Neurona Raises $120M Bolstered by Promising Early-Stage Data for Epilepsy Cell Therapy NRTX-1001

In 2023, Neurona Therapeutics, a clinical-stage biotherapeutics company, announced a $120 million funding round amidst the promising preliminary data of its regenerative cell therapy, NRTX-1001, for mesial temporal lobe epilepsy (MTLE). The open-label, single arm, phase I/II clinical trial showcased a significant reduction (>95%) in seizure frequency in the first patient one year post-treatment and a total seizure elimination since seven months post-treatment. The second patient also showed a remarkable reduction (>90%) in overall monthly seizure frequency at seven months.

The lead candidate, NRTX-1001, consists of universal, off-the-shelf, allogeneic neural cell therapy candidates designed to repair dysfunctional neural networks for individuals with drug-resistant epilepsy. The therapy aims to address the underlying hyperactive neural networks in epileptic seizures by injecting inhibitory GABAergic interneurons.

The positive early readout for NRTX-1001 contributed to the company securing substantial funding despite previously experiencing challenging market conditions. The financing will advance Neurona's lead candidate, with ongoing and planned clinical studies of NRTX-1001 for various epilepsy types and Alzheimer's disease. It will also contribute to the development of other pipeline projects, such as myelinating glial cell and gene-edited cell therapies for unspecified indications.

Neurona's CEO, Cory Nicholas, acknowledges that further evaluation will be conducted with more patients before drawing definitive conclusions about the therapy's long-term potential. The funding will aid advancements in other therapeutic areas and allow Neurona to continue investing in gene-editing cell therapies.