Neurona Gets FDA RMAT Designation for NRTX-1001 in Focal Epilepsy

Neurona Therapeutics, a clinical-stage biotherapeutics company, has announced that their leading product candidate, NRTX-1001, has received the Regenerative Medicine Advanced Therapy (RMAT) designation from the U.S. Food and Drug Administration (FDA) for drug-resistant mesial temporal lobe epilepsy (MTLE). This designation comes after promising results from an ongoing Phase 1/2 clinical trial, which has shown a favorable safety profile and potential to significantly reduce seizure frequency in patients with drug-resistant epilepsy.

NRTX-1001 is a regenerative cell therapy derived from human pluripotent stem cells. The therapy includes fully differentiated neural cells, known as interneurons, which release the inhibitory neurotransmitter gamma-aminobutyric acid (GABA). These interneurons are intended to integrate into the patient’s neural networks to provide long-term inhibition of hyper-excitable neural circuits. The therapy is delivered as a one-time administration.

In the ongoing Phase 1/2 clinical trial, 60% of the participants who received a single low dose of NRTX-1001 became free from their most severe seizures. Additionally, there were no observed neurocognitive impairments from the low dose, and some patients even showed improvements in neuropsychological test scores.

Dr. Cory R. Nicholas, Co-Founder and CEO of Neurona, expressed satisfaction at the FDA's recognition of NRTX-1001's potential, stating that the RMAT designation validates their positive initial data and their novel regenerative approach to treating focal epilepsy. Dr. Kim J. Burchiel, Head of Functional Neurosurgery at Oregon Health Sciences University and a treating physician in the trial, emphasized that NRTX-1001 could revolutionize the treatment of drug-resistant epilepsy by offering an alternative to the current standard of care, which often involves destructive brain surgery.

The RMAT program, overseen by the FDA’s Center for Biologics Evaluation and Research (CBER), aims to expedite the development and review of regenerative medicine therapies that show potential to address unmet medical needs. The designation allows for accelerated development and discussions about potential surrogate endpoints, with the aim of supporting faster approval processes.

The ongoing multicenter Phase 1/2 trial focuses on evaluating the safety and efficacy of NRTX-1001 for drug-resistant unilateral MTLE. Initially involving 10 subjects, the trial has expanded to include up to 16 participants. Patients are monitored for safety and effects on their epilepsy symptoms after a single administration of NRTX-1001. Recruitment for the study is ongoing at several epilepsy centers across the United States.

Epilepsy affects an estimated three million Americans, with 25 to 35 percent experiencing persistent seizures despite treatment with existing medications. MTLE, a common type of focal epilepsy in adults, often begins in the hippocampus. For patients whose seizures are resistant to drugs, surgical options such as removing or ablating the damaged temporal lobe are available but come with significant risks and are not suitable for all patients.

Neurona’s cell therapy aims to provide a less invasive and potentially more effective treatment for these patients. With ongoing trials and further research, the company hopes to offer a new solution for those with drug-resistant epilepsy. The successful initial results and RMAT designation provide a hopeful outlook for the future of epilepsy treatment.