Epizyme commences EPZ-5676 Phase 1 study for MLL-r leukemia

Epizyme, Inc., a company leading the creation of a new class of personalized therapeutics for patients with genetically defined cancers, announced today that it has initiated a Phase 1 Study of EPZ-5676. EPZ-5676 is a novel small molecule inhibitor of DOT1L, a histone methyltransferase (HMT) that is critical to the development of a specific type of acute leukemia defined by rearrangement of the MLL gene (MLL-r leukemia). The Phase 1 study will evaluate the safety, pharmacokinetics, and pharmacodynamics of escalating doses of EPZ-5676 and will also provide a preliminary assessment of efficacy in a cohort of patients with MLL-r leukemia. Epizyme anticipates that approximately 40 patients will be accrued to this Phase 1 study.

“Acute leukemia with MLL rearrangement can occur in both adults and children, usually with devastating outcomes”

"Acute leukemia with MLL rearrangement can occur in both adults and children, usually with devastating outcomes," said Dr. Eric Hedrick, Chief Medical Officer at Epizyme. "Patients with this type of leukemia are already identified using existing diagnostics. The medical community has a high level of awareness about this disease and the need to develop more specific and effective treatments for these patients. We are very excited to begin the clinical evaluation of EPZ-5676, which has been designed to block the specific abnormality that causes this genetically defined type of leukemia."

"EPZ-5676 is the first histone methyltransferase inhibitor (HMTi) to enter human clinical development. It is an important potential therapeutic for MLL-r patients and a significant milestone for the entire field. It is also an example of Epizyme's approach to creating personalized therapeutics for patients with genetically defined cancers," said Dr. Robert Gould, Chief Executive Officer and President at Epizyme.

Epizyme retains all US development and commercialization rights to its DOT1L inhibitor program, including EPZ-5676. The program is partnered with Celgene outside of the United States.