Neurona Therapeutics has secured $102 million to fund its Phase 3 trial of NRTX-1001, an off-the-shelf cell therapy for epilepsy.
The oversubscribed financing round, supported by over 15 investors including Fidelity Management & Research Company, the Column Group, and Alexandria Venture Investments, will advance Neurona’s pipeline of allogeneic cell therapies for chronic neurological disorders.
NRTX-1001, targeting a common epilepsy form, is currently in two open-label Phase 1/2 trials for drug-resistant mesial temporal lobe epilepsy (MTLE). In December, data from ten patients in the unilateral MTLE study showed a 92% median reduction in disabling seizures at 7-12 months post-administration, with good tolerability and no NRTX-1001-related adverse events.
Neurona plans future trials of NRTX-1001 for neocortical focal epilepsy and other indications. NRTX-1001, a GABAergic interneuron cell therapy, received Regenerative Medicine Advanced Therapy (RMAT) designation in June 2024. With RMAT designation, Neurona and the FDA have discussed moving to a single pivotal Phase 3 trial, Epic, in adults with drug-resistant MTLE. This double-blind, randomized trial will compare NRTX-1001 to a sham control, with results intended to support potential drug approval.
https://www.fiercebiotech.com/biotech/cell-therapy-biotech-neurona-nabs-102m-push-epilepsy-candidate-pivotal-phase-3
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