Press Release

Neurona Therapeutics Presents Preclinical Data at the American Academy of Neurology (AAN) Annual Meeting from Lead Cell Therapy Candidate, NRTX-1001, Being Evaluated in a Phase 1/2 Clinical Trial for Chronic Focal Epilepsy

San Francisco, CA, April 1, 2022 – Neurona Therapeutics, a clinical-stage biotherapeutics company advancing regenerative cell therapies for the treatment of neurological disorders, announced the presentation of preclinical data from its lead inhibitory neuron cell therapy candidate, NRTX-1001. NRTX-1001 is being evaluated in a Phase 1/2 clinical trial in people with drug-resistant mesial temporal lobe epilepsy. The data are being presented at the annual meeting of the American Academy of Neurology (AAN) held April 2-7, 2022 in Seattle, WA.

“These studies were part of an important body of work that supported the design of our firstin- human clinical trial and provided important insight into potential biomarkers using magnetic resonance spectroscopy (MRS) to correlate neurometabolic changes with seizure reduction following NRTX-1001 administration,” said Cory R. Nicholas, Ph.D., Neurona’s president and chief executive officer. “We are excited to have begun recruiting for the clinical trial and to evaluate the safety and efficacy of NRTX-1001, which has the potential to improve quality of life for people living with drug-resistant focal epilepsy.

The data, generated from a single targeted administration in a preclinical model of chronic mesial temporal lobe epilepsy, demonstrate that the NRTX-1001 inhibitory neurons persisted in the epileptic hippocampus for the duration of the 7-month study, were well-tolerated, and resulted in significant seizure suppression. The activity of NRTX-1001 was not perturbed by coadministration of the common anti-seizure drugs levetiracetam or diazepam. Moreover, the NRTX-1001-treated group exhibited significantly less mortality and reduced hippocampal sclerotic pathology compared to controls. Of translational relevance as a potential biomarker of seizure reduction, non-invasive 1H MRS analysis revealed a metabolic signature of the epileptic hippocampus that was corrected to normal, non-epileptic levels following NRTX-1001 administration.

About Neurona’s Clinical Trial of NRTX-1001 for MTLE

Neurona’s multi-center, Phase 1/2 clinical trial is designed to evaluate the safety and efficacy of a single administration of NRTX-1001 for drug-resistant mesial temporal lobe epilepsy (MTLE). The first stage of the trial is an open-label dose-escalation study in up to 10 people with MTLE. The second stage will consist of a randomized, blinded investigation of NRTX-1001 compared to a control group to determine safety and efficacy in up to 30 people with MTLE. Patient recruitment is underway at epilepsy centers across the United States. For more information, please visit (NCT05135091).

About NRTX-1001

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 hyperexcitable neural networks that underlie epilepsy, as well as other disorders of the nervous system.

About Mesial Temporal Lobe Epilepsy (MTLE)

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. For some people with seizures that are resistant to 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 neural cell therapies have single-dose curative potential. Based on a novel neural cell lineage developed by the company’s scientific founders, Neurona has built a robust regenerative platform and is developing off-the-shelf, allogeneic neuronal, glial, and gene-edited cell therapy candidates that provide long-term integration and repair of dysfunctional neural networks for multiple neurological disorders. For more information about Neurona, visit

Neurona Therapeutics

Investor and Media Contacts:

Sylvia Wheeler
Wheelhouse LSA

Elizabeth Wolffe, Ph.D.
Wheelhouse LSA