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BerGenBio AS Study Published in Blood Highlights Axl Inhibition as a Novel Therapeutic Approach for Acute Myeloid Leukemia

9/30/2013 9:15:12 AM

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Bergen, Norway, September 30, 2013 – BerGenBio AS (“BerGenBio” or the “Company”), an oncology biopharmaceutical company, announces that a paper entitled “Axl, a prognostic and therapeutic target in acute myeloid leukemia mediates paracrine cross-talk of leukemia cells with bone marrow stroma”1 by Ben-Batalla et al., has been published in Blood, the most cited peer-reviewed research journal in the field of hematology.

The paper reports research directed by Dr. Sonja Loges from the Department of Hematology, Oncology and Bone Marrow Transplantation with Section Pneumology and from the Institute of Tumor Biology, University Medical Center Hamburg-Eppendorf, that identifies a significant role for the Axl receptor in the progression and therapeutic resistance of AML. The Hamburg team’s results reveal a novel Axl-dependent mechanism where AML cells evade treatment by establishing a protective niche in the bone marrow. Growth Arrest-specific protein 6 (Gas6) is released by bone marrow cells and facilitates AML cell proliferation, survival and resistance by activating the Axl receptor. Blocking Axl activity by the selective inhibitor BGB324 is shown to reduce AML proliferation and induce apoptosis (programmed cell death), leading to enhanced survival in preclinical models. These studies, in part sponsored by BerGenBio, support the development of BGB324 as a novel treatment of Axl-expressing AML.

In June 2013, BerGenBio announced that BGB324 had begun a Phase 1 clinical trial designed to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of the drug.

Sonja Loges, MD PhD, Group Leader, Department of Hematology, Oncology and Bone Marrow Transplantation with Section Pneumology & Institute of Tumor Biology, University Comprehensive Cancer Center Hamburg, University Medical Center Hamburg-Eppendorf commented: “Our findings detailed in this paper provide evidence for a specific role of the Gas6-Axl axis in the progression of AML. Additionally, we have shown that the selective Axl inhibitor BGB324 reduces proliferation and induces apoptosis of patient AML cells.”

Richard Godfrey, CEO of BerGenBio commented: “The results of this research support our view that selective Axl-targeting agents hold significant promise in aggressive cancers where this receptor is expressed on tumor cells. As far as we are aware, our novel lead drug, BGB324, is the first Axl-selective inhibitor to enter the clinic and we look forward to future studies exploring the clinical opportunity for BGB324.”

About the Axl kinase receptor

Axl is a member of the Tyro3, Axl, Mer receptor (TAMR) tyrosine kinase family and is a fundamental receptor to cancer biology. It plays a crucial role in the epithelial-mesenchymal transition (EMT) which is a key driver of metastasis (cancer spread) and a mechanism of drug-resistance. The Axl receptor is regarded as one of the most promising new therapeutic targets for cancer drug development.

About BGB324

BGB324 is a novel, oral, highly selective small molecule inhibitor of Axl Kinase. Preclinical in vivo studies have shown that BGB324 has both single agent activity in hematological and some solid tumors and is very effective in preventing and reversing acquired resistance to standard of care cytotoxics and targeted therapies. BGB324 is currently in Phase 1 clinical trial to evaluate its safety, tolerability, pharmacokinetics and pharmacodynamics.

About Acute Myeloid Leukemia

Acute Myeloid Leukemia (AML) is an aggressive cancer of the blood and bone marrow. It is characterized by the rapid growth of abnormal white blood cells that accumulate in the bone marrow and prevent the production of other normal blood cells. It is estimated that 1 in 243 people will be diagnosed with AML during their lifetime. In the United States, the five-year overall survival is 24%.2 It is estimated that 40-45% of patients younger than 65 years can be cured with current therapies, however only 10% of older patients achieve long-term survival.3

For further information please contact:

BerGenBio AS +47 917 86 304

Richard Godfrey, CEO

Media enquiries

FTI Consulting +44 (0)20 7831 3113

Simon Conway / Stephanie Cuthbert


1. Isabel Ben-Batalla, Alexander Schultze, Mark Wroblewski, Robert Erdmann, Michael Heuser, Jonas S. Waizenegger, Kristoffer Riecken, Mascha Binder, Denis Schewe, Stefanie Sawall, Victoria Witzke, Miguel Cubas-Cordova, Melanie Janning, Jasmin Wellbrock, Boris Fehse, Christian Hagel, Jürgen Krauter, Arnold Ganser, James B. Lorens, Walter Fiedler, Peter Carmeliet, Klaus Pantel, Carsten Bokemeyer and Sonja Loges (27 August 2013). Blood; doi:10.1182/blood-2013-03-491431

2. SEER Stat Fact Sheets: Acute Myeloid Leukemia

3. Burnett A, Wetzler M, Lowenberg B (2011). Therapeutic advances in acute myeloid leukemia. Journal of clinical oncology: official journal of the American Society of Clinical Oncology; 29(5):487-494.

About BerGenBio AS

BerGenBio AS is a biopharmaceutical company located in Bergen, Norway. The company is committed to developing first in class therapeutics that inhibit EMT, preventing the formation of cancer stem cells and disrupting the important mechanisms of acquired cancer drug resistance. The company is founded on proprietary platform technology called CellSelect™, which uses information from RNAi screening studies to identify and validate novel drug targets and biomarkers. BGB324 is the first compound in BerGenBio’s pipeline to enter clinical trials, with additional compounds and drug targets at different stages of preclinical development.

About the Department of Hematology, Oncology and Bone Marrow Transplantation with Section Pneumology, University Comprehensive Cancer Center Hamburg, University Medical Center Hamburg-Eppendorf

The Department of Hematology, Oncology and Bone Marrow Transplantation with section Pneumology is part of the Oncologic Center of the Medical University Hospital Hamburg, of the Hubertus Wald Tumorzentrum and of the University Comprehensive Cancer Center Hamburg (UCCH). It has been recognized as a Center of Excellence in Oncology (Onkologisches Spitzenzentrum) by the German Cancer Aid (Deutsche Krebshilfe) since 2007. In the UCCH more than 7000 inpatients and more than 10000 outpatients are treated per year. The Department of Hematology and Oncology administers chemotherapy to more than 2500 patients and treats about 1200 patients in 134 clinical trials per year. Patients with hematologic malignancies constitute about 23% of patients treated in the department. In addition the Department hosts extensive programs for basic and translational cancer research.

About the Institute of Tumor Biology, University Comprehensive Cancer Center Hamburg, University Medical Center Hamburg-Eppendorf

The Department of Tumor Biology is part of the Center of Experimental Medicine and Member of the University Cancer Center Hamburg. Its research is dedicated to the identification and characterization of tumor cells that have disseminated from the primary tumor and may give rise to overt metastases in cancer patients. The early detection of "dormant" tumor cells is important to estimate the prognosis of patients with breast cancer and other carcinomas. It may improve the individual management of targeted therapy options, and in the long term has the potential to contribute to the development of new innovative cancer therapy approaches. To perform excellent translational research, the Institute of Tumor Biology collaborates with other institutes within the UKE, as well as other national and international organizations (Europe, Japan, USA). The Institute also leads extensive joint research projects and organizes international congresses about dormancy and dissemination of tumor cells.

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