Rafael Holdings is a clinical-stage biotechnology holding company primarily focused on developing Trappsol Cyclo (hydroxypropyl beta cyclodextrin), an investigational intravenous drug for Niemann-Pick Disease Type C1 (NPC1), a rare and fatal genetic disorder that disrupts cholesterol metabolism. Trappsol Cyclo is designed to mobilize trapped cholesterol from lysosomes, targeting the root cause of NPC1 rather than managing symptoms. The drug holds FDA Fast Track, Orphan Drug, and Rare Pediatric Disease designations and is currently in a pivotal global Phase 3 trial across 13 countries with 94 patients enrolled. Enrollment completed in May 2024, with a full 96-week readout expected before any NDA filing. Rafael Holdings is pre-revenue and funds development through its balance sheet and capital markets activity. If approved, Trappsol Cyclo would receive seven years of U.S. market exclusivity and up to 12 years in Europe, and may also qualify for a Rare Pediatric Disease Priority Review Voucher, which can be sold to other drug companies. Beyond Trappsol Cyclo, Rafael Holdings holds stakes in several early-stage subsidiaries: LipoMedix (cancer drug development, now exploring partnerships after a Phase 2a miss), Cornerstone Pharmaceuticals (cancer metabolism, restructuring after Phase 3 failures), Rafael Medical Devices (an orthopedic device with recent FDA clearance), and Barer Institute (largely wound down). Rafael Holdings is concentrating capital on the Trappsol Cyclo program and pursuing licensing or partnership options for its other assets.