Cytopia selects JAK2 MPDS candidate

Australian biotechnology firm Cytopia has chosen CYT387, a selective and potent JAK2 inhibitor, for formal preclinical development to treat myeloproliferative disorders (MPDs). This follows extensive laboratory experimentation and includes the recent demonstration of activity in an in vivo model of myeloproliferative disease, the company says

MPDs are a group of diseases including myelofibrosis, polycythemia vera and essential thrombocythemia that affect over 150,000 patients in the USA and more than twice that worldwide. Over 95% of PV cases and in excess of 50% of ET and primary and secondary myelofibrosis cases are believed to be caused by a mutation in the JAK2 kinase enzyme. This renders the kinase permanently active and leads to the overproduction of particular blood cells.

No effective treatments exist for these diseases and untreated progressive disease can lead to leukemia or myelofibrosis. CYT387 is said to possess an excellent selectivity profile against a large panel of kinases and other pharmacological targets. It also has very good pharmacokinetics including oral bioavailability and, in preclinical models, has a promising safety profile, according to Cytopia.