BerGenBio

Regulatory Filings • Jan 4, 2019

BerGenBio Announces Start of Phase II Investigator Initiated Trial Evaluating Selective AXL Inhibitor Bemcentinib in high-risk MDS

BerGenBio Announces Start of Phase II Investigator Initiated Trial Evaluating Selective AXL Inhibitor Bemcentinib in high-risk MDS

· 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, Jan 4th 2019 - 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."

-Ends-

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).

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

Media Relations in Norway

Jan Petter Stiff, Crux Advisers

stiff@crux.no

+47 995 13 891

International Media Relations

David Dible, Mark Swallow, Marine Perrier, Citigate Dewe Rogerson

bergenbio@citigatedewerogerson.com

+44 207 638 9571

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.