Vigil bags $90M to take ex-Amgen assets on path from rare diseases to Alzheimer's

Vigil Neuroscience is barreling forward with a strategy that will see it hop along steppingstones from rare diseases to Alzheimer’s. Having emerged with $50 million late last year, Vigil has gathered up an additional $90 million to validate its ex-Amgen asset in a rare, inherited neurodegenerative disease.

Massachusetts-based Vigil is focused on TREM2, a target that acts as a neurological damage sensor. Loss-of-function mutations in TREM2 are linked to neurodegenerative diseases, and knocking out the target in mice leads to loss of brain white matter. Vigil responded to the data by picking up two drugs from Amgen, raising $50 million and plotting out an R&D plan that goes from niche to mainstream.

With Arie Belldegrun’s Vida Ventures leading a series B financing round, Vigil has shared more details of its plans. The first step is to develop monoclonal antibody VGL101 in the rare neurodegenerative disease adult-onset leukoencephalopathy with axonal spheroids and pigmented glia (ALSP).

ALSP patients suffer white matter lesions. Typically starting after 40 years of age, the symptoms span personality changes through to seizures, dementia and Parkinsonian movement abnormalities. Vigil is betting it can bring the first treatment to the estimated 10,000 ALSP patients in the U.S. by hitting TREM2 to restore the function of microglia, the  central nervous system’s immune defense cells. 

RELATED: With Amgen assets and $50M, Vigil takes aim at brain's 'sentinel cells' to fight neurodegenerative diseases

The concept of targeting immune cells to treat neurodegenerative diseases underpins a small pack of new biotechs. Over the past year or so, Vigil, Tranquis Therapeutics and Muna Therapeutics have collectively raised almost $250 million to explore their spins on the idea. 

Vigil plans to start testing VGL101 in healthy volunteers by the end of the year. The study is the first small clinical step in an R&D strategy that will expand out in multiple directions in the coming years, provided the data support further work. Vigil plans to use knowledge gained in ALSP,  a rare disease with strong ties to microglial deficiency, to study drugs in bigger, more diverse groups of patients. 

Alzheimer’s is on Vigil’s hit list. As a monoclonal antibody, VGL101 may struggle to get into the brain in high enough concentrations to move the needle in common diseases such as Alzheimer’s, leading Vigil to advance a small molecule in its wake. The small-molecule TREM2 agonist program is in lead optimization and is aiming to deliver molecules that readily penetrate the central nervous system.

Other biotechs have stumbled when trying to go from well-defined niche patient populations to the larger and more diverse indications that offer big financial rewards. Vigil’s plan is backed by evidence that TREM2 has a role in Alzheimer’s, with studies showing microglia need the target to evolve into the disease-associated cells that can contain amyloid-beta plaques in patients with the disease.