Neurona Unveils Phase 3 EPIC Study for NRTX-1001 Cell Therapy in Epilepsy

– Based on alignment gained with FDA under the Regenerative Medicine Advanced Therapy (RMAT) designation, Neurona plans to conduct the Phase 3 EPIC (EPIlepsy Cell therapy) trial as the primary basis for a future NRTX-1001 Biologics License Application

– The Phase 3 EPIC trial is a randomized, sham-controlled, double-blind study evaluating NRTX-1001 cell therapy in adults with drug-resistant mesial temporal lobe epilepsy

San Francisco, CA, February 20, 2025 – Neurona Therapeutics, a clinical-stage, privately-held, biotherapeutics company advancing regenerative cell therapies for disorders of the nervous system, today announced plans to initiate the Phase 3 EPIC clinical trial for its lead product candidate, NRTX-1001. NRTX-1001 is the first investigational human cell therapy for drug-resistant epilepsy. The EPIC trial is anticipated to begin in the second half of 2025 and is designed as a randomized, sham-controlled, double-blind study of NRTX-1001 in people living with drug-resistant mesial temporal lobe epilepsy (MTLE), which is the most common type of epilepsy in adults. 

“The advancement of NRTX-1001 into the Phase 3 EPIC study is a significant milestone for the field of regenerative medicine and provides hope for the epilepsy community of patients and caregivers in need of new treatment options,” said Cory R. Nicholas, Ph.D., Neurona’s Chief Executive Officer and Co-Founder. “We are delighted to be moving NRTX-1001 one step closer to a potential marketing application.”

“People living with drug-resistant seizures face a significant burden due to the substantial risks of ongoing seizures. They often have limited therapeutic options, such as surgery to remove or ablate the seizure-prone region of the brain, which can cause irreversible impairment of memory and other neurocognitive functions,” explained Manher A. Joshi, M.D., Neurona’s Chief Medical Officer. “NRTX-1001 is intended to provide a novel tissue-sparing approach to durably restore balanced activity to the brain from a single treatment, while potentially avoiding the serious neurocognitive risks of current tissue-destructive surgical interventions. We are excited about the potential of NRTX-1001 to transform the therapeutic landscape for drug-resistant focal epilepsy.” 

The advancement of NRTX-1001 into the Phase 3 EPIC trial follows productive discussions with the U.S. Food and Drug Administration under an RMAT designation granted in June 2024. Pending the outcome of this Phase 3 trial, and supported by ongoing Phase 1/2 trials, the results are intended to be part of a robust data package to support regulatory submission for drug approval.

In December at the American Epilepsy Society’s Annual Meeting, Neurona Therapeutics presented updated data from both the low-dose (n=5) and high-dose (n=5) cohorts of the Company’s ongoing open-label Phase 1/2 clinical trial of NRTX-1001 GABAergic interneuron cell therapy in adults with drug-resistant, unilateral MTLE. The low-dose cohort demonstrated a 92% median reduction in disabling seizures from baseline, with 80% of subjects reporting >80% seizure reduction, during the primary efficacy evaluation period of 7-12 months after NRTX-1001 administration. Notably, the first two subjects followed for 24 months post-dosing both continued to report >97% seizure reduction after a single administration of NRTX-1001. Although the 7-12-month results are not yet available for the high-dose, this cohort demonstrated a 78% median reduction in disabling seizures from baseline in the interim 4-6-month period after administration of NRTX-1001. Importantly, no cognitive impairments were detected in either cohort, and some subjects had neurocognitive test scores that were markedly improved from baseline levels. NRTX-1001 has been well-tolerated to date at both high and low doses, with no adverse events attributed to the cell therapy.

About Neurona Therapeutics

Neurona is developing allogeneic, off-the-shelf, regenerative neural cell therapy products with the potential to provide long-term targeted repair of the nervous system following a single administration. The Company’s lead product candidate, NRTX-1001, comprising GABAergic interneurons, is currently being studied for safety and efficacy in two ongoing open label multicenter Phase 1/2 trials: NCT05135091 for drug-resistant unilateral mesial temporal lobe epilepsy, and NCT06422923 for drug-resistant bilateral mesial temporal lobe epilepsy, with neocortical focal epilepsy and other indications planned in the future. The Phase 1/2 unilateral MTLE clinical trial is supported by an $8.0 million grant from the California Institute for Regenerative Medicine (CIRM; CLIN2-13355). The FDA granted the Regenerative Medicine Advanced Therapy (RMAT) designation to NRTX-1001 in June 2024 based on positive clinical data from the ongoing Phase 1/2 trial in unilateral MTLE. For more information about Neurona, visit: www.neuronatherapeutics.com.

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