Rafael Holdings, Inc.

Rafael Holdings, Inc. (NASDAQ: RAFA) is a biotechnology company focused on developing pharmaceuticals and holding interests in clinical and early-stage companies that develop pharmaceuticals and medical devices. The company's website is www.rafaelholdings.com.

The company's primary focus is the continued development of Trappsol® Cyclo™, an investigational drug designed to treat Niemann-Pick Disease Type C1, a rare, fatal, and progressive genetic disorder. Trappsol® Cyclo™ has received Orphan Drug Designation, Fast Track Designation, and Rare Pediatric Disease Designation from the FDA. The company is currently conducting a Phase 3 clinical trial called TransportNPC™, which has fully enrolled 94 patients across more than 30 sites in 13 countries. An interim analysis at 48 weeks was completed in June 2025, with an independent Data Monitoring Committee recommending continuation of the study for the full 96-week duration.

Beyond its lead candidate, Rafael Holdings maintains a diversified portfolio of healthcare investments. The company holds approximately 95% ownership in LipoMedix, a clinical-stage pharmaceutical company developing Promitil®, a pegylated liposomal mitomycin-C prodrug for cancer treatment. Rafael Holdings owns a 67% interest in Cornerstone Pharmaceuticals, a cancer metabolism-based therapeutics company, following a restructuring completed in March 2024. The company also holds a 73% equity interest in Rafael Medical Devices, an orthopedic-focused medical device company that received FDA clearance for its VECTR System in December 2024 for minimally invasive ligament and fascia release surgeries. Additionally, Rafael Holdings maintains a majority interest in Day Three Labs, a technology company focused on bioavailability solutions for pharmaceutical and supplement products.

The company owns Barer, a wholly-owned cancer research entity whose operations have been substantially curtailed, and holds a portion of a commercial building in Jerusalem, Israel as its remaining real estate asset. Rafael Holdings operates as a smaller reporting company and is not a well-known seasoned issuer. The company's strategy focuses on developing therapeutics addressing high unmet medical needs while evaluating its portfolio holdings to ensure resources remain concentrated on core assets, particularly Trappsol® Cyclo™'s clinical development and potential commercialization.