Diseases We Target

We Are Going After Diseases that Have Largely Eluded Other Targeted Approaches

We are advancing two clinical-stage drug candidates, tamibarotene and SY-2101, across three genomically defined patient populations in higher-risk myelodysplastic syndrome (HR-MDS), acute myeloid leukemia (AML) and acute promyelocytic leukemia (APL). In addition, we are evaluating SY-5609, a highly selective and potent oral inhibitor of cyclin-dependent kinase 7, or CDK7, as a single agent in patients with select solid tumors and in combination with gemcitabine, a chemotherapy, in pancreatic cancer patients in a Phase 1 clinical trial. SY-5609 is also being evaluated in combination with atezolizumab, a PD-L1 inhibitor, in BRAF-mutant colorectal cancer in an arm of a Phase 1/1b clinical trial sponsored by F. Hoffmann-La Roche AG, or Roche, which is actively enrolling.

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.

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.

Acute Promyelocytic Leukemia (APL)

Acute promyelocytic leukemia (APL) is a subtype of acute myeloid leukemia (AML) that is defined by a fusion of the RARA and PML genes. It represents about 10 percent of AML cases. In APL, there are too many immature blood-forming cells, known as promyelocytes, in the blood and bone marrow. This buildup of promyelocytes leads to a shortage of normal blood cells and platelets in the body. Signs and symptoms of APL include an increased risk of bleeding, infection and fatigue. Intravenous arsenic trioxide (IV ATO), in combination with oral all-trans retinoic acid (ATRA), cures most patients but is extremely burdensome, requiring regular lengthy infusions over nearly a year of treatment, highlighting the need for a more convenient oral medicine.


Colorectal cancer (CRC) is one of the world’s leading causes of morbidity and mortality. Although early diagnosis has significantly improved patient outcomes, CRC remains very difficult to treat, as many patients aren’t diagnosed until the cancer has metastasized to other parts of the body. Treatment difficulty is compounded by the fact that different genetic mutations cause the cancer to grow in different patients.

Mutations in the BRAF gene, which are found in about 10% of patients with metastatic CRC, are associated with a poor prognosis, underscoring the need for better treatment options for these patients.

Pancreatic Cancer

Pancreatic cancer is a particularly aggressive and difficult-to-treat form of cancer in part because most patients do not exhibit symptoms until the cancer has reached an advanced stage. Currently, pancreatic cancer is treated with surgery, chemotherapy, radiation, and, more recently, targeted therapies and immunotherapies.  Patients whose cancer has metastasized have a poor prognosis, and they may face a lack of effective treatments, which underscores the need for new targeted treatment options.

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