Alnylam Pharmaceuticals, Inc. · NASDAQ:ALNY
Alnylam Pharmaceuticals — Q3 2022 Earnings Presentation
32 slides from Alnylam Pharmaceuticals's earnings presentation, Oct 27, 2022
Slide 1: Third Quarter 2022 Financial Results
Nathan (USA) Diagnosed with AHP Third Quarter 2022 Financial Results October 27, 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: 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 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 7: Commercial Highlights
Tolga Tanguler Chief Commercial Officer Commercial Highlights 7
Slide 8: TTR Franchise Update: Q3 2022
TTR Franchise Update: Q3 2022 $170M >2,580 Q3 TTR Franchise Highlights YoY % Growth QoQ % Growth ONPATTRO ($145M) and Patients Worldwide on Commercial AMVUTTRA ($25M) ONPATTRO (~2,400) and U.S. 80% 30% Global Q3 2022 AMVUTTRA (~180) ROW 13% -5% Net Product Revenues at end of Q3 2022 Global 41% 11% $
Slide 9: AMVUTTRA (vutrisiran) Update: Q3 2022
AMVUTTRA® (vutrisiran) Update: Q3 2022 Encouraging Early Launch Performance Following U.S. Approval on June 13th Doubled “New Start Form” Balanced Growth Across Broad Patient Base and Monthly Average Since Launch Account Types Encouraging Access Trends +100% PRESCRIBER BASE • Patient types: – Newly
Slide 10: GIVLAARI (givosiran) Update: Q3 2022
GIVLAARI® (givosiran) Update: Q3 2022 Q3 Highlights $46M >460 U.S. YoY % Growth 39% QoQ % Growth 5% GIVLAARI Global Q3 2022 Patients Worldwide on Commercial ROW 53% -6% Net Product Revenues GIVLAARI at end of Q3 2022 Global 43% 1% $45.2M $45.7M • U.S. QoQ growth of 5% impacted by: ROW 14.5 $40.7M 15
Slide 11: OXLUMO (lumasiran) Update: Q3 2022
OXLUMO® (lumasiran) Update: Q3 2022 Q3 Highlights $16M >230 U.S. YoY % Growth 22% QoQ % Growth -10% OXLUMO Global Q3 2022 Patients Worldwide on Commercial ROW 4% 29% Net Product Revenues OXLUMO at end of Q3 2022 Global 10% 10% • U.S. QoQ growth of -10% impacted by: ROW $19.2M – Demand growth of -1%
Slide 12: Alnylam Pipeline
Akshay Vaishnaw, M.D., Ph.D. President Alnylam Pipeline 12
Slide 13: Patisiran APOLLO-B Phase 3 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 Secon
Slide 14: APOLLO-B Phase 3 Study Results
APOLLO-B Phase 3 Study Results Primary and First Secondary Endpoints Patisiran demonstrated statistically significant and clinically meaningful improvements in functional capacity, health status and quality of life compared to placebo at month 12 Change from Baseline in 6-MWTa Change From Baseline i
Slide 15: APOLLO-B Overall and Cardiac Safety Summary
APOLLO-B Phase 3 Study Results Overall and Cardiac Safety Summarya Patisiran Placebo At least one event, n (%) (n=181) (n=178) • Majority of AEs were mild or moderate in AEs 165 (91.2) 168 (94.4) severity SAEs 61 (33.7) 63 (35.4) Severe AEs 47 (26.0) 52 (29.2) AEs leading to treatment discontinuatio
Slide 16: 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 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 ~ 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 18: Alnylam TTR Franchise
Alnylam TTR Franchise Potential to Expand Value to Patients Globally for Many Years to Come Novel siRNA Conjugates^ Ocular & CNS hATTR Amyloidosis ALN-TTRsc04 ATTR Amyloidosis Patisiran TBD1 Phase 3 ATTR Amyloidosis with CM (incl. WT)‡ Stargardt Disease Biannual Dosing Vutrisiran Vutrisiran Regimen
Slide 19: Cemdisiran Treatment Led to Clinically Meaningful Proteinuria Reduction
Cemdisiran Treatment Led to Clinically Meaningful Proteinuria Reduction Compared with Placebo at Week 32 • Primary endpoint of change from baseline in 24-hour UPCR compared with placebo at Week 32 was -37.4% (90% CI: -61.0, 0.5)a • Secondary endpoint of change from baseline in 24-hour urine total pr
Slide 20: Cemdisiran Phase 2 IgAN Safety Summary
Cemdisiran Phase 2 IgAN Safety Summary Double-Blind Period • No AEs led to treatment or study discontinuation Cemdisiran Phase 2 IgAN Safety Summarya • One death occurred in cemdisiran treatment arm due to cardiorespiratory collapse and was not considered related to At least one treatment emergent a
Slide 21: 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 22: 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 23: R&D Day Save the Date
Save the date! R&D Day December 15, 2022 A VIRTUAL EVENT Registration information coming soon. 23
Slide 24: Financial Summary and Upcoming Milestones
Jeff Poulton Chief Financial Officer Financial Summary and Upcoming Milestones 24
Slide 25: Q3 2022 Financial Summary
Q3 2022 Financial Summary Financial Results ($ millions) Q3 2022 Q3 2021 Reported Growth % CER Growth %3 Net Product Revenues $232 $167 39% 49% Net Revenues from Collaborations $29 $20 45% Royalty Revenues $3 $0 Total Revenues $264 $188 41% 49% Cost of Goods Sold and Collaborations $41 $33 26% Gross
Slide 26: 2022 Updated Full Year Guidance
2022 Updated Full Year Guidance Prior FY 2022 Updated FY 2022 Guidance1 Guidance2 Net Product Revenue $870M – $930M No change (ONPATTRO, GIVLAARI, OXLUMO, AMVUTTRA) Net Revenues from Collaborations & Royalties $175M – $225M $100M – $150M Non-GAAP Combined R&D and SG&A Expenses3 $1,390M – $1,450M No
Slide 27: Alnylam 2022 Goals
Alnylam 2022 Goals Early Mid Late Combined Net Product Revenue Guidance $870 million – $930 million APOLLO-B Phase 3 Topline Results PATISIRAN hATTR/ATTR Amyloidosis Submit sNDA for ATTR with cardiomyopathy FDA Approval U.S. Launch VUTRISIRAN* hATTR/ATTR Amyloidosis EMA Approval Biannual Dose Regime
Slide 28: Q&A Session
Q3 2022 Financial Results Q&A Session 28
Slide 29: Challenge Accepted
To those who say “impossible, impractical, unrealistic,” we say: CHALLENGE ACCEPTED © 2022 Alnylam Pharmaceuticals, Inc. 29
Slide 30: Appendix
Q3 2022 Financial Results Appendix 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
Slide 32: Reconciliation of Revenue and Growth at Constant Currency
Alnylam Pharmaceuticals, Inc. Reconciliation of Revenue and Growth at Constant Currency Please note that the figures presented may not sum exactly due to rounding 32 Constant Currency = CER
Alnylam Pharmaceuticals — Q3 2022 Earnings Presentation on 6ix