Initial Trial Data Looks Promising for a Potential Treatment for Rare Epilepsies

According to a story from, the drug company Ovid Therapeutics Inc. has recently announced that the early data from its phase 2 clinical trial is mostly encouraging. This clinical trial is testing the company’s investigational drug candidate soticlestat as a treatment for rare developmental epileptic encephalopathies (DEE). This group of diseases comprises several different rare epilepsies that are historically difficult to treat. This group of diseases includes Lennox-Gastaut syndrome, Dravet syndrome, and CDLK5 deficiency disorder. 

While there are medications available for epilepsy in general, many of them are of limited effectiveness in this group of diseases. Some forms of DEE do not have any forms of treatment available that have been approved by the US Food and Drug Administration (FDA).

About The Trial

The findings from this clinical trial consists primarily of patients that participated in the phase 1b/2a clinical trial, which tested the drug in adults with various forms of DEE. The phase 2 trial was an extension study of this earlier trial. The safety and tolerability characteristics documented in the extension trial were consistent with those in the earlier study. The results suggest that soticlestat has the potential to provide long term benefits for these patients and includes data out to 48 weeks following the start of treatment.

In the period from 25-36 weeks, the therapy was able to reduce the frequency of seizures by a median of 84 percent. In the 37-58 week range, this reduction increased to a median of 90 percent. Patients with the highest frequency of seizure events at baseline appeared to experience the greatest benefit from the drug. These results suggest that soticlestat has the potential to be a very useful treatment for DEE patients. 

Patients from two other phase 2 clinical trials that have completed their treatment are also being rolled over into the extension study; however, their data has not been included in the announcement because they have not been in the extension study long enough.

One of these studies was testing soticlestat in patients with CDKL5 deficiency disorder and Dup15q syndrome; the other was testing the therapy in patients with Lennox-Gastaut syndrome and Dravet syndrome.

About Soticlestat

Soticlestat is classified as an inhibitor of the cholesterol 24-hydroxylase enzyme. This drug is the first in its class and is the only therapy with this mechanism being tested as a treatment for epileptic conditions. Soticlestat has earned Orphan Drug designation from the FDA as a treatment for Dravet syndrome and Lennox-Gastaut syndrome. 

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