Alnylam Pharmaceuticals, Inc. · NASDAQ:ALNY
Alnylam Pharmaceuticals — Q4 2021 Earnings Presentation
31 slides from Alnylam Pharmaceuticals's earnings presentation, Feb 10, 2022
Slide 1: Fourth Quarter and Full Year 2021 Financial Results
Nathan (USA) Diagnosed with AHP Fourth Quarter and Full Year 2021 Financial Results February 10, 2022 1 © 2022 Alnylam Pharmaceuticals, Inc.
Slide 2: Agenda
Agenda Welcome • Christine Lindenboom Senior Vice President, Investor Relations & Corporate Communications Overview • Yvonne Greenstreet, MBChB, MBA Chief Executive Officer Commercial Highlights • Tolga Tanguler Chief Commercial Officer Alnylam Pipeline • Akshay Vaishnaw, M.D., Ph.D. President Finan
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
Yvonne Greenstreet, MBChB, MBA Chief Executive Officer Overview 4
Slide 5: Notable Accomplishments in 2021
Notable Accomplishments in 2021 Combined net product revenues of Completed enrollment in $662 million two key Phase 3 studies in (83% growth YoY) ATTR amyloidosis w/ CM Advanced multiple investigational products 2 Expanded commercial presence into NDA/sNDA submissions for prevalent diseases >30 coun
Slide 6: Multiple Drivers of Future Growth
Multiple Drivers of Future Growth TTR Franchise Leadership Expansion into Prevalent Diseases Engine for Sustainable Innovation Andreas (Sweden) 6 Diagnosed with hATTR amyloidosis
Slide 7: Alnylam P5x25 Strategy Goals
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: Achieve sustainable non-GAAP
Slide 8: Commercial Highlights
Tolga Tanguler Chief Commercial Officer Commercial Highlights 8
Slide 9: ONPATTRO (patisiran) Update: Year End 2021
ONPATTRO® (patisiran) Update: Year End 2021 Q4 Highlights $475M >2,050 YoY % Growth QoQ % Growth ONPATTRO Global 2021 Patients Worldwide on Commercial U.S. 40% 17% Net Product Revenues ONPATTRO at YE 2021 ROW 66% 14% Global 53% 15% ROW $138.6M 78.5 • Steady patient growth continues across key market
Slide 10: GIVLAARI (givosiran) Update: Year End 2021
GIVLAARI® (givosiran) Update: Year End 2021 Q4 Highlights $128M >350 YoY % Growth QoQ % Growth GIVLAARI Global 2021 Patients Worldwide on Commercial U.S. 81% 35% Net Product Revenues GIVLAARI at YE 2021 ROW 94% 10% Global 84% 28% $40.7M ROW 10.4 • U.S. QoQ growth of 35% impacted by: U.S. $31.8M – De
Slide 11: OXLUMO (lumasiran) Update: Year End 2021
OXLUMO® (lumasiran) Update: Year End 2021 Q4 Highlights $60M >140 QoQ % Growth OXLUMO Global 2021 Patients Worldwide on Commercial U.S. 9% Net Product Revenues OXLUMO at YE 2021 ROW 40% Global 29% ROW $19.2M • U.S. QoQ growth of 9% impacted by: U.S. 13.5 $16.3M – Demand growth +15% due primarily to
Slide 12: Alnylam Pipeline
Akshay Vaishnaw, M.D., Ph.D. President Alnylam Pipeline 12
Slide 13: Patisiran Phase 3 Study (APOLLO-B)
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 14: Vutrisiran Phase 3 Study (HELIOS-A)
Vutrisiran Phase 3 Study Randomized, Open-Label Study in Patients with Hereditary ATTR Amyloidosis with Polyneuropathy • As previously reported, the primary endpoint of change from baseline in mNIS+7 at Month 9 was met1 Efficacy Assessments Vutrisiran vs APOLLO Placebo n=122 Primary Endpoint (at Mon
Slide 15: Rapid and Sustained Reduction in Serum TTR Levels with Vutrisiran
Rapid and Sustained Reduction in Serum TTR Levels with Vutrisiran • Vutrisiran achieved a mean steady-state serum TTR reduction from baseline of 88% (SD: 16%) • TTR reduction with vutrisiran was non-inferior to that observed with the within-study patisiran reference comparator (secondary endpoint) o
Slide 16: Improvement in Neuropathy Impairment and Quality of Life with Vutrisiran
Statistically Significant Improvement in Neuropathy Impairment and Quality of Life with Vutrisiran vs External Placebo at Month 18 • Improvement was observed across all prespecified patient subgroups, components, and subdomains of mNIS+7 and Norfolk QOL-DN (data not shown) • Improvement relative to
Slide 17: Exploratory Imaging Parameters
Exploratory Imaging Parameters Potential Evidence of Reduction in Amyloid Burden Vutrisiran trended toward improvement in all echocardiographic Reduced cardiac technetium uptake on scintigraphy imaging parameters, compared with external placebo group shown in majority of assessable vutrisiran patien
Slide 18: HELIOS-A Safety Summary
HELIOS-A Safety Summarya Majority of AEs mild or moderate in severity HELIOS-A Safety Summarya • No drug-related discontinuations or deaths • Three study discontinuations (2.5%) due to AEs in the APOLLO HELIOS-A vutrisiran arm (two due to death, as previously reported; one Placebo Vutrisiran Patisir
Slide 19: Vutrisiran Phase 3 Outcomes Study (HELIOS-B)
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 20: Stargardt Disease
Stargardt Disease Promising New Opportunity for Vutrisiran Description Therapeutic Hypothesis Rare, inherited, progressive form of blindness caused by accumulation of toxic vitamin A metabolites in retina leading to central vision loss Silence production of TTR in liver* High unmet medical need with
Slide 21: 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 22: Zilebesiran Interim Phase 1 Results
Zilebesiran (ALN-AGT) Interim Phase 1 Results Results for Investigational Therapy Presented at AHA Scientific Sessions1 Dose-Dependent and Durable Reduction of Serum AGT ≥90% Sustained Reductions in SBP and DBP2 Sustained for 12 Weeks After Single Doses of zilebesiran ≥100 mg Mean 24h blood pressure
Slide 23: Alnylam Clinical Development Pipeline
Alnylam Clinical Development Pipeline Focused in 4 Strategic Therapeutic Areas (STArs): EARLY/MID-STAGE LATE STAGE REGISTRATION/ COMMERCIAL Genetic Medicines Cardio-Metabolic Diseases (IND/CTA Filed-Phase 2) (Phase 2-Phase 3) COMMERCIAL1 RIGHTS Infectious Diseases CNS/Ocular Diseases (OLE/Phase 4/II
Slide 24: Alnylam Clinical Development Pipeline
Alnylam Clinical Development Pipeline Focused in 4 Strategic Therapeutic Areas (STArs): EARLY/MID-STAGE LATE STAGE REGISTRATION/ COMMERCIAL Genetic Medicines Cardio-Metabolic Diseases (IND/CTA Filed-Phase 2) (Phase 2-Phase 3) COMMERCIAL1 RIGHTS Infectious Diseases CNS/Ocular Diseases (OLE/Phase 4/II
Slide 25: Financial Summary and Upcoming Milestones
Jeff Poulton Chief Financial Officer Financial Summary and Upcoming Milestones 25
Slide 26: Q4 & Full Year 2021 Financial Summary
Q4 & Full Year 2021 Financial Summary Financial Results ($ millions) Q4 2021 Q4 2020 YoY % Change FY 2021 FY 2020 YoY % Change Net Product Revenues $198.5 $112.8 76% $662.1 $361.5 83% Net Revenues from Collaborations $59.6 $50.7 18% $181.0 $131.3 38% Royalty Revenues $0.4 - - $1.2 - - Total Revenues
Slide 27: 2022 Full Year Guidance
2022 Full Year Guidance1 FY 2021 FY 2022 Projected 2022 Actuals Guidance Growth (using mid-point of guidance) Net Product Revenue (ONPATTRO, GIVLAARI, OXLUMO, Vutrisiran) $662M $900M – $1,000M +44% Net Revenue from Collaborations & Royalties $182M $175M – $225M +10% Non-GAAP Combined R&D and SG&A Ex
Slide 28: Alnylam 2022 Goals
Alnylam 2022 Goals Early Mid Late Combined Net Product Revenue Guidance: $900 million – $1 billion (includes vutrisiran) APOLLO-B Phase 3 Topline Results PATISIRAN hATTR/ATTR Amyloidosis File sNDA for ATTR with CM FDA Approval (4/14/22 PDUFA) U.S. Launch hATTR/ATTR Amyloidosis VUTRISIRAN* EMA Approv
Slide 29: Q&A Session
Q4 and Full Year 2021 Financial Results Q&A Session 29
Slide 30: Challenge Accepted
To those who say “impossible, impractical, unrealistic,” we say: CHALLENGE ACCEPTED © 2022 Alnylam Pharmaceuticals, Inc. 30
Slide 31: Reconciliation of Selected GAAP Measures to Non-GAAP Measures
Alnylam Pharmaceuticals, Inc. Reconciliation of Selected GAAP Measures to Non-GAAP Measures (In thousands) 31 Please note that the figures presented may not sum exactly due to rounding
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