Pharma Focus Asia

NeuCyte Licenses Promising Novel Compound for Treatment of Refractory Epilepsy and Related Neurological Disorders

Thursday, September 26, 2019

NeuCyte, Inc., an innovative biotech company focused on developing novel treatments for neurological disorders, today announced that it has entered into an exclusive license agreement with Trillium Therapeutics to advance an undisclosed preclinical compound with potential utility in treating refractory epilepsy in the form of Dravet syndrome and related disorders.

The compound covered by this agreement has demonstrated highly encouraging activity during studies conducted by the National Institute of Neurological Disorders and Stroke (NINDS) Epilepsy Therapy Screening Program (ETSP). It demonstrates a superior safety and anti-seizure efficacy profile over NINDS benchmark anti-epileptic drugs (AEDs) in eleven animal models. It has also demonstrated broad efficacy in NIH animal studies for pharmaco-resistant epilepsy.  As a result, this novel agent has gained the prestigious NINDS Red Book status and been selected as a promising lead drug candidate.

"This compound is an exceedingly promising antiseizure drug which, although in preclinical development, is quite efficacious in a wide variety of highly predictive seizure models," said Roger J. Porter, MD, Adjunct Professor of Neurology at the University of Pennsylvania, former Deputy Director of NINDS, former Chairman of the White House Committee on Brain and Behavioral Sciences, and former President of American Epilepsy Society. "It may prove very effective for patients with epilepsy." As a first-in-class compound with unique mechanism of action profiles, this drug candidate is likely to have activity across a variety of indications. The favorable efficacy and safety profile has also been validated by NeuCyte's proprietary in vitro human iPSC-derived models.

"Epilepsy is the fourth most common neurological disease affecting more than 65 million people of all ages worldwide. The complex and multifactorial nature of the disorders makes treatment complicated and challenging. Up to 40% of newly diagnosed epilepsy patients are resistant to current therapies which highlights the urgent need to develop first-in-class treatments," said Jacqueline A. French, MD, Chief Medical and Innovation Officer of the Epilepsy Foundation and epileptologist in New York City. "It is very encouraging to see new developments and therapeutic candidates in this disease area."

Under the terms of the agreement, Trillium has granted NeuCyte exclusive, worldwide rights to develop and commercialize the compound.  "NeuCyte is committed to bringing breakthrough therapies to epilepsy patients and believe that our advantageous translational technology platform and CNS expertise enable us to optimally advance such therapeutic candidates," said Tao Huang, PhD, JD, President and Chief Executive Officer of NeuCyte. "This licensing agreement allows us to expand our pipeline and advance our goal of meeting unmet clinical needs and truly improving patient care and quality of life."

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