Agios’ acceleration

How Agios moved so quickly with its cancer metabolism programs

A biomarker-driven strategy in a setting where response was easy to measure, along with a healthy pile of cash, allowed Agios Pharmaceuticals Inc. to go from concept to marketed drug in just over eight years. Now, with a second cancer metabolism candidate slated for registration, the biotech is extending its drug finding model to build an expanded pipeline in rare metabolic disorders and metabolic immuno-oncology.

Agios was founded in 2008 with a $33 million series A round and opened its research laboratory in 2009. On Aug. 4, FDA approved Idhifa enasidenib to treat adults with relapsed or refractory acute myelogenous leukemia (AML) who harbor the isocitrate dehydrogenase 2 (IDH2) mutation.

“The IDH2 target wasn’t even in the company’s portfolio when Agios was started,” CEO David Schenkein told BioCentury.

The company started out with rights to a patent application from Beth Israel Deaconess Medical Center covering a platform used to identify pyruvate kinase M2 isozyme (PKM2) as a possible target.

Co-founder Lewis Cantley’s lab at Harvard Medical School and Beth Israel had found that cancer cells sate their higher energy demands by switching on PKM2. Disabling PKM2’s phosphotyrosine binding region decreased tumor proliferation.

Specifically, Agios’

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