Clinical assessments three-months post-treatment report no serious adverse events and reduced seizure frequency to date in first trial participant

Data Safety Monitoring Board clears advancement of trial to continue enrollment

Pioneering cell therapy approach could provide a disease-modifying treatment for drug-resistant focal epilepsy

Data presented at ISSCR-ASGCT Conference: Emerging Therapies at the Intersection of Genetic and Cellular Technologies

SAN FRANCISCO, CA, USA I September 21, 2022 I Neurona Therapeutics, a clinical-stage biotherapeutics company advancing regenerative cell therapies for the treatment of neurological disorders, today announced the presentation of a clinical case study from the initial dose cohort of the Phase 1/2 first-in-human epilepsy clinical trial of its lead program, NRTX-1001. The data from three months post-cell therapy administration to the first patient in this study show that there were no serious adverse events associated with NRTX-1001 treatment and that the patient has thus far experienced reduced seizure frequency from an average prior history of 30+ seizures per month to four seizures in total during three months of follow-up to date. The data are being presented by Catherine Priest, Ph.D., Neurona’s chief development officer at the ISSCR-ASGCT Conference: Emerging Therapies at the Intersection of Genetic and Cellular Technologies being held in Madison, WI, September 21 -23, 2022.

“The reduced number of seizures reported by the first person to receive NRTX-1001 is very encouraging, and we remain cautiously optimistic that this reduction in seizure frequency will continue and extend to others entering this cell therapy trial. NRTX-1001 administration has been well tolerated thus far in the clinic, which is in line with the extensive preclinical safety data collected by the Neurona team,” said Cory R. Nicholas, Ph.D. Neurona’s president and chief executive officer. “With recent clearance from the Data Safety Monitoring Board we are excited to continue patient enrollment. We are very grateful to these first participants, and thank the clinical teams for the careful execution of this pioneering study.”

In addition to the preclinical data supporting the clinical program, the presentation highlights initial data from the first patient treated in Neurona’s clinical trial who received a starting dose of NRTX-1001. The patient is a young adult male with a 9-year history of seizures and was diagnosed with unilateral mesial temporal lobe epilepsy (MTLE). In the six months prior to the administration of NRTX-1001, the patient experienced an average of 32 seizures per month, despite being on several antiepileptic medications. The patient received NRTX-1001, and the treatment was well tolerated; there have been no serious adverse events to date. The patient has reported having four seizures during the first three months since receiving NRTX-1001.

The clinical trial is funded in part by the California Institute for Regenerative Medicine (DISC2-10525; TRAN1-11611; CLIN2-13355).

About Neurona’s Clinical Trial of NRTX-1001 for Mesial Temporal Lobe Epilepsy (MTLE)
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, with five patients to be treated at a starting dose and five at a higher dose. Patients treated with a single infusion of NRTX-1001 cells will be monitored for safety, tolerability, and effects on their epilepsy disease symptoms. Patient recruitment is underway at epilepsy centers across the United States. For more information, please visit (NCT05135091). The first part of the clinical trial is supported by a recently announced $8.0 million grant from the California Institute for Regenerative Medicine (CIRM; CLIN2-13355).

About NRTX-1001
NRTX-1001 is a regenerative neural cell therapy candidate 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.

About Mesial Temporal Lobe Epilepsy (MTLE)
An estimated three million Americans have epilepsy, and 25 to 35 percent live with ongoing seizures despite treatment with approved drugs, which means that there is a huge unmet medical need in this community. MTLE is the most common type of focal epilepsy in adults and primarily affects the internal structures of the temporal lobe, where seizures often begin in a structure called the hippocampus. For people with seizures that are 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.

About Neurona
Neurona’s regenerative cell therapy candidates 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 about Neurona, visit

SOURCE: Neurona Therapeutics