NRTX-1001

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

SAN FRANCISCO, May 28, 2024 (GLOBE NEWSWIRE) -- Neurona Therapeutics, a clinical-stage biotherapeutics company focused on advancing regenerative cell therapies for the potential treatment of neurological disorders, today announced that Cory R. Nicholas, Ph.D., Neurona’s Co-Founder and Chief Executive Officer, is scheduled to host investor meetings at the Jefferies Global Healthcare Conference being held at the Marriott Marquis in New York, NY on June 4-6, 2024. About Neurona Therapeutics Neurona is developing allogeneic, off-the-shelf, regenerative neural cell therapy products with curative potential, that are designed to provide long-term repair of the nervous system after a single administration. The company’s lead asset 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 temporal lobe epilepsy (UTLE) and bilateral temporal lobe epilepsy (BTLE), with neocortical focal epilepsy (NFE) and other indications planned in the future. A positive clinical update from the Phase 1/2 trial in UTLE was presented at the AAN 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 Media Contacts:Elizabeth Wolffe, Ph.D.Wheelhouse LSAlwolffe@wheelhouselsa.com Investor Contact:Laurence WattsNew Street Investor Relationslaurence@newstreetir.com