Harvard University will partner with Merck & Co. to develop small-molecule treatments for acute myeloid leukemia (AML) and other cancers, in a $20 million-plus collaboration announced today.

The treatments consist of compounds that were developed in the lab of Harvard investigator Matthew Shair, Ph.D. According to Harvard, Dr. Shair’s therapeutic approach to treating AML consists of inhibiting enzymes that regulate the transcription of genetic programs altered in AML and other cancers.

Merck has agreed to take over development of the candidate therapeutics, including clinical development, and will oversee worldwide commercialization of products. The pharma giant will engage in a research collaboration with Dr. Shair’s laboratory to investigate further the biology of transcriptional regulator enzymes.

Last fall, Dr. Shair led a team of Harvard researchers and other collaborators that published a paper in Nature on a molecule designed to fight AML. In the paper—published online September 28, 2015—Dr. Shair and colleagues showed that cortistatin A, which is isolated from sea sponges and later synthesized in his lab, successfully halted the growth of AML cells in mouse models.

“(We) found that it was as efficacious as any other molecule we had seen, without having deleterious effects,” Dr. Shair said in a statement at the time. “This suggests we have identified a promising new therapeutic approach.”

Dr. Shair’s work has been supported by Harvard’s Blavatnik Biomedical Accelerator. Blavatnik nurtures Harvard’s early-stage life science research, with the goal of promoting the development of biomedical advances that will improve health care and benefit society.

“Accelerator funding over the course of several years has enabled my laboratory to advance some of our experimental compounds to a relatively late stage of preclinical development,” Dr. Shair said in a statement. “It’s gratifying to have discovered a new biological target in the fight against AML, but even more fulfilling to have created a promising weapon against it.”

Merck agreed to pay $20 million upfront to Harvard, which the university said will support future research. Harvard is also eligible for undisclosed payments tied to development and commercialization milestones, as well as tiered royalties on any resulting products.

Dr. Shair is a professor in Harvard’s Department of Chemistry and Chemical Biology.








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