Zevra Therapeutics presents new data on MIPLYFFA

Zevra Therapeutics (ZVRA) announced the presentation of several posters highlighting new positive data on MIPLYFFA for the treatment of Niemann-Pick disease type C, NPC, at the International Congress of Inborn Errors of Metabolism, ICIEM. In a poster titled “Arimoclomol upregulates expression of genes belonging to the coordinated lysosomal expression and regulation network,” it was demonstrated that MIPLYFFA activates transcription factors leading to the amplification of NPC1 protein levels and more successful NPC1 processing. In a poster 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 titled “Safety and efficacy of arimoclomol in a pediatric substudy of Niemann-Pick disease type C patients aged 6 to less than24 months at study enrollment,” data evaluating five patients for up to 36 months of treatment showed that MIPLYFFA was well tolerated in children in this age group with no new safety signals observed. In a poster titled “Arimoclomol for the treatment of Niemann-Pick disease type C in a real-world setting: long-term outcomes from an expanded access program in the United States,” the effectiveness and safety of MIPLYFFA for the treatment of NPC, including with and without miglustat as a component of routine clinical care, were further confirmed and were consistent with the benefits demonstrated in the pivotal, double-blind, placebo-controlled Phase 3 study.