Angelman syndrome impedes the brain’s ability to distinguish background noise from whats important. This is a result of a reduced tonic inhibition, caused by a loss of function of the delta (δ)-selective GABAA receptor. While patients with Angelman syndrome have a normal life expectancy, it can cause intellectual disability, delayed development, speech impairment, seizures, anxiety, and issues with balance among other symptoms. While these individuals are highly social, they are not able to live independently.
There is currently no approved therapy for Angelman syndrome. However, Ovid Therapeutics has just announced that they are moving into a Phase 3 trial for a potential new treatment.
The drug is called OV101. It works by activating the delta (δ)-selective GABAA receptors. In preclinical studies, the drug improved symptoms in both Angelman syndrome and Fragile X syndrome. The drug has Orphan Drug Designation and Fast Track designation by the FDA for both of these conditions.
The new Phase 3 Trial for OV101 will last for 12 weeks and will include 50-60 pediatric patients with Angelman syndrome (4-12 years of age). Each patient will be given a dose of OV101 or placebo once daily. The drugs effect will be examined by a score of Clinical Global Impressions of Improvement (CGI-I) as a primary endpoint. These scores will then be examined with a specific focus on the symptoms which are deemed particularly important by patients and their caregivers.
Enrollment for the trial will begin in the second half of 2019. It is only pending FDA concurrence on its protocol.
Researchers are hopeful about the success of this drug based on the clinical progress that they’ve already made. They believe this could be the first approved treatment for the disorder. Ultimately, the goal is to not just improve the lives of those living with the condition, but the families and caregivers who are affected by the condition.
Ovid is also beginning a trial involving patients who have already completed a OV101 study. It will examine the safety and tolerability of OV101 long-term. It will also monitor its efficacy as a long-term treatment.
Additionally, Ovid is looking into potential options for Angelman syndrome patients who are under the age of four.
Stay tuned to hear updates on their Phase 3 clinical trial and these two additional research endeavors! If the Phase 3 trial is successful, a FDA application for OV101’s approval will hopefully be submitted next year.
You can read more about this drug and Ovid’s trial developments here.