Neurona launches phase 3 epilepsy study for lead cell therapy

San Francisco-based biotech Neurona Therapeutics unveiled plans to initiate a phase 3 clinical trial for its lead cell therapy candidate, NRTX-1001, in drug-resistant epilepsy.

The EPIC trial is anticipated to begin in the second half of 2025 and is designed as a randomized, sham-controlled, double-blind study of NRTX-1001 in people living with drug-resistant mesial temporal lobe epilepsy (MTLE), the most common type of epilepsy in adults.

NRTX-1001 is comprised of human MGE-type inhibitory GABAergic interneurons derived from pluripotent stem cells. The therapy is intended to provide a tissue-sparing approach to durably restore balanced activity to the brain from a single treatment, potentially avoiding the serious neurocognitive risks of current tissue-destructive surgical interventions.

At the American Epilepsy Society’s Annual Meeting this past December, Neurona presented updated data from its ongoing phase 1/2 clinical trial of NRTX-1001 in adults with MTLE. The low-dose cohort demonstrated a 92% median reduction in disabling seizures from baseline, with 80% of subjects reporting >80% seizure reduction, during the period of 7-12 months after administration. The high-dose cohort demonstrated a 78% median reduction in disabling seizures from baseline in the interim 4-6 month period after administration.

Neurona intends to use the data from this ongoing phase 1/2 trial, as well as the outcome of the upcoming phase 3 trial, to support regulatory submission to the U.S. FDA.