Rafael Holdings Announces Presentation of Preliminary Data from Ongoing Phase 3 TransportNPC™ Open-Label, Single-Arm Sub-Study in Patients Less Than 3 Years Old

Core Viewpoint - Rafael Holdings, Inc. announced promising preliminary data from the Phase 3 TransportNPC™ study evaluating Trappsol® Cyclo™ for Niemann-Pick Disease Type C1 (NPC1), presented at the 15th International Congress of Inborn Errors of Metabolism (ICIEM) [1][2]. Group 1: Study Overview - The ongoing TransportNPC™ study is a randomized, double-blind, placebo-controlled trial designed to evaluate the safety, tolerability, and efficacy of Trappsol® Cyclo™ at a dose of 2,000 mg/kg administered intravenously [2]. - The study has a total duration of 96 weeks, with a comparative interim analysis conducted at 48 weeks [2][11]. - Enrollment for the study was completed in May 2024, with 94 patients recruited across over 25 sites in 13 countries [11]. Group 2: Sub-Study Results - In the sub-study involving patients under the age of 3, 7 out of 9 patients showed stabilization or improvement in their Clinical Global Impression – Severity (CGI-S) score after 48 weeks [1][7]. - The adverse event profile was consistent with previous studies, with 146 adverse events reported, the majority being mild (69%) or moderate (29%) [7]. Group 3: Clinical Significance and Future Directions - Dr. Orna Staretz-Chacham highlighted the potential of Trappsol® Cyclo™ to address significant unmet medical needs in NPC1 treatment, with encouraging clinical improvements observed [6]. - The full clinical significance of the preliminary data will be evaluated based on the complete results of the ongoing sub-study [5]. Group 4: Drug Information - Trappsol® Cyclo™ is an investigational drug designed to mobilize lysosomal cholesterol, directly targeting the root cause of NPC1 [10]. - The drug has received Orphan Drug Designation in both the U.S. and EU, along with Fast Track and Rare Pediatric Disease Designations in the U.S. [9].