We Are Going After Diseases that Have Largely Eluded Other Targeted Approaches
We are advancing our clinical-stage drug candidate, tamibarotene, across two genomically defined patient populations in higher-risk myelodysplastic syndrome (HR-MDS) and acute myeloid leukemia (AML). We have also done substantial work evaluating tamibarotene for the treatment of acute promyelocytic leukemia (APL), but we are no longer investing capital in the program at this time. In addition, SY-5609, a highly selective and potent oral inhibitor of cyclin-dependent kinase 7, or CDK7, was previously in clinical development for pancreatic cancer. Syros is seeking out-licensing opportunities for further development of SY-5609.
Higher-Risk Myelodysplastic Syndrome (HR-MDS)
Higher-risk Myelodysplastic syndrome (HR-MDS) is a bone marrow disorder in which the bone marrow does not produce enough healthy blood cells. It is closely related to AML, with more than half of the patients progressing to AML. Low blood cell counts, referred to as cytopenias, are a hallmark feature of HR-MDS and are responsible for some of the symptoms that HR-MDS patients experience, including infection, anemia, spontaneous bleeding, or easy bruising. Approved therapies for HR-MDS offer limited efficacy, underscoring the need for better treatment options. We are focused on a subset of HR-MDS patients with RARA overexpression. In these patients, abnormally high expression of the RARA gene contributes to their disease. We estimate about 50 percent of MDS patients are positive for RARA overexpression.
- American Cancer Society: Myelodysplastic Syndrome
- Cancer Care: Myelodysplastic Syndrome
- MDS Foundation
- MDS Alliance
- The Aplastic Anemia and MDS International Foundation
Acute Myeloid Leukemia (AML)
Acute myeloid leukemia (AML) is a cancer of the blood and bone marrow. It is the most common type of acute leukemia. Although several new therapies have been introduced to treat AML and a number of investigational therapies are currently in clinical trials, there remains a significant need for additional targeted approaches since AML is driven by many different genomic mutations and alterations in different patients. We are focused on a subset of AML patients with RARA gene overexpression. In these patients, abnormally high expression of the RARA gene contributes to their disease. We estimate that about 30 percent of AML patients are positive for RARA overexpression.