– Phase II monotherapy study of bemcentinib in high-risk MDS & AML

– Will enrol up to 43 patients at leading MDS centres across Europe

– Investigator sponsored trial led by Prof. Uwe Platzbecker

BERGEN, Norway I Januar 04, 2019 I BerGenBio ASA (OSE: BGBIO), announces that the first patient has been dosed in an investigator-initiated phase II trial of bemcentinib, a selective, potent and orally bio-available AXL inhibitor, in patients with high-risk myelodysplastic syndrome (MDS) who have failed first-line treatment with hypomethylating agents. The trial may also enrol a proportion of patients with acute myeloid leukaemia (AML). The study is being sponsored by GWT-TUD GmbH (a specialist cancer clinical research institution associated with the University of Dresden, Germany) with the support of BerGenBio. 

The trial (BGBIL009 / BERGAMO) aims to confirm the efficacy of bemcentinib monotherapy in patients with high-risk MDS and AML and will enrol up to 43 patients at 8 hospitals in Germany, France, the Netherlands and Italy. The study will allow for the evaluation of potential predictive and pharmacodynamic biomarkers for MDS in bone marrow and blood, including those associated with patient benefit from bemcentinib.  

Prof. Uwe Platzbecker, lead investigator of the trial and director of the Medical Clinic and Policlinic 1, Hematology and Cell Therapy at the University Hospital in Leipzig, Germany, commented: “As treatment of MDS and AML has not changed significantly over the past decades, novel therapies are urgently needed. The survival is still dismal, especially in elderly patients who are not eligible for allogeneic stem cell transplantation and who have failed first line treatment with hypomethylating agents. AXL, a member of the Tyro3, AXL, Mer (TAM) receptor family, mediates proliferation and survival of leukemic cells and is upregulated upon cytostatic treatment. Pre-clinical studies with the inhibitor bemcentinib demonstrated in vitro and in mouse models that leukaemic proliferation was blocked by interference with AXL signalling. Hence, AXL represents a promising new target for the patient population investigated in the BERGAMO trial.”

Richard Godfrey, Chief Executive Officer of BerGenBio, added: “We are very pleased that Prof. Platzbecker and GWT-TUD are initiating this study, which if positive will add valuable information on bemcentinib’s monotherapy use in a larger leukaemia population and provide support for our broader development plans for bemcentinib in these indications. We look forward to reporting results as the trial progresses.”

About MDS

Myelodysplastic syndromes (MDS) are stem cell disorders characterised by a decreased ability of the bone marrow to produce normal blood cells and platelets. MDS is associated with increased risk of developing AML and immune dysfunctions are seen in patients both with lower and higher-risk MDS. Drugs that modify immunological responses can improve blood values and prolong survival in some patients. Thus far, however, the only curative treatment for MDS remains stem cell transplantation. Hence, there is an urgent need for novel therapies to treat MDS.

About AXL

AXL kinase is a cell membrane receptor and an essential mediator of the biological mechanisms underlying aggressive and life-threatening diseases. In cancer, AXL drives tumour survival, treatment resistance and spread, as well as suppressing the body’s immune response to tumours. AXL expression has been established as a negative prognostic factor in many cancers. AXL inhibitors, therefore, have potential value at the centre of cancer combination therapy, addressing significant unmet medical needs and multiple high-value market opportunities.

About Investigator-sponsored trials

Investigator-sponsored clinical trials are clinical trials proposed by front-line patient-facing physicians who act as the regulatory sponsor and are supported by industry in bespoke clinical development partnerships. The industry partner does not assume the role of sponsor according to European or US regulatory guidelines but may offer support in a variety of different ways, such as providing investigational medicinal product at no cost.

About BerGenBio ASA

BerGenBio is a clinical-stage biopharmaceutical company focused on developing transformative drugs targeting AXL as a potential cornerstone of therapy for advanced and aggressive cancers.

The company’s proprietary lead candidate, bemcentinib (formerly known as BGB324), is a potentially first-in-class selective AXL inhibitor in a broad phase II clinical development programme. Ongoing clinical trials are investigating bemcentinib in multiple solid and haematological tumours, in combination with current and emerging therapies (including immunotherapies, targeted therapies and chemotherapy), and as a single agent.

In parallel, BerGenBio is developing companion diagnostics test to identify patient populations most likely to benefit from bemcentinib; this is expected to facilitate more efficient registration trials and support a precision medicine-based commercialisation strategy.

BerGenBio is based in Bergen, Norway with a subsidiary in Oxford, UK. The company is listed on the Oslo Stock Exchange (ticker: BGBIO).