BerGenBio

Regulatory Filings • Jun 14, 2019

BerGenBio presents preliminary Phase II clinical data at EHA 24: bemcentinib in combination with low dose chemotherapy yields durable responses in AML patients unfit for intensive chemotherapy

BerGenBio presents preliminary Phase II clinical data at EHA 24: bemcentinib in combination with low dose chemotherapy yields durable responses in AML patients unfit for intensive chemotherapy

· Two posters to be presented at the 2019 annual congress of the European

Hematology Association on 14th June

· Phase II trial evaluating bemcentinib in combination with low-intensity

chemotherapy in elderly acute myeloid leukaemia (AML) patients unfit for

intensive chemotherapy shows significant efficacy

· Preliminary responses reported in 6/14 (43%) patients, with CR/CRi in 4/14

(29%) patients; this is substantially higher than previously observed/historical

benchmarks in single-agent cytarabine (low-dose chemotherapy)

· Median relapse-free survival 7.9 months in CR/CRi patients (data not mature)

· A second biomarker poster presented by Prof. Gjertsen, University Bergen,

shows pharmacodynamic effects of bemcentinib on AML patients blast cell signal

at just 4 hours post-dosing of bemcentinib

Amsterdam, Netherlands, 14 June 2019 - BerGenBio ASA (OSE:BGBIO) today presents

data showing significant efficacy 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 AML patients unfit for intensive therapy. The data will be

presented by Professor Bjørn Tore Gjertsen MD PhD, Haukeland University Hospital

and University of Bergen, at the 2019 annual congress of the European Hematology

Association (EHA24), in Amsterdam, The Netherlands, Friday, June 14, 2019.

In total, 16 patients were enrolled into the trial. Among the 14 patients

evaluable for efficacy, 6 responses have been reported; 4 patients achieved

complete remission / complete remission with incomplete hematologic recovery

(CR/CRi) and 2 patients achieved partial remission (PR). Five of the six

responses occurred among elderly AML patients (>75 years). Furthermore, two

patients achieved durable stable disease for more than 3 months. The relapse

-free survival rate for patients with CR/CRi is 7.9 months (range: 0.7 to 9.6

months) and continues to mature. The combination treatment of bemcentinib and

LDAC was overall well-tolerated; the most common adverse events (>15% of

patients) included anaemia, neutropenia and diarrhoea. Soluble protein (e.g.

sAXL) and gene expression biomarker data is still maturing and will be reported

in due course. A second biomarker poster from Prof. Gjertsen's group, to be

presented on June 15th, examined the effect of bemcentinib monotherapy from a

phase 1b/2 clinical trial on patient AML cell signal transduction using mass

cytometry. The high-dimensional single cell-level signalling analysis of AML

blasts from 6 evaluable patients revealed significant effects already at 4 hours

post-dosing of bemcentinib

Professor Bjørn Tore Gjertsen commented: "These early results are encouraging,

especially among less fit AML patients with comparatively poor prognosis.

Bemcentinib in combination with LDAC resulted in a substantially higher ORR than

expected for single-agent cytarabine and clearly warrant further investigation

of bemcentinib in an expansion cohort of AML patients unfit for intensive

chemotherapy. Our signal cell-level biomarker analysis of AML blasts from

patients indicates a substantial effect on cell signalling and represents a

potential new biomarker strategy."

Richard Godfrey, Chief Executive Officer of BerGenBio, commented: "There are

currently limited treatment options for AML patients unable to tolerate

intensive chemotherapy, particularly those with relapsed and refractory disease.

These promising preliminary data with bemcentinib in combination with low-dose

cytarabine, reinforce data we recently at presented at ASCO, and suggests that

the addition of our selective AXL inhibitor can substantially improve AML

patient outcomes. I am particularly encouraged by the high response rate

observed in all patient groups studied, the extended duration of responses seen,

which is still maturing, and how well the combination therapy was tolerated. We

are still waiting on much of our biomarker data before we can report that and

are very excited by cell signalling data presented by our collaborators at the

University of Bergen. We will expand this study to include more patients and

report more complete data in the coming months.¨

The posters presented at EHA will be made available at www.bergenbio.com in the

Investors / Presentations section to coincide with the following conference

sessions:

Friday 14 June, 17:30 - 19:00 Central European Time

The Combination of bemcentinib, a novel, oral, selective AXL-Inhibitor and Low

-Dose Cytarabine yields Durable Responses in AML patients Unfit for Intensive

Chemotherapy

· Author: Sonja Loges et al.

· Abstract: PF259

· Location: Poster area

Saturday 15 June, 17:30 - 19:00 Central European Time

Single Cell Signaling Pharmacodynamics in a Phase 1b Clinical Trial of the AXL

inhibitor bemcentinib in Acute Myeloid Leukemia and Myelodysplastic Syndrome

· Author: Monica Hellesøy et al.

· Abstract: PS999

· Location: Poster area

- END -

About AML and the BGBC003 trial

Acute myeloid leukaemia (AML) is a rapidly progressing blood cancer. 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. Up to 28 patients will be enrolled at centres in

the US, Norway, Germany and Italy.

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 Bemcentinib

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. Bemcentinib targets and binds to the intracellular catalytic

kinase domain of AXL receptor tyrosine kinase and inhibits its activity.

Increase in AXL function has been linked to key mechanisms of drug resistance

and immune escape by tumour cells, leading to aggressive metastatic cancers.

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 focused 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, Jessica Hodgson, 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.