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The Leukemia & Lymphoma Society and Epizyme Partner to Develop Personalized Therapeutic for Genetically-Defined Mixed Lineage Leukemia (MLL) Patients

The Leukemia & Lymphoma Society (LLS) and Epizyme announced today that they will jointly support pre-clinical and Phase 1 development of Epizyme’s DOT1L-targeted histone methyltransferase inhibitor (HMTi) for Mixed Lineage Leukemia (MLL), a devastating acute leukemia that affects both pediatric and adult patients. LLS has committed to provide up to $7.5 million in milestone-based funding to Epizyme to support the program through Phase 1. Epizyme is creating personalized therapeutics for genetically-defined oncology and rare disease patients, with a focus on small molecule inhibitors of histone methyltransferases, an important epigenetic target class.

"MLL, a genetically-defined subtype of both ALL and AML, is an acute leukemia with high unmet need and over 1500 new patients each year in the U.S. alone," said Richard Winneker, Senior Vice President, Research at LLS. "LLS is committed to finding cures for all blood cancers, including acute leukemias of this type, and Epizyme has demonstrated strong progress to date in developing a DOT1L HMTi as a personalized therapeutic for MLL patients.”

“Epizyme’s mission is to develop personalized therapeutics for genetically-defined patients,” said Robert Copeland, Ph.D., Executive Vice President and Chief Scientific Officer at Epizyme. “Our DOT1L HMTi is an important therapeutic program for genetically-defined leukemia patients and is a clear example of our focus on developing innovative personalized therapeutics for patients in need. We are thrilled to have LLS’ support and to work with them closely to move our DOT1L program ahead.”

Bruce Chabner, M.D., the Director for Clinical Research at the Massachusetts General Hospital Cancer Center, former head of the Division of Cancer Treatment at the National Cancer Institute, and Epizyme SAB member, added, “Epizyme has made extraordinary progress in advancing the first personalized epigenetic therapy towards a clinical trial. There is a clear unmet need for improving the treatment of MLL patients. Existing diagnostic tests identify these patients, but current therapies are not designed to attack the driver mutation in histone methylation. There is great hope and enthusiasm for personalized therapeutics that address epigenetic targets.”

About The Leukemia & Lymphoma Society

The Leukemia & Lymphoma Society® (LLS) is the world's largest voluntary health agency dedicated to blood cancer. The LLS mission: Cure leukemia, lymphoma, Hodgkin's disease and multiple myeloma, and improve the quality of life of patients and their families. LLS funds lifesaving blood cancer research around the world and provides free information and support services. Founded in 1949 and headquartered in White Plains, NY, LLS has chapters throughout the United States and Canada. To learn more, visit or contact the Information Resource Center at (800) 955-4572.

About Epizyme

Epizyme is leading the discovery and development of small molecule histone methyltransferase (HMT) inhibitors, a new class of targeted therapeutics for the treatment of genetically-defined cancer patients based on breakthroughs in the field of epigenetics. Genetic alterations in the HMTs are strongly associated with the underlying causes of multiple human diseases, including cancer. Epizyme’s patient-driven approach represents the future of personalized therapeutics by creating better medicines for the right patients more quickly and at lower cost than traditional approaches.


For LLS:
Andrea Greif, 914-821-8958
For Epizyme:
MacDougall Biomedical Communications
Jennifer Conrad or Chris Erdman, 781-235-3060

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