Alnylam Pharmaceuticals, Inc. · NASDAQ:ALNY
Alnylam Pharmaceuticals — Q3 2024 Earnings Presentation
27 slides from Alnylam Pharmaceuticals's earnings presentation, Oct 31, 2024
Slide 1: Third Quarter 2024 Financial Results
Third Quarter 2024 Financial Results October 31, 2024 © 2024 Alnylam Pharmaceuticals, Inc. 1
Slide 2: Agenda
Agenda Welcome • Christine Lindenboom Chief Corporate Communications Officer Overview • Yvonne Greenstreet, MBChB, MBA Chief Executive Officer Commercial Highlights • Tolga Tanguler Chief Commercial Officer Alnylam Pipeline • Pushkal Garg, M.D. Chief Medical Officer Financial Summary and Upcoming Mi
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: Q3 2024 Highlights
Q3 2024 Highlights Strong Positioned for Steady Revenue Growth TTR Leadership Pipeline Progress • $420 million net product • Full HELIOS-B results • ALN-HTT02 Phase 1 initiation revenues presented in Huntington’s disease • 34% YoY growth1 • U.S. and EU regulatory • Mivelsiran Phase 1 multidose submi
Slide 6: 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 7: Commercial Highlights
Tolga Tanguler Chief Commercial Officer Commercial Highlights 7
Slide 8: Commercial Portfolio
Commercial Portfolio Continued Strong Performance in Q3 2024 Overall portfolio $420M 34% 2% Combined Net Product Revenue YoY growth1 vs. Q3’23 QoQ growth1 vs. Q2’24 TTR Franchise Rare Franchise 8 1 With FX impact. For growth at CER = constant exchange rate – see the Financial Summary slide for more
Slide 9: TTR Franchise Update: Q3 2024
TTR Franchise Update: Q3 2024 $309M Total TTR Global Q3 2024 Net Product Revenues Q3 TTR Franchise Highlights YoY % Growth QoQ % Growth +34% 0% U.S. 37% 8% $307M $309M ROW 31% -9% $264M Global 34% 0% $254M $82M $90M $230M • U.S. TTR franchise YoY growth of +37% driven by: $53M $65M – Demand (+31%):
Slide 10: We Are On a Path to Leadership in ATTR
We Are On a Path to Leadership in ATTR Rare yet large, untapped and growing category with significant unmet need AMVUTTRA is poised to become the standard of care for first line patients in ATTR-CM1 Our success and strong leadership in hATTR-PN position us to be highly competitive in ATTR-CM We have
Slide 11: Rare Franchise Update: Q3 2024
Rare Franchise Update: Q3 2024 $111M Total Rare Global Q3 2024 Net Product Revenues Q3 Rare Franchise Highlights +34% +8% YoY % Growth QoQ % Growth $111M $103M GIVLAARI 31% 14% $101M $25M OXLUMO 40% -1% $92M $25M Total Rare 34% 8% $83M $29M $22M $15M $19M $16M • GIVLAARI YoY growth of +31% driven by
Slide 12: Alnylam Pipeline
Pushkal Garg, M.D. Chief Medical Officer Alnylam Pipeline 12
Slide 13: HELIOS-B: Compelling Clinical Benefit for Vutrisiran in ATTR-CM
HELIOS-B: Compelling Clinical Benefit for Vutrisiran in ATTR-CM Observed Clinical Benefit Occurs Early and Cascades in Biologically Rational Manner Rapid TTR Cardiac Functional, Outcomes Knockdown Biomarkers and Health Status, and Benefit Echocardiographic QoL Benefit Improvements 13
Slide 14: Vutrisiran Regulatory Filings Submitted in U.S. and EU
Priority Review Voucher to Vutrisiran Regulatory accelerate FDA review period Filings Submitted in U.S. and EU Additional global regulatory submissions – late 2024 14 Note: Vutrisiran is an investigational therapeutic in development for ATTR-CM, and efficacy and safety in this patient population has
Slide 15: Progress with Mivelsiran (ALN-APP)
Progress with Mivelsiran (ALN-APP) Additional sAPPβ Reduction Seen Following Second Administration Phase 1 (Early-onset Alzheimer's Disease) 50 mg SD and Interim MD Data sAPPβ • Rapid, robust, and durable target engagement 0 ‒ Up to 92% reduction in sAPPβ at Month 7 with Mean (SE) Change from Baseli
Slide 16: Expanding Opportunities in CNS: Huntington's Disease
Expanding Opportunities in CNS Addressing Unmet Needs in Huntington’s Disease Progressive & Fatal Broad CNS Distribution and Durable Neurodegenerative Disease HTT-Lowering in NHP5 Control HD Brain Brain • Huntington’s disease (HD) is progressive and fatal, • Widespread distribution across CNS region
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 ONPATTRO® (patisiran) hATTR Amyloidosis with Polyneuropathy AMVUTTRA® (vutrisiran) hATTR A
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+ includ
Slide 19: Financial Summary and Upcoming Milestones
Jeff Poulton Chief Financial Officer Financial Summary and Upcoming Milestones 19
Slide 20: Q3 2024 Financial Summary
Q3 2024 Financial Summary Q3 Reported Q3 CER Financial Results ($ millions) Q3 2024 Q3 2023 Growth % Growth %2 Net Product Revenues $420 $313 34% 35% Net Revenues from Collaborations $57 $427 Royalty Revenues $23 $10 Total Revenues $501 $751 -33% -33% Product Cost of Goods Sold $82 $79 Cost of Colla
Slide 21: 2024 Reiterated Full Year Guidance
2024 Reiterated Full Year Guidance FY 2024 Guidance Key Assumptions Net Product Revenue1 ONPATTRO, AMVUTTRA, GIVLAARI, $1,575M - $1,650M • Uses June 30, 2024 FX rates OXLUMO Net Product Revenue Growth vs. 2023 at reported Fx rates1 27% to 33% • Uses June 30, 2024 FX rates Net Product Revenue Growth
Slide 22: Alnylam 2024 Goals
Alnylam 2024 Goals Early Mid Late Combined Net Product Revenue Guidance L $1,575M – $1,650M HELIOS-B Topline Results L VUTRISIRAN ATTR Amyloidosis sNDA Submission L ALN-TTRsc04* ATTR Amyloidosis Initiate Phase 3 ATTR-CM Study L KARDIA-2 Phase 2 Topline Results L ZILEBESIRAN* Hypertension Initiate KA
Slide 23: Q&A Session
Q3 2024 Financial Results Q&A Session 23
Slide 24: Thank You!
| || Thank You! “LAlnylam
Slide 25: Appendix
Q3 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) Three Months Ended September 30, September 30, 2024 2023 Reconciliation of GAAP to Non-GAAP Research and development: GAAP Research and development $ 270,926 $ 253,179
Slide 27: Reconciliation of Revenue and Growth at Constant Currency
Alnylam Pharmaceuticals, Inc. Reconciliation of Revenue and Growth at Constant Currency September 30, 2024 Three Months Ended Total TTR net product revenue growth, as reported 34 % Add: Impact of foreign currency translation 1 Total TTR net product revenue growth at constant currency 35 % Total Rare
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