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

Core Insights - The Phase 3 TransportNPC study for Trappsol® Cyclo™ will continue based on a positive independent DMC review of safety and efficacy data at the 48-week interim analysis [1][2] - The FDA has accepted the statistical analysis plan for the study, indicating regulatory support for the ongoing clinical development [1] - The study aims to evaluate the treatment of Niemann-Pick Disease Type C1 (NPC1), a rare genetic disorder with significant unmet medical needs [2][8] Company Overview - Rafael Holdings, Inc. is a biotechnology company with a focus on developing treatments for rare and neurodegenerative diseases, including its lead candidate Trappsol® Cyclo™ [9][10] - Cyclo Therapeutics, a wholly owned subsidiary of Rafael Holdings, is dedicated to advancing Trappsol® Cyclo™ through clinical trials [10] - The company recently bolstered its financial position with a $25 million rights offering to support strategic objectives [2] Study Details - The TransportNPC study is a 96-week, randomized, double-blind, placebo-controlled trial involving 94 patients across 25 sites in 13 countries [4][5] - The primary endpoints include changes in the NPC Clinical Severity Scale scores, which measure disease progression relevant to patients and caregivers [5] - An open-label extension study will follow the interventional study, allowing for continued evaluation of Trappsol® Cyclo™ [4][6] Treatment Mechanism - Trappsol® Cyclo™ is an intravenous formulation designed to mobilize lysosomal cholesterol, addressing the root cause of NPC1 [3] - The drug has shown the ability to cross the blood-brain barrier, potentially reaching therapeutic concentrations in the central nervous system [3] Disease Background - Niemann-Pick Disease Type C1 (NPC1) is a rare genetic disorder affecting approximately 1 in 100,000 live births, characterized by cholesterol accumulation in cells [8] - The disease leads to severe symptoms, including intellectual decline and loss of motor skills, often resulting in premature death [8]