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NRTX-1001 Reduces Mesial Temporal Lobe Epilepsy Seizures in 2 Patients

The first participant has been followed-up for 3 months so far after treatment while the second is only 1 week post-treatment.

The neural cell therapy NRTX-1001 was well-tolerated and reduced seizures in the first 2 patients with drug-resistant unilateral mesial temporal lobe epilepsy (MTLE) treated in a first-in-human phase 1/2 study (NCT05135091).1

“The early clinical results with NRTX-1001 in epilepsy are very encouraging, and we look forward to enrolling additional patients in the study,” Cory R. Nicholas, PhD, president and chief executive officer, Neurona Therapeutics, said in a statement.1 “NRTX-1001 is designed to be an off-the-shelf, one-time administration therapy with the potential to durably eliminate seizures and provide a new regenerative cell therapeutic approach in patients for whom anti-seizure medication has failed. It has the potential to be disease-modifying without the tissue-destructive procedural risks associated with lobectomy. Further, there are many who are not currently eligible for lobectomy surgery who may be eligible for NRTX-1001 in the future. We are sincerely grateful to everyone involved in the development of NRTX-1001, including the first participants in this pioneering study, their families, and the respective clinical site teams.”

The patients had up to a 9-year history of seizures and, in the 6 months prior to NRTX-1001 treatment, had averages of 32 and 14 seizures a month while on antiepileptic medications. Both patients received the single dose of NRTX-1001 without any treatment-related serious adverse events (AEs). One patient has had 4 seizures in the first 3 months after treatment and the other has not had any seizures as of the first week after treatment. These data were presented at the ISSCR Boston International Symposium: Translating Pluripotent Stem Cell Discoveries to the Clinic: Preclinical, Manufacturing, and Regulatory Strategies for Success on November 19, 2022, by Nicholas.

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NRTX-1001 is designed to repair neural networks by delivering human interneurons that provide long-term secretion of the gamma-aminobutyric acid (GABA) inhibitory neurotransmitter. Participants in the study receive a single stereotactic intracerebral administration of the interneuron cells and remain on a immunosuppressive regimen for 1 year. Evidence of neural cell viability and local inflammation will be assessed using MRI scans for 2 years following the transplant.The single-arm, dose-escalation first stage of the study will enroll up to 10 participants with MTLE and the second will enroll up to 20 to be treated and 10 for a sham comparator arm.

The study is primarily assessing the incidence and severity of AEs over 1 year after NRTX-1001 administration compared to sham treatment. Secondary end points include the change in seizure frequency and the difference in the 75% responder rate between participants who received NRTX-1001 in stage 2 and those who received the sham treatment. The first patient was dosed in the trial in July 2022.2

“Neurona’s regenerative cell therapy approach has the potential to provide a single-administration, non-destructive alternative for the treatment of drug-resistant focal epilepsy,” Harish Babu, MD, PhD, assistant professor of neurosurgery, SUNY Upstate Medical University and the surgeon who administered the first dose of NRTX-1001, said in an earlier statement.2 “Currently, people with mesial temporal lobe epilepsy who are not responsive to anti-seizure medications have few options, such as an invasive surgery that removes or destroys the affected brain tissue.”

1. Neurona Therapeutics presents encouraging clinical data from first-ever regenerative human cell therapy trial in adults with drug-resistant focal epilepsy and shows supporting manufacturing, delivery, and preclinical data. News release. Neurona Therapeutics. November 21, 2022. https://finance.yahoo.com/news/neurona-therapeutics-presents-encouraging-clinical-130000565.html
2. Neurona Therapeutics announces initial subject dosed in first clinical trial of regenerative human cell therapy, NRTX-1001, in adults with drug-resistant focal epilepsy. News release. Neurona Therapeutics. June 29, 2022. https://www.neuronatherapeutics.com/news/press-releases/062922/