Ovid's fragile X drug shows promise in phase 2

Ovid Therapeuctics’ lead program improved behavioral symptoms, as well as anxiety and mood, in a small phase 2 study in fragile X syndrome. With no serious side effects seen in three different dose regimens, the company is gearing up for a meeting with regulators to discuss the drug’s path forward.

Ovid is developing the drug, known as OV101 or gaboxadol, for the treatment of Angelman syndrome, a nervous system disorder, as well as fragile X, the most common inherited form of intellectual disability and autism. The company tested the drug in 23 patients over 12 weeks, finding a 26.2% mean improvement from their baseline scores on the Aberrant Behavior Checklist-Community for fragile X syndrome, a measure of psychiatric symptoms and behavioral disturbance. Patients also logged a 21.6% mean improvement on a measure of anxiety, depression and mood.

The drug hit its safety and tolerability goals as well—there were no serious side effects, and most of the side effects, 94%, were classed as mild. The most common side effect, upper respiratory infections, affected less than one-fifth of the patients. Of the three patients who quit the study, one did so because of side effects (increased agitation).

RELATED: Journey of a rare disease drug: Ovid’s OV101 for Angelman syndrome

“This was the first interventional clinical study of OV101 in the Fragile X population, and we are encouraged by the safety profile and the positive efficacy signals we see across multiple behavior domains from this small, active-arm study,” said Amit Rakhit, M.D., Ovid’s president and chief medical officer, in a statement.

The two lower doses—5 mg once daily and 5 mg twice a day—seemed to do better than the thrice-daily dose, “which is consistent with the emerging profile of OV101,” he said.

“We are in the process of further evaluating which specific dose to advance in future studies, and we look forward to moving this program forward to discussions with regulators to determine next steps for the development of OV101 in the Fragile X indication,” Rakhit added.

Caused by a mutation in the FMR1 gene, fragile X gives rise to various behavioral challenges such as cognitive impairment, mood swings, hyperactivity, poor sleep and a heightened sensitivity to stimuli, like sound. There is no specific treatment for the syndrome, so treatment focuses on managing symptoms. OV101 is a delta-selective GABAA receptor agonist designed to target the disruption of a process in the brain believed to be the underlying cause of certain neurodevelopmental disorders.