Alzheimer's disease may be the big target in neurodegeneration, but Vigil Neuroscience is taking aim at a smaller one, at least at first. The startup is betting on a new angle—one that focuses on the immune cells of the brain—and zeroing in on rare indications before expanding into larger ones.
The company uncloaked Tuesday with $50 million (PDF) in series A funding and a pair of programs licensed from Amgen designed to harness immune cells called microglia to treat neurodegenerative diseases.
“We can almost think of them as the sentinel cells of the brain’s immune system; they respond to any kind of injury or damage,” said Vigil CEO and President Ivana Magovčević-Liebisch, Ph.D.
Vigil believes it is the first therapeutics company to zero in on microglia, while Tranquis Therapeutics, which launched in July, is targeting the brain’s immune cells more broadly.
Vigil is starting with rare microgliopathies, or disorders of the microglia, and specifically focusing on those caused by single gene mutations. Their patient populations are more uniform, Magovčević-Liebisch pointed out. From there, the company plans to move into more prevalent diseases. It hopes to translate the lessons it learns in these rare diseases to more complex diseases, like Alzheimer’s, where the cause is not so clear-cut.
“We believe that we are going to learn a lot about microglial biology and what is really happening in these patient populations before taking those learnings into larger indications,” Magovčević-Liebisch said.
The funding, drawn from Atlas Venture, Northpond Ventures, Hatteras Venture Partners and Alexandria Venture Investments, will push Vigil’s lead asset, an antibody that activates the protein TREM2, into the clinic in 2021. It will also get its second program, a small molecule with the same target, into IND-enabling studies, as well as lay the groundwork to build Vigil’s pipeline.
TREM2 acts as a damage sensor on microglia, playing a role in how they react to injury, said Marco Colonna, M.D., chair of Vigil’s scientific advisory board, in a statement. Colonna made many of the discoveries underpinning Vigil’s work in his lab at Washington University School of Medicine.
“The loss of function of TREM2 has now been connected to neurodegenerative disease,” Magovčević-Liebisch said. “If you can restore the function of TREM2, you can restore the microglia and increase a number of things that the microglia are involved in to respond to damage. Then you will be able to arrest the damage that is occurring.”
And it’s not stopping at TREM2. With its scientific advisory board, the company is on the lookout for other targets important to microglia function. It could pursue those targets through in-house discovery and development or licensing programs, Magovčević-Liebisch said.