Rafael Holdings, Inc. (“Rafael Holdings”, “Rafael”, “we” or the “Company”) is a biotechnology company that develops pharmaceuticals and holds interests in clinical and early-stage companies that develop pharmaceuticals and medical devices. Our lead candidate is Trappsol® Cyclo™, which is being evaluated in clinical trials for the potential treatment of Niemann-Pick Disease Type C1 (“NPC1”), a rare, fatal and progressive genetic disorder.
Estimated readout windows: registry primary-completion date + 6-12 weeks. Windows already open are shown from "Now". Drug names link to ClinicalTrials.gov.
Partnerships Massachusetts Institute of Technology: exclusive worldwide royalty-bearing patent license (Apr 16, 2026) for products containing hydroxypropyl-beta-cyclodextrin as the active ingredient in the field of Alzheimer’s disease; milestones include dosing the first patient in a U.S. Phase II trial by Mar 31, 2027
Indications
Niemann-Pick disease type C1 (NPC1)(Phase 3)
Alzheimer’s disease (APOE4 carriers)(Stage not disclosed)
Catalysts
— second half of calendar 2026
— second half of calendar 2026
— by Mar 31, 2027
Status note Lead candidate; active ingredient is hydroxypropyl-beta-cyclodextrin. Pivotal Phase 3 TransportNPC study in NPC1: last patient completed the final 96-week visit (announced Jun 2026); pre-NDA meeting with FDA completed.
Per-asset detail extracted from the 10-Q filed 2026-06-11. Fields the filing does not state are marked "not disclosed" rather than filled from other sources.
No lead program on file for this company.
No active ClinicalTrials.gov trials matched to this company as sponsor or collaborator.
Specialist fund holders
Tracked specialist funds: ADAR1 Capital Management, Aisling Capital, Ally Bridge Group, Avoro Capital Advisors, BVF Partners, Baker Bros. Advisors, Boxer Capital, Braidwell LP, Casdin Capital, Cormorant Asset Management, DAFNA Capital, Deerfield Management, EcoR1 Capital, Frazier Life Sciences, Great Point Partners, Ikarian Capital, Logos Global Management, OrbiMed Advisors, Perceptive Advisors, RA Capital Management, RTW Investments, Sphera Biotech, Stonepine Capital, Tang Capital Partners, Vivo Capital.
None of the tracked specialist funds reported a position in the latest 13F snapshot.
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