Focal Epilepsy Cell Therapy NRTX-1001 Shows Efficacy Signals in First Patient Cohort


Neurona Therapeutics also reported that this first patient cohort has completed enrollment in addition to the early efficacy data from the first 2 patients treated.

The first of 2 patients to be treated in Neurona Therapeutics’ phase 1/2 clinical trial (NCT05135091) evaluating NRTX-1001, who is now off the immunosuppression regimen, has maintained a greater than 95% seizure reduction as of 16 months posttreatment.1,2

An allogeneic regenerative neural cell therapy, NRTX-1001 is being assessed to treat drug-resistant mesial temporal lobe epilepsy (MTLE). In addition to these updated early efficacy data from the first 2 patients treated in the trial, the enrollment of patients in this first cohort has completed, according to the company announcement.1

It was noted that at baseline, the first patient, a 26-year-old man, had experienced seizures over the course of the past 7 years and that in the 6 months before receiving the cell therapy, averaged 32 seizures each month.1,2 The first cohort is treating patients with a single, lower dose of NRTX-1001 and a year-long regimen of immunosuppression therapy intended to facilitate integration and persistence of the cell therapy. The cohort enrolled 5 patients in total, and all 5 have received the administration of NRTX-1001.

The second patient to be treated, who has been followed up for 12 months after receiving NRTX-1001, also demonstrated a greater than 95% seizure reduction.1 Furthermore, the company noted that this patient has not experienced any seizures in 6 months. It was noted that at baseline this patient, a 59-year-old woman, had experienced seizures over the course of the past 9 years and that in the 6 months before receiving the cell therapy, averaged 14 seizures each month.2

Neurona added that for these 2 patients, on-protocol neurocognitive testing conducted at baseline and after treatment with NRTX-1001 indicated potential improvements in memory via increased scores on a subset of items measured by the tests.1 In terms of safety, there were no serious or severe adverse events reported in any of the 5 treated patients so far.

“The early data from our ongoing open-label phase 1/2 clinical trial have been promising to date, demonstrating that NRTX-1001 has been well tolerated thus far and may result in substantial, durable seizure reduction in subjects with drug-resistant mesial temporal lobe epilepsy,” Cory R. Nicholas, PhD, Neurona’s chief executive officer, said in a statement.1 “To see the data continue to hold during long-term 12-plus months of follow-up with the first two subjects is extremely rewarding. We look forward to enrolling our second cohort of five subjects and advancing the development of this potentially transformative therapy for people living with drug-resistant epilepsy.”

NRTX-1001 consists of human interneurons that provide long-term secretion of gamma-aminobutyric acid, an inhibitory neurotransmitter, which is expected to repair neural networks. Participants in the trial are receiving NRTX-1001 via a single stereotactic intracerebral administration. Evidence of neural cell viability and local inflammation will be assessed using MRI scans for 2 years following the transplant.

“The first subject’s transition off immunosuppression therapy with continued >95% reduction in overall monthly seizure counts represents a very encouraging development for the potential durability of this treatment,” David Blum, MD, Neurona’s chief medical officer, added.1 “The second subject treated with a single dose of NRTX-1001 has reached the 1-year endpoint, also continues to do well with >95% seizure reduction and has not reported a seizure for the past 6 months. We anticipate updating on the progress of these 2 patients and providing data from the additional patients in this first cohort in the fourth quarter of 2023.”

NRTX-1001 is not the only advanced therapeutic in development for MTLE. In September 2023, uniQure NV’s AMT-260, an investigational adeno-associated virus (AAV) vector-based miRNA gene therapy intended to treat refractory MTLE received clearance of its investigational new drug application from the FDA.3 AMT-260 consists of 2 engineered miRNAs, delivered locally in an AAV9 vector, that are intended to degrade GRIK2, a gene involved in production of glutamate receptor subtype GLUK2. The company is planning to initiate screening activities for a phase 1/2a clinical trial evaluating AMT-260 before the end of the year.

1. Neurona Therapeutics completes clinical enrollment of first cohort and updates on long-lasting impact in first patients treated in ongoing phase I/ii trial of NRTX-1001 cell therapy for drug-resistant focal epilepsy. News release. Neurona Therapeutics. October 19, 2023. Accessed October 23, 2023.
2. Nicholas C. A phase I/II clinical trial of NRTX-1001 HPSC-derived inhibitory interneuron cell therapy for chronic focal epilepsy. Presented at: ISSCR 2023 Annual Meeting.
3. uniQure announces FDA clearance of investigational new drug application for AMT-260 gene therapy for refractory mesial temporal lobe epilepsy. News release. uniQure N.V. September 5, 2023. Accessed September 7, 2023.
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