IND Cleared for Focal Epilepsy Cell Therapy Phase 1/2 Trial


The cell therapy is set to be evaluated in a 2-part, phase 1/2 trial.

This content originally appeared on our sister site, NeurologyLive.

The inhibitory nerve cell therapy NRTX-1001( Neurona Therapeutics) has received investigational new drug (IND) clearance by the FDA and will be evaluated in a first-in-human phase 1/2 clinical trial in patients with drug-resistant mesial temporal lobe epilepsy (MTLE).

NRTX-1001 will be assessed for safety, tolerability, and efficacy in a multicenter trial. The first stage of the trial is an open-label dose-escalation study in up to 10 participants with MTLE and the second stage is a randomized, blinded investigation comparing the cell therapy to a control cohort of 30 participants with MTLE.

NRTX-1001 is designed to secrete the inhibitory neurotransmitter gamma-aminobutyric acid (GABA), providing long-term GABAergic inhibition to repair hyperexcitable neural networks that underlie epilepsy and other central nervous system disorders with a single dose. For now, Neurona indicated that it is intended on focusing NRTX-1001 as a restorative treatment for MTLE.

"The clearance of our first IND is a key milestone for Neurona and a testament to the talent, experience, and hard work of the entire Neurona team," Cory Nicholas, PhD, president and chief executive officer, Neurona, said in a statement. "This milestone is especially rewarding and timely given that November is Epilepsy Awareness Month. Epilepsy is one of the most common neurological disorders, affecting over 3 million people in the US, of whom approximately one-third have drug-resistant disease. NRTX-1001 is a new type of inhibitory cell therapy that is targeted to the focal seizure onset region in the brain and, in a single treatment, has the potential to significantly improve the lives of people living with focal epilepsy.”1

READ MORE: Severe Neurodegenerative, Epileptic Disorders the Target of Taysha Gene Therapies

The only data on NRTX-1001 has come from preclinical models, in which treatment with the cell therapy resulted in seizure-freedom for more than two-thirds of the population compared to 5% of the control group. Additionally, investigators observed signs of reduced mesiotemporal sclerosis, or tissue damage in the affected seizure-onset area of the brain, with treatment from NRTX-1001.

"To our knowledge, NRTX-1001 is the first human cell therapy candidate to enter clinical trials for epilepsy," David Blum, MD, head, Clinical Development, Neurona Therapeutics, said in a statement. "Unlike many other interventions for drug-resistant focal epilepsy, including surgical removal or ablation of brain tissue, NRTX-1001 has the potential to achieve seizure freedom in a non-tissue destructive manner.”1

Most recently, the company presented preclinical data at Neuroscience 2021, the 50th Annual Meeting of the Society for Neuroscience, November 8-11. In an intrahippocampal kainite epilepsy mode, investigators were able to establish a baseline progression of hippocampal damage that is important to the characterization of NRTX-1001. Furthermore, in the same model, treatment with NRTX-1001 was found to be well-tolerated and maintained efficient interneuron cell engraftment and disease-modifying activity when combined with antiseizure medications levetiracetam (Keppra; UCB Pharma) and diazepam (Libervant; Aquestive Therapeutics).2

The final abstract showed that NRTX-1001 transplantation into the hippocampus stably suppressed focal seizures, and significantly reduced hippocampal pathology in the mouse model of MTLE over a 7-month period. No unwanted behavioral effects were observed as a result of treatment. Additionally, the interneuron cells from NRTX-1001 persisted and distributed in the hippocampus throughout the course of the study and were not proliferative.

1. Neurona Therapeutics receives IND clearance to initiate phase 1/2 clinical trial of neural cell therapy NRTX-1001 in chronic focal epilepsy patients. News release. Neurona Therapeutics. November 4, 2021. Accessed November 8, 2021.
2. Neurona Therapeutics presents preclinical data from lead cell therapy candidate NRTX-1001 at Neuroscience 2021. News release. Neurona Therapeutics. November 8, 2021. Accessed November 8, 2021.
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