UNITED STATES SECURITIES AND EXCHANGE COMMISSION WASHINGTON, D.C. 20549 _________________________________ FORM 10-Q _________________________________ ☑ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended March 31, 2021 or ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from ___________ to ___________ Commission File Number: 001-36257 TRAVERE THERAPEUTICS, INC. (Exact name o ...

UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 FORM 10-K ☑ ANNUAL REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the Fiscal Year Ended December 31, 2020 Commission File Number: 001-36257 TRAVERE THERAPEUTICS, INC. (Exact Name of Registrant as specified in its Charter) Delaware 27-4842691 (State or other jurisdiction of incorporation or organization) (I.R.S. Employer Identification No.) 3611 Valley Centre Drive, Suite 300 San Diego, CA 92130 (Addres ...

[PART I – FINANCIAL INFORMATION](index=4&type=section&id=PART%20I%20%E2%80%93%20FINANCIAL%20INFORMATION) [Financial Statements](index=4&type=section&id=Item%201.%20Financial%20Statements) For the nine months ended September 30, 2020, the company reported a **net loss of $47.8 million** on **$147.3 million in net product sales**, with cash and equivalents increasing to **$200.5 million** due to a **$108.7 million equity offering** [Condensed Consolidated Balance Sheets](index=4&type=section&id=Condensed%20Consolidated%20Balance%20Sheets) Consolidated Balance Sheet Highlights (in thousands) | Balance Sheet Item | Sep 30, 2020 (Unaudited) | Dec 31, 2019 | | :--- | :--- | :--- | | **Assets** | | | | Cash and cash equivalents | $200,481 | $62,436 | | Total current assets | $497,682 | $429,064 | | Total assets | $692,220 | $604,800 | | **Liabilities & Equity** | | | | Total current liabilities | $75,426 | $95,449 | | Convertible debt | $212,651 | $204,861 | | Total liabilities | $390,807 | $383,604 | | Total stockholders' equity | $301,413 | $221,196 | [Condensed Consolidated Statements of Operations and Comprehensive Loss](index=5&type=section&id=Condensed%20Consolidated%20Statements%20of%20Operations%20and%20Comprehensive%20Loss) Statement of Operations Summary (in thousands, except per share data) | Metric | Q3 2020 | Q3 2019 | 9 Months 2020 | 9 Months 2019 | | :--- | :--- | :--- | :--- | :--- | | Net product sales | $51,139 | $44,373 | $147,338 | $128,651 | | Total operating expenses | $70,574 | $78,810 | $204,950 | $237,844 | | Operating loss | $(19,435) | $(34,437) | $(57,612) | $(109,193) | | Net loss | $(22,549) | $(36,490) | $(47,809) | $(116,168) | | Net loss per share (basic & diluted) | $(0.44) | $(0.85) | $(1.03) | $(2.76) | [Condensed Consolidated Statements of Cash Flows](index=6&type=section&id=Condensed%20Consolidated%20Statements%20of%20Cash%20Flows) Cash Flow Summary for the Nine Months Ended Sep 30 (in thousands) | Cash Flow Activity | 2020 | 2019 | | :--- | :--- | :--- | | Net cash used in operating activities | $(28,873) | $(53,722) | | Net cash provided by investing activities | $64,184 | $18,283 | | Net cash provided by (used in) financing activities | $102,674 | $(2,172) | | **Net change in cash and cash equivalents** | **$138,045** | **$(37,685)** | [Notes to Unaudited Condensed Consolidated Financial Statements](index=8&type=section&id=Notes%20to%20Unaudited%20Condensed%20Consolidated%20Financial%20Statements) - The company is a biopharmaceutical firm focused on rare diseases, with key clinical programs like Sparsentan and Chenodal, and approved products including Chenodal, Cholbam, and Thiola/Thiola EC[26](index=26&type=chunk)[28](index=28&type=chunk)[32](index=32&type=chunk) Net Product Revenues (in thousands) | Product Line | Q3 2020 | Q3 2019 | 9 Months 2020 | 9 Months 2019 | | :--- | :--- | :--- | :--- | :--- | | Bile acid products | $22,912 | $19,938 | $66,766 | $59,258 | | Tiopronin products | $28,227 | $24,435 | $80,572 | $69,393 | | **Total** | **$51,139** | **$44,373** | **$147,338** | **$128,651** | - In June 2020, the company raised **$108.7 million** in net proceeds from an underwritten public offering of **7.5 million shares** of common stock at **$15.50 per share**[113](index=113&type=chunk) - Post-quarter, on October 21, 2020, the company agreed to acquire Orphan Technologies Limited and its candidate OT-58 for a **$90 million upfront cash payment** and up to **$427 million in contingent payments**[115](index=115&type=chunk)[116](index=116&type=chunk) [Management's Discussion and Analysis of Financial Condition and Results of Operations](index=22&type=section&id=Item%202.%20Management%27s%20Discussion%20and%20Analysis%20of%20Financial%20Condition%20and%20Results%20of%20Operations) Net product sales increased for the first nine months of 2020 due to higher patient counts, operating expenses decreased from R&D program discontinuation, and liquidity was significantly enhanced by an equity offering [Results of Operations](index=25&type=section&id=Results%20of%20Operations) Net Product Sales Analysis (in thousands) | Product Line | 9 Months 2020 | 9 Months 2019 | Change | | :--- | :--- | :--- | :--- | | Bile acid products | $66,766 | $59,258 | $7,508 | | Tiopronin products | $80,572 | $69,393 | $11,179 | | **Total** | **$147,338** | **$128,651** | **$18,687** | - The increase in sales for the three and nine months ended September 30, 2020, was attributed to **higher patient counts** and fluctuations in the timing of new patient starts and refills[136](index=136&type=chunk) - Research and development expenses decreased by **$11.2 million** for the nine months ended September 30, 2020, primarily due to the discontinuation of the fosmetpantotenate program in PKAN[139](index=139&type=chunk)[162](index=162&type=chunk) - For the nine months ended September 30, 2020, the company recognized an **income tax benefit of $18.9 million**, primarily related to CARES Act provisions for net operating loss carrybacks[146](index=146&type=chunk) [Liquidity and Capital Resources](index=26&type=section&id=Liquidity%20and%20Capital%20Resources) - Management believes available cash and short-term investments are sufficient to fund anticipated operations for at least the next **12 months**[148](index=148&type=chunk)[165](index=165&type=chunk) Key Liquidity Metrics (in thousands) | Metric | Sep 30, 2020 | Dec 31, 2019 | | :--- | :--- | :--- | | Cash & Cash Equivalents | $200,481 | $62,436 | | Debt securities | $255,786 | $336,088 | | Net Working Capital | $422,256 | $333,615 | - Cash from financing activities increased significantly to **$102.7 million** for the nine months ended September 30, 2020, primarily due to the June 2020 public offering of common stock[164](index=164&type=chunk) [Quantitative and Qualitative Disclosures About Market Risk](index=29&type=section&id=Item%203.%20Quantitative%20and%20Qualitative%20Disclosures%20About%20Market%20Risk) The company's primary market risk is interest rate changes affecting its debt securities, with a hypothetical **1% adverse change** decreasing value by approximately **$1.4 million** - The company invests excess cash in U.S. government-backed securities and investment-grade corporate debt with maturities not exceeding **two years**[170](index=170&type=chunk) - A hypothetical **1% adverse move** in interest rates would decrease the value of the company's available-for-sale debt securities by approximately **$1.4 million** as of September 30, 2020[170](index=170&type=chunk) [Controls and Procedures](index=29&type=section&id=Item%204.%20Controls%20and%20Procedures) The CEO and CFO concluded that disclosure controls and procedures were effective as of September 30, 2020, with no significant changes to internal control over financial reporting during the quarter - Management, including the CEO and CFO, concluded that the company's disclosure controls and procedures were effective as of the end of the quarter[172](index=172&type=chunk) - No material changes in internal control over financial reporting occurred during the quarter[173](index=173&type=chunk) [PART II – OTHER INFORMATION](index=29&type=section&id=PART%20II%20%E2%80%93%20OTHER%20INFORMATION) [Legal Proceedings](index=29&type=section&id=Item%201.%20Legal%20Proceedings) Legal proceedings include advancing legal fees for former CEO Martin Shkreli and a dismissed antitrust lawsuit from Spring Pharmaceuticals regarding Thiola® samples - The company advanced legal fees for its former CEO, Martin Shkreli, with **$3.8 million** reimbursed by insurance carriers who reserve recovery rights[97](index=97&type=chunk) - A lawsuit by Spring Pharmaceuticals alleging antitrust violations related to Thiola® samples was voluntarily dismissed in April 2020[98](index=98&type=chunk) [Risk Factors](index=29&type=section&id=Item%201A.%20Risk%20Factors) Key risks include potential clinical trial failures, generic competition, manufacturing dependence, significant convertible debt, regulatory and reimbursement changes, and challenges with the planned Orphan Technologies acquisition, exacerbated by COVID-19 impacts [Risks Related to Product Candidate Development](index=29&type=section&id=Risks%20Related%20to%20the%20Development%20of%20our%20Product%20Candidates) - Clinical trials for product candidates like sparsentan may fail to demonstrate safety and efficacy, potentially delaying or preventing regulatory approval, with risks amplified by the COVID-19 pandemic[176](index=176&type=chunk)[179](index=179&type=chunk) - The company is pursuing an accelerated approval pathway (Subpart H) for sparsentan based on a proteinuria endpoint, but regulatory bodies may not deem the data sufficient for approval[188](index=188&type=chunk)[189](index=189&type=chunk) - Delays in patient enrollment for DUPLEX or PROTECT studies, potentially due to COVID-19, could push back timelines for top-line results and regulatory approval filings[193](index=193&type=chunk) [Risks Related to Commercialization](index=33&type=section&id=Risks%20Related%20to%20the%20Commercialization%20of%20Our%20Products) - The company faces generic competition for products like Thiola, with legislative changes such as the CREATES Act designed to encourage this competition[206](index=206&type=chunk)[207](index=207&type=chunk) - Changes in third-party payer reimbursement practices and potential loss of patient insurance coverage due to economic factors like COVID-19 could negatively affect product demand and pricing[211](index=211&type=chunk)[215](index=215&type=chunk) - The company relies on sole-source third-party manufacturers and a single third-party distributor for its commercial products, creating significant supply chain risk due to a lack of internal manufacturing capabilities[216](index=216&type=chunk)[217](index=217&type=chunk) [Risks Related to Business Operations](index=40&type=section&id=Risks%20Related%20to%20Our%20Business) - The COVID-19 pandemic could materially adversely affect business by disrupting clinical trials, impacting the supply chain, and reducing access to capital markets[266](index=266&type=chunk)[267](index=267&type=chunk) - The company may require substantial additional funding for development and commercialization, and market conditions could hinder raising capital on attractive terms[282](index=282&type=chunk) - The planned acquisition of Orphan Technologies is subject to closing conditions and may not be completed, or if completed, integrating the new business presents potential difficulties[288](index=288&type=chunk)[289](index=289&type=chunk) [Risks Related to Indebtedness](index=49&type=section&id=Risks%20Related%20to%20our%20Indebtedness%20and%20Investments) - As of September 30, 2020, the company had approximately **$276 million** of total debt outstanding from its **2.50% Convertible Senior Notes due 2025**, which could adversely affect its financial condition[338](index=338&type=chunk) - The company may lack sufficient cash to repurchase the 2025 Notes after a fundamental change or to pay cash upon conversion, potentially leading to default[340](index=340&type=chunk)[342](index=342&type=chunk) [Unregistered Sales of Equity Securities and Use of Proceeds](index=50&type=section&id=Item%202.%20Unregistered%20Sales%20of%20Equity%20Securities%20and%20Use%20of%20Proceeds) The company reported no unregistered sales of equity securities during the period - None [Defaults Upon Senior Securities](index=50&type=section&id=Item%203.%20Defaults%20Upon%20Senior%20Securities) The company reported no defaults upon senior securities - None [Mine Safety Disclosures](index=50&type=section&id=Item%204.%20Mine%20Safety%20Disclosures) This item is not applicable to the company - Not applicable [Other Information](index=50&type=section&id=Item%205.%20Other%20Information) The company reported no other information for this item - None [Exhibits](index=51&type=section&id=Item%206.%20Exhibits) This section lists exhibits filed with the Form 10-Q, including corporate governance documents, convertible note indentures, agreements, and CEO/CFO certifications - Exhibits include CEO and CFO certifications pursuant to the Sarbanes-Oxley Act of 2002, and indentures related to the company's convertible notes[351](index=351&type=chunk)

Sparsentan Clinical Development - Sparsentan is in Phase 3 development for FSGS and IgAN, rare kidney disorders with no approved medicines, targeting ~40,000 FSGS patients in the U S and EU and ~100,000+ IgAN patients in the U S and EU [58] - Phase 2 DUET study showed Sparsentan more than doubled the reduction of proteinuria vs Irbesartan (44 8% vs 18 5%, p=0 006) [30] - In the DUET study, 28 1% of Sparsentan-treated patients achieved FPRE (UP/C ≤ 1 5 g/g and >40% reduction in UP/C) compared to 9 4% in the Irbesartan group (p=0 040) [33, 35] - eGFR remained stable in Sparsentan-treated patients over 84 weeks in the DUET study [40] - Phase 3 DUPLEX study for FSGS and PROTECT study for IgAN are enrolling, with top-line data expected in 1Q21 and 2H21, respectively [45, 48] Financial Overview - Retrophin reported net sales of $48 4 million in 2Q20 and $175 3 million in FY19 [56] - The company's operating expenses were $71 5 million in 2Q20 and $312 7 million in FY19 [56] - Retrophin had a cash balance of approximately $457 4 million as of June 30, 2020, which is expected to fund operations beyond the planned proteinuria readout of pivotal studies and into 2023 [56, 59] Commercial Portfolio - Retrophin has a diversified product portfolio for rare diseases, including Chenodal, Cholbam, and Thiola [51] - The company estimates an addressable population of 4,000 to 5,000 cystinuria patients for Thiola [51] - Net product sales have grown from $28 million in FY 2014 to $175 million in FY 2019 [53]

PART I – FINANCIAL INFORMATION [Financial Statements](index=4&type=section&id=Item%201.%20Financial%20Statements) Financial statements reflect increased cash and equity from a June 2020 offering, alongside product sales growth and a narrowed net loss [Condensed Consolidated Balance Sheets](index=4&type=section&id=Condensed%20Consolidated%20Balance%20Sheets) Total assets increased due to a substantial rise in cash from an equity offering, while liabilities decreased and equity grew Condensed Consolidated Balance Sheet Highlights (in thousands) | Account | June 30, 2020 (Unaudited) | December 31, 2019 | | :--- | :--- | :--- | | **Assets** | | | | Cash and cash equivalents | $237,170 | $62,436 | | Total current assets | $505,562 | $429,064 | | Total assets | $678,694 | $604,800 | | **Liabilities & Equity** | | | | Total current liabilities | $69,933 | $95,449 | | Convertible debt | $210,009 | $204,861 | | Total liabilities | $360,049 | $383,604 | | Total stockholders' equity | $318,645 | $221,196 | [Condensed Consolidated Statements of Operations and Comprehensive Loss](index=5&type=section&id=Condensed%20Consolidated%20Statements%20of%20Operations%20and%20Comprehensive%20Loss) Net product sales increased, and net loss significantly narrowed for both the quarter and six-month period, aided by lower expenses and a tax benefit Statement of Operations Summary (in thousands, except per share data) | Metric | Q2 2020 | Q2 2019 | Six Months 2020 | Six Months 2019 | | :--- | :--- | :--- | :--- | :--- | | Net product sales | $48,430 | $44,707 | $96,199 | $84,277 | | Research and development | $30,790 | $37,934 | $61,038 | $71,377 | | Selling, general and administrative | $34,971 | $38,970 | $68,110 | $71,639 | | Operating loss | ($23,111) | ($36,529) | ($38,176) | ($74,757) | | Net loss | ($26,068) | ($38,701) | ($25,260) | ($79,678) | | Basic and diluted net loss per share | ($0.58) | ($0.92) | ($0.57) | ($1.91) | - For the six months ended June 30, 2020, the company recorded an income tax benefit of **$18.9 million**, compared to an expense of $470 thousand in the prior year period[18](index=18&type=chunk) [Condensed Consolidated Statements of Cash Flows](index=6&type=section&id=Condensed%20Consolidated%20Statements%20of%20Cash%20Flows) Net cash increased significantly due to financing activities, primarily from common stock issuance, despite cash used in operations Cash Flow Summary for the Six Months Ended June 30 (in thousands) | Activity | 2020 | 2019 | | :--- | :--- | :--- | | Net cash used in operating activities | ($35,608) | ($39,601) | | Net cash provided by investing activities | $107,353 | $13,818 | | Net cash provided by (used in) financing activities | $103,022 | ($1,434) | | **Net change in cash and cash equivalents** | **$174,734** | **($27,216)** | | Cash and cash equivalents, end of period | $237,170 | $75,657 | - The primary source of financing cash flow was **$108.6 million** in net proceeds from the issuance of common stock[21](index=21&type=chunk) [Notes to Unaudited Condensed Consolidated Financial Statements](index=8&type=section&id=Notes%20to%20Unaudited%20Condensed%20Consolidated%20Financial%20Statements) Notes detail the company's biopharmaceutical focus, approved products, clinical programs, debt, and key financial events including a tax benefit and stock offering - The company is a biopharmaceutical firm focused on rare diseases with three approved products: **Chenodal®**, **Cholbam®**, and **Thiola®/Thiola EC®**[26](index=26&type=chunk)[32](index=32&type=chunk) - Key clinical programs include two pivotal Phase 3 studies for **sparsentan** in FSGS and IgAN, and a Phase 3 study for **Chenodal** in CTX[28](index=28&type=chunk) Net Product Revenue by Category (in thousands) | Product Category | Q2 2020 | Q2 2019 | Six Months 2020 | Six Months 2019 | | :--- | :--- | :--- | :--- | :--- | | Bile acid products | $21,573 | $20,929 | $43,854 | $39,319 | | Tiopronin products | $26,857 | $23,778 | $52,345 | $44,958 | | **Total** | **$48,430** | **$44,707** | **$96,199** | **$84,277** | - The company has **$276.0 million** in aggregate principal of 2.50% Convertible Senior Notes due 2025[78](index=78&type=chunk) - A discrete income tax benefit of **$18.9 million** was recorded in the first six months of 2020 due to the CARES Act, which allows for the carryback of net operating losses[106](index=106&type=chunk) - In June 2020, the company sold **7.5 million shares** of common stock in a public offering, raising net proceeds of **$108.6 million**[114](index=114&type=chunk) [Management's Discussion and Analysis of Financial Condition and Results of Operations](index=21&type=section&id=Item%202.%20Management%27s%20Discussion%20and%20Analysis%20of%20Financial%20Condition%20and%20Results%20of%20Operations) Management discusses rare disease focus, COVID-19 impact, increased net product sales, decreased operating expenses, and sufficient cash for future operations - The **COVID-19 pandemic** has the potential to impact business, including slowing the pace of enrollment in clinical trials, but did not have a material adverse effect on financial results for the first half of 2020[118](index=118&type=chunk) Net Product Sales Comparison (in thousands) | Product Line | Six Months 2020 | Six Months 2019 | Change | | :--- | :--- | :--- | :--- | | Bile acid products | $43,854 | $39,319 | $4,535 | | Tiopronin products | $52,345 | $44,958 | $7,387 | | **Total** | **$96,199** | **$84,277** | **$11,922** | - R&D expenses decreased by **$10.3 million** for the first six months of 2020 compared to the same period in 2019, mainly due to the discontinuation of the fosmetpantotenate program in PKAN[139](index=139&type=chunk) - SG&A expenses decreased by **$3.5 million** for the first six months of 2020 compared to 2019, primarily due to lower legal expenses[141](index=141&type=chunk) - The company believes its available cash and short-term investments are **sufficient** to fund operations beyond the next 12 months[148](index=148&type=chunk) [Quantitative and Qualitative Disclosures About Market Risk](index=28&type=section&id=Item%203.%20Quantitative%20and%20Qualitative%20Disclosures%20About%20Market%20Risk) The company's primary market risk exposure is to changes in interest rates on its portfolio of debt securities, with a hypothetical 1% adverse move decreasing value by approximately $1.1 million - The company's main market risk is **interest rate risk** on its portfolio of cash and debt securities[170](index=170&type=chunk) - A hypothetical **1% adverse change** in interest rates would result in a decrease of approximately **$1.1 million** in the fair value of the company's available-for-sale debt securities[170](index=170&type=chunk) [Controls and Procedures](index=28&type=section&id=Item%204.%20Controls%20and%20Procedures) Management concluded that the company's disclosure controls and procedures were effective as of June 30, 2020, with no significant changes in internal control - The CEO and CFO concluded that the company's disclosure controls and procedures were **effective** at a reasonable assurance level as of the end of the quarter[172](index=172&type=chunk) - **No significant changes** to internal control over financial reporting were identified during the quarter ended June 30, 2020[173](index=173&type=chunk) PART II – OTHER INFORMATION [Legal Proceedings](index=28&type=section&id=Item%201.%20Legal%20Proceedings) A lawsuit alleging antitrust violations against the company was voluntarily dismissed in April 2020, with no other material adverse proceedings identified - Information on legal proceedings is incorporated by reference from Note 12 of the financial statements[174](index=174&type=chunk) - A lawsuit by Spring Pharmaceuticals alleging antitrust violations was **voluntarily dismissed** in April 2020[99](index=99&type=chunk) [Risk Factors](index=28&type=section&id=Item%201A.%20Risk%20Factors) The company outlines significant risks including clinical trial failures, commercialization challenges, lack of patent protection, convertible debt, and extensive regulatory compliance [Risks Related to the Development of our Product Candidates](index=28&type=section&id=Risks%20Related%20to%20the%20Development%20of%20our%20Product%20Candidates) Risks include potential clinical trial failures for product candidates like sparsentan, exacerbated by COVID-19, and uncertainty regarding regulatory acceptance of surrogate endpoints - Clinical trials for product candidates like sparsentan may **fail to demonstrate safety and efficacy**, which could prevent or delay regulatory approval[176](index=176&type=chunk) - The **COVID-19 pandemic** has amplified risks and may **delay clinical trial timelines** due to slower patient enrollment and other disruptions[180](index=180&type=chunk)[193](index=193&type=chunk)[269](index=269&type=chunk) - There is **no guarantee** that the FDA or EMA will accept **proteinuria as a surrogate endpoint** for accelerated or conditional approval for sparsentan, despite prior feedback[189](index=189&type=chunk)[190](index=190&type=chunk) - The company **relies on independent investigators and CROs**, whose performance may be substandard or impacted by competing interests, a risk heightened by the introduction of new home health providers due to COVID-19[201](index=201&type=chunk) [Risks Related to the Commercialization of Our Products](index=32&type=section&id=Risks%20Related%20to%20the%20Commercialization%20of%20Our%20Products) Commercialization risks include generic competition, changes in reimbursement practices, and supply chain vulnerability due to reliance on sole-source third-party manufacturers and distributors - The company is subject to **generic competition** for products like Thiola, and recent legislative changes like the CREATES Act are designed to encourage such competition[205](index=205&type=chunk)[206](index=206&type=chunk)[207](index=207&type=chunk) - **Changes in reimbursement policies** from government and private payers, and potential **loss of insurance coverage** by patients due to unemployment, could adversely affect demand and pricing[211](index=211&type=chunk)[212](index=212&type=chunk)[214](index=214&type=chunk) - The company has no manufacturing capabilities and relies on **sole-source third-party manufacturers and a single third-party distributor** for its products[216](index=216&type=chunk)[217](index=217&type=chunk) [Risks Related to our Products and Product Candidates](index=33&type=section&id=Risks%20Related%20to%20our%20Products%20and%20Product%20Candidates) Key product risks include lack of patent protection for commercial products, potential product liability, and dependence on third-party manufacturers for regulatory compliance and supply - The company **does not have patent protection** for its commercial products Thiola, Chenodal, or Cholbam[233](index=233&type=chunk)[236](index=236&type=chunk) - The product candidate **sparsentan** is covered by **use patents expiring in 2030**, but its **composition of matter patent expired in 2019**[237](index=237&type=chunk) - The company faces potential **product liability exposure** in excess of its insurance coverage, which is **$10 million per occurrence** and **$25 million in aggregate** for clinical trials[256](index=256&type=chunk) - The company is **entirely dependent on third-party manufacturers**, which entails risks related to regulatory compliance (cGMP), supply capacity, and quality control[260](index=260&type=chunk)[261](index=261&type=chunk)[262](index=262&type=chunk) [Risks Related to Our Business](index=39&type=section&id=Risks%20Related%20to%20Our%20Business) Business risks include COVID-19 disruptions, uncertain profitability, extensive government regulation, potential need for additional funding, and vulnerability to cyber-attacks - The **COVID-19 pandemic** could **materially adversely affect business** by disrupting clinical trials, the supply chain, and access to capital markets[268](index=268&type=chunk) - The company is subject to **extensive and complex federal and state regulations**, including the Anti-Kickback statute, False Claims Act, and data privacy laws like HIPAA and CCPA, with **significant penalties** for non-compliance[297](index=297&type=chunk)[298](index=298&type=chunk)[308](index=308&type=chunk) - The company may need **substantial additional funding** for its development programs and commercialization, and **access to capital may be difficult**[281](index=281&type=chunk) - Internal computer systems are **vulnerable to security breaches and cyber-attacks**, which could result in data loss, liability, and disruption of development programs[322](index=322&type=chunk)[323](index=323&type=chunk) [Risks Related to our Indebtedness and Investments](index=48&type=section&id=Risks%20Related%20to%20our%20Indebtedness%20and%20Investments) Significant risks relate to the $276 million convertible senior notes, including cash flow requirements, potential inability to repurchase notes, and dilution of existing stockholders upon conversion - The company has approximately **$276 million of total debt** outstanding from its 2025 Convertible Senior Notes, which could adversely affect its financial condition and limit flexibility[333](index=333&type=chunk)[334](index=334&type=chunk) - The company may be **unable to raise the funds necessary to repurchase the notes** upon a 'fundamental change' or to pay cash amounts due upon conversion, which would constitute a default[335](index=335&type=chunk)[336](index=336&type=chunk) - Conversion of the notes into common stock will **dilute the ownership interests** of existing stockholders[341](index=341&type=chunk) [Unregistered Sales of Equity Securities and Use of Proceeds](index=49&type=section&id=Item%202.%20Unregistered%20Sales%20of%20Equity%20Securities%20and%20Use%20of%20Proceeds) The company reported no unregistered sales of equity securities during the period - None[342](index=342&type=chunk) [Defaults Upon Senior Securities](index=49&type=section&id=Item%203.%20Defaults%20Upon%20Senior%20Securities) The company reported no defaults upon senior securities during the period - None[343](index=343&type=chunk) [Mine Safety Disclosures](index=49&type=section&id=Item%204.%20Mine%20Safety%20Disclosures) This item is not applicable to the company - Not applicable[344](index=344&type=chunk) [Other Information](index=49&type=section&id=Item%205.%20Other%20Information) The company reported no other information required to be disclosed in this item - None[345](index=345&type=chunk) [Exhibits](index=50&type=section&id=Item%206.%20Exhibits) This section lists various exhibits filed with the Quarterly Report on Form 10-Q, including corporate governance and debt agreements - Lists various exhibits filed with the report, including indentures for convertible notes, equity incentive plans, and CEO/CFO certifications[347](index=347&type=chunk)

UNITED STATES SECURITIES AND EXCHANGE COMMISSION WASHINGTON, D.C. 20549 _________________________________ FORM 10-Q _________________________________ ☑ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended March 31, 2020 or ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from ___________ to ___________ Commission File Number: 001-36257 RETROPHIN, INC. (Exact name of registran ...

UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 FORM 10-K ☑ ANNUAL REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the Fiscal Year Ended December 31, 2019 Commission File Number: 001-36257 RETROPHIN, INC. (Exact Name of Registrant as specified in its Charter) Delaware 27-4842691 (State or other jurisdiction of incorporation or organization) (I.R.S. Employer Identification No.) 3721 Valley Centre Drive, Suite 200 San Diego, CA 92130 (Address of Princi ...

[PART I – FINANCIAL INFORMATION](index=5&type=section&id=PART%20I%20%E2%80%93%20FINANCIAL%20INFORMATION) [Item 1. Financial Statements](index=5&type=section&id=Item%201.%20Financial%20Statements) This section presents unaudited condensed consolidated financial statements for Q3 and nine months ended September 30, 2019, detailing balance sheets, operations, cash flows, and notes on accounting policies and program discontinuations [Condensed Consolidated Balance Sheets](index=5&type=section&id=Condensed%20Consolidated%20Balance%20Sheets) Condensed Consolidated Balance Sheet Highlights (in thousands) | Account | September 30, 2019 | December 31, 2018 | | :--- | :--- | :--- | | **Assets** | | | | Cash and cash equivalents | $65,188 | $102,873 | | Marketable securities | $341,835 | $368,668 | | Total current assets | $439,718 | $495,678 | | Total assets | $613,816 | $709,160 | | **Liabilities & Equity** | | | | Total current liabilities | $88,017 | $104,621 | | Total liabilities | $367,859 | $390,907 | | Total stockholders' equity | $245,957 | $318,253 | [Condensed Consolidated Statements of Operations and Comprehensive Loss](index=6&type=section&id=Condensed%20Consolidated%20Statements%20of%20Operations%20and%20Comprehensive%20Loss) Statement of Operations Summary (in thousands, except per share data) | Metric | Q3 2019 | Q3 2018 | 9 Months 2019 | 9 Months 2018 | | :--- | :--- | :--- | :--- | :--- | | Net product sales | $44,373 | $40,706 | $128,651 | $120,475 | | Total operating expenses | $78,810 | $76,289 | $237,844 | $195,530 | | Operating loss | $(34,437) | $(35,583) | $(109,193) | $(75,055) | | Net loss | $(36,490) | $(54,516) | $(116,168) | $(95,223) | | Net loss per share (basic & diluted) | $(0.85) | $(1.34) | $(2.76) | $(2.37) | - The company recorded a **$15.0 million impairment** of a long-term investment in Q3 2019. For the nine-month period, significant expenses included a **$25.5 million impairment** of an IPR&D intangible asset and a **$15.0 million investment impairment**, partially offset by an **$18.0 million write-off** of related contingent consideration[18](index=18&type=chunk) [Condensed Consolidated Statements of Cash Flows](index=7&type=section&id=Condensed%20Consolidated%20Statements%20of%20Cash%20Flows) Cash Flow Summary for the Nine Months Ended September 30 (in thousands) | Cash Flow Activity | 2019 | 2018 | | :--- | :--- | :--- | | Net cash used in operating activities | $(53,722) | $(21,393) | | Net cash provided by (used in) investing activities | $18,283 | $(154,489) | | Net cash (used in) provided by financing activities | $(2,172) | $231,338 | | **Net change in cash and cash equivalents** | **$(37,685)** | **$55,426** | [Notes to Unaudited Condensed Consolidated Financial Statements](index=9&type=section&id=Notes%20to%20Unaudited%20Condensed%20Consolidated%20Financial%20Statements) These notes detail accounting policies, financial results, and key events including the discontinuation of three development programs (fosmetpantotenate, CNSA-001, L-UDCA) and related impairment charges - In August 2019, the Phase 3 FORT Study for **fosmetpantotenate** in PKAN failed to meet its primary and secondary endpoints, leading to the discontinuation of the program[27](index=27&type=chunk) - The company declined its option to acquire Censa Pharmaceuticals in August 2019 and discontinued the **CNSA-001** program, resulting in a **$15 million impairment** of its long-term investment[28](index=28&type=chunk)[53](index=53&type=chunk)[104](index=104&type=chunk) - In Q1 2019, the company discontinued the **L-UDCA** program, leading to a **$25.5 million impairment** of the intangible asset and the reversal of an **$18.0 million contingent liability**, resulting in a net **$7.5 million non-cash charge**[29](index=29&type=chunk)[103](index=103&type=chunk) Net Product Revenues (in thousands) | Product Category | Q3 2019 | Q3 2018 | 9 Months 2019 | 9 Months 2018 | | :--- | :--- | :--- | :--- | :--- | | Bile acid products | $19,938 | $18,052 | $59,258 | $55,153 | | Tiopronin products | $24,435 | $22,654 | $69,393 | $65,322 | | **Total** | **$44,373** | **$40,706** | **$128,651** | **$120,475** | - In May 2019, the remaining **$22.6 million principal amount** of the **4.50% senior convertible notes due 2019** was converted by holders into approximately **1.3 million shares** of common stock[84](index=84&type=chunk)[147](index=147&type=chunk) [Management's Discussion and Analysis of Financial Condition and Results of Operations](index=21&type=section&id=Item%202.%20Management's%20Discussion%20and%20Analysis%20of%20Financial%20Condition%20and%20Results%20of%20Operations) Management discusses financial performance, noting increased net product sales, rising operating expenses due to R&D and SG&A, strategic program discontinuations, and analysis of liquidity and capital resources [Overview](index=22&type=section&id=Overview) - The company's key investigational product candidate, **sparsentan**, is being evaluated in two pivotal Phase 3 studies: the **DUPLEX Study for FSGS** and the **PROTECT Study for IgAN**[109](index=109&type=chunk) - Top-line data from the interim analysis of the **DUPLEX Study** (sparsentan in FSGS) are expected in the **first half of 2021** and may support an accelerated approval submission[110](index=110&type=chunk) - Top-line data from the primary endpoint of the **PROTECT Study** (sparsentan in IgAN) are expected in the **first half of 2022** and may also support an accelerated approval submission[111](index=111&type=chunk) - In June 2019, the FDA approved **THIOLA EC**, a new enteric-coated formulation of tiopronin for cystinuria, which became available to patients in July 2019[118](index=118&type=chunk) [Results of Operations](index=24&type=section&id=Results%20of%20Operations) Operating Expense Changes (in thousands) | Expense Category | 9 Months 2019 | 9 Months 2018 | Change | Reason for Change | | :--- | :--- | :--- | :--- | :--- | | Research and development | $104,597 | $91,544 | $13,053 | Increased clinical trial expenses for sparsentan studies | | Selling, general and administrative | $101,418 | $77,675 | $23,743 | Increased compensation and legal expenses | | Change in fair value of contingent consideration | $5,820 | $22,387 | $(16,567) | Changes in projected revenues and passage of time | [Liquidity and Capital Resources](index=25&type=section&id=Liquidity%20and%20Capital%20Resources) - Management believes that available cash and short-term investments will be sufficient to fund operations for at least the **next 12 months**[136](index=136&type=chunk)[151](index=151&type=chunk) Financial Position (in thousands) | Metric | September 30, 2019 | December 31, 2018 | | :--- | :--- | :--- | | Cash & Cash Equivalents | $65,188 | $102,873 | | Marketable securities | $341,835 | $368,668 | | Net Working Capital | $351,701 | $391,057 | - Cash used in operating activities increased to **$53.7 million** for the nine months ended September 30, 2019, compared to **$21.4 million** for the same period in 2018, primarily due to higher R&D and legal expenses[148](index=148&type=chunk) [Quantitative and Qualitative Disclosures About Market Risk](index=29&type=section&id=Item%203.%20Quantitative%20and%20Qualitative%20Disclosures%20About%20Market%20Risk) The company's primary market risk is interest rate changes affecting marketable securities, with a hypothetical **1% adverse change** potentially decreasing their value by **$1.8 million** - The company invests excess cash in investment-grade securities with maturities not exceeding **two years** and does not use derivative instruments[157](index=157&type=chunk) - A hypothetical **1% adverse move** in interest rates would decrease the value of the company's available-for-sale marketable securities by approximately **$1.8 million**[157](index=157&type=chunk) [Controls and Procedures](index=29&type=section&id=Item%204.%20Controls%20and%20Procedures) The CEO and CFO concluded that disclosure controls and procedures were effective as of September 30, 2019, with no significant changes to internal control over financial reporting during the quarter - The CEO and CFO concluded that the company's disclosure controls and procedures were effective at a reasonable assurance level as of **September 30, 2019**[159](index=159&type=chunk) - No significant changes to internal control over financial reporting were identified during the quarter ended **September 30, 2019**[160](index=160&type=chunk) [PART II – OTHER INFORMATION](index=29&type=section&id=PART%20II%20%E2%80%93%20OTHER%20INFORMATION) [Legal Proceedings](index=29&type=section&id=Item%201.%20Legal%20Proceedings) This section incorporates legal proceedings information from Note 12, including a lawsuit from Spring Pharmaceuticals regarding alleged antitrust violations related to Thiola® sales - Information regarding legal proceedings is incorporated by reference from Note 12 to the Unaudited Condensed Consolidated Financial Statements[161](index=161&type=chunk) [Risk Factors](index=29&type=section&id=Item%201A.%20Risk%20Factors) This section details significant risks, including potential clinical trial failures for sparsentan, regulatory approval uncertainty, generic competition, reliance on third-party manufacturers, pricing pressures, and convertible debt risks - The company's clinical trials for product candidates like **sparsentan** may fail to demonstrate safety and efficacy, potentially preventing or significantly delaying regulatory approval[163](index=163&type=chunk) - The company faces immediate generic competition for products like **Thiola** and is involved in a lawsuit with **Spring Pharmaceuticals**, also receiving a civil investigative demand from the FTC regarding this matter[187](index=187&type=chunk)[189](index=189&type=chunk) - The company relies on third-party sole source suppliers for manufacturing its products, posing risks related to supply chain disruption, regulatory compliance, and cost[195](index=195&type=chunk)[232](index=232&type=chunk) - The company's outstanding **$276 million** in **2.50% Convertible Senior Notes due 2025** could adversely affect its financial condition by requiring significant cash flow for debt service and potentially limiting financial flexibility[295](index=295&type=chunk)[296](index=296&type=chunk) [Unregistered Sales of Equity Securities and Use of Proceeds](index=49&type=section&id=Item%202.%20Unregistered%20Sales%20of%20Equity%20Securities%20and%20Use%20of%20Proceeds) The company reported no unregistered sales of equity securities during the reporting period - None[304](index=304&type=chunk) [Defaults Upon Senior Securities](index=49&type=section&id=Item%203.%20Defaults%20Upon%20Senior%20Securities) The company reported no defaults upon its senior securities - None[305](index=305&type=chunk) [Mine Safety Disclosures](index=49&type=section&id=Item%204.%20Mine%20Safety%20Disclosures) This item is not applicable to the company - Not applicable[306](index=306&type=chunk) [Other Information](index=49&type=section&id=Item%205.%20Other%20Information) The company reported no other information required to be disclosed under this item - None[307](index=307&type=chunk) [Exhibits](index=50&type=section&id=Item%206.%20Exhibits) This section lists all exhibits filed with the Form 10-Q, including corporate documents, debt indentures, and CEO/CFO certifications required by Sarbanes-Oxley Act - The report includes certifications from the Chief Executive Officer and Chief Financial Officer pursuant to Sections 302 and 906 of the Sarbanes-Oxley Act of 2002[309](index=309&type=chunk)

[PART I – FINANCIAL INFORMATION](index=5&type=section&id=PART%20I%20%E2%80%93%20FINANCIAL%20INFORMATION) [Financial Statements](index=5&type=section&id=Item%201.%20Financial%20Statements) Net product sales increased to $84.3 million, but net loss widened to $79.7 million due to higher R&D and SG&A expenses and a program discontinuation charge [Condensed Consolidated Statements of Operations](index=6&type=section&id=Condensed%20Consolidated%20Statements%20of%20Operations) Q2 2019 net sales grew to $44.7 million, but increased expenses led to a $38.7 million net loss, with a $25.5 million H1 impairment charge Financial Performance (Three Months Ended June 30) | Financial Metric | Q2 2019 | Q2 2018 | Change | | :--- | :--- | :--- | :--- | | Net Product Sales | $44.7M | $41.3M | +8.1% | | R&D Expenses | $37.9M | $34.5M | +9.8% | | SG&A Expenses | $39.0M | $25.1M | +55.4% | | Operating Loss | ($36.5M) | ($21.6M) | +69.4% | | Net Loss | ($38.7M) | ($22.3M) | +73.5% | | Net Loss Per Share | ($0.92) | ($0.56) | +64.3% | Financial Performance (Six Months Ended June 30) | Financial Metric | H1 2019 | H1 2018 | Change | | :--- | :--- | :--- | :--- | | Net Product Sales | $84.3M | $79.8M | +5.6% | | R&D Expenses | $71.4M | $59.1M | +20.8% | | SG&A Expenses | $71.6M | $51.6M | +38.8% | | Operating Loss | ($74.8M) | ($39.5M) | +89.4% | | Net Loss | ($79.7M) | ($40.7M) | +95.8% | | Net Loss Per Share | ($1.91) | ($1.03) | +85.4% | - In the first half of 2019, the company recorded a **$25.5 million impairment charge** for the L-UDCA IPR&D intangible asset and a corresponding **$18.0 million write-off** of the related contingent consideration, resulting in a net non-cash charge of **$7.5 million**[18](index=18&type=chunk)[105](index=105&type=chunk) [Condensed Consolidated Balance Sheets](index=5&type=section&id=Condensed%20Consolidated%20Balance%20Sheets) Total assets decreased to $647.6 million by June 30, 2019, due to reduced cash and marketable securities; liabilities decreased to $370.5 million from debt conversion Key Balance Sheet Items (in thousands) | Account | June 30, 2019 | Dec 31, 2018 | | :--- | :--- | :--- | | Cash and cash equivalents | $75,657 | $102,873 | | Marketable securities | $350,243 | $368,668 | | Total Assets | $647,574 | $709,160 | | Total Liabilities | $370,485 | $390,907 | | Total Stockholders' Equity | $277,089 | $318,253 | [Condensed Consolidated Statements of Cash Flows](index=7&type=section&id=Condensed%20Consolidated%20Statements%20of%20Cash%20Flows) Net cash used in operating activities increased to $39.6 million for H1 2019, reducing cash and equivalents to $75.7 million by period-end Cash Flow Summary (Six Months Ended June 30, in thousands) | Cash Flow Activity | 2019 | 2018 | | :--- | :--- | :--- | | Net Cash Used in Operating Activities | ($39,601) | ($17,494) | | Net Cash Provided by Investing Activities | $13,818 | $7,056 | | Net Cash (Used in) Provided by Financing Activities | ($1,434) | $383 | | **Net Change in Cash** | **($27,216)** | **($10,089)** | | **Cash at End of Period** | **$75,657** | **$89,305** | [Notes to Unaudited Condensed Consolidated Financial Statements](index=9&type=section&id=Notes%20to%20Unaudited%20Condensed%20Consolidated%20Financial%20Statements) Notes detail program discontinuations, 2019 convertible note conversion, CEO settlement, and ongoing Thiola litigation - The company's three commercial products are **Chenodal**, **Cholbam**, and **Thiola**; key pipeline candidates are **fosmetpantotenate** for **PKAN** and **sparsentan** for **FSGS** and **IgAN**[25](index=25&type=chunk)[26](index=26&type=chunk)[29](index=29&type=chunk) - In May 2019, the remaining **$22.6 million** principal of the 4.50% Senior Convertible Notes due 2019 was converted by holders into approximately **1.3 million shares** of common stock[84](index=84&type=chunk) - The company discontinued the L-UDCA program in Q1 2019, resulting in a **$25.5 million** intangible asset write-off and the reversal of **$18.0 million** in associated contingent consideration[105](index=105&type=chunk) - In August 2019, the company decided not to exercise its option to acquire Censa Pharmaceuticals and will discontinue the joint development of CNSA-001, expecting to impair its **$15 million** investment in Q3 2019[106](index=106&type=chunk)[53](index=53&type=chunk) - The company is involved in a lawsuit with Spring Pharmaceuticals, which alleges the company refused to sell samples of **Thiola** for generic bioequivalence testing[92](index=92&type=chunk) [Management's Discussion and Analysis of Financial Condition and Results of Operations (MD&A)](index=22&type=section&id=Item%202.%20Management%27s%20Discussion%20and%20Analysis%20of%20Financial%20Condition%20and%20Results%20of%20Operations) MD&A highlights 5.6% revenue growth, increased operating expenses from Phase 3 trials and legal costs, and portfolio adjustments including program discontinuations [Overview of Business and Pipeline](index=23&type=section&id=Overview%20of%20Business%20and%20Pipeline) The company focuses on rare disease therapies, with pipeline updates including expected Phase 3 data, THIOLA® EC approval, and program discontinuations - Top-line data from the pivotal **Phase 3 FORT Study** of **fosmetpantotenate** in **PKAN** is expected in the **third quarter of 2019**[114](index=114&type=chunk) - Top-line data from the interim analysis of the **Phase 3 DUPLEX study** of **sparsentan** in **FSGS** are expected in the **first half of 2021**[116](index=116&type=chunk) - Top-line data from the primary endpoint of the **Phase 3 PROTECT study** of **sparsentan** in **IgAN** are expected in the **first half of 2022**[116](index=116&type=chunk) - The FDA approved **THIOLA® EC**, a new enteric-coated formulation of tiopronin, in **June 2019**, which became available to patients in **July 2019**[128](index=128&type=chunk) - The company discontinued the **L-UDCA program** in **Q1 2019** and decided in **August 2019** to discontinue the **CNSA-001 program** and decline its option to acquire Censa Pharmaceuticals[129](index=129&type=chunk)[130](index=130&type=chunk) [Results of Operations](index=25&type=section&id=Results%20of%20Operations) H1 2019 net sales increased by $4.5 million (5.6%), R&D expenses rose by $12.3 million (20.8%), and SG&A by $20.1 million (39.0%) due to trials, compensation, and legal costs Net Product Sales (in thousands) | Product | H1 2019 | H1 2018 | Change | | :--- | :--- | :--- | :--- | | Bile acid products | $39,319 | $37,102 | +$2,217 | | Thiola | $44,958 | $42,667 | +$2,291 | | **Total** | **$84,277** | **$79,769** | **+$4,508** | Operating Expense Changes (H1 2019 vs H1 2018, in thousands) | Expense Category | H1 2019 | H1 2018 | Change | | :--- | :--- | :--- | :--- | | Research and development | $71,377 | $59,096 | +$12,281 | | Selling, general and administrative | $71,639 | $51,568 | +$20,071 | - The increase in **R&D expenses** was due to increased clinical trial costs for the three ongoing **Phase 3 studies**: **fosmetpantotenate** in **PKAN** and **sparsentan** in **FSGS** and **IgAN**[136](index=136&type=chunk) - The increase in **SG&A expenses** was due to higher compensation costs from a larger employee base and increased legal expenses[138](index=138&type=chunk) [Liquidity and Capital Resources](index=27&type=section&id=Liquidity%20and%20Capital%20Resources) As of June 30, 2019, the company held $75.7 million in cash and $350.2 million in marketable securities, with net working capital of $366.4 million, sufficient for 12 months of operations Liquidity Position (in thousands) | Metric | June 30, 2019 | Dec 31, 2018 | | :--- | :--- | :--- | | Cash & Cash Equivalents | $75,657 | $102,873 | | Marketable securities | $350,243 | $368,668 | | Net Working Capital | $366,428 | $391,057 | - In May 2019, the remaining **$22.6 million** of 2019 Notes were converted into approximately **1.3 million shares** of common stock[155](index=155&type=chunk) - The company's primary debt is **$276.0 million** in **2.50% Convertible Senior Notes due 2025**, issued in September 2018[146](index=146&type=chunk) - Cash used in operating activities increased to **$39.6 million** in H1 2019 from **$17.5 million** in H1 2018, attributed to higher R&D and legal expenses[156](index=156&type=chunk) [Quantitative and Qualitative Disclosures about Market Risk](index=30&type=section&id=Item%203.%20Quantitative%20and%20Qualitative%20Disclosures%20about%20Market%20Risk) Primary market risk is interest rate exposure on investments, with a hypothetical 1% adverse change impacting marketable securities by $2.8 million - The company's main market risk is **interest rate risk** on its investment portfolio of cash and marketable securities[166](index=166&type=chunk) - A hypothetical **1%** adverse move in interest rates would result in an approximate **$2.8 million** decrease in the value of the company's available-for-sale marketable securities[166](index=166&type=chunk) [Controls and Procedures](index=30&type=section&id=Item%204.%20Controls%20and%20Procedures) Management concluded disclosure controls were effective as of June 30, 2019, with no material changes to internal control over financial reporting - The CEO and CFO concluded that the company's disclosure controls and procedures were **effective** as of the end of the quarter[168](index=168&type=chunk) - **No material changes** to the company's internal control over financial reporting were identified during the quarter[169](index=169&type=chunk) [PART II – OTHER INFORMATION](index=30&type=section&id=PART%20II%20%E2%80%93%20OTHER%20INFORMATION) [Legal Proceedings](index=30&type=section&id=Item%201.%20Legal%20Proceedings) The company settled disputes with former CEO Martin Shkreli in June 2019 and is defending an antitrust lawsuit from Spring Pharmaceuticals regarding Thiola® samples - The company and former CEO Martin Shkreli reached a **comprehensive settlement** in **June 2019**, dismissing all pending claims and counterclaims with prejudice[91](index=91&type=chunk) - **Spring Pharmaceuticals** filed a lawsuit in **October 2018** alleging the company violated antitrust laws by refusing to sell samples of **Thiola®** for generic testing[92](index=92&type=chunk) [Risk Factors](index=30&type=section&id=Item%201A.%20Risk%20Factors) The company faces significant risks including clinical trial failures, regulatory uncertainty, generic competition, pricing pressures, supply chain reliance, funding needs, litigation, and extensive government regulation [Risks Related to Product Development and Commercialization](index=30&type=section&id=Risks%20Related%20to%20Product%20Development%20and%20Commercialization) Product development and commercialization risks include clinical trial failures, uncertain regulatory approval, generic competition, pricing pressures, and reliance on sole-source manufacturers - Clinical trials may **fail to demonstrate safety and efficacy**, which could **prevent or delay regulatory approval** for product candidates like fosmetpantotenate and sparsentan[172](index=172&type=chunk) - The company is subject to **generic competition**; it is currently in **litigation with Spring Pharmaceuticals** over access to Thiola samples and has received a **civil investigative demand from the FTC** regarding its marketing and pricing practices[198](index=198&type=chunk)[200](index=200&type=chunk) - The company relies on **sole-source third-party manufacturers** for its products and a **single third-party distributor**, creating **significant supply chain and commercialization risk**[206](index=206&type=chunk)[207](index=207&type=chunk) - The **Special Protocol Assessment (SPA)** for the fosmetpantotenate Phase 3 trial and the potential for **accelerated approval (Subpart H)** for sparsentan studies **do not guarantee a favorable regulatory outcome**[183](index=183&type=chunk)[184](index=184&type=chunk) [Risks Related to Business Operations and Financial Condition](index=39&type=section&id=Risks%20Related%20to%20Business%20Operations%20and%20Financial%20Condition) Business operations and financial risks include fluctuating results, uncertain profitability, need for additional funding, product liability, litigation, extensive government regulation, and potential limitations on NOL carryforwards - The company expects **significant fluctuations in operating results** and may need to **raise substantial additional funding** to support its development and commercialization efforts[255](index=255&type=chunk)[260](index=260&type=chunk) - The company is subject to extensive and complex government regulations, including the **Anti-Kickback statute**, **False Claims Act**, and data privacy laws like **HIPAA** and the **California Consumer Privacy Act (CCPA)**[277](index=277&type=chunk)[278](index=278&type=chunk)[287](index=287&type=chunk) - The ability to use **$36.5 million** in federal NOL carryforwards to offset future taxable income may be subject to limitations under Section 382 of the Internal Revenue Code due to ownership changes[300](index=300&type=chunk) - The company faces potential **product liability exposure** and is involved in **litigation matters** that could result in substantial costs and divert management attention[237](index=237&type=chunk)[272](index=272&type=chunk) [Risks Related to Indebtedness](index=49&type=section&id=Risks%20Related%20to%20Indebtedness) The company's $276 million outstanding debt, primarily 2025 Convertible Notes, poses risks including cash flow impact, default risk, and potential dilution of existing stockholders upon conversion - As of June 30, 2019, the company had approximately **$276 million** of total debt outstanding, primarily related to its **2.50% Convertible Senior Notes due 2025**[304](index=304&type=chunk) - The company may not have sufficient funds to repurchase the **2025 Notes** for cash if required after a 'fundamental change' event, which would constitute a default[306](index=306&type=chunk)[307](index=307&type=chunk) - Conversion of the **2025 Notes** into common stock will **dilute the ownership interests** of existing stockholders[312](index=312&type=chunk)[313](index=313&type=chunk) [Other Information (Items 2, 3, 4, 5)](index=50&type=section&id=Item%202.%20Unregistered%20Sales%20of%20Equity%20Securities%20and%20Use%20of%20Proceeds) The company reported no unregistered equity sales, no defaults on senior securities, and no other material disclosures for the period - There were **no unregistered sales of equity securities** or **no defaults upon senior securities** during the quarter[314](index=314&type=chunk)[315](index=315&type=chunk)

RETROPHIN, INC. UNITED STATES SECURITIES AND EXCHANGE COMMISSION WASHINGTON, D.C. 20549 _________________________________ FORM 10-Q _________________________________ þ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended March 31, 2019 or ¨ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from ___________ to ___________ Commission File Number: 001-36257 (Exact name of registran ...