Neurona Therapeutics, a clinical-stage biotherapeutics company advancing regenerative cell therapy candidates to treat neurological disorders, recently announced the successful completion of a $120 million financing.
Neurona’s financial success is an encouraging sign for the stem cell and gene therapy sector, highlighting partnerships with investors and public organizations like the California Institute for Regenerative Medicine (CIRM).
CIRM recently awarded Neurona an $8 million clinical project grant to test NRTX-1001—a neural cell therapy derived from human stem cells—for drug-resistant epilepsy. CIRM has supported this research from initial discovery through the ongoing first-in-human clinical trial.
NRTX-1001 is being evaluated in an ongoing clinical trial and has potential application in Alzheimer’s disease and other disorders of the nervous system.
“This financing is a testament to the hard work and dedication of the Neurona team, the commitment of our collaborators, and encouraging preliminary data from the first patients in the ongoing clinical trial of NRTX-1001 cell therapy,” said Cory R. Nicholas, PhD, Neurona’s chief executive officer and co-founder.
The Potential for Suppressing Seizures in a Single Dose
One patient in the CIRM-funded clinical trial is Annette Adkins, who has experienced a remarkable transformation in her life since receiving the single dose of the neuronal cell therapy NRTX-1001.
In the past, Annette endured frequent seizures, but thanks to this groundbreaking single-dose therapy, she is now hiking again and doing other activities she loves.
Photo Courtesy of OHSU/Christine Torres Hicks
In a recent announcement, the Neurona research team reported that Annette had undergone the procedure safely and had a greater than 90% reduction in seizure frequency.
Annette’s story underscores CIRM’s dedication to investing in meaningful research that provides real-world solutions for unmet medical needs.
Updates from the Trial
Data from the first five participants in Neurona’s ongoing clinical trial became available in December 2023.
All five participants had seizures that anti-seizure medications failed to control.
Each participant received one NRTX-1001 administration and temporary immunosuppression.
The immunosuppression aimed to support long-term persistence of the therapy.
During the six-month baseline period, the first two participants experienced 32 and 14 seizures monthly.
More than one year after treatment, both participants reported over 95% fewer seizures overall.
The remaining three participants received treatment more recently. Researchers followed them for three months after treatment.
Two participants experienced 76% and 87% fewer monthly seizures than during baseline.
Before treatment, those participants experienced 26 and 30 seizures per month, respectively.
Both participants achieved these reductions during the first month after treatment.
They also stopped experiencing their more severe focal impaired-awareness seizures.
To read the full release and more updates from the trials, visit the Neurona website.