BerGenBio

Regulatory Filings • Apr 1, 2019

BerGenBio: Bemcentinib in combination with low-dose chemotherapy achieves efficacy endpoint in AML patients

BerGenBio: Bemcentinib in combination with low-dose chemotherapy achieves efficacy endpoint in AML patients

Phase II trial evaluating bemcentinib in combination with low-dose chemotherapy

in AML patients unfit for intensive therapy

Bemcentinib + low-dose cytarabine (LDAC) achieves efficacy endpoint, warrants

further investigation

· Three out of 10 evaluable patients (30%) reported Complete Responses

(CR/CRi)

· Responses occurred early, improved over time and included poor risk,

previously treated patients

· No overlapping or new toxicities observed

Meanwhile, Company will proceed with late-stage bemcentinib monotherapy trial in

R/R AML patients, expected for H2 2019; monotherapy efficacy and biomarker

correlation with potential for first registration will be explored with key

regulatory authorities

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.