Slide 1: Immunology Pipeline
Evan Beckman, M.D. SVP, Immunology R&D Immunology Pipeline
Slide 2: Safe Harbor Statement
Safe Harbor Statement This presentation contains forward-looking statements about: the sales potential of baminercept alfa (LTbR-Ig) in rheumatoid arthritis the sales potential of RITUXAN(r) (rituximab) in lupus the sales potential of TYSABRI(r) (natalizumab) in Crohn's disease the anticipated devel
Slide 3: Proxy Communication Statement
Proxy Communication Statement Biogen Idec and its directors, executive officers and other members of its management and employees may be deemed to be participants in the solicitation of proxies from the stockholders of Biogen Idec in connection with the Company's 2008 annual meeting of stockholders.
Slide 4: Immunology at Biogen Idec
Immunology at Biogen Idec Experience Veteran team of immunology discovery researchers with record of generating product development candidates (LTbR-Ig, anti-CD40L PEG-Fab, BR3-Fc, anti-BR3 mAb, LFA3-Fc, anti- CD20 mAb, anti-CD23 mAb) BIIB products approved worldwide in a wide range of indications i
Slide 5: Immunology
Immunology Autoimmune RA, SLE, MS, IBD Inflammatory Diseases COPD, Asthma, Atherosclerosis, Metabolic syndrome Infectious Disease Hepatitis Organ Protection Stroke, MI Oncology NHL, CLL, solid tumor Opportunities for Life Cycle Management
Slide 6: Immunology Strategy
Immunology Strategy Disease Lupus Scleroderma Neuro-degenerative RA Asthma IBD Transplant Psoriasis Unmet Need + + + + + + +++ + + + + + + + + + Immune Targets T Cells ^ ^ ^ ^ ^ ^ ^ ^ Adhesion/ Migration ^ ^ ^ ^ ^ ^ ^ ^ B Cells ^ ^ ^ ^ ^ ^ Monocytes/ Dendritic Cells ^ ^ ^ ^ ^ ^ Cytokines/ Chemokines
Slide 7: B Cell Modulation
B Cell Modulation RITUXAN hu Anti-CD20 LTßR-Ig Anti-CD40L Cytokines Chemokines AVONEX(r) LTßR-Ig Anti-TWEAK Tolerance Anti-CD40L Cell Trafficking TYSABRI LTßR-Ig Mechanisms of Interest We have chosen to focus on certain key areas of biology
Slide 8: Biogen Idec Immunology Pipeline
Biogen Idec Immunology Pipeline Stage Phase 3 Phase 2 SLE - Phase 2/3 LN - Ph 3 TNF-IR - Marketed DMARD-IR - Ph3 Phase 1 Market RITUXAN Ocrelizumab (2nd gen. CD20 MAb) Phase 2 Phase 3 Pre-Clinical LTbR-Ig (baminercept alfa) RITUXAN Indication Rheumatoid arthritis Rheumatoid arthritis Lupus - SLE & L
Slide 9: Rheumatoid Arthritis in the United States
Rheumatoid Arthritis in the United States Chronic, progressive disease that results in disability and reduced quality and duration of life The RA biologics market is expected to reach $10B by 2015 Standard of care evolving toward using novel therapies earlier in disease for better outcomes The RA ma
Slide 10: Strategic Opportunities for RA Biologics
Strategic Opportunities for RA Biologics TNF-IR / DMARD-IR Early RA Combination therapy Oral small molecules Personalized approaches RITUXAN, LTbR-Ig, Anti- TWEAK Preclinical candidates Translational medicine
Slide 11: RITUXAN / B Cell Therapies for Rheumatoid Arthritis
RITUXAN / B Cell Therapies for Rheumatoid Arthritis
Slide 12: RITUXAN Mechanism of Action
RITUXAN Mechanism of Action ADCC = antibody-dependent cellular cytotoxicity. CDC = complement-dependent cytotoxicity. Macrophage, monocyte, or natural killer cell CDC Fc?RII, Fc?RIII Cell lysis CD20 CD20 Cell lysis MAC Complement activation (C1qC1rC1s) Apoptosis RITUXAN ADCC B cell
Slide 13: RITUXAN Clinical Development Rheumatoid Arthritis
RITUXAN Clinical Development Rheumatoid Arthritis Anti-TNF Inadequate Responders Anti-TNF Inadequate Responders Methotrexate Naive Methotrexate Inadequate Responders Phase III SERENE (n = 512) Phase III IMAGE Radiographic Study (n = 750) Phase III SUNRISE Controlled Re-treatment Study (n = 555) Phas
Slide 14: LTbR-Ig (baminercept alfa) for Rheumatoid Arthritis
LTbR-Ig (baminercept alfa) A Soluble Lymphotoxin b Receptor for Rheumatoid Arthritis
Slide 15: LTbR-Ig (baminercept alfa; BG9924)
LTbR-Ig (baminercept alfa; BG9924) Fusion protein Human lymphotoxin beta receptor (LTbR) Fc portion of human IgG1 Disulfide-linked glycosylated, dimeric protein
Slide 16: Lymphotoxin Pathway
Lymphtoxin ?/? Pathway is implicated in: Liver, spleen and lymph nodes Gut mucosa and Peyer's patches Surface LTa/b and LIGHT pathways LT? Receptor LT?/? (lymphotoxin) Organization of lymphoid architecture, cellular positioning, etc. Activated lymphocytes and resting B cells Stromal cells, myeloid l
Slide 17: LTbR-Ig Dual Pathway Inhibitor
Lymphtoxin ?/? Pathway is implicated in: Liver, spleen and lymph nodes Gut mucosa and Peyer's patches LIGHT Pathway is implicated in: Mucosal and hepatic inflammation Surface LTa/b and LIGHT pathways LT? Receptor HVEM Receptor LIGHT LT?/? (lymphotoxin) LTbR-Ig Dual Pathway Inhibitor T-cell, dendriti
Slide 18: Synovial Ectopic Lymphoid Structures in RA
RA Synovium Normal Synovium Ectopic lymphoid structures Inhibition of the LT pathway can disrupt ectopic lymphoid organization and limit B- and T-cell activation. LTbR-Ig can disrupt the formation of ectopic lymphoid structures in RA synovium Synovial Ectopic Lymphoid Structures in Chronic Inflammat
Slide 19: LTbR-Ig Phase 2a Data Presented
LTbR-Ig Phase 2a Data Presented Phase 2a data as poster at ACR meeting on November 9th Clinically meaningful ACR responses 8 weeks after the final 4th weekly SC dose
Slide 20: LTbR-Ig (baminercept alfa) Status
LTbR-Ig (baminercept alfa) Status Initiate Phase 2b RA trial in DMARD-IR mid-2007 380 patients, dose ranging trial Primary endpoint ACR50 at 3 months Initiate Phase 2b RA trial in TNF-IR mid-2007 120 patients Primary endpoint ACR50 at 3 months
Slide 21: Systemic Lupus Erythematosus in the US
Systemic Lupus Erythematosus in the US Chronic autoimmune disorder which may affect virtually any part of the body and can range in severity from mild to life- threatening An estimated 425,000 diagnosed patients in US High unmet need due to limited efficacy of current therapies Long-term use of curr
Slide 22: RITUXAN / B Cell Therapies for SLE / Lupus Nephritis
RITUXAN / B Cell Therapies for Systemic Lupus Erythematosus / Lupus Nephritis
Slide 23: RITUXAN Clinical Development SLE and Lupus Nephritis
RITUXAN Clinical Development SLE and Lupus Nephritis Lupus Nephritis Systemic Lupus Erythematosus Phase II/III EXPLORER (n=260) Phase III LUNAR (n=140) Initiated Q2-05 Completed enrollment Q1-07 H1 2008 Initiated Q1-06 Completed enrollment Q4-07 2009 Patient Population Lupus Program Status Phase Stu
Slide 24: BILAG Index in EXPLORER
BILAG Index in EXPLORER EXPLORER Primary Objective - summary To assess the efficacy of rituximab compared to placebo in achieving and maintaining clinical response based on monthly BILAG assessments to week 52 BILAG-2004 Index (Disease Activity Instrument) An index that assesses 97 clinical signs, s
Slide 25: TYSABRI for Crohn's Disease
TYSABRI for Crohn’s Disease 1 biogen idec
Slide 26: Crohn's Disease & Market Opportunity
Crohn's Disease & Market Opportunity Crohn's Disease Chronic and progressive inflammatory disease of the gastrointestinal tract Unmet medical need as many patients fail to respond to current therapies ~500,000 CD patients in the US TYSABRI(r) Market Opportunity Estimated 40,000 - 50,000 CD patients
Slide 27: TYSABRI Induction Data is Comparable to Anti-TNFs
TYSABRI Induction Data is Comparable to Anti-TNFs Placebo N=25 5 mg/kg N=27 10 mg/kg N=28 20 mg/kg N=28 Placebo N=74 40/20 mg N=74 80/40 mg N=75 160/80 mg N=76 Placebo N=73 100 mg N=74 200 mg N=72 400 mg N=72 Placebo N=250 300 mg N=259 Observed 16 81.5 50 64.3 37 54 59 59 31.1 41.9 54.2 54.2 37 51 T
Slide 28: TYSABRI Maintenance Data Compares Favorably to Anti-TNFs
TYSABRI Maintenance Data Compares Favorably to Anti-TNFs ENACT-2 (300 mg/q4w) CHARM (40 mg/eow) ACCENT 1 (5 mg/kg q8w) REMICADE HUMIRA TYSABRI P=0.007 P<0.001 P<0.001 Remission at 1 year Competing Therapy TYSABRI Placebo
Slide 29: Concomitant Immunosuppressives are Not Needed to Maintain Remission
Month 6 Month 12 placebo (ITT) 30 22 natalizumab (ITT) 55 55 placebo (-IMM) 31 21 natalizumab (-IMM) 54 53 placebo (+IMM) 30 23 natalizumab (+IMM) 58 60 - - IMM - - IMM + IMM + IMM Month 6 Month 12 ITT ITT P ^ 0.002 for all comparisons with placebo Placebo TYSABRI Concomitant Immunosuppressives are
Slide 30: TYSABRI Crohn's Disease
TYSABRI Crohn's Disease TYSABRI(r) approved in US on January 14th 2008 Approved for inducing and maintaining clinical response and remission in adult patients with moderately to severely active Crohn's disease (CD) with evidence of inflammation who have had an inadequate response to, or are unable t
Slide 31: Immunology Development Summary
Immunology Development Summary Biogen Idec currently has important marketed products targeting key immunologic pathways Unmet need remains in many autoimmune diseases Leveraging our core expertise in immunology, next generation projects will bring further value in autoimmune disease
Slide 32: Cardiopulmonary and Emerging Therapeutic Areas
Cardiopulmonary and Emerging Therapeutic Areas
Slide 33: Acute Heart Failure
Acute Heart Failure Disease Characteristics & Unmet Medical Need Heart failure patients can't pump enough blood to the body's other organs Significant morbidity and mortality Five year mortality higher than most cancers Frequent cause of hospitalizations Hyponatremia and renal complications extend t
Slide 34: Acute Heart Failure Therapies
Acute Heart Failure Therapies Current therapeutic approaches Fluid restriction to remove water Only current therapy for hyponatremic heart failure patients Diuretics remove water, but also remove sodium Can induce hyponatremia and worsen renal function Positive inotropes inhibit sodium pump, increas
Slide 35: Cardiopulmonary & Emerging Therapeutic Area Pipeline
Cardiopulmonary & Emerging Therapeutic Area Pipeline Stage Acute - Phase 2 Chronic - Phase 2 Phase 1 Market Phase 2 Phase 3 Pre-Clinical ADENTRI Indication Acute Heart Failure - IV Chronic Heart Failure - Oral Phase 2 Aviptadil Preclinical Long Acting rFactor IX Pulmonary arterial hypertension Hemop
Slide 36: Lixivaptan
Lixivaptan Late stage asset lixivaptan Collaboration agreement with Cardiokine announced July 2007 Selective oral V2 vasopressin receptor antagonist Promising Phase 2 activity for hyponatremia (low plasma sodium concentration) Phase 3 recently initiated Randomized, placebo controlled, double blind,
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Slide 38: Research and Development
Cecil B. Pickett, Ph.D. President, R&D Research and Development
Slide 39: Safe Harbor Statement
Safe Harbor Statement This presentation contains forward-looking statements about: the anticipated development and timing of programs in our clinical pipeline our expected filings with regulatory agencies In addition, in the course of the presentation, we may provide additional information of a forw
Slide 40: Proxy Communication Statement
Proxy Communication Statement Biogen Idec and its directors, executive officers and other members of its management and employees may be deemed to be participants in the solicitation of proxies from the stockholders of Biogen Idec in connection with the Company's 2008 annual meeting of stockholders.
Slide 41: Agenda
Agenda R and D Strengths R and D Strategy Biogen Idec Pipeline 2008 R and D Goals Q & A
Slide 42: Biogen Idec's R and D Strengths
Biogen Idec's R and D Strengths Strong R&D fundamentals World class biotherapeutic discovery and development organization Focused drug discovery and development efforts: Neurology Immunology Oncology Cardiopulmonary & Emerging areas Strong link between discovery research, clinical development, and s
Slide 43: R and D Strategy
R and D Strategy Focus on the discovery and development of novel therapeutics to address areas of high unmet medical need Recommend six new development candidates each year: Biologics and Small Molecules Internal Discoveries and in-licensing opportunities Innovative First in Class Molecules as well
Slide 44: Biogen Idec Pipeline
Biogen Idec Pipeline Robust Pipeline: 3 new indication programs for Rituxan 5 novel phase III programs by year end 8 phase II programs 10 pre-clinical/phase I programs A good mix of biologics and small molecules Both internally discovered and in-licensed compounds in development
Slide 45: Biogen Idec Pipeline Late Development
Biogen Idec Pipeline Late Development Program Status Indication BG-12 Phase 3 RRMS Galiximab Phase 3 Relapsed NHL Lumiliximab Phase 3 Relapsed CLL Lixivaptan Phase 3 Acute heart failure with hyponatremia Adentri (IV) Phase 3 in 2008 Acute decompensated congestive heart failure Program Status Indicat
Slide 46: Biogen Idec Pipeline Early Development
Biogen Idec Pipeline Early Development Program Status Indication Anti-IGF-1R Phase 1 Solid Tumors Anti-Cripto-DM4 Preclinical Solid Tumors Hsp90 Inhibitor 3647 Preclinical Solid Tumors Tysabri (natalizumab) Preclinical Multiple Myeloma Raf Inhibitor Preclinical Solid Tumors CD40L-Fab Preclinical SLE
Slide 47: 2008 R and D Goals
2008 R and D Goals Advance registrational studies to achieve accelerated approval dates: Lumiliximab in relapsed CLL Lixivaptan in acute heart failure with hyponatremia BG-12 in RRMS Galiximab in relapsed NHL Adentri (IV) in ADHF Advance proof of concept programs to next decision point: BIIB14 in Pa
Slide 48: 2008 R and D Goals
2008 R and D Goals Advance early stage pipeline cont. Initiate first in human studies Anti-CRIPTO-DM4 in solid tumors Anti-IGF-1R in solid tumors Anti-TWEAK in RA Hsp90 Inhibitor (3647) in solid tumors Neublastin in neuropathic pain Long Acting rFactor IX in hemophilia B Initiate proof of concept st
Slide 49: Data Readouts to Year End 2008
Data Readouts to Year End 2008 Completed: Daclizumab Phase 2 CHOICE in Relapsing Remitting MS Baminercept Phase 2a in RA Rituxan Phase 3 SERENE in DMARD-IR RA Still to Come: Rituxan Phase 2/3 EXPLORER in SLE Rituxan Phase 2/3 OLYMPUS in Primary Progressive MS Baminercept alfa Phase 2b in RA BIIB14 P
Slide 50: Summary
Summary Outstanding people at all levels in the R and D Organization Robust pipeline with important compounds at all stages of development World-class expertise in discovery and development of biologics Building capability in small molecule discovery and development NEED TO FOCUS ON ADVANCING PIPELI