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MPDs are a series of diseases where there is over-production of particularcells within the blood stream. One of these conditions is polycythemia vera(PV) where patients have excess production of red blood cells in the bodywhich can lead to life-threatening conditions such as hemorrhage, thromboticevents (eg stroke) and enlargement of the spleen.
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There are few pharmaceutical treatments for MPD patients and consequentunmet clinical need. Progressive disease can lead MPD patients to developleukemia or myelofibrosis.
Over 95% of patients with PV and about 50% of patients with two relatedMPDs, essential thrombocythemia and primary myelofibrosis, possess a geneticmutation which causes over-activation of the enzyme JAK2. Cytopia isdeveloping selective and potent inhibitors of the JAK2 enzyme to blockexcessive activity.
Cytopia has collaborated with leading laboratories in this field includingthe Royal North Shore Hospital in Sydney, Australia and the Mayo Clinic inCleveland, USA to study the effects of its JAK2 inhibitors in pre-clinicalstudies on MPD patient cells.
The data obtained in these studies show that Cytopia's compounds block themutant JAK2 enzyme and thereby inhibit, in the case of PV, the over-productionof red blood cells. Additionally, when normal human cells were tested in thesame assay, Cytopia's JAK2 inhibitor showed a possible therapeutic window witha greater potency inhibition of disease cells. Extensive screening against apanel of 73 kinase enzymes indicates that CYT387 possesses selectivity forJAK2, potentially minimizing off-target toxicity.
Dr Pardanani of the Mayo clinic said: "In ex vivo studies, we have seenencouraging preliminary results with Cytopia's compounds including theinhibition of erythroid colony growth from PV patients harboring the JAK2V617F mutation."
Cytopia's Research Director Dr Chris Burns stated: "This data is importantvalidation that our selective JAK2 inhibitors stop the proliferation of mutantcells from patients with these diseases at clinically achievable doses."
Cytopia is developing JAK2 inhibitors for the treatment of MPDs and othersolid and blood cancers. In addition, other inhibitors of JAK2 are underdevelopment for cardiovascular and ophthalmic diseases.
About Cytopia
Cytopia Ltd is an Australian headquartered biotechnology company focusedon the discovery and development of new drugs to treat cancer. Cytopiaconducts its research and development via subsidiaries based in Melbourne,Australia and New York and specializes in discovering new molecules that caninhibit enzymes known as kinases, an exciting new class of drugs.www.cytopia.com.au Relevant Links: www.mpdfoundation.org www.mpd-oz.org
SOURCE Cytopia Limited
