Deal gives Celgene license to lead DOT1L candidate and option to other HTM inhibitors.

Celgene is paying Epizyme $90 million up front to established a partnership focused on the discovery, development, and commercialization of histone methyltransferase (HMT)-inhibiting drugs for genetically defined cancers. The up-front fee includes an equity investment in Epizyme and gives Celgene exclusive immediate ex-U.S. rights to its partner’s existing DOT1L HMT inhibitor program against mixed lineage leukemia (MLL).

Under terms of the deal Celgene also retains an exclusive option to license ex-U.S. rights to other Epizyme HMT inhibitor programs over an initial three-year period. Epizyme retains all U.S. rights to resulting products, and could earn up to another $160 million in milestones for each HMT inhibitor licensed by Celgene, and potentially double-digit royalties on ex-U.S. sales. The firms will work jointly to discover and develop HMT inhibitors, and will co-fund global development of collaborative programs.

Celgene’s expertise in epigenetic cancer therapies and Epizyme’s histone myethyltransferase platform are highly complementary, the partners claim. “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 therapies,” comments Robert Gould, Ph.D., Epizyme CEO.  

Epizyme is focused on the discovery and development of small molecule HMT inhibitors. The firm’s initial therapeutic target is MLL, a form of acute leukemia characterized by the presence of a chromosomal alteration (MLL-translocation) that recruits DOT1L activity to aberrant gene locations, leading to increased expression of specific gene products that drive leukemia cell proliferation. The small molecule DOT1L inhibitor is designed to selectively kill MLL cells, while sparing cells that don’t carry the MLL-translocation.

DOT1L is being developed in collaboration with the Leukemia and Lymphoma Society. Last month Eipzyme reported achieving a preclinical milestone in the program, and said it has to date received $2.6 million of potentially $7.5 million in funding under the partnership to support the DOT1L program through Phase I development.

The firm’s second lead program, EZH2, is in development in partnership with Eisai for the treatment of certain nonHodgkin lymphomas and breast cancer subtypes. The compound is designed to target the catalytic center of a multiprotein complex known as polycomb repressive complex 2 (PRC2). Epizyme and Eisai inked their deal for the EZH2 program back in March 2011. 

Previous articlePierre Fabre, Forest Report Positive Data from Third Phase III Depression Trial
Next articlePlus Diagnostics to Co-Market MDxHealth’s ConfirmMDx Prostate Cancer Test in the U.S.