Summary of Catalyst Pharmaceuticals FY Conference Call Company Overview - Catalyst Pharmaceuticals operates in the Central Nervous System (CNS) space, focusing on therapies for rare diseases, particularly Lambert-Eaton Myasthenic Syndrome (LEMS) and Duchenne Muscular Dystrophy (DMD) [3][4] Key Product Insights FIRDAPSE - FIRDAPSE is a successful therapy for LEMS, with over six years on the market and a strong performance record, achieving 15 consecutive quarters of 15% or more growth until impacted by a cyberattack [5] - In the first half of 2025, FIRDAPSE sales grew by 17% compared to the first half of 2024, with a current addressable market of approximately $1.2 billion [5] - The company has split its brand team to focus on idiopathic and oncology-related markets, aiming to sustain growth [6] - The NCCN guidelines inclusion is expected to enhance market penetration, with a focus on integrating these guidelines into care pathways of major oncology practices [8][10] AGAMREE - AGAMREE is a differentiated steroid for DMD, with a significant unmet medical need as only 7% of diagnosed patients are currently on steroids despite 95% having used them at some point [23] - The product has penetrated 93% of the 100 centers of excellence in the U.S., with a dedicated sales force to enhance growth [24][25] - The company is addressing competition from gene therapies and has a strong share of voice in the steroid market [26] - AGAMREE's side effect profile is reported to be superior, with benefits in bone health and reduced anxiety compared to other steroids [32] Market Dynamics - The company is actively identifying patients through AI and machine learning, focusing on misdiagnosed cases of myasthenia gravis to drive growth in the idiopathic market [11][12] - The average physician sees very few LEMS patients, making patient prospecting essential [13] - There are approximately 900 LEMS patients diagnosed annually with small cell lung cancer, presenting a significant opportunity for the company [14] Intellectual Property and Litigation - Catalyst Pharmaceuticals has settled three out of four lawsuits related to its intellectual property, with ongoing litigation against Hetero [18] - The settlements extend the product's market exclusivity, potentially allowing for an additional 10 years of growth [19] Business Development Strategy - The company is focused on acquiring differentiated, profitable products and emphasizes the importance of cultural fit in partnerships [38][39] - Current market conditions are seen as favorable for finding new assets, with 80-90% of opportunities being inbound due to the company's success [39] Future Outlook - Catalyst Pharmaceuticals aims to explore opportunities within the CNS space, particularly in movement disorders, while keeping peak year sales under $500 million to avoid competition with larger players [40] - The company is therapeutic agnostic, believing its existing infrastructure can support various therapeutic areas [41] Conclusion - Catalyst Pharmaceuticals expresses optimism about future growth opportunities and appreciates the support from stakeholders [42]

Summary of Catalyst Pharmaceuticals (CPRX) 2025 Conference Call Company Overview - Catalyst Pharmaceuticals operates under a buy-and-build business model, focusing on orphan and rare diseases, particularly in the CNS (Central Nervous System) sector. This model aims to reduce risk for shareholders and stakeholders by leveraging valuable assets and established infrastructure for patient support and commercial operations [4][5] Key Products and Market Focus - The company has three main assets: - **FIRDAPSE**: For Lambert-Eaton Myasthenic Syndrome (LEMS) with an estimated patient population of 2,500 to 3,000 in the U.S. - **AGAMREE**: A novel corticosteroid for Duchenne muscular dystrophy (DMD) with a patient population of 11,000 to 13,000, launched last year. - **FYCOMPA**: An epilepsy drug recently lost exclusivity, with guidance for 2025 set at $90 to $95 million [6][15][29] Performance Highlights - FIRDAPSE is performing well, with initiatives to improve patient titration and support from specialty pharmacies. The average physician sees one LEMS patient in their career, indicating a need for enhanced education and support [10][11] - AGAMREE is gaining traction, with 45% of patients coming from Influenza treatments and 45% from prednisone, indicating a broad market engagement [46][47] - FYCOMPA's performance is under scrutiny due to the introduction of generics, with expectations of a softer second half of the year [15][16] Growth Potential - FIRDAPSE has a total addressable market estimated at $1.2 billion, with current penetration at 30% for idiopathic LEMS and less than 10% for cancer-associated LEMS. The company sees significant growth opportunities in oncology due to recent guideline changes and testing accessibility [19][34] - AGAMREE's market share is expected to grow as it captures patients transitioning from other steroid treatments, with a focus on pediatric applications [68] Intellectual Property (IP) Status - Catalyst has settled with three out of four generic filers for FIRDAPSE, extending the entry date for generics to February 2035. The remaining litigant is under ongoing discussions [23][24] Business Development Strategy - The company is actively seeking differentiated products under $500 million, focusing on orphan drugs with potential for multiple indications. There is a particular interest in pediatric opportunities and oncology [66][68] Key Value Drivers - Enrollment in the Summit study, which aims to evaluate safety signals for AGAMREE, is crucial for future growth. The prevalence of testing for LEMS is also a significant indirect measure of the company's success [69] Conclusion - Catalyst Pharmaceuticals is positioned for growth in the orphan drug market, with a strong focus on patient support and education. The company is optimistic about its product pipeline and market opportunities, particularly in oncology and pediatric applications, while navigating challenges posed by generics in the epilepsy market [4][68]

Financial Data and Key Metrics Changes - The company reported a first-half performance of $70 million against a guidance of $90 million to $95 million for the year [75][76] - The maximum dose for Firdapse was increased from 80 mg to 100 mg, which is expected to drive patient dosing closer to the new maximum [28][29] - The company anticipates a significant dollar share loss due to the entry of additional generics in the market [80][81] Business Line Data and Key Metrics Changes - Firdapse has a market penetration of approximately 30% in the idiopathic LEMS segment, with an addressable market of about $600 million [9][17] - The cancer-associated LEMS segment has a penetration of only about 10%, with a similar addressable market size [17] - GammaRay for Duchenne's muscular dystrophy has been well received, with 45% of patients coming from prednisone, 45% from Emflaza, and 10% being naive [46] Market Data and Key Metrics Changes - The company is focused on the U.S. market, with a specific emphasis on identifying misdiagnosed patients in the LEMS space [3][4] - The addressable market for both idiopathic and cancer-associated LEMS is estimated at $600 million each, with significant growth potential [17][31] - The company has established partnerships for frictionless testing to improve patient diagnosis and treatment pathways [13][19] Company Strategy and Development Direction - The company aims to grow through acquisitions and lifecycle management of existing products, focusing on commercial execution rather than R&D [2][5] - There is a strong emphasis on building credibility with patient advocacy groups and utilizing non-traditional marketing strategies [86][88] - The company is looking for business development opportunities that are therapeutically agnostic and immediately accretive [91][92] Management's Comments on Operating Environment and Future Outlook - Management expressed optimism about the growth potential in both idiopathic and cancer-associated LEMS, particularly with recent NCCN guideline updates [19][20] - The company is preparing for potential challenges in the gene therapy market, which may create opportunities for GammaRay [57][61] - Management is focused on achieving full enrollment in the SUMMIT study and advancing the cancer LEMS initiative [93][95] Other Important Information - The company has settled with two of the three first filers in patent litigation, potentially extending exclusivity until February 2035 [32] - The company is actively working on life cycle management strategies for GammaRay to enhance its market position [47][58] Q&A Session Summary Question: Can you talk about the assumptions going into the 2025 guidance for Firdapse? - The guidance of $355 million to $360 million is based on increased dosing and a dedicated sales force focused on both Firdapse and GammaRay [28][29][30] Question: What is the status of the NCCN guidelines and frictionless testing? - The NCCN guidelines were updated favorably in July, and the frictionless testing contract was finalized around May-June [19][20] Question: How does the company plan to address the discontinuation rate for Firdapse? - The primary reason for discontinuation is patient mortality, but the company is implementing a titration schedule to improve patient retention [21][22][23] Question: What is the strategy for GammaRay in a competitive market? - The company is positioning GammaRay slightly below branded Emflaza to avoid market blockage and is targeting a broad patient base [41][42][46] Question: How does the company view future SG&A investments? - The company is cautious about overwhelming healthcare providers with sales representatives and is focusing on non-traditional outreach methods [84][86][89]

Financial Data and Key Metrics Changes - The company reported a guidance for Firdapse revenue in 2025 of $355 million to $360 million, with year-to-date trends tracking positively against these assumptions [28] - The addressable market for Firdapse is estimated at over $1 billion, with confidence in reaching this target due to recent increases in maximum dosage from 80 mg to 100 mg [30][31] - The company anticipates a revenue of $100 million to $110 million for GammaRay in 2025, with an addressable market of $1.2 billion [52] Business Line Data and Key Metrics Changes - Firdapse has a market penetration of approximately 30% in the idiopathic LEMS segment, with an addressable market of about 1,700 to 2,000 patients [9] - The cancer-associated LEMS segment has a market penetration of only about 10%, with an addressable market of $600 million [18] - GammaRay has been successful in capturing market share, with 45% of patients coming from prednisone, 45% from Emflaza, and 10% being naive patients [45] Market Data and Key Metrics Changes - The company has established a partnership with Quest Diagnostics for frictionless testing, which is expected to enhance patient diagnosis and treatment pathways [14] - The NCCN guidelines have been updated favorably for LEMS patients, which is anticipated to improve market access and treatment adherence [20] Company Strategy and Development Direction - The company is focused on a "buy and build" strategy, seeking opportunities in the market while managing commercial and regulatory risks [3] - There is a strong emphasis on lifecycle management of existing products and potential acquisitions to drive growth [5] - The company aims to maintain a strong balance sheet while exploring business development opportunities in CNS and other therapeutic areas [90] Management's Comments on Operating Environment and Future Outlook - Management expressed optimism about the growth potential in both idiopathic and cancer-associated LEMS markets, particularly with improved diagnostic capabilities [21] - The company is preparing for potential challenges in the market, including competition from generics and the need for effective patient education [56] - Management highlighted the importance of building credibility with patient advocacy groups and adapting to the evolving landscape of gene therapy [87] Other Important Information - The company has a dedicated sales force for both Firdapse and GammaRay, which is expected to enhance market penetration and patient engagement [83] - There is an upcoming Markman hearing related to patent litigation, which could influence the company's market position and exclusivity [36] Q&A Session Summary Question: What are the assumptions behind the 2025 guidance for Firdapse? - The guidance is based on an increase in maximum dosage and a reassigned sales force dedicated to Firdapse, which is expected to drive patient uptake [28][29] Question: How does the company plan to address the discontinuation rate of Firdapse? - Management noted that the primary reason for discontinuation is patient mortality, but they are implementing a titration schedule to improve patient retention [22][23] Question: What is the strategy for GammaRay in a competitive market? - The company plans to position GammaRay slightly below the branded Emflaza to ensure market access and capitalize on existing patient dissatisfaction with current therapies [41][42] Question: How does the company view the impact of generics on Fycompa? - Management acknowledged that while prescription volumes may remain stable, dollar share is expected to decline as generics enter the market [78][79] Question: What are the key achievements the company aims for in the next year? - The company aims to achieve full enrollment in the SUMMIT study and advance its cancer LEMS initiative [93]

Core Viewpoint - Catalyst Pharmaceuticals and SERB have reached a settlement with Lupin regarding the patent litigation over FIRDAPSE, allowing Lupin to market a generic version starting February 25, 2035, if approved by the FDA [1][2]. Group 1: Settlement Agreement Details - The settlement prohibits Lupin from marketing its generic version of FIRDAPSE in the U.S. before February 25, 2035, unless specific circumstances arise [2]. - All ongoing patent litigation between Catalyst/SERB and Lupin concerning FIRDAPSE patents will be terminated as part of the agreement [2]. - Catalyst has previously settled similar litigation regarding ANDA applications for FIRDAPSE with Teva Pharmaceuticals and Inventia Healthcare Limited [2]. Group 2: Company Overview - Catalyst Pharmaceuticals is focused on developing and commercializing treatments for rare diseases and has a strong commitment to patient care [4]. - The company has been recognized on the Forbes 2025 list as one of America's Most Successful Mid-Cap Companies and on the 2024 Deloitte Technology Fast 500™ list as one of North America's Fastest-Growing Companies [4].

Core Viewpoint - Catalyst Pharmaceuticals (CPRX) has entered into a settlement agreement with Lupin Pharmaceuticals regarding patent litigation over Firdapse, which protects the company's sales from generic competition until February 25, 2035 [1][5][6]. Group 1: Firdapse Overview - Firdapse is approved in the U.S., EU, and Japan for treating Lambert-Eaton Myasthenic Syndrome (LEMS) in adults and has recently expanded its label to include pediatric patients aged six and older [2]. - In 2024, Firdapse accounted for 62% of Catalyst Pharmaceuticals' sales, generating $168.6 million in revenue in the first half of 2025 [5][8]. Group 2: Settlement Details - The settlement prohibits Lupin from marketing its generic version of Firdapse in the U.S. before February 25, 2035, unless specific exceptions arise [6]. - The agreement also resolves all ongoing patent litigation between Catalyst Pharmaceuticals/SERB and Lupin in the U.S. District Court in New Jersey [6][7]. Group 3: Other Products and Market Position - Catalyst Pharmaceuticals markets two additional drugs in the U.S.: Fycompa for epilepsy and Agamree for Duchenne muscular dystrophy, which provide incremental revenue [9]. - The patents for Fycompa are set to expire in 2025 and 2026, leading to anticipated declines in sales due to increased competition from generics [10].

Core Viewpoint - Catalyst Pharmaceuticals and SERB have reached a settlement with Lupin regarding the patent litigation over FIRDAPSE, allowing Lupin to market a generic version starting February 25, 2035, pending FDA approval [1][2]. Group 1: Settlement Agreement Details - The settlement agreement resolves ongoing patent litigation between Catalyst/SERB and Lupin concerning FIRDAPSE [2]. - Lupin is prohibited from marketing its generic version of FIRDAPSE in the U.S. before February 25, 2035, unless specific circumstances arise [2]. - The agreement includes the termination of all ongoing patent litigation related to FIRDAPSE patents in the U.S. District Court for the District of New Jersey [2]. Group 2: Company Background - Catalyst Pharmaceuticals is focused on developing and commercializing treatments for rare diseases and has a strong commitment to patient care [4]. - The company has been recognized on the Forbes 2025 list as one of America's Most Successful Mid-Cap Companies and on the 2024 Deloitte Technology Fast 500™ list as one of North America's Fastest-Growing Companies [4].

Financial Data and Key Metrics Changes - Total revenue for Q2 2025 reached $146.6 million, a 19.4% increase year over year, while total revenue for 2025 grew 30.2% to $288 million [5][29] - The company ended Q2 2025 with a cash position of $652.8 million, reinforcing its ability to invest strategically for long-term growth [5][29] - GAAP net income for Q2 2025 was $52.1 million, or $0.41 per diluted share, reflecting a 27.7% increase year over year [30] Business Line Data and Key Metrics Changes - Firdapse generated net product revenue of $84.8 million in Q2 2025, a 9.7% increase year over year, with year-to-date revenue of $168.6 million, representing a 16.9% increase over 2024 [6][29] - GammaRy achieved net product revenue of $27.4 million in Q2 2025, a 213% increase year over year, with first half revenues reaching $49.4 million, up 398% from the prior year [10][29] - Fycompa reported revenue of $34.3 million in Q2 2025, a 6% decrease year over year, with full year guidance remaining unchanged at $90 million to $95 million [11][29] Market Data and Key Metrics Changes - The company is focusing on expanding its reach in the oncology market, where it believes potentially 90% of cancer-associated LEMS patients remain undiagnosed, representing a significant growth opportunity [9][24] - The updated NCCN guidelines for small cell lung cancer, published on July 25, now include VGCC antibody testing and recommend amifampridine for treatment, which is expected to enhance market access [22] Company Strategy and Development Direction - The company is advancing a focused expansion strategy for Firdapse, centered on educating healthcare providers and improving diagnostic access [9][21] - The company remains committed to disciplined business development, actively evaluating opportunities that align with its long-term growth strategy [16][98] - The leadership changes, including the appointment of a new Chief Medical Officer, are aimed at ensuring scientific rigor and operational excellence [12][14] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in achieving full year 2025 revenue guidance of $545 million to $565 million, supported by strong leading indicators [6][35] - The company anticipates sustained growth driven by the strong performance of its commercial portfolio and the successful execution of its strategic initiatives [35] Other Important Information - The company is awaiting a trial date in ongoing patent litigation for Firdapse, expected in Q4 2025 or Q1 2026 [17] - The company published its 2024 ESG report, underscoring its commitment to sustainable growth and responsible innovation [18] Q&A Session Summary Question: Can you speak more about your strategy for educating oncologists on the LEMS opportunity? - The company is focused on increasing frictionless testing for patients and has implemented digital marketing and educational initiatives to engage oncologists [38][41] Question: What impact are you seeing on Firdapse and GammaRy uptake following the increase in sales force size? - Early indicators show increased engagements and deeper relationships with healthcare providers, with strong performance in enrollments for both products [49][51] Question: How do you view the potential for PTCT's Translarna FDA approval for DMD treatment? - The company believes that GammaRy will continue to retain market share as it is viewed as a differentiated treatment option for DMD [93][95] Question: What is your current stance on near-term strategic initiatives to offset Fycompa's loss of exclusivity? - The company is actively assessing multiple opportunities for business development and lifecycle management to offset potential revenue loss from Fycompa [96][98]

Financial Data and Key Metrics Changes - Catalyst Pharmaceuticals reported total revenue of $146.6 million for Q2 2025, a 19.4% increase year over year, and total revenue for 2025 reached $288 million, reflecting a 30.2% growth [5][28] - The company ended Q2 2025 with a cash position of $652.8 million, indicating strong financial health and capacity for strategic investments [5][28] - GAAP net income for Q2 2025 was $52.1 million, or $0.41 per diluted share, representing a 27.7% increase year over year [29] Business Line Data and Key Metrics Changes - Firdapse generated net product revenue of $84.8 million in Q2 2025, a growth of $7.5 million compared to Q2 2024, with year-to-date revenue of $168.6 million, a 16.9% increase over the previous year [6][28] - GammaRy achieved net product revenue of $27.4 million in Q2 2025, a 213% increase year over year, with first half revenues reaching $49.4 million, up 398% from the prior year [10][28] - Fycompa reported revenue of $34.3 million in Q2 2025, reflecting a 6% decrease year over year, with full year guidance remaining unchanged at $90 million to $95 million [11][28] Market Data and Key Metrics Changes - The company noted that approximately 90% of cancer-associated LEMS patients remain undiagnosed, presenting a significant opportunity for market expansion [9][23] - Prescription approval rates for Firdapse remained above 90%, indicating strong patient adherence and a solid foundation for sustained performance [20] Company Strategy and Development Direction - Catalyst Pharmaceuticals is focused on expanding Firdapse's market presence in oncology, supported by updated NCCN guidelines that recommend its use for cancer patients with LEMS [9][22] - The company is actively pursuing a three-step strategy to enhance diagnostic access and improve patient outcomes, which includes frictionless testing, educational initiatives, and partnerships with oncology practices [21][23] - The leadership team has been strengthened with the addition of new executives, enhancing the company's strategic capabilities [12][15] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in achieving full year 2025 revenue guidance of $545 million to $565 million, supported by strong leading indicators and a balanced commercial portfolio [6][33] - The company remains committed to disciplined financial management and strategic growth, with a focus on long-term value creation [16][33] Other Important Information - Catalyst Pharmaceuticals is awaiting a trial date for ongoing patent litigation related to Firdapse, with expectations for clarity following a scheduled Markman hearing [16] - The company published its 2024 ESG report, emphasizing its commitment to sustainable growth and responsible innovation [17] Q&A Session Summary Question: Can you speak more about your strategy for educating oncologists on the LEMS opportunity? - The company is focused on increasing frictionless testing for patients and enhancing education through digital marketing, congresses, and publications. Success will be tracked through increased VGCC antibody testing and a growing mix of cancer-associated LEMS patients [36][39][41] Question: What impact are you seeing on Firdapse and GammaRy uptake following the increase in sales force size? - Early indicators show increased engagements and deeper relationships with healthcare providers, with strong performance in enrollments and a solid pipeline for both products [47][50][51] Question: How do you view the potential for PTCT's Translarna FDA approval for DMD treatment? - The company believes that GammaRy will continue to retain market share as it is viewed as a differentiated steroid treatment for DMD, unaffected by Translarna's approval [88][90] Question: What is your current stance on near-term strategic initiatives to offset Fycompa's loss of exclusivity? - The company is actively assessing multiple opportunities for business development and lifecycle management to offset potential revenue loss from Fycompa [92][93]

Group 1 - Catalyst Pharmaceutical (CPRX) reported quarterly earnings of $0.68 per share, exceeding the Zacks Consensus Estimate of $0.58 per share, and showing an increase from $0.56 per share a year ago, resulting in an earnings surprise of +17.24% [1] - The company achieved revenues of $146.56 million for the quarter ended June 2025, surpassing the Zacks Consensus Estimate by 5.30%, and up from $122.71 million in the same quarter last year [2] - Catalyst has consistently surpassed consensus EPS estimates for the last four quarters, indicating strong performance [2] Group 2 - The stock has gained approximately 6.5% since the beginning of the year, compared to the S&P 500's gain of 7.1%, suggesting a slight underperformance relative to the broader market [3] - The company's earnings outlook is crucial for investors, as it includes current consensus earnings expectations for upcoming quarters and any recent changes to these expectations [4] - The Zacks Rank for Catalyst is currently 2 (Buy), indicating expectations for the stock to outperform the market in the near future [6] Group 3 - The current consensus EPS estimate for the upcoming quarter is $0.56 on revenues of $138.29 million, while for the current fiscal year, the estimate is $2.25 on revenues of $559.96 million [7] - The Medical - Drugs industry, to which Catalyst belongs, is currently ranked in the top 34% of over 250 Zacks industries, suggesting a favorable industry outlook [8]