Spurred on by OrbiMed partner's diagnosis, Vanqua Bio takes aim at neurodegenerative disease with blue-chip backers
The FDA’s recent approval of Biogen’s Aduhelm has provided a spark plug for neuroscience research across the biopharma spectrum with investors more likely than ever to jump on board. A Chicago biotech now has its own clutch of blue-chip backers to pursue drugs across a range of indications — and it all started from a close colleague’s own diagnosis and search for a cure.
OrbiMed startup Vanqua Bio closed an $85 million Series B led by Omega Funds to put its lead candidate targeting the GCase enzyme in the clinic, first in Gaucher disease and then for a rare form of Parkinson’s, the company said Tuesday.
The company’s lead drug, a small molecule activator of GCase, targets the mutant GBA1 gene pathway, which can cause Gaucher and is correlated with a form of Parkinson’s known as GBA-PD that affects around 10% of all patients and a form of Lewy body dementia known as GBA-LBD.
The biotech launched back in 2019 emboldened by the work of Jonathan Silverstein, a former managing partner at OrbiMed who was diagnosed with GBA-PD some five years ago and kickstarted the Silverstein Foundation to pursue cures for that disease. Funds from the foundation helped pay for pivotal genetics research in the lab of Dimitri Krainc, the chair of neurology at Northwestern University’s Feinberg School of Medicine, which now undergirds Vanqua’s platform.
Vanqua relies both on genetics research out of Krainc’s lab and in vitro modeling technology that tests compounds in patient-derived neurons, potentially offering an early sneak peek at the drug’s efficacy for human tests. The neurons are derived from induced pluripotent stem cells, which are in turn derived and converted from patients’ skin cells.
It’s an approach that looks to fill in the gap left by inadequate animal modeling for neuroscience, where the chances of success in large indications like Alzheimer’s disease and Parkinson’s have left a slew of failures in its wake. According to Vanqua CSO Kevin Hunt, rodent models simply aren’t good predictors of in vivo human testing in neuroscience, and adding a stable preclinical research tool derived from potential patients could eventually help crack the code for a range of diseases with huge unmet clinical needs.
CEO Jim Sullivan, a former VP of research at AbbVie who helped shepherd JAK inhibitor Rinvoq and blood cancer medicine Venclexta into the clinic, and has worked as a venture partner at OrbiMed since 2019, sees Vanqua’s platform as a best of all worlds strategy: combining cutting-edge genetics research and preclinical tools to develop effective molecules with a next-generation modeling approach to up the chances of success.
“Our approach builds on breakthroughs in a couple different areas: one, genetics and pinpointing causes of neurodegenerative disease and two, much more enhanced preclinical models,” he said. “We’re really trying to overcome the limitations of neuroscience research for the last decade.”
The team expects “meaningful” clinical data from its lead drug to be published within the next two years, Sullivan said.
In GBA-PD, which may affect around 800,000 patients worldwide and 100,000 in the US, Vanqua is looking to pair genetic marker researchers pioneered in Krainc’s lab to partner with patient cells and established biology around the GBA1 pathway to tackle Gaucher. But in Alzheimer’s or ALS — or even Parkinson’s, in the case of GBA1 — the genetic correlation is less understood, leaving a big gap where a team like Vanqua could add to the scientific heavy lifting and potentially find a breakthrough along the way.
The Vanqua team acknowledges that cracking the code on Alzheimer’s, for instance, despite the recent approval of Biogen’s Aduhelm, will require a great deal more work in understanding the genetic pathways and developing therapeutic modalities to target them.
But that doesn’t mean they’re not ready to try. After the GCase lead drug, Vanqua has an undisclosed therapy in the preclinical stage that leverages the complement factor pathway to potentially modulate a range of neurodegenerative disorders. The research is still relatively new, but Sullivan said the program could eventually prove to be a game-changer in other large indications.
It’s still early days, but Vanqua has all the trappings of an ambitious platform company and enough scientific cachet to pull in a notable investor syndicate. In addition to round leader Omega, the syndicate is joined by Series A investor OrbiMed and new investors Surveyor Capital, Avoro Ventures, Pontifax, Casdin Capital Management, Eli Lilly, Logos Capital and Osage University Partners.
The biotech expects to use the proceeds to advance its two lead programs and add to its team of 15, potentially doubling in size in the coming years. Vanqua has headquarters in Chicago, near Krainc’s lab from which the team is shuttling researchers over to join the clinical effort. Sullivan highlighted the growing Chicago biotech hub as a major draw for Vanqua and a source of untapped potential for the neuroscience field writ large.