Ovid Therapeutics announces phase III NEPTUNE clinical trial of OV 101 for the treatment of Angelman syndrome did not meet primary endpoint.

Ovid Therapeutics Inc. announced topline results from the Company’s Phase III NEPTUNE clinical trial of OV 101 (gaboxadol) for the treatment of Angelman syndrome. NEPTUNE is a randomized, double-blind, placebo-controlled, Phase III study that enrolled and treated 97 patients diagnosed with Angelman syndrome, 4-12 years of age, and 7 patients diagnosed with Angelman syndrome ages 2-3 years for safety and pharmacokinetic evaluation only. The study was designed to assess the effects of treatment with OV 101 (oral, once-daily dosing) versus placebo over 12 weeks. The sole primary endpoint was change in overall score on the Clinical Global Impression-Improvement-Angelman syndrome (CGI-I-AS) scale. Secondary endpoints included sleep, communication, motor function, socialization, daily living skills and behavior domains. The primary endpoint of the NEPTUNE study was not achieved. Patients given OV 101 showed a 0.7 point improvement in CGI-I-AS over baseline while placebo also showed a 0.8 point improvement in CGI-I-AS (p=NS). Secondary endpoints continue to be evaluated, although initial results show no difference between OV 101 and placebo.