Neurona Therapeutics has announced plans to advance its groundbreaking cell therapy NRTX-1001 into Phase 3 clinical trials, marking a significant milestone in the treatment of drug-resistant epilepsy. The trial, named EPIC (EPIlepsy Cell therapy), is scheduled to commence in the second half of 2025, following productive discussions with the FDA under the Regenerative Medicine Advanced Therapy (RMAT) designation granted in June 2024.
Promising Phase 1/2 Results Drive Development Forward
The decision to proceed with Phase 3 trials is supported by compelling data from ongoing Phase 1/2 studies. The low-dose cohort (n=5) demonstrated remarkable efficacy, achieving a 92% median reduction in disabling seizures during the 7-12 month evaluation period. Notably, 80% of subjects experienced greater than 80% seizure reduction. Two subjects followed for 24 months maintained over 97% seizure reduction after a single treatment.
The high-dose cohort (n=5) has shown preliminary success with a 78% median reduction in disabling seizures during the interim 4-6-month period. Importantly, the therapy has been well-tolerated at both dose levels, with no adverse events attributed to the cell therapy and no cognitive impairments detected.
Novel Therapeutic Approach for Unmet Medical Need
"People living with drug-resistant seizures face a significant burden due to the substantial risks of ongoing seizures," explains Dr. Manher A. Joshi, Neurona's Chief Medical Officer. "Current options often involve invasive surgery that can cause irreversible impairment of memory and other neurocognitive functions."
NRTX-1001 represents a revolutionary approach as the first investigational human cell therapy for drug-resistant epilepsy. The treatment aims to provide a tissue-sparing solution that could restore balanced brain activity through a single administration, potentially avoiding the cognitive risks associated with traditional surgical interventions.
Phase 3 EPIC Trial Design
The Phase 3 EPIC trial will be conducted as a randomized, sham-controlled, double-blind study, specifically targeting adults with drug-resistant mesial temporal lobe epilepsy (MTLE), the most prevalent form of epilepsy in adults. The trial's results are intended to support a future Biologics License Application.
"The advancement of NRTX-1001 into the Phase 3 EPIC study is a significant milestone for the field of regenerative medicine," stated 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."
