Rafael Holdings, Inc.

Rafael Holdings, Inc.

Company overview

Rafael Holdings, Inc. is a Delaware biopharma holding company focused on developing pharmaceuticals and investing in a portfolio of clinical and early-stage companies in therapeutics and medical devices. Its lead program is Trappsol® Cyclo™ (remibetadex) for Niemann-Pick Disease Type C (NPC). A pivotal Phase 3 trial was completed and an NDA was filed with the FDA on October 28, 2026. The company also holds minority and majority interests in several operating entities across its portfolio.

Portfolio companies and ownership (highlights)

• Cyclo Therapeutics, LLC (Cyclo) — Wholly owned subsidiary; develops Trappsol® Cyclo™ (HPβCD IV) for NPC.
• LipoMedix Pharmaceuticals Ltd. (LipoMedix) — Israeli company developing Promitil (liposomal mitomycin-C prodrug); Rafael owns approximately 95% as of July 31, 2026.
• Barer Institute Inc. (Barer) — Holds equity in early-stage cancer assets; majority-owned subsidiary Farber Partners, LLC (Farber).
• Cornerstone Pharmaceuticals, Inc. (Cornerstone) — Focused on cancer metabolism; Rafael holds a majority equity interest (around 67% after the 2024 restructuring).
• Rafael Medical Devices, LLC — Orthopedic medical devices; majority equity interest (83% after July 2026 transactions). VECTR System cleared by the FDA for certain hand and forearm procedures; pursuing additional device opportunities.
• Day Three Labs, Inc. (Day Three) — Bioavailability and cannabinoid-related technology; Day Three Labs Manufacturing (DTLM) is a majority-owned subsidiary. Day Three and its subsidiaries participate in Unlokt™ technology and related activities.

Real estate

As of July 31, 2026, Rafael Holdings retains a portion of a commercial building in Jerusalem, Israel (approximately 12,400 gross square feet, one floor, with 24 indoor parking spaces). The space is fully leased to two tenants.

Financial and operating snapshot (selected figures)

• Employees: As of October 15, 2026, 15 full-time employees and 2 part-time employees.
• Liquidity and investments (as of July 31, 2026):
  • Cash and cash equivalents: approximately $22.1 million
  • Convertible notes receivable (available for sale): approximately $2.0 million
  • Investments: approximately $0.8 million
• Revenue and profitability: Clinical-stage biotechnology company with no approved products and no revenue from product sales. For the year ended July 31, 2026, one major customer tied to Cyclo-related revenue accounted for 34% of total revenues.
• NDA and regulatory context: NDA for Trappsol® Cyclo™ submitted on October 28, 2026. The regulatory pathway may include Priority Review and Rare Pediatric Disease Priority Review Voucher (PRV) considerations; the PRV program has a sunset provision that could affect eligibility after September 30, 2029.
• Intellectual property and licenses:
  • Cyclo holds an MIT license for Trappsol® Cyclo™ related to an Alzheimer’s indication, with a March 31, 2027 diligence deadline to dose the first patient in a U.S. Phase II Alzheimer’s trial (subject to license terms and funding).
  • LipoMedix maintains exclusive licenses with Yissum and Shaare Zedek for Promitil.
  • Cornerstone and Barer maintain various patent families and collaborations, with multiple orphan-designation opportunities across assets.

Operations and business model

• Primary focus is advancing Trappsol® Cyclo™ through clinical development and regulatory review for NPC, including open-label extensions and international pathways.
• Manage and develop a diversified portfolio of bio/pharma and medical-device companies through minority and majority stakes, pursuing strategic investments, collaborations, and potential monetization.
• Manufacturing and supply are outsourced: Rafael and its portfolio companies rely on contract manufacturers and third-party suppliers and must manage associated regulatory compliance.
• Maintain select real estate and pursue opportunistic investments and strategic collaborations to support portfolio development.

What the company does (concise)

Rafael Holdings is a biotechnology holding company that develops and commercializes pharmaceuticals led by Trappsol® Cyclo™ for NPC, while holding substantial or controlling stakes in portfolio companies (Cyclo, LipoMedix, Cornerstone, Rafael Medical Devices, Day Three, Barer/Farber). The company supports development, licensing, and potential monetization of therapeutic and medical-device assets, maintains a Jerusalem real estate asset, and funds early-stage investments. Current liquidity is held in cash, investments, and receivables, and operating costs are focused on regulatory submissions and clinical development.