Biscayne Pharmaceuticals have reported that their antiepileptic compound, BIS-001, was able to eliminate seizures in most animal models of a form of the disorder known as Dravet syndrome. The drug is a synthetic form of huperzine A, which is a plant-derived compound traditionally used in Chinese medicine.
BIS-001 is being developed for use in children with Dravet syndrome, as well as some adults who experience seizures. The results of the company’s preclinical research were published in the journal, Frontiers in Pharmacology.
Dravet syndrome is a rare genetic disorder which results in prolonged, recurrent seizures in early life. Because the seizures are so frequent, children with Dravet syndrome face developmental delays as well as behavioral problems.
The SCN1A sodium channel is thought to be impaired in patients with Dravet syndrome, along with other forms of epilepsy. Currently-approved antiepileptic drugs are often ineffective in these patients, highlighting the urgent need for new therapies.
In order to assess the efficacy of BIS-001, the researchers used mice who carry the same SCN1A mutations and experience similar seizures as patients with Dravet syndrome. The researchers found that BIS-001 was able to eliminate seizures in most of the treated animals, with sustained antiepileptic effects over time.
“BIS-001 demonstrated a high level of efficacy in these models of extremely hard-to-treat epilepsy, eliminating all seizures in most animals and maintaining efficacy over repeat administration, at dose levels we are optimistic will be effective and well-tolerated in patients with our new extended release formulation,” said Dr. Stephen Collins, President and CEO of Biscayne Pharmaceuticals. “We are encouraged by these promising results in a model of devastating childhood epilepsy as we prepare to initiate a Phase 1b clinical trial of BIS-001 in another population with difficult-to-treat seizures – adults with refractory complex partial epilepsy.”
According to Biscayne, BIS-001 could advance into a Phase Ib clinical trial in adult participants by the middle of 2017. Assuming that BIS-001 advances into further trials, the company will look at testing the drug in children after taking the necessary steps to secure regulatory approval.
“The active ingredient of BIS-001 is huperzine A, an acetylcholinesterase (AChE) inhibitor that has a long history of safe use in Chinese medicine,” continued Collins. “It also has exhibited the cognition-enhancing properties seen with some other AChE drugs, but has demonstrated much better central nervous system and systemic tolerability and safety than currently available agents.
“We will be assessing whether BIS-001 supports improved cognition in epilepsy patients, and at the least, we expect it to 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.”