Ovid’s Angelman syndrome therapy falls short at Phase 3

pharmafile | December 2, 2020 | News story | Research and Development Angelman syndrome, Ovid 

Ovid Therapeutics has revealed disappointing results for its therapy OV101 (gaboxadol) in the treatment of Angelman syndrome, admitting that it failed its main goal.

In a Phase 3 study of 97 participants between the age of four and 12 with Angelman syndrome, the therapy failed to fulfil its primary endpoint of change in overall score on the Clinical Global Impression-Improvement-Angelman syndrome (CGI-I-AS) scale. Those receiving Ovid’s therapy showed improvement of 0.7 points from baseline, compared to a change of 0.8 in those receiving placebo.

However, the drugmaker did note that the drug was found to be well-tolerated.

“We are deeply disappointed with the outcome of the NEPTUNE trial which did not achieve its primary endpoint,” remarked Jeremy Levin, Chairman and Chief Executive Officer of Ovid Therapeutics. “Other than the ongoing ELARA study, we plan to pause our OV101 programme in Angelman syndrome pending a full understanding of this outcome and discussions with regulators and investigators.”

Ovid said it is continuing to monitor the trial for efficacy in its secondary endpoints, which include measures of sleep, communication, motor function, socialisation, and daily living and behaviour.

Matt Fellows

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