Rafael announces data from Cyclo Therapeutics’ Phase 3 TransportNPC study

Rafael announced that data from Cyclo Therapeutics’ Phase 3 TransportNPC open-label, single-arm sub-study evaluating Trappsol Cyclo for Niemann-Pick Disease Type C1 were presented at the 15th International Congress of Inborn Errors of Metabolism, ICIEM. Summary Of Data Presented In Dr. Staretz-Chacham’s Poster. At baseline, sub-study patients had a mixture of very mild to severe disease based on the Clinical Global Impression – Severity 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 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 or moderate, 3 reported as severe and the remainder reported as Serious Adverse Events; and No SAEs were considered by the principal investigators as related to or possibly related to study drug.