Cerebral Therapeutics Announces Enrollment of First Patient in a Multicenter Randomized Controlled Phase 2b Trial in Epilepsy

Study builds on published results of initial trial in refractory epilepsy patients

AURORA, Colo.--(BUSINESS WIRE)--Cerebral Therapeutics, Inc., a clinical-stage pharmaceutical company developing innovative treatments for neurological diseases, today announced that the first patient has been enrolled in a Phase 2b study of intracerebroventricular drug delivery of the anti-seizure medication CT-010 in patients with medically refractory epilepsy. The trial is enrolling adult epilepsy patients with temporal lobe onset, with or without secondary generalized seizures.

A clinical feasibility study at St. Vincent’s Hospital Melbourne (published in The Lancet - EClinicalMedicine) established for the first time that intracerebroventricular administration of sodium valproate is a potentially effective strategy in the treatment of refractory epilepsy. The study found that subjects experienced a mean 77 percent seizure reduction and extended periods of seizure freedom. All subjects reported significant quality of life improvement with minimal drug side effects. Based on these promising initial results, Cerebral Therapeutics has developed a propriety formulation, CT-010, and enhanced the infusion system for continued investigation of the safety and effectiveness of the therapy.

“The launch of this important Phase 2b study allows us to further pursue direct brain administration of an anti-epileptic drug that has the potential to dramatically reduce seizures and positively impact the lives of patients with refractory epilepsy,” said Dr. Mark Cook, Professor of Medicine at the University of Melbourne.

“Two out of three people diagnosed with epilepsy still cannot obtain control of seizures with currently available treatments,” said Dr. Jacqueline French, Epilepsy Foundation Chief Medical & Innovation Officer and Professor of Neurology at NYU Langone Health. “We still have a critical need for new and novel therapies to be explored for treatment resistant epilepsy. Results of the 2a are promising, and we look forward to the results of this next trial.”

Dan Abrams, MD, CEO of Cerebral Therapeutics noted that “The company’s brain targeted drug delivery approach combines the best of pharmaceutical and medical device technology to enable CT-010 to reach the site of action in the brain where it can help address refractory epilepsy while minimizing systemic drug exposure and side effects. We are pleased to advance this important new therapy with the initiation of this randomized clinical trial at leading epilepsy centers in Australia. The study will be expanded in 2021 at additional sites in Israel and the United States.”

For more information, visit www.cerebraltherapeutics.com.

About The Study

The Phase 2b, randomized, placebo-controlled, double-blind study is designed to expand on positive results of the ongoing Phase 1b/2a study assessing the safety and efficacy of intracerebroventricular administration of sodium valproate. Key objectives in the study include evaluating the safety and tolerability of intracerebroventricular delivery of proprietary sodium valproate formulation, CT-010, via an implantable pump and cranial port with a catheter and assessing the reduction in the number of seizures in subjects with focal seizures with temporal lobe onset compared to placebo. The study will enroll up to 70 subjects and is beginning in Australia with expansion plans for the United States and Israel. The current sites are St. Vincent’s Hospital Melbourne, The Alfred Hospital, and The Austin Hospital.

About Epilepsy

Epilepsy is the fourth most common neurological condition globally. Approximately 3.4 million people in the United States are living with epilepsy today, and up to one-third continue to experience seizures despite drug treatment. The annual cost for patients with epilepsy in the United States is estimated to be approximately $12.5 billion.

About Cerebral Therapeutics, Inc.

Cerebral Therapeutics, Inc. is a clinical-stage pharmaceutical company dedicated to improving the lives of patients living with severe refractory epilepsy and other neurological diseases. Founded by experts in the fields of neurology, neurosurgery, and drug delivery, Cerebral Therapeutics is pioneering the development of drug-device combination therapies that enable precise targeted delivery to the central nervous system. The goal of this novel approach is to maximize efficacy while greatly reducing systemic drug exposure and enhancing drug regimen compliance. Cerebral Therapeutics’ intracerebroventricular infusion therapy may offer a more efficacious and reliable approach to treating neurological diseases for which oral medications are not able to achieve adequate control and/or are limited by systemic toxicities.

The Lancet - EClinicalMedicine journal can be found here: https://www.thelancet.com/pdfs/journals/eclinm/PIIS2589-5370(20)30070-5.pdf