Two down, 94 to go
CAMBRIDGE, Mass.ŚEpizyme and Celgene International SÓrl, a subsidiary of Celgene Corp., have entered into a strategic partnership to discover, develop and commercialize personalized therapeutics for patients with genetically-defined cancers by inhibiting histone methyltransferases (HMTs), an important epigenetic target class with 96 known members. Epizyme has developed targets for 20 HMTs to date, states Jason Rhodes, the company's executive vice president and chief business officer.
Celgene is its third partnership agreement, Rhodes notes, with GlaxoSmithKline PLC and Eisai preceding Celgene.
Under the terms of the agreement, Celgene receives the exclusive option to license ex-U.S. rights to Epizyme's available HMT inhibitor programs during an initial three-year period and has the right to extend this option period for one year with additional funding. Epizyme retains all U.S. rights to the collaboration programs and receives a $90 million upfront payment, which includes an equity investment. For each HMT inhibitor that Celgene licenses, Epizyme is eligible to earn more than $160 million in milestone payments and up to double-digit royalties on ex-U.S. sales.
Epizyme and Celgene will work jointly to discover and develop HMT inhibitors and will co-fund global development of the collaboration programs. The collaboration includes Epizyme's DOT1L HMT inhibitor program, to which Celgene licenses the ex-U.S. rights at signing. DOT1L is an oncogenic driver gene in a subtype of acute leukemias called mixed lineage leukemia (MLL). The DOT1L program is currently in preclinical development and a companion diagnostic test already exists. Epizyme expects to have human data on the DOT1L program, as well as its EZH2 target for non-Hodgkin's lymphoma (in collaboration with Eisai), in 12 to 18 months. Celgene was eager to have human proof of concept early on and the DOT1L program, which is Epizyme's most advanced, fit the bill.
Four-year-old Epizyme's strategy is to build a new biopharma company based on personalized therapeutics for patients with genetically defined cancers. According to Bob Copeland, Epizyme's chief scientific officer, the Celgene partnership is transformational, enabling Epizyme to significantly expand the scope of its R&D efforts while retaining U.S. rights where the company plans to commercialize its drug candidates independently.
"There were several things about Celgene that were attractive to us," he says. "Notably, their strong commitment to oncology, personalized medicine and epigenetics. When our CEO Bob Gould visited them, a one-hour presentation became three and a half. We took that as a very good sign."
Also, Rhodes notes, "Our two companies have complementary skill sets; ours in discovery and development, and Celgene's in later stages. With this agreement, we gained all the benefits of partnership while retaining U.S. rights and our independence, as well as tremendous access to capital. Through this collaboration, Epizyme gains access to Celgene's leading drug development resources, enabling us to substantially increase the breadth and depth of our efforts, while retaining U.S. rights to our pipeline of personalized therapeutics."
"Celgene is a leader in epigenetic therapies for cancer through our existing drugs, and continues to focus on delivering new drugs with high therapeutic impact in this area," says Dr. Thomas Daniel, president of research at Celgene. "Epizyme's platform, scientific leadership in histone methyltransferases and leading position on promising HMT targets offer an exciting complementary approach. Our collaboration with Epizyme is a key element of our strategy to develop new and innovative therapeutic paradigms."
Copeland notes that Epizyme's HMT inhibitor platform is extremely robust and that after DOT1L and EZH2, the other 94 members of the class will follow in its efforts to discover and develop small-molecule HMT inhibitors. Genetic alterations in 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, he states.
Celgene International SÓrl, located in Boudry in the Canton area of NeuchÔtel, Switzerland, is a wholly owned subsidiary of Celgene. Headquartered in Summit, N.J., Celgene is an integrated global pharmaceutical company engaged primarily in the discovery, development and commercialization of innovative therapies for the treatment of cancer and inflammatory diseases through gene and protein regulation.