Alnylam Pharmaceuticals, Inc. · NASDAQ:ALNY
Alnylam Pharmaceuticals — Q1 2024 Earnings Presentation
27 slides from Alnylam Pharmaceuticals's earnings presentation, May 2, 2024
Slide 1: First Quarter 2024 Financial Results
First Quarter 2024 Financial Results May 2, 2024 © 2024 Alnylam Pharmaceuticals, Inc. 1
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: Alnylam Forward Looking Statements
Alnylam Forward Looking Statements 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. All statements other than historical statements of fact regarding Alnylam’s expectations, b
Slide 4: Overview
Yvonne Greenstreet, MBChB, MBA Chief Executive Officer Overview 4
Slide 5: Ambitious Five-Year Strategy to Drive Growth
Ambitious Five-Year Strategy to Drive Growth 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
Slide 6: Commercial Highlights
Tolga Tanguler Chief Commercial Officer Commercial Highlights 6
Slide 7: Commercial Portfolio Strong Start into 2024
Commercial Portfolio Strong Start into 2024 Overall portfolio $365M 32% 5% Combined Net Product Revenue YoY growth1 vs. Q1.23 QoQ growth1 vs. Q4.23 TTR Franchise Rare Franchise 7 1 With Fx impact. For growth at CER = constant exchange rate – see the Financial Summary slide for more information.
Slide 8: TTR Franchise Update: Q1 2024
TTR Franchise Update: Q1 2024 $264M Total TTR Global Q1 2024 Net Product Revenues Q1 TTR Franchise Highlights +29% +4% YoY % Growth QoQ % Growth $264M U.S. 35% 3% $254M ROW 23% 5% $230M $224M $53M $65M Global 29% 4% $204M $35M $36M $23M • U.S. TTR franchise YoY growth +35% driven by: – Demand (+39%)
Slide 9: AMVUTTRA is Clear Market Leader in Treatment of hATTR-PN
AMVUTTRA is Clear Market Leader in Treatment of hATTR-PN Awareness & Treatment Adherence & Access Diagnosis Choice Compliance ~80% of >50% growth in >99% of patients >95% patients remain prescribers compared to have confirmed access of patients comply with undiagnosed and/or Q1 2023 the AMVUTTRA unt
Slide 10: Significant Growth Opportunity in hATTR-PN
Significant Growth Opportunity in hATTR-PN hATTR PN under treated Winning product profile Expanding our reach and under diagnosed1 • Rapidly knocked down TTR as Launched patient DTC early as three weeks, with a mean campaign to increase 25-30k TTR knockdown of 88% over 18 awareness of disease and pa
Slide 11: Rare Franchise Update: Q1 2024
Rare Franchise Update: Q1 2024 $101M Total Rare Global Q1 2024 Net Product Revenues Q1 Rare Franchise Highlights YoY % Growth QoQ % Growth +40% +9% GIVLAARI 21% -2% $101M OXLUMO 77% 30% $92M Total Rare 40% 9% $82M $83M $29M $22M $72M $15M $19M • GIVLAARI YoY growth +21% driven by: – U.S. (+28%): pri
Slide 12: Alnylam Pipeline
Pushkal Garg, M.D. Chief Medical Officer Alnylam Pipeline 12
Slide 13: Vutrisiran HELIOS-B Phase 3 Study
Vutrisiran Phase 3 Study Randomized, Double-Blind Outcomes Study in ATTR Amyloidosis Patients with Cardiomyopathy N = 655 Primary Endpoint Vutrisiran 1:1 RANDOMIZATION Patient Population SC q3M • Composite outcome of all-cause mortality and recurrent CV • ATTR amyloidosis; events, assessed when last
Slide 14: Uncontrolled Hypertension is a Global Health Crisis
Uncontrolled Hypertension is a Global Health Crisis Leading Preventable Risk Factor for Cardiovascular Morbidity and Mortality Targeting Tonic BP Control to Reduce • ~219MM adults with primary hypertension1 Cardiovascular and Renal Risks 24-Hour • Hypertension is the leading cause of BP Level Strict
Slide 15: Zilebesiran Continues to Demonstrate Potential to Transform Treatment of Hypertension
Zilebesiran Continues to Demonstrate Potential to Transform Treatment of Hypertension Phase 2 Results: Blood Pressure Reduction as Add-On Therapy Primary Endpoint: 24-hour Mean Ambulatory SBP Secondary Endpoint: Office SBP Reduction at Month 3* Reduction at Month 3* • Generally well tolerated • Incr
Slide 16: Zilebesiran Clinical Development Plan
Zilebesiran Clinical Development Plan Exploring Power of Tonic Blood Pressure Control to Improve Cardiovascular Outcomes Phase 1 Phase 2 Phase 3 Zilebesiran safety, tolerability, Launch with and PK/PD in patients with label to reduce mild-to-moderate hypertension1 Zilebesiran monotherapy in pts with
Slide 17: Advancing a Robust and High-Yielding Pipeline of RNAi Therapeutics
Advancing a Robust and High-Yielding Pipeline of RNAi Therapeutics Positioned to Deliver Strong Growth and Innovation Across Multiple Disease Areas and Indications IND-enabling Phase 1 Phase 2 Phase 3 Approved ALN-HTT02* ALN-TTRsc04 Zilebesiran* Vutrisiran Huntington’s Disease ATTR Amyloidosis Hyper
Slide 18: Multiple Sources of Sustainable Innovation Drive Robust Pipeline
Multiple Sources of Sustainable Innovation Drive Robust Pipeline Targeting Nine Alnylam-Led INDs Across Four Tissues by End of 2025 Extrahepatic Delivery Platform Designs Human Genetics 12 months IKARIA™ Reversir™ GEMINI™ By End of 2025 new liver new CNS 5 INDs 2 INDs 2 new tissues with INDs 10+ inc
Slide 19: Financial Summary and Upcoming Milestones
Jeff Poulton Chief Financial Officer Financial Summary and Upcoming Milestones 19
Slide 20: Q1 2024 Financial Summary
Q1 2024 Financial Summary Q1 Reported Q1 CER Financial Results ($ millions) Q1 2024 Q1 2023 Growth % Growth %2 Net Product Revenues $365 $276 32% 32% Net Revenues from Collaborations $119 $36 Royalty Revenues $11 $7 Total Revenues $494 $319 55% 55% Product Cost of Goods Sold $55 $41 Cost of Collabor
Slide 21: 2024 Reiterated Full Year Guidance
2024 Reiterated Full Year Guidance Guidance Key Assumptions Net Product Revenue1 ONPATTRO, AMVUTTRA, GIVLAARI, $1,400M to $1,500M • Uses January 31, 2024 FX rates OXLUMO Net Product Revenue Growth vs. 2023 13% to 21% • Uses January 31, 2024 FX rates at reported Fx rates1 Net Product Revenue Growth v
Slide 22: Alnylam 2024 Goals
Alnylam 2024 Goals Early Mid Late Combined Net Product Revenue Guidance $1,400M – $1,500M HELIOS-B Topline Results (late June/early July) VUTRISIRAN ATTR Amyloidosis sNDA Submission ALN-TTRsc04* ATTR Amyloidosis Initiate Phase 3 ATTR-CM Study KARDIA-2 Phase 2 Topline Results ZILEBESIRAN* Hypertensio
Slide 23: Q&A Session
Q1 2024 Financial Results Q&A Session 23
Slide 24: Thank You!
| || Thank You! “LAlnylam
Slide 25: Appendix
Q1 2024 Financial Results Appendix 25
Slide 26: Reconciliation of Selected GAAP Measures to Non-GAAP Measures
Alnylam Pharmaceuticals, Inc. Reconciliation of Selected GAAP Measures to Non-GAAP Measures (In thousands, except per share amounts) 26 Please note that the figures presented may not sum exactly due to rounding
Slide 27: 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 27 Constant Currency = Constant Exchange Rate, or CER
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