Catalyst Pharmaceuticals, Inc.

“We are thrilled to welcome Dr. Sundaram to our Board of Directors. Her recognized expertise in the pharmaceutical industry and strong leadership qualities perfectly align with our long-term growth strategy. We are confident that her contributions will be instrumental in achieving our goal of advancing early detection and precision treatment of hearing disorders” said Celia Belline, CEO of Cilcare. “We are thrilled to welcome Dr. Sundaram to our Board of Directors. Her recognized expertise in the pharmaceutical industry and strong leadership qualities perfectly align with our long-term growth strategy. We are confident that her contributions will be instrumental in achieving our goal of advancing early detection and precision treatment of hearing disorders” said Celia Belline, CEO of Cilcare. Dr. Sundaram also expressed her enthusiasm for joining the company: “It is an honor to join Cilcare at such a pivotal time in its journey. The company has shown remarkable progress in recent years and is ideally positioned to leverage its established expertise and capabilities to become a key player in hearing care, serving patients worldwide. I look forward to collaborating with the Board and executive team to support the implementation of the company’s strategic growth plans.” “It is an honor to join Cilcare at such a pivotal time in its journey. The company has shown remarkable progress in recent years and is ideally positioned to leverage its established expertise and capabilities to become a key player in hearing care, serving patients worldwide. I look forward to collaborating with the Board and executive team to support the implementation of the company’s strategic growth plans.” Dr. Preethi Sundaram joined Cilcare’s Board of Directors on December 2, 2024. Over the course of her 25-year career in the pharmaceutical industry, she has overseen the development and launch of several essential medical treatments. Since 2021, she has served as Chief Strategy Officer at Catalyst Pharmaceuticals, where she leads the company’s growth strategy with a focus on business development, new product acquisitions, portfolio expansion, and product lifecycle management. Prior to joining Catalyst, Dr. Sundaram held leadership positions at Sanofi starting in 2005, across various therapeutic areas and business units. She served in various leadership roles, including Global Clinical Research Director, Global Project Head, and Global Head, Medical Operations for the General Medicines business unit. She also held positions at Abbott Laboratories, Covance, and Neurovision prior to her tenure at Sanofi. Dr. Sundaram holds several prestigious degrees, including a Bachelor of Science in Optometry from the Elite School of Optometry & BITS (India), a Bachelor of Arts in Psychology from the University of Madras (India), a Doctorate in Optometry from Anglia Ruskin University (UK), and an Executive Business Master’s from the London Business School. With this appointment, Cilcare strengthens its commitment to transforming hearing care and improving the quality of life for patients with hearing disorders. Press contact Marie Peytavy, Marketing & Communication Director: marie.peytavy@cilcare.com About Cilcare Cilcare is a biotechnology company specializing in auditory sciences, developing cutting-edge solutions for the characterization, diagnosis, and treatment of hearing disorders and associated diseases. Founded by three visionary entrepreneurs, the company now employs a team of 45 international collaborators, supported by a scientific advisory board. Since its creation in 2014, Cilcare has dedicated itself to addressing these global challenges by combining an advanced R&D platform, a promising drug candidate portfolio, and the use of artificial intelligence and machine learning to characterize various forms of hearing loss. For the past 10 years, Cilcare has also made its technology available to industry leaders and researchers in Europe, the United States, and Asia to accelerate the development of drugs, gene and cell therapies, and medical devices for hearing disorders. Link to Press Release (French Version) Link to Press Release (English version)

Santhera’s Agamree is indicated for patients aged four years and older with Duchenne muscular dystrophy (DMD). Credit: JohnnyGreig via Getty Images. Swiss biotech Santhera Pharmaceuticals has started launch preparations for its Duchenne muscular dystrophy (DMD) drug Agamree (vamorolone) in the UK, following a recommendation from England’s price watchdog. Approved for use in the National Health Service (NHS) in England, Wales, and Northern Ireland, Agamree is indicated for patients aged four years and older with DMD. The company expects the first sales to commence within this quarter. DMD is a rare progressive genetic disorder that primarily affects boys. It causes muscle weakness and degeneration, leading to loss of mobility and premature death. Standard treatment typically involves corticosteroids to manage inflammation and preserve muscle strength, but these therapies are associated with significant side effects like growth stunting and bone health issues. England’s National Institute for Health and Care Excellence (NICE) issued a positive final guidance for Agamree on 16 January 2025, following confirmation that no appeals were submitted against its December 2024 final draft guidance . This represents a reversal of NICE’s initial decision in March 2024, when the agency declined to recommend the drug due to concerns about its cost-effectiveness compared to standard-of-care corticosteroids. Subsequent submissions from Santhera addressed these issues, with NICE acknowledging that Agamree may offer fewer side effects than current options. Agamree is a first-in-class anti-inflammatory drug that binds to the same receptor as glucocorticoids but modulates its downstream activity differently, reducing the risk of corticosteroid-associated toxicities. The drug was designed as an alternative to traditional corticosteroids to mitigate side effects such as stunted growth and bone metabolism issues. The pivotal VISION-DMD study (NCT03439670) demonstrated that boys treated with Agamree maintained normal bone growth patterns and showed no signs of reduced bone metabolism or mineralisation after 48 weeks, unlike those treated with the corticosteroid prednisone. Patients transitioning from corticosteroids to Agamree reportedly recovered growth and bone health while maintaining efficacy. Agamree received UK regulatory approval in January 2024, building on similar decisions by the US Food and Drug Administration (FDA) and European Commission (EC) in October 2023 and December 2023, respectively. While it is approved in the same indication in Europe, patients as young as two years old in the US have access to the treatment. Santhera sold US commercialisation rights for Agamree to Catalyst Pharmaceuticals in July 2023 in a deal worth up to $231m. According to GlobalData’s Pharma Intelligence Center, Agamree is forecast to generate up to $373m in 2030. GlobalData is the parent company of Pharmaceutical Technology. Other approved treatments for DMD include Sarepta’s gene therapy Elevidys (delandistrogene moxeparvovec-rokl). Initially approved via the accelerated pathway in 2023 for ambulatory boys aged between four and five years, Elevidys’ label was expanded in June 2024 to all DMD patients over four years of age with confirmed DMD mutations. Among others in the same space, US company Regenxbio is also planning a biologics licence application (BLA) filing in 2026 for RGX-202, its investigational DMD gene therapy, following positive data from the Phase I/II portion of its AFINITY DUCHENNE trial (NCT05693142).

Catalyst Pharmaceuticals’ controversial $375,000 rare autoimmune disease drug now won’t face generic competition from Teva until Feb. 25, 2035, thanks to a patent settlement announced by the two companies. The settlement follows Florida-based Catalyst’s suit against Teva for attempting to launch a generic of Firdapse, a treatment for Lambert-Eaton myasthenic syndrome or LEMS, ahead of certain key patent expirations. Other patent-related litigation with India-based generic drugmakers Hetero and Lupin is ongoing, Catalyst said in a Wednesday statement announcing the settlement. The company’s stock $CPRX rose more than 16% when the market closed on Wednesday. Firdapse was first approved in 2018 by the FDA, and pulled in about $258 million in 2023 for Catalyst. In 2021, Catalyst won a lawsuit against the FDA that cut short the approval of a Firdapse copycat. Catalyst claimed that its orphan exclusivity from being first to market should have barred the approval for Jacobus Pharmaceutical’s Ruzurgi, and the court ended up agreeing with Catalyst despite the FDA’s objections. In documents released as part of that Catalyst-FDA suit , Catalyst even went so far as to show how although the FDA, which says publicly that it can’t consider drug prices as part of its analysis of approvals and exclusivities, did consider the $375,000 annual price of Firdapse when approving Ruzurgi, which had a list price that was about $200,000 lower annually.