Promising reduction (>95%) in seizure frequency at the key one-year post treatment endpoint in the first patient to receive NRTX-1001, memory improvements, and seizure-freedom since seven-months post-treatment

Encouraging results seen in second patient dosed with NRTX-1001, where the cell therapy has been well-tolerated with continued reduction (>90%) in overall monthly seizure frequency at seven-months post-treatment

Updated clinical and supporting preclinical data described in two oral presentations at the Annual Meeting of the International Society for Stem Cell Research (ISSCR)

SAN FRANCISCO, CA, USA I June 15, 2023 INeurona Therapeutics, a clinical-stage biotherapeutics company advancing regenerative cell therapies for the treatment of neurological disorders, announced the presentation of new data from the open-label, single arm, starting dose-level of its ongoing Phase I/II clinical trial of a one-time dose of NRTX-1001 in people with drug-resistant mesial temporal lobe epilepsy (MTLE). The preliminary data demonstrate promising reduction (>90%) in seizure frequency in the first and second patients at one year and seven-months post-treatment, respectively. In addition, neuropsychological testing suggests an improvement in memory after NRTX-1001 administration. These preliminary clinical observations, as well as supporting preclinical data, are being presented at the Annual Meeting of the International Society for Stem Cell Research (ISSCR) which is being held June 14 – 17 in Boston, MA and virtually.

“Patient-one has reached the one-year post treatment endpoint and has achieved a >95% overall monthly seizure reduction, including elimination of all seizure events since the seventh month post-administration of NRTX-1001,” said David Blum, M.D., Neurona’s chief medical officer. “In addition, this patient has shown improved memory performance on cognitive tests. We continue to enroll patients in the first cohort of the study and look forward to providing additional updates later this year.”

“Both patients entered the clinical trial with significant seizure activity, impaired cognition, and suboptimal quality of life,” said Cory R. Nicholas, Ph.D., Neurona’s chief executive officer who gave one of the presentations at the meeting. “They were candidates for lobectomy or ablation surgery to remove the epileptic seizure-generating temporal lobe, albeit with an associated risk of causing further, irreversible cognitive deficits. Instead, they courageously chose to be first to receive NRTX-1001 cell therapy. Although our clinical investigation is ongoing in additional patients, it is gratifying to witness the first two patients achieving seizure-relief without additional cognitive impairment to date, which supports the therapeutic potential of NRTX-1001. We are hopeful that these improvements will continue in the ongoing trial and possibly provide others living with drug-resistant focal seizures with a non-destructive cell therapy option in the future that does not risk cognitive decline. Relatedly, the NRTX-1001 trial has received clearance from the FDA to expand enrollment to include adults who have MTLE in the verbal memory-dominant lobe and are at greater risk of cognitive decline from a tissue-destructive lobectomy or ablation surgery. We are grateful to the patients, caregivers, and our clinical collaborators for their participation in this first in human clinical study.”

The first two patients to receive NRTX-1001 in the ongoing clinical trial (NCT05135091), both entered the study with a history of significant monthly seizure activity that was not controlled by anti-seizure medications. The first patient, treated at SUNY Upstate Medical University, had a seven-year history of seizures, and in the six months prior to the administration of NRTX-1001 experienced an average of 32 seizures per month. The second patient treated in the trial, at the Oregon Health & Science University, had a nine-year history of seizures and averaged 14 seizures per month in the six months prior to treatment. Both patients continue to report reduced overall seizure counts of >90% at one year- and seven-months post NRTX-1001 administration, respectively. Per protocol, both patients began neurocognitive testing at six-months post treatment, and a subset of their neurocognitive scores has increased from baseline levels, representing a potential improvement in memory.

Neither subject has experienced a serious adverse event, and the data safety monitoring board has reviewed their progress and allowed the study to continue enrollment.

A second oral presentation at the meeting presented by Catherine Priest, Ph.D., Neurona’s chief development officer, detailed the preclinical and translational data supporting the Phase I/II clinical trial of NRTX-1001.

About Neurona’s Clinical Trial of NRTX-1001 for Mesial Temporal Lobe Epilepsy (MTLE)
The clinical study 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 United States. For more information, please visit (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).

About NRTX-1001
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, and designed to durably silence seizure activity in the epileptic region of the brain. NRTX-1001 is manufactured in Neurona’s in-house GMP facility using proprietary methods. NRTX-1001 interneurons are cryopreserved and shipped to clinical sites to be used as an allogeneic, off-the-shelf investigative therapy.

About Mesial Temporal Lobe Epilepsy
According to the Centers for Disease Control and Prevention an estimated 3.4 million Americans have epilepsy, and 25 to 35 percent live with ongoing seizures despite treatment with approved drugs, illustrating 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.

About Neurona
Neurona is focused on developing regenerative cell therapy candidates that 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 about Neurona, visit

SOURCE: Neurona Therapeutics