Rafael Holdings’ subsidiary Cyclo Therapeutics announced an exclusive licensing agreement with the Massachusetts Institute of Technology for U.S. Patent No. 12285440, which covers the use of cyclodextrins in Alzheimer’s disease patients carrying the ApoE4 genetic mutation.
The patent protects a class of small molecules that improve myelination in ApoE4‑positive patients, a group that represents 50‑70 % of all Alzheimer’s cases. By securing this license, Cyclo expands its therapeutic focus beyond its lead candidate Trappsol Cyclo for Niemann‑Pick disease type C1.
The deal addresses long‑standing investor concerns about pipeline concentration and positions the company to pursue a high‑need, high‑value orphan‑drug market. It also provides a strategic foundation for future product development and potential licensing revenue.
Howard Jonas, CEO and Executive Chairman, said, “Securing this exclusive license from MIT represents a transformative milestone for our company.” Joshua Fine, COO, added, “With Phase 3 results from Transport NPC due in calendar Q3 2026, we’re excited by Trappsol® Cyclo™’s potential across multiple disease areas. Our NPC1 phase 3 study is the largest ever conducted in patients with NPC, yielding crucial clinical and safety data needed to advance the treatment paradigm for patients and families dealing with NPC.”
The announcement was well received by investors, reflecting confidence in the expanded pipeline and the upcoming Phase 3 results for Trappsol® Cyclo™ in Niemann‑Pick disease type C1.
Preclinical studies cited in the announcement demonstrate that cyclodextrins improve cholesterol transport, reduce tau pathology, and enhance learning and memory in mouse models of Alzheimer’s. While the Alzheimer’s indication remains in the preclinical stage, the MIT license provides a solid intellectual‑property foundation that could accelerate future development and create new revenue opportunities.
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