Neurona Therapeutics Announces Initial Subject Dosed in First Clinical Trial of Regenerative Human Cell Therapy in Adults with Drug-Resistant Focal Epilepsy

Neurona Therapeutics recently announced the first patient dosed in a Phase 1/2 clinical trial of its lead program, NRTX-1001, in a first-in-human epilepsy study. An estimated 3 million Americans have epilepsy, and 25% to 35% live with ongoing seizures despite dozens of approved drugs on the market, which means that there is a huge unmet medical need in this community. NRTX-1001 is a regenerative neural cell therapy delivered as a single dose and designed to provide long-term secretion of gamma-aminobutyric acid (GABA), a key inhibitory neurotransmitter, to repair hyper-excitable neural networks associated with mesial temporal lobe epilepsy (MTLE), the most common form of focal epilepsy in adults.

“Administration of NRTX-1001 to the first patient in our clinical trial for MTLE represents a huge milestone for Neurona and the neurology field,” said Cory R. Nicholas, PhD, Neurona’s President and Chief Executive Officer. “This promising program is the result of many years of work, dedication, and innovation by the Neurona team and its collaborators as well as key funding secured by grants received from the California Institute for Regenerative Medicine. We are very excited to take this next step, bringing a first-in-class, novel regenerative treatment to people living with chronic focal seizures. I would like to thank the excellent team at SUNY Upstate Medical University who treated the first patient, as well as our other clinical sites across the country who are part of this multicenter study.”

“Neurona’s regenerative cell therapy approach has the potential to provide a single-administration, non-destructive alternative for the treatment of drug-resistant focal epilepsy, Currently, people with mesial temporal lobe epilepsy who are not responsive to anti-seizure medications have few options, such as an invasive surgery that removes or destroys the affected brain tissue, added Harish Babu, MD, PhD, Assistant Professor of Neurosurgery at SUNY Upstate Medical University and the surgeon who administered the first dose of NRTX-1001.

“The objective of NRTX-1001 is to add cells that have the potential to repair the circuits that are damaged in epilepsy and thus reduce seizure activity,” said Robert Beach, MD, PhD, Professor of Neurology at SUNY Upstate Medical University. “We are delighted to work with the Neurona team to evaluate the potential of this exciting new therapeutic approach.”

Neurona’s multicenter, Phase 1/2 clinical trial is designed to evaluate the safety and efficacy of a single administration of NRTX-1001 for drug-resistant MTLE. The first stage of the trial is an open-label dose-escalation study in up to 10 people with MTLE. Patients treated with a single infusion of NRTX-1001 cells will be monitored for safety, tolerability, neural cell viability, and effects on their epilepsy disease symptoms. Patient recruitment is underway at epilepsy centers across the United States. For more information, visit www.clinicaltrials.gov (NCT05135091). The first part of the clinical trial is supported by a recently announced $8 million grant from the California Institute for Regenerative Medicine (CIRM; CLIN2-13355).

NRTX-1001 is a regenerative neural cell therapy derived from human pluripotent stem cells. The fully-differentiated neural cells, called interneurons, secrete the inhibitory neurotransmitter gamma-aminobutyric acid (GABA). Delivered as a one-time dose, the human interneurons are intended to integrate and innervate on-target, providing long-term GABAergic inhibition to repair hyper-excitable neural networks.

MTLE primarily affects the internal structures of the temporal lobe, where seizures often begin in a structure called the hippocampus. MTLE is the most common type of focal epilepsy in adults. For people resistant to anti-seizure drugs, epilepsy surgery, where the damaged temporal lobe is surgically removed or ablated by laser, can be an option. However, the current surgical options are not available or effective for all, are tissue-destructive, and can have significant adverse effects.

Neurona’s regenerative cell therapies have single-dose curative potential. Neurona is developing off-the-shelf, allogeneic neuronal, glial, and gene-edited cell therapy candidates that are designed to provide long-term repair of dysfunctional neural networks for multiple neurological disorders. For more information, visit www.neuronatherapeutics.com.