Neurona Therapeutics announced its Phase 3 clinical trial to evaluate its stem cell therapy, NRTX-1001. NRTX-1001 is comprised of human MGE-type inhibitory GABAergic interneurons derived from pluripotent stem cells, it’s their lead product candidate.
The therapy is intended for adults with drug-resistant mesial temporal lobe epilepsy (MTLE), the most common form of epilepsy in adults, and according to Neurona, this is the first cell therapy being evaluated for the condition.
The randomized, sham-controlled, double-blind trial is expected to begin in the second half of this year.
“People living with drug-resistant seizures often have limited therapeutic options, such as surgery to remove or ablate the seizure-prone region of the brain, which can cause irreversible impairment of memory and other neurocognitive functions,” explained Manher A. Joshi, M.D., Neurona’s Chief Medical Officer. “NRTX-1001 is intended to provide a novel tissue-sparing approach to durably restore balanced activity to the brain from a single treatment, while potentially avoiding the serious neurocognitive risks of current tissue-destructive surgical interventions. We are excited about the potential of NRTX-1001 to transform the therapeutic landscape for drug-resistant focal epilepsy.”
This follows their Regenerative Medicine Advanced Therapy (RMAT) designation, granted to them in June 2024. Depending on how this trial goes, they intend to submit a Biologics License Application (BLA) as a stepping stone to potential FDA approval.
At the American Epilepsy Society’s Annual Meeting in December, the company presented an update to the ongoing open-label Phase 1/2 clinical trial evaluating NRTX-1001 in adults with the condition. They mentioned that the low-dose cohort demonstrated a median reduction of 92% in disabling seizures compared to baseline, with 80% of subjects experiencing greater than 80% seizure reduction during the primary efficacy evaluation period (7-12 months post-treatment). Additionally, the first two patients followed for 24 months post-treatment continued to report seizure reductions greater than 97%.
For the high-dose group, interim data (4-6 months post-treatment) showed a median reduction of 78% in disabling seizures compared to baseline. The company has not yet released the 7-12 month results for this cohort. Neurona also reported that no cognitive impairment was observed in either dosing group, and some participants demonstrated noticeable improvements in neurocognitive test scores compared to baseline.
