Zevra Therapeutics Presents Positive New Data Supporting Foundational Role of MIPLYFFA® (arimoclomol) for the Treatment of Niemann-Pick Disease Type C at the International Congress of Inborn Errors of Metabolism (ICIEM)

New data from pre-specified analysis shows patients on concomitant miglustat who switched from placebo to MIPLYFFA experienced a decline in annual disease progression

Nomination for Best Poster Award received for poster highlighting MIPLYFFA’s differentiated mechanism of action targets underlying pathology of NPC

CELEBRATION, Fla., Sept. 04, 2025 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company), a commercial-stage company focused on providing therapies for people living with rare disease, today announced the presentation of several posters highlighting new positive data on MIPLYFFA® (MY-PLY-FAH) (arimoclomol) for the treatment of Niemann-Pick disease type C (NPC) at the International Congress of Inborn Errors of Metabolism (ICIEM).

“The efficacy and safety of MIPLYFFA have been substantiated by the most extensive clinical dataset currently available for patients with NPC,” said Adrian Quartel, M.D., FFPM, Zevra’s Chief Medical Officer. “Our commitment to the NPC community drives our ongoing efforts to publish additional data demonstrating the impact of MIPLYFFA across a heterogeneous population of NPC patients, including new findings in patients under the age of two and in patients who transitioned to MIPLYFFA after previously receiving placebo in our pivotal double-blind trial. We are also pleased that our poster detailing the pathways by which MIPLYFFA targets the underlying pathophysiology of NPC has been nominated for the Best Poster Award by ICIEM.”

Data Highlights

In a poster (BP-19) titled “Arimoclomol upregulates expression of genes belonging to the coordinated lysosomal expression and regulation (CLEAR) network,” it was demonstrated that MIPLYFFA activates transcription factors leading to the amplification of NPC1 protein levels and more successful NPC1 processing; thereby addressing the underlying NPC etiology through multiple mechanistic pathways.

In a poster (P-264) titled “Efficacy results across a 12-month double-blind randomized trial and an open-label extension phase of arimoclomol for the treatment of Niemann-Pick disease type C in patients treated with miglustat,” data demonstrated that patients who switched from placebo in the double-blind phase to MIPLYFFA in the open-label extension phase, while on continued concomitant miglustat treatment, experienced a decline in annual disease progression.

In a poster (P-261) titled “Safety and efficacy of arimoclomol in a pediatric substudy of Niemann-Pick disease type C patients aged 6 to