Biotherapeutic company raises $120M to advance drug-resistant epilepsy treatment

A biotherapeutics company has raised $120 million to advance its pipeline of cell therapies, including a lead investigational regenerative neural cell therapy candidate to treat drug-resistant mesial temporal lobe epilepsy.

According to a release from Neurona Therapeutics, financing comes on the heels of positive data from a clinical trial that included five individuals with a history of seizure activity not controlled by medication. Patients received a one-time dose of NRTX-1001 along with temporary immunosuppression to promote the long-term persistence of the cell therapy.

The first two patients, who recorded 32 and 14 seizures per month during the 6-month baseline, respectively, reported a reduction of more than 95% in overall seizure counts more than 1 year after administration of NRTX-1001.

The remaining three patients were given NRTX-1001 more recently, and were followed for 3 months post-dose. Two of the three demonstrated reduced monthly seizure frequencies of 76% and 87% from baseline levels (26 and 30 seizures per month, respectively) since the first month, the company reported in the release.

NRTX-1001 has also been generally well-tolerated, with adverse events mostly mild to moderate and typical of those on a temporary immunosuppression regimen. No severe adverse events were reported from either the cell therapy, delivery procedure or immunosuppression regimen, per the release.

“We are grateful for the significant investment from this reputable syndicate of new and existing investors,” Neurona CEO and co-founder Cory R. Nicholas, PhD, said in the release. “It signifies the conviction that Neurona’s cell therapies have the potential to transform the treatment of previously refractory, devastating neurological disorders.”

Neurona plans to support prospective clinical studies of NRTX-1001 indicated for Alzheimer’s disease, according to the release.