Neurona Expands Investigation of NRTX-1001 Cell Therapy into Mesial Temporal Lobe Epilepsy (MTLE) without Mesial Temporal Sclerosis (MTS)
- First subject received unilateral administration of NRTX-1001 in a new non-MTS cohort; no significant surgical complications or adverse events reported to date -
- Data from this non-MTS cohort, together with results from Neurona’s ongoing Phase 1/2 trials in unilateral and bilateral MTLE and planned Phase 3 EPIC study, are intended to support a future BLA submission for NRTX-1001 in drug-resistant MTLE -
- Neurona to provide an update on its unilateral and bilateral MTLE programs at the American Epilepsy Society’s Annual Meeting in December 2025 -
SAN FRANCISCO, Oct. 07, 2025 (GLOBE NEWSWIRE) -- Neurona Therapeutics, a clinical-stage biotherapeutics company advancing regenerative cell therapies for disorders of the nervous system, today announced that the first adult subject with drug-resistant unilateral mesial temporal lobe epilepsy (MTLE) without Mesial Temporal Sclerosis (MTS) has been treated in its ongoing Phase 1/2 clinical trial of NRTX-1001 cell therapy. The subject was dosed in an operation performed by Dr. Viktoras Palys, M.D., neurosurgeon at the University of Arkansas for Medical Sciences (UAMS), and is part of a separate expansion cohort within the Phase 1/2 trial, which will enroll only non-MTS subjects.
“NRTX-1001 targets the underlying biology of MTLE, which makes it highly differentiated from currently approved treatments for drug-refractory epilepsy,” said Dr. Sisira Yadala, M.D., Neurologist and Director of the Division of Epilepsy and Level 4 Comprehensive Epilepsy Center at the University of Arkansas for Medical Sciences, who oversees treatment for the subject. “Current surgical treatments, whether it be surgical resection or laser ablation, are tissue-destructive and can lead to irreversible cognitive impairment. It is important that Neurona is expanding their NRTX-1001 program into patients without MTS, which represents a notable subset of the drug refractory MTLE population.”
MTLE is among the most common types of focal epilepsy in adults and is characterized by seizures originating primarily from the hippocampus, a core learning and memory center of the brain, from either one hemisphere (unilateral) or both hemispheres (bilateral). The presence of MTS, which is often detectable by MRI, is typically a pathological hallmark of MTLE and involves hippocampal scarring, cell loss, and tissue atrophy. However, a subset of MTLE cases present without detectable MTS and are characterized instead on seizure semiology, scalp EEG, and other techniques, which may include magnetoencephalography (MEG), intracranial EEG, and PET imaging, to localize seizure onset.
Neurona is now actively dosing an expansion cohort of drug-resistant unilateral MTLE subjects without MTS. In total, the non-MTS cohort will enroll up to ten adult subjects who will receive NRTX-1001 via a one-time image-guided intracerebral procedure identical to that used for subjects in the other Phase 1/2 study cohorts. This expansion builds upon encouraging preliminary efficacy and safety data from Neurona’s ongoing multicenter, open-label Phase 1/2 trial (NCT05135091) in unilateral MTLE subjects with MTS.
As part of the overall MTLE clinical program, Neurona is also enrolling a second Phase 1/2 trial (NCT06422923) of NRTX-1001 cell therapy in adult subjects with drug-resistant bilateral MTLE, both with and without MTS. Following discussions with FDA under the Regenerative Medicine Advanced Therapy (RMAT) designation regarding the design of a registrational study, Neurona has also begun preparations for its prospective Phase 3 EPIC (EPIlepsy Cell Therapy) trial, which the company expects to initiate later this year. The results from the Phase 1/2 and Phase 3 trials in both unilateral and bilateral MTLE populations are intended to support a prospective marketing application for the treatment of drug-resistant MTLE.
“We’re excited to expand our clinical development of NRTX-1001 to include MTLE without MTS,” said Cory R. Nicholas, Ph.D., Neurona’s Chief Executive Officer and Co-Founder. “The expansion of our trial to include this cohort underlines our conviction that NRTX-1001 cell therapy has the potential to improve the lives of a broad range of people who suffer from refractory focal epilepsy.”
In April, Neurona presented updated data from its unilateral MTLE trial at the AAN Annual Meeting. 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 this non-MTS MTLE cohort. At the same meeting, Neurona also plans to present additional data from the other cohorts of the ongoing Phase 1/2 study in unilateral MTLE, including updated efficacy and extended durability data for subjects enrolled in Cohorts 1 (low-dose NRTX-1001) and 2 (high-dose NRTX-1001), and early data from the recently-initiated Phase 1/2 study in bilateral MTLE.
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.
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