Bergen, Norway, 1 April 2019 – BerGenBio ASA (OSE:BGBIO) announces that on a top-line, preliminary basis, the first efficacy endpoint has been met in a Phase II clinical trial (BGBC003, NCT02488408) evaluating bemcentinib, a first-in-class selective oral AXL inhibitor, in combination with low-dose cytarabine (LDAC) as a potential new treatment regimen for acute myeloid leukaemia (AML) patients unable to tolerate intensive therapy. A second cohort evaluating bemcentinib in combination with decitabine is also ongoing and data is further maturing.

The pre-specified efficacy endpoint in this trial requires at least three patients out of the first 14 patients (21%) to achieve clinical responses* when treated with the novel drug combination.

The cohort evaluating bemcentinib in combination with LDAC, summarised below, has met its primary endpoint; data is further maturing:

• In total, 15 patients were enrolled of which 10 are evaluable for response to date. Among these, 4 were newly diagnosed and 6 relapsed or refractory (R/R). 
• In newly-diagnosed patients, one complete remission (CR) and one CR with incomplete haematologic recovery(CRi) were observed (2 out of 4) and one CR among R/R patients (1 out of 6), yielding a CR/CRi rate of 30% (3 out of 10). 
• All responders were above the age of 75, at least one had secondary disease, and two had unfavourable cytogenetic profiles. Responses had an early onset (after 1 to 2 cycles), were durable and deepened over time and the combination of bemcentinib with LDAC was well tolerated causing no overlapping or new toxicities compared to each agent given alone.

An extensive translational programme continues to explore potential biomarkers for bemcentinib combination therapy. Further details will be presented at upcoming medical conferences.

Furthermore, the Company is proceeding with preparations for a late-stage bemcentinib monotherapy trial in AML. The study, expected to start in the second half of 2019, will aim to confirm previously reported monotherapy efficacy of bemcentinib in AML patients whose disease has progressed on standard of care therapy. Correlation with relevant biomarkers will be explored with the ambition to pursue an accelerated development path in a selected patient population.

Professor Dr Dr Sonja Loges, attending physician at the University Hospital Hamburg-Eppendorf and lead investigator of the BGBC003 trial, commented: “Data gathered by our group and others have established AXL as a negative prognostic factor in patients with AML and other malignancies. AXL acts both as a driver of leukaemic cell proliferation and as negative regulator of anti-tumour immunity and therefore represents a novel and promising target in AML. Bemcentinib, an oral tablet, targets AXL selectively and I am pleased to see activity in my patients receiving bemcentinib alone or in combination with standard low-intensity therapies while side effects thus far have proven to be limited and manageable. These early results warrant further investigation of bemcentinib in a larger trial addressing AML patients unfit for intensive chemotherapy.” 

Richard Godfrey, Chief Executive Officer of BerGenBio, commented:  “Clearing the efficacy threshold for bemcentinib in combination with LDAC is very encouraging, particularly as we have seen responses in a less fit AML patient population who are considered to have unfavourable prognosis after the failure of first-line therapies, or those with high risk cytogenetics. AML patients represent a challenging patient population for clinical trials, but we are keen to pursue AML monotherapy as our first indication based on the high unmet need, strength of clinical proof-of-concept with biomarker correlation, excellent safety and potential for a fast and first route to registration. We look forward to providing more details on the trial design upon initiation and reporting further data with bemcentinib combinations in this indication as it matures.”

*defined as objective response (OR), as measured by the revised recommendations of the International Working Group (IWG) in AML

– END –

About AML and the BGBC003 trial

AML is the most common form of acute leukaemia in adults where malignant AML blasts interfere with the normal functioning of the bone marrow leading to a multitude of complications like anaemia, infections and bleeding. AML is diagnosed in over 20,000 patients in the US annually and is rapidly lethal if left untreated. Successful treatment typically requires intensive therapy or bone marrow transplantation, and relapse and resistance are common. Consequently, there is an urgent need for effective novel therapies in relapsed/refractory patients, particularly those that are ineligible for intensive therapy or bone marrow transplant. 

The BGBC003 trial is a phase Ib/II multi-centre open label study of bemcentinib in combination with cytarabine (part B2) and decitabine (part B3) in patients with AML who are unsuitable for intensive chemotherapy as a result of advanced age or existing-co-morbidities. 

For more information please access trial NCT02488408 at www.clinicaltrials.gov.

About AXL

AXL kinase is a cell membrane receptor and an essential mediator of the biological mechanisms underlying life-threatening diseases. In cancer, AXL suppresses the body’s immune response to tumours and drives cancer treatment failure across many indications. AXL inhibitors, therefore, have potential high value at the centre of cancer combination therapy, addressing significant unmet medical needs and multiple high-value market opportunities. Research has also shown that AXL mediates other aggressive diseases.

About BerGenBio ASA

BerGenBio is a clinical-stage biopharmaceutical company focused on developing transformative drugs targeting AXL as a potential cornerstone of therapy for aggressive diseases, including immune-evasive, therapy resistant cancers. The company’s proprietary lead candidate, bemcentinib, is a potentially first-in-class selective AXL inhibitor in a broad phase II oncology clinical development programme focussed on combination and single agent therapy in lung cancer and leukaemia. A first-in-class functional blocking AXL antibody (BGB149) and an AXL-ADC (ADCT-601) are undergoing phase I clinical testing. In parallel, BerGenBio is developing a companion diagnostic test to identify those patient populations most likely to benefit from bemcentinib: this is expected to facilitate more efficient registration trials supporting 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). www.bergenbio.com

Contacts

Richard Godfrey CEO, BerGenBio ASA
+47 917 86 304

Rune Skeie, CFO, BerGenBio ASA
rune.skeie@bergenbio.com
+47 917 86 513

International Media Relations

Mary-Jane Elliott, Chris Welsh, Nicholas Brown, Carina Jurs, Consilium Strategic Communications
bergenbio@consilium-comms.com
+44 20 3709 5700

Media Relations in Norway

Jan Petter Stiff, Crux Advisers
stiff@crux.no
+47 995 13 891

Forward looking statements

This announcement may contain forward-looking statements, which as such are not historical facts, but are based upon various assumptions, many of which are based, in turn, upon further assumptions. These assumptions are inherently subject to significant known and unknown risks, uncertainties and other important factors. Such risks, uncertainties, contingencies and other important factors could cause actual events to differ materially from the expectations expressed or implied in this announcement by such forward-looking statements.

This information is subject to the disclosure requirements pursuant to section 5-12 of the Norwegian Securities Trading Act.