Rafael Holdings Announces Continuation of its Phase 3 Study for the Treatment of Niemann-Pick Disease Type C1 (NPC1) Following Independent Data Monitoring Committee (DMC) Review of Prespecified 48-Week Interim Data
Provided By GlobeNewswire
Last update: Jun 18, 2025
Phase 3 TransportNPC study to continue based on the independent DMC review of safety and efficacy data at prespecified 48-week interim analysis
Data on the investigational candidate Trappsol® Cyclo™ (hydroxypropyl-beta-cyclodextrin), indicates that it is well-tolerated and has a safety profile consistent with the previously completed phase 1 and 2 studies and ongoing phase 1 open-label extension study
Read more at globenewswire.comNYSE:RFL (6/26/2025, 3:26:16 PM)
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