Rafael Holdings (RFL) announced that its subsidiary Cyclo Therapeutics’ 96-week pivotal phase 3 TransportNPC study evaluating intravenous Trappsol Cyclo for the potential treatment of Niemann-Pick Disease Type C1 will continue based on the independent Data Monitoring Committee review of safety and efficacy data at the prespecified 48-week interim analysis. Additionally, the Food and Drug Administration has accepted the statistical analysis plan for the TransportNPC study. These developments underscore the company’s continuing commitment to advancing its lead investigational candidate through late-stage clinical development to support global regulatory and commercial readiness.
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