- UNILATERAL MTLE: Low dose resulted in 89% median disabling seizure reduction in participants with MTS during the 7-12-month primary efficacy evaluation period (N=9), and 100% median disabling seizure reduction in participants without MTS during the 4-6-month interim period (N=3) - BILATERAL MTLE: Low dose resulted in 64% median disabling seizure reduction during the 4-6-month interim period (N=4)
- DURABILITY: Participants have maintained reductions in seizure frequency past the 12-month primary endpoint - SAFETY: Cell therapy continues to be well-tolerated, with no serious adverse events attributed to the cells or administration procedure - COGNITION: No substantial group declines in cognitive performance detected in any cohort - PIVOTAL TRIAL: Enrollment of first patient in EPIC Phase 3 trial of low-dose rezanecel in drug-resistant MTLE planned for 2H 2026 - REGULATORY: WHO/INN Programme recommended rezanecel as the non-proprietary name of NRTX-1001
SAN FRANCISCO, April 22, 2026 (GLOBE NEWSWIRE) -- Neurona Therapeutics, a clinical-stage biotherapeutics company developing regenerative cell therapies for epilepsy and other disorders of the nervous system, today presented new clinical results from its ongoing open-label Phase 1/2 trials evaluating rezanecel (NRTX-1001), a one-time, allogeneic interneuron cell therapy, for drug-resistant mesial temporal lobe epilepsy (MTLE), with or without mesial temporal sclerosis (MTS). The data were presented at the 2026 American Academy of Neurology (AAN) Annual Meeting, taking place in Chicago and online, April 18-22. The updates included data from participants enrolled in two Phase 1/2 clinical trials evaluating rezanecel in drug refractory unilateral or bilateral MTLE, with or without MTS. A total of 31 participants have been dosed across both studies with either a low or a high dose of rezanecel. Importantly, data were presented on 15 participants followed for more than 18 months after single-dose administration, providing the most mature assessment to date of rezanecel durability, safety, and quality-of-life outcomes. Neurona’s upcoming Phase 3 EPIC (EPIlepsy Cell therapy) study design was also presented. “We are delighted to share the expanded dataset from our ongoing trials of rezanecel at the 2026 AAN Annual Meeting,” said Eduardo Dunayevich, M.D., Neurona’s Chief Medical Officer. “Across our unilateral MTLE program, we continue to see durable seizure reduction past the one-year timepoint and a consistent safety profile, in line with our prior reports. In addition, our Phase 1/2 study in bilateral MTLE is now demonstrating exciting preliminary efficacy and safety data. Collectively, these results point to the potential of rezanecel to deliver long-lasting, clinically meaningful benefits across a broad spectrum of patients who are drug-refractory and underserved by current standard-of-care treatments, including lobectomy, brain tissue ablation, or stimulator surgeries. We are eager to begin enrolling participants in our planned Phase 3 EPIC trial later this year.” Neurona also announced that the World Health Organization/International Non-proprietary Name (WHO/INN) has accepted and published the name rezanecel to describe Neurona’s allogeneic GABAergic interneuron cell therapy. Rezanecel will replace the former development code NRTX-1001. Results (N=31, data cut as of March 2026): NTE001 Cohort 1 (Low Dose): Unilateral MTLE with MTS Primary Efficacy Endpoint: Months 7-12 Post-Administration (N=9) 89% median reduction in disabling* seizures78% (7/9) responder rate with ≥50% disabling seizure reduction Long-Term Durability: Months 13+ (N=9) 89% median reduction in disabling seizures89% (8/9) responder rate with ≥50% disabling seizure reduction
NTE001 Cohort 2 (High Dose): Unilateral MTLE with MTS Primary Efficacy Endpoint: Months 7-12 Post-Administration (N=9) 58% median reduction in disabling seizures78% (7/9) responder rate with ≥50% disabling seizure reduction
NTE001 Cohort 3 (Low Dose): Unilateral MTLE without MTS Interim Efficacy Endpoint: Months 4-6 Post-Administration (N=3)** 100% median reduction in disabling seizures67% (2/3) responder rate with ≥50% disabling seizure reduction
NTE002 Study (Low Dose): Bilateral MTLE with or without MTS Interim Efficacy Endpoint: Months 4-6 Post-Administration (N=4)** 64% median reduction in disabling seizures50% (2/4) responder rate with ≥50% disabling seizure reduction
*Disabling seizures exclude focal aware seizures without objective signs (‘auras’)**Cohort has yet to reach 12 months of follow up Safety Across All Cohorts No serious adverse events attributed to rezanecel or the administration procedureMost immunosuppression-related adverse events were mild to moderate in severity and resolved in 14 of 15 participants who completed the regimen per protocol; one participant continues to report mild adverse events after immunosuppression completedSerious adverse events that occurred were consistent with underlying disease or unrelated conditions Cognition No substantial group declines in cognitive performance across word retrieval, verbal memory, or visuospatial memory assessments Podium presentation and poster details:
Podium presentation: “Clinical Update: NRTX-1001 GABAergic Interneuron Cell Therapy for Bilateral Drug-Resistant Focal Epilepsy”Date and time: Wednesday Apr 22 at 1-3pmSession: #S41 - Epilepsy: Public Health and EpidemiologyPresenter: Dr. Peter Warnke, UChicago Medicine. Poster title: “Phase 3 EPIC Clinical Trial Design”Date and time: Wednesday Apr 22 at 1145-1245 Session and poster number: Poster session 11, # 10.006 Neurona Therapeutics’ multicenter, open-label Phase 1/2 clinical program is designed to evaluate the safety, tolerability, and preliminary efficacy of a single administration of rezanecel (NRTX-1001) for unilateral or bilateral drug-resistant MTLE with or without MTS. Participants in both studies are monitored for safety, seizure frequency, disease symptoms, EEG, neuropsychological function, and MRI-based imaging outcomes. Neurona is preparing to initiate its Phase 3 EPIlepsy Cell Therapy (EPIC) trial, a randomized, sham-controlled, double-blind study designed to further evaluate rezanecel in adults with drug-resistant MTLE. The trial is planned to start in 2H 2026. For more information about Neurona’s clinical trials, please contact NeuronaMedInfo@neuronatx.com. 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, rezanecel, 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 rezanecel in June 2024, and the EMA granted the Priority Medicines (PRIME) designation to rezanecel in November 2025. For more information about Neurona, visit: www.neuronatherapeutics.com. Investor Contact:Laurence WattsNew Street Investor Relationslaurence@newstreetir.com
