Cyclo Therapeutics, Inc. announced a positive outcome from its recent Type C meeting with the U.S. Food and Drug Administration (FDA), held on December 14, 2023 to discuss a full data review of the Company?s Trappsol® Cyclo? clinical development program to date and proposed strategies for the statistical analysis for the ongoing pivotal Phase 3 study, TransportNPC?, in order to enable a patient population lacking available therapies to have earlier access to a potentially efficacious product with an acceptable safety profile. In summary, the Company?s clinical data (NCT02939547, NCT03893071 and NCT02912793) provided suggestive evidence, based on individual patient data, of potential therapeutic benefit which may be observed at or before 48 weeks of treatment.

Given the significant unmet need within this Orphan Disease and the lack of available effective pharmacotherapy, and that aggregate clinical and nonclinical data argue for a potentially clinically relevant therapeutic effect, the FDA aligned with Cyclo Therapeutics? proposal to continue with the agreed upon Phase 3 96-week study, but change the timing of the planned comparative interim analysis to execute data readout in First Quarter 2025. The Company?s ongoing TransportNPC?

study is a randomized, double-blind, placebo-controlled, parallel group, multicenter study designed to evaluate the safety, tolerability, and efficacy of 2,000 mg/kg doses of Trappsol® Cyclo? administered intravenously and standard of care (SOC), compared to placebo administered intravenously and SOC alone, in patients with NPC1, a rare, genetic disease causing cholesterol accumulation in cells, leading to dysfunction of the liver, lung, spleen and brain and premature death. The Phase 3 study intends to enroll at least 93 pediatric (age 3 years and older) and adult patients with NPC1 and is now active in 12 countries.

Cyclo Therapeutics received Orphan Drug Designation for Trappsol® Cyclo? to treat NPC1 in both the U.S. and EU and Fast Track and Rare Pediatric Disease Designations in the U.S. The Rare Pediatric Disease Designation is one of the chief requirements for sponsors to receive a Priority Review Voucher in the U.S. upon marketing authorization.