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FDA Grants Orphan Drug Designation to Syros’ Investigational Treatment for Acute Myeloid Leukemia

CAMBRIDGE, Mass.–(BUSINESS WIRE)–Syros Pharmaceuticals (NASDAQ: SYRS), a biopharmaceutical company pioneering the discovery and development of medicines to control the expression of disease-driving genes, today announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to SY-1425 for the treatment of acute myeloid leukemia (AML). SY-1425, an oral first-in-class selective retinoic acid receptor alpha (RARα) agonist, is currently in a Phase 2 clinical trial in genomically defined subsets of patients with AML and myelodysplastic syndrome (MDS).

“Treatment of AML remains a significant unmet medical need, with many patients lacking adequate therapeutic options,” said David A. Roth, M.D., Syros’ Chief Medical Officer. “We believe that SY-1425 may provide a meaningful benefit for subsets of AML patients whose disease is driven by abnormally high expression of the RARA or IRF8 genes. Receiving orphan drug designation is an important regulatory milestone in the development of SY-1425. We’re pleased with the continued progress of the ongoing Phase 2 clinical trial, and we look forward to presenting initial clinical data in the fourth quarter of this year.”

The FDA’s Office of Orphan Drug Products grants orphan status to support development of medicines for the treatment of rare diseases that affect fewer than 200,000 people in the United States. Orphan drug designation may provide certain benefits, including a seven-year period of market exclusivity if the drug is approved, tax credits for qualified clinical trials and an exemption from FDA application fees.

Using its gene control platform, Syros discovered subsets of AML and MDS patients with super-enhancers associated with RARA or IRF8. Syros identified proprietary biomarkers related to these super-enhancers. These super-enhancers are believed to drive overexpression of the RARA or IRF8 genes, locking cells in an immature, undifferentiated and proliferative state, leading to disease. In preclinical studies, SY-1425 promoted differentiation of AML cells with high RARA or IRF8 expression and inhibited tumor growth and prolonged survival in patient-derived xenograft models of AML with high RARA expression. Syros estimates that about one-third of AML and MDS patients have either the RARA or IRF8 biomarker, or both.

The ongoing Phase 2 clinical trial of SY-1425 is assessing the safety and efficacy of SY-1425 as a single agent in four AML and MDS patient populations, as well as in combination with azacitidine, a standard-of-care therapy, in newly diagnosed AML patients who are not suitable candidates for standard chemotherapy. All patients in the trial are prospectively selected using biomarkers for high expression of RARA or IRF8. Additional details about the trial can be found using the identifier NCT02807558 at www.clinicaltrials.gov.

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, Syros’ gene control platform has broad potential to create medicines that achieve profound and durable benefit across a range of diseases. Syros is currently focused on cancer and immune-mediated diseases and is advancing a growing pipeline of gene control medicines. Syros’ lead drug candidates are SY-1425, a selective RARα agonist in a Phase 2 clinical trial for genomically defined subsets of patients with acute myeloid leukemia and myelodysplastic syndrome, and SY-1365, a selective CDK7 inhibitor in a Phase 1 clinical trial for patients with advanced solid tumors, including transcriptionally dependent cancers such as triple negative breast, small cell lung and ovarian cancers. Led by a team with deep experience in drug discovery, development and commercialization, Syros is located in Cambridge, Mass.

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