Cyclo Therapeutics' Phase 3 NPC1 Study Advances with Positive Interim Data Review
Cyclo Therapeutics, a subsidiary of Rafael Holdings, is progressing with its Phase 3 TransportNPC study for the treatment of Niemann-Pick Disease Type C1 (NPC1) using Trappsol® Cyclo™. The study continues after a positive interim review by an independent Data Monitoring Committee, confirming safety and efficacy at the 48-week mark. The FDA has approved the statistical analysis plan, and the investigational candidate shows a safety profile consistent with earlier studies. This study is noted for its comprehensive scale and clinical outcomes assessment.
Rafael Holdings, Inc.’s subsidiary, Cyclo Therapeutics, is advancing its pivotal phase 3 TransportNPC study, which evaluates the intravenous treatment Trappsol® Cyclo™ for Niemann-Pick Disease Type C1 (NPC1). The study is set to continue following a positive review by an independent Data Monitoring Committee (DMC) of safety and efficacy data at the 48-week interim mark. The FDA has approved the statistical analysis plan for this study. The investigational candidate has demonstrated a safety profile consistent with earlier phase 1 and 2 studies. This study is considered the most comprehensive of its kind for NPC, both in terms of its scale and the breadth of clinical outcomes assessed. Disclaimer: This news brief was created by Public Technologies (PUBT) using generative artificial intelligence. While PUBT strives to provide accurate and timely information, this AI-generated content is for informational purposes only and should not be interpreted as financial, investment, or legal advice. Rafael Holdings Inc. published the original content used to generate this news brief via GlobeNewswire (Ref. ID: GNW9470521-en) on June 18, 2025, and is solely responsible for the information contained therein. © Copyright 2025 - Public Technologies (PUBT)
