Agios Pharmaceuticals

Agios Pharmaceuticals Awarded Funding From Accelerate Brain Cancer Cure (ABC2) to Support Research into Novel Therapeutics and Diagnostics for Brain Cancer

Agios Pharmaceuticals, the leading biopharmaceutical company focused on discovering and developing novel drugs in the emerging field of cancer metabolism, announced today that it has received funding from Accelerate Brain Cancer Cure (ABC2), a non-profit organization that supports brain cancer research. ABC2’s funding will enable new research investigating IDH1 gene mutations in brain cancer, with the goal of supplementing Agios’s ongoing research into the development of new IDH1 therapeutics and diagnostics.

Recent research by Agios scientists published in the journal Nature established that the mutated form of the enzyme IDH1 produces a metabolite, 2-hydroxyglutarate (2HG), which may contribute to the formation and malignant progression of gliomas, the most common type of brain cancer.[1] This discovery creates the opportunity for therapeutic intervention in brain cancer and other cancers where IDH1 mutations are present using new drugs that can target the IDH1 metabolic pathway and prevent the buildup of 2HG. 2HG also represents a potential metabolic biomarker that may enable rapid diagnosis and earlier treatment of this form of glioma.

“We are delighted to be collaborating with ABC2 on this important research into the role of the IDH1 mutation in brain cancer,” said David Schenkein, M.D., Chief Executive Officer, Agios. “ABC2 has a track record of partnering with biotech companies to bring drugs to patients more quickly. Agios is aggressively developing new therapeutics specifically targeting IDH1 which we hope can have a profound impact on the lives of patients with brain cancer.”

“The Agios approach to cancer metabolism research has revealed a new strategy for treating this deadly disease,” said Max Wallace, Chief Executive Officer, ABC2. “Beyond the compelling scientific evidence, we are excited to be partnering with such an exceptional team with a proven track record of success.”

About the Metabolic Enzyme IDH1 and its Role in Brain Cancer

Research has linked mutations in the metabolic enzyme IDH1 to glioma and other cancers.[2] Approximately 70 percent of low grade gliomas are known to have the IDH1 mutation. New research at Agios, published in the journal Nature [1] has revealed the function of mutations in IDH1 and suggests that aberrant activation of this metabolic pathway may lead to the development of brain cancer.

About Gliomas

A glioma is a type of cancer that starts in the brain or spine. It is called a glioma because it arises from glial cells. The most common site of gliomas is the brain, but gliomas can also affect the spinal cord or any other part of the CNS, such as the optic nerves. High grade gliomas currently cannot be cured and the prognosis for patients is generally poor; of the 20,000 Americans affected each year, more than half die within eighteen months of diagnosis. Gliomas are the most common type of brain cancer.

About Cancer Metabolism

Cancer metabolism is a new and exciting field of biology that provides a novel approach to treating cancer. Cancer cell metabolism is marked by profound changes in nutrient requirements and usage to ensure cell proliferation and survival. Research in the field has demonstrated that cancer cells become addicted to certain fuel sources and metabolic pathways. Identifying and disrupting certain enzymes in these metabolic pathways provides a powerful intervention point for discovery and development of cancer therapeutics.

About Agios Pharmaceuticals

Agios Pharmaceuticals is the first biopharmaceutical company dedicated to the discovery and development of novel therapeutics in the emerging field of cancer metabolism. To support and drive these efforts, Agios is building a robust platform integrating metabolomics, genetics, biochemistry and microscopy to enable target and biomarker identification. Agios’ capabilities to interrogate differential cellular metabolism of diseased cells relative to normal cells will also be applicable to other therapeutics areas including autoimmune, inflammatory and neurological diseases. To date, Agios has put in place a world-class scientific team of more than 60 people, built a fully integrated cell metabolism platform within the largest research laboratory dedicated to cancer metabolism and created an emerging product development pipeline of novel cancer therapeutics. The Company’s founders represent the core thought leaders in the field of cancer metabolism, responsible for key advances, insights and discoveries in the field. Agios Pharmaceuticals is located in Cambridge, Massachusetts. For more information, please visit the company’s website at

[1] Dang et al. Cancer-associated IDH1 mutations produce 2-hydroxyglutarate. Nature 2009;In press. DOI: 10.1038/nature08617

[2] Parsons, D. W. et al. An integrated genomic analysis of human glioblastoma multiforme. Science 321, 1807-12 (2008); Yan, H. et al. IDH1 and IDH2 mutations in gliomas. N Engl J Med 360, 765-73 (2009).