Cory R. Nicholas, PhD, on Initial Efficacy and Safety Results With NRTX-1001 in Mesial Temporal Lobe Epilepsy


The cofounder and CEO of Neurona Therapeutics discussed results from a phase 1/2 clinical trial presented at AES’s annual meeting.

This is the second part of an interview with Cory R. Nicholas, PhD. For the first part, click here.

“So far in the first 2 patients, where we've looked at cognition (which we start doing at the 6 month mark posttransplant and beyond) we've not seen any additional deficits on cognition. In fact, on some of the cognitive scores, their performance has improved, which points to the potential for this type of regenerative cell therapy modality to possibly be restorative to brain function, rather than destructive as the standard of care lobectomy surgeries [can be.]”

Neurona Therapeutics’ NRTX-1001, an investigational allogeneic regenerative neural cell therapy, is currently being evaluated in a phase 1/2 clinical trial (NCT05135091) for the treatment of drug-resistant mesial temporal lobe epilepsy. Thus far, 5 patients have been treated in the trial, and 2 of these patients have reached at least 1 year of follow-up after receiving the cell therapy, which constitutes a one-time treatment. Results from the first 5 patients, with a data cutoff of October 20, 2023, were recently presented at the Annual Meeting of the American Epilepsy Society (AES), held December 1-5, 2023, in Orlando, Florida.

Following the conference, CGTLive™ sat down with Cory R. Nicholas, PhD, the cofounder and CEO of Neurona Therapeutics and an assistant professor, adjunct, at University of California, San Francisco, to learn more about the trial and these early findings. Nicholas spoke about the design of the trial, noting that the phase 1 portion of the trial will ultimately treat a total of 10 patients with a 2-dose escalation design; the 5 patients treated so far received the study’s lower dose. He added that the phase 2 portion of the trial will take the form of a randomized control trial that will treat 20 additional patients with NRTX-1001 and 10 patients with a placebo.

Nicholas also emphasized some of the promising early safety results: the therapy was well-tolerated and no serious adverse events related to NRTX-1001 were reported. In terms of efficacy, he pointed out that the 2 patients who reached 1 year of follow-up have shown a 96% reduction in the occurrence of seizures from their baseline.

1. Blum D, Babu H, Beach R, et al. First-in-human study of NRTX-1001 GABAergic interneuron cell therapy for treatment of focal epilepsy - emerging clinical trial results. Presented at: AES Annual Meeting; December 1-5; Orlando, Florida.
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