First patient enrolled in the pivotal phase III NEPTUNE trial of OV 101 for treatment of Angelman syndrome.- Ovid Therapeutics.

Ovid Therapeutics Inc. announced that the first patient has been randomized in the company�s single, pivotal Phase III NEPTUNE clinical trial evaluating OV 101 (gaboxadol), a novel delta (?)-selective GABAA receptor agonist, in Angelman syndrome. The company anticipates topline data from the trial to be available in mid-2020.

�There are currently no other therapies in clinical development for Angelman syndrome and no approved treatments for this disorder exist today,� said Amit Rakhit, MD, MBA, Chief Medical Officer and Head of Research & Development at Ovid. �We believe the first patient randomized in the pivotal Phase III NEPTUNE trial is a major step toward establishing the clinical effectiveness of OV 101 in Angelman syndrome and one that we hope will result in the first approved medicine for individuals living with this rare neurologic condition.�

NEPTUNE is a randomized, double-blind, placebo-controlled, Phase III clinical trial designed to assess the effects of treatment with OV 101 (oral, once-daily dosing) versus placebo over 12 weeks. The trial will include approximately 60 pediatric patients ages 4 to 12 years diagnosed with Angelman syndrome. The sole primary endpoint will be change in overall score on the Clinical Global Impression-Improvement-Angelman syndrome (CGI-I-AS) scale. Secondary endpoints include sleep, communication, motor function, socialization, daily living skills and behavior domains. A limited number of children, ages 2 to 3 years, will also be enrolled in the study to evaluate OV 101�s pharmacokinetics (PK) along with safety and tolerability in this younger age group. Participants who complete the NEPTUNE trial will be eligible to roll over into the ongoing open-label extension trial (ELARA) should they choose to do so..

.As previously announced, based on Ovid�s interactions with regulatory authorities in the U.S. and Germany regarding a Phase III program and registration path, Ovid, the FDA and Germany�s Federal Institute for Drugs and Medical Devices (BfArM) agree on the study design of the Phase III NEPTUNE trial, including the use of CGI-I-AS as the primary efficacy outcome measure. Both the FDA and BfArM agreed that NEPTUNE, if positive, could support the filings of a New Drug Application (NDA) and Marketing Authorization Application (MAA), respectively.