Neurona Therapeutics Presents Updated Data from the NRTX-1001 Cell Therapy Trial in Adults with Drug-resistant Focal Epilepsy at American Academy of Neurology (AAN) 2023 Annual Meeting

– NRTX-1001 administration is well-tolerated with continued seizure reduction of >90% in the first two patients at nine- and five-months post-treatment, respectively

– Impaired-awareness seizures have been eliminated since one month post treatment in the first patient, who has also demonstrated improved memory performance after treatment

San Francisco, CA, April 24, 2023 – Neurona Therapeutics, a clinical-stage biotherapeutics company advancing regenerative cell therapies for the treatment of neurological disorders, announced the presentation of updated clinical data from its ongoing Phase I/II clinical trial of a single dose of NRTX-1001 in people with drug-resistant mesial temporal lobe epilepsy (MTLE). The data demonstrate >90% reduction in seizure frequency in the first and second patient at nine- and five-months post-treatment, respectively. In addition, neuropsychological testing suggests an improvement in memory in the first patient after NRTX-1001 administration. The data are being presented at the American Academy of Neurology (AAN) 2023 Annual Meeting which is being held April 22- 27 in Boston, MA and virtually.

“It is notable that the early significant seizure reduction observed in this study appears to be durable in these first two patients treated with a single administration of NRTX-1001,” said Robert Beach, M.D., Ph.D., chief of epilepsy and professor of neurology, at SUNY Upstate Medical University and a principal investigator in the study whose patient was the first person treated with NRTX-1001. “It is also encouraging that the first patient has been free from disabling seizures from the second month on, and has shown improved memory performance on multiple cognitive tests, as memory problems can be an issue for individuals with drug-resistant MTLE. NTRX-1001, the first non-destructive human cell therapy for epilepsy, may represent the beginning of a new era in the treatment of this debilitating disease.”

“To date NRTX-1001 has been well tolerated with no serious or severe adverse events, and we have received the green-light from the trial safety board to enroll the remaining patients in the first cohort,” said Cory R. Nicholas, Ph.D., Neurona’s chief executive officer. “We have also recently received clearance from the FDA to expand trial enrollment to include adults who have MTLE in the memory-dominant lobe, in addition to those with MTLE in the non-dominant lobe, which doubles the addressable population of patients. Further, we are investigating the potential use of NRTX-1001 in other related indications that could benefit from inhibitory neuronal cell therapy such as bilateral MTLE, neocortical focal epilepsy, and Alzheimer’s disease with hyperexcitability in the temporal lobe.”

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. The first patient, treated at SUNY Upstate Medical University by Dr. Beach and Dr. Harish Babu, had a nine-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 by Dr. David Spencer and Dr. Kim Burchiel, had an eight-year history of seizures and averaged 14 seizures per month in the six months prior to treatment. Both patients have reported reduced seizure counts by >90% at nine- and five-months post NRTX-1001 administration, respectively. In addition, the first patient has not experienced any impaired awareness seizures since one month post-treatment. 

Per protocol, the first patient underwent neurocognitive testing at six- and nine-months post treatment, which showed a measurable improvement in verbal and spatial memory after NRTX-1001 administration. The second patient will also be tested on the same measures soon.

NRTX-1001 is a one-time dose of an injectable suspension of high-purity inhibitory neurons, called interneurons, derived from human stem cells 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 to guide stem cells into specific types 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.

About Neurona’s Clinical Trial of NRTX-1001 for Mesial Temporal Lobe Epilepsy (MTLE)

Neurona’s multicenter, Phase I/II 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 United States. For more information, please 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).

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, providing long-term GABAergic inhibition to repair hyper-excitable neural networks.

About Mesial Temporal Lobe Epilepsy 

An estimated three 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’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 about Neurona, visit www.neuronatherapeutics.com

Investor and Media Contacts:

Sylvia Wheeler
Wheelhouse LSA
swheeler@wheelhouselsa.com

Elizabeth Wolffe, Ph.D.
Wheelhouse LSA
lwolffe@wheelhouselsa.com