Catalyst Pharmaceuticals, Inc. (NASDAQ:CPRX) Q2 2020 Earnings Conference Call August 11, 2020 8:30 AM ET Company Participants Alicia Grande - Chief Financial Officer Patrick McEnany - Chairman and Chief Executive Officer Jeffrey Del Carmen - Chief Commercial Officer Steven Miller - Chief Operating Officer and Chief Scientific Officer Gary Ingenito - Chief Medical Regulatory Officer Conference Call Participants Charles Duncan - Cantor Fitzgerald Leland Gershell - Oppenheimer Operator Hello and welcome to the ...

UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 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 ☐ 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, 2020 OR ☐ TRANSITION REPORT UNDER SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 Commission File No. 001-33057 CAT ...

Catalyst Pharmaceuticals, Inc. (NASDAQ:CPRX) Q1 2020 Earnings Conference Call May 12, 2020 8:30 AM ET Company Participants Alicia Grande - Chief Financial Officer Patrick McEnany - Chairman and Chief Executive Officer Dan Brennan - Chief Commercial Officer Steven Miller - Chief Operating Officer and Chief Scientific Officer Conference Call Participants Joe Catanzaro - Piper Sandler Charles Duncan - Cantor Fitzgerald Leland Gershell - Oppenheimer Scott Henry - ROTH Capital Operator Greetings and welcome to ...

[PART I. FINANCIAL INFORMATION](index=3&type=section&id=PART%20I.%20FINANCIAL%20INFORMATION) [Financial Statements](index=4&type=section&id=Item%201.%20FINANCIAL%20STATEMENTS) The company presents its unaudited consolidated financial statements for Q1 2020, including key statements and notes [Consolidated Balance Sheets](index=4&type=section&id=Consolidated%20balance%20sheets) Total assets grew to $117.9 million while liabilities decreased, boosting stockholders' equity to $99.7 million Consolidated Balance Sheet Highlights | | March 31, 2020 (unaudited) | December 31, 2019 | | :--- | :--- | :--- | | **Total Current Assets** | $116,943,553 | $111,363,623 | | **Total Assets** | $117,879,651 | $112,376,230 | | **Total Current Liabilities** | $17,635,513 | $24,098,742 | | **Total Liabilities** | $18,203,934 | $24,746,274 | | **Total Stockholders' Equity** | $99,675,717 | $87,629,956 | [Consolidated Statements of Operations and Comprehensive Income (Loss)](index=5&type=section&id=Consolidated%20statements%20of%20operations%20and%20comprehensive%20income%20(loss)) The company achieved a net income of $10.4 million in Q1 2020, a significant turnaround from a loss in Q1 2019 Q1 Statement of Operations | | For the Three Months Ended March 31, 2020 | For the Three Months Ended March 31, 2019 | | :--- | :--- | :--- | | **Product revenue, net** | $29,136,472 | $12,448,438 | | **Total operating costs and expenses** | $18,436,725 | $13,436,207 | | **Operating income (loss)** | $10,699,747 | $(987,769) | | **Net income (loss)** | $10,426,015 | $(644,503) | | **Diluted net income (loss) per share** | $0.10 | $(0.01) | [Consolidated Statements of Cash Flows](index=7&type=section&id=Consolidated%20statements%20of%20cash%20flows) Net cash from operations turned positive at $7.2 million, increasing total cash and equivalents to $101.8 million Q1 Cash Flow Summary | | For the Three Months Ended March 31, 2020 | For the Three Months Ended March 31, 2019 | | :--- | :--- | :--- | | **Net cash provided by (used in) operating activities** | $7,213,078 | $(8,016,429) | | **Net cash provided by (used in) investing activities** | $5,000,000 | $10,449,393 | | **Net cash provided by (used in) financing activities** | $26,149 | $89,350 | | **Net increase (decrease) in cash and cash equivalents** | $12,239,227 | $2,522,314 | | **Cash and cash equivalents—end of period** | $101,750,937 | $19,081,714 | [Notes to Unaudited Consolidated Financial Statements](index=8&type=section&id=Notes%20to%20unaudited%20consolidated%20financial%20statements) Notes detail accounting policies, revenue recognition for Firdapse®, legal matters, and collaborative agreements - The company is a biopharmaceutical firm focused on rare neuromuscular and neurological diseases, with its first product, **Firdapse®**, launched in January 2019 for Lambert-Eaton Myasthenic Syndrome (LEMS)[19](index=19&type=chunk)[20](index=20&type=chunk) - Revenue is recognized when the customer obtains control of Firdapse®, with **net revenue recorded after deducting variable considerations** like rebates and discounts[36](index=36&type=chunk)[37](index=37&type=chunk)[43](index=43&type=chunk) - The company **filed a lawsuit against the FDA** in June 2019, challenging the approval of a competitor drug, Ruzurgi®, for violating its Orphan Drug Exclusivity[89](index=89&type=chunk) - The company has a license agreement for Firdapse®, paying **tiered royalties of 7-10% on net sales** in North America and Japan[91](index=91&type=chunk)[92](index=92&type=chunk) [Management's Discussion and Analysis of Financial Condition and Results of Operations (MD&A)](index=26&type=section&id=Item%202.%20MANAGEMENT'S%20DISCUSSION%20AND%20ANALYSIS%20OF%20FINANCIAL%20CONDITION%20AND%20RESULTS%20OF%20OPERATIONS) Management discusses Q1 2020 performance, Firdapse® commercialization, trial progress, and pandemic impacts [Overview](index=26&type=section&id=Overview) The company details Firdapse® commercialization, COVID-19 impacts on trials, and competitive legal challenges - The **COVID-19 pandemic has delayed** the diagnosis of new LEMS patients and pushed back top-line results for the MuSK-MG and SMA Type 3 trials[111](index=111&type=chunk)[114](index=114&type=chunk) - The company faces **competition from Jacobus Pharmaceuticals' Ruzurgi®**, which is believed to be prescribed off-label to adults at a lower price[88](index=88&type=chunk)[123](index=123&type=chunk) - A **lawsuit was filed against the FDA** in June 2019 challenging the approval of Ruzurgi®, with a decision expected later in the year[124](index=124&type=chunk)[125](index=125&type=chunk) - The company is **expanding its commercial territory for Firdapse®** to Japan and has submitted an NDS in Canada with priority review status[134](index=134&type=chunk)[135](index=135&type=chunk) [Results of Operations](index=32&type=section&id=Results%20of%20Operations) Firdapse® revenue more than doubled to $29.1 million, driving net income despite increased operating expenses Q1 2020 vs Q1 2019 Performance | Metric | Q1 2020 | Q1 2019 | Change (%) | | :--- | :--- | :--- | :--- | | **Net Revenue (Firdapse®)** | $29,136,472 | $12,448,438 | +134.1% | | **Cost of Sales** | $4,200,000 | $1,700,000 | +147.1% | | **R&D Expenses** | $4,222,811 | $3,307,959 | +27.7% | | **SG&A Expenses** | $10,063,048 | $8,416,460 | +19.6% | | **Net Income (Loss)** | $10,426,015 | $(644,503) | N/A | - **Non-GAAP net income for Q1 2020 was $11.9 million** ($0.11 per diluted share), excluding $1.5 million in non-cash stock-based compensation[169](index=169&type=chunk) [Liquidity and Capital Resources](index=34&type=section&id=Liquidity%20and%20Capital%20Resources) The company maintains a strong liquidity position with $101.8 million in cash and investments, sufficient for operations - The company held cash, cash equivalents, and investments of approximately **$101.8 million at the end of Q1 2020**[170](index=170&type=chunk) - **Net cash provided by operating activities was $7.2 million** in Q1 2020, a significant improvement from the $8.0 million used in Q1 2019[179](index=179&type=chunk) - The company has contractual obligations including **royalty payments on Firdapse® sales** and purchase commitments of about $950,000 per year[185](index=185&type=chunk) - **$92.5 million remains available for future sales** under the company's 2017 Shelf Registration Statement[178](index=178&type=chunk) [Quantitative and Qualitative Disclosure About Market Risk](index=38&type=section&id=Item%203.%20QUANTITATIVE%20AND%20QUALITATIVE%20DISCLOSURE%20ABOUT%20MARKET%20RISK) Market risk exposure is primarily limited to interest rate fluctuations on cash and short-term investments - The company's market risk is confined to **interest rate fluctuations** affecting its cash and investments, held in highly liquid funds and U.S. Treasuries[193](index=193&type=chunk) [Controls and Procedures](index=39&type=section&id=Item%204.%20CONTROLS%20AND%20PROCEDURES) Management concluded that disclosure controls and procedures were effective with no material changes in the quarter - The principal executive and financial officers concluded that as of March 31, 2020, the company's **disclosure controls and procedures were effective**[196](index=196&type=chunk) - **No changes in internal controls** that could materially affect financial reporting occurred during the three months ended March 31, 2020[196](index=196&type=chunk) [PART II. OTHER INFORMATION](index=40&type=section&id=PART%20II.%20OTHER%20INFORMATION) [Legal Proceedings](index=40&type=section&id=Item%201.%20LEGAL%20PROCEEDINGS) The company details its ongoing lawsuit against the FDA regarding the approval of a competitor drug, Ruzurgi® - In June 2019, the company **sued the FDA, challenging the approval of Ruzurgi®** on the grounds that it violated the company's statutory rights[198](index=198&type=chunk) - The lawsuit seeks an order to **vacate the FDA's approval of Ruzurgi®**, with a decision expected later in 2020[198](index=198&type=chunk)[199](index=199&type=chunk) [Risk Factors](index=40&type=section&id=Item%201A.%20RISK%20FACTORS) Investors are referred to the 2019 Annual Report for a comprehensive discussion of business risk factors - The company refers readers to its **2019 Annual Report on Form 10-K** for a comprehensive description of significant risk factors affecting the business[201](index=201&type=chunk) [Exhibits](index=41&type=section&id=Item%206.%20EXHIBITS) This section lists all exhibits filed with the Form 10-Q, including Sarbanes-Oxley certifications - Filed exhibits include **Sarbanes-Oxley Act certifications** (Sections 302 and 906) and XBRL financial data files[203](index=203&type=chunk)

Financial Data and Key Metrics Changes - In 2019, Catalyst Pharmaceuticals achieved GAAP net income of $32 million, or $0.31 per basic share, compared to a GAAP net loss of $34 million in 2018 [58] - For Q4 2019, GAAP net income was $7.9 million, or $0.08 per basic share, compared to a GAAP net loss of $14.5 million in Q4 2018 [60] - Full-year 2019 net revenues from Firdapse were $102 million, with Q4 2019 net revenues of $30 million [9][60] Business Line Data and Key Metrics Changes - Firdapse launch exceeded expectations, with 532 patients prescribed by the end of Q4 2019, up from 490 at the end of Q3 2019 [25] - The average co-pay for patients decreased to $1.08 per month, with 94% of patients having zero out-of-pocket costs [27] - The discontinuation rate for newly enrolled patients decreased to under 25% in Q4, down from over 35% in the first three quarters [28] Market Data and Key Metrics Changes - Approximately 35% of diagnosed adult LEMS patients have received a Firdapse prescription since its launch [24] - The company is expanding its global footprint, with a New Drug Submission for Firdapse in Canada and ongoing discussions for approval in Japan [18][51] Company Strategy and Development Direction - Catalyst aims to establish Firdapse as the standard of care for adult LEMS patients and expand its label to include other indications [69] - The company is focusing on business development in rare diseases and has hired a new Vice President of Business Development [17] - Plans for additional proof-of-concept studies for neuromuscular conditions are underway, including Kennedy's disease and hereditary neuropathy [53] Management's Comments on Operating Environment and Future Outlook - Management expressed cautious optimism regarding the impact of COVID-19 on business operations, emphasizing the robustness of the supply chain [10][55] - The company anticipates continued growth in patient enrollments and revenue, with guidance for 2020 net product revenues expected to be between $135 million and $155 million [9][75] Other Important Information - Catalyst has a strong balance sheet with approximately $95 million in cash and no funded debt [20] - The company is pursuing a lawsuit against the FDA regarding the approval of a competing product, Ruzurgi, with expected decisions in Q2 or Q3 2020 [21][22] Q&A Session Summary Question: Current tone of business and guidance for 2020 - Management indicated that the guidance of $135 million to $155 million does not include global expansion and is driven by new patient starts and maintaining current patients [72][75] Question: Impact of COVID-19 on patient follow-up and prescriptions - All prescriptions are delivered directly to patients by mail, and the company is ensuring that patients are informed about product availability [87][88] Question: Discontinuation rates and reasons for patient drop-off - Discontinuations were primarily due to patients switching to a competitor's product, but trends are improving with new patient enrollments increasing [92][94] Question: MuSK-MG trial optimism - Management remains cautiously optimistic about the MuSK-MG trial due to the homogeneous patient population and previous positive outcomes [82][84] Question: Market dynamics regarding competitor share - The share of the competitor's product has leveled off, and efforts are focused on supporting existing patients and generating awareness among new patients [103][104]

Table of Contents 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, 2019 OR ☐ TRANSITION REPORT PURSUANT TO 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 of jurisdiction of incorporation ...

| --- | --- | --- | --- | --- | --- | --- | --- | |-------|--------------|-------|-------|-----------------------------------------------------------------------|-------|-------|-----------------| | | | | | | | | | | | | | | | | | | | | | | | | | | | | | | | | Catalyst Corporate Presentation San Francisco, CA January 13-16, 2020 | | | Pharmaceuticals | | | | | | | | | | | | Confidential | | | | | | | Safe Harbor 2 Confidential This presentation contains forward-looking statements that are subject to a numbe ...

Catalyst Pharmaceuticals, Inc. (NASDAQ:CPRX) Q3 2019 Results Earnings Conference Call November 13, 2019 8:30 AM ET Company Participants Alicia Grande - Chief Financial Officer Patrick McEnany - Chairman and Chief Executive Officer Steve Miller - Chief Operating Officer and Chief Scientific Officer Dan Brennan - Chief Commercial Officer Conference Call Participants Charles Duncan - Cantor Fitzgerald Joe Catanzaro - Piper Jaffray Leland Gershell - Oppenheimer Scott Henry - ROTH Capital Operator Greetings. Wel ...

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 September 30, 2019 OR ☐ TRANSITION REPORT UNDER SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 Commission File No. 001-33057 | --- | --- | |-------------------------------------------------------------------------------------------------------|------------------------ ...

Catalyst Pharmaceuticals, Inc. (NASDAQ:CPRX) Q2 2019 Earnings Conference Call August 8, 2019 8:30 AM ET Company Participants Alicia Grande - Chief Financial Officer Pat McEnany - Chairman and Chief Executive Officer Dan Brennan - Chief Commercial Officer Steve Miller - Chief Operating Officer and Chief Scientific Officer Gary Ingenito - Chief Medical Officer and Head of Regulatory Affairs Conference Call Participants Charles Duncan - Cantor Fitzgerald Joe Catanzaro - Piper Jaffray Leland Gershell - Oppenhei ...