Press Release

Neurona Therapeutics Completes Clinical Enrollment of First Cohort and Updates on Long-lasting Impact in First Patients Treated in Ongoing Phase I/II Trial of NRTX-1001 Cell Therapy for Drug-resistant Focal Epilepsy 

– The first subject to receive NRTX-1001 tapered off immunosuppression and continues to experience stable seizure reduction (>95%) in the second year post-administration

– Encouraging results were seen in the second subject, where the cell therapy has been well-tolerated with continued reduction (>95%) in overall monthly seizure frequency at one-year post-treatment

– Preliminary data from all five subjects treated in the first dose cohort is expected to be announced in Q4 2023

San Francisco, CA, October 19, 2023 – Neurona Therapeutics, a clinical-stage biotherapeutics company advancing regenerative cell therapy candidates for the treatment of neurological disorders, announced completion of enrollment of the first cohort of five subjects at the starting dose-level in its ongoing open-label, single-arm 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). 

The five subjects in the first cohort received a one-time administration of NRTX-1001 along with immunosuppression administered in the first year, which is intended to promote integration and long-term persistence of NRTX-1001. No serious or severe adverse events from the cell therapy or immunosuppression regimen have been reported thus far in the ongoing clinical trial. The first subject in the clinical trial completed the immunosuppression regimen per protocol, has discontinued the immunosuppression, and continues to show >95% stable seizure reduction 16 months from cell administration. The second subject in the clinical trial is now 12 months from cell administration, has also reported >95% seizure reduction, and has been free of seizures for the past six months.

“The early data from our ongoing open-label Phase I/II clinical trial have been promising to date, demonstrating that NRTX-1001 has been well tolerated thus far and may result in substantial, durable seizure reduction in subjects with drug-resistant mesial temporal lobe epilepsy,” Cory R. Nicholas, Ph.D., Neurona’s chief executive officer. “To see the data continue to hold during long-term 12-plus months of follow-up with the first two subjects is extremely rewarding. We look forward to enrolling our second cohort of five subjects and advancing the development of this potentially transformative therapy for people living with drug-resistant epilepsy.”

“The first subject’s transition off immunosuppression therapy with continued >95% reduction in overall monthly seizure counts represents a very encouraging development for the potential durability of this treatment,” said David Blum, M.D., Neurona’s chief medical officer. “The second subject treated with a single dose of NRTX-1001 has reached the one-year endpoint, also continues to do well with >95% seizure reduction and has not reported a seizure for the past six months. We anticipate updating on the progress of these two patients and providing data from the additional patients in this first cohort in the fourth quarter of 2023.”

The first two subjects to receive NRTX-1001 in the ongoing clinical trial (NCT05135091) entered the study with a history of significant monthly seizure activity that was not controlled by anti-seizure medications. The first subject had a seven-year history of seizures and, in the six months before the administration of NRTX-1001, experienced an average of 32 seizures per month. The second subject treated in the trial had a nine-year history of seizures and averaged 14 monthly seizures in the six months before treatment. Both subjects continue to report a >95% reduction in overall seizure counts at 16 months and one-year after NRTX-1001 administration, respectively. Per protocol, both subjects had neurocognitive testing post-treatment, and a subset of their neurocognitive scores has increased from baseline levels, representing potential improvements in memory.

Three additional subjects have been treated with NRTX-1001, which completes the first cohort. None have had serious or severe adverse events to date. 

About Neurona’s Clinical Trial of NRTX-1001 for Mesial Temporal Lobe Epilepsy (MTLE)

The 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 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 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 neural circuits and are designed to 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 

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

About Neurona

Neurona is focused on developing regenerative cell therapy candidates with single-dose curative potential. Neurona’s investigational allogeneic neural 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 subject to risks and uncertainties, including risks and uncertainties inherent in the clinical development of therapeutic candidates, risks that earlier 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 and Media Contacts:

Sylvia Wheeler
Wheelhouse LSA

Elizabeth Wolffe, Ph.D.
Wheelhouse LSA