Biscayne Neurotherapeutics announced results from a Phase 1b trial of its novel antiepileptic compound, BIS-001ER. In the trial, the new extended release version of BIS-001 met or surpassed all pre-defined endpoints, including dosing frequency, serum drug exposure and safety. Biscayne intends to initiate a BIS-001ER proof of concept efficacy study in adults with refractory focal seizures in early 2018.
"These results exceeded our expectations and are consistent with the encouraging safety profile seen in numerous studies of BIS-001 in animals, as well as its record of safe use for hundreds of years as a traditional Chinese medicine,” Stephen Collins, MD, PhD, President and Chief Executive Officer of Biscayne Neurotherapeutics, said. “This successful Phase 1b study sets the stage for the proof of concept efficacy trial in adults with refractory focal epilepsy we will initiate early next year. Biscayne will also soon be launching a Series C financing that will enable us to pursue an accelerated clinical program for BIS-001ER in Dravet syndrome and other hard-to-treat epilepsies."
In the Phase 1b study in healthy volunteers, BIS-001ER achieved its goal of twice-daily dosing, a regimen preferred by many epilepsy physicians and patients. On average, serum drug exposure levels were almost twice as high as the levels researchers think are needed to achieve strong efficacy results (based on dosages in animal models that suppressed 100% of seizures in at least half the subjects.) Adverse events were as expected and were generally mild to moderate, transient and non-dose limiting.
BIS-001 is a highly potent form of huperzine A, a synthetic extract of a traditional Chinese medicine with a long history of safe use. Huperzine A is an acetylcholinesterase (AChE) inhibitor with high brain penetration that offers a unique mechanism of action for the treatment of epilepsy. It has shown promising efficacy in highly predictive preclinical models of refractory epilepsy, providing complete elimination of seizures in the majority of animals. Biscayne's extended release formulation of BIS-001 is designed to enhance tolerability across a range of doses and ensure patient convenience and medication adherence. Biscayne is developing BIS-001ER to treat refractory forms of focal epilepsy, including Focal Impaired Awareness Seizures (previously known as Complex Partial Seizures.) It has been awarded a U.S. FDA Orphan Drug designation for BIS-001 for the treatment of Dravet syndrome, a devastating seizure condition affecting children.
Epilepsy is a chronic disorder characterized by recurrent, unprovoked seizures. Epilepsy affects about three million people in the US and over 50 million people worldwide. In more than half of patients, the cause is unknown. Many patients with epilepsy have more than one type of seizure and may also have other symptoms of neurological problems. Up to one in 3 patients with epilepsy is unable to control their seizures with current therapies whether taken alone or in combination.