Epizyme, Inc., a clinical stage biopharmaceutical company, engages in the discovery, development, planning, and commercialization of personalized therapeutics for the treatment of patients with genetically defined cancers. The company has built a proprietary product platform that it uses to create small molecule inhibitors of a 96-member class of enzymes known as histone methyl transferases (HMTs). HMTs are part of the system of gene regulation, referred to as epigenetics, that controls gene expression. As of December 31, 2013, the company had three collaboration partners, Celgene Corporation (Celgene), Eisai Co. Ltd. (Eisai), and Glaxo Group Limited (an affiliate of GlaxoSmithKline) (GSK). EPZ-5676—DOT1L Inhibitor EPZ-5676 The company develops EPZ-5676 as an intravenously administered small molecule inhibitor of DOT1L for the treatment of acute leukemias with alterations in the MLL gene, specifically rearrangements of MLL as a consequence of chromosomal translocation, referred to as MLL-r, and partial tandem duplications of the MLL gene, referred to as MLL-PTD. The company initiated a Phase 1 clinical trial of this product candidate in 2012. EPZ-6438—EZH2 Inhibitor The company develops EPZ-6438 as an orally available small molecule inhibitor of EZH2 for the treatment of non-Hodgkin lymphoma patients who have an oncogenic point mutation in EZH2 and for the treatment of certain INI1-deficient solid tumors, such as synovial sarcoma, a soft tissue sarcoma, and malignant rhabdoid tumor, a pediatric cancer. In June 2013, Eisai and the company initiated a Phase 1/2 clinical trial of EPZ-6438. In August 2013, the company was granted orphan drug designation for EPZ-5676 for the treatment of acute myeloid leukemia (AML), and acute lymphoblastic leukemia (ALL), by the United States Food and Drug Administration (FDA), and in January 2014, the European Commission granted orphan drug designation for EPZ-5676 for the treatment of AML and ALL. HMT Collaborations In 2012, the company entered into a collaboration and license agreement with Celgene to discover, develop and commercialize small molecule HMT inhibitors targeting DOT1L and any other targets from its product platform for patients with genetically defined cancers, excluding targets covered by its two other existing collaborations. The company has a collaboration and license agreement with Eisai under which it granted Eisai an exclusive worldwide license to its small molecule HMT inhibitors directed to EZH2, including EPZ-6438, while retaining an opt-in right to co-develop, co-commercialize and share profits with Eisai as to licensed products in the United States. In 2011, the company entered into a collaboration and license agreement with GlaxoSmithKline to discover, develop and commercialize novel small molecule HMT inhibitors directed to available targets from its product platform. The company granted GlaxoSmithKline the option to obtain exclusive worldwide license rights to HMT inhibitors directed to three targets. Intellectual Property In 2013, two U.S. patents covering the composition of matter of DOT1L and EZH2 product candidates were issued. These patents would expire in 2032. History The company was incorporated under the laws of the state of Delaware in 2007.
epizyme inc (EPZM:NASDAQ GS)
400 Technology Square
Cambridge, MA 02139
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