Biopharmaceutical firm Ovid Therapeutics has commenced the Phase I clinical trial of OV101 for the treatment of adolescents with Angelman syndrome or Fragile X syndrome.

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OV101 (gaboxadol) is a delta (δ)-selective GABAA receptor agonist being developed to target the disruption of tonic inhibition, which is necessary for the human brain to interpret correct excitatory and inhibitory neurological signals.

The single-dose, single-arm, open-label Phase I trial is designed to assess the pharmacokinetics (PK), safety and tolerability of OV101 in patients aged 13-17.

Ovid Therapeutics chief medical and portfolio officer Amit Rakhit said: “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.

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"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."

"The initiation of this trial represents the first step in our strategy to develop OV101 for an adolescent population."

Upon completion of the required preclinical testing, the firm intends to further investigate OV101 in a pediatric population.

OV101 was found to selectively stimulate the δ-subunit of GABAA receptors in the extrasynaptic space, as well as regulated neuronal activity by tonic inhibition in preclinical models.

The results from preclinical studies found improved symptoms in both Angelman and Fragile X syndromes.

It is expected that the product will restore tonic inhibition and alleviate multiple symptoms of the disorders.

Image: A Fragile X syndrome patient. Photo: courtesy of Peter Saxon/Wikipedia.

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