Ovid Therapeutics (NASDAQ: OVID) reported that its Phase 3 NEPTUNE trial evaluating OV101 for the treatment of Angelman syndrome failed to meet its primary endpoint.
Angelman syndrome is a rare genetic disorder that affects the brain’s ability to correctly process excitatory and inhibitory neurological signals, a function of a specific GABA receptor. OV101, or gaboxadol, is a selective agonist of that receptor.
The trial’s primary endpoint was the change in overall score on the clinical global impression-improvement-Angelman syndrome scale. Patients who received OV101 showed a 0.7-point improvement over baseline, compared with a 0.8-point improvement in patients who received placebo.
In a statement, Amit Rakhit, M.D., Ovid’s president and CMO, said, “NEPTUNE is our first study focused on the pediatric and adolescent population in Angelman syndrome, and we will fully assess all the data from this trial to understand this outcome and determine next steps, if any, for OV101 in this and other conditions, including Fragile X syndrome.”
The company will pause the OV101 program in Angelman syndrome pending full analysis of trial data and discussions with FDA.
