Press Release

Neurona Therapeutics Presents Clinical Development Plan for Lead Cell Therapy Program at the 16th Conference on Antiepileptic Drug and Device Trials (AEDD TRIALS XVI)

– Supporting data in preclinical model of drug-resistant epilepsy demonstrate seizure-freedom in 70% of the NRTX-1001 cell therapy treatment group versus 5% in the control group

– Data demonstrate accurate and reproducible on-target administration of the NRTX-1001 cell therapy using the intended clinical delivery device

– Neurona expects to file an Investigational New Drug (IND) Application this year to begin a Phase 1/2a clinical trial in people with drug-resistant focal epilepsy

SAN FRANCISCO, June 16, 2021 – Neurona Therapeutics, a biotherapeutics company advancing restorative neural cell therapies for the treatment of chronic neurological disorders, today announced the presentation of clinical trial plans and supporting preclinical data for its lead inhibitory neuron cell therapy candidate, NRTX-1001, which is being developed for the treatment of drug-resistant focal epilepsy. The presentation was made at the 16th Conference on Antiepileptic Drug and Device Trials (AEDD TRIALs XVI) sponsored by The Epilepsy Study Consortium in collaboration with the University of Pennsylvania and the Epilepsy Foundation.

“The data presented at AEDD TRIALS XVI demonstrate that NRTX-1001 can be accurately and safely delivered to the hippocampus, which is the tissue most frequently affected in focal epilepsy, using a clinically-relevant MRI-guided delivery procedure,” said Kim J. Burchiel, M.D., John Raaf professor and chairman emeritus of the Department of Neurological Surgery at Oregon Health & Science University, and a collaborator on the delivery studies. “Neurona has also generated compelling preclinical data in a focal seizure model that support clinical advancement of NRTX-1001 as a potential restorative option for people living with chronic focal epilepsy.”

“We look forward to advancing NRTX-1001 into a first-in-human clinical study in people with chronic mesial temporal lobe epilepsy (MTLE), ” said Cory Nicholas, Ph.D., Neurona’s president and chief executive officer. “NRTX-1001 is a neuronal cell therapy that targets seizure-onset regions in the brain where it can functionally integrate, persist long-term, and restore balance to hyperactive neural networks following a one-time administration.”

Neurona plans to conduct a multicenter, open label, dose escalation clinical study to investigate safety and preliminary efficacy of NRTX-1001 in up to 15 adults with chronic MTLE. Patients will receive a single administration of the cell therapeutic. The primary endpoint of the proposed clinical study is safety and tolerability, however other endpoints will be evaluated including change in seizure frequency and severity, quality of life and neuropsychological outcomes.

Data presented at the AEDD meeting demonstrated successful on-target delivery of NRTX-1001 in an anatomically relevant preclinical model using standard equipment of the type that will be used in the trial. Further, the data showed on-target cell distribution, persistence, and tolerability across the intended clinical dosing range, which was confirmed by MRI contrast and histological analysis. No adverse behavioral changes were observed in the treatment group over the study duration.

Neurona has generated supporting safety and efficacy data in a preclinical model of drug-resistant mesiotemporal seizures with stable seizure-freedom achieved for the duration of the study, up to nine months, in 70% of the cell treatment group versus 5% in the control group. Robust and reproducible seizure suppression was achieved with multiple, independently produced lots of NRTX-1001 across a broad dosing range. Moreover, the NRTX-1001 treatment significantly decreased damage in the temporal lobe.

About MTLE

Mesial temporal lobe epilepsy (MTLE) involves the internal structures of the temporal lobe. Seizures often begin in a structure of the brain called the hippocampus. MTLE accounts for almost 80% of all temporal lobe seizures. For people with seizures that are resistant to drugs, epilepsy surgery, where the damaged temporal lobe is surgically removed or laser ablated, can be an option for some. However, current surgical options are not available or effective for all, are tissue-destructive, and can have significant adverse effects.

About NRTX-1001

NRTX-1001 is an inhibitory nerve cell therapy derived from human pluripotent stem cells using Neurona’s proprietary manufacturing process. The inhibitory neurons, or interneurons, secrete the inhibitory neurotransmitter, gamma-aminobutyric acid (GABA). Delivered as a one-time administration, the human interneurons integrate and innervate on-target, providing local long-term GABAergic inhibition to rebalance and repair hyperexcitable neural networks that underlie epilepsy as well as other disorders of the nervous system. Neurona is initially focused on developing NRTX-1001 as a restorative treatment for MTLE, which is the most common type of focal epilepsy.

About Neurona

Neurona’s 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 restorative 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