CTI BioPharma presents data from Phase 3 trial of pacritinib in AML patients at ASH 2014

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CTI BioPharma Corp. (CTI) (NASDAQ and MTA: CTIC) today announced data showing treatment with pacritinib, an investigational oral multikinase inhibitor in Phase 3 clinical development, preferentially killed acute myeloid leukemia (AML) cells with FLT3 mutations, overcame stromal protection and suppressed leukemic outgrowth from stroma adherent AML cells in both medium-term (7-14 days) and long-term (5-6 weeks) assays. The findings from this study were presented by Dr. Ceri Marrin, Consultant Haematologist, University Hospital of Wales at Cardiff University, during an oral presentation at the 56th American Society of Hematology (ASH) Annual Meeting and Exposition held December 6-9 in San Francisco, CA.

"About 30 percent of patients with AML have a mutation in FLT3, a known poor prognostic factor, and an important target for drug development. Traditional FLT3 inhibitors are unable to kill FLT3 mutated cells when grown on stromal cells of the microenvironment, and this is believed to be an important resistance factor for other FLT3 antagonists. Stromal interaction confers protection through upregulation of alternative survival pathways including MEK and JAK2. The current data show that pacritinib is able to evade this mechanism of resistance, likely through its ability to suppress other signaling pathways, such as JAK2," said Alan Burnett, M.D., Past Professor and Head of Haematology, Department of Medical Genetics, Haematology and Pathology at the School of Medicine at Cardiff University. "While the data indicates that pacritinib was able to suppress growth of AML cells as a single agent, the synergistic effect of treatment with pacritinib in combination with either cytarabine or a MEK inhibitor suggest interesting directions for future clinical evaluation."

"This study provides further evidence that pacritinib inhibits signaling pathways that are important to a broad spectrum of blood-related cancers. We believe pacritinib has the potential to be a significant new treatment option for AML given its ability to target multiple pathways, including suppression of microenvironmental tumor interactions that are key to overcoming longer-term drug resistance in this disease," said James A Bianco, M.D., President and CEO of CTI. "A Phase 2 trial in patients with relapsed AML and FLT3 mutations is currently underway and a first-line study in elderly patients with AML is expected to be initiated in the near future."

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