Rafael Holdings, Inc. RFL
Rafael Holdings, Inc. (NYSE: RFL) is a rare genetic drug developer. Its lead program, Trappsol Cyclo (hydroxypropyl beta cyclodextrin), is in Phase 3 for Niemann-Pick Disease Type C1 (NPC1). Its next catalyst on record is Trappsol Cyclo: Rafael Holdings remains on track to submit an NDA for Trappsol Cyclo in the fourth quarter of 2026. (Q4 2026).
Upcoming catalysts
No catalyst is dated to the next 30 days or to an FDA action date.
Next further out: Trappsol Cyclo — Rafael Holdings remains on track to submit an NDA for Trappsol Cyclo in the fourth quarter of 2026. (Q4 2026). 1 catalyst on record beyond the next 30 days; subscribers see each with its filing sentence.
Pipeline
| Program | Indication | Modality | Stage |
|---|---|---|---|
| Trappsol Cyclo (pivotal Phase 3) | Niemann-Pick Disease Type C1 | small molecule | Phase 3 |
| Cyclo MIT-licensed myelination small molecules | Alzheimer's disease / APOE4 carriers | small molecule | Pre-clinical |
| Barer/Farber early-stage cancer metabolism programs (SHMT transferred to Forme Therapeutics; one licensed to Ludwig) | cancer | small molecule | Pre-clinical |
| VECTR System (Rafael Medical Devices, 510(k) cleared) | minimally invasive ligament/fascia release surgery (carpal and cubital tunnel) | unclear | NA_NON_THERAPEUTIC |
Recent results and decisions
- 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
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
- Expected move for each dated catalyst, from comparable past events
- 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.