OV101 for the Treatment of AS

Quick Overview

Dr. Amit Rakhit, President of Ava Therapeutics, spoke at the 2019 FAST Science Summit about the progress of OV101 (Gaboxadol) in the treatment of Angelman Syndrome (AS). He discussed the Phase 2 STARS program and the ongoing Phase 3 NEPTUNE trial, which is a pivotal registrational study for AS. The NEPTUNE trial is a randomized, double-blind, placebo-controlled study in pediatric populations aged 4 to 12 years. The primary endpoint is the Clinical Global Impressions of Improvement (CGI-I), and secondary endpoints include measurements of sleep using actigraphy and the Vineland scale. Dr. Rakhit also mentioned the open-label extension study called ELARA, which allows participants from both STARS and NEPTUNE to continue receiving OV101. He emphasized the importance of caregiver input in assessing the efficacy of the treatment. The most common side effects of OV101 are gastrointestinal issues.


In this talk, Dr. Amit Rakhit discusses the progress of OV101, a potential treatment for Angelman Syndrome (AS). He highlights the importance of therapeutic options for rare neurology conditions and the potential impact of OV101 in improving the lives of individuals with AS.

The Journey of OV101

Dr. Rakhit begins by acknowledging the growth and development of the AS community over the years. He emphasizes the increasing number of therapies in development and the potential for significant advancements in the treatment of AS. Drawing parallels with other neurological conditions like multiple sclerosis and spinal muscular atrophy, he highlights the progress made in these areas and the potential for similar advancements in AS.

Understanding OV101

OV101, also known as Gaboxadol, is a compound that aims to address the deficiency of a compound responsible for communication between neurons in the brain. Dr. Rakhit explains that OV101 acts as a replacement for this compound, restoring balance and improving symptoms associated with AS. He mentions the positive results observed in mouse models of AS, which led to the initiation of clinical trials.

The STARS Program

Dr. Rakhit discusses the STARS program, a Phase 2 study of OV101 in individuals with AS. He explains that the study focused on safety, tolerability, and exploratory measures of efficacy. The primary endpoint of the study was the Clinical Global Impressions of Improvement (CGI-I), which assesses overall improvements in sleep, behavior, motor function, and communication. The study showed statistically significant improvements in the once-a-day arm of OV101, leading to further discussions with regulatory authorities and the design of the Phase 3 program.


The NEPTUNE trial is a Phase 3 program of OV101 in pediatric populations aged 4 to 12 years old. It is a randomized, double-blind, placebo-controlled study with a primary endpoint of CGI-I. Dr. Rakhit explains that the trial aims to refine the assessment of CGI-I for AS and provides specific guidance on how to assess behavior, motor function, sleep, and communication. The trial started in September and is expected to complete enrollment in early 2020, with data expected in mid-2020.

The ELARA Program

Dr. Rakhit mentions the ELARA program, an open-label extension study for individuals who complete the NEPTUNE trial. This three-year-long study allows participants to continue receiving OV101 and assesses long-term effects. He explains that all siblings with AS can join the ELARA program upon completion of the NEPTUNE trial.


Dr. Rakhit expresses gratitude to the AS community, clinicians, scientists, and companies working in this space. He emphasizes the importance of collaboration and the potential for novel treatments to make a difference in the lives of individuals with rare conditions like AS. He concludes by expressing excitement for the future and the progress that can be made in the treatment of AS.

Talk details

  • Title: OV101 for the Treatment of AS
  • Author(s): Amit Rakhit
  • Author(s)’ affiliation: Ovid Therapeutics
  • Publication date: 2019-12-27
  • Collection: 2019 FAST Science Summit