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BerGenBio

Earnings Release Aug 18, 2017

3555_rns_2017-08-18_ed7eece0-aa0d-4701-aed3-f91a21524347.pdf

Earnings Release

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BerGenBio

Developing first-in-class Axl inhibitors to treat aggressive cancer

Second Quarter and First Half 2017 presentation August 18th 2017

Disclaimer

Certain statements contained in this presentation constitute forward-looking statements. Forward-looking statements are statements that are not historical facts and they can be identified by the use of forward-looking terminology, including the words "anticipate", "believe", "intend", "estimate", "expect", "will", "may", "should" and words of similar meaning. By their nature, forward-looking statements involve a number of risks, uncertainties and assumptions that could cause actual results or events to differ materially from those expressed or implied by the forward-looking statements. Accordingly, no assurance is given that such forward-looking statements will prove to have been correct. They speak only as at the date of the presentation and no representation or warranty, expressed or implied, is made by BerGenBio ASA or its affiliates ("BerGenBio"), or by any of their respective members, directors, officers or employees that any of these forward-looking statements or forecasts will come to pass or that any forecast result will be achieved and you are cautioned not to place any undue influence on any forward-looking statement. BerGenBio is making no representation or warranty, expressed or implied, as to the accuracy, reliability or completeness of this presentation, and neither BerGenBio nor any of its directors, officers or employees will have any liability to you or any other person resulting from the use of this presentation.

Copyright of all published material, including photographs, drawings and images in this presentation remain with BerGenBio and relevant third parties, as appropriate. Consequently, no reproduction in any form of the presentation, or parts thereof, is permitted without the prior written permission, and only with appropriate acknowledgements.

Q2 and 1H 2017 Achievements

R&D programmes are progressing to plan – solid foundation to build value

Corporate Successful IPO (April) raised NOK 400 million to finance development strategy
Â
Clinical collaboration agreement with Merck & Co (MSD) (March)
Â
Two Phase II combination trials with MSD's immune checkpoint inhibitor KEYTRUDA® in patients
Â
with advanced lung (NSCLC) and triple negative breast cancer (TNBC)
BGB324 Phase II clinical development program opened and enrolling
Â
Monotherapy study in AML/MDS enrolling patients
Â
Combination study with erlotinib opened in lung cancer –
enrolling first and second line cohorts
Â
Sites active and patient recruitment ongoing in BGB324/KEYTRUDA study in patients with TNBC,
Â
Site activation underway for BGB324/KEYTRUDA study in patients with NSCLC
Â
Investigator-sponsored studies in lung cancer (docetaxel combo) and melanoma (combination
Â
with current targeted & I-O therapies) opened and dosed first patients
Biomarker studies underway in association with clinical studies
Â
Scientific and clinical presentations made at AACR and ASCO international cancer conferences
Â
Pipeline Clinical candidate BGB149 was nominated, a humanised
anti-Axl monoclonal antibody
Â
Cell line development and antibody manufacturing is underway with a leading biologics CRO
Â
Cash NOK 440 million at end of Q2 2017
Â
Cash runway into 2019, and sufficient to deliver key read-outs from four Phase II trials in 2H 2018
Â
Awarded BIA Grant by NFR and IFU grant from Innovasjon
Norge of NOK 24 m
Â

1. AXL biology and clinical development strategy

  • 2. BGB324 Clinical trial progress report
  • 3. BGB324 and I-O therapy
  • Merck clinical collaboration studies
  • 4. BGB324 Investigator-sponsored studies
  • BGB324 combination study with docetaxel in NSCLC patients
  • 5. BGB324 Commercialisation strategy
  • 6. Finance report
  • 7. Outlook
  • 8. Q&A

BerGenBio – First-in-class Axl inhibitors for multiple aggressive cancers

90% of cancer deaths result from tumors spreading, becoming immune evasive and drug resistant

Axl is a key mediator of these traits in a broad range of cancers

BerGenBio is a world-leader in Axl biology and is developing an exciting pipeline of Axl inhibitors

BGB324 initially addressing an annual market potential of USD 11 Billion

BGB324 – First-in-class, highly selective oral Axl inhibitor

  • Â Highly selective and potent
  • Â Orally bioavailable
  • Â Well tolerated: suitable for long-term therapy
  • Â Wide therapeutic index: suitable for combination with existing drugs
  • Â Orphan status in US for AML
  • Â Licensed from Rigel Inc. 2011

Investigational Medicinal Product

  • Â Patients take medicines home, one-a-day dose
  • Â 100mg capsules, standard pharmaceutical formulation
  • Â 3yr shelf life
  • Â Cost effective treatment

Strategic rationale for target indications Major unmet need, strong scientific basis, large addressable market (~USD11Bn)

1) SEER Program – National Cancer Institute (National Institute of Health) http://seer.cancer.gov/; 3) Cancer.net; 4) Figure for male and female breast cancer; 5) Excluding rectum; 6) Estimates by Alacrita Consulting

  • 1. AXL biology and clinical development strategy
  • 2. BGB324 Clinical trial progress report
  • 3. BGB324 and I-O therapy
  • Merck clinical collaboration studies
  • 4. BGB324 Investigator-sponsored studies
  • BGB324 combination study with docetaxel in NSCLC patients
  • 5. BGB324 Commercialisation strategy
  • 6. Finance report
  • 7. Outlook
  • 8. Q&A

Strategic pipeline will drive value creation

Compelling Phase Ib clinical data for BGB324

BGB324 has generated strong efficacy data in patients with no other existing treatment options

10 |

Status of BGB324 phase II clinical trials

BerGenBio sponsored clinical trials

  • 1. AXL biology and clinical development strategy
  • 2. BGB324 Clinical trial progress report
  • 3. BGB324 and I-O therapy
  • Merck clinical collaboration studies
  • 4. BGB324 Investigator-sponsored studies
  • BGB324 combination study with docetaxel in NSCLC patients
  • 5. BGB324 Commercialisation strategy
  • 6. Finance report
  • 7. Outlook
  • 8. Q&A

Strong rationale for combining BGB324 with checkpoint inhibitors

Checkpoint inhibitors do not work for all patients in all cancers

AXL up-regulation is the greatest change in non-responders

AXL drives immune evasion

13 |

Hugo, 2016

Phase II studies of BGB324 in combination with KEYTRUDATM

The ONLY combination study with KEYTRUDA addressing the fundamental mechanism of cancer cell resistance to CPIs

Sales of KEYTRUDA were USD 1.4bn in 2016

  • KEYTRUDATM (pembrolizumab) is approved in the US for the treatment of:
  • First-line treatment of metastatic NSCLC high PD-L1 expression
  • PDL-1 positive Metastatic NSCLC Unresectable / metastatic melanoma
  • Recurrent / metastatic HNSCC
  • Microsatellite instability-high (MSI-H) or a mismatch repair deficient (dMMR) solid tumours

Merck collaboration studies – BGB324 in combination with KEYTRUDA

Patient population Parameters Read out (exp. 2H 2018) § Previously treated unresectable or metastatic triple negative breast cancer § Measurable disease § Fresh tissue biopsy 10 Endpoint: § Objective response rate 20 Endpoint: § Safety § Duration of response § Time to progression § Survival at 12 months § Response by biomarker expression BGBC007 Phase II – Triple negative breast cancer 28 patients (up to 56) § Continuous treatment with BGB324 in combination with KEYTRUDA Biomarker: § Tissue sample and blood based biomarker collection and processing § Assay development and qualification § PD-L1 assay to be performed by Merck Patient population Parameters Read out (exp. 2H 2018) BGBC008 Phase II – Adenocarcinoma of the lung Status § Protocol approved in US, UK & Norway (under review in Spain) § Patient recruitment ongoing at active sites

  • § Previously treated unresectable adenocarcinoma of the lung
  • § Measurable disease
  • § Fresh tissue biopsy
22 patients (up to 48)
§
Continuous treatment with BGB324 in
combination with KEYTRUDA

Biomarker:

  • § Tissue sample and blood based biomarker collection and processing
  • § Assay development and qualification
  • § PD-L1 assay to be performed by Merck
Status
10
Endpoint:
§
Objective response rate
20
Endpoint:
§
Protocol approved in US,
UK, Norway & Spain
§
Safety
§
Duration of response
§
Progression free survival
§
Survival at 12 months
§
Response by biomarker expression
§
Site startup ongoing
§
First patient dosing
imminent
  • 1. AXL biology and clinical development strategy
  • 2. BGB324 Clinical trial progress report
  • 3. BGB324 and I-O therapy
  • Merck clinical collaboration studies
  • 4. BGB324 Investigator-sponsored studies
  • BGB324 combination study with docetaxel in NSCLC patients
  • 5. BGB324 Commercialisation strategy
  • 6. Finance report
  • 7. Outlook
  • 8. Q&A

Rationale for combining BGB324 and docetaxel in NSCLC patients

Chemotherapy still is an important part of the treatment landscape for advanced NSCLC1

BGB324 enhances the effect of chemotherapy in animal models

(1) As per ASCO guidelines 2017 (2) Wilson et al. Cancer Res (2014) (2) Wilson et al. Cancer Res (2014)

Investigator-sponsored study: Phase I/II trial in NSCLC of BGB324 with docetaxel

Sponsor Investigator: Dr David Gerber, UTSW Dallas

''The vast majority of my lung cancer patients progress onto chemotherapy, combining this with BGB324 may significantly improve the performance of the chemo and could lead to meaningful disease modification in some patients.''

18

  • 1. AXL biology and clinical development strategy
  • 2. BGB324 Clinical trial progress report
  • 3. BGB324 and I-O therapy
  • Merck clinical collaboration studies
  • 4. BGB324 Investigator-sponsored studies
  • BGB324 combination study with docetaxel in NSCLC patients

5. BGB324 Commercialisation strategy

  • 6. Finance report
  • 7. Outlook
  • 8. Q&A

Summary of BerGenBio's clinical development plan to deliver Phase II data in high-value indications by end 2018

IND: Investigational New Drug – required prior to initiation of clinical trials in the USA

BGB324 – Set to become a highly attractive and valuable asset

AA: Accelerated approval; BT: Breakthrough designation; PRIME: Priority Medicine status in EU

  • 1. AXL biology and clinical development strategy
  • 2. BGB324 Clinical trial progress report
  • 3. BGB324 and I-O therapy
  • Merck clinical collaboration studies
  • 4. BGB324 Investigator-sponsored studies
  • BGB324 combination study with docetaxel in NSCLC patients
  • 5. BGB324 Commercialisation strategy
  • 6. Finance report
  • 7. Outlook
  • 8. Q&A

Successful IPO and broadened shareholder base

IPO and listing on OSE § Share facts *

  • Â Completed 7 April 2017, ticker BGBIO
  • Â Raised NOK 400 million in gross proceeds
  • Â New and existing investors participated (approximately 2,000 shareholders)
Currency NOK
Market Oslo (NOK)
ISIN code NO0010650013
Ticker code BGBIO
Industry Biotechnology
Market Capitalization NOK
1.1 bn
Number of Shares 49,757,200
Number
of shareholders
1,954

§ Shareholding by investor type in IPO

Key financials Q2 and 1H 2017

(NOK million) Q2 '17 Q2 '16 YTD '17 YTD '16 FY '16
Operating revenues - - - - -
Operating expenses 33.8 66.5 99.6 87.2 131.6
Operating profit (loss) (33.8) (66.5) (99.6) (87.2) (131.6)
Profit (loss) after tax (34.1) (66.2) (99.1) (86.5) (129.8)
Basic and diluted
earnings (loss) per
share (NOK)
(0.70) (225.83) (2.41) (307.27) (419.68)
Cash position end of
period
440.3 105.2 440.3 105.2 161.8

 Operating expenses in Q1 2017 impacted by NOK 27.8 million (USD 3.3 million) Phase II milestone payment to Rigel Pharmaceuticals Inc, and Q2 2016 impacted by the first instalment of the Phase II milestone

 Net proceeds from the IPO approximately NOK 375 million – received in April

  • 1. AXL biology and clinical development strategy
  • 2. BGB324 Clinical trial progress report
  • 3. BGB324 and I-O therapy
  • Merck clinical collaboration studies
  • 4. BGB324 Investigator-sponsored studies
  • BGB324 combination study with docetaxel in NSCLC patients
  • 5. BGB324 Commercialisation strategy
  • 6. Finance report
  • 7. Outlook
  • 8. Q&A

Key progress and future milestones

Lung cancer (NSCLC) study with BGB324 in combination with TARCEVA opened (first and second line cohorts)
IPO –
NOK 400m to fund BGB324 Phase II clinical programme, Axl
CDx
and BGB149 into the clinic
Data presentations at AACR and ASCO
Investigator-sponsored Phase
II trial opened, first NSCLC patients dosed with BGB324 in combination with docetaxel
Investigator-sponsored Phase II trial opened,
first melanoma patients dosed with BGB324 in combination with
KEYTRUDA
or targeted therapy
Phase II initiated in TNBC study with BGB324 in combination with KEYTRUDA
-
Sites active and patient recruitment ongoing
Phase II initiated in NSCLC study with BGB324 in combination with KEYTRUDA
-
First patient dosed
2H 2017
Presentation of interim data from Phase II study
of BGB324 in AML/MDS
2H
2017
Presentation of Interim data from Phase II study of BGB324 in EGFR+ NSCLC 2H
2017
Initiation of Phase I for
BGB149
2H 2018
Phase II Clinical proof-of-concept
data from BGB324 studies
-
AML/MDS –
single agent/combination
-
NSCLC (EGFR+) –
combination with TARCEVA
2H 2018
-
NSCLC (adenocarcinoma) –
combination with KEYTRUDA
-
TNBC –
combination with KEYTRUDA

Summary and outlook

  • Â First-in-class drugs targeting aggressive cancers with large unmet need
  • Â Universal mechanism of action confers broad clinical potential across many tumour types
  • Â \$11bn addressable market from ongoing sponsored studies

 Multiple Phase II programmes with BGB324 in significant cancer indications are open and recruiting

 Recent IPO provides funding for clinical and pipeline development through high-value inflection points

  • Â Clear strategy to develop and commercialise assets
  • Â High value, first-in-class drug candidates are attractive targets for partnering and M&A
  • Â Go-to market possibilities in enriched patient populations
  • Â High visibility with strong news flow and multiple value driving inflection points though 2018

Thank you.

For further information please visit www.bergenbio.com

Developing first-in-class Axl inhibitors to treat aggressive cancer

Glossary

AA Accelerated
approval
FDA US Food and Drug
Administration
ADC Antibody drug
conjugate
GLP Good
Laboratory Practice
ALK Alkaline
phosphatase
IHC Immunohistochemistry
AML Acute myeloid leukemia mAb Monoclonal
antibody
BLA Biologic license application MDS Myeloid dysplastic syndrome
BT Breakthrough therapy NDA New drug application
CAB Clinical advisory board NSCLC Non-small cell lung cancer
CBR Clinical benefit rate pAxl Phosphorylated Axl (activated Axl)
CDx Companion diagnostic PD Progressive disease
CLIA Clinical Laboratory Improvement Amendments PR Partial response
CLL Chronic lymphocytic leukemia RCC Renal carcinoma
CPI Checkpoint inhibitor RP2D Recommended Phase II Dose
CR Complete response RTK Receptor tyrosine kinase
CTL Cytotoxic T-lymphocytes TAM Tyro, Axl, Mer
(family of kinases)
ECG Electrocardiogram TNBC Triple negative breast cancer
EGFR Epidermal growth factor receptor sAxl Soluble Axl
ELISA Enzyme-linked immunosorbent assay SD Stable disease
EMT Epithelial-to-mesenchymal transition SoC Standard of Care
EU5 France, Germany, Italy, Spain, United Kingdom QTcF QT inverval, a measure of time in the heart's electrical cycle

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