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 select solid tumors. 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. As in AML, about 30 percent of MDS patients are RARA-positive.


Breast Cancer

Breast cancer is the most common type of cancer in women, and despite advances in treatment, there remains a need for new therapies for different types and stages of the disease. Most breast cancer patients have a form of the disease known as hormone receptor-positive, or HR-positive, breast cancer. If caught early, the outlook for these patients is generally good, and for patients with metastatic HR-positive breast cancer, hormone-based therapies, together with a new class of drugs known as CDK4/6 inhibitors, have improved survival outcomes. Patients eventually relapse, however, and second-line therapies have limited efficacy. Triple-negative breast cancer represents another area of ongoing unmet need within breast cancer. These patients don’t respond to either hormonal therapies or anti-HER2 therapies, leaving them with limited treatment options.


Colorectal Cancer

Colorectal cancer starts in either the colon or the rectum. It is the third most commonly diagnosed cancer in men and women. Early screening and treatment have significantly improved outcomes in recent decades. For patients with metastatic colorectal cancer, however, there remains a significant need for better treatments. Therapies targeted towards specific mutations such as BRAF or KRAS are currently being investigated in clinical trials, but chemotherapy remains the standard of care.


Lung Cancer

Lung cancer is the second most common cancer in men and women. The two main types of lung cancer are small cell lung cancer (SCLC) and non-small cell lung cancer (NSCLC). Treatment options and outlook vary depending on the type of lung cancer and stage of the disease. Patients with NSCLC may be treated with a combination of surgery, chemotherapy, radiation, and targeted therapies. For SCLC, chemotherapy and radiation remain the mainstays of treatment. While SCLC is less common, it is typically more aggressive than NSCLC and patients are often diagnosed after the cancer has already started to spread, further underscoring the need for new treatment options.


Ovarian Cancer

Ovarian cancer is the fifth leading cause of cancer-related deaths among women. If caught in the early stages, ovarian cancer is often successfully treated with surgery, chemotherapy and radiation. Common symptoms of ovarian cancer include bloating, abdominal pain, trouble eating or feeling full quickly, and urinary symptoms. These symptoms are often attributed to other causes, however, meaning many women are not diagnosed until after the cancer has begun to spread and becomes markedly more difficult to treat. Chemotherapy remains the standard of care. Despite the emergence of some targeted therapies for ovarian cancer, there remains a need for better treatment options.


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. There continues to be a need for better treatment options to improve the prognosis for patients with pancreatic cancer, with a focus on new therapies that target specific genetic mutations and pathway alterations that contribute to the cancer-causing processes in cells.

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