Rafael Holdings, Inc. is a biotechnology company that develops pharmaceuticals and holds interests in clinical and early-stage enterprises that develop pharmaceuticals and medical devices. Its lead product candidate is Trappsol® Cyclo™, an investigational therapy being evaluated in a pivotal Phase 3 clinical trial for the treatment of Niemann‑Pick Disease Type C1, a rare and fatal genetic disorder. In addition to Cyclo, the company owns majority stakes in LipoMedix,…
Rafael Holdings, Inc. is a biotechnology company that develops pharmaceuticals and holds interests in clinical and early-stage enterprises that develop pharmaceuticals and medical devices. Its lead product candidate is Trappsol® Cyclo™, an investigational therapy being evaluated in a pivotal Phase 3 clinical trial for the treatment of Niemann‑Pick Disease Type C1, a rare and fatal genetic disorder. In addition to Cyclo, the company owns majority stakes in LipoMedix, Barer, Cornerstone, Rafael Medical Devices, and Day Three, and retains a portion of a commercial building in Jerusalem, Israel as its sole remaining real estate asset.
The company generates revenue primarily from the eventual sale of its approved pharmaceutical products, licensing of its proprietary technologies, and royalty arrangements with partners. Rafael Medical Devices derives income from the sale of its VECTR System and related orthopedic instruments, while Day Three earns fees from licensing its Unlokt™ platform to third‑party manufacturers. Additionally, the real estate holding in Jerusalem provides rental income from leased office space.
The company operates through the following segments.
- Cyclo: develops cyclodextrin‑based biopharmaceuticals, with Trappsol® Cyclo™ as its lead candidate for Niemann‑Pick Disease Type C1 and continues a legacy fine‑chemical business selling cyclodextrins to pharmaceutical and nutritional industries.
- LipoMedix: a clinical‑stage Israeli company focused on the liposomal mitomycin‑C prodrug Promitil® as a potential cancer therapy.
- Barer: a wholly‑owned cancer research entity whose activities have been curtailed, now principally holding interests in SHMT inhibitor programs through its Farber subsidiary and licensing agreements with the Ludwig Institute of Cancer Research.
- Cornerstone: a majority‑owned cancer metabolism‑based therapeutics company whose lead asset is CPI‑613® (devimistat) for indications including pancreatic cancer and hematologic malignancies.
- Rafael Medical Devices: an orthopedic‑focused medical device company that has obtained FDA clearance for the VECTR System, a tool for minimally invasive ligament and fascia release procedures such as carpal and cubital tunnel release.
- Day Three: a technology firm that enables third‑party manufacturers to improve the bioavailability of active ingredients, notably cannabinoids, through its Unlokt™ platform.
Within the biotechnology and medical device sectors, Rafael Holdings competes with large pharmaceutical firms, specialized biotech companies, and academic research groups that are pursuing therapies for rare diseases, cancer therapeutics, and orthopedic devices. Its competitive advantages stem from orphan drug designations and Fast Track status for Trappsol® Cyclo™, a diversified portfolio of early‑stage assets that reduces reliance on any single program, and FDA clearance of the VECTR System that provides an immediate commercial pathway in orthopedics.
The company serves patients suffering from Niemann‑Pick Disease Type C1 through its Cyclo subsidiary, oncology patients via LipoMedix and Cornerstone therapeutics, orthopedic surgeons and hospitals that purchase Rafael Medical Devices’ VECTR System, pharmaceutical and supplement manufacturers that license Day Three’s Unlokt™ technology, and commercial tenants leasing its Jerusalem office space.