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 Tr