Per the terms of the agreement, Celgene will receive an exclusive option to license ex-U.S. rights to Epizyme’s available HMT inhibitor programs during an initial three-year period. In addition, Celgene also gains the right to extend its option period for a year with additional funding. As a result of the strategic partnership, Epizyme will receive an upfront payment of $90 million, which includes an equity investment. For each HMT inhibitor that Celgene licenses through the partnership, Epizyme stands to receive more than $160 million in milestone payments, as well as up to double-digit royalties on ex-U.S. sales. The two companies will be working together to develop the HMT inhibitors and will both contribute funding for global development of the programs.

 

“Our Celgene partnership is a transformational step in Epizyme’s growth and is made possible by Celgene’s vision and commitment to patients,” Robert Gould, Ph.D., CEO and president of Epizyme, said in a press release. “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 US rights to our pipeline of personalized therapeutics.”

 

HMTs are a class of epigenetic enzymes, with 96 members total, many of which have been associated with cancer and other serious diseases. Targeting HMTs represents a new approach to affect pathways of disease-causing gene expression. Adding small chemical units such as methyl groups at specific locations on a histone can alter the structure of the chromatin and signal cells to run transcription on or off for certain genes, Epizyme notes on its site. “The selective addition of methyl groups to specific sites on the histones is controlled by the action of a unique class of enzymes known as the histone methyltransferases (HMTs). Once the methyl group has been deposited on the histone site, the affected genes continue to be regulated (turned on or off) until this chemical unit is removed by other enzymes, known as histone demethylases,” it adds, meaning that HMTs could provide long-lasting modification of gene expression.  

 

The partnership will make use of Epizyme’s HMT inhibitor platform, including its DOT1L HMT inhibitor program, which is in preclinical development targeted at treating mixed lineage leukemia. Celgene licensed the ex-U.S. rights to the program at the partnership’s signing.

 

“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,” Thomas Daniel, M.D., president of Research for Celgene, said in a press release regarding the agreement. “Epizyme's platform, scientific leadership in histone methyltransferases and leading position on promising HMT targets offers an exciting complementary approach. Our collaboration with Epizyme is a key element of our strategy to develop new and innovative therapeutic paradigms.”