Company Focus and Strategy - The company is focused on developing and commercializing novel medicines for rare diseases, with a commitment to a robust pipeline of best-in-class medicines [99]. - In 2021, the company made a strategic decision to diversify its product portfolio through acquisitions in rare disease therapeutic categories outside of neuromuscular diseases [100]. - The company is exploring several potential opportunities to acquire or in-license drug products in development, although no definitive agreements have been made to date [100]. - The company is exploring growth opportunities beyond FIRDAPSE® through acquisitions and in-licensing of product opportunities [171]. Product Information and Development - FIRDAPSE® was approved by the FDA on November 28, 2018, and launched in January 2019, with a dedicated sales force of approximately 30 personnel [107]. - The company is actively pursuing regulatory approval for FIRDAPSE® for pediatric LEMS patients, although success is not guaranteed [115]. - A Phase 3 registrational study for FIRDAPSE® in Japan was initiated by DyDo Pharma, with completion anticipated in 2023 [122]. - The company has expanded its commercial territory for FIRDAPSE® to include Japan and has the option to expand further into Asia and Latin America upon achieving certain milestones [119]. - The company is assessing the potential for its version of vigabatrin tablets to be approved by the FDA and the implications for milestone payments or royalties [171]. Financial Performance - For the three months ended March 31, 2022, the company recognized $43.0 million in net revenue from product sales of FIRDAPSE®, an increase of approximately $12.8 million or 42.4% compared to $30.2 million in the same period of 2021 [145]. - Net income for the three months ended March 31, 2022, was $13.2 million, an increase of 71.4% compared to $7.7 million for the same period in 2021 [156]. - Cash and cash equivalents and investments totaled $197.8 million as of March 31, 2022, up from $191.3 million at December 31, 2021 [157]. - Net cash provided by operating activities was $8.5 million for the three months ended March 31, 2022, compared to $3.8 million for the same period in 2021 [164]. - The company had cash and investments of approximately $198 million as of March 31, 2022, which is expected to support operations for at least the next 12 months [129]. - The company anticipates sufficient resources to support operations for at least the next 12 months [158]. Expenses and Costs - The cost of sales for the three months ended March 31, 2022, was approximately $5.9 million, compared to $4.7 million for the same period in 2021, reflecting an increase of 25.5% [146]. - Research and development expenses for the three months ended March 31, 2022, were approximately $3.4 million, representing 13% of total operating costs, compared to $3.0 million or 15% in the same period of 2021 [148]. - Selling, general and administrative expenses for the three months ended March 31, 2022, were approximately $16.4 million, an increase of 29.2% from $12.7 million in the same period of 2021 [150]. - The increase in selling, general and administrative expenses was primarily due to contributions to organizations supporting LEMS patients of approximately $2.1 million and increases in sales commissions and employee compensation [150]. - Total stock-based compensation for the three months ended March 31, 2022, was $1.9 million, compared to $1.6 million for the same period in 2021 [152]. Legal and Regulatory Matters - The company has filed lawsuits against Jacobus and PantherRx for patent infringement related to FIRDAPSE®, which are currently in the discovery stage [126]. - The company is currently involved in lawsuits against Jacobus Pharmaceutical Company for patent infringement, with the outcome uncertain [171]. - The company is pursuing additional patent applications for FIRDAPSE® to further protect its intellectual property, although there is no assurance that additional patents will be issued [125]. Market and Economic Considerations - The company is monitoring potential changes in reimbursement and coverage policies from government and private payors, which could impact FIRDAPSE® sales [171]. - The company is assessing the impact of the Canadian Court's decision to overturn the approval of Ruzurgi® and its potential effects on FIRDAPSE® sales in the U.S. [171]. - The company is focused on the successful commercialization of FIRDAPSE® in Canada and is awaiting the outcome of appeals regarding Ruzurgi® [171]. - The company is closely monitoring the state of the economy and its potential impact on business operations [171]. Investment and Liquidity - The company emphasizes liquidity and preservation of principal in its investment activities, focusing on cash and short-term investments [174]. - The company does not use derivative financial instruments in its investment portfolio, prioritizing capital preservation [174]. - The company may require additional working capital depending on future success with FIRDAPSE® sales [159]. - The effective income tax rate increased to 24.2% for the three months ended March 31, 2022, from 22.5% in the same period of 2021 [154].

Catalyst Pharmaceuticals, Inc. (NASDAQ:CPRX) Q4 2021 Earnings Conference Call March 17, 2022 8:30 AM ET Company Participants Patrick McEnany - Co-Founder, Chairman, President and CEO Alicia Grande - VP, CFO and Treasurer Steven Miller - COO and Chief Scientific Officer Jeff Del Carmen - Chief Commercial Officer Gary Ingenito - Chief Medical and Regulatory Officer Conference Call Participants Joseph Catanzaro - Piper Sandler Charles Duncan - Cantor Fitzgerald Scott Henry - ROTH Capital Partners Joon Lee - Tr ...

Table of Contents Title of Each Class Ticker SymbolName of Exchange on Which Registered Common Stock, par value $0.001 per share CPRX NASDAQ Capital Market Emerging Growth Company ☐ UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 FORM 10-K [Mark One] ☒ ANNUAL REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the Fiscal Year Ended December 31, 2021 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 Commission F ...

Financial Data and Key Metrics Changes - Catalyst Pharmaceuticals reported record Firdapse revenue of $35.9 million for Q3 2021, a 23% increase compared to Q3 2020 [6] - For the nine-month period, Firdapse revenues reached $99.7 million, up 13% year-over-year from $87.9 million [7] - Net income before income taxes for Q3 2021 was $14.1 million, a 20% increase from $11.7 million in Q3 2020 [7] - GAAP net income for Q3 2021 was $10.3 million, or $0.10 per share, compared to $43.3 million in Q3 2020, which included a one-time non-cash deferred tax asset [34] Business Line Data and Key Metrics Changes - Firdapse sales were driven by steady new patient enrollments and favorable reimbursement dynamics, with new patient enrollments up 26% year-over-year [21] - The company has reached approximately 800 adult LEMS patients since the launch of Firdapse, with a focus on expanding its reach to the remaining undiagnosed patients [22] Market Data and Key Metrics Changes - The company has only reached about 25% of the estimated 3,000 U.S. LEMS patients, indicating significant growth potential [10] - Approximately 50% of LEMS patients are thought to be tumor LEMS patients, primarily linked to small cell lung cancer, highlighting a key growth area for the company [23] Company Strategy and Development Direction - Catalyst is focused on expanding the label for Firdapse to include pediatric LEMS patients and is preparing a supplementary MDA submission for this purpose [27] - The company is also working on a long-acting formulation of Firdapse to improve patient experience and therapeutic consistency [26] - Catalyst has decided to end the MuskMG program to concentrate R&D efforts on more promising areas, indicating a strategic shift towards maximizing resource utilization [15] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in continued organic growth through the end of the year and beyond, despite challenges posed by the Delta variant of COVID-19 [7][19] - The company is well-prepared to ensure uninterrupted access to Firdapse for LEMS patients and is committed to addressing patient needs [12] Other Important Information - Catalyst ended the quarter with $174.8 million in cash and short-term investments, providing financial flexibility for future growth opportunities [8] - The company has made substantial investments in Firdapse and is focused on expanding its intellectual property portfolio, with two patents extending exclusivity to 2034 [13][31] Q&A Session Summary Question: What are the key growth drivers for Firdapse in 2022? - Management indicated that they may provide guidance in early Q1 2022 and highlighted the importance of increasing awareness and the breadth of prescribers as key growth factors [40][41] Question: Can you provide details on the pediatric filing for LEMS? - Management confirmed that pediatric LEMS is an autoimmune condition similar to adult LEMS, with an estimated 10 to 15 known cases, and emphasized the importance of ensuring treatment access for all patients [46][47] Question: What would be the impact if a competitor's product is pulled from the market? - Management expressed concern for a smooth transition for patients and indicated that they expect a significant influx of patients over a 30 to 60-day period if that event occurs [55][57] Question: What challenges have you faced in business development diligence? - Management noted that valuation was a significant challenge in their recent assessments, leading them to walk away from a potential acquisition due to discomfort with the valuation [62]

Table of Contents Title of Each ClassTicker SymbolName of Exchange on Which Registered Common Stock, par value $0.001 per share CPRX NASDAQ Capital Market Emerging growth company ☐ 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 September 30, 2021 OR ☐ TRANSITION REPORT UNDER SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 Commission ...

| --- | --- | --- | --- | --- | |--------------------------------|-------|---------------------------------------------------------------|-------|---------------------------------------------------------| | | | | | | | | | | | | | | | CANTON | | Catalyst Pharmaceuticals VIRTUAL GLOBAL HEALTHCARE 2021 | | | | | | | | Alle SMILE SMAN AND AMERICAN 1 | | SMILLER COMME SMILLE ssiller Allin SMILLe SMILLE Ship Million | | | Safe Harbor This presentation contains forward‐looking statements that are subject to a num ...

Catalyst Pharmaceuticals, Inc. (NASDAQ:CPRX) Q2 2021 Earnings Conference Call August 10, 2021 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 Gary Ingenito - Chief Medical and Regulatory Officer Conference Call Participants Charles Duncan - Cantor Fitzgerald Operator Greetings and welcome to the Catalyst Pharmaceuticals ...

Table of Contents Title of Each ClassTicker SymbolName of Exchange on Which Registered Common Stock, par value $0.001 per share CPRX NASDAQ Capital Market Emerging growth company ☐ 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, 2021 OR ☐ TRANSITION REPORT UNDER SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 19 ...

Catalyst Pharmaceuticals, Inc. (NASDAQ:CPRX) Q1 2021 Earnings Conference Call May 11, 2021 8:30 AM ET Company Participants Alicia Grande – Vice President, Chief Financial Officer and Treasurer Patrick McEnany – Chairman and Chief Executive Officer Jeffrey Del Carmen – Chief Commercial Officer Steven Miller – Chief Operating Officer and Chief Scientific Officer Brian Elsbernd – Chief Legal and Compliance Officer Conference Call Participants Charles Duncan – Cantor Fitzgerald Leszek Sulewski – Truist Operator ...

Table of Contents Title of Each ClassTicker SymbolName of Exchange on Which Registered Common Stock, par value $0.001 per share CPRX NASDAQ Capital Market 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 March 31, 2021 OR ☐ TRANSITION REPORT UNDER SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 Commission File No. 00 ...