Neurona Therapeutics Announces Priority Medicines (PRIME) Designation Granted by the European Medicines Agency (EMA) to NRTX-1001 in the Treatment of Drug-Resistant Focal Epilepsy

EMA’s PRIME designation bolsters Neurona’s global development strategy and underscores the company’s commitment to bringing NRTX-1001 interneuron cell therapy to patients worldwide.

SAN FRANCISCO, Nov. 05, 2025 (GLOBE NEWSWIRE) -- Neurona Therapeutics, a clinical-stage biotherapeutics company advancing regenerative cell therapies for disorders of the nervous system, today announced that its lead product candidate, NRTX-1001, has been granted the PRIority MEdicines (PRIME) designation by the European Medicines Agency (EMA) for the treatment of adults with drug-resistant focal epilepsy. EMA created the PRIME designation to recognize the potential of a medicine to address a significant unmet medical need and to facilitate enhanced dialogue in support of accelerated development and regulatory review in the European Union.

NRTX-1001 is an off-the-shelf, allogeneic GABAergic interneuron cell therapy candidate that is being evaluated in two ongoing multicenter, open-label Phase 1/2 clinical trials in adults with drug-resistant mesial temporal lobe epilepsy (MTLE), which is the most common type of focal epilepsy in adults. Preparations are also underway to start screening potential subjects with drug-resistant MTLE for Neurona’s previously announced pivotal Phase 3 EPIC (EPIlepsy Cell therapy) randomized controlled trial.

For focal epilepsy patients whose disease does not adequately respond to medications, the set of current treatment options is limited to highly invasive surgical procedures, including lobectomy resection or ablation surgeries, which are brain tissue-destructive. Destructive surgeries carry a significant risk of irreversible neurocognitive impairment; the risk of such impairment is elevated enough to exclude many drug-resistant patients from eligibility.

In contrast, NRTX-1001 is an investigative cell therapy alternative that is being evaluated in ongoing clinical trials for people living with drug-resistant seizures, administered in a one-time, minimally-invasive delivery procedure. By integrating into dysregulated neural circuits, secreting GABA, and restoring inhibitory tone from a single dose, NRTX-1001 has the potential to substantially and durably reduce or eliminate seizures in focal epilepsy patients whose disease is inadequately controlled by traditional anti-seizure medications. Additionally, due to its non-destructive design, NRTX-1001 may avoid the risk of neurocognitive impairment associated with invasive surgical procedures.

“Receiving PRIME designation from the EMA is a major milestone for Neurona and strong external validation of NRTX-1001’s potential to change the treatment paradigm for people living with drug-resistant focal epilepsy,” said Cory R. Nicholas, Ph.D., Neurona’s Chief Executive Officer and Co-Founder. “This designation empowers our team to work hand-in-hand with the EMA toward efficient development of NRTX-1001 for a broad population of focal epilepsy patients who are presently underserved by current pharmacological and surgical options.”

The EMA awarded the PRIME designation to NRTX-1001 based on robust preclinical evidence, emerging clinical data, and the unmet need in patients with drug-resistant focal epilepsy. With this PRIME designation, Neurona is eligible for early scientific advice, protocol assistance, accelerated assessment, and other regulatory incentives in Europe. Under European Union regulations, this designation also simplifies NRTX-1001’s regulatory pathway by allowing for a single centralized MAA submission.

Neurona presented updated data from its unilateral MTLE trial at the AAN Annual Meeting in April 2025. The results included a 92% median reduction from baseline in disabling seizures during the 7-12-month efficacy endpoint period, an 80% (4/5) responder rate with >75% seizure reduction at the 12-month endpoint, and durable seizure control in all (4/4) responders followed for 18-24 months after a single low-dose of NRTX-1001 (Cohort 1). All subjects (5/5) in Cohort 1 had significantly improved quality-of-life test scores, with no subject to date experiencing persistent decline in cognition. Importantly, NRTX-1001 continues to be well-tolerated with no serious adverse events to date attributed to the cell therapy.

Neurona plans to present updated data from across the clinical development program for NRTX-1001 in MTLE at the Annual Meeting of the American Epilepsy Society (AES) in December 2025, including early data from recently added cohorts of bilateral and non-MTS MTLE subjects.

About Neurona Therapeutics

Neurona is developing allogeneic, off-the-shelf, regenerative neural cell therapies with the potential to provide long-term targeted repair of the nervous system following a single administration. Neurona’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 (MTLE), and NCT06422923 for drug-resistant bilateral MTLE, with expansion to neocortical focal epilepsy and other indications planned in the future. The Phase 1/2 MTLE clinical trials are supported by grants from the California Institute for Regenerative Medicine (CIRM; CLIN2-13355 and CLIN2-17135). The FDA granted the Regenerative Medicine Advanced Therapy (RMAT) designation to NRTX-1001 in June 2024. Consistent with Neurona’s discussion with the FDA, the Phase 3 EPIC (EPIlepsy Cell therapy) trial is planned to start in 2H 2025. For more information about Neurona, visit: www.neuronatherapeutics.com.

Investor Contact:
Laurence Watts
New Street Investor Relations
laurence@newstreetir.com

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