Neurona Therapeutics Announces FDA Clearance of Expanded Inclusion Criteria for Phase 1/2 Clinical Trial of Regenerative Cell Therapy in Patients With Dominant Hemisphere Drug-Resistant Focal Epilepsy

Neurona Therapeutics recently announced the US FDA has cleared an amendment to Neurona’s ongoing Phase 1/2 clinical trial of NRTX-1001 in people with drug-resistant mesial temporal lobe epilepsy (MTLE) enabling expansion of enrollment to include adults who have dominant-lobe disease, as well as those whose disease is focused in the non-dominant lobe. In addition, the independent Data and Safety Monitoring Board (DSMB) overseeing the clinical trial completed a prespecified review of data from the first two patients and unanimously recommended progression of the trial to enroll the remaining patients in the first dose cohort.

David Blum, MD, Chief Medical Officer of Neurona, said “When MTLE originates in the verbal-memory dominant hemisphere, typically the left hemisphere in right-handed individuals, there are risks with the current standard of care, lobectomy surgery, because it puts them at risk of memory impairment post-surgery. Individuals with dominant lobe disease have an even greater need for less invasive options that have the potential to be disease-modifying, like NRTX-1001, and we are encouraged that our clinical trial is now open to these individuals.”

“We are delighted with the FDA’s decision, which enables enrollment of patients with focal epilepsy that originates in either the dominant or the non-dominant hemisphere and will effectively double the eligible patient population. In addition, with the DSMB’s clearance to continue the study, we are working with major epilepsy centers across the US to enroll the remaining patients into the first cohort of the NRTX-1001 trial,” added Cory R. Nicholas, PhD, Neurona’s Chief Executive Officer. “We remain cautiously optimistic that the early signs of safety and potential efficacy will continue for our existing and future patients. With additional data from the full cohort, we will determine how to most efficiently advance this novel regenerative cell therapy with the potential to transform the lives of patients for whom anti-seizure medication has failed.”

Two patients have been treated to date in this clinical trial (NCT05135091), both of whom entered the study with a history of significant monthly seizure activity that was not controlled by anti-seizure medications. Following a single treatment with NRTX-1001, which has been well tolerated with no serious or severe adverse events, both patients have thus far experienced impressive, early reduction in seizure activity.

The first patient had a 9-year history of seizures, and in the six months prior to the administration of NRTX-1001 experienced an average of 32 seizures per month, despite being on several antiepileptic medications. The second patient treated in the trial also had drug-resistant seizures, with an average of 14 seizures per month in the six months prior to treatment. Both patients have reported reduced seizure counts by >90% at 6 and 3 months post NRTX-1001 administration, respectively.

NRTX-1001 is an injectable suspension of high-purity inhibitory neurons, called interneurons, derived from human stem cells and designed to replace damaged neurons in the region of the brain where seizures originate. NRTX-1001 is manufactured in Neurona’s in-house GMP facility, using proprietary methods to guide the cells into a specific, pallial-type, lineage of interneurons that secrete the inhibitory neurotransmitter gamma-aminobutyric acid (GABA). NRTX-1001 interneurons are cryopreserved and shipped to clinical sites to be used as an allogeneic, off-the-shelf investigative therapy.

Neurona’s multicenter, Phase 1/2 clinical trial is designed to evaluate the safety and efficacy of a single administration of NRTX-1001 for drug-resistant MTLE. The first stage of the trial is an open-label dose-escalation study in up to 10 people with MTLE, with five patients to be treated at a starting dose and five at a higher dose. Patients treated with a single infusion of NRTX-1001 cells will be monitored for safety, tolerability, and effects on their epilepsy disease symptoms. Patient recruitment is underway at epilepsy centers across the US. For more information, visit www.clinicaltrials.gov (NCT05135091). The first part of the clinical trial is supported by an $8.0 million grant from the California Institute for Regenerative Medicine (CIRM; CLIN2-13355).

NRTX-1001 is a regenerative neural cell therapy candidate derived from human pluripotent stem cells. The fully differentiated neural cells, called interneurons, secrete the inhibitory neurotransmitter gamma-aminobutyric acid (GABA). Delivered as a one-time dose, the human interneurons are intended to integrate and innervate on-target, providing long-term GABAergic inhibition to repair hyper-excitable neural networks.

An estimated three million Americans have epilepsy, and 25% to 35% live with ongoing seizures despite treatment with approved drugs, which means that there is a huge unmet medical need in this community. MTLE is a common type of focal epilepsy in adults and primarily affects the internal structures of the temporal lobe, where seizures often begin in a structure called the hippocampus. For people with seizures resistant to anti-seizure drugs, epilepsy surgery – where the damaged temporal lobe is surgically removed or ablated by laser – can be an option. However, the current surgical options are not available or effective for all patients, are tissue-destructive, and can have significant adverse effects.

Neurona’s regenerative cell therapy candidates have single-dose curative potential. Neurona is developing off-the-shelf, allogeneic neural cell therapy candidates that are designed to provide long-term repair of dysfunctional neural networks for multiple neurological disorders. For more information, visit www.neuronatherapeutics.com