Rafael Holdings Inc（RFL）资讯与事件RFLRafael Holdings Inc

Rafael Holdings Subsidiary Submits NDA for NPC Treatment

Rafael Holdings announced that its subsidiary, Cyclo Therapeutics, has submitted a New Drug Application, or NDA, to the U.S. Food and Drug Administration seeking approval of remibetadex for the treatment of the neurovisceral manifestations of Niemann-Pick Disease Type C, or NPC, in adult and pediatric patients from birth. Although the Phase 3 TransportNPC study did not achieve statistical significance on its primary endpoint, remibetadex showed a directionally favorable 64% relative slowing of disease progression compared with placebo and, in a prespecified analysis of patients receiving background therapy, a 71% relative slowing of disease progression. The NDA also includes an overall survival analysis in which remibetadex was associated with an 85% reduction in the risk of mortality in patients with infantile-onset NPC compared with matched registry controls. Remibetadex was generally well tolerated, although serious adverse events occurred more frequently with remibetadex than with placebo, 35.9% vs. 16.7%. "This submission is an important milestone for Rafael Holdings and, most importantly, for patients living with NPC. I believe the totality of the data, including the Phase 3 trial results and the overall survival analysis, supports the potential of remibetadex for patients with limited treatment options. Pending FDA approval, we are excited to bring remibetadex to patients and families who urgently need new options to treat NPC," said Howard Jonas, chairman and CEO of Rafael Holdings.

Rafael Holdings Announces Phase 3 Data for Trappsol Cyclo

Rafael Holdings announced topline efficacy and safety data from its Cyclo Therapeutics subsidiary's pivotal Phase 3 TransportNPC study evaluating Trappsol Cyclo in 94 patients diagnosed with Niemann-Pick Disease Type C, NPC. Trappsol Cyclo was well tolerated and demonstrated a directionally favorable treatment effect on the primary endpoint, although the result did not achieve statistical significance. Notably, a prespecified analysis in patients receiving background miglustat and/or leucine therapy demonstrated a statistically significant and clinically meaningful treatment benefit. Based upon its previously announced pre-NDA meeting with the FDA, the Company remains on track to submit an NDA for Trappsol Cyclo in the fourth quarter of 2026.

Rafael reveals findings from Cyclo Therapeutics' Phase 3 TransportNPC trial

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.