Pharma Focus Europe

Neurona Therapeutics Obtains FDA RMAT Designation for NRTX-1001 in Treating Focal Epilepsy

Wednesday, June 19, 2024

Neurona Therapeutics, a company in the clinical stage focusing on regenerative cell therapies for disorders of the nervous system, has received RMAT designation from the FDA for its main product candidate, NRTX-1001, which aims to treat drug-resistant mesial temporal lobe epilepsy (MTLE).

Neurona, expressed his enthusiasm about the FDA's recognition of NRTX-1001’s potential, noting the company's novel approach to treating focal epilepsy. He mentioned that Neurona is focused on completing the ongoing Phase 1/2 study and working with the FDA to further develop their product. “We are committed to safely and rapidly providing our cell therapy to those suffering from drug-resistant focal epilepsy,” Nicholas said.

Dr. Kim J. Burchiel, who leads functional neurosurgery at Oregon Health Sciences University, highlighted the potential of NRTX-1001 to change the treatment landscape for drug-resistant epilepsy. Unlike existing surgical treatments, which can cause significant harm, this cell therapy might offer seizure control without damaging brain tissue. “NRTX-1001 could meet a critical need for patients who do not respond to current treatments,” he observed.

NRTX-1001 employs regenerative cell therapy derived from human pluripotent stem cells. These cells, known as interneurons, produce the inhibitory neurotransmitter gamma-aminobutyric acid (GABA). This therapy, administered as a single treatment, is intended to integrate, persist, and provide long-term GABAergic inhibition, potentially repairing overactive neural networks. Ongoing studies are assessing its safety and effectiveness in treating drug-resistant unilateral and bilateral mesial temporal lobe epilepsy, with additional studies planned for other types of epilepsy.

The RMAT program, managed by the FDA’s Center for Biologics, Evaluation, and Research (CBER), is designed to speed up the development of regenerative medicines and cell therapies for serious diseases. Therapies granted RMAT designation can receive expedited review processes and consult with CBER staff about potential surrogate endpoints and expedited approval pathways.

Neurona’s clinical trial for NRTX-1001 in unilateral mesial temporal lobe epilepsy:
The current Phase 1/2 trial evaluates the safety and effectiveness of a single dose of NRTX-1001 for patients with drug-resistant unilateral MTLE. Originally designed as an open-label dose-escalation study, it now involves up to 16 participants. The trial tracks patient safety and seizure management. Enrollment is ongoing at various epilepsy centers across the United States. Additional information can be found at (NCT05135091), and the trial is supported by the California Institute for Regenerative Medicine (CIRM; CLIN2-13355).

Epilepsy affects about three million people in the U.S., with 25 to 35 percent continuing to experience seizures despite medication, highlighting a significant unmet medical need. MTLE, a common form of focal epilepsy in adults, typically affects the internal structures of the temporal lobe, where seizures often originate. For those with drug-resistant MTLE, surgical treatments, which can be invasive and result in severe side effects, are sometimes considered.



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