Since October 2018, scientists at Neuron23 have worked quietly to find their place in the world of precision medicine. That place is using artificial intelligence and data science to target neurodegenerative and neuroinflammatory diseases in patients with specific genetic mutations.
Founded through a partnership with Germany’s Origenis, Neuron23 launches Wednesday with $113.5 million in financing. The funds, which comprise $30 million in series A funding from Westlake Village BioPartners and Kleiner Perkins and an $80 million series B led by Redmile Group, will propel two to three programs into the clinic in the next two years.
RELATED: Parkinson's discovery could yield new treatment strategy for young onset patients
“We wanted to make sure that when we came out, we were really in the race,” Neuron23 CEO Nancy Stagliano, Ph.D., said. “We thought it was the right time given the focus and resurgence of interest in the CNS industry.”
Neuron23’s lead program targets Leucine-rich repeat kinase 2 (LRRK2), a gene that codes for a kinase enzyme of the same name. Mutations in this gene are strongly associated with 3% of patients with Parkinson’s disease. What’s exciting, Stagliano said, is that finding a small-molecule inhibitor for LRRK2, which has a very clear genetic linkage to disease, can provide a foundation of biological knowledge to help treat the other 97% of Parkinson’s patients.
The second program is pursuing brain-penetrant inhibitors for tyrosine kinase 2 (TYK2), a kinase that leads to multiple sclerosis.
RELATED: Harper, Seidenberg's Westlake Village BioPartners reels in $500M across 2 funds
It can be difficult to get small-molecule inhibitors into the brain, because the brain has evolved to keep toxins and other pathogens out of it through its blood-brain barrier. Small molecules can get through the bloodstream but, due to their structure or other properties, can be quickly pumped out from the brain.
“One of the strengths of our partnership with Origenis, the additional chemistry partner we founded the company with, is getting these brain-penetrant molecules against these kinds of target kinases,” Stagliano said. “LRRK2 is extremely exciting and an area of hot pursuit in our industry, because it systemically has also been linked to Crohn’s disease. So, we can look at that second group of LRRK2 molecules that don't get into the brain and we can say, what can we use those for?”
In addition to its work on brain-penetrant molecules that inhibit both of these kinases’ functions in the brain, Neuron23 will design inhibitors that don’t penetrate the brain to treat systemic diseases like Crohn’s caused by LRRK2 or autoimmune diseases caused by TYK2.
Neuron23’s funding will push those treatments through the clinic, allow the company to recruit more talent and make room for additional disease programs.