Core Insights - Zevra Therapeutics, Inc. (NASDAQ:ZVRA) is highlighted as a promising investment opportunity in the biotech sector, particularly due to its recent positive data regarding MIPLYFFA® (arimoclomol) for Niemann-Pick Disease Type C [1][2] - The company focuses on developing therapies for rare diseases, specifically targeting conditions with limited treatment options, including Idiopathic Hypersomnia and Urea Cycle Disorders [2] Group 1 - On September 4, Zevra Therapeutics announced new data supporting the effectiveness of MIPLYFFA® in treating Niemann-Pick Disease Type C at the International Congress of Inborn Errors of Metabolism [1] - A pre-specified analysis indicated that patients on concomitant miglustat who switched from placebo to MIPLYFFA experienced a decline in annual disease progression [2] - Zevra Therapeutics is a commercial-stage company with a product portfolio that emphasizes rare neurological and sleep conditions [2]

Core Insights - Zevra Therapeutics, Inc. announced the presentation of four posters on MIPLYFFA® (arimoclomol) at the International Congress of Inborn Errors of Metabolism (ICIEM) in Kyoto, Japan, from September 2-6, 2025 [1][2] Product Overview - MIPLYFFA is approved in the U.S. for treating Niemann-Pick disease type C (NPC) and has shown a unique mechanism of action targeting the underlying pathophysiology of NPC [2][6] - The drug received FDA approval on September 20, 2024, and has demonstrated the ability to halt disease progression over a one-year trial compared to placebo, as measured by the NPC Clinical Severity Scale [6] Clinical Data Presentation - A poster detailing the safety and efficacy of arimoclomol in a pediatric substudy for patients aged 6 to <24 months will be presented on September 3, 2025 [3] - A poster selected for a Best Poster award will present long-term outcomes from an expanded access program in the U.S. on September 4, 2025 [4] - Additional presentations will include efficacy results from a 12-month double-blind randomized trial and an open-label extension phase for patients treated with miglustat [4] Regulatory and Market Position - MIPLYFFA has received Orphan Medicinal Product designation by the European Medicines Agency (EMA) for NPC treatment, and a Marketing Authorization Application is currently under review by the EMA [6] - The extensive clinical development program for MIPLYFFA includes data from over 270 NPC patients worldwide, showcasing long-term clinical outcomes [6] Company Mission and Strategy - Zevra Therapeutics focuses on developing therapies for rare diseases with limited treatment options, aiming to bring life-changing therapeutics to affected individuals [17]