Neurona to Present NRTX-1001 Clinical Update at AAN Annual Meeting

Neurona Therapeutics, a biotherapeutic firm specializing in regenerative cell therapies for neurological conditions, has announced an upcoming presentation of new data from their Phase I/II clinical trial for NRTX-1001. This experimental cell therapy is aimed at treating adults with mesial temporal lobe epilepsy (MTLE) that is unresponsive to medication. The data will be showcased at the American Academy of Neurology's Annual Meeting, scheduled for April 13-18, 2024, in Denver, Colorado.
The presentation, titled "First-in-Human Trial of NRTX-1001 GABAergic Interneuron Cell Therapy for Treatment of Focal Epilepsy - Emerging Clinical Trial Results," is set to be delivered orally by John Hixson, M.D., Neurona's Senior Medical Director, on Monday, April 15, 2024, at 3:42 p.m. Mountain Time.
NRTX-1001 is an innovative neural cell therapy derived from stem cells, which upon full differentiation, become interneurons that release the inhibitory neurotransmitter GABA. The therapy is administered as a single dose, with the goal of these cells integrating into the brain and reducing seizure activity in the affected region. The manufacturing process for NRTX-1001 is carried out using proprietary methods in Neurona's GMP facility.
MTLE is a prevalent form of focal epilepsy in adults, typically initiating seizures in the hippocampus, a part of the temporal lobe. Despite the availability of anti-seizure medications, a significant portion of patients continue to experience seizures, indicating a need for alternative treatments. Current surgical interventions are not always viable or effective and can lead to tissue damage and adverse effects.
The clinical trial for NRTX-1001 is structured to assess the safety and effectiveness of a single dose of the therapy in treating drug-resistant MTLE. It begins with an open-label dose-escalation phase involving up to 10 participants, with the first group receiving an initial dose and the second group a higher dose. Participants will be monitored for safety and changes in their epilepsy symptoms following the infusion of NRTX-1001 cells. Recruitment for the trial is ongoing across various epilepsy centers in the United States, with more details available at www.clinicaltrials.gov (NCT05135091). The initial phase of the trial is funded by an $8.0 million grant from the California Institute for Regenerative Medicine (CIRM).
Neurona Therapeutics is dedicated to developing cell therapies with the potential for a single-dose cure, targeting long-term repair of impaired neural networks across a range of neurological disorders. 

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