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CAMBRIDGE, Mass.--(BUSINESS WIRE)--Syros Pharmaceuticals today announced that SY-1425, a potent and selective retinoic acid receptor alpha (RARα) agonist, was observed to inhibit the growth of cancer cells and prolong survival in in vivo models of acute myeloid leukemia (AML) with a novel RARA biomarker discovered by the Company. The biomarker was found in approximately 25 percent of AML and myelodysplastic syndrome (MDS) patient tissue samples. These data were presented at the American Association of Cancer Research (AACR) Annual Meeting in New Orleans.

“SY-1425 represents a promising therapeutic option for novel genomically defined subsets of AML and MDS patients” Tweet this “SY-1425 represents a promising therapeutic option for novel genomically defined subsets of AML and MDS patients,” said Nancy Simonian, MD, Chief Executive Officer of Syros. “Based on our preclinical data, which show a strong link between our biomarker and response to treatment with SY-1425, we are committed to rapidly advancing this first-in-class therapy for these currently underserved patients. Positive results from a Phase 2 clinical study would not only be beneficial to patients but also validate our pioneering approach of analyzing the non-coding region of DNA to advance the field of genomics-based medicine.”

The data presented at AACR shows that the RARA biomarker discovered by Syros is predictive of response to treatment with SY-1425 in AML cell lines and patient-derived xenograft (PDX) models of AML. Treatment with SY-1425 was observed to inhibit cancer growth and prolong survival in PDX models of AML with the RARA biomarker but not in models of AML without the biomarker. Highlights of the data include:

Using its gene control platform, Syros identified a specialized regulatory region of non-coding DNA known as a super-enhancer that is associated with the RARA gene in subsets of AML and MDS patients. The super-enhancer is believed to lock cells in an immature, proliferative state. Treatment with SY-1425 in cancer cells with the RARA super-enhancer appears to promote differentiation of these cells. Syros in-licensed SY-1425, which is approved in Japan as tamibarotene to treat acute promyelocytic leukemia, to develop and commercialize SY-1425 in North America and Europe for all cancers.

Syros plans to file an Investigational New Drug (IND) application with the U.S. Food and Drug Administration and initiate a Phase 2 clinical trial of SY-1425 in mid-2016 in subsets of AML and MDS patients with the RARA biomarker. The Company continues to research the role of the RARA super-enhancer in other cancers and plans to pursue additional indications upon achieving proof-of-concept in AML and MDS.

About Syros Pharmaceuticals

Syros Pharmaceuticals is pioneering the understanding of the non-coding region of the genome to advance a new wave of medicines that control expression of disease-driving genes. Syros has built a proprietary platform that is designed to systematically and efficiently analyze this unexploited region of DNA in human disease tissue to identify and drug novel targets linked to genomically defined patient populations. Because gene expression is fundamental to the function of all cells, the Company’s gene control platform has broad potential to achieve profound and durable benefit across a range of diseases. Syros is focused on cancer and immune-mediated diseases and is advancing a growing pipeline, including its lead drug candidates SY-1425, a selective RARα agonist for genomically defined subsets of patients identified by its platform, for a range of cancers including acute myeloid leukemia and myelodysplastic syndrome, and SY-1365, a selective CDK7 inhibitor for a range of blood cancers and solid tumors. Led by a team with deep experience in drug discovery, development and commercialization, Syros is located in Cambridge, Mass.