Ovid Therapeutics Inc. (NASDAQ:OVID), a biopharmaceutical company committed to developing medicines that transform the lives of people with rare neurological diseases, today announced positive topline results from the signal-finding Phase 2 ROCKET trial of OV101 (gaboxadol), a novel delta (δ)-selective GABAA receptor agonist, in males with Fragile X syndrome. Fragile X syndrome is the most common inherited form of intellectual disability and autism, with no approved therapies. In addition, Ovid today provided preliminary findings from SKYROCKET, a non-drug interventional study in Fragile X syndrome.
The ROCKET trial was a signal-finding, randomized, double-blind, parallel-group trial to evaluate the safety, tolerability and efficacy of OV101 in males ages 13 to 22 with a confirmed diagnosis of Fragile X syndrome. The primary objective of the study was to assess the safety and tolerability of OV101 over 12 weeks of treatment in three different active dose arms. The secondary objective was to evaluate changes in behavior after 12 weeks of treatment. A total of 23 participants were randomized into the study across three active-arm dose cohorts: OV101 5 mg once-daily (QD), OV101 5 mg twice-daily (BID), and OV101 5 mg three-times-daily (TID).
OV101 met its primary objective and appeared to be well tolerated over 12 weeks of treatment with no serious adverse events reported across all three dose cohorts. OV101 demonstrated a statistically significant effect on secondary behavioral endpoints in the three combined study groups as follows: 26.2% mean improvement in the Aberrant Behavior Checklist-Community for Fragile X syndrome (ABC-CFXS) total score from baseline to week 12 (p=0.002); and a 21.6% mean improvement in the Anxiety, Depression and Mood Scale (ADAMS) total score from baseline to week 12 (p=0.004). Statistically significant improvements were also observed across various ABC-CFXS and ADAMS subscales. In addition, OV101 demonstrated a statistically significant mean reduction of 0.4 in the Clinical Global Impressions Severity scale (CGI-S) total score (p=0.002) from baseline to week 12.
“The data from the ROCKET trial demonstrate that OV101 may have a meaningful effect on improving the lives of some individuals living with Fragile X syndrome,” said Dr. Elizabeth Berry-Kravis, Professor of Pediatrics and Neurological Sciences and Co-Director of the Molecular Diagnostics Section of the Genetic Laboratory at Rush University Medical Center. “Fragile X is a challenging neurodevelopmental genetic condition, with complex symptomatology that significantly impacts patients and their families. I am encouraged by the safety results of this Phase 2 study of OV101 and am excited to further investigate its therapeutic potential in patients living with Fragile X syndrome.”
“Developing treatment options in Fragile X syndrome has historically been challenging, and there continues to be a high unmet medical need,” said Amit Rakhit, M.D., MBA, President and Chief Medical Officer of Ovid Therapeutics. “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. 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.”