Cyclo Therapeutics Shares Positive Phase 3 TransportNPC™ Data for Patients Under 3

Cyclo Therapeutics, Inc. (Nasdaq: CYTH), a biotechnology firm in the clinical stage, is focused on the development of transformative medicines for rare and challenging diseases. In recent news, the company has shared findings from its ongoing pivotal Phase 3 global study, TransportNPC™, which evaluates Trappsol® Cyclo™ as a treatment for Niemann-Pick Disease Type C1 (NPC1). This announcement was made at the 21st Annual WORLD Symposium™ in San Diego, CA.

The TransportNPC™ study is considered the most comprehensive of its kind, investigating an investigational therapy for NPC1. It is notable for its extensive patient size, global reach, and the duration and outcomes of the study. Enrollment for the study was completed in May 2024. Additionally, Cyclo Therapeutics has enrolled ten patients in a single-arm sub-study designed to treat newborns to children aged three, as per their Pediatric Investigational Plan (PIP). The sub-study aims to evaluate Trappsol® Cyclo™ in the youngest demographic affected by NPC1, focusing on the visceral aspects of the disease. Theoretically, early administration could potentially prevent overall symptom development of the disease. This sub-study is being conducted outside the United States.

At the WORLD Symposium™, Dr. Ronen Spiegel, a Clinical Associate Professor and Director of the Pediatric B Department at Emek Medical Center, delivered a presentation on the treatment of young NPC1 patients with Trappsol® Cyclo™. The sub-study results showed consistent lipid clearance, both centrally and systemically, in a prior 14-week Phase 1 study with adult NPC patients. Patients in the sub-study exhibited a range of disease severities at baseline, with clinical improvements being more pronounced in those with mild to moderate symptoms. Moreover, most patients had shown stabilization or improvement in their condition after 24 and 48 weeks.

The adverse effect profile aligns with previous findings from earlier studies, with most effects being mild to moderate. As of December 2024, 107 adverse effects were reported, with a majority being mild (77%) or moderate (22%), and only a single case reported as severe. Among these, 19 adverse effects were classified as serious, but none were deemed related to the investigational drug.

Dr. Spiegel expressed optimism about the preliminary data from the TransportNPC™ sub-study, highlighting the potential of Trappsol® Cyclo™ to meet the considerable unmet medical needs in NPC treatment. He emphasized the importance of slowing down disease progression through cholesterol mobilization to prevent neurodegeneration in untreated patients. The company is eagerly awaiting more data to further assess the drug's impact on NPC progression.

Cyclo Therapeutics CEO, N. Scott Fine, also commented on the encouraging data from Trappsol® Cyclo™ in NPC treatment, acknowledging the devastating nature of the disease and the significant unmet need for effective therapies. The company anticipates interim data from the TransportNPC™ study in the first half of 2025, which could pave the way for a new treatment option for NPC patients.

The TransportNPC™ study is a randomized, double-blind, placebo-controlled, multicenter study designed to evaluate the safety, tolerability, and efficacy of 2,000 mg/kg doses of Trappsol® Cyclo™ compared to a placebo. The study spans 96 weeks, with a 48-week interim analysis. Meeting the statistical significance in interim data could lead the company to submit marketing applications based on these findings.

At the symposium, Dr. Caroline Hastings also presented posters on Trappsol® Cyclo™, focusing on its long-term efficacy and safety in NPC1 treatment. The company’s website hosts these presentations for further information.

Cyclo Therapeutics has received Orphan Drug Designation for Trappsol® Cyclo™ in the U.S. and EU, along with Fast Track and Rare Pediatric Disease Designations in the U.S., underscoring the potential of this investigational therapy for NPC1.