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Neurona Therapeutics to Present Preclinical Data Supporting Regenerative Cell Therapy, NRTX-1001, in Clinical Development for Chronic Focal Epilepsy, at the ISSCR 2022 Annual MeetingOral 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, June 17, 2022 (GLOBE NEWSWIRE) -- 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 eliminaed 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 Title: HUMAN INHIBITORY NEURON CELL THERAPY ENTERS PHASE I/II CLINICAL INVESTIGATION FOR CHRONIC FOCAL EPILEPSY About Neurona’s Clinical Trial of NRTX-1001 for Mesial Temporal Lobe Epilepsy (MTLE) About NRTX-1001 About Mesial Temporal Lobe Epilepsy (MTLE) About Neurona Investor and Media Contacts:
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