Rafael Holdings Secures Exclusive MIT Patent License to Advance Alzheimer’s Disease Therapy

Key Points for Investors

• Rafael Holdings, Inc. (NYSE: RFL) has announced a major milestone: its subsidiary, Cyclo Therapeutics, has signed an exclusive licensing agreement with the Massachusetts Institute of Technology (MIT) for U.S. Patent No. 12285.
• This license covers the use of cyclodextrins as an Active Pharmaceutical Ingredient (API) for the treatment of Alzheimer’s Disease (AD) in patients with the ApoE4 genetic mutation—a group representing 50-70% of all AD patients.
• The agreement strengthens Rafael’s intellectual property position around its lead candidate, Trappsol® Cyclo™, in the large and high-risk ApoE4-positive Alzheimer’s population.
• The mechanism of action for Trappsol® Cyclo™ targets cholesterol transport issues believed to underlie AD pathology, especially in ApoE4 carriers.
• Preclinical data suggest Trappsol® Cyclo™ may improve cholesterol delivery between CNS cells, reduce tau-pathology, and improve learning and memory in animal models.
• Upcoming catalysts include the release of Phase 3 results for Trappsol® Cyclo™ in Niemann-Pick Disease Type C1 (NPC1) expected in Q3 2026—the largest NPC1 clinical trial to date.

Details of the Licensing Agreement

On April 22, 2026, Rafael Holdings revealed a significant step forward in its neurodegenerative disease program by securing exclusive rights from MIT to a patent covering the use of cyclodextrins in Alzheimer’s patients carrying the ApoE4 mutation. This agreement is particularly noteworthy given the high prevalence of the ApoE4 gene variant among AD patients and its established role as a key risk factor for early-onset and sporadic Alzheimer’s.

The MIT patent provides Rafael Holdings with a formidable layer of protection for its Trappsol® Cyclo™ investigational therapy, setting the company apart in a highly competitive field and enhancing its potential value for shareholders.

Scientific and Clinical Rationale

The ApoE4 variant is believed to disrupt cholesterol metabolism in the brain, contributing to the formation of amyloid plaques and tau tangles—hallmarks of Alzheimer’s pathology. Trappsol® Cyclo™ has demonstrated, in preclinical studies, an ability to correct cholesterol trafficking issues, reduce tau pathology, and improve cognitive outcomes in animal models. This suggests a robust therapeutic rationale for Cyclo Therapeutics’ approach, especially in the large subgroup of ApoE4-positive patients who are at highest risk.

What Shareholders Need to Know

• This licensing deal is a transformative milestone for Rafael Holdings—it not only solidifies their IP portfolio but also positions the company as a leader in the development of disease-modifying therapies for Alzheimer’s.
• Potential Share Price Impact: The announcement of a prestigious MIT license, focused on a massive and high-need patient population, is likely to be viewed very positively by the market. The move substantially increases the long-term commercial potential of Trappsol® Cyclo™ and could attract new investor interest.
• Upcoming Catalysts: Investors should pay close attention to the anticipated Phase 3 results for NPC1 in Q3 2026. A positive outcome could further validate the approach and unlock additional value for shareholders.

About Rafael Holdings, Inc.

Rafael Holdings is a biotechnology company focused on developing innovative therapies for rare and devastating diseases. Its lead candidate, Trappsol® Cyclo™, is currently being studied in a pivotal Phase 3 trial for Niemann-Pick Disease Type C1 (NPC1)—a rare, fatal genetic disorder. The company also maintains a portfolio of interests in other clinical- and early-stage pharmaceutical and medical device companies, including a focus on cancer metabolism and orthopedic devices.

Forward-Looking Statements

The company notes that this press release contains forward-looking statements, which involve risks and uncertainties that could lead to materially different results. Investors are encouraged to review the risk factors outlined in Rafael Holdings’ SEC filings, including the Annual Report on Form 10-K for the year ended July 31, 2025.

Contact

For investor inquiries, please contact Barbara Ryan at [email protected] or (203) 274-2825.

Disclaimer: This article is for informational purposes only and does not constitute investment advice. Investors should perform their own due diligence and consult with financial professionals before making investment decisions. The information herein is based on company disclosures as of April 22, 2026, and may be subject to change without notice.

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