Ovid Therapeutics, a privately held biopharmaceutical company committed to developing medicines that transform the lives of people with rare neurological diseases, has initiated a phase I clinical trial to evaluate the pharmacokinetics (PK), safety and tolerability of OV101 in adolescents diagnosed with Angelman syndrome or Fragile X syndrome. OV101 (gaboxadol), a delta (δ)-selective GABAA receptor agonist, is believed to be the first investigational drug to target the disruption of tonic inhibition, a key mechanism that allows a healthy human brain to decipher excitatory and inhibitory neurological signals correctly without being overloaded. Tonic inhibition is believed to play a significant role in the neurodevelopmental symptoms characteristic of disorders such as Angelman syndrome and Fragile X syndrome.

“Those with neurodevelopmental disorders such as Angelman syndrome and Fragile X syndrome are affected from birth. Our goal is to provide medicines to these individuals at all ages. The initiation of this trial represents the first step in our strategy to develop OV101 for an adolescent population. This is an important step because having access to medicines at a younger age has the potential to have a transformative effect on the lives of these patients and their families,” said Amit Rakhit, M.D., MBA, chief medical and portfolio officer of Ovid Therapeutics. “We look forward to continuing to partner with the Angelman and Fragile X syndrome communities as we work to develop therapies that can have the greatest impact on the challenges they face every day.”

The phase I single dose, single-arm, open-label, clinical trial of OV101 will measure PK parameters. The trial is expected to enroll adolescent patients, ages 13 to 17, who have been diagnosed with either Angelman syndrome or Fragile X syndrome.

In addition to this clinical trial in adolescents, Ovid is planning to initiate clinical development in a pediatric population upon completing the required preclinical testing.