Cyclo Therapeutics Presents Positive Phase 3 TransportNPC™ Data in Children Under 3
Cyclo Therapeutics, Inc. (Nasdaq: CYTH), a clinical-stage biotechnology company focused on developing life-changing treatments for patients with rare and challenging diseases, today announced the presentation of data from its ongoing pivotal Phase 3 global study, TransportNPC™, at the 21st Annual WORLDSymposium™ in San Diego, CA. The study is evaluating Trappsol® Cyclo™ for the treatment of Niemann-Pick Disease Type C1 (NPC1).
The study is considered one of the most comprehensive pivotal studies of an investigational therapy for NPC1 due to its large patient sample, global scope, duration, and clinical outcomes.
The TransportNPC™ study completed enrollment in May 2024. The company also enrolled 10 patients in a single-arm sub-study, per its adopted Pediatric Investigational Plan (PIP), that focuses on treating newborns to children under 3 years of age. Two patients discontinued the sub-study after 48 weeks due to caregiver decisions. The sub-study is crucial as it targets the visceral aspects of NPC1, with Trappsol® Cyclo™ showing potential in delivering optimal results when administered early in the disease course, potentially acting preventatively to limit symptom development. The study is being conducted in countries outside of the United States, in line with the company’s PIP.
Dr. Ronen Spiegel, Clinical Associate Professor and Director of Pediatric B Department at Emek Medical Center, presented an oral session at WORLDSymposium™ titled “Trappsol® Cyclo™: Open Label Treatment in the TransportNPC™ Sub-Study in Patients Under the Age of 3 Diagnosed with Niemann-Pick Disease Type C1.”
Key Data Highlights:
- Clearance of lipids, both centrally and systemically, was consistently shown in the 14-week Phase 1 study of adult NPC patients.
- At baseline, sub-study patients had a mix of mild to severe disease, based on the Clinical Global Impression – Severity (CGI-S) scale. Clinical improvements were most significant in patients with clinically mild to moderate disease.
- Seven of eight patients who reached 24 weeks and six of seven who reached 48 weeks showed stabilization or improvement in CGI-C.
- Adverse event (AE) profile aligned with previous studies, including a double-blind Phase 3 study running concurrently. As of December 2024, out of 107 AEs, the majority (77%) were mild, 22% were moderate, and 1 AE was severe. Only one mild AE was considered possibly related to the study drug, with no serious AEs linked to the drug.
Dr. Spiegel commented, “The preliminary findings from the TransportNPC™ sub-study are promising. Trappsol® Cyclo™ shows potential in addressing the unmet medical need for NPC patients, slowing disease progression through cholesterol mobilization, particularly for those with established disease who would otherwise face neurodegeneration. We look forward to further data from the ongoing study.”
N. Scott Fine, CEO of Cyclo Therapeutics, said, “We’re encouraged by the data from the TransportNPC™ study and sub-study. This growing body of evidence continues to show the potential of Trappsol® Cyclo™ in treating NPC, a disease with immense unmet need. We look forward to interim data from the TransportNPC™ study in H1 2025 and hope to provide a new treatment option for patients soon.”
TransportNPC™ Study Overview
The TransportNPC™ study is a randomized, double-blind, placebo-controlled, multicenter study aimed at evaluating the safety, tolerability, and efficacy of 2,000 mg/kg doses of Trappsol® Cyclo™ administered intravenously, compared to placebo and standard of care. The study focuses on NPC1, a rare genetic disorder that leads to cholesterol accumulation in cells, affecting organs such as the liver, lung, spleen, and brain, and causing premature death. The study lasts for 96 weeks, with a 48-week interim analysis. If the 48-week data meets statistical significance, Cyclo Therapeutics plans to submit marketing applications to the FDA and EMA.
In addition to Dr. Spiegel’s oral presentation, two posters were presented at WORLDSymposium™. Dr. Caroline Hastings, Pediatric Hematologist-Oncologist at UCSF Benioff Children’s Hospital, presented data from four clinical studies and the ongoing expanded access program for Trappsol® Cyclo™ in NPC. Dr. Spiegel also presented a poster on the sub-study for patients under 3 years of age with NPC1.
Cyclo Therapeutics’ Trappsol® Cyclo™ has received Orphan Drug Designation in the U.S. and EU for NPC1, along with Fast Track and Rare Pediatric Disease Designations in the U.S. The latter designation could grant the company a Priority Review Voucher upon marketing authorization.
About WORLDSymposium™
WORLDSymposium™ is an annual research conference focused on lysosomal diseases. Since its inception in 2002, the conference has grown into a major international event, attracting over 2,000 participants from more than 50 countries. The conference provides a platform for researchers to share the latest advances in lysosomal disease research and therapy.
