Catalyst Pharmaceuticals Inc. (NASDAQ:CPRX) Q1 2023 Earnings Conference Call May 11, 2023 8:30 AM ET Company Participants Patrick McEnany - Chairman, Chief Executive Officer Alicia Grande - Vice President, Chief Financial Officer Jeff Del Carmen - Chief Commercial Officer Steven Miller - Chief Operating Officer, Chief Scientific Officer Conference Call Participants Les Sulewski - Truist Securities Joe Catanzaro - Piper Sandler Pete Stavropoulos - Cantor Fitzgerald Operator Greetings and welcome to the Catal ...

[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) This section presents the unaudited consolidated financial statements for Catalyst Pharmaceuticals, Inc. as of March 31, 2023, and for the three months ended March 31, 2023, and 2022 [Consolidated Balance Sheets](index=4&type=section&id=Consolidated%20Balance%20Sheets) Total assets increased to **$407.2 million**, primarily due to the FYCOMPA® acquisition, with stockholders' equity rising to **$333.7 million** Consolidated Balance Sheet Highlights (in thousands) | Account | March 31, 2023 (unaudited) | December 31, 2022 | | :--- | :--- | :--- | | **Assets** | | | | Cash and cash equivalents | $148,247 | $298,395 | | Total current assets | $198,911 | $320,806 | | License and acquired intangibles, net | $184,083 | $32,471 | | **Total assets** | **$407,227** | **$375,630** | | **Liabilities & Equity** | | | | Total current liabilities | $56,709 | $57,588 | | **Total liabilities** | **$73,566** | **$75,209** | | **Total stockholders' equity** | **$333,661** | **$300,421** | [Consolidated Statements of Operations and Comprehensive Income](index=5&type=section&id=Consolidated%20Statements%20of%20Operations%20and%20Comprehensive%20Income) Revenues nearly doubled to **$85.4 million**, with net income more than doubling to **$29.6 million**, primarily due to the FYCOMPA® acquisition Q1 2023 vs Q1 2022 Statement of Operations (in thousands, except per share data) | Metric | Q1 2023 (unaudited) | Q1 2022 (unaudited) | | :--- | :--- | :--- | | Product revenue, net | $85,304 | $43,033 | | Total revenues | $85,366 | $43,089 | | Total operating costs and expenses | $49,757 | $25,723 | | Operating income | $35,609 | $17,366 | | **Net income** | **$29,568** | **$13,241** | | **Diluted EPS** | **$0.26** | **$0.12** | [Consolidated Statements of Cash Flows](index=7&type=section&id=Consolidated%20Statements%20of%20Cash%20Flows) Operating cash flow was **$12.1 million**, with **$162.4 million** used for the FYCOMPA® acquisition, reducing cash to **$148.2 million** Cash Flow Summary for Three Months Ended March 31 (in thousands) | Cash Flow Activity | 2023 | 2022 | | :--- | :--- | :--- | | Net cash provided by operating activities | $12,070 | $8,478 | | Net cash used in investing activities | ($162,367) | $0 | | Net cash provided by (used in) financing activities | $149 | ($1,551) | | **Net decrease in cash** | **($150,148)** | **$6,927** | | **Cash at end of period** | **$148,247** | **$178,372** | [Notes to Unaudited Consolidated Financial Statements](index=8&type=section&id=Notes%20to%20Unaudited%20Consolidated%20Financial%20Statements) Notes detail the FYCOMPA® acquisition, revenue recognition for FIRDAPSE® and FYCOMPA®, and contractual obligations - The company is a commercial-stage biopharmaceutical firm focused on rare neurological and epileptic diseases, with main products FIRDAPSE® and FYCOMPA®[55](index=55&type=chunk) - On January 24, 2023, the company acquired the U.S. rights to FYCOMPA® from Eisai Co., Ltd. and is now marketing it in the United States[29](index=29&type=chunk) - The company sells FIRDAPSE® through an exclusive U.S. distributor and sells FYCOMPA® directly to major wholesalers and distributors[64](index=64&type=chunk) Net Product Revenue by Product (in thousands) | Product | For the Three Months Ended March 31, 2023 | For the Three Months Ended March 31, 2022 | | :--- | :--- | :--- | | FIRDAPSE® | $57,526 | $43,033 | | FYCOMPA® | $27,778 | — | | **Total product revenue, net** | **$85,304** | **$43,033** | [Item 2. Management's Discussion and Analysis of Financial Condition and Results of Operations (MD&A)](index=31&type=section&id=Item%202.%20MANAGEMENT%27S%20DISCUSSION%20AND%20ANALYSIS%20OF%20FINANCIAL%20CONDITION%20AND%20RESULTS%20OF%20OPERATIONS) Management discusses Q1 2023 financial performance, highlighting revenue and net income growth from the FYCOMPA® acquisition [Overview](index=31&type=section&id=Overview) Catalyst, a biopharmaceutical company, expanded its portfolio with the FYCOMPA® acquisition for rare neurological diseases - The company's flagship U.S. commercial product is FIRDAPSE® for LEMS, and it recently acquired the U.S. rights to FYCOMPA® for epilepsy from Eisai, closing the deal on **January 24, 2023**[153](index=153&type=chunk) - The company is pursuing a strategy to broaden and diversify its drug portfolio through acquisitions of early or late-stage products, companies, or technology platforms in rare disease and CNS therapeutic categories[203](index=203&type=chunk) [Results of Operations](index=36&type=section&id=Results%20of%20Operations) Total revenues increased **98%** to **$85.4 million**, driven by FIRDAPSE® and FYCOMPA® sales, leading to a **124%** net income increase Q1 2023 vs Q1 2022 Revenue Breakdown (in millions) | Product | Q1 2023 | Q1 2022 | | :--- | :--- | :--- | | FIRDAPSE® Sales | $57.5 | $43.0 | | FYCOMPA® Sales | $27.8 | $0.0 | | **Total Product Revenue** | **$85.3** | **$43.0** | - The increase in net revenues was due to the acquisition of FYCOMPA® and a **21%** increase in FIRDAPSE® sales volumes, which included patients transferred from RUZURGI® in 2022[238](index=238&type=chunk) - Amortization of intangible assets was **$6.5 million** in Q1 2023, a new expense related to the FYCOMPA® and RUZURGI® rights acquisitions, compared to **$0** in Q1 2022[192](index=192&type=chunk) - Selling, general and administrative (SG&A) expenses increased by **$13.3 million**, primarily due to **$8.5 million** in transition service fees for FYCOMPA® and a **$1.5 million** increase in employee compensation[219](index=219&type=chunk) Net Income Comparison (in millions, except per share data) | Metric | Q1 2023 | Q1 2022 | | :--- | :--- | :--- | | Net Income | $29.6 | $13.2 | | Diluted EPS | $0.26 | $0.12 | [Liquidity and Capital Resources](index=37&type=section&id=Liquidity%20and%20Capital%20Resources) Cash decreased to **$148.2 million** due to the **$162 million** FYCOMPA® acquisition, but remains sufficient for future operations - Cash and cash equivalents decreased to **$148.2 million** at March 31, 2023, from **$298.4 million** at December 31, 2022, mainly because the company used approximately **$162 million** to acquire the U.S. rights to FYCOMPA®[246](index=246&type=chunk) - Net cash from operating activities was **$12.1 million** for Q1 2023, with an increase in accounts receivable from FYCOMPA® sales partially offsetting cash generated from net income[250](index=250&type=chunk) - Contractual obligations include royalties on FIRDAPSE® sales, deferred payments and future royalties for the RUZURGI® settlement, and potential milestone and royalty payments for the FYCOMPA® acquisition[252](index=252&type=chunk)[268](index=268&type=chunk) [Item 3. Quantitative and Qualitative Disclosure About Market Risk](index=42&type=section&id=Item%203.%20QUANTITATIVE%20AND%20QUALITATIVE%20DISCLOSURE%20ABOUT%20MARKET%20RISK) Market risk is limited to interest rate fluctuations on cash and equivalents, with a focus on capital preservation - The company's primary market risk is interest rate fluctuations affecting its cash and cash equivalents invested in money market funds and U.S. Treasuries[273](index=273&type=chunk) [Item 4. Controls and Procedures](index=42&type=section&id=Item%204.%20CONTROLS%20AND%20PROCEDURES) Disclosure controls and procedures were effective as of March 31, 2023, with no material changes to internal controls - Management concluded that as of **March 31, 2023**, the company's disclosure controls and procedures were effective[274](index=274&type=chunk) - No material changes were made to the company's internal control over financial reporting during the first quarter of 2023[274](index=274&type=chunk) [PART II. OTHER INFORMATION](index=42&type=section&id=PART%20II.%20OTHER%20INFORMATION) [Item 1. Legal Proceedings](index=42&type=section&id=Item%201.%20LEGAL%20PROCEEDINGS) The company initiated patent lawsuits against generic manufacturers for FIRDAPSE® and FYCOMPA®, triggering 30-month FDA approval stays - In January 2023, the company received Paragraph IV notice letters from three generic drug manufacturers seeking to market a generic version of FIRDAPSE®[118](index=118&type=chunk)[174](index=174&type=chunk) - The company filed patent infringement lawsuits against the three generic manufacturers on **March 1, 2023**, triggering an automatic **30-month** stay of FDA approval for the generic FIRDAPSE® products until **May 2026** or a court decision[118](index=118&type=chunk)[261](index=261&type=chunk) - In February 2023, the company received Paragraph IV notice letters for generic versions of FYCOMPA® tablets and oral suspension, and subsequently filed lawsuits on **April 5, 2023**, triggering a **30-month** stay for those applications as well[202](index=202&type=chunk)[262](index=262&type=chunk)[297](index=297&type=chunk) [Item 1A. Risk Factors](index=43&type=section&id=Item%201A.%20RISK%20FACTORS) No material changes to risk factors were reported, referring readers to the 2022 Annual Report on Form 10-K for details - The company refers to its **2022 Annual Report on Form 10-K** for a description of significant risk factors affecting the business[280](index=280&type=chunk) [Item 2. Unregistered Sales of Equity Securities and Use of Proceeds](index=43&type=section&id=Item%202.%20UNREGISTERED%20SALES%20OF%20EQUITY%20SECURITIES%20AND%20USE%20OF%20PROCEEDS) No shares were repurchased under the **$40 million** program in Q1 2023, as cash is being retained for business development Issuer Purchases of Equity Securities (Q1 2023) | Period | Total Number of Shares Purchased | Average Price Paid Per Share | Dollar Value of Shares that May Yet Be Purchased (in thousands) | | :--- | :--- | :--- | :--- | | Jan 2023 | — | $ — | $21,003 | | Feb 2023 | — | $ — | $21,003 | | Mar 2023 | — | $ — | $21,003 | - The company is not currently purchasing shares under its repurchase program, instead opting to retain cash for business development[281](index=281&type=chunk)

Financial Data and Key Metrics Changes - The company reported total revenues of $214 million for 2022, a 52% year-over-year increase from $141 million in 2021 [23][111] - Fourth quarter total net revenues were $61 million, a 59% increase compared to $38 million in Q4 2021 [23][111] - GAAP net income for 2022 was $83 million, or $0.80 per basic share, representing a 110% increase compared to $39 million in 2021 [23][111] - Non-GAAP net income for 2022 was $114 million, or $1.10 per basic share, a 93% increase from $59 million in 2021 [23][111] - The effective tax rate for 2022 was 21%, down from 25% in 2021, with expectations of a normalized rate of 23% to 25% in future periods [3][35] Business Line Data and Key Metrics Changes - FIRDAPSE net sales for 2022 were $214 million, a 52% increase year-over-year, with Q4 sales of $61 million showing a 6% growth quarter-over-quarter [50][23] - The company expects FIRDAPSE's net revenues for 2023 to be between $245 million and $255 million [13][27] - FYCOMPA is forecasted to generate approximately $130 million in net revenues for 2023, following its acquisition [13][51] Market Data and Key Metrics Changes - The company identified a total LEMS patient population of about 3,000, with current penetration estimated at around 25% [66] - The company is actively working to increase the share of voice among thoracic oncologists to expand the use of FIRDAPSE [16] Company Strategy and Development Direction - The company aims to grow and diversify revenues while strengthening its position in the neuroscience space [38] - The acquisition of FYCOMPA is seen as a strategic move to expand into rare epileptic diseases and enhance the product portfolio [19][51] - The company plans to file a supplemental NDA to increase the maximum indicated daily dosage of FIRDAPSE from 80 mg to 100 mg [112] Management's Comments on Operating Environment and Future Outlook - Management expressed optimism about the long-term growth prospects of FIRDAPSE, supported by advancements in its intellectual property estate [14] - The company anticipates continued organic growth driven by strategic initiatives and a strong commercial strategy [27][52] - Management highlighted the importance of maintaining operational excellence to drive new patient starts and favorable prescription approval rates [27] Other Important Information - The company ended 2022 with cash and equivalents of $298 million, providing financial flexibility for strategic initiatives [60][111] - The company has been recognized for its achievements, including being named one of Forbes 2023 America's Best Small Companies [49] Q&A Session Summary Question: What is the penetration of the total LEMS opportunity? - Management estimates the penetration is around 25%, indicating significant opportunity for growth [66] Question: What percent of patients are currently on the 100 mg dose? - Management has not disclosed specific numbers but indicated that some patients are on the 100 mg dose, with about 40% of patients currently on 80 mg [66] Question: What is the strategy for reinvigorating the FYCOMPA brand? - The focus will be on fully integrating the epilepsy franchise and targeting physicians currently prescribing FYCOMPA [68] Question: How many antibody tests were provided and utilized by physicians in Q4? - There has been a significant increase in the number of tests conducted, with consistent high levels compared to previous years [74] Question: What data will be presented to regulators for the 100 mg dose? - The company plans to use safety data from patients already on the 100 mg dose and data from the previous RUZURGI approval to support the supplemental NDA [75]

Financial Performance - For the year ended December 31, 2022, the company recognized $213.9 million in net revenue from product sales of FIRDAPSE®, a 55% increase from $138.0 million in 2021, driven by a 49% increase in sales volume [46]. - Selling, general and administrative expenses increased to approximately $58.2 million in 2022, up from $49.6 million in 2021, accounting for 52% of total operating costs [31]. - Other income for 2022 was $2.9 million, up from $0.3 million in 2021, attributed to higher yields on investments and increased invested balances [50]. - Total selling, general, and administrative expenses for 2022 were $58.2 million, a 17.2% increase from $49.6 million in 2021 [58]. - Employee stock-based compensation increased to $7.9 million in 2022 from $6.1 million in 2021, reflecting a 29.5% rise [59]. - As of December 31, 2022, the company had cash and investments of approximately $298 million, which decreased to $136 million after the FYCOMPA® acquisition [92]. Research and Development - The company incurred total research and development expenses of $19.8 million for the year ended December 31, 2022, representing a 16.8% increase from $16.9 million in 2021 [30]. - Research and development expenses for 2022 were approximately $19.8 million, representing about 18% of total operating costs, an increase of $2.9 million from 2021 [48]. - The increase in research and development expenses was primarily due to a one-time acquisition of RUZURGI® inventory costing approximately $4.1 million [48]. - The company expects substantial research and development expenses to continue in 2023 as it seeks to acquire innovative technology platforms in rare disease categories [48]. Acquisitions and Partnerships - The acquisition of FYCOMPA® included an upfront payment of $160 million and a potential additional payment of $25 million if a patent extension is approved [34]. - The company is exploring additional opportunities to acquire companies with commercial drug products or products in development to diversify its portfolio [34]. - The company has entered into a definitive agreement with Endo for the development and commercialization of generic Sabril® tablets, with an upfront payment of $500,000 and a potential milestone payment of $2.0 million [61]. - The company is exploring additional acquisition opportunities in rare disease and CNS therapeutic categories to diversify its product portfolio [91]. Product Development and Marketing - FYCOMPA® is the first and only AED targeting the AMPA receptor, with ongoing efforts to market this product [54]. - The company expects to hire approximately 34 sales and marketing personnel to support the marketing of FYCOMPA® [34]. - FYCOMPA® is expected to be marketed by the company through its own sales team starting May 2023, with plans to hire approximately 34 sales and marketing personnel [89]. - The company plans to expand FIRDAPSE® commercialization efforts into Japan and other Asian territories upon approval [93]. Regulatory and Legal Matters - The company filed lawsuits against three generic drug manufacturers to protect its patents for FIRDAPSE® following Paragraph IV Certification Notice Letters received in January 2023 [33]. - The company has filed lawsuits against three generic drug manufacturers following Paragraph IV Certification Notice Letters received in January 2023, which challenge the validity of six patents listed in the FDA Orange Book for FIRDAPSE® [128]. - The company is required to conduct a pediatric safety study and has established a pregnancy surveillance program for FIRDAPSE® [112]. Market and Competitive Landscape - The pharmaceutical industry is highly competitive, with many organizations having greater resources than the company [66]. - The company faces significant competition in the biotechnology and pharmaceutical industries, with competitors having greater financial resources and experience [204]. - The commercial success of the company's drug products will depend on third-party payor coverage and reimbursement, which may not be sufficient to ensure profitability [212]. Patient Access and Support Programs - The company has a co-pay assistance program that reduces out-of-pocket costs for LEMS patients to no more than $10.00 per month, currently averaging less than $2.00 per month [88]. - The company is committed to patient access programs to ensure no LEMS patient is denied access to FIRDAPSE® for financial reasons [93]. - Catalyst Pathways® is a personalized treatment support program that assists patients in managing the dosing and titration regimen for FIRDAPSE® [82]. Intellectual Property and Patent Protection - The company aims to maintain its patent protection for FIRDAPSE® to prevent generic competition after orphan drug exclusivity expires [86]. - The company holds patent rights for FIRDAPSE® with a patent expiration date of April 7, 2034, protecting specific dosing regimens [127]. - Catalyst Pharmaceuticals emphasizes the importance of protecting its intellectual property and regulatory exclusivities as a strategic priority for future success [128]. Operational Challenges - The company is reliant on third-party contract manufacturers for production, which poses risks related to compliance with current good manufacturing practices (cGMP) and potential disruptions in supply [209]. - The ongoing COVID-19 pandemic may adversely affect the company's business operations and financial results [219]. - The company may encounter difficulties in managing growth, which could adversely affect its financial condition and operational results [211].

Financial Data and Key Metrics Changes - Catalyst Pharmaceuticals reported a record quarterly net product revenue of $57.2 million for Q3 2022, representing a 59.3% increase compared to Q3 2021 [5][44] - GAAP net income for Q3 2022 was $22.7 million, or $0.22 per basic share, reflecting a 120.2% increase year-over-year [6][45] - Non-GAAP net income for Q3 2022 was $28.6 million, or $0.28 per basic share, which is an 83.3% increase compared to Q3 2021 [7][48] - The company ended Q3 2022 with $256 million in cash and short-term investments, marking a $35.3 million increase from the previous quarter [9][55] Business Line Data and Key Metrics Changes - Firdapse net sales for Q3 2022 were $57.2 million, showing an 8% growth quarter-over-quarter and a 59.3% growth year-over-year [22] - The discontinuation rate of reimbursed patients in Q3 was 35% lower than in Q2, indicating improved patient retention [23] - Average co-pay for patients enrolled in Catalyst Pathways was less than $2 per month, facilitating access to treatment [24] Market Data and Key Metrics Changes - Approximately 50% of LEMS patients have a comorbidity of small cell lung cancer, representing a significant market opportunity for Catalyst [11] - The company estimates that there are about 1,500 small cell lung cancer LEMS patients in the U.S., with over 80% currently undiagnosed [27] Company Strategy and Development Direction - Catalyst aims to broaden and diversify its product portfolio through acquisitions and partnerships, with ongoing due diligence for potential commercial stage products [15][42] - The company has launched a marketing initiative targeting thoracic oncologists to educate them about LEMS and Firdapse, expected to ramp up in early 2023 [11][28] - Catalyst is focused on expanding access to Firdapse for all LEMS patients, including pediatric patients aged six and older, following recent FDA approval [14][32] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in achieving 2022 objectives, revising Firdapse revenue guidance upward to $205 million to $210 million [8] - The company anticipates continued organic growth driven by new patient enrollments and improved diagnostic efforts [24][80] - Management acknowledged the potential for challenges to their patent position but indicated preparedness for such events [80] Other Important Information - Catalyst was named Company of the Year by BioFlorida and ranked 39th in Fortune Magazine's 2022 Top 100 Fastest Growing Companies [17][18] - The company was added to the S&P SmallCap 600 Index on September 15, 2022, reflecting its market capitalization and growth [19] Q&A Session Summary Question: How many patients are currently offered Firdapse, and how many are small cell lung cancer patients? - Management indicated that about 30% of new enrollments are small cell lung cancer LEMS patients, with expectations for growth in this area [60] Question: What is the target for small cell lung cancer patients in 2023? - The company expects to increase the percentage of small cell lung cancer patients among new enrollments from 30% to about 35% to 40% in 2023 [61] Question: Is the patient growth from breadth or depth in the prescriber base? - The prescriber base is broad, with 75% of prescribers treating only one patient, indicating potential for future growth as physicians gain experience [66] Question: Are there differences in persistence between treatment naïve patients and those switching from Ruzurgi? - No significant differences in persistence were observed between the two groups, with both groups showing similar treatment adherence [68] Question: What is the current status of patients on Ruzurgi for other indications? - Approximately 100 patients with congenital myasthenic syndromes are being treated with Ruzurgi under INDs, but no specific plans to pursue this indication currently exist [72]

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, 2022 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 incor ...

Financial Data and Key Metrics Changes - Catalyst Pharmaceuticals reported an all-time high in total FIRDAPSE net revenue of $53 million for Q2 2022, representing a 57.7% increase compared to the same period last year [6] - Net income before income taxes for Q2 2022 was $28.2 million, a 77.6% increase compared to $15.9 million for Q2 2021 [7] - GAAP net income for Q2 2022 was $21.6 million, or $0.21 per basic share and $0.20 per diluted share, reflecting a 77.5% increase year-over-year [49] - The company ended Q2 2022 with $220.8 million in cash and investments and no funded debt [7][57] Business Line Data and Key Metrics Changes - FIRDAPSE net sales for Q2 2022 were $53 million, showing a 23.3% growth quarter-over-quarter and a 57.7% growth year-over-year [22] - The company successfully transitioned nearly 100% of adult LEMS patients from Ruzurgi to FIRDAPSE without a lapse in therapy [23] - Payer approval rates for adult LEMS patients transitioning from Ruzurgi were greater than 90% [24] Market Data and Key Metrics Changes - The company expects steady organic growth in the second half of 2022, primarily driven by new patient enrollments of already diagnosed LEMS patients not yet on FIRDAPSE [26] - Leading indicators for Q3 are positive, with July net new patients ahead of forecast due to low discontinuation rates and consistent new enrollments [27] Company Strategy and Development Direction - Catalyst Pharmaceuticals aims to broaden and diversify its product portfolio through collaborative partnerships and acquisitions of commercial stage assets [17] - The company is actively engaged in advanced stages of due diligence for several acquisition opportunities [18] - The company has made significant progress in building its ESG program, publishing its inaugural set of industry-specific disclosures [20] Management's Comments on Operating Environment and Future Outlook - Management expressed optimism about the future, believing the company is well-positioned to execute its growth strategy and deliver long-term value for stakeholders [21] - The company anticipates that 2022 will continue to be a transformative year with increased momentum to diversify and invest in innovative opportunities for rare diseases [60] Other Important Information - The company resolved issues with Jacobus Pharmaceuticals, acquiring certain intellectual property rights, including U.S. and Mexico rights for Ruzurgi [12] - A supplemental new drug application for FIRDAPSE to support its use in pediatric LEMS patients has been accepted by the FDA for review [15] Q&A Session Summary Question: What are the key factors to achieve the upper end of revenue guidance? - Management indicated that strong new patient enrollments and low discontinuation rates are crucial for achieving the upper end of revenue guidance [64] Question: What information was provided to the FDA regarding the pediatric LEMS supplemental application? - The company submitted pediatric safety data and simulations on how adult data translates to pediatric patients [67] Question: Will potential business development activities be accretive to revenue opportunities next year? - Management expects that any potential business development activities would be accretive to revenues next year, leveraging established commercial infrastructure [68] Question: What is the expected pace of naïve new patient enrollments in the second half of the year? - Management expects the pace of growth to continue, with strong new enrollments and steady discontinuation rates [74] Question: What does the newly acquired patent cover? - The patent contains claims for a new and novel previously unknown impurity that appears in all products containing amifampridine [78]

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 June 30, 2022 OR ☐ TRANSITION REPORT UNDER SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 Commission File ...

| --- | --- | |------------------------------|-------| | | | | Dedicated To Making A | | | Meaningful Difference In The | | | Lives Of Patients | | | Suffering From Rare Diseases | | NASDAQ: CPRX May 2022 1 Confidential Safe Harbor 2 Confidential This presentation contains forward-looking statements that are subject to a number of risks and uncertainties, many of which are outside our control. All statements regarding our strategy, future operations, financial position, estimated revenues or losses, project ...

Financial Data and Key Metrics Changes - The company reported record net product revenue of $43 million for Q1 2022, a 42.5% increase compared to Q1 2021 [6] - Net income before income taxes for Q1 2022 was $17.5 million, a 76.7% increase from $9.9 million in Q1 2021 [7] - GAAP net income for Q1 2022 was $13.2 million, or $0.13 per basic share, representing a 72.8% increase year-over-year [39] - The company ended the quarter with $198 million in cash and short-term investments, with no funded debt [46] Business Line Data and Key Metrics Changes - FIRDAPSE net sales for Q1 2022 were $43 million, reflecting a 42% growth compared to the same quarter last year [21] - The transition of patients from Ruzurgi to FIRDAPSE was successful, with over 90% of adult LEMS patients transitioned by the end of March 2022 [22] - The company anticipates the conversion of remaining patients to be completed by the end of Q2 2022 [10] Market Data and Key Metrics Changes - The company estimates approximately 3,000 LEMS patients in the U.S., with about half undiagnosed or misdiagnosed [12] - The company has identified over 500 potential new patients based on claims data and field intelligence [62] Company Strategy and Development Direction - The company aims to diversify its product portfolio through collaborative partnerships and acquisitions of commercial-stage assets [14] - The company is actively evaluating several potential opportunities for acquisition but has not entered into any agreements to date [15] - The company has fortified its intellectual property with three new patents, enhancing the commercial durability of FIRDAPSE [13] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in sustaining organic growth driven by newly diagnosed patients and those misdiagnosed [12] - The company reiterated its full-year 2022 total revenue guidance of $195 million to $205 million, representing a 38% to 45% increase compared to 2021 [8] - Management highlighted the importance of the favorable court ruling that removed Ruzurgi from the market, which is expected to drive additional patient enrollment [9] Other Important Information - The company has submitted a supplemental NDA for pediatric label expansion to the FDA, aiming to provide access to FIRDAPSE for pediatric LEMS patients [17] - The company received a favorable Canadian Federal Court ruling enforcing data protection for FIRDAPSE, resulting in the removal of Ruzurgi from the Canadian market [18] Q&A Session Summary Question: Transition of Ruzurgi patients to FIRDAPSE - Management confirmed that over 90% of adult LEMS patients transitioned from Ruzurgi to FIRDAPSE, with payer approval rates greater than 95% [52][53] Question: Trends in new patient enrollment - Management noted that April's new patient enrollments remained strong, almost matching March's figures, and attributed this to improved access to healthcare [54] Question: Business development activities - Management indicated they are deep into due diligence on several promising commercial opportunities, focusing on FDA-approved drugs that have not yet launched [56] Question: Revenue contributors for Q2 - Management expects the full impact of the Ruzurgi transition and strong organic growth from new patient enrollments to contribute significantly to Q2 revenue [60] Question: Pediatric LEMS patients on FIRDAPSE - Management estimated there are fewer than 20 pediatric LEMS patients currently receiving FIRDAPSE through expanded access [71] Question: Targeting tumor LEMS patients - Management reported that around 22% of new enrollments are from tumor LEMS patients, with ongoing educational efforts aimed at oncologists to increase this percentage [72] Question: Organic growth rate - Management indicated an organic growth rate of approximately 15% to 20% year-over-year, with expectations for continued strong growth as patient visits increase [75]