Syros Pharmaceuticals, Inc. (NASDAQ: SYRS) saw its shares rise in premarket trading Monday after the Cambridge, Mass-based company announced a critical update that it received from the U.S. Food and Drug Administration. Specifically, the agency has granted orphan drug designation to SY-1425 for the treatment of acute myeloid leukemia (AML).

Orphan status supports the development of medicines for the treatment of rare diseases that affect fewer than 200,000 people in the United States. Also 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.

In preclinical studies, SY-1425 promoted differentiation of AML cells 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 (myelodysplastic syndrome) patients have either the RARA or IRF8 biomarker, or both.

The current and 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.

David A. Roth, M.D., Syros’ Chief Medical Officer, commented:

“Treatment of AML remains a significant unmet medical need, with many patients lacking adequate therapeutic options. 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.”

Shares of Syros closed Friday at $20.17, with a consensus analyst price target of $26.50 and a 52-week range of $10.22 to $24.38. Following the release of the earnings report, the stock was last seen up 4% at $21.00 in early trading indications Monday.

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