Press release
Zevra Therapeutics Announces Arimoclomol Research Featured in Two Poster Presentations at the 19th Annual WORLDSymposium™ 2023
Interim data analysis of four-year, open-label extension from Phase 2/3 clinical trial suggests arimoclomol may reduce long-term progression of Niemann-Pick
About this update from Zevra Therapeutics, Inc.
[{"type":"text","content":"Interim data analysis of four-year, open-label extension from Phase 2/3 clinical trial suggests arimoclomol may reduce long-term progression of Niemann-Pick Disease Type C (NPC) CELEBRATION, Fla., Feb. 24, 2023 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: KMPH) (“Zevra” or the “Company,” formerly known as KemPharm, Inc.), today announced that arimoclomol, the Company’s orally-delivered, first-in-class investigational product candidate being developed as a treatment for Niemann-Pick disease type C (NPC), is being featured in two poster presentations during the 19th Annual WORLDSymposium™ 2023, the annual research conference dedicated to lysosomal diseases being held February 22-26, 2023 in Orlando, Florida, USA. Research presented at WORLDSymposium™ 2023 included an interim analysis from the ongoing four-year open-label extension of the Phase 2/3 clinical trial of arimoclomol. Results from this analysis, based on up to four years of continuous treatment, suggest that arimoclomol may reduce the long-term progression of NPC. Zevra plans to include these data as part of the updated New Drug Application (NDA) for arimoclomol, which the Company expects to resubmit to the U.S. Food and Drug Administration (FDA) as early as the third quarter of 2023. “We are pleased to share this important research of arimoclomol at WORLDSymposium™ 2023, which expands our understanding of the potential for arimoclomol to decrease the long-term progression of NPC,” said Travis C. Mickle, Ph.D., President of Zevra. “We are particularly encouraged by the top-line efficacy data from the four-year open label study of arimoclomol given the significant need to develop improved therapies to treat NPC, a devastating lysosomal disease characterized by progressive impairment of mobility, cognition, speech and swallowing before a premature death. We look forward to including these data which further characterize the potential clinical benefit of arimoclomol in NPC, along with a 48-month safety analysis, in our updated NDA for arimoclomol.” \"I am encouraged that the extension studies have provided further data supporting a beneficial effect of arimoclomol in patients with Niemann-Pick disease, type C, for whom safe and effective therapies are urgently needed,” said Marc C. Patterson, M.D., Neurology, Pediatrics and Medical Genetics, Mayo Clinic, Rocheste...