Zevra Therapeutics Presents New Data on the Long-Term Safety and Efficacy of Arimoclomol as a Treatment for Niemann-Pick Disease Type C at the SIMD 45th Annual Meeting

The oral presentation highlighted data from Zevra’s Expanded Access Program demonstrating a consistent safety profile

CELEBRATION, Fla., April 15, 2024 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company), a rare disease therapeutics company, today announced that new long-term real-world data from the arimoclomol expanded access program (EAP: NCT04316637) for the treatment of Niemann-Pick disease type C (NPC) was featured in an oral presentation during the Society for Inherited Metabolic Disorders (SIMD) 45^th Annual Meeting taking place at the Sheraton/Le Meridien Charlotte Hotel Complex in Charlotte, NC, from April 14-17, 2024.

“These real-world data collected in the arimoclomol US EAP represent the first evidence to support the use of arimoclomol in adults with NPC, demonstrating a clinically meaningful slowing of disease progression,” noted Kristina Julich, M.D., Assistant Professor, Department of Neurology, at The University of Texas at Austin, Dell Medical School. “NPC presents a therapeutic challenge, lacking approved treatment options in the U.S. and invariably leading to progressive loss of independence due to physical and cognitive impairments. We are very pleased with the positive new data that brings hope for a community with a high unmet medical need.”

“These observed long-term clinical benefits add to the body of evidence for arimoclomol that supports its tolerability and effectiveness in the treatment of NPC, bringing us one step closer to helping patients suffering from this relentless and fatal disease,” remarked Neil F. McFarlane, President and Chief Executive Officer of Zevra. “We remain committed to continue collaborating closely with the FDA as it reviews arimoclomol's NDA ahead of our PDUFA date on September 21, 2024.”

Highlights from the Presentation:

The results were presented by Kristina Julich, M.D., Chief, Neurogenetics Center, and Assistant Professor of Pediatric Neurosciences at Dell Medical School from The University of Texas.

The first patient in the EAP was enrolled in June 2020. As of July 19, 2023, 41 participants from 11 U.S. centers were ≥18 years at arimoclomol initiation; 26 had baseline Physician-reported 5-domain NPC Clinical Severity Scale (5DNPCCS) assessments with ≥1 year of follow-up. Among these 26 adults, mean (standard deviation, SD) age at NPC diagnosis and arimoclomol initiation was 23.7 (9.0) and 28.5 (6.5) years, respectively. Patients continued arimoclomol treatment for a mean of 21 months (range: 12-32) and 69% (18/26) had recorded miglustat use. Adults treated with arimoclomol, including those with and without miglustat use, generally had a stable disease course over two years of treatment and follow-up and the safety profile was consistent with that observed in the Phase 2/3 study where no new safety adverse events were identified.