Press Release

Neurona Therapeutics Announces Presentation of Updated Clinical Data at the Upcoming American Academy of Neurology (AAN) Annual Meeting from Lead Cell Therapy Candidate, NRTX-1001

– NRTX-1001 is being evaluated in an ongoing Phase I/II clinical trial in adults with drug-resistant focal epilepsy

San Francisco, CA, March 07, 2024 – Neurona Therapeutics, a clinical-stage biotherapeutics company advancing regenerative cell therapy candidates for the treatment of neurological disorders, today announced that updated data from its ongoing Phase I/II clinical trial of NRTX-1001, an investigational allogeneic cell therapy candidate that is being developed for treatment of drug-resistant mesial temporal lobe epilepsy (MTLE), will be presented at the American Academy of Neurology (AAN) 2024 Annual Meeting, which will be held April 13-18, 2024, in Denver, CO.

AAN 2024 Presentation Details:

Title: First-in-Human Trial of NRTX-1001 GABAergic Interneuron Cell Therapy for Treatment of Focal Epilepsy - Emerging Clinical Trial Results
Format: Oral (abstract # 5721, presentation #002)
Session: S19: Epilepsy Clinical Trials and Long-term Studies
Presenter: John Hixson, M.D., Senior Medical Director, Neurona Therapeutics
Date: Monday, April 15, 2024
Time: 3:42 p.m. MT

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 on-target and durably silence seizure activity in the epileptic region of the brain. NRTX-1001 is manufactured in Neurona’s in-house GMP facility using proprietary methods. 

About Mesial Temporal Lobe Epilepsy (MTLE)

According to the Centers for Disease Control and Prevention, an estimated 3.4 million Americans have epilepsy, and 25-35% live with ongoing seizures despite treatment with approved drugs, illustrating a substantial 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 subjects, are tissue-destructive, and can have significant adverse effects.

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

The ongoing clinical study 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 drug-resistant MTLE, with five subjects in the first cohort treated at a starting dose and five subjects in the second cohort to be treated at a higher dose. Subjects treated with a single infusion of NRTX-1001 cells will be monitored for safety and effects on their epilepsy disease symptoms. Subject 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). With $5.5 billion in funding and more than 150 active stem cell programs in its portfolio, CIRM is one of the world’s largest institutions dedicated to helping people by bringing the future of cellular medicine closer to reality.

About Neurona Therapeutics

Neurona is focused on developing regenerative cell therapy candidates with single-dose curative potential. Neurona’s investigational allogeneic, off-the-shelf, cell therapy candidates are designed to provide long-term repair of dysfunctional neural networks for multiple neurological disorders. For more information about Neurona, visit

Forward-Looking Statements

This press release includes forward-looking statements that are subject to risks and uncertainties, including risks and uncertainties inherent in the clinical development of therapeutic candidates, risks that early clinical data collected from a small number of subjects may not be predictive of data when NRTX-1001 is tested in larger clinical trials, the possibility that timelines may change, and difficulties associated with obtaining sufficient data to demonstrate safety and efficacy to support regulatory approval. NRTX-1001 is an investigational candidate and is being evaluated in ongoing clinical trials. NRTX-1001 has not been approved by any regulatory authority for commercial use or deemed to be safe or effective for any indication.

Investor Contacts:

Sylvia Wheeler
Wheelhouse LSA

Elizabeth Wolffe, Ph.D.
Wheelhouse LSA

Media Contact:

Aljanae Reynolds
Wheelhouse LSA