Earnings Release • Aug 18, 2017
Earnings Release
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Developing first-in-class Axl inhibitors to treat aggressive cancer
Second Quarter and First Half 2017 presentation August 18th 2017
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.
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| Corporate | Successful IPO (April) raised NOK 400 million to finance development strategy  Clinical collaboration agreement with Merck & Co (MSD) (March)  Two Phase II combination trials with MSD's immune checkpoint inhibitor KEYTRUDA® in patients  with advanced lung (NSCLC) and triple negative breast cancer (TNBC) |
|---|---|
| BGB324 | Phase II clinical development program opened and enrolling  Monotherapy study in AML/MDS enrolling patients  Combination study with erlotinib opened in lung cancer – enrolling first and second line cohorts  Sites active and patient recruitment ongoing in BGB324/KEYTRUDA study in patients with TNBC,  Site activation underway for BGB324/KEYTRUDA study in patients with NSCLC  Investigator-sponsored studies in lung cancer (docetaxel combo) and melanoma (combination  with current targeted & I-O therapies) opened and dosed first patients Biomarker studies underway in association with clinical studies  Scientific and clinical presentations made at AACR and ASCO international cancer conferences  |
| Pipeline | Clinical candidate BGB149 was nominated, a humanised anti-Axl monoclonal antibody  Cell line development and antibody manufacturing is underway with a leading biologics CRO  |
| Cash | NOK 440 million at end of Q2 2017  Cash runway into 2019, and sufficient to deliver key read-outs from four Phase II trials in 2H 2018  Awarded BIA Grant by NFR and IFU grant from Innovasjon Norge of NOK 24 m  |
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
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
BGB324 has generated strong efficacy data in patients with no other existing treatment options
10 |
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
• Sales of KEYTRUDA were USD 1.4bn in 2016
| 22 patients (up to 48) § Continuous treatment with BGB324 in combination with KEYTRUDA |
|
|---|---|
| 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 |
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)
''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
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
AA: Accelerated approval; BT: Breakthrough designation; PRIME: Priority Medicine status in EU
| 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 |
| (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
| 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 |
 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
Developing first-in-class Axl inhibitors to treat aggressive cancer
| 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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