Neurona Therapeutics, a clinical-stage biotherapeutics company, is developing regenerative cell therapies for the treatment of neurological disorders.
They recently presented updated clinical data from their ongoing Phase I/II trial of NRTX-1001 in people with drug-resistant mesial temporal lobe epilepsy (MTLE) at the American Academy of Neurology 2023 Annual Meeting.
The data showed a >90% reduction in seizure frequency in the first and second patients at nine and five months post-treatment, respectively. Additionally, the first patient demonstrated an improvement in memory after NRTX-1001 administration.
NRTX-1001, a non-destructive human cell therapy, has been well tolerated with no severe adverse events. The trial has received approval to enroll the remaining patients in the first cohort and clearance from the FDA to expand trial enrollment to include adults with MTLE in the memory-dominant lobe.
Neurona is also investigating NRTX-1001’s potential use in other related indications, such as bilateral MTLE, neocortical focal epilepsy, and Alzheimer’s disease with hyperexcitability in the temporal lobe.
The ongoing multicenter Phase I/II clinical trial aims to evaluate the safety and efficacy of a single administration of NRTX-1001 for drug-resistant MTLE. Patient recruitment is underway at epilepsy centers across the US.
NRTX-1001 is a one-time dose of an injectable suspension of inhibitory neurons derived from human stem cells designed to durably silence seizure activity in the epileptic region of the brain. It is manufactured using proprietary methods to guide stem cells into specific types of interneurons that secrete the inhibitory neurotransmitter gamma-aminobutyric acid (GABA).
MTLE is a common type of focal epilepsy in adults, affecting the internal structures of the temporal lobe. Current surgical options are not effective for all patients and can have significant adverse effects.
Neurona’s regenerative cell therapy candidates, like NRTX-1001, have the potential to provide long-term repair of dysfunctional neural networks for multiple neurological disorders.