Neurona Therapeutics has dosed the first patient in a Phase 1/2 clinical trial of NRTX-1001, a regenerative cell therapy, for drug-resistant bilateral mesial temporal lobe epilepsy (MTLE). This trial follows positive preliminary data from a similar study in unilateral MTLE and is funded by a $14 million grant from the California Institute for Regenerative Medicine (CIRM). Data from both the bilateral and unilateral MTLE trials will support a future Biologics License Application (BLA) for NRTX-1001.
This advancement is crucial for patients with bilateral MTLE, a debilitating condition for which current treatment options are limited. Surgical intervention carries a high risk of irreversible memory loss, making it unsuitable for these patients. Neurostimulator devices offer another avenue, but their efficacy has proven limited in clinical trials. NRTX-1001 represents a potentially game-changing treatment paradigm by directly addressing the underlying biological mechanisms of MTLE while preserving neurocognitive function. This could significantly improve the quality of life for this underserved patient population.
The bilateral MTLE trial is a multicenter, open-label study enrolling ten adult subjects. Participants will receive a single, image-guided intracerebral administration of NRTX-1001 to both temporal lobes. The study will assess safety, tolerability, and preliminary efficacy over two years. In parallel, Neurona has fully enrolled an eight-subject expansion cohort in its unilateral MTLE trial and anticipates starting its Phase 3 EPIC trial in the latter half of 2025. Prior unilateral MTLE trial data revealed a 92% median reduction in disabling seizures and an 80% responder rate with over 75% seizure reduction at the 12-month endpoint. Notably, NRTX-1001 has been well-tolerated across trials with no reported drug-related adverse events.
The initiation of the bilateral MTLE trial marks a significant step forward in Neurona’s clinical development program. The study’s findings, combined with upcoming data from the unilateral MTLE and Phase 3 trials, will be pivotal in determining the therapy’s potential to address unmet needs within the broader epilepsy community and possibly expand its application to other neurological disorders. This development also represents a crucial step in exploring the safety and efficacy of bilateral interneuron cell delivery, potentially opening doors for future cell therapy applications in conditions like Alzheimer’s disease.
Jon Napitupulu is Director of Media Relations at The Clinical Trial Vanguard. Jon, a computer data scientist, focuses on the latest clinical trial industry news and trends.
