Financial Position and Funding - The company has sufficient funds to support operations for at least the next 12 months, but future commercialization success of FIRDAPSE®, FYCOMPA®, and AGAMREE® is uncertain [246]. - The company had cash and cash equivalents of approximately $137.6 million as of December 31, 2023, and raised approximately $140.1 million from a public offering of 10 million shares on January 9, 2024 [273]. - The company did not repurchase any common stock during the fiscal year ended December 31, 2023, with approximately $21 million remaining available under the Share Repurchase Program [385]. - The company is retaining cash for business development activities rather than repurchasing shares, indicating a focus on growth opportunities [385]. Product Development and Commercialization - FIRDAPSE® is currently being commercialized in the U.S. and Canada, with a focus on increasing disease awareness among healthcare providers [248]. - The company acquired U.S. rights to FYCOMPA® in Q1 2023 and has successfully launched its marketing efforts, adding a second product to its portfolio [248]. - AGAMREE® is set to launch in the U.S. by the end of Q1 2024, following an exclusive license agreement with Santhera Pharmaceuticals [248]. - The FDA accepted a supplemental NDA to increase the maximum daily dosage of FIRDAPSE® from 80 mg to 100 mg, with a PDUFA action date of June 4, 2024 [262]. - The company has hired approximately 35 sales and marketing personnel to support the marketing of FYCOMPA® [265]. - The company aims to obtain approval for FIRDAPSE® in Japan and expand into other territories in Asia and Latin America, with a sub-licensee, DyDo, currently pursuing marketing approval in Japan [275]. - The company has begun steps to expand FIRDAPSE® activities into several countries in Asia and Latin America following the acceptance of DyDo's NDA by the Japanese regulatory agency [294]. - The FDA approved AGAMREE® for the treatment of DMD in patients aged two and older on October 26, 2023 [310]. - The FDA approved AGAMREE® (vamorolone) oral suspension 40 mg/ml for the treatment of Duchenne Muscular Dystrophy (DMD) on October 26, 2023, with a planned commercial launch in Q1 2024 [389]. Strategic Investments and Partnerships - A $75 million payment was made to Santhera for the exclusive North American license for AGAMREE®, with an additional $36 million milestone payment due upon NDA approval [267]. - The company made a strategic investment in Santhera, acquiring 1,414,688 shares at CHF 9.477 per share, totaling approximately $15.7 million USD, which includes $13.5 million USD in fair value and $2.2 million USD in transaction costs [268]. - The company made a strategic investment of approximately $15.7 million in Santhera, acquiring 11.26% of its outstanding shares to support AGAMREE®'s Phase IV studies [312]. - The company entered into a definitive agreement with Eisai on January 24, 2023, to support clinical trials for FYCOMPA®, evaluating multiple dosages [335]. Market and Competitive Landscape - The company faces intense competition in the pharmaceutical industry, with many organizations having greater resources [346]. - The company must achieve significant market share and high per-patient prices for FIRDAPSE® and AGAMREE® to achieve meaningful gross margins due to small target patient populations [352]. - FIRDAPSE® has been widely covered and reimbursed by private and public payors for adult LEMS patients, indicating strong market acceptance [290]. Legal and Regulatory Matters - The company is actively involved in legal proceedings regarding the approval of RUZURGI®, which may impact its market position [256]. - The company filed lawsuits against the generic manufacturers on March 1, 2023, which could delay FDA approval of the ANDAs until May 2026 [298]. - The company has received a favorable court decision regarding RUZURGI® in Canada, allowing it to be sold again [322]. - The company is subject to regulatory requirements for product promotion and advertising, which could impact its marketing strategies [371]. Workforce and Corporate Governance - Approximately 57% of the company's workforce is female, with 58% at the leadership level [351]. - The company is highly dependent on its executive officers and key employees, with no employment or retention agreements in place, which could impede business objectives if key personnel are lost [387]. Research and Development - A Phase IV study of AGAMREE® in DMD will be funded by the investment proceeds, focusing on additional indications for the drug [268]. - The company is focused on in-licensing and developing novel medicines for rare diseases, emphasizing a patient-centric approach in its operations [388]. - The company contracted for an exclusive North America license for AGAMREE®, a novel corticosteroid for the treatment of Duchenne Muscular Dystrophy (DMD) [357]. - The incidence of DMD is estimated to be about 1 in 3,600 live male births in the United States, primarily affecting boys [341]. Intellectual Property - The USPTO issued Patent Nos. 11,845,977 and 11,873,525 for methods of treating LEMS with FIRDAPSE® under different dosing conditions [296]. - In January 2023, three generic drug manufacturers submitted ANDAs for a generic version of FIRDAPSE®, claiming that the company's six patents are invalid or unenforceable [298]. - The patent protection for FYCOMPA® includes U.S. patent no. 6,949,571, expiring on May 23, 2025, and U.S. patent no. 8,772,497, expiring on July 1, 2026 [355]. - FIRDAPSE® received five-year "new chemical entity" exclusivity and seven-year orphan drug exclusivity upon its approval [326]. - The company has in-licensed the FIRDAPSE® trademark from SERB S.A., registered in the U.S. in March 2015 [328]. - AGAMREE® has New Chemical Entity exclusivity expiring in October 2028 and Orphan Drug Exclusivity expiring in October 2030 [313]. Guidance and Speculation - The company’s guidance is speculative and based on assumptions that may not materialize, cautioning investors against undue reliance on such guidance [404].

CORAL GABLES, Fla., Feb. 27, 2024 (GLOBE NEWSWIRE) -- Catalyst Pharmaceuticals, Inc. ("Catalyst") (NASDAQ:CPRX), a commercial-stage, patient-centric biopharmaceutical company focused on in-licensing, developing, and commercializing novel high-quality medicines for patients living with rare and difficult to treat diseases, today announced an upcoming poster about the establishment of a registry to study long-term safety and quality of life (QoL) in patients with Duchenne muscular dystrophy (DMD) who are trea ...

CORAL GABLES, Fla., Feb. 27, 2024 (GLOBE NEWSWIRE) -- Catalyst Pharmaceuticals, Inc. ("Catalyst") (Nasdaq: CPRX), a commercial-stage, patient-centric biopharmaceutical company focused on in-licensing, developing, and commercializing novel high-quality medicines for patients living with rare and difficult to treat diseases, today announced an upcoming poster about the establishment of a registry to study long-term safety and quality of life (QoL) in patients with Duchenne muscular dystrophy (DMD) who are tre ...

CORAL GABLES, Fla., Feb. 21, 2024 (GLOBE NEWSWIRE) -- Catalyst Pharmaceuticals, Inc. ("Catalyst" or "Company") (NASDAQ:CPRX), a commercial-stage, patient-centric biopharmaceutical company focused on in-licensing, developing, and commercializing novel high-quality medicines for patients living with rare and difficult to treat diseases, today announced that the peer-reviewed journal Neurology has published Santhera Pharmaceutical's ("Santhera") study titled "Efficacy and Safety of Vamorolone Over 48 Weeks in ...

CORAL GABLES, Fla., Feb. 14, 2024 (GLOBE NEWSWIRE) -- Catalyst Pharmaceuticals, Inc. (“Catalyst”) (Nasdaq: CPRX), a commercial-stage, patient-centric biopharmaceutical company focused on in-licensing, developing, and commercializing novel high-quality medicines for patients living with rare diseases, today announced that it will release its fourth quarter and full-year 2023 financial results after the market close on February 28, 2024. Catalyst's management team will host a conference call and webcast the f ...

CORAL GABLES, Fla., Feb. 14, 2024 (GLOBE NEWSWIRE) -- Catalyst Pharmaceuticals, Inc. (“Catalyst”) (Nasdaq: CPRX), a commercial-stage, patient-centric biopharmaceutical company focused on in-licensing, developing, and commercializing novel high-quality medicines for patients living with rare diseases, today announced that it will release its fourth quarter and full-year 2023 financial results after the market close on February 28, 2024. Catalyst's management team will host a conference call and webcast the f ...

Catalyst Pharmaceuticals, Inc. (NASDAQ:CPRX) Q3 2023 Results Conference Call November 9, 2023 8:30 AM ET Company Participants Ali Grande - Chief Financial Officer Patrick McEnany - Chairman and Chief Executive Officer Steven Miller - Chief Operating Officer and Chief Scientific Officer Jeffrey Del Carmen - Chief Commercial Officer Conference Call Participants Les Sulewski - Truist Securities Charles Duncan - Cantor Fitzgerald Joe Catanzaro - Piper Sandler Scott Henry - ROTH Capital Partners Operator Hello, ...

[PART I. FINANCIAL INFORMATION](index=3&type=section&id=PART%20I.%20FINANCIAL%20INFORMATION) [Item 1. Financial Statements](index=3&type=section&id=Item%201.%20Financial%20Statements) The financial statements show a Q3 2023 net loss due to a significant IPR&D expense, despite strong revenue growth from recent acquisitions, and a decrease in cash from acquisition funding [Consolidated Balance Sheets](index=4&type=section&id=Consolidated%20Balance%20Sheets) This section presents the company's financial position, highlighting changes in assets, liabilities, and equity due to recent acquisitions - **Consolidated Balance Sheet Highlights (in thousands)** | Balance Sheet Item | Sep 30, 2023 (unaudited) | Dec 31, 2022 | | :--- | :--- | :--- | | Cash and cash equivalents | $120,971 | $298,395 | | Accounts receivable, net | $48,049 | $10,439 | | License and acquired intangibles, net | $167,108 | $32,471 | | Total assets | $413,211 | $375,630 | | Total liabilities | $64,663 | $75,209 | | Total stockholders' equity | $348,548 | $300,421 | - The significant decrease in cash and cash equivalents is primarily due to cash used for acquisitions, while the increase in license and acquired intangibles reflects the addition of FYCOMPA® to the company's portfolio[9](index=9&type=chunk) [Consolidated Statements of Operations](index=5&type=section&id=Consolidated%20Statements%20of%20Operations) This section details the company's revenues, expenses, and net income (loss) over specific periods, reflecting the impact of recent strategic transactions - **Statement of Operations Summary (in thousands, except per share data)** | Metric | Q3 2023 | Q3 2022 | Nine Months 2023 | Nine Months 2022 | | :--- | :--- | :--- | :--- | :--- | | Total revenues | $102,688 | $57,244 | $287,636 | $153,446 | | Research and development | $83,662 | $8,310 | $91,178 | $15,696 | | Operating income (loss) | $(37,188) | $25,102 | $45,120 | $71,037 | | Net income (loss) | $(30,764) | $22,748 | $36,566 | $57,608 | | Diluted EPS | $(0.29) | $0.20 | $0.32 | $0.52 | - The company reported a net loss in Q3 2023 despite a **79% increase in revenue**, driven by a massive surge in R&D expenses, primarily due to the immediate expensing of acquired in-process research and development (IPR&D) for vamorolone[12](index=12&type=chunk)[151](index=151&type=chunk) [Consolidated Statements of Cash Flows](index=8&type=section&id=Consolidated%20Statements%20of%20Cash%20Flows) This section summarizes the cash inflows and outflows from operating, investing, and financing activities, highlighting the impact of recent acquisitions on liquidity - **Cash Flow Summary for the Nine Months Ended Sep 30 (in thousands)** | Cash Flow Activity | 2023 | 2022 | | :--- | :--- | :--- | | Net cash provided by operating activities | $87,952 | $75,970 | | Net cash provided by (used in) investing activities | $(255,184) | $9,209 | | Net cash provided by (used in) financing activities | $(10,192) | $(559) | | Net increase (decrease) in cash | $(177,424) | $84,620 | - Investing activities show a significant cash outflow of **$255.2 million** in the first nine months of 2023, primarily for asset acquisitions including FYCOMPA® (**$162.3 million**) and the IPR&D for vamorolone (**$79.3 million**)[21](index=21&type=chunk) [Notes to Unaudited Consolidated Financial Statements](index=9&type=section&id=Notes%20to%20Unaudited%20Consolidated%20Financial%20Statements) The notes detail the company's business, accounting policies, and the financial impact of recent strategic moves, including asset acquisitions, a significant IPR&D charge, ongoing patent litigation, and subsequent FDA approval of AGAMREE® - The company's product portfolio has expanded with the acquisition of U.S. rights to FYCOMPA® in January 2023 and an exclusive North American license for vamorolone in July 2023[26](index=26&type=chunk)[27](index=27&type=chunk) - **Net Product Revenue by Product (in thousands)** | Product | Q3 2023 | Q3 2022 | Nine Months 2023 | Nine Months 2022 | | :--- | :--- | :--- | :--- | :--- | | FIRDAPSE® | $66,224 | $57,173 | $188,648 | $153,255 | | FYCOMPA® | $36,393 | $— | $98,750 | $— | | **Total** | **$102,617** | **$57,173** | **$287,398** | **$153,255** | - The acquisition of vamorolone rights from Santhera was accounted for as an asset acquisition, with the purchase price allocated to IPR&D (**$81.5 million**) and an investment in Santhera (**$13.5 million**), with the IPR&D immediately expensed to R&D as the product had not yet received regulatory approval[150](index=150&type=chunk)[151](index=151&type=chunk) - Subsequent to the quarter's end, on October 26, 2023, AGAMREE® (vamorolone) was approved by the FDA for treating Duchenne muscular dystrophy (DMD), triggering a milestone payment obligation[171](index=171&type=chunk) [Item 2. Management's Discussion and Analysis of Financial Condition and Results of Operations](index=34&type=section&id=Item%202.%20Management%27s%20Discussion%20and%20Analysis%20of%20Financial%20Condition%20and%20Results%20of%20Operations) Management attributes Q3 2023 revenue growth to FYCOMPA® and FIRDAPSE® sales, explains the net loss by a one-time IPR&D expense, and confirms sufficient liquidity despite acquisition-related cash outflows [Overview](index=34&type=section&id=Overview) This section provides a high-level summary of the company's business, strategic focus, and key developments, including product portfolio and intellectual property defense - The company is a commercial-stage biopharmaceutical firm focused on rare diseases, with three key products: FIRDAPSE® (for LEMS), FYCOMPA® (for epilepsy), and the newly licensed AGAMREE® (vamorolone) for Duchenne muscular dystrophy (DMD)[173](index=173&type=chunk)[174](index=174&type=chunk) - The company is actively defending its intellectual property for FIRDAPSE® and FYCOMPA® against multiple generic drug manufacturers that have filed Abbreviated New Drug Applications (ANDAs), with lawsuits filed triggering a 30-month stay on FDA approval for the generics[181](index=181&type=chunk)[192](index=192&type=chunk) - Following the FDA approval of AGAMREE® (vamorolone) on October 26, 2023, the company is planning a commercial launch in the U.S. during the first quarter of 2024[174](index=174&type=chunk)[199](index=199&type=chunk) [Results of Operations](index=39&type=section&id=Results%20of%20Operations) This section provides a detailed analysis of the company's financial performance, explaining the drivers behind revenue, cost of sales, and operating expenses - **Q3 2023 vs Q3 2022 Operating Results (in thousands)** | Item | Q3 2023 | Q3 2022 | Change ($) | Change (%) | | :--- | :--- | :--- | :--- | :--- | | Net Product Revenue | $102,617 | $57,173 | $45,444 | 79.5% | | Cost of Sales | $14,167 | $9,665 | $4,502 | 46.6% | | R&D Expenses | $83,662 | $8,310 | $75,352 | 906.8% | | SG&A Expenses | $33,560 | $13,649 | $19,911 | 145.9% | | Net Income (Loss) | $(30,764) | $22,748 | $(53,512) | -235.2% | - The increase in net product revenue was driven by the acquisition of FYCOMPA® (contributing **$36.4 million** in Q3 2023) and a **9% increase** in FIRDAPSE® sales volume[218](index=218&type=chunk)[219](index=219&type=chunk) - The primary driver for the increase in R&D expenses was the **$81.5 million** IPR&D charge for the vamorolone license acquisition in Q3 2023[223](index=223&type=chunk) - SG&A expenses increased significantly due to commercialization costs for FYCOMPA®, including transition service fees and the hiring of a dedicated sales force[226](index=226&type=chunk) [Liquidity and Capital Resources](index=42&type=section&id=Liquidity%20and%20Capital%20Resources) This section discusses the company's ability to meet its financial obligations, detailing cash position, working capital, and significant cash flow activities - As of September 30, 2023, the company had cash and cash equivalents of **$121.0 million** and working capital of **$134.2 million**, down from **$298.4 million** and **$263.2 million**, respectively, at year-end 2022[235](index=235&type=chunk) - Major cash outflows in 2023 included approximately **$162 million** for the FYCOMPA® acquisition, approximately **$95 million** for the vamorolone license and Santhera equity investment, and a **$10 million** payment to Jacobus for the RUZURGI® acquisition[235](index=235&type=chunk) - The company filed a **$500 million** shelf registration statement on September 8, 2023, providing flexibility for future capital raises, though no offerings have been completed under it yet[241](index=241&type=chunk) - A regulatory milestone payment of **$36 million** for AGAMREE® became due upon its FDA approval on October 26, 2023, and will be paid in the fourth quarter of 2023[250](index=250&type=chunk) [Item 3. Quantitative and Qualitative Disclosure About Market Risk](index=46&type=section&id=Item%203.%20Quantitative%20and%20Qualitative%20Disclosure%20About%20Market%20Risk) The company's primary market risk is interest rate exposure on its cash and cash equivalents, managed through investments in highly liquid instruments without derivatives - The company's market risk is confined to interest rate risk on its cash and cash equivalents[257](index=257&type=chunk)[258](index=258&type=chunk) [Item 4. Controls and Procedures](index=46&type=section&id=Item%204.%20Controls%20and%20Procedures) Management concluded that the company's disclosure controls and procedures were effective as of September 30, 2023, with no material changes to internal controls - As of September 30, 2023, the company's disclosure controls and procedures were deemed effective by management[262](index=262&type=chunk) [PART II. OTHER INFORMATION](index=47&type=section&id=PART%20II.%20OTHER%20INFORMATION) [Item 1. Legal Proceedings](index=47&type=section&id=Item%201.%20Legal%20Proceedings) The company is engaged in significant patent litigation, having filed lawsuits against generic manufacturers for FIRDAPSE® and FYCOMPA®, triggering 30-month FDA approval stays - The company has initiated patent infringement lawsuits against three generic manufacturers for FIRDAPSE® and one manufacturer for FYCOMPA® following their Paragraph IV ANDA filings[264](index=264&type=chunk)[266](index=266&type=chunk) - These legal actions have triggered a statutory **30-month stay**, preventing the FDA from approving the generic versions of FIRDAPSE® until at least May 2026, and also staying the approval for the FYCOMPA® generics, absent an earlier court decision[264](index=264&type=chunk)[266](index=266&type=chunk) [Item 1A. Risk Factors](index=48&type=section&id=Item%201A.%20Risk%20Factors) This section refers readers to the 2022 Form 10-K for detailed risk factors, noting no material changes to previously disclosed risks - The report directs investors to the Risk Factors section of the 2022 Form 10-K for a comprehensive discussion of potential risks[268](index=268&type=chunk) [Item 2. Unregistered Sales of Equity Securities and Use of Proceeds](index=48&type=section&id=Item%202.%20Unregistered%20Sales%20of%20Equity%20Securities%20and%20Use%20of%20Proceeds) The company did not repurchase common stock in Q3 2023, prioritizing cash for business development, with $21 million remaining under the repurchase program - No shares were repurchased during the three months ended September 30, 2023[269](index=269&type=chunk) - The company is prioritizing cash for business development over share repurchases at present[269](index=269&type=chunk) [Item 5. Other Information](index=49&type=section&id=Item%205.%20Other%20Information) The company announced a CEO transition, with Richard R. Daly appointed to succeed retiring Patrick J. McEnany, effective January 1, 2024 - A CEO transition is underway, with Richard R. Daly appointed to take over from the retiring Patrick J. McEnany on January 1, 2024[271](index=271&type=chunk)

Financial Data and Key Metrics Changes - Total revenues for Q2 2023 were $99.6 million, representing an 87.5% increase year-over-year [24][75] - FIRDAPSE product net revenues were $64.9 million, a 22.3% increase year-over-year [24][75] - GAAP net income for Q2 2023 was $37.8 million, or $0.36 per basic share, reflecting a 74.7% increase year-over-year [25][76] - Non-GAAP net income for Q2 2023 was $60.4 million, or $0.57 per basic share, representing a 99.2% increase year-over-year [96][97] Business Line Data and Key Metrics Changes - FIRDAPSE revenues increased by 22% year-over-year, driven by steady new patient starts and low discontinuation rates [58] - FYCOMPA net revenues for Q2 2023 were $34.6 million, contributing to confidence in achieving the full-year revenue estimate of $130 million [36][75] - The company has a pipeline of over 450 diagnosed LEMS patients not yet on FIRDAPSE, with approximately 50% of new enrollments generated from these leads [33] Market Data and Key Metrics Changes - The company anticipates a significant increase in the LEMS patient population due to educational efforts targeting thoracic oncologists [60] - A quantitative analysis of the small cell lung cancer LEMS market size is expected to provide further insights into the patient population [35] Company Strategy and Development Direction - The company aims to continue growing the FIRDAPSE and FYCOMPA brands while preparing for the launch of Vamorolone in Q1 2024 [31][52] - The strategy includes geographic expansion of FIRDAPSE and the addition of new products leveraging existing expertise in rare diseases [30][31] - The company plans to integrate Vamorolone into its neuromuscular franchise, anticipating minimal sales and marketing personnel expansion [37] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in achieving revised revenue guidance of $380 million to $390 million for 2023, driven by strong demand for FIRDAPSE [51][63] - The company is preparing for a one-time charge of $75 million related to the acquisition of Vamorolone, which will significantly increase R&D expenses in Q3 2023 [27][100] - Management highlighted the importance of addressing the unmet medical needs of DMD patients with Vamorolone, which has shown potential to reduce corticosteroid-associated side effects [62][70] Other Important Information - The company ended Q2 2023 with cash and cash equivalents of $178.8 million, which will support ongoing R&D programs and strategic initiatives [25][81] - The FDA has granted Vamorolone orphan drug and fast track designations, with a PDUFA action date set for October 26, 2023 [44][52] Q&A Session Summary Question: What are the dynamics of new enrollments from Tumor LEMS in Q2? - Management noted that new sources of leads are stemming from increased VGCC testing, with a focus on autoimmune and small cell lung cancer LEMS patients [3][5] Question: What are the expectations for the future launch of Vamorolone? - Management indicated that more details will be provided in the Q3 conference call, with a significant portion of DMD patients currently on steroids expected to consider switching to Vamorolone [6][8] Question: What are the recent trends in FYCOMPA prescriptions? - Management reported a smooth transition and stabilization in new prescriptions, with slight growth observed in recent data [12][13] Question: What is the outlook for gross margins and SG&A expenses? - Management expects margins to remain consistent and anticipates an uptick in SG&A expenses due to the expansion of the sales force for Vamorolone [14][15] Question: How does the company plan to leverage synergies between FIRDAPSE and FYCOMPA? - Management highlighted the collaboration between sales teams and the potential for cross-promotion, which is expected to enhance growth for both products [120][123]

Table of Contents UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 FORM 10-Q [Mark One] ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the Quarterly Period Ended June 30, 2023 OR ☐ TRANSITION REPORT UNDER SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 Commission File No. 001-33057 CATALYST PHARMACEUTICALS, INC. (Exact name of registrant as specified in its charter) Delaware 76-0837053 (State or other jurisdiction of incorporat ...