Rafael Holdings, Inc. RFL
Rafael Holdings, Inc. (NYSE: RFL) is a rare genetic drug developer. Its lead program, Trappsol Cyclo (remibetadex), is in Phase 3 for Niemann-Pick Disease Type C (NPC). Its next catalyst on record is Trappsol Cyclo: FDA filing decision on NDA (December 2026).
Upcoming catalysts
- December 2026Trappsol Cyclo — FDA filing decision on NDA
Within 60 days of receipt, the FDA will determine whether the application is complete and acceptable for filing and, if accepted, whether Priority Review will be granted.
Next further out: Trappsol Cyclo — PDUFA action date assignment (date not stated). 1 catalyst on record beyond the next 30 days; subscribers see each with its filing sentence.
Pipeline
| Program | Indication | Modality | Stage |
|---|---|---|---|
| Trappsol Cyclo (remibetadex) | Niemann-Pick Disease Type C | small molecule | Phase 3 |
| Promitil (LipoMedix) | Cisplatin-Ineligible Head and Neck Cancer | small molecule | Phase 2 |
| Trappsol Cyclo (remibetadex) | Alzheimer's disease (APOE4 carriers) | small molecule | Pre-clinical |
Recent results and decisions
- Oct 28, 2026submitted Rafael Holdings subsidiary Cyclo Therapeutics submitted an NDA to the FDA for remibetadex to treat Niemann-Pick Disease Type C.
Cyclo Therapeutics, LLC, has submitted a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) seeking approval of remibetadex for the proposed indication of the treatment of the neurovisceral manifestations of Niemann-Pick Disease Type C (NPC) in adult and pediatric patients fro
Filing - Sep 30, 2026missed Phase 3 TransportNPC missed primary endpoint (4DNPCSS at Week 96: -0.81 difference, p=0.19; 64% slowing); prespecified miglustat/leucine subgroup (n=78) p=0.046.
Trappsol Cyclo did not achieve statistical significance but demonstrated a directionally favorable treatment effect versus placebo on the primary endpoint of change in the 4-domain NPC Clinical Severity Scale (4DNPCSS) at Week 96
Filing - Sep 30, 2026missed The Phase 3 TransportNPC™ study did not achieve statistical significance on its primary endpoint but showed directionally favorable results in subgroup analyses.
Although the study did not achieve statistical significance on its primary endpoint, remibetadex showed a directionally favorable 64% relative slowing of disease progression compared with placebo (p=0.19).
Filing
2 more on record.
For subscribers
- Cash runway against each catalyst, with the company's own runway statement
- Track record: every Phase 3 readout, FDA decision and guidance slip since 2014
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- Phase 2 evidence behind each Phase 3 readout
- Companies on the same target or class
- Full catalyst list with filing sentences, deals, registered trials, Excel export
Other phase 3 rare genetic companies
The free Monday brief
Last week's readouts and FDA decisions and the dated catalysts in the week ahead, each with its filing sentence.