Press Release

Neurona Therapeutics Presents Clinical Data for NRTX-1001 Regenerative Cell Therapy for Drug-Resistant Focal Epilepsy at American Epilepsy Society Annual Meeting

– NRTX-1001 administration was well-tolerated, with notable seizure reduction reported in early data from the first two patients in Phase I/II clinical trial

– Additional preclinical data highlight robust and reproducible in-house manufacturing of NRTX-1001, the clinical delivery system, and candidate metabolic biomarkers of therapeutic activity

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

San Francisco, CA, December 5, 2022 – Neurona Therapeutics, a clinical-stage biotherapeutics company advancing regenerative cell therapies for the treatment of neurological disorders, announced the presentation of clinical data from Neurona’s ongoing Phase I/II clinical trial of NRTX-1001 in people with mesial temporal lobe epilepsy (MTLE) as well as supporting preclinical and manufacturing data at the annual meeting of the American Epilepsy Society. The meeting is being held in Nashville, TN, December 2-6, 2022.

In a poster entitled: NRTX-1001: First-in-class human inhibitory neuron cell therapy for phase I/II clinical investigation in chronic focal epilepsy, data were provided demonstrating the characterization, delivery, and initial clinical trial progress from the first patients treated in the groundbreaking clinical trial (NCT05135091). Two patients have been treated to date, both of whom entered the study with a history of significant monthly seizure activity that was not controlled by anti-seizure medications. Following treatment with NRTX-1001, which was well tolerated with no serious or severe adverse events, both patients have thus far seen a notable reduction in seizure activity. The first patient had a 9-year history of seizures, and 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 reported four seizures during the first three months since receiving NRTX-1001. The second patient treated in the trial also had drug-resistant seizures, with an average of 14 seizures per month in the six months prior to treatment and, in the first month post-treatment, reported one seizure since receiving the cell therapy. 

“We are very encouraged by the early clinical data from our NRTX-1001 trial in people with drug-resistant focal epilepsy who have tolerated the treatment well and have reported reductions in seizure activity thus far,” said David Blum, chief medical officer of Neurona. “Currently, there are few effective therapeutic options for people with this type of epilepsy, including a lobectomy surgery to remove or ablate brain tissue, which can have tissue-destructive adverse effects on memory and vision. NRTX-1001 is a regenerative cell therapeutic with the potential to durably eliminate seizures after a single administration. We look forward to enrolling additional patients into the NRTX-1001 clinical trial and advancing this novel therapy for patients for whom anti-seizure medication has failed.”

A second presentation entitled: NRTX-1001: Human inhibitory neuron cell therapy suppresses seizures and reduces histopathology in a mouse model of focal epilepsy with high repeatability across multiple studies and manufacturing lots, provided data from the manufacturing and preclinical characterization of NRTX-1001, highlighting the reproducibility of Neurona’s proprietary in-house manufacturing process. 

NRTX-1001 is an injectable suspension of high-purity inhibitory neurons, called interneurons, derived from human stem cells and designed to replace damaged neurons in the region of the brain where seizures originate. The cells are manufactured in Neurona’s in-house GMP facility, using proprietary methods to guide the stem cells into a specific, pallial-type, lineage of interneurons that secrete the inhibitory neurotransmitter gamma-aminobutyric acid (GABA). NRTX-1001 interneurons are cryopreserved and shipped to clinical sites to be used as an allogeneic, off-the-shelf investigative therapy.

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 an $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 

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 a 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 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 patients, 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

Investor and Media Contacts:

Sylvia Wheeler
Wheelhouse LSA

Elizabeth Wolffe, Ph.D.
Wheelhouse LSA