Cyclo Therapeutics, a clinical stage biotechnology company that develops cyclodextrin-based products for the treatment of Niemann-Pick Disease Type C (NPC) and Alzheimer’s Disease, today announced that 3 additional patients have completed the 48-week Phase I/II study. Results show encouraging trends in efficacy, consistent with findings of the first four patients who completed the study, as reported in the company’s Interim Analysis in May 2020.
The group data show that six of the seven patients (86% of those who completed) met the primary endpoint of the Phase I/II study, which was to achieve at least a one-point reduction in two or more of the 17-Domain NPC Clinical Severity Scale (CSS) measures. Domains in which improvement were seen varied from patient to patient, in keeping with the heterogeneous nature of the disease. Features that improved included swallowing, ambulation, ability to manage seizures, saccadic eye movements, fine motor skills and cognition. Patients whose overall NPC Severity Scores showed worsening still had areas of improvement, with only one patient who worsened without having a single domain of improvement.
Additionally, five of seven patients also improved in the Clinician’s Global Impression of Improvement scale (CGI-I), a second primary outcome measure. Of the five patients (71% of those who completed), one improved very much; two were much improved; two were minimally improved. The other 2 patients remained unchanged. Per the treating physicians, no change in score or stabilization can be viewed as a positive outcome given the progressive nature of the disease.
“The data on these 3 additional patients provide valuable insights as we continue to collaborate with both the FDA and EMA on our global Phase III pivotal program,” said Cyclo Therapeutics’ Chairman and CEO, N. Scott Fine. “Of those 7 patients who have completed the study, 5 improved in at least one of the 5-Domain NPC Clinical Severity Scale (5D-NPC-CSS) measure. This measure of the five domains (Swallow, Fine Motor, Ambulation, Cognition and Speech) is determined by NPC families and their caregivers in collaboration with the FDA to be the most important for their quality of life.”