Alnylam Pharmaceuticals, Inc. · NASDAQ:ALNY
Alnylam Pharmaceuticals — Q1 2025 Earnings Presentation
30 slides from Alnylam Pharmaceuticals's earnings presentation, May 1, 2025
Slide 1: First Quarter 2025 Financial Results
First Quarter 2025 Financial Results May 1, 2025 1 © 2025 Alnylam Pharmaceuticals, Inc.
Slide 2: Agenda
Agenda Welcome • Christine Lindenboom Chief Corporate Communications Officer Overview • Yvonne Greenstreet, M.D., MBA Chief Executive Officer Commercial Highlights • Tolga Tanguler Chief Commercial Officer Pipeline • Pushkal Garg, M.D. Chief Medical Officer Financial Summary and Upcoming Milestones
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
Overview Yvonne Greenstreet, M.D., MBA Chief Executive Officer 4
Slide 5: Strong Q1 Financial Results and Significant Advancement Toward Global TTR Leader
Strong Q1 Financial Results and Significant Advancement Toward Our Goal of Becoming the Global TTR Leader Commercial Execution Portfolio Advancement Reiterated Guidance Combined net product revenues: Regulatory approvals of On track to meet 2025 financial $469 million AMVUTTRA® (vutrisiran) in ATTR-
Slide 6: Positioned for Success Through 2025 and Beyond
Positioned for Success Through 2025 and Beyond Growth Strong TTR Through Financial Leadership Innovation Performance Best-in-Class Team + Award-Winning Culture 6
Slide 7: Continued Progress Against Ambitious Five-Year Goals
Continued Progress Against Ambitious Five-Year 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; 10+ in late stages; 4+ INDs per year PERFORMANCE: ≥40% revenue CAGR through YE 2025
Slide 8: Commercial Highlights
Commercial Highlights Tolga Tanguler Chief Commercial Officer 8
Slide 9: Alnylam Continued To Deliver Strong Performance in Q1'25
Alnylam Continued To Deliver Strong Performance in Q1’25 Growth Achieved Across Franchises; Primarily Driven by U.S. TTR Franchise Q1 2025 Overall Portfolio $469 M +28% +4% YoY growth1 QoQ growth1 Combined Net Product Revenues vs. Q1’24 vs. Q4’24 TTR Franchise Rare Franchise 9 1 With FX impact. For
Slide 10: Q1 2025: Rare Franchise Demand Growth Remains Healthy
Q1 2025: Rare Franchise Demand Growth Remains Healthy $109M Total Rare Global Q1 2025 Q1 2025 Rare Franchise Highlights Net Product Revenues YoY % Growth QoQ % Growth +8% +1% $111M $109M GIVLAARI 15% 4% $108M $103M $101M $25M OXLUMO -1% -3% $25M $28M $29M $25M Total Rare 8% 1% $15M $19M $14M Revenue
Slide 11: Q1 2025: Robust 36% TTR Growth Driven by U.S. Performance
Q1 2025: Robust 36% TTR Growth Driven by U.S. Performance $359M Total TTR Global Q1 2025 Q1 2025 TTR Franchise Highlights Net Product Revenues YoY % Growth QoQ % Growth U.S. 45% 5% +36% +5% $359M ROW 24% 5% $343M $307M $309M Global 36% 5% $112M $103M $264M $82M $90M • U.S. YoY +45% growth highlights
Slide 12: hATTR-PN: AMVUTTRA Drives Category Growth and Leads New Patient Share
hATTR-PN | AMVUTTRA Drives Category Growth and Leads New Patient Share in U.S. AMVUTTRA captured ~70% PN category growth accelerated as expected of new patient starts in Q1’25 hATTR-PN U.S. New Patient Share in Q1'25 Patients on treatment 2nd Product Entry ~30% ~70% Jan’23 Apr’23 Jul’23 Oct’23 Jan’2
Slide 13: ATTR-CM: Momentum Building for Second Half of 2025
ATTR-CM | Momentum Building for Second Half of 2025 Launch Today Momentum Approval 1H 2025 2H 2025 Optimizing access pathways at ~170 priority health systems (~80% of Rx volume) Treatment sites expansion (2x increase since Q4’24, achieving >2,000 sites1) Continued payer engagements (e.g., VBA expans
Slide 14: ATTR-CM: U.S. Launch Off To A Strong Start
ATTR-CM | U.S. Launch Off To A Strong Start Access Health System Treatment & Affordability Formulary Coverage Choice Patients Are Rapid Progress on AMVUTTRA’s First-Line Accessing AMVUTTRA Formulary Inclusion Profile Is Resonating Across Payer Mix Commercial, Medicare FFS, and Medicare Advantage ~60
Slide 15: Continue to Communicate Progress
Continue to Communicate Progress Q3’25 Q2’25 Earnings 2025 Today Earnings Guidance Q1’25 Customer ✓ Utilization Earnings Additional Launch PDUFA, Indicators $1,600 to U.S. Launch Early Launch $1,725 Million Indicators & Final Label Customer & Launch Feedback Total TTR Readiness Indicators Product Sa
Slide 16: Pipeline
Pipeline Pushkal Garg, M.D. Chief Medical Officer 16
Slide 17: AMVUTTRA Therapeutic Profile Supports First-Line Potential
AMVUTTRA Therapeutic Profile Supports First-Line Potential Population Representative Composite of all- All-cause mortality Broad Impact on of Today’s Patients cause mortality & (secondary endpoint)1,2 ATTR-CM Parameters with recurrent CV events Substantial Improvement (primary endpoint)1,2 of CV Out
Slide 18: AMVUTTRA Regulatory Progress
AMVUTTRA Regulatory Progress U.S. Brazil European Union APPROVED APPROVED POSITIVE CHMP OPINION Japan United Kingdom Canada Additional countries expected Q2 2025 expected Q3 2025 expected Q4 2025 expected 2026-2027 18
Slide 19: Continued Evidence Generation from HELIOS-B to Support AMVUTTRA Differentiation
Continued Evidence Generation from HELIOS-B to Support AMVUTTRA Differentiation Echocardiographic Baseline Heart Failure Functional Capacity, Health Parameters1 Severity2 Status, Quality of Life3 • Vutrisiran improved diastolic • Consistent benefit across • Significantly more vutrisiran function and
Slide 20: Nucresiran TRITON Phase 3 Program
Nucresiran TRITON Phase 3 Program Potential to Launch in hATTR-PN Ahead of ATTR-CM Assumed Launch CVO primary endpoint ~2030 (event driven) • Randomized, double-blind, event-driven outcomes study • Initiation expected first half 2025 Targeting Launch mNIS+7 primary several years earlier endpoint • E
Slide 21: Robust and High-Value Pipeline of RNAi Therapeutics
Robust and High-Value Pipeline of RNAi Therapeutics PHASE 1 PHASE 2 PHASE 3 APPROVED ONPATTRO® (patisiran) hATTR Amyloidosis with Polyneuropathy TTR AMVUTTRA® (vutrisiran) ATTR Amyloidosis with Cardiomyopathy and hATTR Amyloidosis with Polyneuropathy Nucresiran (ALN-TTRsc04) ATTR Amyloidosis GIVLAAR
Slide 22: Financial Summary and Upcoming Milestones
Financial Summary and Upcoming Milestones Jeff Poulton Chief Financial Officer 22
Slide 23: Q1 2025 Financial Summary
Q1 2025 Financial Summary Q1 2025 vs Q1 2025 vs ($ in millions except where noted as percentages) Q1 2024 Q1 2025 Q1 2024 Q1 2024 (Reported) (CER2) Total Combined Net Product Revenues $365 $469 28% 30% Net Revenues from Collaborations & Royalties 129 126 (3%) Collaboration Revenue 119 99 (16%) Royal
Slide 24: 2025 Full-Year Guidance
2025 Full-Year Guidance Item FY 2025 Guidance Key Assumptions Total TTR Net Product Revenues (PN & CM) • AMVUTTRA-CM approval and launch in Germany (AMVUTTRA, ONPATTRO) $1,600 to $1,725 million and Japan in the second half of 2025 Total Rare Net Product Revenues (GIVLAARI, OXLUMO) $450 to $525 milli
Slide 25: Alnylam 2025 Goals
Alnylam 2025 Goals Combined Net Product Revenue Guidance $2,050M – $2,250M 2025 U.S. FDA Approval March 20, 2025 VUTRISIRAN ATTR Amyloidosis Additional Global Approvals (Japan, EU) Q2, Q3 Initiate Phase 3 Study in ATTR-CM H1 NUCRESIRAN* ATTR Amyloidosis (ALN-TTRsc04) Initiate Phase 3 Study in hATTR-
Slide 26: Q&A Session
Q&A Session Q1 2025 Financial Results 26
Slide 27: 27
27
Slide 28: Appendix
Appendix Q1 2025 Financial Results 28
Slide 29: Reconciliation of Selected GAAP Measures to Non-GAAP Measures
Alnylam Pharmaceuticals, Inc. Reconciliation of Selected GAAP Measures to Non-GAAP Measures (In thousands) 29 Please note that the figures presented above may not sum exactly due to rounding
Slide 30: Reconciliation of Product Revenue and Growth at Constant Currency
Alnylam Pharmaceuticals, Inc. Reconciliation of Product Revenue and Growth at Constant Currency 30 Please note that the figures presented above may not sum exactly due to rounding Constant Currency = Constant Exchange Rate, or CER
Alnylam Pharmaceuticals — Q1 2025 Earnings Presentation on 6ix