Biscayne Neurotherapeutics, a clinical-stage company developing novel treatments for neurological disorders, announced that lead compound BIS-001 has received an Orphan Drug designation from the US Food and Drug Administration (FDA) for the treatment of Dravet syndrome, a severe form of childhood epilepsy that has no approved therapies.
Separately, the company reported that the Australian regulatory authorities have cleared Biscayne to begin a phase 1b trial of BIS-001 at the Royal Melbourne Hospital. Enrollment is expected to commence in the coming weeks. BIS-001 is in clinical development as a novel treatment for adults with refractory complex partial seizures.
"Dravet syndrome is a devastating, poorly-treated condition that afflicts young children with high rates of disability and mortality, so we are especially pleased that BIS-001 has been designated to receive R&D and commercialization incentives under the Orphan Drug programme," noted Stephen Collins, MD, PhD, president and chief executive officer of Biscayne Neurotherapeutics.
Dr. Collins continued, "We also were notified this week that our Phase 1b clinical trial of BIS-001 as a treatment for adults with refractory complex partial seizures has been approved and enrollment of our first subjects is imminent. BIS-001 demonstrated an encouraging safety profile in a phase 1a trial. This new study is primarily intended to ensure that our new extended release formulation has an acceptable safety profile and good pharmacokinetics. We expect to report data later this year and anticipate starting a phase 2a trial in 2018. Our goal is to pursue an accelerated clinical programme in a number of hard-to-treat epilepsies in the next few years."
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 animal subjects. Data from a Phase 1a trial provided valuable pharmacokinetic information and showed that BIS-001appeared safe. Biscayne has developed a new extended release formulation of BIS-001 that is designed to provide good tolerability across a range of doses and ensure patient convenience and medication adherence. The company is initially developing BIS-001 to treat refractory forms of epilepsy, including refractory partial complex seizures and Dravet syndrome.