Rafael Holdings, Inc.'s subsidiary, Cyclo Therapeutics, is advancing its pivotal phase 3 TransportNPC study, which evaluates the intravenous treatment Trappsol® Cyclo™ for Niemann-Pick Disease Type C1 (NPC1). The study is set to continue following a positive review by an independent Data Monitoring Committee $(DMC.AU)$ of safety and efficacy data at the 48-week interim mark. The FDA has approved the statistical analysis plan for this study. The investigational candidate has demonstrated a safety profile consistent with earlier phase 1 and 2 studies. This study is considered the most comprehensive of its kind for NPC, both in terms of its scale and the breadth of clinical outcomes assessed.
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