 133,869 $ 123,647 Corporate debt securities 185,474 224,055 Securities of government sponsored entities 80,794 40,855 Total marketable debt securities $ 400,137 $ 388,557 The following is a summary of short-term marketable debt securities classified as available-for-sale as of March 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 $ 134,218 $ — $ (349) $ 133,869 Corporate debt securities Less than 1 118,324 — (883) 117,441 Securities of government-sponsored entities Less than 1 27,845 9 (107) 27,747 Total maturity less than 1 year 280,387 9 (1,339) 279,057 Corporate debt securities 1 to 2 68,268 109 (344) 68,033 Securities of government-sponsored entities 1 to 2 53,140 117 (210) 53,047 Total maturity 1 to 2 years 121,408 226 (554) 121,080 Total available-for-sale marketable debt securities $ 401,795 $ 235 $ (1,893) $ 400,137 The following is a summary of short-term marketable debt securities classified as available-for-sale as of December 31, 2022 ( in thousands ): Remaining Contractual Maturity Amortized Cost Unrealized Gains Unrealized Losses Aggregate Estimated Fair Value Marketable debt securities: Commercial paper Less than 1 $ 124,301 $ 2 $ (656) $ 123,647 Corporate debt securities Less than 1 155,841 — (1,355) 154,486 Securities of government-sponsored entities Less than 1 7,473 — (80) 7,393 Total maturity less than 1 year 287,615 2 (2,091) 285,526 Corporate debt securities 1 to 2 70,195 33 (659) 69,569 Securities of government-sponsored entities 1 to 2 33,702 6 (246) 33,462 Total maturity 1 to 2 years 103,897 39 (905) 103,031 Total available-for-sale securities $ 391,512 $ 41 $ (2,996) $ 388,557 During the three months ended March 31, 2023, investment activity for the Company included $94.1 million in maturities and $103.2 million in purchases, all relating to debt-based marketable securities. During the three months ended March 31, 2022, investment activity for the Company included $64.7 million in maturities and $26.3 million in purchases, all relating to debt-based marketable securities. As of March 31, 2023 and December 31, 2022, the accrued interest receivable related to the Company's marketable debt securities was $2.0 million and $1.9 million 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 March 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 $ 133,869 $ 349 $ — $ — $ 133,869 $ 349 Corporate debt securities 79,397 826 70,900 401 150,297 1,227 Securities of government-sponsored entities 51,225 292 2,486 25 53,711 317 Total $ 264,491 $ 1,467 $ 73,386 $ 426 $ 337,877 $ 1,893 The following is a summary of available-for-sale marketable debt securities in an unrealized loss position with no credit losses reported as of December 31, 2022 ( in thousands ): Less Than 12 Months 12 Months or Greater Total Description of Securities Fair Value Unrealized Losses Fair Value Unrealized Losses Fair Value Unrealized Losses Commercial paper $ 117,853 $ 656 $ — $ — $ 117,853 $ 656 Corporate debt securities 99,066 1,041 107,964 973 207,030 2,014 Securities of government-sponsored entities 31,402 263 4,456 63 35,858 326 Total $ 248,321 $ 1,960 $ 112,420 $ 1,036 $ 360,741 $ 2,996 As of March 31, 2023 and December 31, 2022, the amortized cost of the available-for-sale marketable debt securities in an unrealized loss position was $339.8 million and $363.7 million, respectively. As of March 31, 2023 and December 31, 2022, 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 three months ended March 31, 2023 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 |
VARIABLE INTEREST ENTITIES
VARIABLE INTEREST ENTITIES | 3 Months Ended |
Mar. 31, 2023 | |
Organization, Consolidation and Presentation of Financial Statements [Abstract] | |
VARIABLE INTEREST ENTITIES | VARIABLE INTEREST ENTITIES On March 8, 2022, the Company entered into a Collaboration Agreement with PharmaKrysto Limited (“PharmaKrysto”), a privately held pre-clinical stage company related to PharmaKrysto's early-stage cystinuria discovery program, and concurrently therewith entered into a Stock Purchase Agreement with PharmaKrysto (together, the "Agreements"). Pursuant to the terms of the Agreements, the Company paid PharmaKrysto's shareholders $0.6 million in cash to purchase 5% of the outstanding common shares of PharmaKrysto and $0.4 million to PharmaKrysto as a one-time signing fee. Under the Collaboration Agreement, the Company will fund all research and development expenses for the pre-clinical activities associated with the cystinuria program, which are estimated to be approximately $5.0 million. The Agreements require the Company to purchase an additional 5% of the outstanding common shares for $1.0 million upon the occurrence of a specified pre-clinical milestone, and grant an option to the Company to purchase 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 March 31, 2023 and December 31, 2022 were immaterial. The results of operations were not significant for the three months ended March 31, 2023 and 2022. The Company is not required to provide additional funding other than the contractually required amounts disclosed above. The creditors and beneficial holders of PharmaKrysto have no recourse to the general credit or assets of the Company. |
LEASES
LEASES | 3 Months Ended |
Mar. 31, 2023 | |
Leases [Abstract] | |
LEASES | LEASES As of March 31, 2023, 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 delivered in September 2020. 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. The aggregate base rent due over the initial term of the lease is approximately $0.5 million. Utilizing a discount rate equal to the Company's estimated incremental borrowing rate, the Company established an ROU asset and corresponding lease liability of $0.4 million. Following is a schedule of the future minimum rental commitments for the Company's operating leases reconciled to the lease liability and ROU asset as of March 31, 2023 ( in thousands ): March 31, 2023 2023 (remaining nine months) $ 4,754 2024 6,501 2025 6,673 2026 6,889 2027 7,064 Thereafter 4,781 Total undiscounted future minimum payments 36,662 Present value discount (5,791) Total lease liability 30,871 Unamortized lease incentives (5,329) Cash payments in excess of straight-line lease expense (5,253) Total ROU asset $ 20,289 The weighted-average remaining lease term and weighted-average discount rate of the Company's operating leases are as follows: March 31, 2023 December 31, 2022 Weighted-average remaining lease term in years 5.4 5.7 Weighted-average discount rate 6.48 % 6.48 % |
FAIR VALUE MEASUREMENTS
FAIR VALUE MEASUREMENTS | 3 Months Ended |
Mar. 31, 2023 | |
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 March 31, 2023, the fair value of the Company's 2.5% Convertible Senior Notes due 2025 was $65.2 million and the fair value of the Company's 2.25% Convertible Senior Notes due 2029 was $307.8 million. As of December 31, 2022, the fair value of the Company's 2.5% Convertible Senior Notes due 2025 was $62.9 million and the fair value of the Company's 2.25% Convertible Senior Notes due 2029 was $283.0 million. The fair values were estimated utilizing market quotations and are considered Level 2. The following table presents the Company’s assets and liabilities, measured and recognized at fair value on a recurring basis, classified under the appropriate level of the fair value hierarchy as of March 31, 2023 ( in thousands ): As of March 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 $ 161,376 $ 161,376 $ — $ — Marketable debt securities, available-for-sale 400,137 — 400,137 — Total $ 561,513 $ 161,376 $ 400,137 $ — Liabilities: Business combination-related contingent consideration $ 75,200 $ — $ — $ 75,200 Total $ 75,200 $ — $ — $ 75,200 The following table presents the Company’s assets and liabilities, measured and recognized at fair value on a recurring basis, classified under the appropriate level of the fair value hierarchy as of December 31, 2022 ( in thousands ): As of December 31, 2022 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 $ 61,688 $ 61,688 $ — $ — Marketable debt securities, available-for-sale 388,557 — 388,557 — Total $ 450,245 $ 61,688 $ 388,557 $ — Liabilities: Business combination-related contingent consideration $ 71,200 $ — $ — $ 71,200 Total $ 71,200 $ — $ — $ 71,200 The Company acquired two businesses, related to the Cholbam and Chenodal products, whose purchase price included potential future payments that are contingent on the achievement of certain milestones and percentages of future net sales derived from the products acquired. The Company recorded contingent consideration liabilities at their fair value on the acquisition date and revalues them at the end of each reporting period. In estimating the fair value of the Company’s contingent consideration, the Company uses a Monte Carlo Simulation. The determination of the contingent consideration liabilities requires significant judgements including the appropriateness of the valuation model and reasonableness of estimates and assumptions included in the forecasts of future net sales and the discount rates applied to such forecasts. Changes in these estimates and assumptions could have a significant impact on the fair value of the contingent consideration liabilities. Discount rates used to determine the fair value at March 31, 2023 and December 31, 2022 are as follows: Revenue Discount Payment Discount Cholbam Chenodal March 31, 2023 6.75% 6.75% 7.65% December 31, 2022 7.75% 8.00% 8.10% Based on the fair value hierarchy, the Company classified the fair value measurement of contingent consideration within Level 3 because valuation inputs are based on projected revenues discounted to a present value. The following table sets forth a summary of changes in the estimated fair value of the Company's Level 3 business combination-related contingent consideration for the three months ended March 31, 2023 ( in thousands ): Fair Value Measurements of Acquisition-Related Contingent Consideration 2023 2022 Balance at January 1, $ 71,200 $ 67,100 Changes in the fair value of contingent consideration 6,756 9,080 Contractual payments included in accrued liabilities at March 31, (2,756) (2,680) Balance at March 31, $ 75,200 $ 73,500 |
INTANGIBLE ASSETS
INTANGIBLE ASSETS | 3 Months Ended |
Mar. 31, 2023 | |
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. In March 2023, the Company capitalized a $23.0 million milestone payment to Ligand (and Bristol-Myers Squibb Company ("BMS")) that was triggered upon the accelerated approval of FILSPARI in February 2023. Pursuant to the Ligand License Agreement, the Company is obligated to pay to Ligand (and 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 months ended March 31, 2023, the Company capitalized $0.5 million to intangible assets for royalties owed on net sales of FILSPARI. The cost of the $23.0 million milestone payment 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 March 31, 2023 and December 31, 2022 ( in thousands ): March 31, 2023 December 31, 2022 Finite-lived intangible assets $ 329,767 $ 302,935 Less: accumulated amortization (167,820) (158,833) Net carrying value $ 161,947 $ 144,102 As of March 31, 2023 and December 31, 2022, the Company had goodwill of $0.9 million. The following table summarizes amortization expense for the three months ended March 31, 2023 and 2022 ( in thousands ): Three Months Ended March 31, 2023 2022 Research and development $ 2,394 $ 286 Selling, general and administrative 6,593 6,270 Total amortization expense $ 8,987 $ 6,556 |
CONVERTIBLE NOTES PAYABLE
CONVERTIBLE NOTES PAYABLE | 3 Months Ended |
Mar. 31, 2023 | |
Debt Disclosure [Abstract] | |
CONVERTIBLE NOTES PAYABLE | CONVERTIBLE NOTES PAYABLE The composition of the Company’s convertible senior notes are as follows ( in thousands ): March 31, 2023 December 31, 2022 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 (8,400) (8,750) Unamortized debt issuance costs - 2.50% convertible senior notes due 2025 (780) (859) Total convertible senior notes, net of unamortized debt discount and debt issuance costs $ 375,974 $ 375,545 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 March 31, 2023, 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. We determined the expected life of the debt is equal to the seven-year term of the 2029 Notes. The effective interest rate on the 2029 Notes is 2.74%. Convertible Senior Notes Due 2025 On September 10, 2018, the Company completed a registered underwritten public offering of $276.0 million aggregate principal amount of 2.50% Convertible Senior Notes due 2025 ("2025 Notes"), and entered into a base indenture and supplemental indenture agreement (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 March 31, 2023, 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 three months ended March 31, 2022, which includes the write-off of related deferred financing costs of $3.4 million. After giving effect to the repurchase, the total remaining principal amount outstanding under the 2025 Notes as of March 31, 2023 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 March 31, 2023. The 2025 and 2029 Notes are classified on the Company's Consolidated Balance Sheets at March 31, 2023 as long-tern convertible debt. The following table sets forth total interest expense recognized related to the 2025 and 2029 Notes ( in thousands ): Three Months Ended March 31, 2023 2022 Contractual interest expense $ 2,209 $ 1,843 Amortization of debt issuance costs 429 337 Total interest expense for the 2025 and 2029 Notes $ 2,638 $ 2,180 Total interest expense recognized for the three months ended March 31, 2023 was $2.9 million. Total interest expense recognized for the three months ended March 31, 2022 was $2.5 million. |
ACCRUED EXPENSES
ACCRUED EXPENSES | 3 Months Ended |
Mar. 31, 2023 | |
Payables and Accruals [Abstract] | |
ACCRUED EXPENSES | ACCRUED EXPENSES Accrued expenses at March 31, 2023 and December 31, 2022 consisted of the following ( in thousands ): March 31, 2023 December 31, 2022 Compensation related costs $ 26,839 $ 35,267 Research and development 24,909 26,070 Sales discounts, rebates, and allowances 11,451 13,486 Selling, general and administrative 9,064 8,791 Accrued royalties 7,218 7,755 Miscellaneous accrued expenses 6,285 4,373 Total accrued expenses $ 85,766 $ 95,742 |
NET LOSS PER COMMON SHARE
NET LOSS PER COMMON SHARE | 3 Months Ended |
Mar. 31, 2023 | |
Earnings Per Share [Abstract] | |
NET LOSS PER COMMON SHARE | NET LOSS PER COMMON SHARE Basic and diluted net loss per common share is calculated by dividing net loss applicable to common stockholders by the weighted-average number of common shares outstanding during the period. 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 are exercisable immediately 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. Since the $0.0001 price per share represents little consideration and is non-substantive in relation to the $20.9999 price per pre-funded warrant and the $21.00 price per share of the common stock offered to the public, and as the warrants are immediately exercisable with no further vesting conditions or contingencies associated with them, the shares underlying the warrants are therefore included in the calculation of basic net loss per common share. The Company’s potentially dilutive shares, which include outstanding stock options, restricted stock units, and shares issuable upon conversion of the 2025 Notes and 2029 Notes, are considered to be common stock equivalents and are not included in the calculation of diluted net loss per share because their effect is anti-dilutive. Basic and diluted net loss per share is calculated as follows (net loss amounts are stated in thousands) : Three Months Ended March 31, 2023 2022 Shares Net Loss Loss per common share Shares Net Loss Loss per common share Basic and diluted loss per share 68,174,099 $ (86,331) $ (1.27) 63,132,841 $ (75,971) $ (1.20) The following common stock equivalents have been excluded because they were anti-dilutive: Three Months Ended March 31, 2023 2022 Convertible debt 11,697,952 8,336,091 Options 10,546,780 9,864,889 Restricted stock 3,278,797 1,900,016 Total anti-dilutive shares 25,523,529 20,100,996 |
COMMITMENTS AND CONTINGENCIES
COMMITMENTS AND CONTINGENCIES | 3 Months Ended |
Mar. 31, 2023 | |
Commitments and Contingencies Disclosure [Abstract] | |
COMMITMENTS AND CONTINGENCIES | COMMITMENTS AND CONTINGENCIES Commitments Certain of the Company's contractual arrangements with contract manufacturing organizations ("CMOs") require binding forecasts or commitments to purchase minimum amounts for the manufacture of drug product supply, which may be material to the Company's financial statements. As of March 31, 2023, we have commitments to purchase $36.3 million in active pharmaceutical ingredient, to be delivered in 2023, which is planned to support commercial sales of FILSPARI. Contingencies In October 2021, our Kolbam distributor in France notified us that the French authorities were seeking reimbursement for a portion of Kolbam sales in France during the periods from 2015-2020. During this period, the Company had aggregate revenues from sales of Kolbam in France of approximately $8.0 million. At this time, the Company is not able to estimate the potential liability that may be incurred, if any. Legal Proceedings From time to time in the normal course of business, the Company is subject to various legal matters such as threatened or pending claims or litigation. Although the results of claims and litigation cannot be predicted with certainty, the Company does not believe it is a party to any claim or litigation the outcome of which, if determined adversely to it, would individually or in the aggregate be reasonably expected to have a material adverse effect on its results of operations or financial condition. |
SHARE-BASED COMPENSATION
SHARE-BASED COMPENSATION | 3 Months Ended |
Mar. 31, 2023 | |
Equity [Abstract] | |
SHARE-BASED COMPENSATION | SHARE-BASED COMPENSATION Stock Options The following table summarizes stock option activity during the three months ended March 31, 2023: Shares Underlying Options Weighted Average Exercise Price Weighted Average Remaining Contractual Life (years) Aggregate Intrinsic Value (in thousands) Outstanding at December 31, 2022 9,932,422 $ 21.56 5.79 $ 24,658 Granted 1,011,250 22.35 — — Exercised (148,466) 15.47 — 995 Forfeited/canceled (70,488) 27.62 — — Outstanding at March 31, 2023 10,724,718 $ 21.68 5.95 $ 30,730 Vested and expected to vest at March 31, 2023 10,724,718 $ 21.68 5.95 $ 30,730 At March 31, 2023, unamortized stock compensation for stock options was $38.8 million, with a weighted-average recognition period of 2.8 years. At March 31, 2023, outstanding options to purchase 7.4 million shares of common stock were exercisable with a weighted-average exercise price per share of $20.65. In connection with the retirement of the Company's former Chief Financial Officer, the Board of Directors approved a modification to extend the deadline to exercise each stock option held to the earlier of three months following the last vesting date or the original expiration date of the option, and to continue vesting on the original schedule of any underlying unvested stock options and restricted stock units. The modification resulted in incremental compensation cost of $2.6 million for the three months ended March 31, 2023. Restricted Stock Units Service Based Restricted Stock Units The following table summarizes the Company’s service based restricted stock unit activity during the three months ended March 31, 2023: Number of Restricted Stock Units Weighted Average Grant Date Fair Value Unvested at December 31, 2022 2,343,709 $ 24.65 Granted 1,631,792 22.33 Vested (444,020) 24.62 Forfeited/canceled (26,180) 24.02 Unvested at March 31, 2023 3,505,301 $ 23.58 At March 31, 2023, unamortized stock compensation for service based restricted stock units was $73.8 million a weighted-average recognition period of 3.1 years. Performance Based Restricted Stock Units The following table summarizes the Company’s performance based restricted stock unit activity during the three months ended March 31, 2023: Number of Restricted Stock Units Weighted Average Grant Date Fair Value Unvested at December 31, 2022 157,048 $ 25.24 Granted 66,250 22.40 Vested — — Forfeited/canceled (22,840) 24.86 Unvested at March 31, 2023 200,458 $ 24.34 At March 31, 2023, unamortized stock compensation for performance based restricted stock units was $2.6 million, with a weighted-average recognition period of 1.1 years. Share-Based Compensation The following table sets forth total share-based compensation for the three months ended March 31, 2023 and 2022 ( in thousands ): Three Months Ended March 31, 2023 2022 Research and development $ 4,481 $ 3,168 Selling, general and administrative 9,283 5,018 Total share-based compensation $ 13,764 $ 8,186 |
INVENTORY
INVENTORY | 3 Months Ended |
Mar. 31, 2023 | |
Inventory Disclosure [Abstract] | |
INVENTORY | INVENTORY Inventory consisted of the following at March 31, 2023 and December 31, 2022 ( in thousands ): March 31, 2023 December 31, 2022 Raw materials $ 3,077 $ 3,627 Finished goods 3,635 3,295 Total inventory $ 6,712 $ 6,922 |
ACCOUNTS RECEIVABLE
ACCOUNTS RECEIVABLE | 3 Months Ended |
Mar. 31, 2023 | |
Receivables [Abstract] | |
ACCOUNTS RECEIVABLE | ACCOUNTS RECEIVABLEAccounts receivable, net of reserves for prompt pay discounts and expected credit losses, was $21.5 million and $16.6 million at March 31, 2023 and December 31, 2022, respectively. The total reserves for both periods were immaterial.The Company's evaluation and accounting for credit losses for the current period included an assessment of our aged trade receivables balances and their underlying credit risk characteristics. Our evaluation of past events, current conditions, and reasonable and supportable forecasts about the future resulted in an expectation of immaterial credit losses. |
EQUITY OFFERINGS
EQUITY OFFERINGS | 3 Months Ended |
Mar. 31, 2023 | |
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. 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). An additional $51.9 million were sold under the Company's effective registration statement on Form S-3 (Registration Statement No. 333-259311), which included $20.1 million in the year ended December 31, 2022. The Company did not sell any shares under the ATM Agreement during the three months ended March 31, 2023. As of March 31, 2023, an aggregate of $19.5 million remained eligible for sale under the ATM Agreement. |
SUBSEQUENT EVENTS
SUBSEQUENT EVENTS | 3 Months Ended |
Mar. 31, 2023 | |
Subsequent Events [Abstract] | |
SUBSEQUENT EVENTS | SUBSEQUENT EVENTSOn May 1, 2023, we announced that the pivotal Phase 3 DUPLEX Study did not achieve its two-year primary endpoint with statistical significance over the active control irbesartan. We are continuing to analyze the data to further evaluate the potential for sparsentan as a treatment for FSGS and plan to meet with the regulators to explore a potential path to a submission for sparsentan in FSGS. |
BASIS OF PRESENTATION AND SIG_2
BASIS OF PRESENTATION AND SIGNIFICANT ACCCOUNTING POLICIES (Policies) | 3 Months Ended |
Mar. 31, 2023 | |
Accounting Policies [Abstract] | |
Principles of Consolidation | Principles of ConsolidationThe 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. |
Revenue Recognition and Deductions from Revenue | Revenue RecognitionThe 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. 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 three months ended March 31, 2023 and 2022, the Company recorded a net product revenue increase of $0.4 million and $0.1 million, respectively, related to performance obligations satisfied in previous periods. Government Rebates: The Company calculates the rebates that it will be obligated to provide to government programs and deducts these estimated amounts from its gross product sales at the time the revenues are recognized. Allowances for government rebates and discounts are established based on an estimated allocation of payers and the government-mandated discounts applicable to government-funded programs. Rebate discounts are included in other current liabilities in the accompanying consolidated balance sheets. Commercial Rebates: The Company calculates the rebates it incurs 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 other current liabilities in the accompanying consolidated balance sheets. Prompt Pay Discounts: The Company offers discounts to certain customers for prompt payments. The Company accrues for the calculated prompt pay discount based on the gross amount of each invoice for those customers at the time of sale. Product Returns: Consistent with industry practice, the Company offers its customers a limited right to return product purchased directly from the Company, which is principally based upon the product’s expiration date. 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 judgement to evaluate whether the milestones are probable of being achieved and estimates the amount to include in the transaction price utilizing the most likely amount method. If it is probable that a significant revenue reversal will not occur, the estimated amount is included in the transaction price. Milestone payments that are not within the Company or the licensee’s control, such as regulatory approvals, are considered to be constrained due to a high degree of uncertainty and are not included in the transaction price until such uncertainty is resolved. At the end of each reporting period, the Company re-evaluates the probability of achievement of development milestones and any related constraint and adjusts the estimate of the overall transaction price, if necessary. The Company recognizes aggregate sales-based milestones and royalty payments from product sales of which the license is deemed to be the predominant item to which the royalties relate, at the later of when the related sales occur or when the performance obligation to which the sales-based milestone or royalty has been allocated has been satisfied. Revenue from collaboration and licensing agreements may also include sales of inventory, at cost plus a margin, and is recorded in license and collaboration revenue. The Company utilizes significant judgement to develop estimates of the stand-alone selling price for each distinct performance obligation based upon the relative stand-alone selling price. Variable consideration that relates specifically to the Company’s efforts to satisfy specific performance obligations is allocated entirely to those performance obligations. The stand-alone selling price for license-related performance obligations requires judgement in developing assumptions to project probability-weighted cash flows based upon estimates of forecasted revenues, clinical and regulatory timelines and discount rates. The stand-alone selling price for clinical development performance obligations is based on forecasted expected costs of satisfying a performance obligation plus an appropriate margin. If the licenses to intellectual property are determined to be distinct from the other performance obligations identified in the arrangement and have stand-alone functionality, the Company recognizes revenues from non-refundable, upfront fees allocated to the license when the license is transferred to the licensee and the licensee is able to benefit from the license. For licenses that are not distinct from other promises, the Company applies judgment to assess the nature of the combined performance obligation to determine whether the combined performance obligation is satisfied over time or a point in time and, if over time, the appropriate method of measuring progress for purposes of recognizing revenue from non-refundable, upfront fees. The Company evaluates the measure of progress each reporting period and, if necessary, adjusts the related revenue recognition accordingly. The selection of the method to measure progress towards completion requires judgment and is based on the nature of the products or services to be provided. Revenue is recorded proportionally as costs are incurred. The Company generally utilizes the cost-to-cost method of progress because it best measures the transfer of control to the customer which occurs as the Company incurs costs. Under the cost-to-cost measure of progress, the extent of progress towards completion is measured based on the ratio of costs incurred to date to the total estimated costs at completion of the performance obligation. The Company uses judgment to estimate the total costs expected to complete the clinical development performance obligations, which include subcontractor costs, labor, materials, other direct costs and an allocation of indirect costs. The Company evaluates these cost estimates and the progress each reporting period and adjusts the measure of progress, if necessary. |
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 ingredient to the Company's collaboration partner, 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. Sales of FILSPARI for the three months ended March 31, 2023 primarily consisted of zero-cost inventories, which favorably impacted gross margin for related sales. Prior to the February 2023 FDA approval of FILSPARI (sparsentan), the Company recognized approximately $7.5 million in research and development expenses related to the production of active pharmaceutical ingredient to support the commercial launch of FILSPARI. The Company expects to continue to benefit from the sale of previously expensed inventories through at least 2024. |
Research and Development Expense | Research and Development Expenses Research and development includes expenses related to sparsentan, pegtibatinase, and the Company's other pipeline programs. The Company expenses all research and development costs as they are incurred. The Company's research and development costs are composed of salaries and bonuses, benefits, share-based compensation, license fees, milestones under license agreements, costs paid to third-party contractors to perform research, conduct clinical trials, develop drug materials and delivery devices, manufacture drug product supplies to support clinical development, and associated overhead expenses and facilities costs. The Company charges direct internal and external program costs to the respective development programs. The Company also incurs indirect costs that are not allocated to specific programs because such costs benefit multiple development programs and allow us to increase our pharmaceutical development capabilities. These consist of internal shared resources related to the development and maintenance of systems and processes applicable to all of our programs. |
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 one Phase 1/2 clinical trial and three Phase 3 clinical trials in process that are in varying stages of activity, with ongoing non-clinical support trials. As such, clinical trial expenses will vary depending on all the factors set forth above and may fluctuate significantly from quarter to quarter. |
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. The additional cost basis is subsequently amortized over the remaining life of the license agreement. Consistent with all prior periods since Thiola was acquired, the Company has not accrued any liability for future royalties in excess of the annual contractual minimum at March 31, 2023 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 judgements regarding the purpose and design of the entity, the value of the licensed assets to the entity, the value of the entity’s total assets and the significant activities of the entity. If the Company has a variable interest in the entity as a whole, the Company assesses whether or not the Company is a primary beneficiary of that VIE, based on a number of factors, including: (i) which party has the power to direct the activities that most significantly affect the VIE’s economic performance, (ii) the parties’ contractual rights and responsibilities pursuant to the collaboration agreement, and (iii) which party has the obligation to absorb losses of or the right to receive benefits from the VIE that could be significant to the VIE. If the Company determines that it is the primary beneficiary of a VIE at the onset of the collaboration, the collaboration is treated as a business combination and the Company consolidates the financial statements of the VIE into the Company’s consolidated financial statements. On a quarterly basis, the Company evaluates whether it continues to be the primary beneficiary of the consolidated VIE. If the Company determines that it is no longer the primary beneficiary of a consolidated VIE, it deconsolidates the VIE in the period in which the determination is made. Assets and liabilities recorded as a result of consolidating the financial results of the VIE into the Company’s consolidated balance sheet do not represent additional assets that could be used to satisfy claims against the Company’s general assets or liabilities for which creditors have recourse to the Company’s general assets. |
Recently Adopted and Issued Accounting Pronouncements | Recently Adopted Accounting Pronouncements In August 2020, the FASB issued ASU No. 2020-06, Accounting for Convertible Instruments and Contracts in an Entity's Own Equity. The ASU includes amendments to the guidance on convertible instruments and the derivative scope exception for contracts in an entity's own equity in Subtopic 815-40 and simplifies the accounting for convertible instruments which include beneficial conversion features or cash conversion features by removing certain separation models in Subtopic 470-20. Additionally, the ASU will require entities to use the "if-converted" method when calculating diluted earnings per share for convertible instruments. The ASU is effective for fiscal years beginning after December 15, 2021, including interim periods within those fiscal years. The adoption of the new standard impacted the Company's accounting for its Convertible Senior Notes Due 2025 (2025 Notes), discussed in Note 10, which were previously accounted for using the cash conversion model applied under ASC 470-20, Debt with Conversion and Other Options ("ASC 470-20"). The Company adopted ASU 2020-06 on January 1, 2022 using the modified retrospective method. The cumulative effect of the accounting change as of January 1, 2022 increased the carrying amount of the 2025 Notes by $44.7 million, reduced additional paid-in capital by $74.9 million, and reduced accumulated deficit by $30.2 million. 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. |
Financial Instruments and Fair Value | 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. |
Income (Loss) Per Common Share | Basic and diluted net loss per common share is calculated by dividing net loss applicable to common stockholders by the weighted-average number of common shares outstanding during the period. 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 are exercisable immediately 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. Since the $0.0001 price per share represents little consideration and is non-substantive in relation to the $20.9999 price per pre-funded warrant and the $21.00 price per share of the common stock offered to the public, and as the warrants are immediately exercisable with no further vesting conditions or contingencies associated with them, the shares underlying the warrants are therefore included in the calculation of basic net loss per common share. 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. |
BASIS OF PRESENTATION AND SIG_3
BASIS OF PRESENTATION AND SIGNIFICANT ACCCOUNTING POLICIES (Tables) | 3 Months Ended |
Mar. 31, 2023 | |
Accounting Policies [Abstract] | |
Schedule of Cost and Goods Sold | The following table summarizes cost of goods sold for the three months ended March 31, 2023 and 2022 ( in thousands ): Three Months Ended March 31, 2023 2022 Cost of goods sold - product sales $ 2,088 $ 2,138 Cost of goods sold - license and collaboration 3,037 — Total cost of goods sold $ 5,125 $ 2,138 |
REVENUE RECOGNITION (Tables)
REVENUE RECOGNITION (Tables) | 3 Months Ended |
Mar. 31, 2023 | |
Revenue from Contract with Customer [Abstract] | |
Schedule of Net Product Revenue | The following table summarizes net product sales for the three months ended March 31, 2023 and 2022 ( in thousands ): Three Months Ended March 31, 2023 2022 Bile acid products $ 26,105 $ 25,075 Tiopronin products 21,174 21,368 FILSPARI 3,004 — Total net product sales $ 50,283 $ 46,443 |
MARKETABLE DEBT SECURITIES (Tab
MARKETABLE DEBT SECURITIES (Tables) | 3 Months Ended |
Mar. 31, 2023 | |
Investments, Debt and Equity Securities [Abstract] | |
Schedule of Marketable Debt Securities | Marketable debt securities consisted of the following ( in thousands ): March 31, 2023 December 31, 2022 Marketable debt securities: Commercial paper $ 133,869 $ 123,647 Corporate debt securities 185,474 224,055 Securities of government sponsored entities 80,794 40,855 Total marketable debt securities $ 400,137 $ 388,557 |
Schedule of Available for Sale Securities | The following is a summary of short-term marketable debt securities classified as available-for-sale as of March 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 $ 134,218 $ — $ (349) $ 133,869 Corporate debt securities Less than 1 118,324 — (883) 117,441 Securities of government-sponsored entities Less than 1 27,845 9 (107) 27,747 Total maturity less than 1 year 280,387 9 (1,339) 279,057 Corporate debt securities 1 to 2 68,268 109 (344) 68,033 Securities of government-sponsored entities 1 to 2 53,140 117 (210) 53,047 Total maturity 1 to 2 years 121,408 226 (554) 121,080 Total available-for-sale marketable debt securities $ 401,795 $ 235 $ (1,893) $ 400,137 The following is a summary of short-term marketable debt securities classified as available-for-sale as of December 31, 2022 ( in thousands ): Remaining Contractual Maturity Amortized Cost Unrealized Gains Unrealized Losses Aggregate Estimated Fair Value Marketable debt securities: Commercial paper Less than 1 $ 124,301 $ 2 $ (656) $ 123,647 Corporate debt securities Less than 1 155,841 — (1,355) 154,486 Securities of government-sponsored entities Less than 1 7,473 — (80) 7,393 Total maturity less than 1 year 287,615 2 (2,091) 285,526 Corporate debt securities 1 to 2 70,195 33 (659) 69,569 Securities of government-sponsored entities 1 to 2 33,702 6 (246) 33,462 Total maturity 1 to 2 years 103,897 39 (905) 103,031 Total available-for-sale securities $ 391,512 $ 41 $ (2,996) $ 388,557 |
Schedule 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 March 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 $ 133,869 $ 349 $ — $ — $ 133,869 $ 349 Corporate debt securities 79,397 826 70,900 401 150,297 1,227 Securities of government-sponsored entities 51,225 292 2,486 25 53,711 317 Total $ 264,491 $ 1,467 $ 73,386 $ 426 $ 337,877 $ 1,893 The following is a summary of available-for-sale marketable debt securities in an unrealized loss position with no credit losses reported as of December 31, 2022 ( in thousands ): Less Than 12 Months 12 Months or Greater Total Description of Securities Fair Value Unrealized Losses Fair Value Unrealized Losses Fair Value Unrealized Losses Commercial paper $ 117,853 $ 656 $ — $ — $ 117,853 $ 656 Corporate debt securities 99,066 1,041 107,964 973 207,030 2,014 Securities of government-sponsored entities 31,402 263 4,456 63 35,858 326 Total $ 248,321 $ 1,960 $ 112,420 $ 1,036 $ 360,741 $ 2,996 |
LEASES (Tables)
LEASES (Tables) | 3 Months Ended |
Mar. 31, 2023 | |
Leases [Abstract] | |
Schedule of Future Minimum Rent Commitments | 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 March 31, 2023 ( in thousands ): March 31, 2023 2023 (remaining nine months) $ 4,754 2024 6,501 2025 6,673 2026 6,889 2027 7,064 Thereafter 4,781 Total undiscounted future minimum payments 36,662 Present value discount (5,791) Total lease liability 30,871 Unamortized lease incentives (5,329) Cash payments in excess of straight-line lease expense (5,253) Total ROU asset $ 20,289 |
Schedule of Weighted-average Remaining Lease Term and Discount Rate | The weighted-average remaining lease term and weighted-average discount rate of the Company's operating leases are as follows: March 31, 2023 December 31, 2022 Weighted-average remaining lease term in years 5.4 5.7 Weighted-average discount rate 6.48 % 6.48 % |
FAIR VALUE MEASUREMENTS (Tables
FAIR VALUE MEASUREMENTS (Tables) | 3 Months Ended |
Mar. 31, 2023 | |
Fair Value Disclosures [Abstract] | |
Schedule of Fair Value on a Recurring Basis | The following table presents the Company’s assets and liabilities, measured and recognized at fair value on a recurring basis, classified under the appropriate level of the fair value hierarchy as of March 31, 2023 ( in thousands ): As of March 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 $ 161,376 $ 161,376 $ — $ — Marketable debt securities, available-for-sale 400,137 — 400,137 — Total $ 561,513 $ 161,376 $ 400,137 $ — Liabilities: Business combination-related contingent consideration $ 75,200 $ — $ — $ 75,200 Total $ 75,200 $ — $ — $ 75,200 The following table presents the Company’s assets and liabilities, measured and recognized at fair value on a recurring basis, classified under the appropriate level of the fair value hierarchy as of December 31, 2022 ( in thousands ): As of December 31, 2022 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 $ 61,688 $ 61,688 $ — $ — Marketable debt securities, available-for-sale 388,557 — 388,557 — Total $ 450,245 $ 61,688 $ 388,557 $ — Liabilities: Business combination-related contingent consideration $ 71,200 $ — $ — $ 71,200 Total $ 71,200 $ — $ — $ 71,200 |
Schedule of Discount Rates Used | Discount rates used to determine the fair value at March 31, 2023 and December 31, 2022 are as follows: Revenue Discount Payment Discount Cholbam Chenodal March 31, 2023 6.75% 6.75% 7.65% December 31, 2022 7.75% 8.00% 8.10% |
Schedule of Fair Value Measurements of Acquisition-related Contingent Consideration | The following table sets forth a summary of changes in the estimated fair value of the Company's Level 3 business combination-related contingent consideration for the three months ended March 31, 2023 ( in thousands ): Fair Value Measurements of Acquisition-Related Contingent Consideration 2023 2022 Balance at January 1, $ 71,200 $ 67,100 Changes in the fair value of contingent consideration 6,756 9,080 Contractual payments included in accrued liabilities at March 31, (2,756) (2,680) Balance at March 31, $ 75,200 $ 73,500 |
INTANGIBLE ASSETS (Tables)
INTANGIBLE ASSETS (Tables) | 3 Months Ended |
Mar. 31, 2023 | |
Goodwill and Intangible Assets Disclosure [Abstract] | |
Schedule of Finite-lived Amortizable Intangible Assets | The following table sets forth amortizable intangible assets as of March 31, 2023 and December 31, 2022 ( in thousands ): March 31, 2023 December 31, 2022 Finite-lived intangible assets $ 329,767 $ 302,935 Less: accumulated amortization (167,820) (158,833) Net carrying value $ 161,947 $ 144,102 |
Schedule of Amortization Expense | The following table summarizes amortization expense for the three months ended March 31, 2023 and 2022 ( in thousands ): Three Months Ended March 31, 2023 2022 Research and development $ 2,394 $ 286 Selling, general and administrative 6,593 6,270 Total amortization expense $ 8,987 $ 6,556 |
CONVERTIBLE NOTES PAYABLE (Tabl
CONVERTIBLE NOTES PAYABLE (Tables) | 3 Months Ended |
Mar. 31, 2023 | |
Debt Disclosure [Abstract] | |
Schedule of Long-term Debt Instruments | The composition of the Company’s convertible senior notes are as follows ( in thousands ): March 31, 2023 December 31, 2022 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 (8,400) (8,750) Unamortized debt issuance costs - 2.50% convertible senior notes due 2025 (780) (859) Total convertible senior notes, net of unamortized debt discount and debt issuance costs $ 375,974 $ 375,545 The following table sets forth total interest expense recognized related to the 2025 and 2029 Notes ( in thousands ): Three Months Ended March 31, 2023 2022 Contractual interest expense $ 2,209 $ 1,843 Amortization of debt issuance costs 429 337 Total interest expense for the 2025 and 2029 Notes $ 2,638 $ 2,180 |
ACCRUED EXPENSES (Tables)
ACCRUED EXPENSES (Tables) | 3 Months Ended |
Mar. 31, 2023 | |
Payables and Accruals [Abstract] | |
Schedule of Accrued Expenses | Accrued expenses at March 31, 2023 and December 31, 2022 consisted of the following ( in thousands ): March 31, 2023 December 31, 2022 Compensation related costs $ 26,839 $ 35,267 Research and development 24,909 26,070 Sales discounts, rebates, and allowances 11,451 13,486 Selling, general and administrative 9,064 8,791 Accrued royalties 7,218 7,755 Miscellaneous accrued expenses 6,285 4,373 Total accrued expenses $ 85,766 $ 95,742 |
NET LOSS PER COMMON SHARE (Tabl
NET LOSS PER COMMON SHARE (Tables) | 3 Months Ended |
Mar. 31, 2023 | |
Earnings Per Share [Abstract] | |
Schedule of Basic and Diluted Net Loss Per Share | Basic and diluted net loss per share is calculated as follows (net loss amounts are stated in thousands) : Three Months Ended March 31, 2023 2022 Shares Net Loss Loss per common share Shares Net Loss Loss per common share Basic and diluted loss per share 68,174,099 $ (86,331) $ (1.27) 63,132,841 $ (75,971) $ (1.20) |
Schedule 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 March 31, 2023 2022 Convertible debt 11,697,952 8,336,091 Options 10,546,780 9,864,889 Restricted stock 3,278,797 1,900,016 Total anti-dilutive shares 25,523,529 20,100,996 |
SHARE-BASED COMPENSATION (Table
SHARE-BASED COMPENSATION (Tables) | 3 Months Ended |
Mar. 31, 2023 | |
Equity [Abstract] | |
Schedule of Stock Option Issuances and Balances Outstanding | The following table summarizes stock option activity during the three months ended March 31, 2023: Shares Underlying Options Weighted Average Exercise Price Weighted Average Remaining Contractual Life (years) Aggregate Intrinsic Value (in thousands) Outstanding at December 31, 2022 9,932,422 $ 21.56 5.79 $ 24,658 Granted 1,011,250 22.35 — — Exercised (148,466) 15.47 — 995 Forfeited/canceled (70,488) 27.62 — — Outstanding at March 31, 2023 10,724,718 $ 21.68 5.95 $ 30,730 Vested and expected to vest at March 31, 2023 10,724,718 $ 21.68 5.95 $ 30,730 |
Schedule of Service Based Restricted Stock Activity | The following table summarizes the Company’s service based restricted stock unit activity during the three months ended March 31, 2023: Number of Restricted Stock Units Weighted Average Grant Date Fair Value Unvested at December 31, 2022 2,343,709 $ 24.65 Granted 1,631,792 22.33 Vested (444,020) 24.62 Forfeited/canceled (26,180) 24.02 Unvested at March 31, 2023 3,505,301 $ 23.58 |
Schedule of Performance Based Restricted Stock Activity | The following table summarizes the Company’s performance based restricted stock unit activity during the three months ended March 31, 2023: Number of Restricted Stock Units Weighted Average Grant Date Fair Value Unvested at December 31, 2022 157,048 $ 25.24 Granted 66,250 22.40 Vested — — Forfeited/canceled (22,840) 24.86 Unvested at March 31, 2023 200,458 $ 24.34 |
Schedule of Share Based Compensation Expenses | The following table sets forth total share-based compensation for the three months ended March 31, 2023 and 2022 ( in thousands ): Three Months Ended March 31, 2023 2022 Research and development $ 4,481 $ 3,168 Selling, general and administrative 9,283 5,018 Total share-based compensation $ 13,764 $ 8,186 |
INVENTORY (Tables)
INVENTORY (Tables) | 3 Months Ended |
Mar. 31, 2023 | |
Inventory Disclosure [Abstract] | |
Schedule of Inventory | Inventory consisted of the following at March 31, 2023 and December 31, 2022 ( in thousands ): March 31, 2023 December 31, 2022 Raw materials $ 3,077 $ 3,627 Finished goods 3,635 3,295 Total inventory $ 6,712 $ 6,922 |
BASIS OF PRESENTATION AND SIG_4
BASIS OF PRESENTATION AND SIGNIFICANT ACCCOUNTING POLICIES - Cost of Goods Sold (Details) - USD ($) $ in Thousands | 3 Months Ended | |
Mar. 31, 2023 | Mar. 31, 2022 | |
New Accounting Pronouncements or Change in Accounting Principle [Line Items] | ||
Cost of goods sold | $ 5,125 | $ 2,138 |
Net product sales | ||
New Accounting Pronouncements or Change in Accounting Principle [Line Items] | ||
Cost of goods sold | 2,088 | 2,138 |
License and collaboration revenue | ||
New Accounting Pronouncements or Change in Accounting Principle [Line Items] | ||
Cost of goods sold | $ 3,037 | $ 0 |
BASIS OF PRESENTATION AND SIG_5
BASIS OF PRESENTATION AND SIGNIFICANT ACCCOUNTING POLICIES - Narrative (Details) $ in Thousands | 3 Months Ended | ||
Mar. 31, 2023 USD ($) clinicalTrial | Dec. 31, 2022 USD ($) | Jan. 01, 2022 USD ($) | |
New Accounting Pronouncements or Change in Accounting Principle [Line Items] | |||
Research and development | $ 7,500 | ||
Number of Phase1/2 clinical trials in process | clinicalTrial | 1 | ||
Number of Phase 3 clinical trials in process | clinicalTrial | 3 | ||
Reduction in additional paid-in capital | $ (1,291,863) | $ (1,059,975) | |
Reduction in accumulated deficit | $ (1,100,554) | $ (1,014,223) | |
Cumulative Effect, Period of Adoption, Adjustment | Accounting Standards Update 2020-06 | |||
New Accounting Pronouncements or Change in Accounting Principle [Line Items] | |||
Convertible notes payable | $ 44,700 | ||
Reduction in additional paid-in capital | 74,900 | ||
Reduction in accumulated deficit | $ 30,200 |
REVENUE RECOGNITION - Additiona
REVENUE RECOGNITION - Additional Information (Details) - USD ($) $ in Millions | 3 Months Ended | |
Mar. 31, 2023 | Mar. 31, 2022 | |
Disaggregation of Revenue [Line Items] | ||
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 other 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. | |
Net revenue | $ 0.4 | $ 0.1 |
Geographic Concentration Risk | Revenue Benchmark | United States | ||
Disaggregation of Revenue [Line Items] | ||
Concentration risk, percentage | 98% | |
Geographic Concentration Risk | Revenue Benchmark | Canada | ||
Disaggregation of Revenue [Line Items] | ||
Concentration risk, percentage | 1% | |
Geographic Concentration Risk | Revenue Benchmark | Rest of World | ||
Disaggregation of Revenue [Line Items] | ||
Concentration risk, percentage | 1% |
REVENUE RECOGNITION - Net Produ
REVENUE RECOGNITION - Net Product Revenue (Details) - USD ($) $ in Thousands | 3 Months Ended | |
Mar. 31, 2023 | Mar. 31, 2022 | |
Disaggregation of Revenue [Line Items] | ||
Total revenue | $ 56,993 | $ 48,487 |
Net product sales | ||
Disaggregation of Revenue [Line Items] | ||
Total revenue | 50,283 | 46,443 |
Bile acid products | ||
Disaggregation of Revenue [Line Items] | ||
Total revenue | 26,105 | 25,075 |
Tiopronin products | ||
Disaggregation of Revenue [Line Items] | ||
Total revenue | 21,174 | 21,368 |
FILSPARI | ||
Disaggregation of Revenue [Line Items] | ||
Total revenue | $ 3,004 | $ 0 |
COLLABORATION AND LICENSE AGR_2
COLLABORATION AND LICENSE AGREEMENTS (Details) $ in Thousands | 3 Months Ended | 12 Months Ended | |||
Sep. 15, 2021 USD ($) performance_obligation | Mar. 31, 2023 USD ($) | Mar. 31, 2022 USD ($) | Dec. 31, 2021 USD ($) | Dec. 31, 2022 USD ($) | |
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items] | |||||
Revenue | $ 56,993 | $ 48,487 | |||
Deferred revenue, current portion | 10,975 | $ 11,976 | |||
License | |||||
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items] | |||||
Revenue | 6,710 | $ 2,044 | |||
Sale of Active Pharmaceutical Ingredient | |||||
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items] | |||||
Revenue | 3,300 | ||||
Clinical Development Activity | |||||
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items] | |||||
Revenue | 3,400 | ||||
Collaborative Arrangement | Vifor Pharma | |||||
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items] | |||||
Upfront payment | $ 55,000 | ||||
Maximum milestone payments | $ 845,000 | ||||
Percentage of royalty on net sales receives | 40% | ||||
Force majeure event period threshold | 6 months | ||||
Revenue | $ 55,000 | ||||
Number of performance obligations | performance_obligation | 2 | ||||
Deferred revenue | $ 43,000 | 19,800 | |||
Deferred revenue, current portion | $ 11,000 | ||||
Collaborative Arrangement | Vifor Pharma | Regulatory and Market Access Milestone | |||||
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items] | |||||
Maximum milestone payments | 135,000 | ||||
Collaborative Arrangement | Vifor Pharma | Sales-based Milestone Payments | |||||
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items] | |||||
Maximum milestone payments | $ 655,000 | ||||
Collaborative Arrangement | Vifor Pharma | License | |||||
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items] | |||||
Revenue | $ 12,000 |
MARKETABLE DEBT SECURITIES - Ma
MARKETABLE DEBT SECURITIES - Marketable Securities (Details) - USD ($) $ in Thousands | Mar. 31, 2023 | Dec. 31, 2022 |
Debt Securities, Available-for-sale [Line Items] | ||
Total marketable debt securities | $ 400,137 | $ 388,557 |
Commercial paper | ||
Debt Securities, Available-for-sale [Line Items] | ||
Total marketable debt securities | 133,869 | 123,647 |
Corporate debt securities | ||
Debt Securities, Available-for-sale [Line Items] | ||
Total marketable debt securities | 185,474 | 224,055 |
Securities of government sponsored entities | ||
Debt Securities, Available-for-sale [Line Items] | ||
Total marketable debt securities | $ 80,794 | $ 40,855 |
MARKETABLE DEBT SECURITIES - Av
MARKETABLE DEBT SECURITIES - Available for Sale Securities (Details) - USD ($) $ in Thousands | Mar. 31, 2023 | Dec. 31, 2022 |
Debt Securities, Available-for-sale [Line Items] | ||
Marketable debt securities, available-for-sale, amortized cost basis, current | $ 280,387 | $ 287,615 |
Marketable debt securities, available for sale, unrealized gain, current | 9 | 2 |
Marketable debt securities, available for sale, unrealized loss, current | (1,339) | (2,091) |
Marketable debt securities, available-for-sale, current | 279,057 | 285,526 |
Marketable debt securities, available-for-sale, amortized cost basis, noncurrent | 121,408 | 103,897 |
Marketable debt securities, available for sale, unrealized gain, noncurrent | 226 | 39 |
Marketable debt securities, available for sale, unrealized loss, noncurrent | (554) | (905) |
Marketable debt securities, available-for-sale, noncurrent | 121,080 | 103,031 |
Marketable debt securities, available-for-sale, amortized cost | 401,795 | 391,512 |
Marketable debt securities, available for sale, unrealized gains | 235 | 41 |
Marketable debt securities, available-for-sale, unrealized loss | (1,893) | (2,996) |
Total available-for-sale marketable debt securities | 400,137 | 388,557 |
Commercial paper | ||
Debt Securities, Available-for-sale [Line Items] | ||
Marketable debt securities, available-for-sale, amortized cost basis, current | 134,218 | 124,301 |
Marketable debt securities, available for sale, unrealized gain, current | 0 | 2 |
Marketable debt securities, available for sale, unrealized loss, current | (349) | (656) |
Marketable debt securities, available-for-sale, current | 133,869 | 123,647 |
Total available-for-sale marketable debt securities | 133,869 | 123,647 |
Corporate debt securities | ||
Debt Securities, Available-for-sale [Line Items] | ||
Marketable debt securities, available-for-sale, amortized cost basis, current | 118,324 | 155,841 |
Marketable debt securities, available for sale, unrealized gain, current | 0 | 0 |
Marketable debt securities, available for sale, unrealized loss, current | (883) | (1,355) |
Marketable debt securities, available-for-sale, current | 117,441 | 154,486 |
Marketable debt securities, available-for-sale, amortized cost basis, noncurrent | 68,268 | 70,195 |
Marketable debt securities, available for sale, unrealized gain, noncurrent | 109 | 33 |
Marketable debt securities, available for sale, unrealized loss, noncurrent | (344) | (659) |
Marketable debt securities, available-for-sale, noncurrent | 68,033 | 69,569 |
Total available-for-sale marketable debt securities | 185,474 | 224,055 |
Securities of government sponsored entities | ||
Debt Securities, Available-for-sale [Line Items] | ||
Marketable debt securities, available-for-sale, amortized cost basis, current | 27,845 | 7,473 |
Marketable debt securities, available for sale, unrealized gain, current | 9 | 0 |
Marketable debt securities, available for sale, unrealized loss, current | (107) | (80) |
Marketable debt securities, available-for-sale, current | 27,747 | 7,393 |
Marketable debt securities, available-for-sale, amortized cost basis, noncurrent | 53,140 | 33,702 |
Marketable debt securities, available for sale, unrealized gain, noncurrent | 117 | 6 |
Marketable debt securities, available for sale, unrealized loss, noncurrent | (210) | (246) |
Marketable debt securities, available-for-sale, noncurrent | 53,047 | 33,462 |
Total available-for-sale marketable debt securities | $ 80,794 | $ 40,855 |
MARKETABLE DEBT SECURITIES - Ad
MARKETABLE DEBT SECURITIES - Additional Information (Details) - USD ($) $ in Millions | 3 Months Ended | ||
Mar. 31, 2023 | Mar. 31, 2022 | Dec. 31, 2022 | |
Investments, Debt and Equity Securities [Abstract] | |||
Proceeds from sale of debt securities | $ 94.1 | $ 64.7 | |
Purchase of debt securities | 103.2 | $ 26.3 | |
Accrued interest receivable | 2 | $ 1.9 | |
Available-for-sale marketable debt securities in an unrealized loss position | $ 339.8 | $ 363.7 |
MARKETABLE DEBT SECURITIES - Se
MARKETABLE DEBT SECURITIES - Securities in an Unrealized Loss Position (Details) - USD ($) $ in Thousands | Mar. 31, 2023 | Dec. 31, 2022 |
Debt Securities, Available-for-sale [Line Items] | ||
Less than 12 months, fair value | $ 264,491 | $ 248,321 |
Less than 12 months, unrealized losses | 1,467 | 1,960 |
12 months or greater, fair value | 73,386 | 112,420 |
12 months or greater, unrealized losses | 426 | 1,036 |
Total, fair value | 337,877 | 360,741 |
Total, unrealized losses | 1,893 | 2,996 |
Commercial paper | ||
Debt Securities, Available-for-sale [Line Items] | ||
Less than 12 months, fair value | 133,869 | 117,853 |
Less than 12 months, unrealized losses | 349 | 656 |
12 months or greater, fair value | 0 | 0 |
12 months or greater, unrealized losses | 0 | 0 |
Total, fair value | 133,869 | 117,853 |
Total, unrealized losses | 349 | 656 |
Corporate debt securities | ||
Debt Securities, Available-for-sale [Line Items] | ||
Less than 12 months, fair value | 79,397 | 99,066 |
Less than 12 months, unrealized losses | 826 | 1,041 |
12 months or greater, fair value | 70,900 | 107,964 |
12 months or greater, unrealized losses | 401 | 973 |
Total, fair value | 150,297 | 207,030 |
Total, unrealized losses | 1,227 | 2,014 |
Securities of government-sponsored entities | ||
Debt Securities, Available-for-sale [Line Items] | ||
Less than 12 months, fair value | 51,225 | 31,402 |
Less than 12 months, unrealized losses | 292 | 263 |
12 months or greater, fair value | 2,486 | 4,456 |
12 months or greater, unrealized losses | 25 | 63 |
Total, fair value | 53,711 | 35,858 |
Total, unrealized losses | $ 317 | $ 326 |
VARIABLE INTEREST ENTITIES (Det
VARIABLE INTEREST ENTITIES (Details) $ in Millions | Mar. 08, 2022 USD ($) |
Collaborative Arrangement | PharmaKrysto, LTD | |
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items] | |
Collaboration agreement payment | $ 0.4 |
Research and development expenses, estimated | 5 |
Collaborative arrangement, option to purchase additional shares of VIE, amount | 1 |
Collaborative arrangement, option to purchase remaining shares of VIE, amount | 5 |
Milestone payments contingently due | $ 16 |
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 | $ 0.6 |
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 | |||
Mar. 31, 2023 USD ($) lease | Mar. 31, 2022 USD ($) | Dec. 31, 2022 USD ($) | Dec. 31, 2020 USD ($) | |
Lessee, Lease, Description [Line Items] | ||||
Operating lease right of use assets | $ 20,289 | $ 21,000 | ||
Lease liability | $ 30,871 | |||
Operating lease extension term | 5 years | |||
Operating lease expense | $ 1,200 | $ 1,200 | ||
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 | |||
Esprit Investments Limited | ||||
Lessee, Lease, Description [Line Items] | ||||
Number of operating leases | lease | 1 | |||
Operating lease right of use assets | $ 400 | |||
Lease liability | $ 400 | |||
Operating lease extension term | 5 years | |||
Base rent due over the initial term of the lease | $ 500 |
LEASES - Future Minimum Rent Co
LEASES - Future Minimum Rent Commitments (Details) - USD ($) $ in Thousands | Mar. 31, 2023 | Dec. 31, 2022 |
Leases [Abstract] | ||
2023 (remaining nine months) | $ 4,754 | |
2024 | 6,501 | |
2025 | 6,673 | |
2026 | 6,889 | |
2027 | 7,064 | |
Thereafter | 4,781 | |
Total undiscounted future minimum payments | 36,662 | |
Present value discount | (5,791) | |
Total lease liability | 30,871 | |
Unamortized lease incentives | (5,329) | |
Cash payments in excess of straight-line lease expense | (5,253) | |
Total ROU asset | $ 20,289 | $ 21,000 |
LEASES - Weighted-Average Remai
LEASES - Weighted-Average Remaining Lease Term and Discount Rate (Details) | Mar. 31, 2023 | Dec. 31, 2022 |
Leases [Abstract] | ||
Weighted-average remaining lease term in years | 5 years 4 months 24 days | 5 years 8 months 12 days |
Weighted-average discount rate | 6.48% | 6.48% |
FAIR VALUE MEASUREMENTS - Addit
FAIR VALUE MEASUREMENTS - Additional Information (Details) $ in Millions | 3 Months Ended | |
Mar. 31, 2023 USD ($) business | Dec. 31, 2022 USD ($) | |
Business Acquisition [Line Items] | ||
Number of businesses acquired | business | 2 | |
Senior Notes Due 2025 | Senior Notes | ||
Business Acquisition [Line Items] | ||
Interest rate percentage | 2.50% | |
Fair value of convertible debt | $ 65.2 | $ 62.9 |
Senior Notes Due 2029 | Senior Notes | ||
Business Acquisition [Line Items] | ||
Interest rate percentage | 2.25% | |
Fair value of convertible debt | $ 307.8 | $ 283 |
FAIR VALUE MEASUREMENTS - Asset
FAIR VALUE MEASUREMENTS - Assets and Liabilities (Details) - USD ($) $ in Thousands | Mar. 31, 2023 | Dec. 31, 2022 |
Assets: | ||
Cash and Cash Equivalents | $ 161,376 | $ 61,688 |
Marketable debt securities, available-for-sale | 400,137 | 388,557 |
Total | 561,513 | 450,245 |
Liabilities: | ||
Business combination-related contingent consideration | 75,200 | 71,200 |
Total | 75,200 | 71,200 |
Quoted prices in active markets (Level 1) | ||
Assets: | ||
Cash and Cash Equivalents | 161,376 | 61,688 |
Marketable debt securities, available-for-sale | 0 | 0 |
Total | 161,376 | 61,688 |
Liabilities: | ||
Business combination-related contingent consideration | 0 | 0 |
Total | 0 | 0 |
Significant other observable inputs (Level 2) | ||
Assets: | ||
Cash and Cash Equivalents | 0 | 0 |
Marketable debt securities, available-for-sale | 400,137 | 388,557 |
Total | 400,137 | 388,557 |
Liabilities: | ||
Business combination-related contingent consideration | 0 | 0 |
Total | 0 | 0 |
Significant unobservable inputs (Level 3) | ||
Assets: | ||
Cash and Cash Equivalents | 0 | 0 |
Marketable debt securities, available-for-sale | 0 | 0 |
Total | 0 | 0 |
Liabilities: | ||
Business combination-related contingent consideration | 75,200 | 71,200 |
Total | $ 75,200 | $ 71,200 |
FAIR VALUE MEASUREMENTS - Disco
FAIR VALUE MEASUREMENTS - Discount Rates Used (Details) | Mar. 31, 2023 | Dec. 31, 2022 |
Payment Discount | ||
Fair Value Measurement Inputs and Valuation Techniques [Line Items] | ||
Measurement input | 0.0765 | 0.0810 |
Cholbam | Revenue Discount | ||
Fair Value Measurement Inputs and Valuation Techniques [Line Items] | ||
Measurement input | 0.0675 | 0.0775 |
Chenodal | Revenue Discount | ||
Fair Value Measurement Inputs and Valuation Techniques [Line Items] | ||
Measurement input | 0.0675 | 0.0800 |
FAIR VALUE MEASUREMENTS - Acqui
FAIR VALUE MEASUREMENTS - Acquisition-related Contingent Consideration (Details) - USD ($) $ in Thousands | 3 Months Ended | |
Mar. 31, 2023 | Mar. 31, 2022 | |
Fair Value Measurements of Acquisition-Related Contingent Consideration (Level 3) | ||
Beginning balance | $ 71,200 | $ 67,100 |
Changes in the fair value of contingent consideration | 6,756 | 9,080 |
Contractual payments included in accrued liabilities at March 31, | (2,756) | (2,680) |
Ending balance | $ 75,200 | $ 73,500 |
INTANGIBLE ASSETS - Additional
INTANGIBLE ASSETS - Additional Information (Details) - USD ($) $ in Thousands | 1 Months Ended | 3 Months Ended | ||
Mar. 31, 2023 | Mar. 31, 2023 | Mar. 31, 2022 | Dec. 31, 2022 | |
Finite-Lived Intangible Assets [Line Items] | ||||
Payments to date under terms of agreement | $ 27,337 | $ 5,136 | ||
Goodwill | $ 900 | 900 | $ 900 | |
Ligand License Agreement | ||||
Finite-Lived Intangible Assets [Line Items] | ||||
Substantial payments payable upon achievement of milestones | 114,100 | $ 114,100 | ||
Payments to date under terms of agreement | $ 23,000 | |||
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 | $ 500 |
INTANGIBLE ASSETS - Amortizable
INTANGIBLE ASSETS - Amortizable Intangible Assets (Details) - USD ($) $ in Thousands | Mar. 31, 2023 | Dec. 31, 2022 |
Goodwill and Intangible Assets Disclosure [Abstract] | ||
Finite-lived intangible assets | $ 329,767 | $ 302,935 |
Less: accumulated amortization | (167,820) | (158,833) |
Net carrying value | $ 161,947 | $ 144,102 |
INTANGIBLE ASSETS - Amortizatio
INTANGIBLE ASSETS - Amortization (Details) - USD ($) $ in Thousands | 3 Months Ended | |
Mar. 31, 2023 | Mar. 31, 2022 | |
Finite-Lived Intangible Assets [Line Items] | ||
Total amortization expense | $ 8,987 | $ 6,556 |
Research and development | ||
Finite-Lived Intangible Assets [Line Items] | ||
Total amortization expense | 2,394 | 286 |
Selling, general and administrative | ||
Finite-Lived Intangible Assets [Line Items] | ||
Total amortization expense | $ 6,593 | $ 6,270 |
CONVERTIBLE NOTES PAYABLE - Sch
CONVERTIBLE NOTES PAYABLE - Schedule of Carrying Amount of Debt (Details) - Senior Notes - USD ($) $ in Thousands | Mar. 31, 2023 | Dec. 31, 2022 |
Debt Instrument [Line Items] | ||
Total convertible senior notes, net of unamortized debt discount and debt issuance costs | $ 375,974 | $ 375,545 |
Senior Notes Due 2029 | ||
Debt Instrument [Line Items] | ||
Interest rate percentage | 2.25% | |
Convertible senior notes | $ 316,250 | 316,250 |
Unamortized debt issuance costs | $ (8,400) | (8,750) |
Senior Notes Due 2025 | ||
Debt Instrument [Line Items] | ||
Interest rate percentage | 2.50% | |
Convertible senior notes | $ 68,904 | 68,904 |
Unamortized debt issuance costs | $ (780) | $ (859) |
CONVERTIBLE NOTES PAYABLE - Add
CONVERTIBLE NOTES PAYABLE - Additional Information (Details) $ / shares in Units, $ in Thousands | 3 Months Ended | |||
Mar. 11, 2022 USD ($) | Sep. 10, 2018 USD ($) | Mar. 31, 2023 USD ($) day $ / shares | Mar. 31, 2022 USD ($) | |
Debt Instrument [Line Items] | ||||
Debt instrument, repurchase amount Including accrued and unpaid interest | $ 213,800 | |||
Loss on extinguishment of debt | $ 0 | $ 7,578 | ||
Write off of deferred debt financing costs | 3,400 | |||
Interest expense recognized | $ 2,900 | $ 2,500 | ||
Senior Notes | Senior Notes Due 2029 | ||||
Debt Instrument [Line Items] | ||||
Long-term debt, excluding current maturities | 316,300 | |||
Interest rate percentage | 2.25% | |||
Proceeds from issuance of debt | 306,400 | |||
Debt issuance costs, net | 9,900 | $ 9,900 | ||
Accrued interest | $ 600 | |||
Debt instrument, convertible, threshold percentage of stock price trigger | 130% | |||
Conversion ratio | 0.031374 | |||
Conversion price (in dollars per share) | $ / shares | $ 31.87 | |||
Notes payable | $ 100,000 | |||
Long-term debt, term | 7 years | |||
Effective interest percentage | 2.74% | |||
Senior Notes | Senior Notes Due 2029 | Debt Conversion, Scenario One | ||||
Debt Instrument [Line Items] | ||||
Debt instrument, convertible, threshold trading days | day | 20 | |||
Debt instrument, convertible, threshold consecutive trading days | day | 30 | |||
Debt instrument, convertible, threshold percentage of stock price trigger | 130% | |||
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% | |||
Proceeds from issuance of debt | 267,200 | |||
Debt issuance costs, net | $ 8,800 | |||
Accrued interest | $ 100 | |||
Debt instrument, convertible, threshold percentage of stock price trigger | 130% | |||
Conversion ratio | 0.0257739 | |||
Conversion price (in dollars per share) | $ / shares | $ 38.80 | |||
Long-term debt, term | 7 years | |||
Effective interest percentage | 2.98% | |||
Debt instrument, repurchase amount | $ 207,100 | |||
Long-term debt, excluding current maturities, repaid if converted | $ 68,900 | |||
Senior Notes | Senior Notes Due 2025 | Debt Conversion, Scenario One | ||||
Debt Instrument [Line Items] | ||||
Debt instrument, convertible, threshold trading days | day | 20 | |||
Debt instrument, convertible, threshold consecutive trading days | day | 30 | |||
Debt instrument, convertible, threshold percentage of stock price trigger | 130% | |||
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 - Schedule of Interest Expense (Details) - Senior Notes - USD ($) $ in Thousands | 3 Months Ended | |
Mar. 31, 2023 | Mar. 31, 2022 | |
Senior Notes Due 2029 | ||
Debt Instrument [Line Items] | ||
Contractual interest expense | $ 2,209 | $ 1,843 |
Amortization of debt issuance costs | 429 | 337 |
Total interest expense for the 2025 and 2029 Notes | 2,638 | 2,180 |
Senior Notes Due 2025 | ||
Debt Instrument [Line Items] | ||
Contractual interest expense | 2,209 | 1,843 |
Amortization of debt issuance costs | 429 | 337 |
Total interest expense for the 2025 and 2029 Notes | $ 2,638 | $ 2,180 |
ACCRUED EXPENSES (Details)
ACCRUED EXPENSES (Details) - USD ($) $ in Thousands | Mar. 31, 2023 | Dec. 31, 2022 |
Payables and Accruals [Abstract] | ||
Compensation related costs | $ 26,839 | $ 35,267 |
Research and development | 24,909 | 26,070 |
Sales discounts, rebates, and allowances | 11,451 | 13,486 |
Selling, general and administrative | 9,064 | 8,791 |
Accrued royalties | 7,218 | 7,755 |
Miscellaneous accrued expenses | 6,285 | 4,373 |
Total accrued expenses | $ 85,766 | $ 95,742 |
NET LOSS PER COMMON SHARE - Nar
NET LOSS PER COMMON SHARE - Narrative (Details) - Underwritten Equity Offering | Feb. 28, 2023 $ / shares shares |
Subsidiary, Sale of Stock [Line Items] | |
Sales price per share (in dollars per share) | $ 21 |
Common Stock | |
Subsidiary, Sale of Stock [Line Items] | |
Pre-funded warrants exercisable for share of common stock (in shares) | shares | 1 |
Pre Funded Warrant | |
Subsidiary, Sale of Stock [Line Items] | |
Number of securities called by warrants or rights (in shares) | 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 |
NET LOSS PER COMMON SHARE - Bas
NET LOSS PER COMMON SHARE - Basic and Diluted (Details) - USD ($) $ / shares in Units, $ in Thousands | 3 Months Ended | |
Mar. 31, 2023 | Mar. 31, 2022 | |
Earnings Per Share [Abstract] | ||
Basic shares outstanding (in shares) | 68,174,099 | 63,132,841 |
Diluted shares outstanding (in shares) | 68,174,099 | 63,132,841 |
Net loss (Basic) | $ (86,331) | $ (75,971) |
Net loss (Diluted) | $ (86,331) | $ (75,971) |
Basic net loss per common share (in dollars per share) | $ (1.27) | $ (1.20) |
Diluted net loss per common share (in dollars per share) | $ (1.27) | $ (1.20) |
NET LOSS PER COMMON SHARE - Ant
NET LOSS PER COMMON SHARE - Antidilutive Shares (Details) - shares | 3 Months Ended | |
Mar. 31, 2023 | Mar. 31, 2022 | |
Total anti-dilutive shares | ||
Anti-dilutive shares excluded from the calculation (in shares) | 25,523,529 | 20,100,996 |
Convertible debt | ||
Total anti-dilutive shares | ||
Anti-dilutive shares excluded from the calculation (in shares) | 11,697,952 | 8,336,091 |
Options | ||
Total anti-dilutive shares | ||
Anti-dilutive shares excluded from the calculation (in shares) | 10,546,780 | 9,864,889 |
Restricted stock | ||
Total anti-dilutive shares | ||
Anti-dilutive shares excluded from the calculation (in shares) | 3,278,797 | 1,900,016 |
COMMITMENTS AND CONTINGENCIES (
COMMITMENTS AND CONTINGENCIES (Details) - USD ($) $ in Thousands | 1 Months Ended | 3 Months Ended | |
Oct. 31, 2021 | Mar. 31, 2023 | Mar. 31, 2022 | |
Loss Contingencies [Line Items] | |||
Purchase commitment | $ 36,300 | ||
Total revenue | $ 56,993 | $ 48,487 | |
Kolbam | FRANCE | |||
Loss Contingencies [Line Items] | |||
Total revenue | $ 8,000 |
SHARE-BASED COMPENSATION - Stoc
SHARE-BASED COMPENSATION - Stock Option Activity (Details) - USD ($) $ / shares in Units, $ in Thousands | 3 Months Ended | 12 Months Ended |
Mar. 31, 2023 | Dec. 31, 2022 | |
Aggregate Intrinsic Value (in thousands) | ||
Number of shares exercisable (in shares) | 7,400,000 | |
Weighted average exercise price (in dollars per share) | $ 20.65 | |
Stock Options | ||
Shares Underlying Options | ||
Beginning balance (in shares) | 9,932,422 | |
Granted (in shares) | 1,011,250 | |
Exercised (in shares) | (148,466) | |
Forfeited/canceled (in shares) | (70,488) | |
Ending balance (in shares) | 10,724,718 | 9,932,422 |
Vested and expected to vest (in shares) | 10,724,718 | |
Weighted Average Exercise Price | ||
Beginning balance (in dollars per share) | $ 21.56 | |
Granted (in dollars per share) | 22.35 | |
Exercised (in dollars per share) | 15.47 | |
Forfeited/canceled (in dollars per share) | 27.62 | |
Ending balance (in dollars per share) | 21.68 | $ 21.56 |
Vested and expected to vest (in USD per share) | $ 21.68 | |
Weighted Average Remaining Contractual Life (years) | 5 years 11 months 12 days | 5 years 9 months 14 days |
Vested and expected to vest | 5 years 11 months 12 days | |
Aggregate Intrinsic Value (in thousands) | ||
Aggregate intrinsic value, beginning balance | $ 24,658 | |
Exercised | 995 | |
Aggregate intrinsic value, ending balance | 30,730 | $ 24,658 |
Vested and expected to vest | 30,730 | |
Unamortized stock compensation expense | $ 38,800 | |
Weighted-average recognition period (in years) | 2 years 9 months 18 days | |
Stock Options | Chief Executive Officer | ||
Aggregate Intrinsic Value (in thousands) | ||
Incremental compensation cost | $ 2,600 |
SHARE-BASED COMPENSATION - Serv
SHARE-BASED COMPENSATION - Service Based Restricted Stock Activity (Details) - Restricted stock units $ / shares in Units, $ in Millions | 3 Months Ended |
Mar. 31, 2023 USD ($) $ / shares shares | |
Number of Restricted Stock Units | |
Beginning balance (in shares) | shares | 2,343,709 |
Granted (in shares) | shares | 1,631,792 |
Vested (in shares) | shares | (444,020) |
Forfeited/canceled (in shares) | shares | (26,180) |
Ending balance (in shares) | shares | 3,505,301 |
Weighted Average Grant Date Fair Value | |
Beginning balance (in dollars per share) | $ / shares | $ 24.65 |
Granted (in dollars per share) | $ / shares | 22.33 |
Vested (in dollars per share) | $ / shares | 24.62 |
Forfeited/canceled (in dollars per share) | $ / shares | 24.02 |
Ending balance (in dollars per share) | $ / shares | $ 23.58 |
Unamortized stock compensation expense | $ | $ 73.8 |
Weighted-average recognition period (in years) | 3 years 1 month 6 days |
SHARE-BASED COMPENSATION - Perf
SHARE-BASED COMPENSATION - Performance Based Restricted Stock Activity (Details) - Performance Shares $ / shares in Units, $ in Millions | 3 Months Ended |
Mar. 31, 2023 USD ($) $ / shares shares | |
Number of Restricted Stock Units | |
Beginning balance (in shares) | shares | 157,048 |
Granted (in shares) | shares | 66,250 |
Vested (in shares) | shares | 0 |
Forfeited/canceled (in shares) | shares | (22,840) |
Ending balance (in shares) | shares | 200,458 |
Weighted Average Grant Date Fair Value | |
Beginning balance (in dollars per share) | $ / shares | $ 25.24 |
Granted (in dollars per share) | $ / shares | 22.40 |
Vested (in dollars per share) | $ / shares | 0 |
Forfeited/canceled (in dollars per share) | $ / shares | 24.86 |
Ending balance (in dollars per share) | $ / shares | $ 24.34 |
Unamortized stock compensation expense | $ | $ 2.6 |
Weighted-average recognition period (in years) | 1 year 1 month 6 days |
SHARE-BASED COMPENSATION - St_2
SHARE-BASED COMPENSATION - Stock based Compensation Expense (Details) - USD ($) $ in Thousands | 3 Months Ended | |
Mar. 31, 2023 | Mar. 31, 2022 | |
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||
Stock-based compensation expense | $ 13,764 | $ 8,186 |
Research and development | ||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||
Stock-based compensation expense | 4,481 | 3,168 |
Selling, general and administrative | ||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||
Stock-based compensation expense | $ 9,283 | $ 5,018 |
INVENTORY - Schedule of Invento
INVENTORY - Schedule of Inventory (Details) - USD ($) $ in Thousands | Mar. 31, 2023 | Dec. 31, 2022 |
Inventory Disclosure [Abstract] | ||
Raw materials | $ 3,077 | $ 3,627 |
Finished goods | 3,635 | 3,295 |
Total inventory | $ 6,712 | $ 6,922 |
ACCOUNTS RECEIVABLE (Details)
ACCOUNTS RECEIVABLE (Details) - USD ($) $ in Thousands | Mar. 31, 2023 | Dec. 31, 2022 |
Receivables [Abstract] | ||
Accounts receivable, net | $ 21,537 | $ 16,646 |
EQUITY OFFERINGS (Details)
EQUITY OFFERINGS (Details) - USD ($) $ / shares in Units, $ in Thousands | 1 Months Ended | 3 Months Ended | 12 Months Ended | ||
Feb. 28, 2023 | Mar. 31, 2023 | Mar. 31, 2022 | Dec. 31, 2022 | Feb. 29, 2020 | |
Class of Stock [Line Items] | |||||
Proceeds from the issuance of pre-funded warrants, net of issuance costs | $ 24,630 | $ 0 | |||
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 | ||||
Proceeds from the issuance of common stock, net of issuance costs | $ 20,100 | ||||
Aggregate offering amount authorized | $ 100,000 | ||||
Remaining offering amount authorized | $ 19,500 | ||||
At-The-Market Offering Under Previous Registration Statement | |||||
Class of Stock [Line Items] | |||||
Amount sold to date | 28,600 | ||||
At-The-Market Offering Under Current Registration Statement | |||||
Class of Stock [Line Items] | |||||
Amount sold to date | $ 51,900 |
Uncategorized Items - tvtx-2023
Label | Element | Value |
Accounting Standards Update [Extensible Enumeration] | us-gaap_AccountingStandardsUpdateExtensibleList | Accounting Standards Update 2020-06 Cumulative Effect, Period of Adoption [Member] |