Zevra Therapeutics announces publication of results on Phase 2/3 NPC-002

Zevra Therapeutics (ZVRA) announced the publication of “Long-term Efficacy and Safety of Arimoclomol in Niemann-Pick Disease Type C: Final Results of the Phase 2/3 NPC-002 48-month Open-label Extension Trial” in the peer-reviewed journal, Molecular Genetics and Metabolism. This paper presents long-term efficacy and safety outcomes for NPC patients treated with MIPLYFFA in the 48-month open-label extension phase following the end of the 12-month, pivotal, placebo-controlled, double-blind, Phase 2/3 trial. Efficacy was calculated using the 5- and rescored 4-domain NPC Clinical Severity Scale, the only validated measurement of NPC progression. The data from the OLE phase showed a sustained reduction in disease progression for at least 5 years in a heterogeneous population of NPC patients receiving MIPLYFFA in addition to routine clinical care, with no new safety concerns. These results align with the pivotal Phase 2/3 trial, which showed that MIPLYFFA halted disease progression compared to placebo over the one-year duration.