Alnylam Pharmaceuticals, Inc. · NASDAQ:ALNY
Alnylam Pharmaceuticals — Q1 2022 Earnings Presentation
29 slides from Alnylam Pharmaceuticals's earnings presentation, Apr 28, 2022
Slide 1: First Quarter 2022 Financial Results
Nathan (USA) Diagnosed with AHP First Quarter 2022 Financial Results April 28, 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 • Pushkal Garg, M.D. Chief Medical Officer Fin
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: Multiple Drivers of Future Growth
Multiple Drivers of Future Growth TTR Franchise Leadership Expansion into Prevalent Diseases Engine for Sustainable Innovation Andreas (Sweden) 5 Diagnosed with hATTR amyloidosis
Slide 6: Alnylam P5x25 strategy targets
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 7: Commercial Highlights
Tolga Tanguler Chief Commercial Officer Commercial Highlights 7
Slide 8: ONPATTRO (patisiran) Update: Q1 2022
ONPATTRO® (patisiran) Update: Q1 2022 Q1 Highlights $137M >2,200 YoY % Growth QoQ % Growth ONPATTRO Global Q1 2022 Patients Worldwide on Commercial U.S. 26% 4% Net Product Revenues ONPATTRO at end of Q1 2022 ROW 42% -5% Global 34% -1% ROW $138.6M $137.0M 78.5 • Steady patient growth continues across
Slide 9: GIVLAARI (givosiran) Update: Q1 2022
GIVLAARI® (givosiran) Update: Q1 2022 Q1 Highlights $35M >400 YoY % Growth QoQ % Growth GIVLAARI Global Q1 2022 Patients Worldwide on Commercial U.S. 33% -22% Net Product Revenues GIVLAARI at end of Q1 2022 ROW 68% 11% Global 43% -13% $40.7M ROW 10.4 • U.S. QoQ growth of -22% impacted by: U.S. $35.3
Slide 10: OXLUMO (lumasiran) Update: Q1 2022
OXLUMO® (lumasiran) Update: Q1 2022 Q1 Highlights $15M >160 YoY % Growth QoQ % Growth OXLUMO Global Q1 2022 Patients Worldwide on Commercial U.S. 284% -5% Net Product Revenues OXLUMO at end of Q1 2022 ROW 19% -32% Global 59% -24% ROW $19.2M • U.S. QoQ growth of -5% impacted by: U.S. 13.5 $16.3M – De
Slide 11: Alnylam Pipeline
Pushkal Garg, M.D. Chief Medical Officer Alnylam Pipeline 11
Slide 12: Patisiran Phase 3 APOLLO-B Study
Patisiran Phase 3 Study Randomized, Double-Blind, Placebo-Controlled Study in ATTR Amyloidosis Patients with Cardiomyopathy N = 360 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 13: Vutrisiran HELIOS-A Phase 3 Study
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 14: Improvement in Neuropathy Impairment and Quality of Life at Month 18
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 15: 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 16: 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 17: Vutrisiran HELIOS-B Phase 3 Study
Vutrisiran Phase 3 Study Randomized, Double-Blind Outcomes Study in ATTR Amyloidosis Patients with Cardiomyopathy Primary Endpoint N = 655 • 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 18: 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 • E
Slide 19: 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 20: 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 21: Financial Summary and Upcoming Milestones
Jeff Poulton Chief Financial Officer Financial Summary and Upcoming Milestones 21
Slide 22: Q1 2022 Financial Summary
Q1 2022 Financial Summary Financial Results ($ millions) Q1 2022 Q1 2021 Reported Growth % CER Growth %3 Net Product Revenues $186.9 $135.8 38% 43% Net Revenues from Collaborations $25.9 $41.8 (38%) Royalty Revenues $0.4 - - Total Revenues $213.3 $177.6 20% 24% Cost of Goods Sold and Collaborations
Slide 23: 2022 Updated Full Year Guidance
2022 Updated Full Year Guidance Prior FY 2022 Updated FY 2022 Guidance1 Guidance2 Net Product Revenue $900M – $1,000M $870M – $930M (ONPATTRO, GIVLAARI, OXLUMO, vutrisiran)3 Net Revenues from Collaborations & Royalties $175M – $225M No change Non-GAAP Combined R&D and SG&A Expenses4 $1,400M – $1,500
Slide 24: Alnylam 2022 Goals
Alnylam 2022 Goals Early Mid Late Combined Net Product Revenue Guidance $870 million – $930 million (includes vutrisiran) APOLLO-B Phase 3 Topline Results PATISIRAN hATTR/ATTR Amyloidosis File sNDA for ATTR-CM FDA Approval (7/14/22 PDUFA) U.S. Launch hATTR/ATTR Amyloidosis VUTRISIRAN* EMA Approval B
Slide 25: Q&A Session
Q1 2022 Financial Results Q&A Session 25
Slide 26: Challenge Accepted
To those who say “impossible, impractical, unrealistic,” we say: CHALLENGE ACCEPTED © 2022 Alnylam Pharmaceuticals, Inc. 26
Slide 27: Appendix
Q1 2022 Financial Results Appendix 27
Slide 28: Reconciliation of Selected GAAP Measures to Non-GAAP Measures
Alnylam Pharmaceuticals, Inc. Reconciliation of Selected GAAP Measures to Non-GAAP Measures (In thousands) 28 Please note that the figures presented may not sum exactly due to rounding
Slide 29: Reconciliation of Revenue and Operating Expense Growth at Constant Currency
Alnylam Pharmaceuticals, Inc. Reconciliation of Revenue and Operating Expense Growth at Constant Currency 29 Please note that the figures presented may not sum exactly due to rounding
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