Rafael Holdings, Inc. announced that data from Cyclo Therapeutics’ Phase 3 TransportNPC™ open-label, single-arm sub-study evaluating Trappsol® Cyclo™ (hydroxypropyl-beta-cyclodextrin) for Niemann-Pick Disease Type C1 (“NPC1”) were presented at the 15th International Congress of Inborn Errors of Metabolism (ICIEM).
The Company’s ongoing TransportNPC™ study is a randomized, double-blind, placebo-controlled, parallel group, multicenter study designed to evaluate the safety, tolerability, and efficacy of 2,000 mg/kg doses of Trappsol® Cyclo™ administered intravenously and standard of care (SOC), compared to placebo administered intravenously and SOC alone, in patients with NPC1, a rare, genetic disease causing cholesterol accumulation in cells, leading to dysfunction primarily of the liver, lung, spleen and brain and premature death. The study duration is a 96-week study, with a 48-week comparative interim analysis.
- At baseline, sub-study patients had a mixture of very mild to severe disease based on the Clinical Global Impression – Severity (CGI-S) scale;
- Seven of nine patients who have reached 48 weeks participation in the study had an outcome of stabilization or improvement in CGI-S, with three patients showing improvement, and two patients showing deterioration of their CGI-S score;
- Adverse Event (AE) profile appears to be consistent with prior findings from earlier studies, and from the larger Phase 3 TransportNPC™ study that is irrespective of age and disease severity;
- As of May 14, 2025, there were 146 AEs, with the majority reported as mild (69%) or moderate (29%), 3 reported as severe and the remainder reported as Serious Adverse Events(SAEs) (14%); and
- No SAEs were considered by the principal investigators as related to or possibly related to study drug.
Trappsol® Cyclo™ (hydroxypropyl-beta-cyclodextrin) is an investigational drug designed to deliver a first-in-class propriety cyclodextrin formulation that is administered intravenously (IV) in order to mobilize lysosomal cholesterol. Trappsol® Cyclo™ is designed to directly impact the root cause of Niemann-Pick Disease Type C1 (NPC1) by mobilizing cholesterol from late-stage endosomes and lysosomes. Trappsol® Cyclo™ has also been found in the completed Phase 1 study (CTD-TCNPC-101) and a Phase 2 study (CTD-TCNPC-201) to cross the blood-brain barrier after IV administration, suggesting that therapeutic concentrations have the potential to be reached in the central nervous system over the infusion time window.