STAT+: Ovid’s treatment for a rare, untreatable disease fails in a key trial
Ovid Therapeutics’ experimental drug for a devastating rare disease proved no better than placebo in a pivotal clinical trial, the company said Tuesday, a bitter disappointment for families dealing with the untreatable condition.
The study enrolled 97 children with Angelman syndrome, a rare genetic disorder that causes seizures and severe cognitive impairment, and randomized them to receive either Ovid’s drug, OV101, or placebo. After 12 weeks, a daily oral dose of OV101 was no better than placebo at improving the symptoms of Angelman, which include impaired sleep, inability to speak, delayed motor development, and intellectual disability.
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