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.

Go deeper with GlobalData

Premium Insights

The gold standard of business intelligence.

Find out more

Related Company Profiles

View all

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.

See Also:

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

How well do you really know your competitors?

Access the most comprehensive Company Profiles on the market, powered by GlobalData. Save hours of research. Gain competitive edge.

Company Profile – free sample

Thank you!

Your download email will arrive shortly

Not ready to buy yet? Download a free sample

We are confident about the unique quality of our Company Profiles. However, we want you to make the most beneficial decision for your business, so we offer a free sample that you can download by submitting the below form

By GlobalData
Visit our Privacy Policy for more information about our services, how we may use, process and share your personal data, including information of your rights in respect of your personal data and how you can unsubscribe from future marketing communications. Our services are intended for corporate subscribers and you warrant that the email address submitted is your corporate email address.

"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.