TargeGen has announced the successful completion of a 59-patient, multicentre clinical trial to examine TG101348, a new oral, potent and highly selective inhibitor of JAK2 for patients with myelofibrosis.

There are no currently approved specific therapies for myelofibrosis, polycythemia vera or essential thrombocythemia, which are collectively estimated to affect more than 200,000 patients in the US alone.

The current clinical trial was conducted in two phases at six centres in the USA. The first dose-escalation phase enrolled 28 patients, followed by a dose-confirmation phase that enrolled 31 patients.

The primary goals of the open-label, dose-escalation study included identification of a maximum tolerated dose (MTD), characterisation of the safety profile, and evaluation of drug effects of TG101348 on surrogate markers, biomarkers and presumptive clinical endpoints.

The results of this study have been accepted for oral presentation at the American Society of Hematology (ASH) Annual Meeting in New Orleans, 5-8 December 2009.

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