Posted by 100% of patients who completed the trial improved or remained stable, and 89% met the efficacy outcome measure of improvement in at least 2 domains of the 17-domain NPC Severity Scale
Trial data suggest that Trappsol® Cyclo™ overcomes the NPC1 defect by removing trapped cholesterol from cells both systemically and in the central nervous system (CNS)
Pharmacokinetic analysis confirms that Trappsol® Cyclo™ crosses the blood-brain-barrier after intravenous infusion and further supports neurological benefit
Cyclo Therapeutics, Inc., a clinical stage biotechnology company dedicated to developing life-changing medicines through science and innovation for patients and families suffering from diseases, announced topline data from its Phase 1/2 clinical trial, which demonstrated promising safety and efficacy results for Trappsol® Cyclo™ in the treatment of Niemann-Pick Disease type C1, a rare, genetic disease causing cholesterol accumulation in cells, leading to dysfunction of liver, lung, spleen and brain and premature death.
The multi-center, randomized, double-blind, parallel group trial without a placebo arm randomized 12 patients ranging in age from 2 to 39 years across clinical sites in the UK, Israel and Sweden. Patients were treated intravenously with Trappsol® Cyclo™, the Company’s proprietary formulation of hydroxypropyl beta cyclodextrin, over 8-9 hours for a total of 24 doses in a 48-week treatment period. Three doses of Trappsol® Cyclo™ were evaluated: 1500 mg/kg, 2000 mg/kg, and 2500 mg/kg. All patients received study drug, and the top line data summarizes the results from all dose levels combined.