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Rafael Holdings Phase 3 trial misses primary endpoint but shows significant survival benefit
RFLRafael Holdings, Inc.
Rafael Holdings announced topline results from its pivotal Phase 3 TransportNPC study evaluating Trappsol Cyclo in Niemann-Pick Disease Type C. The primary endpoint did not achieve statistical significance (p=0.19), though a prespecified analysis of patients on background therapy showed a significant benefit (p=0.046). An overall survival analysis indicated an 85% reduction in mortality risk compared to external controls. The company remains on track to submit a New Drug Application to the FDA in the fourth quarter of 2026. Rafael Holdings is a biotechnology company that develops pharmaceuticals and holds interests in clinical-stage companies.