Neurona Therapeutics, a clinical-stage biotherapeutics company, is initiating its Phase 3 EPIC clinical trial for NRTX-1001, a regenerative cell therapy for drug-resistant mesial temporal lobe epilepsy (MTLE) in adults. This marks the first investigational human cell therapy to reach Phase 3 for this indication and is based on the regulatory pathway established through the Regenerative Medicine Advanced Therapy (RMAT) designation. The Phase 3 EPIC trial, planned for the second half of 2025, will be a randomized, sham-controlled, double-blind study.
This trial represents a significant advancement in the treatment of drug-resistant epilepsy. Current options for drug-resistant MTLE, such as surgical removal or ablation of the affected brain region, carry significant risks of irreversible neurocognitive impairment. NRTX-1001 offers a potential alternative that aims to restore balanced brain activity with a single administration, possibly avoiding the invasive and potentially damaging effects of surgery. The success of this therapy could reshape the treatment landscape for this prevalent form of epilepsy, providing a less invasive and potentially more effective solution for patients.
The Phase 3 trial builds upon promising data from Neurona’s ongoing Phase 1/2 trials. Preliminary results from the open-label Phase 1/2 study showed substantial median seizure reduction and no reported cognitive impairments. Some patients even experienced notable improvements in neurocognitive test scores. The Phase 3 trial, with its rigorous design, will provide more robust data on the efficacy and safety of NRTX-1001, ultimately aiming to support a future Biologics License Application.
The commencement of the Phase 3 EPIC trial marks a critical step towards potentially delivering a novel therapeutic option to patients with drug-resistant MTLE. Positive results from this trial could lead to the first disease-modifying therapy for this condition, offering new hope for patients and potentially revolutionizing the field of epilepsy treatment. The outcome of the trial is expected to have a substantial impact on Neurona’s future, shaping the company’s position as a leader in regenerative cell therapies for neurological disorders.
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.
