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Ovid Therapeutics Announces First Patient Randomized in Pivotal Phase 3 NEPTUNE Trial of OV101 for the Treatment of Angelman Syndrome
OV101 May Become the First Medicine for Individuals with Angelman Syndrome; Topline Results Expected in Mid-2020 NEW YORK, Sept. 12, 2019 (GLOBE NEWSWIRE) --
About this update from Ovid Therapeutics Inc.
[{"type":"text","content":"OV101 May Become the First Medicine for Individuals with Angelman Syndrome; Topline Results Expected in Mid-2020\nNEW YORK, Sept. 12, 2019 (GLOBE NEWSWIRE) -- Ovid Therapeutics Inc. (NASDAQ: OVID), a biopharmaceutical company committed to developing medicines that transform the lives of people with rare neurological diseases, today announced that the first patient has been randomized in the company’s single, pivotal Phase 3 NEPTUNE clinical trial evaluating OV101 (gaboxadol), a novel delta (δ)-selective GABAA receptor agonist, in Angelman syndrome. The company anticipates topline data from the trial to be available in mid-2020.\n “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 3 NEPTUNE trial is a major step toward establishing the clinical effectiveness of OV101 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 3 clinical trial designed to assess the effects of treatment with OV101 (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 OV101’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. “In the U.S., it is estimated there are approximately 24,000 individuals with Angelman syndrome,” said Amanda Moore, CEO of the Angelman Syndrome Foundation. “Those with Angelman syndrome require 24-hour care throughout their lives and are unable to live independently. The Angelman synd...