–EFFICACY: 92% median reduction in disabling seizures during 7-12-month efficacy endpoint period, and 80% (4/5) of patients responding with >75% seizure reduction in Cohort 1.
–DURABILITY: All responders (4/4) in Cohort 1 continue to have durable seizure control for 18-24 months after a single-dose of NRTX-1001.
–COGNITION AND QUALITY OF LIFE: All patients (5/5) in Cohort 1 had significantly improved quality-of-life test scores, with no patient experiencing persistent decline in cognition.
– SAFETY: NRTX-1001 continues to be well-tolerated at both high and low doses with no adverse events attributed to the cell therapy.
–NEXT STEPS: Phase 3 EPIC trial planned to start enrolling in 2H 2025, as aligned with FDA under RMAT designation, to support submission of a Biologics License Application (BLA).
SOUTH SAN FRANCISCO, Calif., April 08, 2025 (GLOBE NEWSWIRE) -- Neurona Therapeutics, a clinical-stage biotherapeutics company developing regenerative cell therapies for epilepsy and other disorders of the nervous system, today presented updated data from its open-label Phase 1/2 clinical trial of NRTX-1001 in adults with drug-resistant, unilateral mesial temporal lobe epilepsy (MTLE) at the American Academy of Neurology (AAN) 2025 Annual Meeting in San Diego, CA.
The ongoing multicenter Phase 1/2 trial for unilateral MTLE has enrolled 18 subjects in Cohorts 1 and 2 across 13 comprehensive epilepsy centers in the United States, and 10 subjects have been followed for 9 to 24 months after NRTX-1001 administration. Patients in the low-dose Cohort 1 (N=5) achieved median disabling seizure reduction of 92% from baseline during the primary efficacy evaluation period in months 7-12 post-treatment, and a 97% median reduction of disabling seizures after month 13. In the high dose Cohort 2 (N=5), patients achieved median disabling seizure reduction of 72% from baseline in months 4-9, the most recent 6-month epoch, and have not yet reached month 12.
“We are pleased with the results to-date from the ongoing trial of NRTX-1001 cell therapy,” said Dr. Jerry Shih, M.D., Neurologist and Director of the University of California San Diego (UCSD) Comprehensive Epilepsy Center, who has treated 4 subjects in the trial alongside UCSD Neurosurgeon and Surgical Director of Epilepsy, Dr. Sharona Ben-Haim, M.D. “Our first subject in the study is now 19 months post-cell administration and has experienced a 98% reduction in his drug-resistant disabling seizures. He successfully stopped the temporary immunosuppression regimen 6 months ago, and continues to have a durable outcome, free from any study-related adverse effects. If these results continue to be observed in the upcoming Phase 3 EPIC trial, cell therapy has the potential to become a paradigm-shifting treatment option for patients with drug-refractory focal epilepsy.”
Said Dr. John Hixson, M.D., VP of Clinical Development at Neurona, “The results of the trial thus far point to the promise of NRTX-1001 being a cell therapy that can address a major unmet need for patients with drug-resistant focal seizures, providing durable seizure control and avoiding the significant risk of permanent neurocognitive impairment from current tissue-destructive surgeries like laser ablation and brain resection. To that end, NRTX-1001 could represent a new treatment option that preserves brain tissue and function.” Dr. Hixson continued, “We are pleased with the continued durability signal from the ongoing NRTX-1001 study, supporting a single ‘one-and-done’ dose. We are also very encouraged by the lack of cell-related adverse effects and absence of neurocognitive impairment thus far. In summary, the data give us confidence to proceed with our planned Phase 3 EPIC trial of NRTX-1001, as we seek to bring this novel therapy to patients as safely and expeditiously as possible.”
Results:
Low Dose Effect on Disabling Seizures (N=5):
High Dose Effect on Disabling Seizures (N=5):
Safety – Low and High Dose Cohorts (N=10):
Neurocognition – Low and High Dose Cohorts (N=10):
The open-label Phase 1/2 clinical trial was designed to evaluate the safety and preliminary efficacy of a single-dose administration of NRTX-1001 inhibitory neuron cell therapy for drug-resistant unilateral MTLE. This ongoing trial has been expanded to include 8 additional subjects for a total of 18 subjects across both cohorts: low-dose cohort 1 (N=9) and high-dose cohort 2 (N=9). Study subjects are being monitored for safety, tolerability, and effects on their epilepsy disease symptoms. In addition, a second multicenter, open-label Phase 1/2 clinical trial has been initiated to evaluate NRTX-1001 in adults with drug-resistant bilateral MTLE. For more information, please visit www.clinicaltrials.gov (NCT05135091 and NCT06422923).
In February, the company announced plans to initiate its Phase 3 EPIC (EPIlepsy Cell therapy) trial, which is expected to be a single pivotal study and primary basis of a future BLA submission for NRTX-1001 in drug-resistant MTLE, based on alignment gained with FDA under the RMAT designation. 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 adults with drug-resistant MTLE, the most common type of epilepsy in adults.
Earlier this month, Neurona closed an upsized and oversubscribed $102 million private financing with support from new and existing investors.
AAN Presentation Details:
Title: First-in-Human Trial of NRTX-1001 GABAergic Interneuron Cell Therapy for Focal Epilepsy - Updated Clinical Trial Results
Presenting Author: John D. Hixson, M.D., VP of Clinical Development, Neurona
Presentation: Tuesday April 8, 2025; Poster Session 8 (8:00 AM-9:00 AM); Number: 9-003
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. 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 (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. For more information about Neurona, visit: www.neuronatherapeutics.com.
Investor Contact:
Laurence Watts
New Street Investor Relations
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