Cyclo Therapeutics presents data from study, substudy of Trappsol Cyclo

Cyclo Therapeutics announced the presentation of preliminary data from its ongoing pivotal Phase 3 study and substudy evaluating Trappsol Cyclo for the treatment of Niemann-Pick Disease Type C1. The data were presented in two poster presentations at the SSIEM Annual Symposium 2024 being held September 3-6 in Porto, Portugal. 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 completed enrollment in May. Additionally, the company completed enrollment with 10 patients in a substudy per the adopted Paediatric Investigational Plan, or PIP, treating newborn to three years of age. The substudy is evaluating Trappsol Cyclo in the youngest age groups, focusing on its ability to target the visceral aspects of the disease. Administering treatment early in the disease course may lead to the most optimal results and could potentially have a preventative effect on overall symptom development. The company’s ongoing Phase 3 study will evaluate change in 5D-NPC-CSS between Trappsol Cyclo and placebo as primary endpoint for the EU. A total of 104 patients have been enrolled into the study and substudy, the majority of patients enrolled fall within the ASIS score limits targeted with the average ASIS at recruitment was 1.04; average age at inclusion was 20.2 years. Average participation time in the blinded phase of the study is 44 weeks as of June 30, 2024. As of June 30, six patients terminated study participation before week 96; following protocol, six patients switched to open-label Trappsol Cyclo before week 96 due to disease worsening. A total of 625 adverse events were reported, 80% were Grade 1 in severity most related to underlying NPC disease; Grade 2 were 16.7%, and Grade 3 were 5.3%. Serious adverse events reported, possibly related or related to treatment. One patient death unrelated to study-drug. No patients have withdrawn due to safety concerns. Safety profile found consistent with that of completed studies. Topline data from the 48-week interim analysis is anticipated for H1 2025. 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 goal of the substudy is to evaluate the safety of biweekly IV Trappsol Cyclo for 192 weeks in very young patients, in order to obtain descriptive data regarding safety, global severity, and improvement in response to HPssCD from investigators and patients/caregivers. Ten patients were recruited, of which two terminated the study after 48 weeks. AE profile is in line with prior findings from compassionate use, from earlier studies, and from a double-blind Phase 3 study running in parallel. Overall, AEs are limited, majority are mild or moderate and 1 AE severe; most considered unrelated to study drug. No SAEs were considered as related to or possibly related to study drug. At baseline patients had a mixture of very mild to severe disease based on CGI. Progress in patients with neurological symptoms from the early pediatric age is understood to be more rapid than in patients with late pediatric, juvenile, or even adult neurological onset of NPC. Disease progression is carefully monitored throughout duration of the study. The substudy has completed enrollment and the treatment has been well tolerated with a safety profile consistent with prior studies, including the larger TransportNPC study. In addition to the clinical endpoints, this study will provide additional critical information to address the concept of early intervention and prevention of disease progression. By the first half of 2025, all currently enrolled patients will have completed at least 48 weeks of treatment; Three patients will likely have completed 96 weeks of treatment. This will be the first data in NPC1 on treatment in this age group over a period of 48 weeks.