The financing was led by the Global Health Sciences Fund of Quark Venture and GF Securities, along with Mesa Verde Venture Partners. Existing Biscayne Pharmaceuticals investors and new private investors also participated.
Biscayne Neurotherapeutics will primarily use the funds to conduct a Phase 1b trial of a new extended release form of its novel antiepileptic compound, BIS-001, in development for adults with refractory complex partial seizures.
Stephen Collins, MD, PhD, President and Chief Executive Officer of Biscayne Neurotherapeutics, said: "Patients with severe epilepsies urgently need more effective therapies with fewer disabling side effects. BIS-001 has demonstrated exceptional anti-seizure activity preclinically and an encouraging safety profile in a Phase 1a trial.
"We are delighted that our new and existing investors have provided the financing to advance our new extended release formulation of BIS-001 into a Phase 1b clinical trial that is scheduled to begin by mid-year. If all goes well, we expect to initiate a Phase 2a trial in 2018 and to pursue an accelerated clinical program in a number of hard-to-treat epilepsies."
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 the animals. In a Phase 1a trial in patients with drug-resistant epilepsy, BIS-001 appeared safe and well tolerated at dosages planned for future studies.
Biscayne has now developed an extended release formulation of BIS-001, which is designed to enhance tolerability across a range of doses and ensure patient convenience and medication adherence.
Biscayne is initially developing BIS-001 to treat refractory forms of focal epilepsy, including refractory partial complex seizures and Dravet syndrome, a devastating refractory condition afflicting children.
Karimah Es Sabar, Chief Executive Officer, Quark Venture, said: "The goal of the Global Health Sciences Fund is to invest in the best science the world has to offer, so we are pleased to partner with Biscayne Neurotherapeutics, whose innovative science has emerged from researchers at Harvard and Yale.
"We are impressed with the company's leadership and their unique approach to finding a solution for these devastating diseases, for which there are currently no adequate therapies available."
Carey Ng, PhD, MBA, Managing Director of Mesa Verde Venture Partners, said: "We are committed to building partnerships with promising life sciences companies addressing important unmet needs.
"Biscayne's team has deep epilepsy expertise and a lead compound with a novel mechanism of action, a long history of safe use and impressive preclinical data in a well-validated model of severe epilepsy. We welcome this opportunity to support the company as it advances the clinical program for BIS-001."
Steven Schachter MD, Professor of Neurology at Harvard Medical School and a Scientific Co-Founder of Biscayne, commented, "In addition to its powerful anti-seizure activity in preclinical models of severe epilepsy, BIS-001 has exhibited the cognition-enhancing properties seen with other AChE drugs, but with much better central nervous system and systemic tolerability and safety than currently available agents. Our clinical program will assess both anti-seizure efficacy and whether BIS-001 supports improved cognition in epilepsy patients.
"At a minimum, we are optimistic it will be devoid of the detrimental effects on cognition seen with many existing antiepileptic drugs. We are eager to test BIS-001 in a variety of conditions, given the high unmet therapeutic need that exists across the spectrum of seizure disorders."
As part of the financing agreement, the Global Health Sciences Fund will appoint one director and one observer to the Biscayne Neurotherapeutics Board of Directors, and Mesa Verde Venture Partners will also name a director.
Samuel Reich, a Co-Founder and Executive Chairman of Biscayne Neurotherapeutics, noted, "We welcome the involvement and investment of Quark Venture and GF Securities, as well as Mesa Verde Venture Partners and the company's other new and existing investors.
"Our new venture investors bring us diverse biopharmaceutical industry expertise, along with new funding to progress our highly promising lead therapy for people with severely disabling epilepsy. We also appreciate the fact that they have the depth of resources needed to support our future efforts as BIS-001 advances in clinical trials."
Epilepsy is a chronic disorder, the hallmark of which is 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 1 in 3 patients with epilepsy is unable to control their seizures with current therapies whether taken alone or in combination.