apples-to-oranges comparisons when stacking up a low-risk program to develop another statin drug for lowering cholesterol, as opposed to a new drug for lupus, which uses a completely new mode of action for a disease that doesn’t have effective therapies. Amgen has been known in the past to take on some of these lower-risk projects, like making longer-lasting versions of erythropoietin (Aranesp) and pegfilgrastim (Neulasta). But now Amgen’s pipeline is a bit more daring, with about two-thirds to three-fourths of its candidates being aimed at diseases where no one has a marketed product that works the same way, Miletich says.
One of the most interesting examples that Miletich offered up is a new antibody drug emerging in Amgen’s pipeline, called AMG-785. This is based on biological work that says a protein called sclerostin normally neutralizes production of osteoblasts, the cells that build new bone. Some patients with rare genetic abnormalities, who lack the gene to make sclerostin, have been shown to end up with severe deformities from bone overgrowth by the time they are in their 30s, Miletich says.
Even more interestingly, patients with only one of two functioning copies of the sclerostin gene have no symptoms of disease, but they have really strong bone mineral density, which means they don’t develop osteoporosis later in life, and usually “they don’t break bones” in accidents, Miletich says.
So Amgen’s vision is to build on that fundamental genetic insight to create a drug that does that same thing, blocking sclerostin in a carefully calibrated way. With some clever in-house protein engineering, Miletich says Amgen showed an experimental antibody drug could block
