Zevra Therapeutics announces presentations on MIPLYFFA at NNPDF conference

Zevra Therapeutics (ZVRA) announced an oral presentation and two poster presentations on MIPLYFFA are being featured at the National Niemann Pick Disease Foundation Conference, taking place July 10-13, 2025, in Concord, North Carolina. The presentations review data on MIPLYFFA, the first treatment approved by the U.S. Food and Drug Administration for the treatment of Niemann-Pick disease type C. MIPLYFFA is indicated for use in combination with miglustat for the treatment of neurological manifestations of NPC in adult and pediatric patients 2 years of age and older. Title: Advances in Niemann-Pick Disease Type C Treatment: The Role of Arimoclomol. Title: Long-Term Effectiveness and Safety Evaluation of Arimoclomol Treatment in Patients With Niemann-Pick Disease Type C – Data From the Pivotal Study and Open-Label Extension: Summary: In the pivotal trial, arimoclomol in combination with miglustat halted disease progression through 12 months compared with placebo as measured by the R4DNPCCSS. The effectiveness and safety of arimoclomol with miglustat was further confirmed in a 48-month open-label extension. Arimoclomol was generally well tolerated with no new safety signals observed during the OLE. Title: Arimoclomol Upregulates Expression of Genes Belonging to the Coordinated Lysosomal Expression and Regulation Network: Summary: The presented in vitro data provide mechanistic evidence of how arimoclomol can target NPC through multiple mechanistic pathways making it relevant in NPC. Increased translocation of the transcription factors TFE3 and TFEB from the cytosol to the nucleus is a crucial step that results in upregulation of a series of downstream processes that may improve lysosomal function and cell viability. Overall, the data support that arimoclomol does not only upregulate expression of certain CLEAR genes and specifically NPC1 at the transcriptional level, but also that this overexpression results in amplification of NPC1 protein levels and more successful NPC1 processing ultimately leading to increased cholesterol clearance from the lysosomal compartments. The effects of arimoclomol in mutant NPC cells found across the in vitro studies are consistent, and downstream effects expected to result from the activation of a specific process in one study could be confirmed in another study to provide an understanding of the mechanism of action of arimoclomol.