Neurona Therapeutics secured $102 million in an oversubscribed financing round, fueled by positive clinical data from its ongoing Phase 1/2 trial of NRTX-1001 for drug-resistant epilepsy. The funding will support the advancement of NRTX-1001 into a pivotal Phase 3 trial (EPIC) designed to support a Biologics License Application (BLA). This investment follows a prior $120 million round in February 2024 and demonstrates strong investor confidence in the therapy’s potential.
This development is crucial because it signals significant progress toward a potential new treatment option for drug-resistant mesial temporal lobe epilepsy (MTLE), a debilitating condition with limited effective therapies. The successful financing allows Neurona to expedite the clinical development of NRTX-1001, potentially bringing much-needed relief to patients sooner. The initiation of a pivotal Phase 3 trial represents a critical milestone on the path to regulatory approval and commercialization.
The Phase 1/2 trial of NRTX-1001 has shown promising results, with patients experiencing substantial and durable seizure reduction. Interim data revealed a 92% median seizure reduction in the low-dose cohort and a 78% reduction in the high-dose cohort. Notably, the therapy has been well-tolerated without any reported cognitive impairments. The Phase 3 EPIC trial will be a randomized, double-blind study comparing NRTX-1001 to a sham control, with a crossover option for control group participants. This rigorous design will provide robust data to assess the efficacy and safety of NRTX-1001.
The successful financing and the FDA’s alignment on the Phase 3 trial design position Neurona Therapeutics to potentially revolutionize the treatment landscape for drug-resistant MTLE. Positive results from the EPIC trial could lead to the first FDA-approved regenerative cell therapy for this indication, offering new hope for patients who currently have limited treatment options. The substantial financial backing also strengthens Neurona’s position to expand its pipeline of allogeneic cell therapies for other chronic neurological disorders.
