Cell Therapy Biotech Neurona Secures $102M for Epilepsy Treatment Advancement

Neurona Therapeutics, a California-based biotech company, has successfully raised $102 million in an oversubscribed financing round to advance its innovative cell therapy for epilepsy treatment. The funding, backed by more than 15 investors including Fidelity Management & Research Company, the Column Group, and Alexandria Venture Investments, will primarily support the company's lead candidate, NRTX-1001, as it progresses into a pivotal phase 3 trial.

Promising Phase 1/2 Results Fuel Investor Confidence

NRTX-1001, an allogeneic cell therapy candidate targeting drug-resistant mesial temporal lobe epilepsy (MTLE), has shown encouraging results in ongoing phase 1/2 trials. Data presented in December from ten patients in the unilateral MTLE study revealed a 92% median reduction in disabling seizures from baseline in the low-dose cohort, measured seven to 12 months post-administration.

The therapy has demonstrated a favorable safety profile, with no adverse events attributed to NRTX-1001 reported to date. This positive data has not only attracted substantial investor interest but also earned the candidate a regenerative medicine advanced therapy (RMAT) designation from the FDA in June 2024.

Advancing to Pivotal Phase 3 Trial

With the new funding in place, Neurona is preparing to launch a single pivotal phase 3 trial, dubbed Epic. This double-blind, randomized study will evaluate NRTX-1001 against a sham control in adults with drug-resistant MTLE. The results from this trial are expected to support regulatory submission for potential drug approval.

Dr. Cory Nicholas, President and CEO of Neurona Therapeutics, commented on the significance of this milestone: "This substantial funding round validates the potential of our innovative approach to treating epilepsy. We're excited to move NRTX-1001 into late-stage clinical testing and closer to potentially offering a new treatment option for patients with drug-resistant MTLE."

Navigating a Challenging Funding Landscape

Neurona's successful fundraising comes amid a challenging period for the cell and gene therapy sector. The company had previously conducted an undisclosed series financing round of $120 million in early 2024, following a staff reduction of 25% in 2023 due to the difficult funding environment.

This latest capital injection highlights Neurona's resilience and the strength of its clinical data in attracting investor support. It stands in contrast to the broader trend in the cell and gene therapy field, which has seen at least 20 layoff rounds in the first quarter of 2025 alone, accounting for approximately 31% of all biopharma layoffs during that period.

As Neurona advances its epilepsy treatment towards late-stage clinical trials, the company's progress will be closely watched by industry observers and patients alike, potentially signaling a new chapter in the treatment of drug-resistant epilepsy.