Rafael Holdings Announced Topline Efficacy And Safety Data From Its Cyclo Therapeutics Subsidiary’s Phase 3 TransportNPC Study Evaluating Trappsol Cyclo In 94 Patients Diagnosed With Niemann-Pick Disease Type C; Trappsol Cyclo Did Not Achieve Statistical Significance
Trappsol® Cyclo™ was well tolerated and demonstrated a directionally favorable treatment effect on the primary endpoint, although the result did not achieve statistical significance. Notably, a prespecified analysis in
Rafael's medicine for a rare disease worked safely but did not prove it helped enough. When a medicine doesn't show strong proof it works, doctors and investors watch carefully what happens next.
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