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Ovid Therapeutics Announces Positive Topline Results from the Phase 2 ROCKET Trial of OV101 for the Treatment of Fragile X Syndrome
OV101 met the study’s primary objective of safety and tolerabilityOV101 produced statistically significant reductions in behavioral and functional symptoms in
About this update from Ovid Therapeutics Inc.
[{"type":"text","content":"OV101 met the study’s primary objective of safety and tolerabilityOV101 produced statistically significant reductions in behavioral and functional symptoms in individuals with Fragile X syndromeResults support the continued development of OV101 in Fragile X syndrome NEW YORK, May 07, 2020 (GLOBE NEWSWIRE) -- 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.\n 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 Sev...