Zevra Publishes Promising Phase 2/3 Data For MIPLYFFA In Treating Niemann-Pick Type C
(RTTNews) - Zevra Therapeutics Inc. (ZVRA), a commercial-stage company, on Tuesday announced publication of encouraging Phase 2/3 data for MIPLYFFA in treating Niemann-Pick type C disease in pediatric patients of two years or younger. The results were published in the journal of Molecular Genetics and Metabolism Reports.
Niemann-Pick disease is a rare congenital disorder that affects the metabolism of fats in the body, and may impact the brain, nerves, liver, spleen, and bone marrow. Patients are usually diagnosed as infants, but those of Niemann-Pick type C (NPC) may not show symptoms until adulthood. The disease has no known cure and may be fatal.
The drug MIPLYFFA (arimoclomol) was approved for the treatment of Niemann-Pick disease by the U.S. Food and Drug Administration (FDA) in September 2024. The drug activates the E3 and EB transcription factors to upregulate the CLEAR genes. This can lead to the removal of unesterified cholesterols from fibroblast lysosomes, halting progression of the disease. MIPLYFFA was granted Orphan Medicinal Product designation by the European Medicines Agency (EMA).
MIPLYFFA was administered to five NPC patients in a pediatric sub study that was part of the Phase 2/3 open-label extension (OLE) trial. Patients were between 12 to 24 months at the time of enrollment and were dosed with arimoclomol with concomitant miglustat over 36 months to assess the drug's safety, tolerability, and pharmacokinetics.
The published data indicates that MIPLYFFA was generally well-tolerated and showed a good safety profile. Development assessments determined that there were variable delays in patients, implying an underlying phenotypic heterogenicity.
A marketing authorization application (MAA) was filed with the EMA to clear arimoclomol as a treatment for Niemann-Pick disease.
ZVRA closed Tuesday at $13, up 2.77%. In after-hours, the stock is trading at $12.93, down 0.54%.
