Rafael Holdings Announces Abstracts Accepted for Oral and Poster Presentations at the 15th International Congress of Inborn Errors of Metabolism (ICIEM)

Core Viewpoint - Rafael Holdings, Inc. announced that its subsidiary Cyclo Therapeutics will present two abstracts on Trappsol Cyclo™ for Niemann-Pick Disease Type C1 at the upcoming ICIEM conference in Kyoto, Japan [1] Group 1: Presentation Details - The first presentation will be an oral session titled "Hydroxypropyl-beta-cyclodextrin for the Long-Term Treatment of Niemann Pick Type C1: Efficacy and Safety Data from 4 Clinical Studies and the Ongoing Expanded Access Program" by Dr. Caroline Hastings on September 3, 2025 [1] - The second presentation will be a poster session titled "Trappsol Cyclo Open Label Treatment in the TransportNPC Sub-Study in Patients Under the Age of 3 Diagnosed with Niemann Pick Disease Type C1" by Dr. Orna Staretz Chacham on the same day [1] Group 2: About Trappsol Cyclo™ - Trappsol Cyclo™ is a first-in-class proprietary cyclodextrin formulation administered intravenously that mobilizes lysosomal cholesterol, targeting the root cause of Niemann-Pick Disease Type C1 [2] - It has been shown to cross the blood-brain barrier, suggesting therapeutic concentrations in the central nervous system [2] Group 3: Study Program - The Phase 3 TransportNPC study is a randomized, double-blind, placebo-controlled trial evaluating the safety and efficacy of Trappsol Cyclo™ in patients aged 3 years and older, with a total enrollment of 94 patients across 25 sites in 13 countries [3] - The study duration is 96 weeks, with an interim analysis recommending continuation after 48 weeks [3] Group 4: Disease Overview - Niemann-Pick Disease Type C1 is a rare genetic disorder affecting approximately 1 in 100,000 live births, characterized by cholesterol accumulation leading to organ damage and life-limiting symptoms [8] - About 95% of individuals with NPC have mutations in the NPC1 gene, while 5% have mutations in the NPC2 gene [8] Group 5: Company Overview - Rafael Holdings, Inc. is a biotechnology company with a focus on developing treatments for rare and neurodegenerative diseases, including a 100% interest in Cyclo Therapeutics [9] - Cyclo Therapeutics is dedicated to developing Trappsol Cyclo™ for Niemann-Pick Disease Type C1 and is conducting multiple clinical trials for this indication [10]