Myeloproliferative Diseases Driven by the Activation of Individual Kinases
Myeloproliferative diseases are a group of related diseases characterized by the overproduction of certain blood components such as red blood cells. Polycythemia Vera (PV) is a prototypical example of a myeloproliferative disease caused by the mutation-activated form of a single kinase (JAK2 V617F) that drives the disease. In PV, the JAK2 kinase mutation (V617F) causes patients to overproduce red blood cells. Over time, they may suffer stroke, other thrombotic events and significant enlargement of the spleen as a consequence. Other myeloproliferative diseases that involve the JAK2 (V617F) mutation include Essential Thrombocytopenia (ET) and Myelofibrosis (MF). The prevalence of PV, ET, and MF collectively is estimated at approximately 200,000 patients in the US. There is no specific therapy currently approved for any of these conditions which are associated with significant morbidity and mortality. TargeGen® is developing an internally discovered JAK2 inhibitor (TG101348) which has demonstrated excellent results in published pre-clinical and clinical testing. In October 2009 TargeGen® completed its first human clinical trial with TG101348 in MF patients and Phase II studies are expected to begin in Q3 2010.