The designation allows for expedited development and review of therapeutics for serious diseases with high unmet medical need.
“This designation is an important milestone for both Ovid and the Angelman community as it enables increased dialog with the FDA, speeding our ability to bring this potential therapeutic option to people living with Angelman syndrome. We believe that OV101, with its novel mechanism of action, has the potential to be an innovative and impactful therapy,” Amit Rakhit, MD, chief medical and portfolio management officer of Ovid, said in a press release.
OV101 binds selectively to the delta-selective GABAA receptor agonist to restore what is known as tonic inhibition – the brain’s ability to filter real neural signals. Disruption of this process is thought to underlie Angelman syndrome.
In preclinical studies, OV101 was shown to improve several Angelman syndrome symptoms, including motor function, sleep, behavior, and cognition parameters. Following a Phase 1 clinical trial (NCT03109756) where OV101 showed a similar and safe pharmacodynamics profile in adults and adolescents, the therapy is now being evaluated in a Phase 2 study.
Patients will be assigned randomly to either oral OV101 once daily or twice daily, and the results will be compared to those receiving a placebo twice daily.
The trial outcomes are measured after 12 weeks of treatment and include the incidence of adverse events in OV101-treated groups versus placebo (primary goal) and additional (secondary goal) functional parameters, including motor function, sleep, and cognition.
“In addition to the regulatory milestones of orphan drug and fast track designations for Angelman syndrome, we achieved significant clinical progress with our OV101 program. As a result of positive Phase 1 data, we were recently able to expand our ongoing Phase 2 STARS clinical trial to include both adults and adolescents with Angelman syndrome. We look forward to data from the STARS trial in the second half of 2018,” Rakhit said.