The company is currently evaluating OV101 in the STARS study, a randomized, double-blind, placebo-controlled Phase 2 trial designed to explore the efficacy endpoints and assess the safety parameters of OV101 in adult patients with Angelman syndrome.
OV101 is believed to be the only clinically tested delta (δ)-selective GABA-A receptor agonist under development to normalize tonic inhibition in patients with Angelman syndrome.
It also has the potential to improve neuronal function, influence sleep, motor control and cognition, and enhance outcomes in patients living with the disease.
Ovid Therapeutics chairman and CEO Jeremy Levin said: “Angelman syndrome is a rare, genetic neurological disorder that causes profound developmental and neurologic disabilities.
“The granting of orphan drug designation is a significant milestone for the OV101 clinical development program and highlights the high unmet medical need for new therapies that may transform the lives of patients with Angelman syndrome and their families.”
Danish chemist Povl Krogsaard-Larsen originally synthesized OV101 in 1977. It has a unique mechanism of action in the brain.
OV101 is a small molecule derived from the mushroom, Amanita muscaria, also known as fly agaric or fly amanita.
It is the first and only clinically tested selective extrasynaptic GABAA receptor agonist (SEGA). Through its action as a SEGA, OV101 selectively regulates tonic inhibition.