Neurona Therapeutics, Inc.

SAN FRANCISCO, Sept. 27, 2024 (GLOBE NEWSWIRE) -- Neurona Therapeutics , a clinical-stage biotherapeutics company advancing regenerative cell therapies for disorders of the nervous system, today announced that Cory R. Nicholas, Ph.D., Neurona’s Co-Founder and Chief Executive Officer, will participate in the following investor conferences in October: 2024 William Blair’s Innovator Series: Private Biotechnology Stars Format: Fireside Chat Date/Time: Thursday, October 3, at 6:00 am PT / 8:00 am CT Location: Virtual Leerink Partners Biopharma Private Company Connect Format: 1x1 Investor Meetings Date/Time: Tuesday, October 22 and Wednesday, October 23 Location: Virtual A replay of the fireside chat at the 2024 William Blair’s Innovator Series: Private Biotechnology Stars will be available in the News section of the Neurona website under Events for 7 days following the event. About Neurona Therapeutics Neurona is developing allogeneic, off-the-shelf, regenerative neural cell therapy products with the potential to provide long-term targeted repair of the nervous system following a single administration. The Company’s lead product candidate NRTX-1001, comprising GABAergic interneurons, is currently being studied for safety and efficacy in two ongoing open label multicenter Phase 1/2 trials ( NCT05135091 ) for drug-resistant unilateral mesial temporal lobe epilepsy and ( NCT06422923 ) for drug-resistant bilateral mesial temporal lobe epilepsy, with neocortical focal epilepsy and other indications planned in the future. The Phase 1 of the unilateral MTLE clinical trial is supported by an $8.0 million grant from the California Institute for Regenerative Medicine (CIRM; CLIN2-13355). The FDA granted the Regenerative Medicine Advanced Therapy (RMAT) designation to NRTX-1001 in June 2024 based on positive clinical data from the ongoing Phase 1/2 trial in unilateral MTLE, for which the most recent data update was presented at the American Academy of Neurology Annual Meeting in April 2024. In February 2024, Neurona raised $120 million in a private financing co-led by Viking Global Investors and Cormorant Asset Management. For more information about Neurona, visit: . Investor Contact: Laurence Watts New Street Investor Relations laurence@newstreetir.com

SAN FRANCISCO, Sept. 10, 2024 (GLOBE NEWSWIRE) -- Neurona Therapeutics, a clinical-stage biotherapeutics company advancing regenerative cell therapies for disorders of the nervous system, today announced that the California Institute for Regenerative Medicine (CIRM) has awarded a $3.8M grant to support the development of a next generation neural cell therapy candidate. “We’re grateful for the ongoing partnership with CIRM to help support the development of novel regenerative cell therapy candidates for the prospective treatment of epilepsy and other neurological disorders,” said Cory R. Nicholas, Ph.D., Neurona’s Chief Executive Officer and Co-Founder. “We believe this expanded relationship, recent positive clinical data, and the FDA’s RMAT designation collectively validate our therapeutic approach and highlight the potential for NRTX-1001 to address the large unmet medical need in drug-resistant epilepsy.” “CIRM is delighted to make this award to Neurona Therapeutics in view of the highly encouraging initial data from its ongoing Phase 1/2 clinical trial in adults with drug-resistant epilepsy. Today’s grant brings CIRM’s cumulative commitment to the company to $18.5M across five separate grants,” said Abla Creasey, PhD, Vice President of Therapeutics Development at CIRM. “This award highlights the promise of regenerative medicine technology, including Neurona’s pipeline of cell therapy candidates for patients with epilepsy and other CNS diseases.” In April, Neurona Therapeutics presented updated data at the American Academy of Neurology (AAN) Annual Meeting from its ongoing open-label Phase 1/2 clinical trial of its lead program, NRTX-1001, in adults with drug-resistant, unilateral mesial temporal lobe epilepsy (MTLE). In the first cohort of five subjects who received the lower dose of NRTX-1001, the data showed a median seizure reduction from baseline of 75%, with four of five subjects reporting >50% seizure reduction from baseline, in the six months after treatment. Further, two subjects had been followed for 16- and 21-months after administration of NRTX-1001 and reported durable, >95% seizure reduction from baseline and increased quality-of-life test scores. To date, NRTX-1001 has been well-tolerated in all treated subjects, and neuropsychological testing suggests the absence of neurocognitive impairment. About Neurona Therapeutics Neurona is developing allogeneic, off-the-shelf, regenerative neural cell therapy products with the potential to provide long-term targeted repair of the nervous system following a single administration. The Company’s lead product candidate NRTX-1001, comprising GABAergic interneurons, is currently being studied for safety and efficacy in two ongoing open label multicenter Phase 1/2 trials (NCT05135091) for drug-resistant unilateral mesial temporal lobe epilepsy and (NCT06422923) for drug-resistant bilateral mesial temporal lobe epilepsy, with neocortical focal epilepsy and other indications planned in the future. The Phase 1 of the unilateral MTLE clinical trial is supported by an $8.0 million grant from the California Institute for Regenerative Medicine (CIRM; CLIN2-13355). The FDA granted the Regenerative Medicine Advanced Therapy (RMAT) designation to NRTX-1001 in June 2024 based on positive clinical data from the ongoing Phase 1/2 trial in unilateral MTLE, for which the most recent data update was presented at the American Academy of Neurology Annual Meeting in April 2024. In February 2024, Neurona raised $120 million in a private financing co-led by Viking Global Investors and Cormorant Asset Management. For more information about Neurona, visit: www.neuronatherapeutics.com. Investor Contact:Laurence WattsNew Street Investor Relationslaurence@newstreetir.com

- RMAT designation follows positive data update from the ongoing Phase 1/2 clinical trial of NRTX-1001 cell therapy, demonstrating a favorable safety profile to date and the potential to significantly reduce seizure frequency in drug-resistant epilepsy - - 60% (3 of 5 subjects) who received a single low dose administration of NRTX-1001 have become free from their most disabling seizure type - - The low dose of NRTX-1001 has not resulted in neurocognitive impairment, and some neuropsychological test scores have improved from baseline - - The RMAT provides validation of the preliminary clinical data and the potential for NRTX-1001 to address a key unmet medical need for epilepsy - June 18, 2024 -- Neurona Therapeutics, a clinical-stage biotherapeutics company advancing regenerative cell therapies for disorders of the nervous system, today announced that the U.S Food and Drug Administration (FDA) has granted the company’s lead product candidate, NRTX-1001, the RMAT expedited program designation for drug-resistant mesial temporal lobe epilepsy (MTLE). “We’re pleased the FDA has recognized the potential of NRTX-1001, and we see the RMAT designation as validating both our positive initial data as well as our novel regenerative approach to treating focal epilepsy,” said Cory R. Nicholas, Ph.D., Neurona’s Co-Founder and Chief Executive Officer. “In the near-term, we are focused on completing the ongoing Phase 1/2 study and engaging with the FDA to discuss our product development plan. The Neurona team is committed to bringing our cell therapy to people living with drug-resistant focal epilepsy as safely and expeditiously as possible.” “NRTX-1001 represents a potential paradigm shift in the treatment of drug-refractory epilepsy where the current standard of care involves surgical removal or ablation of the affected brain region,” said Kim J. Burchiel, M.D., Head of Functional Neurosurgery at Oregon Health Sciences University, who treated the second subject in the Phase 1/2 trial. “Unlike current surgical options, which are destructive to brain tissue and can result in serious adverse effects including neurocognitive impairment, NRTX-1001 cell therapy has shown the potential to control seizures without such adverse effects. If these findings are confirmed, NRTX-1001 would satisfy a major unmet medical need for people with drug-resistant epilepsy.” NRTX-1001 is a regenerative 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 administration, the human interneurons are intended to integrate, persist, and provide long-term GABAergic inhibition to repair hyper-excitable neural networks. NRTX-1001 is currently being studied for safety and efficacy in two ongoing open label multicenter Phase 1/2 trials for drug-resistant unilateral mesial temporal lobe epilepsy (unilateral MTLE) and for drug-resistant bilateral mesial temporal lobe epilepsy (bilateral MTLE), with neocortical focal epilepsy and other indications planned in the future. Managed by the FDA’s Center for Biologics, Evaluation and Research (CBER), the RMAT program is dedicated to advancing investigational regenerative medicines, including cell therapies, that are intended to treat, modify, reverse, or cure serious diseases where preliminary clinical evidence indicates that the regenerative medicine therapy has the potential to address unmet medical needs. Investigational therapies that receive RMAT designation are eligible to receive expedited development review and planning guidance from senior CBER managers. RMAT also creates a pathway for early discussions about potential surrogate endpoints and ways to support accelerated approval. The ongoing multicenter Phase 1/2 clinical trial is designed to evaluate the safety and efficacy of a single administration of NRTX-1001 for drug-resistant unilateral MTLE. The first stage of the trial is an open-label dose-escalation study in 10 subjects, recently expanded to up to 16 subjects, with 8 patients to be treated at a starting dose and 8 at a higher dose. Patients receive a single administration of NRTX-1001 and are monitored for safety 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 stage of the clinical trial is supported by the California Institute for Regenerative Medicine (CIRM; CLIN2-13355). An estimated three million Americans have epilepsy, and 25 to 35 percent live with ongoing seizures, despite treatment with approved drugs, illustrating a significant 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 MTLE and seizures that are resistant to 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 is developing allogeneic, off-the-shelf, regenerative neural cell therapy products with curative potential to provide long-term repair of the nervous system after a single administration. The company’s lead product NRTX-1001, comprising GABAergic interneurons, is currently being studied for safety and efficacy in two ongoing open label multicenter Phase 1/2 trials (NCT05135091) for drug-resistant unilateral mesial temporal lobe epilepsy and (NCT06422923) for drug-resistant bilateral mesial temporal lobe epilepsy, with neocortical focal epilepsy and other indications planned in the future. A positive clinical update from the Phase 1/2 trial in unilateral TLE was presented at the American Academy of Neurology 2024 Annual Meeting. In February 2024, Neurona raised $120 million in a private financing co-led by Viking Global Investors and Cormorant Asset Management. For more information about Neurona, visit: www.neuronatherapeutics.com. The content above comes from the network. if any infringement, please contact us to modify.