GAINESVILLE -
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 (final) and 94th (over enrolled) patients. Additionally, the Company has enrolled ten (10) patients in its substudy per their adopted Paediatric Investigational Plan (PIP) 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
Professor
'We are grateful for the continued support of our investors, including
The Company's ongoing TransportNPC study is a randomized, double-blind, placebo-controlled, parallel group, multicenter study designed to evaluate the safety, tolerability, and efficacy of 2,000 mg/kg doses of Trappsol Cyclo administered intravenously and standard of care (SOC), compared to placebo administered intravenously and SOC alone, in patients with NPC1, a rare, genetic disease causing cholesterol accumulation in cells, leading to dysfunction of the liver, lung, spleen and brain and premature death. The study duration is a 96-week study, with a 48-week comparative interim analysis. Should the 48-week interim data meet statistical significance, the Company, in alignment with the FDA and EMA, intends to submit marketing applications for approval based on the 48-week interim data.
The Company's ongoing single-arm sub-study is evaluating patients from birth to less than 3 years of age with NPC1 irrespective of symptoms to evaluate safety and to obtain descriptive data on global disease severity and the response to Trappsol Cyclo. The substudy is being conducted in countries outside of
About Niemann-Pick Disease Type C1 (NPC)
NPC is a rare genetic disease affecting 1 in 100,000 live births globally. Approximately 95% of individuals with NPC have mutations in the NPC1 gene and 5% have mutations in the NPC2 gene. NPC affects nearly every cell in the body due to a deficiency in either the NPC1 or NPC2 protein, which are required for the transport and processing of cholesterol within the cell. As cholesterol accumulates within cells, NPC causes symptoms that affect the brain, liver, spleen, lung, and other organs and often leads to premature death.
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