BIO CEO and Investor Conference February 10, 2009 Sylvie...

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BIO CEO and Investor Conference February 10, 2009 Sylvie Grégoire President, Shire HGT

Transcript of BIO CEO and Investor Conference February 10, 2009 Sylvie...

  • BIO CEO and Investor ConferenceFebruary 10, 2009

    Sylvie Grégoire President, Shire HGT

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    THE “SAFE HARBOR” STATEMENT UNDER THE PRIVATE SECURITIES LITIGATION REFORM ACT OF 1995 Statements included herein that are not historical facts are forward-looking statements. Such forward-looking statements involve a number of risks and uncertainties and are subject to change at any time. In the event such risks or uncertainties materialize, the Company’s results could be materially adversely affected. The risks and uncertainties include, but are not limited to, risks associated with: the inherent uncertainty of research, development, approval, reimbursement, manufacturing and commercialization of the Company’s Specialty Pharmaceutical and Human Genetic Therapies products, as well as the ability to secure and integrate new products for commercialization and/or development; government regulation of the Company’s products; the Company’s ability to manufacture its products in sufficient quantities to meet demand; the impact of competitive therapies on the Company’s products; the Company’s ability to register, maintain and enforce patents and other intellectual property rights relating to its products; the Company’s ability to obtain and maintain government and other third-party reimbursement for its products; and other risks and uncertainties detailed from time to time in the Company’s filings with the Securities and Exchange Commission.

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    Product Information

    Today’s presentation include information about certain products that are not approved for marketing in the United States. This information is for investor purposes only, and should not be relied upon by health care professionals. It is intended to be informative with respect to Shire HGT’s pipeline, and is not in any way intended to be promotional in nature.

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    Shire Purpose Statement

    We enable people with life-altering conditions to

    lead better lives.

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    Shire HGT Business Model SupportsStrong Growth

    Rare diseases with unmet need

    High impact Rx / high margin products

    Global development / commercialization

    3 Marketed products

    Pipeline gaps filled

    8 potential launches by 2015

    #1 or #2 in our markets

    Growth via internal Research and BD

    Strong RoW growth with minimal infrastructure

    BUSINESSMODEL

    PORTFOLIOSTRENGTH

    FUTUREGROWTH

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    Orphan Disease Landscape ProvidesSignificant Opportunities for Development

    Infectious Diseases

    9%

    Neuro logical Disorders

    11%Other

    11%

    Cancer and related

    47%

    Transplantation2%Cardiovascular

    Diseases2%

    Blood Disorders

    1%

    Respiratory Disorders

    4%

    Auto immune Disorders

    5%

    Genetic Disorders

    8%

    ~ 7,000 orphan diseases

    Individually rare but collectively affect 1 in 10

    25 million Americans

    25-30 million EU

    50% Pediatric

    85% Serious or life-threatening

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    Long Term Ratios Reflect R&D Investment with Minimal Commercial Infrastructure

    Spec Pharma HGT Total Spec Pharma HGT TotalTotal sales % 85% 15% 100% 70% 30% 100%

    Gross Margin 85% 89% 86% 86% 87% 86%

    R&D (14%) (47%) (19%) (14%) (25%) (17%)

    S&M (37%) (19%) (35%) (31%) (16%) (26%)

    G&A - - (11%) - - (9%)

    EBITDA Margin 34% 23% 21% 41% 46% 34%

    2007 TARGET

    Note: All expense ratios are a percentage of product sales

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    HGT’s Global Revenue Diversification Meets Corporate Milestone in 2008

    2015 Total: $1,706

    U.S.A Revenue, 17%

    EU5 + Canada Revenue, 42%

    RoW Revenue, 41%

    0%

    5%

    10%

    15%

    20%

    25%

    30%

    35%

    40%

    45%

    50%

    2008 Total: $505

    U.S.A Revenue, 15%

    EU5 + Canada Revenue, 60%

    RoW Revenue, 25%

    0%

    10%

    20%

    30%

    40%

    50%

    60%

    70%

    USA Revenue20%

    RoW Revenue40%

    EU5 + CanadaRevenue 40%

    USA Revenue15%

    EU5 + CanadaRevenue 60%

    RoW Revenue25%

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    CommercialPh II/IIIPh I/IIPreclinical IND Tox

    POC

    HGT Pipeline in October 2007

    REPLAGAL®Fabry Disease

    ELAPRASE®Hunter Syndrome

    VelagluceraseGaucher Disease

    HGT 2310 Idursulfase-IT/Hunter CNSHGT 1410 Sanfilippo ERT

    HGT 1110MLD ERT

    Marketed Product Support

    Grow ERT Franchise

    New Technology: CNS Diseases

    Research

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    CommercialPh II/IIIPh I/IIPreclinical IND Tox

    POC

    HGT Pipeline Gaps Filled Through Recent BD and Research Success

    REPLAGAL®Fabry Disease

    ELAPRASE®Hunter Syndrome

    VelagluceraseGaucher Disease

    HGT 2310 Idursulfase-IT/Hunter CNSHGT 1410 Sanfilippo ERT

    HGT 1111MLD ERT

    HGT 3310 (AMIGAL™)Fabry Disease

    HGT 3410 (PLICERA™)Gaucher Disease

    HGT 3510 (AT2220)Pompe Disease

    Marketed Product Support

    Grow ERT Franchise

    New Platform: Pharmacological

    Chaperones

    New Technology: CNS Diseases

    Research

    Expand pipeline through Internal Research Capability

    New in past 12 mo

    HGT 2610Krabbe/GLD ERT

    FIRAZYR®*HAE

    Expansion to New Products and Targets

    * Shire owns a 98% interest in Jerini AG; FIRAZYR® is a registered trademark of Jerini AG

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    Prioritization CriteriaRare Diseases

    • Competition• Proof of Concept• Development Feasibility• IP/Exclusivity

    • Prevalence• Unmet Need• Commercial Fit

    Selecting Products for In-Licensing/Acquisition

    Disease Criteria

    Product Criteria

    Business Development Opportunities

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    Shire HGT- Key Strengths

    Fully integrated business, exclusively focused on rare diseases

    Global reach – development, regulatory, commercial

    Demonstrated success in bringing rare disease products to market

    Simultaneous global filings; early market access

    Setting new standards for MPS launches with ELAPRASE®

    Combine entrepreneurial culture with sufficient resources to achieve success

    Why we are a good partner

  • Replagal

    67

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    Fabry Market Continues to Demonstrate Strong Growth

    Source: EvaluatePharma

    $0

    $100

    $200

    $300

    $400

    $500

    $600

    $700

    2001 2002 2003 2004 2005 2006 2007

    $M

    2001-2007 CAGR 99%

  • Elaprase

    72

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    Manage New Patient Accruals

    Early TX and CNS Patients

    Maintain Compliance

    Geographic expansion

    Customer Service Model

    Focus on early treatment

    Published case studies

    HOS working groups

    Hunter-CNS program

    Persistency programs

    Publish Long Term Clinical Data

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  • 18Efficacy

    Goal: To establish FIRAZYR as a new global standard of care for HAE patients

    Safety Convenience

    Treats attacks quickly

    Treats all kinds of attacks

    Not a “blood derived”product

    Not immunogenic

    Pre-filled “Ready toUse” Syringe

    Subcutaneous dosing

    Room temp storage

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    HGT Revenue Growth Reflects Launch of 8 New Products*

    20112008 20102009 2012 - 2015

    *Subject to regulatory approvals

    REPLAGAL

    ELAPRASE

    FIRAZYR

    velaglucerase

    MLD/1111

    AMIGAL

    Pompe/3510

    PLICERA

    Hunter CNS

    Sanfilippo/1410

    REPLAGAL

    ELAPRASE

    FIRAZYR

    velaglucerase

    MLD/1111

    REPLAGAL

    ELAPRASE

    FIRAZYR

    velaglucerase

    REPLAGAL

    ELAPRASE

    FIRAZYR

    REPLAGAL

    ELAPRASE

    FIRAZYR

    Krabbe/2610

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    Shire HGT Positioned for Strong Growth

    LYSOSOMALSTORAGEDISEASES

    LYSOSOMALSTORAGEDISEASES

    LYSOSOMALSTORAGEDISEASES

    RAREGENETIC

    DISEASES

    RAREGENETIC

    DISEASES

    ORPHANDISEASESShir

    e HGT’s F

    uture Gr

    owth

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    To be as brave as the people we help.

  • Questions

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    HGT Key Criteria for Long Term Revenue Growth

    REPLAGAL® - approved in 44 countriesELAPRASE® - approved in 43 countriesFIRAZYR® - approved in 30 countries

    HGT Pipeline:velaglucerase alfaHGT-1111 (MLD)AMIGAL (ex-US)PLICERA (ex-US)HGT- 3510 (Pompe, ex-US)HGT-1410 (Sanfilippo-A)HGT- 2310 (Hunter-CNS)HGT-2610 (Krabbe)

    Strong IP/Orphan

    Speed of Registration

    Premium Pricing

    High Levels of Reimbursement

    Unmet Niche Markets

    Symptomatic Disease

    Global infrastructure

    HGT Global Products