Neurona Therapeutics Raises $102M for NRTX-1001 Phase 3 Trial

Neurona Therapeutics, a clinical-stage, privately-held, biotherapeutics company developing regenerative cell therapies for disorders of the nervous system, announced the successful completion of an upsized and oversubscribed $102 million financing. New and existing investors in the financing round include Fidelity Management & Research Company, The Column Group, Soleus Capital, Viking Global Investors, Cormorant Asset Management, Schroders Capital, LYFE Capital, Euclidean Capital, UCB Ventures, Willett Advisors, UC Investments, YK Bioventures, Berkeley Frontier Fund, Ysios Capital, Alexandria Venture Investments, and Spur Capital Partners.

Proceeds from the financing will be used to advance the company’s wholly owned pipeline of allogeneic cell therapy candidates for chronic neurological disorders, including its lead investigational candidate, NRTX-1001, for drug-resistant mesial temporal lobe epilepsy (MTLE), which is among the most common types of epilepsy. In an ongoing multicenter, open-label Phase 1/2 trial in adults with drug-resistant MTLE, NRTX-1001 has been well-tolerated to date and has demonstrated the potential to provide substantial and durable seizure reduction.

“This robust financing speaks not only to the positive results we are generating in our ongoing Phase 1/2 trial of NRTX-1001, but also to the hard work and dedication of the Neurona team and our collaborators,” said Cory R. Nicholas, Ph.D., Neurona’s Chief Executive Officer and Co-Founder. “We are grateful for the collective investment from this highly reputable syndicate – which comes on top of the $120 million we raised in February 2024 – as we prepare to recruit patients for the Phase 3 EPIC trial in the second half of 2025.”

The advancement of NRTX-1001 into the Phase 3 EPIC trial follows productive discussions with the U.S. Food and Drug Administration under the RMAT designation granted in June 2024. In the EPIC trial, adults with drug-resistant MTLE will be randomized 2:1, in a double-blind design, to NRTX-1001 or a sham-control group. Upon reaching the primary endpoint, subjects in the control group will have the opportunity to cross-over and receive NRTX-1001. Pending the outcome of the Phase 3 EPIC trial, and supported by ongoing Phase 1/2 trials, the results will be part of a growing data package to support regulatory submission for potential drug approval.

At the Annual Meeting of the American Epilepsy Society in December 2024, Neurona presented updated data from both the low-dose (n=5) and high-dose (n=5) cohorts of the company’s ongoing open-label Phase 1/2 clinical trial of NRTX-1001 GABAergic interneuron cell therapy in adults with drug-resistant, unilateral MTLE. The low-dose cohort demonstrated a 92% median reduction in disabling seizures from baseline, with 80% of subjects reporting >80% seizure reduction, during the primary efficacy evaluation period of 7-12 months after NRTX-1001 administration. Notably, the first two subjects followed for 24 months post-dosing both continued to report >97% seizure reduction after a single administration of NRTX-1001. Although the 7-12-month results are not yet available for the high-dose cohort, this cohort demonstrated a 78% median reduction in disabling seizures from baseline in the interim 4-6-month period after administration of NRTX-1001. Importantly, no cognitive impairments were detected in either cohort, and some subjects had neurocognitive test scores that were markedly improved from baseline levels. NRTX-1001 has been well-tolerated to date at both high and low doses, with no adverse events attributed to the cell therapy.