GSK-1482160 is being evaluated for treatment of inflammatory pain (such as arthritis). This compound acts on P2X7 receptors, expressed by cells of innate and adaptive immunity. P2X7 receptors are involved in the production of pro-inflammatory cytokines that are thought to be an important mediator of inflammation. By blocking P2X7 receptors, less inflammatory chemicals are released, which possibly results in less inflammatory pain. Because of its ability to target P2X7R with high selectivity and to be radiolabelled with 11C, GSK-1482160 was suggested to be a useful biomarker for neuroinflammation via positron emission tomography (PET).

Pinometostat, also known as EPZ-5676, is a small molecule inhibitor of histone methyltransferase with potential antineoplastic activity. Upon intravenous administration, EPZ-5676 specifically blocks the activity of the histone lysine-methyltransferase DOT1L, thereby inhibiting the methylation of nucleosomal histone H3 on lysine 79 (H3K79) that is bound to the mixed lineage leukemia (MLL) fusion protein which targets genes and blocks the expression of leukemogenic genes. Epizyme is developing pinometostat, a small molecule inhibitor of DOT1L, for the treatment of patients with MLL-r, a genetically defined acute leukemia. Epizyme is conducting a phase 1 clinical trial in pediatric patients. Epizyme is evaluating preclinical combinations of pinometostat with other anti-cancer agents in MLL-r leukemia. Pinometostat is being developed in collaboration with Celgene. Epizyme retains all U.S. rights to pinometostat and has granted Celgene an exclusive license to pinometostat outside of the U.S.