Phase III trial is initiated for pediatric patients for OV 101 in Angelman Syndrome. Ovid Therapeutics

Ovid Therapeutics Inc. announced plans to move ahead with a single pivotal Phase III trial of OV 101 in pediatric patients with Angelman syndrome based on its End-of-Phase II Meeting with the FDA. If successful, the Phase III efficacy and safety trial called NEPTUNE is intended to support a New Drug Application (NDA) for OV 101 in Angelman syndrome.

OV 101 is a novel delta-selective GABAA receptor agonist. Angelman syndrome is a rare genetic disorder that is characterized by a heterogeneous constellation of symptoms, including disturbances in communication, sleep, motor function, and behavior, and for which there are no approved medicines or established treatment paradigms.

Based upon review and discussion of the Phase II 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 III 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 OV 101. 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.