CATALYST PHARMACEUTICALS, INC.

Catalyst Pharmaceuticals, Inc. is 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. The company currently sells three commercial-stage drug products: FIRDAPSE® (amifampridine), AGAMREE® (vamorolone), and FYCOMPA® (perampanel). Its core business model revolves around generating revenue from product sales, supplemented by license and other revenue from sublicensees in international markets. The company emphasizes a patient-first approach, providing personalized treatment support programs like Catalyst Pathways® and financial assistance to ensure access to its medications.

The company's revenue generation is primarily from product sales, with a mix of recurring income from ongoing sales and transactional income from milestone payments in collaborative arrangements. Primary customer segments include patients with rare neurological and neuromuscular disorders, with a focus on conditions like Lambert-Eaton Myasthenic Syndrome (LEMS) and Duchenne Muscular Dystrophy (DMD). The company also engages with healthcare providers, rare disease advocacy organizations, and specialty pharmacies to support its ecosystem.

FIRDAPSE® (amifampridine) is approved for the treatment of adult and pediatric patients (ages six and older) with Lambert-Eaton Myasthenic Syndrome (LEMS). In May 2024, the FDA approved an sNDA increasing the maximum daily dosage from 80 mg to 100 mg, offering greater treatment flexibility. FIRDAPSE® is marketed in the U.S. by a field-based force of approximately 23 personnel, including sales and thought leader liaisons, with two national account directors focusing on the oncology market for LEMS patients with cancer. Non-personal promotion targets 20,000 neurologists and 16,000 oncologists. The company also offers a no-cost LEMS diagnostic testing program. Internationally, FIRDAPSE® is marketed in Canada through sublicensee KYE Pharmaceuticals, Inc., and in Japan through DyDo Pharma, Inc., which began commercialization on January 21, 2025. The company holds six U.S. patents for FIRDAPSE® expiring between 2032 and 2037.

AGAMREE® (vamorolone) is a novel corticosteroid approved by the U.S. FDA on October 26, 2023, for treating Duchenne muscular dystrophy (DMD) in patients aged two years and older. The company acquired exclusive North American rights to AGAMREE® from Santhera Pharmaceuticals Holding AG in July 2023 for an upfront payment of $75 million ¹ and a regulatory milestone payment of $36 million ² in Q4 2023. AGAMREE® was launched in March 2024, initially utilizing the FIRDAPSE® commercial force, but a dedicated field-based force of approximately 16 personnel was established for AGAMREE® effective April 1, 2025. The company is conducting a SUMMIT registry study with up to 250 patients to evaluate long-term safety and quality of life data. AGAMREE® has New Chemical Entity exclusivity expiring in October 2028 and Orphan Drug Exclusivity expiring in October 2030, further protected by seven Orange Book listed patents expiring between May 28, 2029, and July 16, 2040. In July 2024, the company licensed exclusive Canadian commercial rights for AGAMREE® to KYE, which received Health Canada approval on October 2, 2025.

FYCOMPA® (perampanel) CIII is an anticonvulsant approved for focal onset seizures in patients four years and older, and primary generalized tonic-clonic seizures in patients 12 years and older. The company acquired the U.S. rights to FYCOMPA® from Eisai Co., Ltd. on January 24, 2023, for an upfront cash payment of $160 million ³. Patent protection for FYCOMPA® tablets and oral solution was primarily from two patents: U.S. patent no. 6,949,571, which expired on May 23, 2025, and U.S. Patent No. 8,772,497, expiring on July 1, 2026. Following the expiration of the first patent, generic versions of FYCOMPA® tablets and oral suspension have entered the market. The company ceased active marketing efforts for FYCOMPA® effective December 31, 2025, but continues to sell the product.

For the fiscal year ended December 31, 2025, total revenues were approximately $589.0 million ⁴, including $588.8 million ⁵ in net product revenue. This compares to total revenues of approximately $491.7 million ⁶ and net product revenue of $489.3 million ⁷ for the fiscal year ended December 31, 2024. Gross profit is not explicitly stated, but cost of sales was approximately $87.3 million ⁸ in 2025, compared to $68.8 million ⁹ in 2024. Operating income was approximately $257.8 million ¹⁰ in 2025, up from $195.1 million ¹¹ in 2024. Net income was approximately $214.3 million ¹² ($1.75 ¹³ per basic share and $1.68 ¹⁴ per diluted share) in 2025, compared to $163.9 million ¹⁵ ($1.38 ¹⁶ per basic share and $1.31 ¹⁷ per diluted share) in 2024. Cash and cash equivalents were approximately $709.2 million ¹⁸ at December 31, 2025, compared to $517.6 million ¹⁹ at December 31, 2024. Total liabilities were $149.7 million ²⁰ at December 31, 2025, compared to $123.8 million ²¹ at December 31, 2024. Free cash flow is not explicitly reported, but net cash provided by operating activities was $208.7 million ²² in 2025, compared to $239.8 million ²³ in 2024.

Year-over-year, total revenues increased by approximately $97.3 million ²⁴, driven primarily by the commercialization of AGAMREE®. FIRDAPSE® net product revenue increased by approximately $52.3 million ²⁵, or 17.1% ²⁶, from $306.0 million ²⁷ in 2024 to $358.4 million ²⁸ in 2025, due to increased sales volumes. AGAMREE® net product revenue grew from approximately $46.0 million ²⁹ in 2024 (from its March 2024 launch) to $117.1 million ³⁰ in 2025. Conversely, FYCOMPA® net product revenue decreased by approximately $23.9 million ³¹, or 17.4% ³², from $137.3 million ³³ in 2024 to $113.3 million ³⁴ in 2025, due to generic competition. Cost of sales increased by $18.5 million ³⁵ from $68.8 million ³⁶ in 2024 to $87.3 million ³⁷ in 2025, primarily due to higher royalty payments and product costs associated with increased sales of FIRDAPSE® and AGAMREE®. Operating expenses increased, with selling, general and administrative expenses rising by approximately $16.0 million ³⁸ to $193.8 million ³⁹ in 2025, mainly due to increased employee compensation and consulting fees for business development, partially offset by reduced contributions to patient assistance programs. Research and development expenses remained relatively consistent at approximately $12.7 million ⁴⁰ in 2025. Other income, net, increased by approximately $4.6 million ⁴¹ to $25.7 million ⁴² in 2025, driven by higher invested balances and an increase in the fair value of the investment in Santhera.

Significant operational developments during the period include the FDA approval of an sNDA for FIRDAPSE® in May 2024, increasing the maximum daily dosage to 100 mg. AGAMREE® was commercially launched in March 2024, and its field-based commercial and medical forces were separated into two dedicated units for FIRDAPSE® and AGAMREE® effective April 1, 2025. The National Comprehensive Cancer Network (NCCN) Clinical Practice Guidelines for Small Cell Lung Cancer were updated on August 6, 2025, to include LEMS, amifampridine, and VGCC antibody tests, recommending amifampridine as a treatment. The company settled patent litigation with Inventia Healthcare Limited on July 30, 2024, and with Teva Pharmaceuticals USA, Inc. on January 8, 2025, and Lupin Pharmaceuticals, Inc. on August 26, 2025, regarding FIRDAPSE® generics, with Teva and Lupin agreeing not to market generic versions before February 25, 2035. The patent litigation against Hetero USA, Inc. remains ongoing, with trial scheduled for March 23, 2026. Health Canada approved AGAMREE® for DMD on October 2, 2025, for which KYE Pharmaceuticals, Inc. holds Canadian commercial rights. The company's Board of Directors authorized a new share repurchase program on October 1, 2025, to repurchase up to $200 million ⁴³ of common stock by December 31, 2026, and as of February 23, 2026, 1,740,713 shares ⁴⁴ had been repurchased for approximately $39.9 million ⁴⁵.

The company anticipates that revenues from its sublicense agreements with KYE Pharmaceuticals, Inc. for FIRDAPSE® and AGAMREE® in Canada, and with DyDo Pharma, Inc. for FIRDAPSE® in Japan, will be immaterial in 2026 as distribution ramps up in each jurisdiction. No specific revenue or earnings guidance for the upcoming period was formally issued in the filing.

A major growth area for the company is the continued commercialization of FIRDAPSE® for LEMS and the expansion of disease awareness. This includes efforts to educate physicians treating LEMS patients who also have small-cell lung cancer, leveraging the August 6, 2025, inclusion of LEMS and amifampridine in the NCCN Clinical Practice Guidelines for Small Cell Lung Cancer. The company is actively working to integrate these guidelines into standard oncology practice. Furthermore, the company plans to evaluate FIRDAPSE® for the treatment of other conditions in the future. The personalized treatment support program, Catalyst Pathways®, and patient assistance programs are cornerstones of this strategy, aiming to ensure no LEMS patient is denied access for financial reasons within legal restrictions.

Another significant growth vector is the continued commercialization of AGAMREE® for DMD and the pursuit of additional indications for the product. The company is currently conducting a Phase 1 study in healthy adults to compare single doses of vamorolone, prednisone, and deflazacort, and to define the immunosuppressive dose of AGAMREE® for future indications and its use with gene and cell therapies for DMD that require concurrent immunosuppression. Results from both parts of this study are expected by the end of the second quarter of 2026. The SUMMIT registry study, enrolling up to 250 patients, aims to gather long-term patient safety and quality of life data, potentially demonstrating benefits in areas like stature, bone health, behavior, and cardiovascular health. The recent addition of DMD to the Recommended Uniform Screening Panel by the U.S. Department of Health and Human Services is expected to support earlier detection and timely treatment access. A joint steering committee with Santhera Pharmaceuticals Holding AG is overseeing AGAMREE®'s lifecycle management and development.

Operationally, the company expects selling, general, and administrative expenses to remain substantial in future periods as it continues to sell FIRDAPSE® and AGAMREE® and pursues business expansion. However, these expenses are anticipated to be offset in part by reduced selling, general, and administrative expenses for FYCOMPA® due to the entry of generic competitors. Research and development activities are expected to become more significant in the future as the company seeks to develop additional indications for FIRDAPSE® and AGAMREE® and advances its portfolio expansion efforts. The company is also in the process of transitioning final goods manufacturing for AGAMREE® to a U.S. location, with completion estimated by the end of 2026, and expects to have an additional third-party manufacturer for AGAMREE® before the end of 2026.

Regarding capital allocation, the company's Board of Directors authorized a new share repurchase program on October 1, 2025, to repurchase up to $200 million ⁴⁶ of common stock between October 1, 2025, and December 31, 2026. As of February 23, 2026, the company had repurchased 1,740,713 shares ⁴⁷ for an aggregate purchase price of approximately $39.9 million ⁴⁸. The company intends to use existing cash on hand to fund this program and believes it can do so without impairing its business development strategy. The company does not anticipate paying cash dividends on its common stock in the foreseeable future.

The company explicitly flagged several structural headwinds and execution risks to its growth plan. These include the uncertainty of prevailing in the ongoing Paragraph IV litigation with Hetero USA, Inc. regarding FIRDAPSE® patents, which could allow a generic version to market before February 25, 2035. For AGAMREE®, there is a risk that DMD patients transitioning to gene therapy treatments may delay or stop AGAMREE® use, and uncertainty exists regarding whether steroids will remain the foundational standard of care for DMD as new drugs are approved. For FYCOMPA®, reduced revenue is expected due to generic competition following the expiration of the '571 patent on May 23, 2025, and the '497 patent on July 1, 2026, potentially requiring impairment of the intangible asset for FYCOMPA®. The company also faces risks related to the accuracy of its forecasts for FYCOMPA® sales in a generic market.

Geographic, regulatory, and macro factors identified as constraints include the impact of tariffs on the cost of sales for products manufactured outside the U.S., and the potential impact of "Most Favored Nation" pricing, such as proposed in the GUARD and GLOBE Medicare demonstration projects, on net product revenues. Changes in healthcare industry regulations, including those from the Inflation Reduction Act of 2022 and potential future changes by the current Administration (e.g., Most Favored Nation pricing or 100% tariffs on branded pharmaceuticals without U.S. manufacturing), could impact drug pricing and reimbursement. The company also notes the potential for a prolonged U.S. government shutdown to impact its business.

The company faces material risks including intense competition from pharmaceutical and biotechnology companies with greater resources, and the potential for generic competition upon loss of exclusivity or patent protection for its products. Tariffs, such as those signaled by the Trump Administration for up to 100% ⁴⁹ on imported branded or patented pharmaceutical products, could adversely affect the business by increasing costs for API and finished drug products sourced from outside the U.S., and retaliatory tariffs could impact international sales. The successful commercialization of products is highly dependent on adequate reimbursement and pricing policies from third-party payors, with significant uncertainty around new legislation like the Inflation Reduction Act of 2022 and potential Most Favored Nation (MFN) pricing models (GENEROUS, GLOBE, GUARD) that could reduce net product revenues. Given the small target patient populations for FIRDAPSE® and AGAMREE®, the company must achieve significant market penetration and high per-patient prices to maintain meaningful gross margins, and patient discontinuation rates higher than forecast could negatively impact revenues. The ongoing Paragraph IV patent litigation with Hetero USA, Inc. for FIRDAPSE® patents expiring in 2032, 2034, and 2037, scheduled for trial on March 23, 2026, presents a significant legal and financial risk, as an unfavorable outcome could allow generic market entry prior to February 25, 2035. For AGAMREE®, there is a risk that new gene therapy treatments for DMD could delay or stop AGAMREE® use, and that steroids may not remain the foundational standard of care. The company is highly dependent on third-party suppliers and contract manufacturers for its products, and any failure to comply with cGMP or supply disruptions could cause significant delays and lost sales. Cybersecurity threats, including ransomware and phishing attacks, pose a risk of system failures, data breaches, and disruption to product development programs, potentially leading to liability and increased costs.

Management's overall tone is one of patient-centricity and strategic expansion, emphasizing a commitment to providing innovative, best-in-class medications for rare and difficult-to-treat diseases. They highlight the importance of their current commercial-stage products, FIRDAPSE®, AGAMREE®, and FYCOMPA®, while actively seeking to broaden and diversify the product portfolio through immediate or near-term accretive acquisitions in rare disease therapeutic categories. Management explicitly states that they believe they have sufficient funds to support operations for at least the next 12 months ⁵⁰ from the date of the report, absent the use of cash for potential business development opportunities. They also note that they can execute the recently authorized share repurchase program of up to $200 million ⁵¹ without impairing the advancement of their business development strategy. The three strategic priorities emphasized for the period ahead are: first, to continue commercializing FIRDAPSE® for LEMS and improving disease awareness, including evaluating it for other conditions; second, to continue commercializing AGAMREE® for DMD, seeking to develop additional indications, and conducting the SUMMIT registry study for long-term data; and third, to actively seek to acquire additional drug products, focusing on clinically differentiated, de-risked opportunities in rare disease therapeutic areas.