Neurona Raises $120M, Buoyed by Early-Stage Data for Epilepsy Cell Therapy

Pictured: 3D illustration of stem cells used for cell therapy

Pictured: 3D illustration of stem cells used for cell therapy

Neurona Therapeutics will use the funds to advance its investigational regenerative neural cell therapy NRTX-1001 for mesial temporal lobe epilepsy, the most common form of focal epilepsy.

Pictured: 3D illustration of stem cells used for cell therapy/iStock, Maksim Tkachenko

Neurona Therapeutics on Thursday announced that it has secured $120 million in funding, which will help it advance its pipeline of investigational off-the-shelf cell therapies.

The California-based biotech will use the money to advance its lead candidate NRTX-1001, a regenerative neural cell therapy candidate being assessed in a Phase I/II trial for drug-resistant mesial temporal lobe epilepsy (MTLE), the most common form of focal epilepsy that can trigger ongoing seizures despite medical treatment.

Neurona’s financing on Thursday was co-led by Viking Global Investors and Cormorant Asset Management. Other institutional supporters—such as UC Investments, UCB Ventures, Euclidean Capital, The Column Group and Alexandria Venture Investments—also participated in the funding round.

“Neurona has pioneered development of a fully-differentiated cell therapy for drug-resistant focal epilepsy that is designed to be disease-modifying, repairing the affected neural network, and is yielding very promising initial clinical data,” Raymond Kelleher, managing director at Cormorant, said in a statement, adding that NRTX-1001 could be a “game-changer” with its potential to control seizures and preserve neurocognitive function.

According to the biotech’s website, NRTX-1001 is a cell therapy comprised of human inhibitory GABAergic interneurons, which can potentially address the underlying hyperactive neural networks in epileptic seizures.

In December 2023, at the annual meeting of the American Epilepsy Society, Neurona presented data from two patients enrolled in the Phase I/II study of NRTX-1001, who saw at least a 95% drop in overall seizure frequency more than one year after treatment. NRTX-1001 was also safe overall, with adverse events categorized as mild to moderate in severity.

Neurona is also testing NRTX-1001 in Alzheimer’s disease, for which it is currently in the preclinical evaluation stage.

Beyond NRTX-1001, Neurona will also channel some of Thursday’s haul into its other pipeline projects including investigational myelinating glial cells and gene-edited cells, both for yet-undisclosed indications.

Neurona’s funding comes nearly a year after the biotech laid off 18 employees, or 25% of its total headcount, to “streamline” its budget “in the face of the current tight funding environment and extend available cash to support the ongoing clinical trial of NRTX-1001,” a spokesperson told Fierce Biotech at the time.

The financing could also be a sign of an uptick in the industry’s fundraising activities. This week alone, there have been two initial public offerings—Kyverna and Metagenomi—which follow the earlier debuts of Alto Neuroscience, Fractyl Health, ArriVent Biopharma and CG Oncology.

Tristan Manalac is an independent science writer based in Metro Manila, Philippines. Reach out to him on LinkedIn or email him at tristan@tristanmanalac.com or tristan.manalac@biospace.com.

Tristan is an independent science writer based in Metro Manila, with more than eight years of experience writing about medicine, biotech and science. He can be reached at tristan.manalac@biospace.com, tristan@tristanmanalac.com or on LinkedIn.
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