ET is a rare blood cancer resulting from the overproduction of platelets which increases the risk of blood clots and bleeding. It is one of the MPN family of rare bone marrow diseases, and affects approximately 80,000 – 100,000 patients in the U.S. There is a significant unmet need for new ET therapies that can effectively reduce patient platelet levels without general bone marrow suppression, while slowing the progression of the underlying disease. By normalizing elevated platelets, the primary clinical feature of ET, we believe bomedemstat can address an unmet need in the 20% of the high risk ET patients who are intolerant or resistant to hydroxyurea, the current standard-of-care for those patients with ET at greatest risk for blood clots.
Our preliminary data from the ongoing Phase 2 study demonstrates bomedemstat is well-tolerated and a significant proportion of patients have achieved a normal platelet count within the first eight weeks of treatment. We are continuing to enroll patients in this trial and expect to complete enrollment in 2021.
Bomedemstat has received Orphan Drug and Fast Track Designations for ET from the FDA. For more information about our current clinical trials investigating bomedemstat for ET, visit our patients page.