Rare Disease Report

Targeted AML Treatment Gets FDA Orphan Drug Designation

AUGUST 21, 2017
Mathew Shanley
Today, Syros Pharmaceuticals announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to its leading drug candidate SY-1425, intended to treat acute myeloid leukemia (AML).

Currently in a Phase 2 clinical trial, SY-1425 is an oral first-in-class selective retinoic acid receptor alpha (RARα) agonist. The study is assessing the safety and efficacy of the drug in subsets of patients with AML and myelodysplastic syndrome (MDS).

AML, a cancer of the blood and bone marrow, progresses almost instantaneously and is characterized by the rapid growth of white blood cells. When expanded, these cells obstruct the production of typical blood cells.

It is estimated that one-third of AML and MDS patients have either the RARA or IRF8 biomarker, or both.

“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,” said David A. Roth, M.D., Syros’ Chief Medical Officer in a press release. 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.”

Through the utilization of its gene control platform, the biopharmaceutical company discovered subsets of patients with AML and MDS that have super-enhancers associated with RARA or IRF8 genes. These super-enhancers lock cells in an undeveloped, homogenous and proliferative state, often leading to disease.

In preclinical studies, SY-1425 promoted differentiation of AML cells with high RARA or IRF8 expression and inhibited tumor growth and improved overall survival in patient-derived xenograft models of AML with high RARA expression.

The ongoing Phase 2 study is evaluating SY-1425 as a single agent in 4 AML and MDS patient populations, and as a combination therapy with azacitidine in newly-diagnosed AML patients who are not eligible for chemotherapy or radiation.

Patients enrolled in the trial are selected using biomarkers for high expression of RARA or IRF8. Additional details about the trial can be found here: NCT02807558.
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