Cyclo Therapeutics, Inc., a clinical-stage biotechnology firm, recently announced the completion of patient enrollment for its pivotal Phase 3 study, TransportNPC™, which examines Trappsol® Cyclo™ for treating systemic and neurological symptoms of Niemann-Pick Disease Type C1 (NPC1). This study is notable for being the most comprehensive in terms of patient size, global reach, duration, and clinical outcomes for NPC1.
The TransportNPC™ study has enrolled 104 patients, including 94 in the main study and an additional 10 in a substudy focusing on newborns to children up to three years old. This substudy aims to evaluate the effect of Trappsol® Cyclo™ on the youngest patients, targeting visceral aspects of the disease with the potential to prevent overall symptom development if administered early.
Cyclo Therapeutics anticipates topline data from the 48-week interim analysis by the first half of 2025. If this data is significant, the company plans to submit a New Drug Application (NDA) to the FDA and a Marketing Authorization Application (MAA) to the EMA in the latter half of 2025. The NDA submission could qualify the company for a Priority Review Voucher (PRV).
N. Scott Fine, CEO of Cyclo Therapeutics, emphasized the importance of reaching this milestone, noting that half of the 104 patients will have completed the 96-week endpoint at the time of the 48-week analysis. This dual-timepoint data will provide essential long-term insights to support the company's submission to health authorities. Fine expressed optimism about the potential impact of this treatment for the NPC community.
Professor Caroline Hastings, MD, the global principal investigator for the TransportNPC™ study, highlighted the progress of the trial and its potential to offer a much-needed treatment option for NPC, which currently lacks effective approved treatments for its systemic and neurological symptoms. With nearly two decades of research, the study aims to transform the treatment landscape for NPC.
Joshua Fine, CFO of Cyclo Therapeutics, acknowledged the continued support from investors, including Rafael Holdings, Inc., and other long-term shareholders, as the company moves towards achieving clinical development goals.
The TransportNPC™ study is designed as a randomized, double-blind, placebo-controlled, parallel group, multicenter trial. It evaluates the safety, tolerability, and efficacy of 2,000 mg/kg doses of Trappsol® Cyclo™ administered intravenously alongside the standard of care (SOC), compared to a placebo and SOC alone. NPC1 is a rare genetic condition causing cholesterol accumulation in cells, leading to dysfunction in the liver, lungs, spleen, brain, and premature death. The study spans 96 weeks with a 48-week interim analysis. Depending on the interim results, Cyclo Therapeutics plans to seek marketing approval.
The single-arm substudy focuses on evaluating NPC1 patients from birth to under three years of age, regardless of symptoms, to gather descriptive data on disease severity and response to Trappsol® Cyclo™. Conducted outside the U.S., this substudy follows an adopted Pediatric Investigational Plan (PIP).
Cyclo Therapeutics has received Orphan Drug Designation for Trappsol® Cyclo™ in both the U.S. and the EU, along with Fast Track and Rare Pediatric Disease Designations in the U.S., the latter of which is crucial for securing a Priority Review Voucher.
Niemann-Pick Disease Type C1 (NPC) is an uncommon genetic disease affecting approximately 1 in 100,000 live births worldwide. It is characterized by mutations in the NPC1 or NPC2 genes, leading to cholesterol transport and processing issues within cells, which causes severe systemic and neurological symptoms and often results in premature death.
Cyclo Therapeutics is committed to developing transformative medicines through scientific innovation, focusing on conditions like NPC1. The company's Trappsol® Cyclo™ is currently under investigation in multiple clinical trials and has shown promise in early Alzheimer's disease treatment.
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