Firm will also expand R&D into field of inborn errors of metabolism.

Agios Pharmaceuticals raised $78 million in an oversubscribe Series C round of fundraising with new and existing investors. The firm, which is focused on developing drugs targeting cancer metabolism, will use the cash injection to progress its pipeline into the clinic and expand R&D to identify targets in the field of inborn errors of metabolism (IEM), a class of genetic diseases caused by mutations or defects in single metabolic genes.

In comparison with normal cells, cancer cells have modified requirements for metabolites and change the way they process sugars, fats, amino acids, and other energy sources to fuel continued proliferation, Agios explains. These proliferation-supporting processes represent a cellular phenomenon known as metabolic rewiring. The firm is exploiting its scientific expertise to generate a portfolio of anticancer drugs that essentially starve cancer cells by interrupting these cancer-specific metabolic processes. Agios’ drug discovery engine integrates techniques in metabolomics, biology, genetics, biochemistry, and bioinformatics, to identify and prioritize metabolism targets and metabolic biomarkers, and devise clinical strategies to identify the patients most likely to respond to a specific metabolism treatment. 

The firm has two lead discovery programs. One is targeting the IDH1 and IDH2 metabolic pathways for the treatment of brain cancer, amyotrophic lateral sclerosis (AML), and other cancers that carry IDH1 or IDH2 mutations. The cancer-related mutations lead to production of a metabolite, 2-hydroxyglutarate (2HG), which appears to contribute to the formation and malignant progression of gliomas and other cancers, Agios claims. 2HG itself may therefore represent a cancer biomarker for diagnostics development. Its second program is centered on regulation of the M2 form of the glucose metabolism enzyme pyruvate kinase (PKM2).

In addition to its in-house programs, the firm has an ongoing collaboration with Celgene, signed in April 2010, to discover and develop disease-modifying anticancer treatments based on Agios’ cancer metabolism research platform. Under terms of the deal Agios received a $130 million up-front payment, incluing an equity investment. Celgene retains a period of exclusivity that gives it the option to license resulting products at the end of Phase I studies. In October the firms extended this period of exclusivity from three to four years, in return for which Agios received another $20 million payment. 

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