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Dr. Tefferi is a world-renowned expert in the treatment ofmyeloproliferative disorders (MPDs) and has authored over 500 researchpublications and given many hundreds of invited lectures in haematology. Healso serves on the editorial boards of a number of leading haematologyjournals.
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Myelofibrosis patients suffer a bone marrow disorder in which the marrowis replaced by fibrous scar tissue. Patients will be treated in this studywith Cytopia's novel orally available compound CYT387. This drug inhibits themutant JAK2 enzyme to specifically treat haematological malignancies,including myelofibrosis.
Many of these particular diseases can lead to life threatening conditionssuch as leukemia. There are currently no effective long term treatments forthese conditions.
"Cytopia is delighted that a clinician of Dr. Tefferi's standing hasagreed to chair our Phase I/II program," said Mr. Andrew Macdonald, CEO. "Dr.Tefferi and our other clinical investigators will study and document theclinical benefits of using this highly promising JAK2 inhibitor to treat theseblood disorders."
Following the completion of very encouraging in-vivo studies at OregonHealth and Science University early in the year, CYT387 has been undergoingrigorous IND-enabling toxicology studies in recent months. It is anticipatedthat an Investigational New Drug application will be lodged with the US Foodand Drug Administration by the end of the year to support studies in Australiaand the United States.
About JAK2
The discovery of a specific single activating mutation in the JAK2 enzymein MPDs in 2005 has focused attention on developing a therapy for thesediseases through selective inhibition of JAK2. To successfully address thesechronic diseases with a JAK2 inhibitor, the specificity and resultanttolerability profile is a key element of the product profile required for a"best in class" inhibitor.
Cytopia is a world leader in JAK2 kinase chemistry and holds a broad rangeof JAK2 related patents. The main area of focus for the JAK2 program has beenthe development of orally available JAK2 inhibitors to treatmyeloproliferative disorders, which include myelofibrosis, polycythemia vera(PV) and essential thrombocythemia (ET) CYT387 is a specific JAK2 inhibitorwith excellent potential for safe and efficacious chronic human dosing. It hasbeen derived from Cytopia's knowledge of basic biology of JAK kinases, astrong structural biology program that has delivered multiple co-crystalstructures of JAK2 complexed with inhibitors, and the subsequent developmentof an extensive chemical library of JAK2 inhibitors through an integratedcomputational and medicinal chemistry platform.
Cytopia is developing a suite of JAK2 inhibitors for multiple indicationsincluding the treatment of certain cancers, particularly lymphomas and solidtumours where JAK2 has been shown to be upregulated, and for cardiovasculardiseases such as pulmonary hypertension.
About Cytopia
Cytopia Ltd is an Australian biotechnology company focused on thediscovery and development of new drugs to treat cancer and other diseases.Cytopia conducts its research and drug development through subsidiaries basedin Melbourne, Australia and San Francisco, USA and specializes in developingnew small molecule compounds with an improved therapeutic profile for thetreatment of cancer.
The lead program for the company is CYT997, a vascular disrupting agent(VDA) for the treatment of various cancers, and currently being trialed inPhase 1 and Phase 2 clinical studies. Cytopia also is building on its range ofJAK inhibitors and kinase e
