CHARLESTON, S.C. – Neuroene Therapeutics announced that the FDA has granted orphan drug designation for the company’s proprietary compound: NT102. NT102 was developed as part of a novel patented platform of mitochondrial and neuroprotective chemicals for the treatment of neurological diseases such as pharmacoresistant epilepsies. In preclinical studies funded by the National Institutes of Health (grant R44NS097047), NT102 was shown to be well-tolerated and orally bioavailable, with excellent brain penetration. NT102 has broad seizure protection in several preclinical animal models and in particular has excellent protection against seizures in Dravet syndrome – a rare childhood syndrome marked by severe encephalopathy and epilepsy. Under the Orphan Drug Act, the FDA may grant orphan drug designation to drugs intended to treat a rare disease or condition; generally a disease or condition affecting fewer than 200,000 individuals in the U.S.
Neuroene’s collaborators at the University of Utah Anticonvulsant Drug Development Program (ADD) performed efficacy studies in a Dravet Syndrome mouse model with a mutation in the same gene as most Dravet Syndrome patients and displays a phenotype similar to human Dravet patients including heat-induced seizures and spontaneous recurrent seizures. The ADD found that while only combinations of FDA-approved antiseizure drugs were effective at reducing seizures in this model, NT102 showed potent single-agent efficacy.
“This is an exciting development for Neuroene Therapeutics, and patients and families of those suffering Dravet syndrome,” said Drs. Sherine Chan and James Chou, co-founders of Neuroene Therapeutics. “We could also apply our compounds to more broad-spectrum epilepsies in the future, as they are efficacious in many animal models of medication-resistant seizures. But we now have a new potential treatment for Dravet syndrome.”
Neuroene Therapeutics: Understanding Dravet Syndrome
Dravet syndrome is a rare childhood syndrome affecting 1 in 15,700 infants born in the U.S. and is characterized by encephalopathy and epilepsy. Difficult to diagnose because many symptoms are not apparent in the first year of a child’s life. Seizures are the most prevalent symptom and can present following an increase in core temperature (e.g. fever, hot bath), with continued frequency occurring thereafter, often progressing in severity.
Full diagnosis requires gene testing ordered by a neurologist, but according to the Dravet Syndrome Foundation, those who are eventually diagnosed include at least four of the following symptoms:
- Normal or near-normal cognitive and motor development before seizure onset.
- Two or more seizures, with or without a fever, before age one.
- A history including myoclonic, hemiclonic, or generalized tonic-clonic seizure activity.
- Two or more seizures lasting longer than 10 minutes.
- No response to first-line antiepileptic drug therapy and continued seizures past age two.
In 80–90% of Dravet syndrome cases, the disease is caused by a mutation in the SCN1A gene, encoding a subunit of the voltage gated sodium channel Nav1.1. Insufficient activity of this sodium channel alters excitatory/inhibitory balance in the brain, causing hyperexcitability.
Dravet syndrome is classified in the most severe end of the spectrum of SCN1A-related disorders and had previously been well-known to be pharmacoresistant.
About Neuroene Therapeutics
Founded in 2015, Neuroene Therapeutics is a biotech company focused on discovering, developing, and commercializing novel therapeutics for difficult-to-treat neurological disorders. Neuroene’s pipeline of candidates include NT102, which was granted Orphan Drug Designation from the FDA for the treatment of Dravet syndrome, a rare pharmacoresistant childhood epilepsy. Neuroene’s products also include compounds in development for Parkinson’s disease and rare mitochondrial disorders. For more information, research, and drug development updates, please visit www.NeuroeneTherapeutics.com.
Sherine Chan, Co-Founder and CEO