Ovid: Towards Improved Outcomes in Rare Neurodevelopmental Disorders Via Targeted Treatments

Quick Overview

Ovid Therapeutics is developing targeted treatments for rare neurodevelopmental disorders, such as Angelman Syndrome and Dup15q. The company is focusing on the shared deficits across these disorders, aiming to create interventions that could be useful for multiple conditions. Ovid’s approach is based on the concept of tonic inhibition, which is the ability to identify signal in the background of noise. The company’s compound, OV101, binds to extrasynaptic receptors that mediate this tonic inhibition. In animal studies, OV101 has been shown to increase tonic inhibition and normalise behavioural features. The compound has already been evaluated in more than 3,000 individuals and is known to be safe and well-tolerated. Ovid is planning to launch a trial in adults in the near future, with the primary goal of assessing safety and identifying the most informative measures for a Phase 3 trial.


I’m thrilled to be here, and thanks to Guy and the organisers for giving me the opportunity. I’m going to keep things pretty high level, but what I’d like to give people a sense of is what OVID is, some of the conceptual things that led to its founding, and then a little taste of the biology underlying this upcoming trial in Angelman Syndrome.

The Need for OVID

Neurodevelopmental disorders are debilitating. We see them all around us, and whether the rare disorders we’re working on are AS or Dup15q, they’re numerous and come at an enormous cost both to families and to society in terms of financial burden. OVID was really founded to address this. There’s surprisingly little in terms of commercial work in this space, and so we see that as a real opportunity and a way that we can impact and hopefully make a change for the better.

Shared Deficits and Overlaps

Regardless of the genetic etiology of any given disorder that one may be studying, there are a lot of shared deficits. When we dig a little deeper, either in terms of clinical presentation or mechanisms, they diverge, certainly. But the hope is at OVID that we can capture some of these overlaps and use that as entry points for different disorders.

The Role of GABA Receptors

Central to the work that we’re pursuing is that there’s actually two functionally distinct classes of GABA receptors. You have these synaptic GABA receptors, and they mediate phasic excitation. Our interest are these extra-synaptic GABA receptors that mediate a very different kind of neural activity, this tonic inhibition.

OV101: A Potential Solution

OV101, also known as THIP, also known as Gaboxadol, very specifically binds these extrasynaptic receptors, these delta subunit containing receptors that mediate this tonic inhibition. It’s the only known agonist specific to this receptor population, and so data from animal studies shows that it can actually increase this tonic inhibition, and in the Angelman mouse, normalize behavioural features that are observed.

The Upcoming Trial

The trial that we’ll launch in the fall will be in adults, and the primary goal here will be to assess safety and carry out some exploratory work to evaluate those measures that might be most informative in a Phase 3 trial. Sites are just getting underway, but we’re incredibly fortunate to have folks in this room with so much expertise guiding us on the design of the trial, and enrolling patients hopefully in the near future.


It is a pretty exciting time for us. We’re looking at a variety of other indications and opportunities there, and additional compounds that we think may be useful. Hopefully at the next meeting I’ll be able to talk about some of that work that we’ll be able to move forward.

Talk details

  • Title: Ovid: Towards Improved Outcomes in Rare Neurodevelopmental Disorders Via Targeted Treatments
  • Author(s): Brett Abrahams
  • Author(s)’ affiliation: Ovid Therapeutics
  • Publication date: 2016-09-06
  • Collection: 2016 ASF-Dup15q Scientific Symposium