RFL News & Events

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.

Rafael Holdings announced that its subsidiary Cyclo Therapeutics' 96-week pivotal phase 3 TransportNPC study evaluating intravenous Trappsol Cyclo for the potential treatment of Niemann-Pick Disease Type C1 will continue based on the independent Data Monitoring Committee review of safety and efficacy data at the prespecified 48-week interim analysis. Additionally, the Food and Drug Administration has accepted the statistical analysis plan for the TransportNPC study. These developments underscore the company's continuing commitment to advancing its lead investigational candidate through late-stage clinical development to support global regulatory and commercial readiness.

Reports Q3 revenue $362,000 vs $336,000 last year. "We are pleased to have completed our merger with Cyclo Therapeutics and look forward to reporting the topline data from the 48-week interim analysis of the pivotal Phase 3 TransportNPC study evaluating Trappsol Cyclo for the treatment of Niemann-Pick Disease Type C1 anticipated later this month," said Howard Jonas, Chief Executive Officer, Executive Chairman and Chairman of the Board of Rafael Holdings. Jonas added, "We have enhanced our financial position with the closing of a $25 million rights offering earlier this month which will support advancing this potential new treatment option for patients suffering from this rare genetic disease."