Neurona Therapeutics, a clinical-stage biotherapeutics company advancing regenerative cell therapy candidates for the treatment of 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 the initial discovery research stage to 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, 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 cohort of five participants in Neurona’s ongoing clinical trial were shared in December 2023.
The five participants entered the study with a history of seizure activity that was not controlled by anti-seizure medications. These subjects received a one-time administration of NRTX-1001 as well as temporary immunosuppression to promote the long-term persistence of cell therapy.
The first two subjects, who experienced 32 and 14 seizures per month, respectively, during the six-month baseline, have continued to report a reduction of more than 95% in overall seizure counts more than one year after taking the dose of NRTX-1001.
The remaining three subjects were treated with NRTX-1001 more recently and have been followed for three months after the dose. Two of the three subjects demonstrated reduced monthly seizure frequencies of 76% and 87% from baseline levels (26 and 30 seizures per month, respectively) since the first month, with elimination of their more severe focal impaired-awareness seizures.
To read the full release and more updates from the trials, visit the Neurona website.
The Stem Cellar 