Ovid Therapeutics (NASDAQ:OVID) reported positive topline results from its Phase 2 trial of OV101 in males with Fragile X syndrome.
A total of 23 participants were randomized to receive either 5 mg of OV101 either once, twice, or three times daily.
The trial met its primary objective of demonstrating OV101’s safety and tolerability. OV101 also demonstrated statistically significant reductions in behavioral and functional symptoms, including a 26.2% mean improvement in Aberrant Behavior Checklist-Community for Fragile X syndrome total score and a 21.6% mean improvement in Anxiety, Depression and Mood Scale total score, both from baseline to week 12.
“Developing treatment options in Fragile X syndrome has historically been challenging, and there continues to be a high unmet medical need,” Dr. Amit Rakhit, Ovid’s president and CMO, said in a statement.
“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. Lower and middle dosing regimens appear to be superior to the higher, three-times-daily dose, which is consistent with the emerging profile of OV101,” he added.
