Neurona Therapeutics has gained $102m to support the advancement of its investigational cell therapy, NRTX-1001, into Phase III testing for drug-resistant mesial temporal lobe epilepsy (MTLE).
The financing 鈥� secured after Neurona鈥檚 $120m fundraising round in February 2024 鈥� will also help expand the company鈥檚 pipeline of allogeneic cell therapy candidates for chronic neurological disorders. Investors in the latest round include Fidelity Management & Research Company, The Column Group, Soleus Capital, Viking Global Investors, and Cormorant Asset Management.
NRTX-1001 is a designed to deliver neural cells that secrete the neurotransmitter gamma-aminobutyric acid (GABA), silencing seizure activity in the epileptic region of the brain. NRTX-1001 is derived from human pluripotent stem cells, which are manufactured in-house by Neurona.
The company aims to position it as a potential alternative to brain surgery, which remains the standard intervention for drug-resistant MTLE but carries risks of memory impairment and other neurocognitive deficits. Neurona has stated that NRTX-1001, administered as a one-time treatment, could provide a durable reduction in seizure frequency while preserving brain function.
The Phase III EPIC trial is expected to begin enrolling patients in H2 2025 following what Neurona described as 鈥減roductive discussions鈥� with the US Food and Drug Administration (FDA). The agency granted NRTX-1001 a regenerative medicine advanced therapy (RMAT) designation in June 2024.
The trial will enrol adults with drug-resistant MTLE in a randomised, double-blind design, assigning patients to either NRTX-1001 or a sham-control group in a 2:1 ratio. Patients in the control arm will have the option to receive the therapy once the primary endpoint is reached.
Early-stage clinical data suggest the therapy could offer meaningful benefits. In an ongoing Phase I/II trial (NCT05135091), NRTX-1001 has been well tolerated, and interim results presented at the American Epilepsy Society annual meeting in December 2024 showed that two patients who received a low-dose treatment reported a more than 97% reduction in seizures two years after administration.
There is a growing drive for innovative treatments as up to one-third of the estimated 50 million people with epilepsy do not respond to existing therapies. While current therapies include surgery, neurostimulation devices, and dietary interventions, there is increasing interest in regenerative and precision medicine approaches.
Neurona is also exploring the potential of NRTX-1001 for other neurological conditions, including Alzheimer鈥檚 disease, where it is currently in preclinical evaluation.
