Trappsol Cyclo Shows Promise in Stabilizing or Improving Niemann-Pick Disease Type C1 in Young Patients
核心洞察
Preliminary data from Cyclo Therapeutics' TransportNPC trial shows that 87% of patients under three years old with Niemann-Pick Disease Type C1 (搜索) (NPC1) demonstrated stabilization or improvement after 24 weeks of Trappsol Cyclo treatment.
Six out of seven patients who continued Trappsol Cyclo treatment for 48 weeks showed continued improvement, suggesting sustained efficacy over a longer duration.
Trappsol Cyclo targets cholesterol mobilization, addressing neurodegeneration associated with NPC1, and Cyclo Therapeutics plans to seek regulatory approval from the FDA and EMA based on these results.
Cyclo Therapeutics has announced positive preliminary data from its Phase III TransportNPC trial, indicating that Trappsol Cyclo, a hydroxypropyl beta cyclodextrin formulation, shows promise in treating Niemann-Pick Disease Type C1 (搜索) (NPC1) in young patients. The open-label sub-study within the TransportNPC trial (NCT04860960) evaluated Trappsol Cyclo in eight patients under the age of three. The results, presented at the 21st Annual WORLDSymposium, revealed that 87% of participants who reached the 24-week mark showed signs of clinical improvement or stabilization, as measured by the Clinical Global Impression – Change (CGI-C) Scale.
Encouraging Results in Young NPC1 Patients
The TransportNPC trial is a Phase III double-blind, placebo-controlled, multi-center study designed to assess the safety and tolerability of Trappsol Cyclo in 104 patients. The sub-study focused on younger patients to evaluate the drug's impact on the visceral aspects of the disease, with the hypothesis that early intervention may have a preventative effect on overall symptom development. Of the patients who continued onto week 48, six out of seven showed continued improvement, suggesting a sustained benefit with longer-term treatment.
Clinical Perspective
Dr. Ronen Spiegel, trial investigator and director for the Center of Rare Disease at Emek Medical Center, commented on the findings: "The preliminary findings from the TransportNPC sub-study are encouraging and further underscore the potential of Trappsol Cyclo to address the significant unmet medical need in the treatment of NPC. Slowing down disease progression through cholesterol mobilization is an important consideration for patients with established disease, who can expect to experience neurodegeneration without treatment."
Niemann-Pick Disease Type C1
Niemann-Pick Disease Type C1 (搜索) is a rare genetic disorder affecting approximately 1 in 100,000 live births globally. It is characterized by mutations in the NPC1 or NPC2 genes, leading to the accumulation of cholesterol in cells and causing dysfunction in the brain, liver, spleen, lungs, and other organs. This accumulation often results in neurodegeneration and premature death. Trappsol Cyclo aims to address this by facilitating cholesterol mobilization.
Regulatory Pathway
Cyclo Therapeutics is now planning to use the positive data from this sub-group to support marketing applications to the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA). The company anticipates an interim data readout from the main TransportNPC study in the first half of 2025. N. Scott Fine, Chief Executive Officer of Cyclo Therapeutics, stated, "We continue to be encouraged by the data demonstrated by Trappsol Cyclo in the treatment of NPC... We look forward to our interim data readout from the TransportNPC study, which is expected in H1 2025 and ultimately providing a much needed treatment option for patients."
Study Design and Further Presentations
The ongoing TransportNPC study is evaluating 2,000 mg/kg doses of Trappsol Cyclo administered intravenously, compared to placebo, in patients with NPC1 over a 96-week period, with a 48-week interim analysis. In addition to the oral presentation by Dr. Spiegel, Cyclo Therapeutics presented two posters at the WORLDSymposium, highlighting long-term efficacy and safety data, as well as the drug's impact across individual 5D domains.