Neurona Doses First Patient in Phase 1/2 Trial of NRTX-1001 Cell Therapy in Adults with Drug-Resistant, Bilateral Mesial Temporal Lobe Epilepsy (MTLE)

First patient received bilateral administration of NRTX-1001; no significant surgical complications or adverse events reported to date

Trial funded by a $14 million grant from California Institute for Regenerative Medicine

Data from this bilateral trial, together with results from the previously announced Phase 3 EPIC study in unilateral MTLE, are intended to support a future BLA submission for NRTX-1001 in drug-resistant MTLE –

Full enrollment of an eight-subject expansion cohort in unilateral MTLE trial also announced

Excerpt from the Press Release:

SAN FRANCISCO, June 18, 2025 (GLOBE NEWSWIRE) — Neurona Therapeutics, a clinical-stage biotherapeutics company advancing regenerative cell therapies for disorders of the nervous system, today announced dosing of the first patient in its Phase 1/2 clinical trial of NRTX-1001 cell therapy in adults with drug-resistant bilateral mesial temporal lobe epilepsy (MTLE). MTLE is the most common type of epilepsy in adults and is characterized by seizures originating primarily in the hippocampus from either one hemisphere of the brain (unilateral) or both hemispheres (bilateral).

Building upon preliminary efficacy and safety data from its ongoing Phase 1/2 trial in unilateral MTLE, most recently presented at the 2025 American Academy of Neurology (AAN) Annual Meeting, Neurona has initiated a second Phase 1/2 trial of NRTX-1001 inhibitory interneuron cell therapy in bilateral MTLE and has additionally announced completed enrollment of an expansion cohort of eight patients for its unilateral MTLE study. Neurona previously announced the design of its upcoming Phase 3 EPIC (EPIlepsy Cell Therapy) trial, based on its discussion with FDA under the Regenerative Medicine Advanced Therapy (RMAT) designation. The results from the Phase 1/2 and Phase 3 trials in both unilateral and bilateral MTLE populations are intended to support a prospective marketing application for the treatment of drug-resistant MTLE.

The bilateral MTLE study (NCT06422923) is a multicenter, open-label trial that will enroll ten adult subjects who will receive a one-time image-guided intracerebral administration of NRTX-1001 into both temporal lobe regions of the brain.

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