Diseases We Target

Starting with Cancer, We Are Going After Diseases that Have Largely Eluded Other Targeted Approaches

We have three investigational medicines – one for genomically defined subsets of patients with higher-risk myelodysplastic syndrome (MDS) and acute myeloid leukemia (AML), another for acute promyelocytic leukemia (APL), and the third for patients with pancreatic cancer and BRAF-mutant colorectal cancer. We are also building a pipeline of programs in earlier stages of research for cancer, as well as genetic diseases, that lack effective treatment options.

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 who are RARA-positive. In these patients, abnormally high expression of the RARA gene contributes to their disease. We estimate that about 30 percent of AML patients are RARA-positive.

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.

Myelodysplastic Syndrome (MDS)

Myelodysplastic syndrome (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 about a third of patients being considered at higher risk of progressing to AML. Low blood cell counts, referred to as cytopenias, are a hallmark feature of MDS and are responsible for some of the symptoms that MDS patients experience, including infection, anemia, spontaneous bleeding, or easy bruising. Approved therapies for higher-risk MDS offer limited efficacy, underscoring the need for better treatment options. We are focused on a subset of higher-risk MDS patients who are RARA-positive. In these patients, abnormally high expression of the RARA gene contributes to their disease. We estimate about 50 percent of MDS patients are RARA-positive.


Colorectal cancer (CRC) is one of the leading causes of mortality and morbidity in the world. Although early diagnosis has significantly improved outcomes, many patients aren’t diagnosed until the cancer has spread, or metastasized, to other parts of the body, making it very difficult to treat. Adding to the complexity is 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. This is 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. For patients whose cancer has spread, or metastasized, there is a lack of effective treatments and a poor prognosis, underscoring the need for new targeted treatment options.

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