Neurona Therapeutics Receives FDA’s Regenerative Medicine Advanced Therapy Designation for NRTX-1001 in Focal Epilepsy

Neurona Therapeutics recently announced the US FDA has granted the company’s lead product candidate, NRTX-1001, the RMAT expedited program designation for drug-resistant mesial temporal lobe epilepsy (MTLE).

“We’re pleased the FDA has recognized the potential of NRTX-1001, and we see the RMAT designation as validating both our positive initial data as well as our novel regenerative approach to treating focal epilepsy,” said Cory R. Nicholas, PhD, Neurona’s Co-Founder and Chief Executive Officer. “In the near-term, we are focused on completing the ongoing Phase 1/2 study and engaging with the FDA to discuss our product development plan. The Neurona team is committed to bringing our cell therapy to people living with drug-resistant focal epilepsy as safely and expeditiously as possible.”

“NRTX-1001 represents a potential paradigm shift in the treatment of drug-refractory epilepsy where the current standard of care involves surgical removal or ablation of the affected brain region,” added Kim J. Burchiel, MD, Head of Functional Neurosurgery at Oregon Health Sciences University, who treated the second subject in the Phase 1/2 trial. “Unlike current surgical options, which are destructive to brain tissue and can result in serious adverse effects including neurocognitive impairment, NRTX-1001 cell therapy has shown the potential to control seizures without such adverse effects. If these findings are confirmed, NRTX-1001 would satisfy a major unmet medical need for people with drug-resistant epilepsy.”

NRTX-1001 is a regenerative cell therapy candidate derived from human pluripotent stem cells. The fully differentiated neural cells, called interneurons, secrete the inhibitory neurotransmitter gamma-aminobutyric acid (GABA). Delivered as a one-time administration, the human interneurons are intended to integrate, persist, and provide long-term GABAergic inhibition to repair hyper-excitable neural networks. NRTX-1001 is currently being studied for safety and efficacy in two ongoing open label multicenter Phase 1/2 trials for drug-resistant unilateral mesial temporal lobe epilepsy (unilateral MTLE) and for drug-resistant bilateral mesial temporal lobe epilepsy (bilateral MTLE), with neocortical focal epilepsy and other indications planned in the future.

Managed by the FDA’s Center for Biologics, Evaluation and Research (CBER), the RMAT program is dedicated to advancing investigational regenerative medicines, including cell therapies, that are intended to treat, modify, reverse, or cure serious diseases where preliminary clinical evidence indicates that the regenerative medicine therapy has the potential to address unmet medical needs. Investigational therapies that receive RMAT designation are eligible to receive expedited development review and planning guidance from senior CBER managers. RMAT also creates a pathway for early discussions about potential surrogate endpoints and ways to support accelerated approval.

The ongoing multicenter Phase 1/2 clinical trial is designed to evaluate the safety and efficacy of a single administration of NRTX-1001 for drug-resistant unilateral MTLE. The first stage of the trial is an open-label dose-escalation study in 10 subjects, recently expanded to up to 16 subjects, with 8 patients to be treated at a starting dose and 8 at a higher dose. Patients receive a single administration of NRTX-1001 and are monitored for safety and effects on their epilepsy disease symptoms. Patient recruitment is underway at epilepsy centers across the United States. For more information, please visit www.clinicaltrials.gov (NCT05135091). The first stage of the clinical trial is supported by the California Institute for Regenerative Medicine (CIRM; CLIN2-13355).

An estimated 3 million Americans have epilepsy, and 25% to 35% live with ongoing seizures, despite treatment with approved drugs, illustrating a significant unmet medical need in this community. MTLE is a common type of focal epilepsy in adults and primarily affects the internal structures of the temporal lobe, where seizures often begin in a structure called the hippocampus. For people with MTLE and seizures that are resistant to drugs, epilepsy surgery – where the damaged temporal lobe is surgically removed or ablated by laser – can be an option. However, the current surgical options are not available or effective for all patients, are tissue-destructive, and can have significant adverse effects.

Neurona is developing allogeneic, off-the-shelf, regenerative neural cell therapy products with curative potential to provide long-term repair of the nervous system after a single administration. The company’s lead product 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 and (NCT06422923) for drug-resistant bilateral mesial temporal lobe epilepsy, with neocortical focal epilepsy and other indications planned in the future. A positive clinical update from the Phase 1/2 trial in unilateral TLE was presented at the American Academy of Neurology 2024 Annual Meeting. In February 2024, Neurona raised $120 million in a private financing co-led by Viking Global Investors and Cormorant Asset Management. For more information, visit www.neuronatherapeutics.com.