Ovid Therapeutics to move into Phase 3 OV101 trial in pediatric patients

Ovid Therapeutics announced plans to move ahead with a single pivotal Phase 3 trial of OV101 in pediatric patients with Angelman syndrome based on its End-of-Phase 2 Meeting with the U.S. Food and Drug Administration. If successful, the Phase 3 efficacy and safety trial called NEPTUNE is intended to support a New Drug Application for OV101 in Angelman syndrome. OV101 is a novel delta-selective GABAA receptor agonist. Based upon review and discussion of the Phase 2 STARS trial results, the clinical trial design, and the applicability of Clinical Global Impressions of Improvement (CGI-I) as a primary endpoint, the FDA and Ovid are in general agreement that the pivotal Phase 3 NEPTUNE clinical trial to be completed prior to an NDA submission, will include the following elements: Single 12-week, two-arm, randomized, double-blind, placebo-controlled trial; Once-daily dose; Approximately 50-60 pediatric patients aged 4 to 12 years, diagnosed with Angelman syndrome randomized to either placebo or OV101; Primary endpoint of change in overall CGI-I score. CGI-I will be used as a single primary endpoint, mainly due to the rare nature of Angelman syndrome, the lack of treatment options, the nonexistence of assessment instruments specific to Angelman syndrome, the heterogeneity of the disorder, and the lack of sensitivity or appropriateness of other validated measures. Based on feedback from the FDA, Ovid will develop a framework for study investigators to ensure uniform use of the validated CGI-I scale by focusing on specific symptoms that are relevant and important to patients with Angelman syndrome and their caregivers. Ovid expects to begin enrollment of the NEPTUNE trial in the second half of 2019, pending FDA concurrence on the study protocol and supporting framework and materials. Updates and further details of this trial, including secondary endpoints, will be provided upon trial initiation. In addition to the planned Phase 3 NEPTUNE trial, Ovid is initiating the ELARA clinical trial, an open-label extension study for individuals with Angelman syndrome who have completed any prior OV101 study. ELARA will use once-daily dosing and will assess long term safety and tolerability in addition to efficacy measures. Trial initiation activities are underway. The company is also exploring options for patients under four years of age.
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