Alnylam Pharmaceuticals, Inc. · NASDAQ:ALNY
Alnylam Pharmaceuticals — Q3 2021 Earnings Presentation
39 slides from Alnylam Pharmaceuticals's earnings presentation, Oct 28, 2021
Slide 1: Third Quarter 2021 Financial Results
Glaucienne Diagnosed with AHP (Brazil) Third Quarter 2021 Financial Results October 28, 2021 © 2021 Alnylam Pharmaceuticals, Inc. 1
Slide 2: Agenda
Agenda Welcome • Christine Lindenboom Senior Vice President, Investor Relations & Corporate Communications Overview • John Maraganore, Ph.D. Chief Executive Officer Leadership Transition • Yvonne Greenstreet, MBChB, MBA President and Chief Operating Officer Commercial Highlights • Tolga Tanguler Chi
Slide 3: Forward Looking Statements & Non-GAAP Financial Measures
Alnylam Forward Looking Statements & Non-GAAP Financial Measures This presentation contains forward-looking statements within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934, including expectations regarding our aspiration to become a l
Slide 4: Overview
John Maraganore, Ph.D. Chief Executive Officer Overview 4
Slide 5: Transformational Medicines, Robust R&D Pipeline, High-Yield Product Engine
Mayah Diagnosed with PH1 (USA) TRANSFORMATIONAL ROBUST & HIGH-YIELD ORGANIC 5 MEDICINES R&D PIPELINE PRODUCT ENGINE
Slide 6: John Maraganore and Yvonne Greenstreet
John Maraganore Yvonne Greenstreet Chief Executive Officer President & Chief Operating Officer 6
Slide 7: Leadership Transition
Yvonne Greenstreet, MBChB, MBA President and Chief Operating Officer Leadership Transition 7
Slide 8: Our New 5-Year Strategy
Our New 5-Year Strategy Patients: Over 0.5 million on Alnylam RNAi therapeutics globally Products: 6+ marketed products in rare and prevalent diseases Pipeline: Over 20 clinical programs, with 10+ in late stages and 4+ INDs per year Performance: ≥40% revenue CAGR through YE 2025 Profitability: Achie
Slide 9: Please join us for Alnylam R&D Day
Please join us for “D/Alnylam R&D. Day November 19, 2021 AVIRTUAL EVENT Registration information coming soon.
Slide 10: Commercial Highlights
Tolga Tanguler Chief Commercial Officer Commercial Highlights 10
Slide 11: ONPATTRO Launch Update: Q3 2021
ONPATTRO® (patisiran) Launch Update: Q3 2021 Steady and Continued Growth $120M >1,875 Q3 U.S. Highlights Steady, continuous patient growth; notable growth in ONPATTRO Global Q3 2021 Patients Worldwide on Commercial demand and new prescribers Net Product Revenues ONPATTRO at end of Q3 2021 Increase d
Slide 12: ONPATTRO Global Commercialization
ONPATTRO Global Commercialization Increasing Access and Value Recognition • Progress with global ONPATTRO availability UNITED STATES CEMEA LATIN AMERICA ASIA ‒ Over 30 countries now selling ONPATTRO through direct reimbursement, named patient sales, or reimbursed expanded access ‒ Pricing and reimbu
Slide 13: GIVLAARI Launch Update: Q3 2021
GIVLAARI® (givosiran) Launch Update: Q3 2021 Continued Progress with Uptake and Access $32M >300 Q3 U.S. Highlights >10 Value-Based Agreements (VBAs) finalized GIVLAARI Global Q3 2021 Patients Worldwide on Commercial Net Product Revenues GIVLAARI at end of Q3 2021 >94% covered U.S. lives with confir
Slide 14: GIVLAARI Global Commercialization
GIVLAARI Global Commercialization Ensuring GIVLAARI Availability Around the World • Progress with global GIVLAARI availability UNITED STATES CEMEA LATIN AMERICA ASIA ‒ Recent launches in Japan and Spain ‒ Pricing and reimbursement achieved in UK and France 14 CEMEA: Canada, Europe, Middle East, Nort
Slide 15: OXLUMO Launch Update: Q3 2021
OXLUMO® (lumasiran) Launch Update: Q3 2021 Continuous Launch Execution $15M >120 Q3 U.S. Highlights Net growth in new patients; revenues reflect OXLUMO Global Q3 2021 Patients Worldwide on Commercial transition of initial bolus of commercial patients from Net Product Revenues OXLUMO at end of Q3 202
Slide 16: OXLUMO Global Commercialization
OXLUMO Global Commercialization Ensuring OXLUMO Availability Around the World • Progress with global OXLUMO availability UNITED STATES CEMEA LATIN AMERICA ASIA ‒ Launches underway in Germany and France ‒ Timely transitions from Expanded Access Program ‒ Broad utilization across age groups and EGFR c
Slide 17: Alnylam Pipeline
Akshay Vaishnaw, M.D., Ph.D. President of R&D Alnylam Pipeline 17
Slide 18: Patisiran Phase 3 Study in ATTR Cardiomyopathy
Patisiran Phase 3 Study Randomized, Double-Blind, Placebo-Controlled Study in ATTR Amyloidosis Patients with Cardiomyopathy N ~ 300 Primary Endpoint Patient Population Patisiran • Change in 6-MWT at 12 months IV q3w† 1:1 RANDOMIZATION • ATTR amyloidosis; wild-type or 0.3 mg/kg any TTR mutation Key S
Slide 19: Vutrisiran HELIOS-A Phase 3 Study
Vutrisiran Phase 3 Study Randomized, Open-Label Study in Hereditary ATTR Amyloidosis Patients with Polyneuropathy N ~ 164 Vutrisiran 3:1 RANDOMIZATION Patient Population SC q3M 25 mg • hATTR amyloidosis; 9-Month Efficacy 18-Month Efficacy any TTR mutation • Assessment vs. • Assessment vs. Open-Label
Slide 20: HELIOS-A 18-Month Topline Results: Efficacy
HELIOS-A 18-Month Topline Results: Efficacy Sustained Treatment Benefit with Vutrisiran Through 18 Months Positive results for all Month 18 secondary efficacy endpoints, relative to external APOLLO placebo Month 18 Secondary Endpoint P-value mNIS+7 6.5E-20 Norfolk QoL-DN total score 1.8E-10 10-MWT 1
Slide 21: HELIOS-A 18-Month Topline Results: Safety
HELIOS-A 18-Month Topline Results: Safety Continued signs of encouraging safety and tolerability profile during 18-month treatment period • No drug-related discontinuations or deaths • Three discontinuations due to adverse events in vutrisiran arm during 18-month treatment period (none considered re
Slide 22: Vutrisiran Phase 3 Outcomes Study in ATTR Cardiomyopathy
Vutrisiran Phase 3 Study Randomized, Double-Blind Outcomes Study in ATTR Amyloidosis Patients with Cardiomyopathy Primary Endpoint N ~ 600 • Composite outcome of all-cause mortality and recurrent CV events (when Vutrisiran last patient reaches Month 30) Patient Population SC q3M 1:1 RANDOMIZATION •
Slide 23: IKARIA Platform: Proprietary siRNA Design with Novel Chemistry
IKARIATM Platform: Proprietary siRNA Design with Novel Chemistry Super-specific siRNAs May Enable Higher Doses to Achieve Annual Dosing Exquisite specificity High potency in NHP Human dose projection TTR 0.5KD in NHP mg/kg at 0.5 single sc mg/kg dose % TTR Remaning (Normalized to Prebleed) 1.25 Vutr
Slide 24: ILLUMINATE-C Phase 3 Results
ILLUMINATE-C Phase 3 Results Single Arm, Open-Label Study in PH1 Patients with Impaired Renal Function, Including Advanced Disease (N=21) Lumasiran achieved substantial reductions in plasma oxalate relative to baseline • Both in dialysis-independent and -dependent patients Lumasiran demonstrated an
Slide 25: Alnylam Clinical Development Pipeline
Alnylam Clinical Development Pipeline Focused in 4 Strategic Therapeutic Areas (STArs): Genetic Medicines Cardio-Metabolic Diseases EARLY/MID-STAGE LATE STAGE REGISTRATION/ COMMERCIAL (IND/CTA Filed-Phase 2) (Phase 2-Phase 3) COMMERCIAL1 RIGHTS Infectious Diseases CNS/Ocular Diseases (OLE/Phase 4/II
Slide 26: Alnylam Clinical Development Pipeline
Alnylam Clinical Development Pipeline Focused in 4 Strategic Therapeutic Areas (STArs): Genetic Medicines Cardio-Metabolic Diseases EARLY/MID-STAGE LATE STAGE REGISTRATION/ COMMERCIAL (IND/CTA Filed-Phase 2) (Phase 2-Phase 3) COMMERCIAL1 RIGHTS Infectious Diseases CNS/Ocular Diseases (OLE/Phase 4/II
Slide 27: Zilebesiran Phase 2 Clinical Development Plan
Zilebesiran Phase 2 Clinical Development Plan Monotherapy Phase 2 Study (N ~375) • IND opened May 2021 • Evaluate efficacy and safety of zilebesiran as a monotherapy in patients with mild-to-moderate hypertension • Exploring both quarterly and biannual dosing regimens • Study initiated June 2021 Add
Slide 28: Alnylam Clinical Development Pipeline
Alnylam Clinical Development Pipeline Focused in 4 Strategic Therapeutic Areas (STArs): Genetic Medicines Cardio-Metabolic Diseases EARLY/MID-STAGE LATE STAGE REGISTRATION/ COMMERCIAL (IND/CTA Filed-Phase 2) (Phase 2-Phase 3) COMMERCIAL1 RIGHTS Infectious Diseases CNS/Ocular Diseases (OLE/Phase 4/II
Slide 29: Financial Summary and Upcoming Milestones
Jeff Poulton Chief Financial Officer Financial Summary and Upcoming Milestones 29
Slide 30: Global ONPATTRO Q3 2021 Performance
Global ONPATTRO Q3 2021 Performance Revenue ($M) Q3 Highlights YoY % Growth QoQ % Growth U.S. 31% -2% ROW ROW 59% 12% U.S. $120.3M Global 46% 6% $113.8M 69.1 $102.0M 61.4 • Steady and continuous patient growth continues across $90.4M 52.5 key markets (>1,875 commercial patients at end of Q3) $82.5M
Slide 31: Global GIVLAARI Q3 2021 Performance
Global GIVLAARI Q3 2021 Performance Revenue ($M) Q3 Highlights YoY % Growth QoQ % Growth U.S. 85% 0% ROW $31.8M ROW 106% 15% $30.6M U.S. 9.5 Global 91% 4% 8.3 $24.7M • >300 global patients on therapy since launch $22.1M 6.9 5.4 22.4 22.4 • U.S. QoQ demand growth +9% primarily due to an $16.7M increa
Slide 32: Global OXLUMO Q3 2021 Performance
Global OXLUMO Q3 2021 Performance Revenue ($M) Q3 Highlights QoQ % Growth U.S. -20% ROW $16.3M ROW -2% U.S. 9.8 $14.9M Global -9% 9.7 • >120 patients on commercial treatment in U.S. and Europe since launch $9.1M • QoQ sales decreased in both U.S. and ROW, despite 7.7 growth in patients on therapy, d
Slide 33: Q3 2021 Financial Summary
Q3 2021 Financial Summary Financial Results ($ millions) Q3 2021 Q3 2020 YoY % Change Net Product Revenues $167.0 $99.2 68% Net Revenues from Collaborations $20.1 $26.6 (24%) Royalty Revenues $0.5 - - Total Revenues $187.6 $125.9 49% Cost of Goods Sold and Cost of $32.7 $21.8 50% Collaborations and
Slide 34: 2021 Full Year Guidance Reiterated
2021 Full Year Guidance Reiterated FY 2021 Guidance1 Net Product Revenue $640M - $665M (ONPATTRO, GIVLAARI, OXLUMO) Net Revenue from Collaborations $150M - $200M & Royalties Non-GAAP Combined R&D and $1,175M - $1,275M SG&A Expenses2 1 As of August 3, 2021 34 2 2021 Non-GAAP Combined R&D and SG&A Exp
Slide 35: Upcoming Milestones in Late 2021
Upcoming Milestones in Late 2021 Program Upcoming Milestones Vutrisiran • Full 18-month results from HELIOS-A Phase 3 study at medical conference in early 2022 Zilebesiran • Additional Phase 1 data at American Heart Association Scientific Sessions, November 13-15 (ALN-AGT) • Initiate KARDIA-2 Phase
Slide 36: Q&A Session
Q3 2021 Financial Results Q&A Session 36
Slide 37: Challenge Accepted
To those who say “impossible, impractical, unrealistic,” we say: CHALLENGE ACCEPTED © 2021 Alnylam Pharmaceuticals, Inc. 37
Slide 38: Appendix
Q3 2021 Financial Results Appendix 38
Slide 39: Reconciliation of Selected GAAP Measures to Non-GAAP Measures
Alnylam Pharmaceuticals, Inc. Reconciliation of Selected GAAP Measures to Non-GAAP Measures (In thousands) 39 Please note that the figures presented may not sum exactly due to rounding
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