Ovid Therapeutics’ First Patient Randomized in Pivotal Phase 3 NEPTUNE Trial

Ovid Therapeutics (NASDAQ:OVID) has announced it has randomized the first patient in its Phase 3 NEPTUNE clinical trial of OV101 to treat Angelman syndrome.

As quoted in the press release:

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 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.

Click here to read the full press release.