The Use of Gaboxadol (OV101) in the Treatment of Angelman Syndrome: Downstream Therapeutics Working at the Neuronal Synapse

Quick Overview

Gaboxadol (OV101) is being investigated as a potential treatment for Angelman Syndrome. The drug acts at the neuronal synapse and has shown minimal side effects in previous clinical trials. Future clinical trials are being planned, with the aim of starting in the second half of 2016. The drug will need to be taken continuously to maintain its effects. The company, Ovid Therapeutics, is focused on the well-being of patients and aims to bring an important medicine to the Angelman community. The drug will need to be approved by regulatory agencies such as the FDA and EMA before it can be made available to patients. The company is also considering obtaining orphan drug status to help accelerate the approval process. The trial will initially focus on young adults, but there are plans to include younger children in future trials. The company is also planning to expand the trials globally, including in Australia and the UK. The drug has the potential to improve various symptoms of Angelman Syndrome, but the specific outcome measures for the trials have not yet been determined. The drug is expected to have a good safety profile, with minimal side effects. The company is committed to working closely with patients and their families throughout the drug development process.


In this talk, we will discuss the use of Gaboxadol (OV101) in the treatment of Angelman Syndrome. We will also explore the downstream therapeutics working at the neuronal synapse and provide an overview of future clinical trials.


Dr. Jeremy Levin and Dr. Matthew During from Ovid Therapeutics are leading the discussion on Angelman Syndrome at the 2015 FAST Science Summit. Dr. Levin is the chairman and CEO of Ovid Therapeutics and has extensive experience in the pharmaceutical industry. Dr. During is a Harvard and Yale trained neuroscientist and the founder of Ovid Therapeutics.

Importance of Angelman Syndrome Research

Angelman Syndrome is a rare genetic disorder that affects individuals from infancy. The symptoms include developmental delays, intellectual disability, speech impairments, and movement and balance issues. Currently, there are no approved therapies that target the underlying causes of Angelman Syndrome.

The Potential of Gaboxadol (OV101)

Gaboxadol (OV101) is a drug that has shown promise in the treatment of Angelman Syndrome. It acts on the extrasynaptic GABA receptor and impacts cellular function in a way that can potentially improve the symptoms of the syndrome. The drug has been tested in over 3,000 adults in clinical trials for sleep disorders and has shown minimal side effects.

Future Clinical Trials

Ovid Therapeutics is planning to conduct clinical trials for Gaboxadol (OV101) in the second half of 2016. The trials will focus on young adults initially, with the aim of expanding to younger children in the future. The exact duration of the trials is yet to be determined, but it is expected to be approximately three months. The trials will be double-blind, meaning that some participants will receive a placebo to ensure accurate results.

Considerations for the Trials

The trials will require a genetic diagnosis of Angelman Syndrome for inclusion. Ovid Therapeutics is also considering the functional aspects of the syndrome when designing the trials, aiming to capture a broad range of individuals and symptoms. The company is committed to ensuring the safety and efficacy of the drug and will work closely with the FDA and other regulatory agencies to meet all requirements.

Global Reach

Ovid Therapeutics is dedicated to making the drug available globally. While the initial trials will take place in the United States, the company plans to extend the trials to other countries, including the UK and Europe. The process of obtaining approval in different countries may vary, but Ovid Therapeutics is actively working on establishing partnerships and collaborations to expedite the process.


The use of Gaboxadol (OV101) in the treatment of Angelman Syndrome shows great promise. Ovid Therapeutics is committed to conducting rigorous clinical trials to ensure the safety and efficacy of the drug. The company is working closely with regulatory agencies and the Angelman Syndrome community to design trials that will have the greatest impact on patients’ lives.

Talk details

  • Title: The use of Gaboxadol (OV101) in the treatment of Angelman Syndrome. Down stream therapeutics working at the neuronal synapse. Discussion of future clinical trials
  • Author(s): Jeremy Levin, Matthew During
  • Author(s)’ affiliation: Ovid Therapeutics
  • Publication date: 2015-12-04
  • Collection: 2015 FAST Science Summit