Fenfluramine Hydrochloride

Phase 3 study met primary and most key secondary clinical endpoints This marks the third developmental and epileptic encephalopathies (DEE) population for fenfluramine with positive phase 3 results Results underscore the impact of UCB's continued investment in scientific innovation to advance new treatments for DEEs CDKL5 deficiency disorder (CDD) is an ultra-rare, severe DEE with an onset in early infancy with a high unmet need, and has limited treatment options CDD affects approximately 1 in 40,000 to 60,000 live births, with a median age of onset of six weeks1,2 UCB plans to submit for regulatory approval to bring this potential treatment option to people living with CDD as soon as possible ATLANTA, June 27, 2025 /PRNewswire/ -- UCB, a global biopharmaceutical company, today announced that the Phase 3 study investigating the safety and efficacy of adjunctive fenfluramine in CDKL5 deficiency disorder (CDD) met its primary and most key secondary endpoints.3 The study is a randomized, double-blind, placebo-controlled, fixed-dose, multi-center study examining the efficacy and safety of adjunctive fenfluramine treatment in 87 individuals aged one through 35, with a CDD diagnosis and uncontrolled seizures.4 "These results pave the way for creating significant therapeutic progress and represent an important milestone in UCB's mission to bring meaningful innovation to individuals and families affected by developmental and epileptic encephalopathies (DEEs). We are grateful to the patients, families, and researchers who made this progress possible, and we look forward to working with the health authorities to make treatment available as soon as possible," said Fiona du Monceau, Executive Vice President, Patient Evidence, UCB. The primary endpoint of the study is based on the median percent change in countable motor seizure frequency (CMSF) between baseline and the titration plus maintenance phase, comparing fenfluramine with the placebo group.3 Full results will be presented at an upcoming scientific meeting. CDD is an ultra-rare DEE with refractory infantile-onset epilepsy and severe global neurodevelopmental delays resulting in intellectual, motor, cortical visual, and sleep impairments as major features.1 It is caused by pathogenic variants in the cyclin dependent kinase-like 5 (CDKL5) gene located on the X chromosome. It is estimated that CDD affects approximately 1 in 40,000 to 60,000 live births.2 In the study, fenfluramine was generally well tolerated, and the safety profile was consistent with previous studies in DS/LGS.3 UCB is currently conducting an open-label, flexible-dose, long-term 54-week extension phase of the study to characterize the long-term safety and tolerability of fenfluramine in pediatric and adult individuals with CDD.4 In the United States, fenfluramine oral solution is indicated for the treatment of seizures associated with Dravet syndrome and Lennox-Gastaut syndrome in patients 2 years of age and older.5 It is not approved for use in CDD by any regulatory authority worldwide. For further information, contact UCB: US Communications Becky Malone T +1.919.605.9600 [email protected] Corporate Communications, Media Relations Laurent Schots T +32.2.559.92.64 [email protected] Investor Relations Antje Witte T +32.2.559.94.14 [email protected] Sahar Yazdian T +32.2.559.91.37 [email protected] About UCB UCB, Brussels, Belgium () is a global biopharmaceutical company focused on the discovery and development of innovative medicines and solutions to transform the lives of people living with severe diseases of the immune system or of the central nervous system. With approximately 9,000 people in approximately 40 countries, the company generated revenue of € 6.15 billion in 2024. UCB is listed on Euronext Brussels (symbol: UCB). Follow us on Twitter: @UCB_news. 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UCB's efforts to acquire other products or companies and to integrate the operations of such acquired companies may not be as successful as UCB may have believed at the moment of acquisition. Also, UCB or others could discover safety, side effects or manufacturing problems with its products and/or devices after they are marketed. The discovery of significant problems with a product similar to one of UCB's products that implicate an entire class of products may have a material adverse effect on sales of the entire class of affected products. Moreover, sales may be impacted by international and domestic trends toward managed care and health care cost containment, including pricing pressure, political and public scrutiny, customer and prescriber patterns or practices, and the reimbursement policies imposed by third-party payers as well as legislation affecting biopharmaceutical pricing and reimbursement activities and outcomes. Finally, a breakdown, cyberattack or information security breach could compromise the confidentiality, integrity and availability of UCB's data and systems. Given these uncertainties, the public is cautioned not to place any undue reliance on such forward-looking statements. These forward-looking statements are made only as of the date of this document, and do not reflect any potential impacts from the evolving event or risk as mentioned above as well as any other adversity, unless indicated otherwise. The company continues to follow the development diligently to assess the financial significance of these events, as the case may be, to UCB. UCB expressly disclaims any obligation to update any forward-looking statements in this document, either to confirm the actual results or to report or reflect any change in its forward-looking statements with regard thereto or any change in events, conditions or circumstances on which any such statement is based, unless such statement is required pursuant to applicable laws and regulations. References 1 Zuberi et al. ILAE classification and definition of epilepsy syndromes with onset in neonates and infants: Position statement by the ILAE Task Force on Nosology and Definitions. Epilepsia.2022;63(6):1349-97. 2 Overview of Medical-scientific Research in the Netherlands (OMON). A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Fixed-Dose, Multicenter Study to Examine the Efficacy and Safety of ZX008 in Subjects with CDKL5 Deficiency Disorder Followed by an Open-Label Extension. 2022. Available at: . Accessed: June 2025. 3 UCB data on file. 2025 4 ClinicalTrials.gov. NCT05064878. Available at: . Accessed: June 2025. 5 FINTEPLA (fenfluramine) U.S. prescribing information. Smyrna, GA: UCB, Inc. . Accessed June 2025. FINTEPLA® is a registered trademark of the UCB Group of Companies. ©2025 UCB, Inc., Smyrna, GA 30080. All rights reserved. US-FA-2500517 Date of preparation: June 2025 SOURCE UCB WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Renowned Neurologist and Epilepsy Advocate Honored for Decades of Innovation in Therapeutics, Research, and Patient Care BOWIE, Md., May 14, 2025 (GLOBE NEWSWIRE) -- Internationally acclaimed neurologist and epilepsy researcher Dr. Orrin Devinsky will be honored with the Lifetime Accelerator Award during the Epilepsy Therapies & Diagnostics Development Symposium in Leesburg, Va. on Thursday, May 29, 2025. This award is given in recognition of Dr. Devinsky’s groundbreaking work advancing epilepsy treatment, pioneering therapeutic development, and his unwavering dedication to improving the lives of people with epilepsy. Each year, the Lifetime Accelerator Award is presented to a physician, scientist, industry leader, or other individual who has demonstrated a lifelong commitment to bringing new therapies to people with epilepsy. Lifetime Accelerator Award recipients are chosen by an independent committee of global thought leaders and clinical investigators in epilepsy and seizure therapy discovery and development. Dr. Devinsky has been a trailblazer in the development of new therapies for epilepsy, most notably serving as the Principal Investigator of the pivotal Phase III cannabidiol (CBD) trials for Dravet Syndrome and Lennox-Gastaut Syndrome. Both published in the New England Journal of Medicine, these trials were instrumental in demonstrating the efficacy of CBD in reducing seizures, a milestone that helped change the therapeutic landscape for drug-resistant epilepsies. His research has also centered on high-need areas in the epilepsy community, including therapeutic evolution for developmental and epileptic encephalopathies. His work was pivotal in advancing lorcaserin and fenfluramine He is a recognized leader in epilepsy mortality prevention, having founded and served as Principal Investigator of the North American SUDEP (Sudden Unexplained Death in Epilepsy) Registry. Additionally, he co-developed the QOLIE (Quality of Life in Epilepsy), a standardized and widely adopted tool for assessing the impact of epilepsy beyond seizures. Dr. Devinsky has authored over 700 peer-reviewed journal articles and co-founded the Epilepsy Therapy Project and Epilepsy.com, the largest online platform for epilepsy information and support. He is also the author of Epilepsy: Patient and Family Guide, a widely used resource for families navigating epilepsy care. In addition to his clinical and academic work, Dr. Devinsky founded FACES (Finding A Cure for Epilepsy and Seizures), which has supported numerous early-stage and “n-of-1” studies to bring promising treatments for rare and complex neurological disorders to the forefront. His research has received support from the Epilepsy Foundation, National Institutes of Health, National Science Foundation, Centers for Disease Control, Department of Defense, Simons Foundation and other organizations. He has played a key role in building and leading large-scale initiatives. “I’m deeply honored to receive the Lifetime Accelerator Award,” said Dr. Devinsky. “This recognition reflects the collective efforts of the incredible teams, families, researchers, and patients I’ve had the privilege to work with throughout my career. Advancing care for people with epilepsy has been the mission of my life, and I remain as committed as ever to pushing the boundaries of what’s possible in neurological care.” Dr. Devinsky’s distinguished academic and clinical career spans decades of leadership and innovation. A Yale University graduate (B.S., M.S., magna cum laude), he earned his M.D. from Harvard University (cum laude) before completing prestigious fellowships at the NIH’s National Institute of Neurological Disorders and Stroke (NINDS). He is board-certified in Neurology and Clinical Neurophysiology and has served numerous academic and hospital leadership roles, including Director of the NYU Comprehensive Epilepsy Center since 1989. Throughout his career, Dr. Devinsky has held influential positions in professional societies such as the American Epilepsy Society and American Academy of Neurology, serving on committees, advisory boards, and editorial boards for over three decades. He remains a trusted voice across academia, biotech, and public health. The Lifetime Accelerator Award acknowledges his exceptional impact and enduring legacy in the field of neuroscience and beyond. To watch Dr. Devinsky receive his Lifetime Accelerator Award, register for the livestream of the Foundation’s Shark Tank Competition. About EpilepsyEpilepsy is considered by world experts to be the most common serious brain disorder worldwide with no age, racial, social class, national or geographic boundaries. The U.S. Centers for Disease Control & Prevention (CDC) estimates that 3.4 million people in the United States are affected by epilepsy. Epilepsy is the underlying tendency of the brain to produce seizures which are sudden abnormal bursts of electrical energy that disrupt brain functions. About the Epilepsy Foundation of AmericaThe Epilepsy Foundation is a national nonprofit organization dedicated to improving the lives of people affected by epilepsy through education, advocacy, research, and connection. For more than five decades, the Epilepsy Foundation has shone a light on epilepsy to promote awareness and understanding, and to advocate for laws that matter to people with epilepsy, while also funding epilepsy research and supporting epilepsy investigators and specialists in their early careers. In partnership with the CDC, the Epilepsy Foundation has helped to improve access to care for people with epilepsy and trained more than 600,000 people in seizure recognition and first aid. The Epilepsy Foundation continues to focus on serving the epilepsy community through advocacy, education, direct services, and research for new therapies. To learn more visit epilepsy.com or call 1.800.332.1000; in Spanish at 866.748.8008 or laepilepsia.org. Follow us on Facebook, Instagram, Twitter, LinkedIn, TikTok, and YouTube. MEDIA CONTACT: Kaitlyn Gallagher 301.918.3756 kgallagher@efa.org