BerGenBio

Regulatory Filings • Jul 20, 2020

BERGENBIO ANNOUNCES FIRST PATIENT DOSED IN RECURRENT GLIOBLASTOMA INVESTIGATOR SPONSORED PHASE I/II STUDY ASSESSING SELECTIVE AXL INHIBITOR BEMCENTINIB

BERGENBIO ANNOUNCES FIRST PATIENT DOSED IN RECURRENT GLIOBLASTOMA INVESTIGATOR SPONSORED PHASE I/II STUDY ASSESSING SELECTIVE AXL INHIBITOR BEMCENTINIB

Bergen, Norway, 20 July 2020 - BerGenBio ASA (OSE: BGBIO), a clinical-stage

biopharmaceutical company developing novel, selective AXL kinase inhibitors for

severe unmet medical need, announces that the first patient has been dosed and

continues on therapy in a trial assessing bemcentinib in recurrent glioblastoma

(GBM). The trial is sponsored by Prof. Ichiro Nakano, MD, Professor in the

Department of Neurosurgery and co-leader of the Neuro-Oncology Program at

University of Alabama at Birmingham and funded by the National Cancer Institute

(NCI).

This is an open label, multi-centre, intra-tumoral tissue pharmacokinetic (PK)

study of bemcentinib in patients with recurrent glioblastoma for whom a surgical

resection is medically indicated. The study will enrol up to 20 recurrent GBM

patients, at up to 15 sites in the USA. 10 patients will be treated prior to

surgery and 10 patients will have no pre-surgical treatment. However, all

patients will receive treatment with bemcentinib following surgery. The

endpoints of the study include an evaluation of bemcentinib's ability to cross

the blood brain barrier, AXL expression, pharmacokinetics, safety and

tolerability, as well as efficacy assessments including Progression Free

Survival and Overall Survival. More information about the trial can be found at

https://clinicaltrials.gov/ct2/show/NCT03965494 (https://clinicaltrials.gov/ct2/s

how/NCT03965494)

Increased expression of the receptor tyrosine kinase AXL is significantly

correlated with poor prognosis in GBM patients and preclinical data has

suggested that bemcentinib may be a promising therapeutic agent for GBM,

particularly in post-irradiation mesenchymal-transformed GBM tumors[1]. A

comprehensive translational research programme will run in parallel with the

clinical trial, this will be conducted by Prof. Jeff Supko, Harvard Medical

School and Director of the Clinical Pharmacology Laboratory, Massachusetts

General Hospital (Boston, USA).

Prof. Burt Nabors MD, the Chairman of the trial and Director of Neuro-Oncology

at University of Alabama at Birmingham (UAB) and Director of UAB's Centre for

Clinical Translational Science's Clinical Research Unit, commented: "GBM is

among the most lethal of adult cancers. The median survival of patients remains

less than two years despite the current available therapies, including surgery,

radiation, and chemotherapy; development of more effective therapies is urgently

needed. We welcome the opportunity to offer patients access to the

investigational AXL inhibitor bemcentinib in this pilot study and look forward

to initiating additional trial sites across the Adult Brain Tumour Consortium in

the USA later this year."

Richard Godfrey, Chief Executive Officer of BerGenBio, commented: "We

congratulate Prof. Nakano and Prof. Nabors on the start of this exciting

clinical study, which we believe will provide us with important data regarding

the ability of bemcentinib to cross the blood-brain barrier and potentially

treat GBM patients. This clinical trial is based on pioneering preclinical

research carried out by our collaborators, conducted at high profile research

hospitals in the USA and is funded by National Cancer Institute (NCI). We look

forward to reporting the potential of bemcentinib to improve patient outcomes in

this very aggressive cancer."

- END -

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 and 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,

leukaemia and COVID-19. A first-in-class functional blocking anti-AXL antibody,

tilvestamab, is undergoing Phase I clinical testing. In parallel, BerGenBio is

developing companion diagnostic tests to identify those patient populations most

likely to benefit from bemcentinib or tilvestamab: this is expected to

facilitate more efficient registration trials and support a precision medicine

-based commercialisation strategy. For further information, please visit:

www.bergenbio.com (http://www.bergenbio.com)

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 Glioblastoma

Glioblastoma (GBM) ranks among the deadliest of all human cancers with no

curative options available[2]. It is the most aggressive of the gliomas, a

collection of tumors arising from glia or their precursors within the central

nervous system. Gliomas are divided into four grades, grade 4 or glioblastoma

multiforme (GBM) is the most aggressive of these and is the most common in

humans. Most patients with GBMs die of their disease in less than a year[3].

For more information, please contact

Richard Godfrey

CEO, BerGenBio ASA

media@bergenbio.com

+47 917 86 304

International Media Relations

Mary-Jane Elliott, Chris Welsh, Carina Jurs,

Lucy Featherstone, Maya Bennison

Consilium Strategic Communications

bergenbio@consilium-comms.com

+44 7780 600290

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.

[1] 3. Sadahiro H, Kang KD, Gibson JT, et al. Activation of the Receptor

Tyrosine Kinase AXL Regulates the Immune Microenvironment in Glioblastoma.

Cancer Res. 2018;78(11):3002-3013.

[2,3] 3 1. Cloughesy, T., Finocchiaro, G., Belda-Iniesta, C., et al. (2016).

Randomized, Double-Blind, Placebo-Controlled, Multicenter Phase II Study of

Onartuzumab plus Bevacizumab versus Placebo plus Bevacizumab in Patients with

Recurrent Glioblastoma: Efficacy, Safety, and Hepatocyte Growth Factor and O6

-Methylguanine-DNA Methyltransferase Biomarker Analyses. J Clin Oncol,

JCO2015647685. Gilbert, M.R., Sulman, E.P., and Mehta, M.P. (2014). Bevacizumab

for newly diagnosed glioblastoma. N Engl J Med 370, 2048-2049.