Neurona Therapeutics to Present Preclinical Data Supporting Regenerative Cell Therapy, NRTX-1001, in Clinical Development for Chronic Focal Epilepsy, at the ISSCR 2022 Annual Meeting

– Oral presentation will outline design of first ever Phase 1/2 clinical trial of regenerative human cell therapy in epilepsy

– Presentations will detail single-cell transcriptomic profiling of NRTX-1001 product composition and fate post-administration into preclinical models

San Francisco, CA, June 17, 2022 – Neurona Therapeutics, a clinical-stage biotherapeutics company advancing regenerative cell therapies for the treatment of neurological disorders, today announced the presentation of investigational new drug (IND)-enabling data that support the company’s lead inhibitory neuronal cell therapy candidate, NRTX-1001, which has entered a first-in-human clinical trial for the treatment of drug-resistant focal epilepsy. The data are being presented in oral and poster presentations at the annual meeting of the International Society for Stem Cell Research (ISSCR) held June 15-18, 2022 in San Francisco, CA.

“Supported by compelling preclinical data, Neurona has opened a clinical trial to evaluate NRTX-1001 in adults with mesial temporal lobe epilepsy (MTLE),” said Cory R. Nicholas, Ph.D., Neurona’s president and chief executive officer. “Currently, people living with drug-resistant MTLE have few options, including surgery to remove or ablate the affected temporal lobe. However, these tissue-destructive surgeries can have serious adverse effects and not all patients are eligible. NRTX-1001 has the potential to durably restore neural inhibition to seizure-onset foci following a single administration of cells.”

Data presented by Marina Bershteyn, Ph.D., who co-directs Neurona’s discovery research department, describe the characterization of NRTX-1001, a cell therapeutic produced from human pluripotent stem cells using Neurona’s proprietary process in the company’s in-house manufacturing facility. NRTX-1001 cells were characterized in functional and single-cell RNAseq assays prior to administration, which confirmed high on-target purity of hippocampal/cortical-type interneurons, so-called GABAergic pallial-type interneurons, that match the specific inhibitory cell lineage affected in the temporal lobe. Post-administration into preclinical models, RNAseq analysis of single human NRTX-1001 nuclei demonstrated appropriate maturation of the cells into hippocampal/cortical-type interneuron sub-lineages. 

Separately, in an oral session, Dr. Nicholas will present data demonstrating that the NRTX-1001 interneurons stably persisted in a preclinical MTLE model, reproducibly eliminated focal seizures in the majority of the treatment group, reduced temporal lobe tissue damage, and improved survival rates. A dose-finding study in the MTLE model identified a broad maximum effective dosing range and no detectable dose-limiting toxicities. Additionally, the MRI-guided clinical delivery system displayed consistent and safe deposition of the NRTX-1001 interneurons in a second preclinical model. 

Supported by these promising data, Neurona is initiating a first ever clinical trial of human cell therapy for epilepsy. The trial is a multicenter, open-label, dose-escalation study followed by a randomized controlled evaluation of NRTX-1001 safety and efficacy in people with drug-resistant MTLE.

Details of the presentations are as follows:

Title: RAPID DETECTION OF PALLIAL GABAERGIC INTERNEURON SUBTYPES BY SINGLE NUCLEI RNA SEQUENCING AFTER PRECLINICAL TRANSPLANTATION OF NRTX-1001, A CELLULAR THERAPEUTIC IN CLINICAL DEVELOPMENT FOR EPILEPSY
Authors: Marina Bershteyn, Robin Zhou, Luis Fuentealba, Geetha Subramanyam, Meliz Sezan, Daniel Cherkowsky, Adrian Bates, Steven Havlicek, Yves Maury, Alessandro Bulfone, Gautam Banik, Catherine Priest, Cory R. Nicholas
Poster #: 124
Date: Wednesday June 15, 2022. 7:30 - 8:30 PM PT

Title: HUMAN INHIBITORY NEURON CELL THERAPY ENTERS PHASE I/II CLINICAL INVESTIGATION FOR CHRONIC FOCAL EPILEPSY
Authors: Catherine Priest, Gautam Banik, David Blum, Alessandro Bulfone, Mansi Parekh, Philip Hampel, Hannah Kim, Andrew Adler, Luis Fuentealba, Michael Watson, Seonok Lee, Sonja Kriks, Steven Havlicek, Robin Zhou, Yves Maury, Marina Bershteyn, Cory R. Nicholas
Session: Stem Cells – From Development to Therapy
Date: Friday June 17, 2022. 1:55 – 2:05 PM PT

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

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. Patient recruitment is underway at epilepsy centers across the United States. For more information, please visit www.clinicaltrials.gov (NCT05135091). The first stage of the clinical trial is supported by a recently announced $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 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 hyperexcitable neural networks.

About Mesial Temporal Lobe Epilepsy (MTLE)

MTLE primarily affects the internal structures of the temporal lobe, where seizures often begin in a structure called the hippocampus. MTLE is the most common type of focal epilepsy in adults. For people 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, are tissue-destructive, and can have significant adverse effects.

About Neurona

Neurona’s regenerative cell therapies have single-dose curative potential. Neurona is developing off-the-shelf, allogeneic neuronal, glial, and gene-edited cell therapy candidates 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