Cyclo Therapeutics completes enrollment in TransportNPC Trial of NPC1

Cyclo Therapeutics announced the last patient has been enrolled in the Company’s pivotal Phase 3 study evaluating Trappsol Cyclo for the treatment of systemic and neurological symptoms of Niemann-Pick Disease Type C1 or NPC1. The TransportNPC study is the most comprehensive controlled pivotal study regarding patient size, global footprint, duration and clinical outcomes of an investigational therapy for NPC1. The study has dosed its 93rd and 94th patients. Additionally, the Company has enrolled ten patients in its substudy per their adopted Paediatric Investigational Plan treating newborn to 3 years of age. The substudy is evaluating Trappsol Cyclo in the youngest age subsets as it targets also the visceral aspects of the disease and may achieve its most optimal results when administered early in the disease course, thus having the potential of a preventative effect in overall symptom development. “The completion of enrollment represents by far the most significant milestone for Cyclo Therapeutics to date. Not only is this study the largest controlled pivotal study for NPC1 ever to be conducted with 104 patients enrolled, but we will also have approximately half of those 104 patients who completed the 96-week timepoint at the time of our 48-week interim analysis, thus providing important additional long-term data supporting our potential submissions to the Health Authorities,” commented N. Scott Fine, Chief Executive Officer of Cyclo Therapeutics. “With the positive support, feedback and alignment from our recent health authority interactions with both the FDA and EMA, we are excited to take this critical step toward the interim data readout, which is expected in H1 2025 and most importantly, the potential to provide a much-needed treatment option for the NPC community.”