Regeneron Pharmaceuticals, Inc. · NASDAQ:REGN
Regeneron Pharmaceuticals — 27th Annual J.P. Morgan Healthcare Conference 2009
36 slides from Regeneron Pharmaceuticals's conference presentation, Jan 9, 2009
Slide 1: 27th Annual J.P. Morgan Healthcare Conference January 2009
27th Annual J. P. Morgan Healthcare Conference January 2009
Slide 2: Safe Harbor Statement
Except for historical information, the matters contained in this presentation may constitute forward-looking statements that involve risks and uncertainties, including uncertainties related to product development and clinical trials, unforeseen safety issues resulting from the administration of prod
Slide 3: Regeneron Overview
Regeneron Overview ARCALYST(r) (rilonacept) marketed for treatment of orphan auto-inflammatory disease (CAPS) Broad Development Pipeline - 9 Phase 3 trials by mid- 2009 Oncology - Symptomatic malignant ascites data in mid-2009; 4 Phase 3 programs enrolling patients Eye Disease - Phase 3 program in w
Slide 4: Broad Development Pipeline
ARCALYST(r) (rilonacept) CAPS Gout Flare Prevention Phase 1 1st-line metastatic prostate cancer 2nd-line metastatic non-small cell lung cancer 2nd-line metastatic colorectal cancer 1st-line metastatic pancreatic cancer VEGF Trap-Eye Phase 2 Phase 3 Marketed REGN88 (IL-6R antibody) REGN421 (Dll4 anti
Slide 5: ARCALYST (rilonacept)
REGENERON ARCALYST® (rilonace pt) SEEEN
Slide 6: ARCALYST Approved and Launched in 2008
ARCALYST(r) (rilonacept) Approved and Launched in 2008 ARCALYST(r) (rilonacept) is the only therapy approved for treatment of Cryopyrin-Associated Periodic Syndromes (CAPS) Rare, inherited, auto-inflammatory diseases Familial Cold Auto-inflammatory Syndrome (FCAS) Muckle-Wells Syndrome (MWS) Approve
Slide 7: Rilonacept in Gout: Unmet medical need
Rilonacept in Gout Unmet medical need 1% of US population suffers from gout - one of the most painful rheumatic diseases > 750,000 gout patients start on allopurinol each year to lower uric acid levels; during first months of therapy many experience acute flares of joint pain and inflammation 1.4MM
Slide 8: Primary Endpoint: Mean Number of Gout Flares per Patient through Week 12
Phase 2 Primary Endpoint: Mean Number of Gout Flares per Patient through Week 12 Placebo (n=42) Rilonacept (n=41) 0.79 0.15 Mean number of gout flares per patient 0.15 p = 0.0011 (33 flares) (6 flares) 0.79 81% ^
Slide 9: Proportion of Patients with a Gout Flare through Week 12
Phase 2 Proportion of Patients with a Gout Flare through Week 12 Placebo (n=42) Rilonacept (n=41) 45 15 Proportion of patients with a gout flare p < 0.004 45.2% 14.6% (19 patients) (6 patients) 9 of 42 > 1 flare 0 of 41 > 1 flare
Slide 10: Rilonacept Program in Gout
Phase 3 Rilonacept Program in Gout Gout flares induced by urate-lowering drugs Two studies: rilonacept vs. placebo Acute gout rilonacept vs. NSAIDs vs. NSAIDs plus rilonacept Safety study Program to start 1Q09
Slide 11: Aflibercept (VEGF Trap)
7» REGENERON Aflibercept (VEGF Trap)
Slide 12: Aflibercept Phase 3 Oncology Program
Aflibercept (VEGF Trap) Phase 3 Oncology Program Partnered with sanofi-aventis All studies approximately 1/3 enrolled Each study monitored by Independent Data Monitoring Committee Total Number of Patients to be Enrolled Initial Data Expected VELOUR study: 2nd line metastatic colorectal cancer (+ fol
Slide 13: Aflibercept Single-Agent Phase 3 - SMA
Aflibercept Single-Agent Phase 2 - SMA Symptomatic Malignant Ascites (SMA) in Advanced Ovarian Cancer (AOC) Condition in which fluid containing cancer cells collects in the abdomen Treated by paracentesis - procedure in which fluid is directly drained from the abdomen Randomized, placebo-controlled
Slide 14: Aflibercept Summary
Aflibercept Summary SMA target enrollment reached Data expected mid-2009 4 Phase 3 studies approximately 1/3 enrolled Initial data expected 2010 Additional studies Colorectal cancer Phase 2 study: First line treatment with FOLFOX6+/- aflibercept recruiting patients Over 1600 patients treated with af
Slide 15: VEGF Trap-Eye
> REGENERON VEGF Trap-Eye cy
Slide 16: VEGF Trap-Eye - The Opportunity
VEGF Trap-Eye - The Opportunity Potential differentiation relative to standard of care being explored in patients with neovascular form of age-related macular degeneration (wet AMD) Possible visual acuity gains Possible less frequent dosing with similar gain in vision Potential maintenance of visual
Slide 17: Ranibizumab Findings in Wet AMD
Ranibizumab Findings in Wet AMD 12-Month Change in Visual Acuity vs. Baseline (Letters) Infrequent Dosing Monthly Dosing 12-Month Change in Visual Acuity vs. Baseline (Letters)
Slide 18: CLEAR-IT 2: Phase 2 Study Design
CLEAR-IT 2: Phase 2 Study Design 0.5 mg q4 wks VEGF Trap-Eye 0 4 8 12 Weeks 0.5 mg q12 wks VEGF Trap-Eye 2 mg q4 wks VEGF Trap-Eye 2 mg q12 wks VEGF Trap-Eye 4 mg q12 wks VEGF Trap-Eye Primary and Secondary Endpoints 16 prn ....1 year prn ....1 year prn ....1 year prn ....1 year prn ....1 year
Slide 19: Encouraging 52-Week Phase 2 Results for VEGF Trap-Eye in Wet AMD
Encouraging 52-Week Phase 2 Results for VEGF Trap-Eye in Wet AMD Significant improvements in visual acuity and retinal thickness through one year 9.0 letter average gain for 2.0 mg q4 week dose at 52 weeks After week 12, patients from all dose groups, on average, required only two additional injecti
Slide 20: 52-Week Mean Change in Visual Acuity
VEGF Trap-Eye Phase 2 Study 52-Week Mean Change in Visual Acuity Change in Visual Acuity (letters) 0 4 8 12 16 20 24 28 32 36 40 44 48 52 0.5q4 0 5 7.1 8.8 9.3 8.6 7.6 7.3 8 8.1 6.9 5.7 5.5 5.4 2q4 0 6.5 6.2 8.3 10 9.9 9.5 9.5 10.1 9.1 9.7 8.4 7.2 9 9.0* 5.4† 0.5q4 2q4 *P<0.0001 †P >0.05 Fixed-dosin
Slide 21: 52-Week Mean Change in Retinal Thickness
VEGF Trap-Eye Phase 2 Study 52-Week Mean Change in Retinal Thickness Posterior Pole Scans, LOCF analysis. 0.5q4: n=32; 2q4: n=31 0 4 8 12 16 20 24 28 32 36 40 44 48 52 0.5q4 0 -123.7 -144.2 -153.5 -163.3 -148.2 -145.2 -135.9 -140.8 -137.3 -135.8 -118.7 -129.6 -125 2q4 0 -151.3 -177.1 -169.2 -182.7 -
Slide 22: Mean Change in Total Active CNV Size
Mean Change in Total Active CNV Size through 48 weeks by Fluorescein Angiography 0 4 8 12 16 20 24 28 32 36 48 0.5q4 0 -1.73 -1.73 -1.26 -1.29 -1.12 -1.55 -1.53 -1.43 -1.72 -1.42 2q4 0 -1.83 -1.61 -1.64 -1.55 -1.76 -2.52 -2.41 -2.62 -3.07 -3.41 Week Change From Baseline (mm2) - -1.42** 0.5q4 Mandato
Slide 23: VEGF Trap-Eye: Two Phase 3 Studies Underway
VEGF Trap-Eye Phase 3 Program in Wet AMD VIEW 1 enrolling 1200 patients in U.S. and Canada VIEW 2 enrolling 1200 patients in rest of world 4 arms in both trials - - VEGF Trap-Eye - ranibizumab - 0.5 mg q4 weeks - 0.5 mg q4 weeks - 2.0 mg q4 weeks - 2.0 mg q8 weeks (after 3 monthly doses) Fixed dosin
Slide 24: VEGF Trap-Eye Phase 2 Program in DME
VEGF Trap-Eye Phase 2 Program in DME Double-masked, randomized, controlled study 5 arms: 4 VEGF Trap-Eye regimens vs. laser treatment Enrolling approximately 200 patients in U.S., Canada, EU and Australia 52 weeks of treatment with 6 month additional safety follow-up Primary efficacy endpoint: Chang
Slide 25: Antibodies
) SS REGENERON Antibodies (4 Fe ae Pap LEP AN ee nip i tS \ se aoe > \ a , \ : & / + os yt, 3 y € q 1 ~ \ Y 49
Slide 26: VelociSuite: Target Identification and Validation
VelociSuite of Technologies: Target Identification and Validation Together, used to identify and validate drug targets: Rapidly replaces gene with reporter to see where gene is active Shows the result of deleting or adding extra copies of genes Allows direct testing in mammalian models of whether th
Slide 27: VelociSuite: Human Antibody Generation and Manufacturing
VelociSuite of Technologies: Human Antibody Generation and Manufacturing Genetically humanized over 6 megabases of mouse immune genome VelocImmune mice mount a robust immune response and generate antibodies as efficiently as normal mice Antibodies with most desirable therapeutic characteristics are
Slide 28: Sanofi-Aventis Antibody Collaboration
Global collaboration to discover, develop, and commercialize therapeutic human antibodies Sanofi-aventis funds $475 million of discovery research over five years through 2012 Sanofi-aventis funds 100% of development costs for collaboration antibodies Goal is to bring average of 2 to 3 new antibodies
Slide 29: REGN88: IL-6R Antibody
REGN88: IL-6R Antibody Phase 1 trial initiated in rheumatoid arthritis IL-6 plays important role in regulation of inflammatory and immune responses Clinical proof-of-concept for IL-6R antibody in rheumatoid arthritis demonstrated by Roche's tocilizumab - FDA approval pending Regeneron antibody is hi
Slide 30: REGN421: Dll4 Antibody
REGN421: Dll4 Antibody Delta-like ligand 4 inhibition being studied for its role in anti-angiogenesis IND filed December 2008 Phase 1 single-agent, dose-ranging study to begin in patients with advanced malignancies
Slide 31: REGN475: Selective Anti-NGF Antibody
REGN475: Selective Anti-NGF Antibody Clinical proof-of-concept for nerve growth factor (NGF) inhibition in treatment of pain demonstrated by Pfizer's tanezumab, currently in Phase 3 trials for osteoarthritis Challenge: Obtain an antibody that binds NGF selectively without cross-reacting, even at hig
Slide 32: Expanding Research and Development, Clinical, and Manufacturing
Expanding Research and Development, Clinical, and Manufacturing Regeneron's headquarters and research laboratories located in Tarrytown, NY 230,000 SF of world-class lab and office space under construction Expected occupancy in mid-2009 Biologics manufacturing capacity being expanded from 22,000 to
Slide 33: Strong Financial Position
Strong Financial Position Year-end 2008 cash and securities: ~$528MM No debt: In 2008, retired $200MM of convertible notes
Slide 34: Monetizing Technology
Monetizing Technology VelociGene(r) Knock-Out and Transgenic Models sanofi-aventis (August 2008) Terms: $4.3MM/year for 5 years VelocImmune(r) Technology Licenses Corporate Astellas Pharma (March 2007) and AstraZeneca/Cambridge Antibody Technology (February 2007) Terms: $20MM/year each for up to 6 y
Slide 35: Collaboration Agreements
Collaboration Agreements sanofi-aventis Bayer HealthCare sanofi-aventis - Upfront/milestone payments $130MM $95MM $85MM - Development costs paid by partner * 100% ~50% 100%** - Profit split - Regeneron share US 50% 100% 50% 100% Japan ~35% royalty 50% 35-45% 100% ROW 50% 50% 35-45% 100% Milestones r
Slide 36: 27th Annual J.P. Morgan Healthcare Conference January 2009
27th Annual J.P. Morgan Healthcare Conference January 2009
Regeneron Pharmaceuticals — 27th Annual J.P. Morgan Healthcare Conference 2009 on 6ix