Myelofibrosis (MF) is a progressive cancer in which the bone marrow (the spongy tissue inside bones that helps blood cells grow) is gradually replaced by fibrous, scar-like tissue. This scarring process slowly reduces the marrow’s ability to make blood cells. As a result, the body cannot make enough of the cells that fight infection (white blood cells), carry oxygen (red blood cells) and help clot blood (platelets). Not having enough of these cells can result in anemia (leaving patients feeling weak or tired), infections and bleeding.
As the disease progresses, the body attempts to make blood cells outside the bone marrow, notably in organs such as the spleen and liver, which slowly become enlarged. There are over 50,000 patients with MF in the U.S., E.U. and Japan, many of which have a poor quality of life owing to chronic fatigue, anemia, pain, itching, fever and night sweats. Median survival post-diagnosis is approximately 5 years.
There remains a high unmet need for alternative disease-modifying therapies that improve symptom management. We are evaluating bomedemstat for treatment in patient with myelofibrosis, which has the potential to be a differentiated, first-in-class, disease-modifying therapy for a patient population with very limited therapeutic options. For more information on our Phase 2 clinical trial, visit our clinical study website or clinicaltrials.gov.