Rare Disease Report

New Hope for Dravet Syndrome, Lennox-Gastaut Syndrome and Tuberous Sclerosis Complex Patients?

AUGUST 21, 2017
James Radke
Seizures are common symptoms in diseases such as Dravet Syndrome, Lennox-Gastaut Syndrome and Tuberous Sclerosis Complex. A new study is underway, and it may provide patients with these and other epilepsy encephalopathies some relief.

The Phase 1b/2a clinical trial, sponsored by Takeda and Ovid Pharmaceuticals, tests TAK-935 (OV935) in adults with developmental and/or epileptic encephalopathies, such as those observed in Dravet Syndrome, Lennox-Gastaut Syndrome and Tuberous Sclerosis Complex. Additional studies are also in the planning stages with the hope that early intervention can greatly improve outcomes in children with these conditions.

TAK-935 (OV935) is a cholesterol 24-hydroxylase (CH24H) inhibitor that has shown to have anti-epileptic activity in preclinical studies.

“We are excited to achieve this important milestone as this is the first clinical trial in our collaboration with Ovid and TAK-935/OV935 is the first CH24H inhibitor in clinical development,” said Dr. Emiliangelo Ratti, head of Takeda’s Central Nervous System Therapeutic Area Unit. “While there are many treatments available for epilepsy, few therapeutic options exist for patients with developmental and/or epileptic encephalopathies, which cause intractable seizures that are often associated with cognitive, neurologic and behavioral symptoms. Our collaboration with Ovid is geared to leverage each company’s strengths to jointly develop this investigational medicine for people living with rare epilepsies.”

“We believe that TAK-935/OV935 has the potential to target the increased pro-epileptic signaling that occurs in developmental and/or epileptic encephalopathies and provide a treatment for people with rare epilepsies who currently do not have sufficient options,” said Amit Rakhit, M.D., MBA, chief medical and portfolio management officer of Ovid Therapeutics. “This trial is the beginning of a planned broader development program for TAK-935/OV935. We intend to investigate TAK-935/OV935 in younger populations because rare epilepsies, such as Dravet syndrome, Lennox-Gastaut syndrome and Tuberous Sclerosis Complex, are diagnosed early in life and this may provide an opportunity to intervene early in the disease course.”

The Phase 1b/2a trial is a randomized, double-blind, placebo-controlled, dose-escalation study that will enroll approximately 20 adult patients with developmental and/or epileptic encephalopathies, including Dravet Syndrome, Lennox-Gastaut Syndrome and Tuberous Sclerosis Complex.

The primary endpoint of the study is to characterize the safety and tolerability of TAK-935 (OV935).

For more information about the trial, click here.

Dravet syndrome is an epilepsy encephalopathy beginning in childhood – generally in the first year of life. The seizures are often resistant to standard anti-epileptic drugs.

Lennox-Gastaut syndrome is a severe form of epilepsy, and seizures commonly begin before the age of 4 years. Seizure types vary among patients but may include tonic, atonic, atypical absence, and myoclonic seizures.

Tuberous Sclerosis Complex involves the uncontrolled growth of benign tumors in multiple organs, including the brain, kidneys, heart, lungs, eyes, and skin. Symptoms vary widely in these patients but seizures are fairly common.

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