Agios has led the field of cancer metabolism with its novel IDH1 and IDH2 programs, and continues to make important advances in the field of rare genetic diseases with its PKR program. These programs exemplify our strategy of applying our foundational expertise in cellular metabolism and precision medicine to translated science from our labs into first-of-their-kind experimental therapies.
In addition to advancing our lead programs, we continue to discover novel metabolic targets that meet a high bar for future development. Our “wave two” projects are in early to late drug discovery phase, and our “wave three” projects are in validation phase.
The most advanced of our wave two programs is focused on MTAP-deleted cancers. Agios scientists have discovered a novel pathway comprised of multiple targets with a shared vulnerability in MTAP-deleted tumors. Our scientists have demonstrated that this pathway can be modulated by small molecule inhibitors, resulting in robust anti-tumor activity in animal models. MTAP (methylthioadenosine phosphorylase) is a metabolic enzyme that is deleted in approximately 15 percent of all cancers. This deletion is readily detected by a simple genomic test, thus allowing the selection of patients predicted to be sensitive to the therapy. This program is being developed in collaboration with Celgene.