Rafael's Cyclo Therapeutics files NDA for remibetadex in Niemann-Pick Disease Type C

Rafael Holdings, Inc. disclosed on October 29, 2026 that Cyclo Therapeutics submitted a New Drug Application to the FDA on October 28, 2026 seeking approval of remibetadex for the treatment of Niemann-Pick Disease Type C.¹

The application draws on the Phase 3 "TransportNPC" study, which wrapped up in June 2026. That trial did not meet its primary endpoint in the overall patient population, with a p-value of 0.19, but a prespecified analysis of patients also receiving background miglustat and/or leucine therapy showed a 71% slowing of disease progression, with a p-value of 0.046. The filing is also backed by a comparison of overall survival against matched registry controls.¹ The NDA additionally requests a rare pediatric disease priority review voucher.¹

Separately, the United States Adopted Names Council has assigned the nonproprietary name remibetadex to the compound previously known as intravenous hydroxypropyl-β-cyclodextrin.¹

Elsewhere in the pipeline, Cyclo entered an exclusive, worldwide license in April 2026 with the Massachusetts Institute of Technology covering hydroxypropyl-β-cyclodextrin for use in Alzheimer's disease.¹ In Israel, an investigator-initiated Phase II trial testing Promitil with radiotherapy began in June 2026 in head and neck cancer patients who are ineligible for cisplatin.¹ Rafael Medical Devices continued its U.S. commercial build-out of the FDA-cleared VECTR System, including expanding its regional distributor network.¹

On financials, the company reported cash and cash equivalents of $22.1 million as of July 31, 2026.¹ For the fiscal fourth quarter, Rafael recorded a net loss attributable to the company of $9.2 million, or $0.18 per share, compared with a $12.1 million loss, or $0.28 per share, in the year-ago period.¹ For the full fiscal year, the net loss attributable to Rafael Holdings was $29.6 million, or $0.58 per share, versus $30.5 million, or $1.04 per share, a year earlier.¹