BerGenBio Receives Orphan-drug Designation from FDA for BGB324 in the Treatment of Acute Myeloid Leukaemia

BERGEN, Norway, November 19, 2014 /PRNewswire/ --

BerGenBio AS, an oncology biopharmaceutical company, today announces that the US Food and Drug Administration (FDA) has granted orphan-drug designation for BGB324 for treatment of acute myeloid leukaemia (AML).

Earlier this month BerGenBio announced that the first patient has been dosed in its multi-centre Phase 1b trial of BGB324, a selective inhibitor of Axl, in patients with AML. The two-part trial will primarily investigate the safety and tolerability of BGB324 when administered as a single agent and in combination with a standard-of-care drug (cytarabine); secondary endpoints will also explore evidence of clinical response and assess novel biomarkers. The study will be conducted at six sites in Norway, Germany and the United States. BerGenBio expects data to be available from this trial in 2015. Additional clinical trials with BGB324 in non-small cell lung cancer are in preparation.

AML is an aggressive cancer of the blood and bone marrow characterised 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 about 1 in 227 men and 1 in 278 women will be diagnosed with AML during their lifetime^[1] and in the United States the five-year overall survival is 24%^[2].

Richard Godfrey, Chief Executive Officer of BerGenBio, commented:

"The FDA's decision to grant BerGenBio orphan-drug status for BGB324 is a significant milestone for the Company and recognises the need for innovative new ways to treat AML. The designation will give BerGenBio access to various development incentives from the Agency, including tax credits for qualified clinical testing. Additionally BerGenBio will be exempt from prescription drug user fees for BGB324 for this indication and, if the drug receives marketing approval, it will enjoy seven years of market exclusivity in the United States."

About orphan-drug designation 

The FDA grants orphan-drug designation to development-stage novel therapeutics that are intended for use in treating rare diseases and medical conditions that affect fewer than 200,000 patients in the US. Orphan-drug status provides sponsors with development and commercial incentives under the Orphan Drug Act, including tax credits for qualified clinical testing.

About BGB324 

BGB324 is a first-in-class, highly selective small molecule inhibitor of the Axl receptor tyrosine kinase. It blocks the epithelial-mesenchymal transition (EMT), which is a key driver in drug-resistance and metastasis.

About acute myeloid leukaemia 

Acute myeloid leukaemia (AML) is an aggressive cancer of the blood and bone marrow. It is characterised 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 227 men and 1 in 278 women will be diagnosed with AML during their lifetime. In the United States, the five-year overall survival is 24%. 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. BerGenBio has shown that more than 50% of AML patients have elevated levels of Axl and by inhibiting Axl with BGB324 in preclinical studies a substantial and potentially therapeutic reduction in leukemic burden can be achieved.

About BerGenBio AS 

BerGenBio AS is a clinical stage biopharmaceutical company. The Company is committed to developing innovative therapeutics that inhibit EMT, prevent the formation of cancer stem cells and disrupt the cellular mechanisms that drive acquired cancer drug resistance. The Company is founded on proprietary platform technology, 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 in AML and NSCLC, with additional compounds and drug targets at different stages of preclinical development. http://www.bergenbio.com

[1] http://www.cancer.org/cancer/leukemia-acutemyeloidaml/detailedguide/leukemia-acute-myeloid-myelogenous-key-statistics (last accessed 19.11.14)

[2] http://www.cancer.net/cancer-types/leukemia-acute-myeloid-aml/statistics (last accessed 19.11.14)

SOURCE BerGenBio