Cyclo Therapeutics presented data from its pivotal Phase 3 TransportNPC™ trial of Trappsol® Cyclo™ for Niemann-Pick Disease Type C1 (NPC1) at the 21st Annual WORLDSymposium™. The data highlighted positive results from a sub-study of the trial focusing on children aged newborn to three years, showing stabilization or improvement in a majority of patients after 24 and 48 weeks of treatment. This sub-study, conducted outside the US, explores Trappsol® Cyclo™’s potential to impact the visceral manifestations of NPC1 and possibly prevent overall symptom progression when administered early.
This research is vital for NPC1 patients, particularly those diagnosed very young, as current treatment options are limited and the disease progresses rapidly, often leading to premature death. Demonstrating efficacy in this young age group could significantly alter the disease trajectory and improve long-term outcomes, offering a potential preventative approach. This is especially crucial considering NPC1’s impact on multiple organs, including the brain, liver, spleen, and lungs.
The Phase 3 TransportNPC™ trial is a randomized, double-blind, placebo-controlled study comparing Trappsol® Cyclo™ with standard of care to placebo and standard of care. It includes a 48-week interim analysis. If this interim analysis demonstrates statistical significance, the company plans to submit marketing applications based on these findings. The sub-study focusing on younger patients complements the broader trial, investigating the drug’s impact at different stages of the disease.
Positive results from both the main trial and the sub-study could lead to the first disease-modifying therapy specifically targeting early intervention in NPC1. This could drastically improve the quality of life for these young patients and their families while also paving the way for further research into preventative strategies for other rare genetic diseases. The anticipated interim data readout in the first half of 2025 is a critical milestone for the company and the NPC1 community.
Jon Napitupulu is Director of Media Relations at The Clinical Trial Vanguard. Jon, a computer data scientist, focuses on the latest clinical trial industry news and trends.
