dm+d

Unassigned

New Medicines

Angelman syndrome

Information

New molecular entity
Ovid Therapeutics
Ovid Therapeutics

Development and Regulatory status

Discontinued
Discontinued
Discontinued
Yes
Yes
Apr 21Development of OV101 discontinued. Ovid plan to pursue OV882 for Angelman syndrome[10].
Dec 20PIII NEPTUNE trial of OV101 has failed to meet its primary endpoint. Ovid is currently revieweing secondary endpoints of sleep, communication, motor function and socialisation. All programmes have been paused other than the open-label expansion for which results are expected Q1 2021 [9].

Category

Delta-selective GABAA receptor agonist - thought to impact neuronal activity by restoring levels of tonic inhibition that exist when intrinsic GABAA levels are low. [1,2]
Prevalence is estimated to be ~1 in 12,000-20,000 people in the general population, affecting males and females equally, but many go undiagnosed. Angelman Syndrome is a genetic disorder mostly affecting the nervous system. It is marked by developmental delay, intellectual disability, severe speech impairment and movement and balance problems. [3,6]
Angelman syndrome
Oral