Algeta ASA
Second Quarter and First Half 2009 Results
August 2009
Andrew Kay, President & CEO Thomas Ramdahl, EVP and CTO
Øystein Soug, CFO
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Disclaimer
THIS PRESENTATION HAS BEEN PREPARED BY ALGETA ASA (THE ”COMPANY”) EXCLUSIVELY FOR INFORMATION PURPOSES. THIS PRESENTATION HAS NOT BEEN REVIEWED OR REGISTERED WITH ANY PUBLIC AUTHORITY OR STOCK EXCHANGE. THE DISTRIBUTION OF THIS PRESENTATION AND ANY OFFERING, SUBSCRIPTION, PURCHASE OR SALE OF SECURITIES ISSUED BY THE COMPANY IN CERTAIN JURISDICTIONS IS RESTRICTED BY LAW. POTENTIAL INVESTORS ARE REQUIRED BY THE COMPANY TO INFORM THEMSELVES ABOUT AND TO COMPLY WITH ALL APPLICABLE LAWS AND REGULATIONS IN FORCE IN ANY JURISDICTION IN WHICH IT INVESTS AND MUST OBTAIN ANY CONSENT, APPROVAL OR PERMISSION REQUIRED UNDER THE LAWS AND REGULATIONS IN FORCE IN SUCH JURISDICTION. THE COMPANY SHALL NOT HAVE ANY RESPONSIBILITY OR LIABILITY FOR THESE OBLIGATIONS. THIS PRESENTATION DOES NOT CONSTITUTE AN OFFER TO SELL OR A SOLICITATION OF AN OFFER TO BUY ANY SECURITIES IN ANY JURISDICTION TO ANY PERSON TO WHOM IT IS UNLAWFUL TO MAKE SUCH AN OFFER OR SOLICITATION IN SUCH JURISDICTION.
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Agenda
• Introduction / 2009 highlights
• Alpharadin positioning and clinical development
• Market opportunity & commercialization
• Alpha-pharmaceutical pipeline
• Outlook & Summary
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• Developing novel targeted therapies for patients with cancer– New-generation ‘alpha-pharmaceuticals’ from world-leading proprietary
technology platform– Tumor-targeted with potent and localized cancer cell killing activity– Potential safety benefits
• Lead candidate Alpharadin in global phase III clinical trial (ALSYMPCA)
– Targeting bone metastases in patients with advanced prostate cancer – an area of high medical need and significant commercial potential
– Overall survival and improved QoL demonstrated in phase II– Remarkable benign side-effect profile
• Management with experience of developing and bringing major cancer products to market
• Strong cash position– Shareholders include major international life sciences and institutional investors
Algeta – An Emerging Force in Oncology
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Q2 & H1 2009 Highlights
• Momentum building in Alpharadin clinical development
– >100 sites in 17 countries now recruiting– Successful end of phase II meeting with FDA (Jan 09) – Preparations underway for enrolment of US patients into ALSYMPCA
– First US patient expected H2 09
• Commercial strategy for Alpharadin progressing
– Pricing market research supports blockbuster commercial potential
• Successful fundraising – NOK 250m / US$37m
– Private placement led by Abingworth, with continued participation by major investors
– Joe Anderson (Abingworth) and Shahzad Malik (Advent) join Board– Supports phase III development
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Q2 & H1 2009 Highlights
• Key appointments enhance commercial and clinical development
– Andrew Kay, President & CEO (ex Renovo, Novartis, AZ)– Gillies O’Bryan-Tear, Chief Medical Officer (ex GSK, Genzyme, BMS)
• Alpha platform progress highlights future pipeline potential
– Academic collaborators presented exciting research at Society for Nuclear Medicine meeting (June 09)
– Coupling alpha-emitter (thorium-227) to monoclonal antibody could extend alpha-pharmaceutical benefits to soft tissue tumors, such as:
– Target specificity– Potent and localized tumor cell-killing– Safety
– Major cancer types targeted initially – Lymphoma - Th227-rituximab – Breast - Th227-trastuzumab
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Key Financials(MNOK)
Profit & Loss Balance sheet
2Q09 2Q08 2009 2008 2Q09 2Q08
Net income 0 0 0 0 Non-current assets 7 7
Payroll 10 7 20 16 Current assets 297 224
of which cash 293 210
Other costs 34 48 65 77
Total assets 304 231
Depreciation 0 0 1 1
Shareholders' equity 251 181
EBIT -45 -56 -86 -94
Current liabilities 53 50
Net financial 4 3 5 6
Total equity & liability 304 231
EBT -41 -53 -81 -87
Market cap 1,467 381
YTDQuarter End quarter
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Agenda
• Introduction / 2009 highlights
• Alpharadin positioning and clinical development
• Market opportunity & commercialization
• Alpha-pharmaceutical pipeline
• Outlook & Summary
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Why target bone metastases?
• Bone metastases occur in up to 90% of certain late-stage cancers− E.g. prostate, breast, lung
• For many patients there isno effective treatment
• Bone metastases profoundly impair patient QoL and further reduce life expectancy
“The morbidity and mortality associated with prostate cancer can
almost universally be attributed to the consequences of metastases to the bone.”
Vassella. (1999) Cancer and Metastasis Reviews 17: 331–336
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Bone metastases: high incidence in major cancers
Incidence in US Incidence in EU
Breast
78,800
Prostate
96,400
Lung
87,700
Sources: prostate metastasis incidence data are company estimates, based on mortality data from SEER for US and Ferlay et al (Annals of Oncology. 2007) for EU. Incidence data for breast and lung cancers in EU are derived from ‘top 5’ incidence data contained in Datamonitor Stakeholder Opinion report published March 2008.
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Alpharadin: highly targeted to bone metastases
• Alpharadin (radium-223 in solution) acts as a calcium mimic – Naturally targets new bone growth in and around bone metastases
• Emits alpha particles that induce irreparable double-strand DNA breaks in adjacent tumor cells– Short penetration nature of alpha emitters (2-10 cell diameters) means highly
localized tumor cell killing and minimal damage to surrounding normal tissue– Effective against chemo- and radio-resistant phenotypes, and hypoxic tumors
• Alpharadin is cleared rapidly into gut
Alpharadin is a new class alpha-pharmaceutical
Targets new bone in metastases Irradiates adjacent tumor cells
Bone marrowTumorcells
Osteoclast
Osteoblast
Newlyformed
bone
Radium-223deposition
Alphaparticleradiation
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Goal: A first choice treatment for bone metastases
Phase I and II clinical trials in over 340 HRPC patients with bone metastases demonstrated:
– Impressive Overall Survival in phase II (>40% increase, p=0.017)– Enhanced Quality of Life– Benign side-effect profile
– Ideal addition to cancer treatment regimes– Controls pain– Easy to use - out-patient procedure
Launch indication – bone metastases in HR prostate cancer
Sources: US market research commissioned from Decision Resources (May 2008) EU market research commissioned from Bridgehead (May 2008)
Market research with leading oncologists suggests Alpharadin hasan “ideal clinical profile” for treating bone metastases
“Clinicians saw many advantages to Alpharadin…and struggled to find disadvantages.”
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Clinical landscape and Alpharadin positioning in HRPC
BONE METASTASES
Alpharadin
HRPC
Approved treatment• Taxotere (Sanofi-Aventis)
Phase III candidates• Abiraterone (Cougar)• Zibotentan (AstraZeneca)• Avastin (Genentech)• Provenge (Dendreon)• XRP6258 (Sanofi-Aventis)• Abraxane (Abraxis BioSciences)
Disease development (time)
Patient diagnosed with HRPC
Bone metastases identified
Hormone dependent Hormone refractory
Non-metastatic Metastatic
BisphosphonatesDenosumab
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BC1-02 phase II: significant increase in survival
• Median overall survival 46.4 weeks in the placebo group and 65.3weeks in the Alpharadin group; 4.5 months difference.Hazard ratio 2.103, p = 0.017.
• 30% (10pts) of the patients were alive at 24 months in the Alpharadin group versus 13% (4 pts) in the placebo group
Published in the Lancet
Phase II BC1-02 trial
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Benign adverse event profile
Alpharadin (n=33) Placebo (n=31)Mild Mod. Severe Total Mild Mod. Severe Total
Diarrhea 6 3 0 9 5 5 0 10
Constipation 6 5 1 12 0 2 0 2
Vomiting 4 4 0 8 3 3 0 6
Nausea 6 3 0 9 6 3 1 10
Fatigue 3 5 0 8 7 0 0 7
Bone pain 2 7 1 10 7 7 2 16
Myalgia 3 2 0 5 3 1 0 4
Tumor flare 2 3 1 6 1 5 1 7
Anemia 0 5 0 5 2 3 2 7
Table lists adverse events in more than 15% of the BC1-02 study population during treatment
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ALSYMPCA: global phase III objectives
– Patients with symptomatic HRPC and bone metastases for whom no cytotoxic drug treatment is planned
– Alpharadin plus best standard of care– Placebo plus best standard of care
– Overall survival
– Time to occurrence of specified disease events– Changes and time to progression in serum PSA and
total ALP concentrations– Acute and long-term safety profile– Quality of life– Health economics
Setting
Study arms
Primary endpoint
Secondary endpoints
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ALSYMPCA: successful meetings with regulators
Europe (EMEA/CHMP)
• ALSYMPCA trial design endorsed during scientific advice in Q4 2007– Overall survival as end-point– Target patient population
• Multiple national reviews concluded successfully in Europe (and RoW)
USA (FDA)
• Highly successful end-of-phase II meeting with FDA (30 Jan. 2009)• US patients to be enrolled into the on-going global ALSYMPCA trial
EMEA: European Medicines AgencyCHMP: Committee for Medicinal Products for Human Use
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ALSYMPCA: Site initiation on track
0
20
40
60
80
100
120
June
Jul
Aug
Sep
t
Oct
Nov
Dec
Jan
Feb
Mar
Apr
May
June
Jul
2008 2009
Active TrialSites
• ALSYMPCA Target 750 patients
• >100 sites in 17 countries now recruiting
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ALSYMPCA: country overview
Australia South America• Brazil
Asia• India
• Hong Kong
• Singapore
North America• Canada
• USA
Europe• UK
• Spain
• Germany
• Czech Republic
• France
• Poland
• Norway
• Slovakia
• Italy
• Sweden
• Belgium
• Netherlands
• Israel
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Agenda
• Introduction / 2009 highlights
• Alpharadin positioning and clinical development
• Market opportunity & commercialization
• Alpha-pharmaceutical pipeline
• Outlook & Summary
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Peak sales potential of $1.3 - 1.9 billion (EU and USA)*
High medical need
• No effective therapy for Taxotere ineligible, refuses and Taxotere failures
• Combination therapy with Taxotere– Alpharadin placebo side-effect
profile & survival data positions it as the ideal combination product
• Effective therapy needed for bone metastases in breast, lung, kidney
Additional upside in sales
• ROW sales
• Rx earlier in prostate cancer for micro metastases
• Lung, renal etc
Life cycle
• $518-761 million
• Breast Cancer
• (Lung, renal etc)
Market growth
• $274-403 million
• HRPC bone metastases
• Combination with Taxotere/others
Launch market
• $590-867 million
• HRPC bone metastases• Taxotere failures
• Taxotere ineligible / refusal
AlpharadinGoal: 1st Choice treatment for patients with bone metastases
Note: Peak Sales forecasts price range $15,000 - $22,000
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Alpharadin: new class alpha-pharmaceutical
• 1st choice treatment for bone metastases
• Huge launch market in HRPC
– premium pricing over branded docetaxel
• Ideal addition to cancer treatment regimes
• Growth from life cycle management – breast, (lung, kidney etc)
USA EU
Medical oncologists √ √
Radiation oncologists √ √
Payers √ √
Regulators (EMEA & FDA) √ √
Strong endorsement from all key stakeholders
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Agenda
• Introduction / 2009 highlights
• Alpharadin positioning and clinical development
• Market opportunity & commercialization
• Alpha-pharmaceutical pipeline
• Outlook & Summary
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• New-generation ‘alpha-pharmaceuticals’ from world-leading proprietary technology platform
– Tumor-targeted with potent and localized cancer cell killing activity– Potential safety benefits
• TH-1 technology– Coupling alpha-emitter (thorium-227) to monoclonal antibodies and
other tumor-targeting molecules– Extend ’alpha’ benefits to soft tissue tumors
• Initial targets and indications• CD20 on lymphoma cells• HER-2 on breast cancer cells
• Exciting research results presented at Society for Nuclear Medicine (June 2009)
Alpha-pharmaceutical pipeline
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Thorium-227 alpha-radioimmunotherapy
Specific binding of mAb to cancer cells
Monoclonal
antibody (mAb)
Thorium-227
Alpha particles from Thorium-227 only kill tumor cells with specific
binding due to the short radiation
range
50-100 µm long, high energy ionizing alpha
track
Alpha particle
(helium nucleus)
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• 227Th-rituximab
• Results presented at SNM meeting in Toronto
– Proof of concept demonstrated– Selective for CD20-positive tumor cells in vitro– Cancer cell killing effect– Complete xenograft regression in 60% mice treated– Therapeutic relevant doses well tolerated in mice– Support previous findings reported in Blood (Dahle J. et al 2007)
Key research findings – lymphoma
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• 227Th-trastuzumab
• First reported results with trastuzumab at SNM meeting in Toronto
– Selectively bound HER-2 presenting tumor cells in vitro and in vivo– Dose-dependent cancer cell killing effect– Tumor regression observed in vivo– Promising safety profile
Key research findings – breast cancer
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Agenda
• Introduction / 2009 highlights
• Alpharadin positioning and clinical development
• Market opportunity & commercialization
• Alpha-pharmaceutical pipeline
• Outlook & Summary
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Algeta outlook 2009
• Continue ALSYMPCA phase III trial recruitment– Initiate clinical trial at sites in USA and RoW
• Continue preparations for commercialization of Alpharadin− Conclude partnering deal in 2009
• Report final data for phase II BC1-03/BC1-04 studies− ECCO (Berlin, Germany: 20-24 Sept 2009)
• Initiate trial for breast cancer with bone metastases– BC1-09, phase II
• Finalise protocol for Taxotere combination HRPC & bone metastases
– BC1-10, phase II
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Summary
• Momentum building in Alpharadin clinical development
• Patient recruitment for ALSYMPCA phase III study on track
• First US site coming online in H2 09 with further sites activating thereafter
• Commercial strategy for Alpharadin progressing – first partnering deal anticipated in 2009
• Exciting pipeline developments from alpha platform
• Strong, experienced management team in place
• Well-funded and supported by leading sector and institutional investors
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Questions
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For further information:
Andrew KayPresident & CEO
Algeta ASAKjelsåsveien 172 APO BOX 54 Kjelsås
NO 0411 OsloNorway
Tel: +47 23 00 79 [email protected]
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