Cyclo Therapeutics, Inc. engages in the development of cyclodextrin-based biopharmaceuticals for the treatment of disease. Its product pipeline include Trappsol Cyclo, which treats Neimann-Pick Type C disease and Alzheimer's disease. The company was founded by Charles E. Rick Strattan on August 9, 1990 and is headquartered in Gainesville, FL.
Clinical Trials
14
7 active
Approvals
0
Total approvals
Agencies
0
Regulatory bodies
Founded
1990
Active, not recruiting
7
50.0%
Completed
2
14.3%
No Longer Available
1
7.1%
Recruiting
2
14.3%
Unknown
1
7.1%
Withdrawn
1
7.1%
No approval data available
- Rafael Holdings' subsidiary Cyclo Therapeutics has entered into an exclusive licensing agreement with MIT covering U.S. Patent No.12285440 for cyclodextrin use in ApoE4-positive Alzheimer's disease patients. - The ApoE4 gene variant is present in 50-70% of Alzheimer's patients and is believed to be a key risk factor for early onset sporadic disease development. - Preclinical data suggest Trappsol® Cyclo™ can improve cholesterol delivery between CNS cells, reduce tau pathology, and enhance learning and memory in mice. - The company expects Phase 3 results from their Transport NPC trial in Q3 2026, representing the largest study ever conducted in Niemann-Pick disease patients.
- Rafael Holdings announced preliminary data from its Phase 3 TransportNPC sub-study showing that 7 of 9 patients under 3 years old with NPC1 demonstrated stabilization or improvement after 48 weeks of treatment with Trappsol Cyclo. - The investigational drug hydroxypropyl-beta-cyclodextrin showed a favorable safety profile with the majority of adverse events reported as mild to moderate, and no serious adverse events were considered related to the study drug. - The data was presented at the 15th International Congress of Inborn Errors of Metabolism, representing the first clinical evidence for treating NPC1 in this youngest patient population over a 48-week period.
- Rafael Holdings successfully completed a $25 million rights offering in June 2025, with CEO Howard Jonas backstopping the majority of unsubscribed shares through a private placement. - The funding is specifically earmarked for regulatory approval efforts and potential commercial launch of Trappsol Cyclo, pending positive results from the ongoing Phase 3 TransportNPC trial. - Trappsol Cyclo is being evaluated as a treatment for Niemann-Pick Disease Type C1, a rare, fatal, and progressive genetic disorder with significant unmet medical need. - The company expects net proceeds of approximately $24.9 million after expenses, positioning it for potential regulatory milestones and market entry.
- Preliminary data from Cyclo Therapeutics' TransportNPC trial shows that 87% of patients under three years old with Niemann-Pick Disease Type C1 (NPC1) demonstrated stabilization or improvement after 24 weeks of Trappsol Cyclo treatment. - Six out of seven patients who continued Trappsol Cyclo treatment for 48 weeks showed continued improvement, suggesting sustained efficacy over a longer duration. - Trappsol Cyclo targets cholesterol mobilization, addressing neurodegeneration associated with NPC1, and Cyclo Therapeutics plans to seek regulatory approval from the FDA and EMA based on these results. - The ongoing TransportNPC study is a randomized, double-blind, placebo-controlled trial evaluating the safety and efficacy of Trappsol Cyclo in a broader NPC1 patient population.
