Travere Therapeutics(TVTX) - 2023 Q3 - Quarterly Report

PART I – FINANCIAL INFORMATION This section provides the company's unaudited consolidated financial statements, including balance sheets, statements of operations, stockholders' equity, and cash flows, along with management's discussion and analysis of financial condition and results of operations, market risk disclosures, and controls and procedures Item 1. Financial Statements This section presents the unaudited consolidated financial statements, including balance sheets, statements of operations, stockholders' equity, and cash flows, along with detailed notes. Key financial highlights include a significant increase in cash and marketable securities, a shift to net income for the three-month period due to the sale of the bile acid business, and increased operating expenses driven by commercialization efforts and R&D investments Consolidated Balance Sheets This section provides a snapshot of the company's financial position, detailing assets, liabilities, and stockholders' equity at specific points in time Consolidated Balance Sheet Highlights (in thousands) | Item | Sep 30, 2023 | Dec 31, 2022 | | :-------------------------- | :----------- | :----------- | | Cash and cash equivalents | $144,244 | $61,688 | | Marketable debt securities | $490,399 | $388,557 | | Total current assets | $686,230 | $486,437 | | Total assets | $832,850 | $672,585 | | Total current liabilities | $139,433 | $142,163 | | Total liabilities | $553,084 | $629,734 | | Total stockholders' equity | $279,766 | $42,851 | Consolidated Statements of Operations and Comprehensive Income (Loss) This section outlines the company's financial performance over specific periods, detailing revenues, expenses, and net income or loss Consolidated Statements of Operations Highlights (in thousands) | Item | 3 Months Ended Sep 30, 2023 | 3 Months Ended Sep 30, 2022 | 9 Months Ended Sep 30, 2023 | 9 Months Ended Sep 30, 2022 | | :------------------------------------------ | :-------------------------- | :-------------------------- | :-------------------------- | :-------------------------- | | Total revenue | $37,095 | $28,075 | $100,179 | $80,121 | | Operating loss | $(92,585) | $(82,604) | $(293,905) | $(233,111) | | Net income (loss) | $150,735 | $(69,656) | $(21,226) | $(212,659) | | Net income from discontinued operations | $239,976 | $14,407 | $266,511 | $33,173 | | Net income (loss) per common share (Basic & Diluted) | $1.97 | $(1.09) | $(0.29) | $(3.34) | Consolidated Statements of Stockholders' Equity This section details changes in the company's equity over time, reflecting contributions, distributions, and net income or loss Changes in Stockholders' Equity (9 Months Ended Sep 30, 2023 vs 2022, in thousands) | Item | 2023 | 2022 | | :------------------------------------------------ | :----- | :----- | | Balance - December 31 | $42,851 | $302,112 | | Equity offering, net of issuance costs | $191,199 | — | | Issuance of pre-funded common stock warrants, net | $24,630 | — | | Share based compensation | $36,273 | $28,751 | | Net loss | $(21,226) | $(212,659) | | Balance - September 30 | $279,766 | $100,676 | Consolidated Statements of Cash Flows This section reports the cash generated and used by the company through its operating, investing, and financing activities Consolidated Statements of Cash Flows Highlights (9 Months Ended Sep 30, 2023 vs 2022, in thousands) | Cash Flow Activity | 2023 | 2022 | | :------------------------------------------ | :--------- | :--------- | | Net cash used in operating activities | $(209,548) | $(131,651) | | Net cash provided by investing activities | $72,726 | $4,148 | | Net cash provided by financing activities | $218,628 | $117,022 | | Net increase in cash and cash equivalents | $82,556 | $(14,416) | | Cash and cash equivalents, end of period | $144,244 | $151,337 | Notes to Unaudited Consolidated Financial Statements This section provides detailed explanations and additional information supporting the consolidated financial statements NOTE 1. DESCRIPTION OF BUSINESS This note describes the company's core business, strategic focus, key product developments, and recent divestitures - Travere Therapeutics is a biopharmaceutical company focused on identifying, developing, and delivering life-changing therapies for people living with rare kidney and metabolic diseases[37] - The company sold its bile acid product portfolio (Chenodal and Cholbam) to Mirum Pharmaceuticals for an upfront cash payment of $210.0 million, with potential for up to $235.0 million in sales-based milestones[38] - FILSPARI (sparsentan) received accelerated FDA approval on February 17, 2023, to reduce proteinuria in adults with primary IgAN[40] - Topline results from the two-year confirmatory endpoints in the PROTECT Study (sparsentan for IgAN) were reported in September 2023 and are intended to support traditional approval of FILSPARI[42] - The Phase 3 study of sparsentan for FSGS did not meet its primary endpoint, and the company is engaging with regulators to explore a potential path forward[43] - Pegtibatinase (TVT-058) for classical homocystinuria (HCU) showed positive topline results from cohort 6 in the Phase 1/2 COMPOSE Study in May 2023, with preparation for a pivotal Phase 3 clinical trial by the end of 2023[44] NOTE 2. BASIS OF PRESENTATION AND SIGNIFICANT ACCOUNTING POLICIES This note outlines the principles and methods used in preparing the financial statements, including revenue recognition, inventory, R&D, and consolidation policies - The consolidated financial statements are prepared in accordance with GAAP for interim financial information and SEC rules for Form 10-Q[47] - Revenue is recognized when customers obtain control of promised goods or services, with variable consideration (discounts, rebates, co-pay assistance) estimated and recognized as a reduction of the transaction price[49][73] - Inventory costs are capitalized after regulatory approval when future commercialization is probable; costs prior to approval are expensed as R&D[56][57] - All research and development costs are expensed as incurred, including those for sparsentan, pegtibatinase, and other pipeline programs[58] - The company consolidates PharmaKrysto Limited as a Variable Interest Entity (VIE) due to its funding obligations and control over the cystinuria program[66][96] - The sale of the bile acid business is presented as discontinued operations due to its strategic shift and major effect on financial results[68] - Adopted ASU 2020-06 on January 1, 2022, using the modified retrospective method, which impacted the accounting for Convertible Senior Notes Due 2025[69] NOTE 3. REVENUE RECOGNITION This note details the company's policies for recognizing revenue from product sales and collaboration agreements, including variable consideration estimates - Product sales consist of FILSPARI and tiopronin products (Thiola and Thiola EC), with over 98% of net product sales from the United States[71] - FILSPARI sales are recognized upon delivery to specialty pharmacies, while other product sales are recognized upon delivery to the patient[72] - Deductions from revenue, such as government rebates, commercial rebates, prompt pay discounts, product returns, and co-pay assistance, are estimated as variable consideration and reduce the transaction price[73] Net Product Sales (in thousands) | Product | 3 Months Ended Sep 30, 2023 | 3 Months Ended Sep 30, 2022 | 9 Months Ended Sep 30, 2023 | 9 Months Ended Sep 30, 2022 | | :---------------- | :-------------------------- | :-------------------------- | :-------------------------- | :-------------------------- | | Tiopronin products | $25,888 | $25,369 | $73,112 | $72,154 | | FILSPARI | $8,044 | — | $14,509 | — | | Total net product sales | $33,932 | $25,369 | $87,621 | $72,154 | NOTE 4. COLLABORATION AND LICENSE AGREEMENTS This note describes key agreements, including the CSL Vifor partnership for sparsentan, and the associated revenue recognition - The company has a license and collaboration agreement with CSL Vifor for the commercialization of sparsentan in Europe, Australia, and New Zealand[80] - Under the agreement, the company received an upfront payment of $55.0 million and is eligible for up to $135.0 million in regulatory/market access milestones and up to $655.0 million in sales-based milestones, plus tiered double-digit royalties up to 40%[80] - $43.0 million of the transaction price was allocated to clinical development activities and is recognized as deferred revenue over the development period using the cost-to-cost method[84] License and Collaboration Revenue (in thousands) | Item | 3 Months Ended Sep 30, 2023 | 3 Months Ended Sep 30, 2022 | 9 Months Ended Sep 30, 2023 | 9 Months Ended Sep 30, 2022 | | :-------------------------------- | :-------------------------- | :-------------------------- | :-------------------------- | :-------------------------- | | Clinical development activities | $3,163 | $2,706 | $9,300 | $7,967 | | Sale of active pharmaceutical ingredients | — | — | $3,300 | — | | Total license and collaboration revenue | $3,163 | $2,706 | $12,600 | $7,967 | NOTE 5. MARKETABLE DEBT SECURITIES This note provides details on the company's marketable debt securities portfolio, including composition and fair value changes Marketable Debt Securities (in thousands) | Type | Sep 30, 2023 | Dec 31, 2022 | | :---------------------------------- | :----------- | :----------- | | Commercial paper | $49,243 | $123,647 | | Corporate debt securities | $334,281 | $224,055 | | Securities of government sponsored entities | $106,875 | $40,855 | | Total available-for-sale | $490,399 | $388,557 | - Investment activity for the nine months ended September 30, 2023, included $284.6 million in maturities and $381.3 million in purchases of debt-based marketable securities[89] - Unrealized losses increased due to fluctuations in short-term interest rates, but the company does not intend to sell these investments before recovery, and no credit losses were reported[94] NOTE 6. VARIABLE INTEREST ENTITIES This note explains the company's involvement with PharmaKrysto Limited as a Variable Interest Entity and its primary beneficiary status - The company entered into a Collaboration Agreement with PharmaKrysto Limited for an early-stage cystinuria discovery program, acquiring 5% of shares for $0.6 million and paying a $0.4 million signing fee[95] - Travere will fund all research and development expenses for the preclinical activities (estimated $5.0 million) and has an option to purchase the remaining shares for $5.0 million upon a subsequent preclinical milestone[95] - PharmaKrysto is deemed a Variable Interest Entity (VIE), and Travere is the primary beneficiary due to its control over the R&D program and funding obligations[96] NOTE 7. LEASES This note details the company's operating lease commitments, including future payments and associated expenses - As of September 30, 2023, the company had two operating leases for office space in San Diego, California, and Dublin, Ireland[98][100] Future Minimum Rental Commitments for Operating Leases (in thousands) | Year | Amount | | :-------------------------- | :----- | | 2023 (remaining three months) | $1,582 | | 2024 | $6,501 | | 2025 | $6,673 | | 2026 | $6,889 | | 2027 | $7,064 | | Thereafter | $4,781 | | Total undiscounted payments | $33,490 | - The weighted-average remaining lease term is 4.9 years, and the weighted-average discount rate is 6.48%[101] - Operating lease expense for the three and nine months ended September 30, 2023, was $1.2 million and $3.7 million, respectively[101] NOTE 8. FAIR VALUE MEASUREMENTS This note describes the fair value hierarchy used for financial instruments and the valuation of convertible senior notes - The company utilizes a fair value hierarchy (Level 1, 2, 3) for financial instruments, classifying marketable debt securities within Level 2[102][103] Fair Value of Convertible Senior Notes (in thousands) | Note Type | Sep 30, 2023 | Dec 31, 2022 | | :---------------------------------- | :----------- | :----------- | | 2.5% Convertible Senior Notes due 2025 | $65,400 | $62,900 | | 2.25% Convertible Senior Notes due 2029 | $241,600 | $283,000 | Assets Measured at Fair Value (Sep 30, 2023, in thousands) | Asset | Total Fair Value | Level 1 | Level 2 | Level 3 | | :---------------------------------- | :--------------- | :------ | :------ | :------ | | Cash and cash equivalents | $144,244 | $78,367 | $65,877 | — | | Marketable debt securities, available-for-sale | $490,399 | — | $490,399 | — | | Total | $634,643 | $78,367 | $556,276 | — | NOTE 9. INTANGIBLE ASSETS This note outlines the company's intangible assets, including capitalized milestone payments and royalties, and their amortization - A $23.0 million milestone payment to Ligand (and BMS) was capitalized in March 2023 upon the accelerated approval of FILSPARI[107] - Royalties owed on net sales of FILSPARI were capitalized to intangible assets, totaling $1.1 million for the three months and $2.2 million for the nine months ended September 30, 2023[107] Amortizable Intangible Assets (in thousands) | Item | Sep 30, 2023 | Dec 31, 2022 | | :-------------------------- | :----------- | :----------- | | Finite-lived intangible assets | $195,971 | $158,783 | | Less: accumulated amortization | $(89,821) | $(62,463) | | Net carrying value | $106,150 | $96,320 | Total Amortization Expense (in thousands) | Category | 3 Months Ended Sep 30, 2023 | 3 Months Ended Sep 30, 2022 | 9 Months Ended Sep 30, 2023 | 9 Months Ended Sep 30, 2022 | | :-------------------------------- | :-------------------------- | :-------------------------- | :-------------------------- | :-------------------------- | | Research and development | $2,447 | $1,906 | $7,261 | $3,817 | | Selling, general and administrative | $8,461 | $3,173 | $20,097 | $9,008 | | Total amortization expense | $10,908 | $5,079 | $27,358 | $12,825 | NOTE 10. CONVERTIBLE NOTES PAYABLE This note details the company's convertible senior notes, including principal amounts, interest rates, maturity dates, and conversion terms Convertible Senior Notes Composition (in thousands) | Item | Sep 30, 2023 | Dec 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 - 2029 Notes | $(7,699) | $(8,750) | | Unamortized debt issuance costs - 2025 Notes | $(622) | $(859) | | Total convertible senior notes, net | $376,833 | $375,545 | - The 2029 Notes have an aggregate principal of $316.3 million, bear 2.25% interest, and mature on March 1, 2029, with an initial conversion rate of 31.3740 shares per $1,000 principal[112][114] - The 2025 Notes had an initial principal of $276.0 million, bear 2.50% interest, and mature on September 15, 2025[116] - $207.1 million of the 2025 Notes was repurchased in March 2022, leaving $68.9 million outstanding[121] - A $7.6 million loss on extinguishment of debt was recognized in 2022 due to the repurchase of the 2025 Notes[121] Total Interest Expense for Convertible Notes (in thousands) | Item | 3 Months Ended Sep 30, 2023 | 3 Months Ended Sep 30, 2022 | 9 Months Ended Sep 30, 2023 | 9 Months Ended Sep 30, 2022 | | :------------------------------------------ | :-------------------------- | :-------------------------- | :-------------------------- | :-------------------------- | | Contractual interest expense | $2,210 | $2,170 | $6,629 | $6,223 | | Amortization of debt issuance costs | $430 | $428 | $1,288 | $1,194 | | Total interest expense | $2,640 | $2,598 | $7,917 | $7,417 | NOTE 11. ACCRUED EXPENSES This note provides a breakdown of the company's accrued expenses, including compensation, R&D, and sales-related costs Accrued Expenses (in thousands) | Item | Sep 30, 2023 | Dec 31, 2022 | | :-------------------------------- | :----------- | :----------- | | Compensation related costs | $26,844 | $35,267 | | Research and development | $26,249 | $26,070 | | Sales discounts, rebates, and allowances | $10,385 | $13,486 | | Selling, general and administrative | $8,707 | $8,791 | | Accrued royalties | $8,002 | $7,755 | | Miscellaneous accrued expenses | $15,525 | $4,373 | | Total accrued expenses | $95,712 | $95,742 | NOTE 12. NET INCOME (LOSS) PER COMMON SHARE This note presents the calculation of basic and diluted net income or loss per common share, including the treatment of potentially dilutive securities Net Income (Loss) Per Common Share (Basic and Diluted) | Period | 2023 | 2022 | | :-------------------------- | :----- | :----- | | 3 Months Ended Sep 30 | $1.97 | $(1.09) | | 9 Months Ended Sep 30 | $(0.29) | $(3.34) | - Pre-funded warrants issued in February 2023 are included in the calculation of basic net loss per common share due to their low exercise price and immediate exercisability[128] - Potentially dilutive shares, including outstanding stock options, restricted stock units, and convertible notes, were excluded from diluted net loss per share calculations because their effect was anti-dilutive[130] NOTE 13. COMMITMENTS AND CONTINGENCIES This note discloses the company's significant contractual commitments and potential contingent liabilities, including legal matters - The company has commitments to purchase $22.1 million in active pharmaceutical ingredients in 2023 to support commercial sales of FILSPARI[131] - No legal matters are currently expected to have a material adverse effect on the company's results of operations or financial condition[132] NOTE 14. SHARE-BASED COMPENSATION This note details the company's share-based compensation plans, including stock option activity and related expenses Stock Option Activity (9 Months Ended Sep 30, 2023) | Item | Shares Underlying Options | Weighted Average Exercise Price | | :-------------------------- | :------------------------ | :------------------------------ | | Outstanding at Dec 31, 2022 | 9,932,422 | $21.56 | | Granted | 1,157,750 | $21.69 | | Exercised | (226,663) | $14.30 | | Forfeited/canceled | (410,559) | $25.52 | | Outstanding at Sep 30, 2023 | 10,452,950 | $21.58 | - Unamortized stock compensation for stock options was $29.9 million, with a remaining weighted-average recognition period of 2.5 years[133] - A modification to the former CFO's stock options resulted in an incremental compensation cost of $2.6 million for the nine months ended September 30, 2023[135] Total Share-Based Compensation for Continuing Operations (in thousands) | Category | 3 Months Ended Sep 30, 2023 | 3 Months Ended Sep 30, 2022 | 9 Months Ended Sep 30, 2023 | 9 Months Ended Sep 30, 2022 | | :-------------------------------- | :-------------------------- | :-------------------------- | :-------------------------- | :-------------------------- | | Research and development | $4,372 | $3,372 | $13,372 | $10,182 | | Selling, general and administrative | $6,949 | $5,216 | $22,730 | $18,664 | | Total share-based compensation | $11,321 | $8,588 | $36,102 | $28,846 | NOTE 15. INVENTORY This note provides a breakdown of the company's inventory, including raw materials, work in process, and finished goods Inventory Composition (in thousands) | Item | Sep 30, 2023 | Dec 31, 2022 | | :---------------- | :----------- | :----------- | | Raw materials | $16,879 | $2,397 | | Work in process | $972 | — | | Finished goods | $2,922 | $2,126 | | Total inventory | $20,773 | $4,523 | NOTE 16. ACCOUNTS RECEIVABLE This note details the company's accounts receivable, net of reserves, and related credit loss estimates - Accounts receivable, net of reserves, was $14.6 million at September 30, 2023, compared to $16.6 million at December 31, 2022[140] - Reserves for prompt pay discounts and expected credit losses were immaterial for both periods[140][141] NOTE 17. EQUITY OFFERINGS This note describes recent equity offerings, including common share and pre-funded warrant sales, and their net proceeds - In February 2023, the company completed an underwritten public offering, selling approximately 9.7 million common shares and 1.25 million pre-funded warrants, generating net proceeds of approximately $215.8 million[142] - The pre-funded warrants were equity-classified and recorded as a component of additional paid-in capital, with $24.6 million allocated to them[143] - No shares were sold under the At-the-Market (ATM) Agreement during the nine months ended September 30, 2023[146] NOTE 18. DIVESTITURES This note details the sale of the bile acid business, including the upfront payment, potential milestones, and the resulting gain from discontinued operations - The sale of the bile acid business (Chenodal and Cholbam) to Mirum Pharmaceuticals closed on August 31, 2023, for an upfront cash payment of $210.0 million[147] - The company is eligible to receive up to $235.0 million upon the achievement of certain sales-based milestones[148] - The divestiture resulted in a gain, net of tax, of $225.9 million and is classified as a discontinued operation for all periods presented[149][151] Net Income from Discontinued Operations (in thousands) | Period | 2023 | 2022 | | :-------------------------- | :--------- | :--------- | | 3 Months Ended Sep 30 | $239,976 | $14,407 | | 9 Months Ended Sep 30 | $266,511 | $33,173 | Item 2. Management's Discussion and Analysis of Financial Condition and Results of Operations This section provides management's perspective on the company's financial condition and operational results. It highlights the strategic focus on rare kidney and metabolic diseases, updates on product pipeline and approved products (including FILSPARI's launch and the sale of the bile acid business), detailed analysis of revenue and operating expenses, and a discussion of liquidity and capital resources Overview This section provides a high-level introduction to the company's strategic focus on rare kidney and metabolic diseases and its investment approach - Travere Therapeutics is a biopharmaceutical company focused on identifying, developing, and delivering life-changing therapies for people living with rare kidney and metabolic diseases[155] - The company invests revenues from its commercial portfolio into its pipeline with the goal of delivering new treatments for diseases with limited or no approved therapies[157] Our Pipeline and Approved Products This section provides updates on the company's product candidates and approved therapies, including clinical trial results and commercialization efforts - FILSPARI (sparsentan) received accelerated FDA approval on February 17, 2023, for primary IgAN and became commercially available in the U.S. by late February 2023[160] - Topline two-year confirmatory secondary endpoint results from the PROTECT Study for FILSPARI in IgAN were announced in September 2023, showing long-term kidney function preservation and clinically meaningful eGFR differences, narrowly missing statistical significance for total slope in the U.S. but achieving it for chronic slope in the EU[170] - The Phase 3 DUPLEX Study of sparsentan for FSGS did not achieve its primary efficacy endpoint (eGFR total slope) with statistical significance over 108 weeks, but secondary and exploratory endpoints trended favorably[176] - The company is engaging with regulators to explore a potential path forward[176] - Pegtibatinase (TVT-058) for classical homocystinuria (HCU) showed positive topline results from cohort 6 of the Phase 1/2 COMPOSE Study in May 2023, with plans for a pivotal Phase 3 clinical trial by the end of 2023[181][182] - Thiola and Thiola EC (tiopronin tablets) are approved for cystinuria, but generic versions of Thiola and Thiola EC have been approved, with generic Thiola EC expected to enter the market by April 1, 2026[187][188] - The bile acid business (Chenodal and Cholbam) was sold to Mirum Pharmaceuticals on August 31, 2023, for an upfront payment of $210.0 million and potential milestones, resulting in a $225.9 million gain[189][190] Results of Operations This section analyzes the company's financial performance, detailing changes in total revenue, operating expenses, and net income or loss Total Revenue (in thousands) | Period | 2023 | 2022 | Change | | :-------------------------- | :----- | :----- | :----- | | 3 Months Ended Sep 30 | $37,095 | $28,075 | $9,020 | | 9 Months Ended Sep 30 | $100,179 | $80,121 | $20,058 | - The increase in total net product revenues was primarily due to the launch of FILSPARI in February 2023 and subsequent sales[193] - License and collaboration revenue increased for the nine months ended September 30, 2023, primarily due to a $3.3 million sale of active pharmaceutical ingredients to CSL Vifor[194] Total Operating Expenses (in thousands) | Period | 2023 | 2022 | Change | | :-------------------------- | :------- | :------- | :------- | | 3 Months Ended Sep 30 | $129,680 | $110,679 | $19,001 | | 9 Months Ended Sep 30 | $394,084 | $313,232 | $80,852 | - Research and development expenses increased by $3.4 million (3 months) and $16.0 million (9 months) year-over-year, driven by increased headcount and investment in pegtibatinase development[201] - Selling, general and administrative expenses increased by $15.4 million (3 months) and $61.5 million (9 months) year-over-year, primarily due to commercial preparations and activities for the U.S. launch of FILSPARI[203] - A change in the estimated useful life of the Thiola intangible asset contributed a $7.4 million increase in SG&A expense for the nine months ended September 30, 2023[203] - Net income from discontinued operations was $239.9 million (3 months) and $266.5 million (9 months) in 2023, largely due to the $225.9 million gain from the sale of the bile acid business[207] Liquidity and Capital Resources This section discusses the company's financial position, including cash, marketable securities, working capital, and funding sources for future operations Liquidity and Capital Resources (in thousands) | Item | Sep 30, 2023 | Dec 31, 2022 | | :-------------------------- | :----------- | :----------- | | Cash and Cash Equivalents | $144,244 | $61,688 | | Marketable debt securities | $490,399 | $388,557 | | Net Working Capital | $546,797 | $344,274 | | Net Working Capital Ratio | 4.92 | 3.42 | - Significant funding sources in 2023 include a $210.0 million upfront cash payment from the bile acid product portfolio sale and $215.8 million net proceeds from an underwritten public offering[212] - Management believes available cash and short-term investments, along with anticipated cash from operations, will be sufficient to fund operations beyond the next 12 months[210] - Future financial obligations include funding operations, operating lease payments, interest on outstanding debt, royalties on existing products, and R&D expenses for pipeline products[213] - Outstanding debt includes $316.3 million principal of 2.25% Convertible Senior Notes due 2029 and $68.9 million principal of 2.50% Convertible Senior Notes due 2025[229][230] Other Matters This section refers to discussions on new accounting standards and critical accounting estimates - Refer to Note 2 for discussions on the adoption of new accounting standards and recently issued accounting pronouncements[238][239] - Critical accounting estimates are detailed in the Annual Report on Form 10-K for the fiscal year ended December 31, 2022[240] Item 3. Quantitative and Qualitative Disclosures About Market Risk The company's primary market risk exposures are interest rate risk on its investment portfolio, credit risk, and foreign currency exchange rate risk. Inflationary pressures have mainly impacted labor costs - Primary market risk exposure is to changes in interest rates, affecting approximately $634.6 million in cash equivalents and marketable debt securities[241] - A 100 basis point change in interest rates would have an approximate $4.5 million impact on the company's investments[241] - The company is exposed to foreign currency exchange rate risk from contracts with non-U.S. vendors but does not currently hedge this risk[243] - Inflationary pressures have primarily impacted operations through increased labor costs[244] Item 4. Controls and Procedures Management concluded that disclosure controls and procedures were not effective as of September 30, 2023, due to a material weakness in internal control over financial reporting related to the accounting for a pre-launch inventory contract. Remediation efforts are ongoing - Disclosure controls and procedures were not effective as of September 30, 2023[247] - A material weakness in internal control over financial reporting was identified, related to the evaluation of accounting for a pre-launch inventory contract affecting the timing of research and development expense recognition[249] - The material weakness has not been fully remediated as of the date of the report, but a remediation plan is underway[250] PART II – OTHER INFORMATION This section provides additional information beyond the financial statements, covering legal proceedings, risk factors, equity sales, defaults, mine safety, other disclosures, and a list of exhibits Item 1. Legal Proceedings Information regarding legal proceedings is incorporated by reference from Note 13 to the unaudited consolidated financial statements, indicating no material adverse effects are currently expected - Information on legal proceedings is incorporated by reference from Note 13, indicating no material adverse effects are expected[251] Item 1A. Risk Factors This section outlines various speculative and high-degree risks that could adversely affect the company's business, financial condition, and stock price. These risks span commercialization challenges, product development uncertainties, operational dependencies, intellectual property concerns, regulatory and market dynamics, financial obligations, and broader macroeconomic and geopolitical factors Risks Related to the Commercialization of Our Products This section details risks associated with achieving market acceptance, competition, pricing, reimbursement, manufacturing, and maintaining orphan drug status for the company's commercial products - The company's future prospects are highly dependent on the successful commercialization of its products, particularly FILSPARI, and achieving market acceptance among physicians, patients, and healthcare payers[253] - FILSPARI's commercial success is uncertain due to challenges in estimating market potential, educating physicians, and patient adoption, especially given the narrowly missed statistical significance in eGFR total slope for U.S. regulatory review[255] - The company faces substantial generic and other competition, particularly for Thiola and Thiola EC, which could materially impact net product revenues[264][271] - Generic Thiola EC is expected to enter the market by April 1, 2026[271] - Healthcare reform initiatives, unfavorable pricing regulations, and changes in third-party payer reimbursement practices could adversely affect product pricing and demand[272][273] - Dependence on third parties for manufacturing and distribution poses risks to profit margins, timely product delivery, and compliance with regulatory requirements[276][277] - Orphan drug status for FILSPARI (IgAN), sparsentan (FSGS), and pegtibatinase (HCU) may not confer marketing exclusivity if a clinically superior competitor emerges or if re-evaluation criteria are not met[281] Risks Related to the Development of our Product Candidates This section addresses the inherent uncertainties, costs, and potential failures in clinical trials, regulatory approvals, and the confirmation of clinical benefits for product candidates - Clinical trials are expensive, time-consuming, and may fail to demonstrate the safety and efficacy of product candidates, potentially leading to delays or abandonment of development programs[282][283] - Success in nonclinical testing and early clinical trials does not guarantee success in later clinical trials; for example, the DUPLEX study for FSGS did not meet its primary eGFR endpoint with statistical significance over 108 weeks[290] - Communications and feedback from regulatory authorities do not guarantee specific outcomes or timelines for regulatory review, and expedited pathways may not lead to faster development or approval[291] - FILSPARI's accelerated approval for IgAN is contingent upon confirmation of clinical benefit in the PROTECT Study, which narrowly missed statistical significance in eGFR total slope for U.S. regulatory review[294] - Interim, topline, and preliminary data from clinical trials may change materially as more patient data become available and audit procedures are completed[301] - The company and its partners are subject to ongoing regulatory obligations and continued review for approved products, which may result in costly post-marketing requirements, restrictions, or even product withdrawal[304][305] Risks Related to our Products and Product Candidates This section covers risks concerning market acceptance, potential side effects, market size estimates, intellectual property protection, pricing regulations, and manufacturing dependencies - Products may not achieve or maintain expected levels of market acceptance or commercial success due to factors like side effects (e.g., FILSPARI's REMS for liver injury and birth defects), efficacy, pricing, and competition[311][312] - The market opportunities for rare disease products (IgAN, FSGS, HCU) may be smaller than estimated, which could adversely affect revenue generation[314][316] - Product candidates may cause undesirable side effects or have other properties that could delay or prevent their regulatory approval or commercialization[317] - The company lacks patent protection for certain commercial products (Thiola), and existing patents (Thiola EC, sparsentan) may be challenged, narrowed, or invalidated, adversely affecting intellectual property value[321][329] - Unfavorable pricing regulations, third-party reimbursement practices, and healthcare reform initiatives (e.g., PPACA, IRA) could negatively impact product pricing and demand[334][337][339] - Reliance on third-party manufacturers increases the risk of insufficient product quantities, unacceptable costs, and delays in clinical development and commercialization[346][347] - Materials necessary to manufacture products and product candidates may not be available on commercially reasonable terms or at all, potentially delaying development and commercialization[352] Risks Related to Our Business This section discusses risks related to the company's operating history, workforce, external events, financial fluctuations, funding needs, stock price volatility, acquisitions, legal liabilities, regulatory compliance, data security, tax laws, internal controls, and financial industry stability - The company's limited operating history and significant growth make future prospects difficult to evaluate, and profitability in the future is uncertain[353][357] - Dependence on a highly experienced and skilled workforce means that an inability to attract, retain, and engage employees could hinder effective growth[359] - Health epidemics or pandemics could materially adversely affect business operations, clinical trials, and supply chains[363] - Operating results are likely to fluctuate significantly due to R&D investments and commercialization expenses, potentially leading to substantial losses[365] - The company may need substantial additional funding for R&D and commercialization, and capital may not be available when needed or on acceptable terms, especially given volatile market conditions[371][372] - The market price for the company's common stock may be volatile due to various factors, including clinical trial results, regulatory developments, and general market conditions[375] - There is a risk that the company might not receive some or all of the potential milestone payments from the sale of its bile acid product portfolio[377] - The company may be unable to successfully integrate new products or businesses it acquires, potentially failing to achieve anticipated benefits[378] - Product liability lawsuits could result in substantial liabilities that exceed insurance coverage, harming the business[382] - The company is subject to significant ongoing regulatory obligations and oversight (e.g., Anti-Kickback Statute, False Claims Act, HIPAA), which may result in additional expense, fines, or limit commercial success[385][390][391][398] - Failure to obtain and maintain required regulatory approvals would materially impair the ability to generate revenue[402] - Stringent and changing U.S. and foreign laws related to data privacy and security (e.g., GDPR, CCPA) pose risks of regulatory investigations, litigation, fines, and business disruptions[407][409] - The company's information technology systems and data, or those of third parties, are vulnerable to cyberattacks and security incidents, which could lead to operational interruptions, data loss, and reputational harm[415][421] - Uncertainties in the interpretation and application of existing, new, and proposed tax laws and regulations could materially affect tax obligations and the effective tax rate[424][427] - The company has identified a material weakness in its internal control over financial reporting, which, if not remediated, could result in material misstatements and adversely affect future results and stock price[439][441] - Adverse developments affecting the financial services industry (e.g., bank failures) could adversely affect current and projected business operations and financial condition[443][445] Risks Related to our Indebtedness and Investments This section highlights risks associated with the company's debt obligations, including financial flexibility, ability to repurchase notes, potential for default, and stock dilution from conversions - The company's indebtedness of approximately $385 million could adversely affect its financial condition by requiring a portion of cash flow for interest and principal payments, and limiting financial flexibility[448][449] - The company may be unable to raise the necessary funds to repurchase the 2025 Notes and 2029 Notes following a fundamental change or to pay any cash amounts due upon conversion[453][454] - A default under the 2025 Notes or 2029 Notes could lead to the immediate acceleration of principal and interest, which may have a material adverse effect on the company's financial condition[455] - Conversion of the Notes may dilute the ownership interest of existing stockholders and could depress the market price of the common stock[457] General Risk Factors This section addresses broad market, economic, and geopolitical conditions that could adversely impact the company's business and financial operations - Unstable market, economic, and geopolitical conditions, including inflation, rising interest rates, bank failures, and international conflicts, may have serious adverse consequences on the company's business, financial condition, and stock price[459][460] - Economic downturns could make necessary debt or equity financing more difficult, costly, and dilutive[459] Item 2. Unregistered Sales of Equity Securities and Use of Proceeds There were no unregistered sales of equity securities or use of proceeds during the quarter ended September 30, 2023 - No unregistered sales of equity securities or use of proceeds occurred during the quarter ended September 30, 2023[461] Item 3. Defaults Upon Senior Securities There were no defaults upon senior securities during the quarter ended September 30, 2023 - No defaults upon senior securities occurred during the quarter ended September 30, 2023[462] Item 4. Mine Safety Disclosures This item is not applicable to the company - This item is not applicable[463] Item 5. Other Information None of the company's directors or officers adopted or terminated a Rule 10b5-1 trading arrangement or a non-Rule 10b5-1 trading arrangement during the quarter ended September 30, 2023 - No Rule 10b5-1 or non-Rule 10b5-1 trading arrangements were adopted or terminated by directors or officers during the quarter ended September 30, 2023[464] Item 6. Exhibits This section lists the exhibits filed with the Form 10-Q, including corporate organizational documents, the Asset Purchase Agreement, CEO/CFO certifications, and XBRL-related documents - Exhibits include corporate documents (Certificate of Incorporation, Bylaws), the Asset Purchase Agreement dated July 16, 2023, CEO and CFO certifications (Sarbanes-Oxley Act), and Inline XBRL documents[465]