Despite a Rocky Start, Possible Angelman Syndrome Drug Enters Late Stage Trial

According to a story from The Boston Globe, the drug developer Ovid Therapeutics plans to move forward with a late stage clinical trial that will test the company’s investigational drug called OV101 that is in development as a treatment for the rare disease Angelman syndrome. In an earlier trial, the therapy offering improvement in one endpoint but failed to impact a number of others.

About Angelman Syndrome

Angelman syndrome is a type of genetic disorder that impacts the nervous system. This disorder is not inherited from parents, but instead occurs as a spontaneous genetic alteration, typically the deletion of a segment from chromosome 15 called UBE3A. A mutation of this gene can also caused Angelman syndrome. Symptoms of this disorder can include seizures, developmental delays, a small head, an excited and happy demeanor, trouble with balance and movement, intellectual disability, and speech issues. Patients may share certain facial characteristics. Symptoms become noticeable at about one year old. Treatment is supportive and symptomatic, and there is currently no cure. Patients have a strong desire for social interaction, and despite limited language are able to understand much of what is happening around them. Life expectancy is generally close to that of an unaffected person. To learn more about Angelman syndrome, click here.

A Rough Start

While Ovid was quick to hail the results from the earlier trial as a positive sign, investors and the US Food and Drug Administration (FDA) were not so sure. However, the company has since met with FDA representatives and convinced them to allow a late stage trial with an alternative endpoint called the clinical global impressions of improvement. The endpoint is a seven point scale that a doctor uses in order to determine if a patient is seeing improvement.

This may seem too vague to be scientifically useful, but it does make some sense to use it for a disease like Angelman syndrome in which patients often present very differently. It was a line of argument that was successfully able to convince FDA officials at least. The new trial will test OV101 in 60 patients between ages 4 and 12 with initial data expected by the middle of 2020.


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