Epizyme is developing pinometostat, a small molecule inhibitor of DOT1L, for the treatment of patients with MLL-r, a genetically defined acute leukemia. We are conducting a phase 1 clinical trial in pediatric patients. We are also 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.