Cyclo Therapeutics Shares Promising Initial Safety Data from Phase 3 NPC1 Study

Cyclo Therapeutics, Inc., a biotechnology company focused on developing transformative medicines, has shared promising preliminary results from its ongoing pivotal Phase 3 study, TransportNPC™, and a related substudy on Trappsol® Cyclo™ for treating Niemann-Pick Disease Type C1 (NPC1). These findings were unveiled at the SSIEM Annual Symposium 2024 in Porto, Portugal.

The TransportNPC™ study is a major, globally controlled clinical trial designed to assess the efficacy and safety of Trappsol® Cyclo™ in NPC1 patients. This comprehensive study, which had completed patient enrollment by May 2024, includes 104 participants and evaluates various clinical outcomes. Additionally, a substudy involving ten infants under the age of three has been conducted as part of the Paediatric Investigational Plan (PIP), aiming to understand the treatment's impact on younger patients, especially in addressing the visceral symptoms of NPC1.

N. Scott Fine, CEO of Cyclo Therapeutics, expressed satisfaction with the study's progress and emphasized the significant potential of Trappsol® Cyclo™ in meeting the critical need for effective NPC1 therapies. He highlighted the company's commitment to advancing this global program, with interim data expected in the first half of 2025.

Professor Caroline Hastings, MD, the Global Principal Investigator for TransportNPC™, echoed these sentiments, noting the transformative potential of Trappsol® Cyclo™ for NPC patients. She emphasized the encouraging preliminary safety data, suggesting that the treatment could address both systemic and neurological symptoms, which are crucial for the NPC community.

Key highlights from the poster presentations include:

TRANSPORTNPC™: Phase 3 Global Trial
The Phase 3 study involves a 96-week, double-blind, placebo-controlled assessment of Trappsol® Cyclo™. The primary endpoint for the EU is the change in 5D-NPC-CSS scores, which measure ambulation, fine motor skills, speech, swallowing, and cognition. The study's participants have an average age of 20.2 years, with a broad age range from 3 to 65 years. As of June 30, 2024, participants had been in the blinded phase for an average of 44 weeks. Six patients exited the study before week 96, and another six transitioned to open-label Trappsol® Cyclo™ due to disease worsening.

Safety and Tolerability
A total of 625 adverse events (AEs) were reported, with 80% being mild, 16.7% moderate, and 5.3% severe. There were nine serious adverse events (SAEs), some possibly related to the treatment. Importantly, no patients withdrew due to safety concerns, and the overall safety profile was consistent with previous studies. Topline data from a 48-week interim analysis is anticipated in the first half of 2025. If the interim results are statistically significant, the company plans to seek regulatory approval based on this data.

TRANSPORTNPC™ Substudy for Patients Under 3 Years
This substudy focuses on evaluating the safety of biweekly IV Trappsol® Cyclo™ (2000 mg/kg) for 192 weeks in infants and very young children. Ten patients were enrolled, with two exiting the study after 48 weeks. The adverse event profile was consistent with earlier findings, with most events being mild or moderate. No SAEs were linked to the study drug. The substudy aims to provide critical insights into early intervention and disease progression prevention in this age group.

By the first half of 2025, all currently enrolled patients will have completed at least 48 weeks of treatment, and some will have reached 96 weeks. This data will be pivotal in understanding the treatment's long-term effects on young NPC1 patients.

Cyclo Therapeutics, Inc. continues to drive forward with its mission to develop life-changing therapies. Trappsol® Cyclo™, designated as an orphan drug in both the U.S. and Europe, is undergoing multiple clinical trials for NPC1 and other potential indications, including early Alzheimer's disease.

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