Alnylam Pharmaceuticals, Inc. · NASDAQ:ALNY
Alnylam Pharmaceuticals — Q2 2025 Earnings Presentation
32 slides from Alnylam Pharmaceuticals's earnings presentation, Jul 31, 2025
Slide 1: Second Quarter 2025 Financial Results
Second Quarter 2025 Financial Results July 31, 2025 1 © 2025 Alnylam Pharmaceuticals, Inc.
Slide 2: Agenda
Agenda Welcome • Christine Akinc 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 Research & Development Officer Financial Summary and Upcoming M
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: 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 5
Slide 6: Durable TTR Leadership Drove Q2 Performance
Durable TTR Leadership Drove Q2 Performance Innovative Pipeline Advancing TTR Leadership Growth Through Innovation Strong Financial Performance $544M Q2 Total TTR ATTR-CM Initiated TRITON-CM $672M Q2 Total Net Net Revenues Phase 3 Study Product Revenues (+77% YoY) nucresiran (+64% YoY) ATTR-CM Patie
Slide 7: Continued Progress Against Fulfillment of Ambitious Goals
Continued Progress Against Fulfillment of Ambitious 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
Slide 8: Commercial Highlights
Commercial Highlights Tolga Tanguler Chief Commercial Officer 8
Slide 9: Accelerating Performance in Q2 2025
Accelerating Performance in Q2 2025 Strong Growth Achieved Across Franchises and Regions Q2 2025 Overall Portfolio $672M +64% +43% YoY growth1 QoQ growth1 Total Net Product Revenues vs. Q2’24 vs. Q1’25 TTR Franchise Rare Franchise 9 1 With FX impact. For growth at CER = constant exchange rate – see
Slide 10: Q2 2025: Driving Consistent Rare Franchise Growth
Q2 2025: Driving Consistent Rare Franchise Growth $128M Total Rare Global Q2 2025 Net Product Revenues Q2 2025 Rare Franchise Highlights YoY % Growth QoQ % Growth +24% +17% GIVLAARI 30% 21% $128M OXLUMO 15% 11% $111M $108M $109M TOTAL Rare 24% 17% $31M $103M $25M $25M $25M $28M • GIVLAARI YoY +30% g
Slide 11: Q2 2025: Growth Inflection Driven by U.S. ATTR-CM Launch
Q2 2025: Growth Inflection Driven by U.S. ATTR-CM Launch $544M Total TTR Global Q2 2025 Net Product Revenues Q2 2025 TTR Franchise Highlights YoY % Growth QoQ % Growth +77% +51% U.S. 125% 80% $544M ROW 18% 10% $131M Global 77% 51% $30M • U.S. Q2 ‘25 vs. Q1 ‘25 (QoQ) +80% growth highlights: $359M Rev
Slide 12: Q2 2025: Step Change in Growth Indicates ~$150 Million in U.S. ATTR-CM Launch Revenue
Q2 2025: Step Change in Growth Indicates ~$150 Million in U.S. ATTR-CM Launch Revenue PN Only PN+CM U.S. AMVUTTRA +$170M U.S. ONPATTRO $383M Average quarterly growth Q1’24 – Q1‘25 +10% +$18M +$10M +$24M +$14M $214M Q2 growth $203M $171M $185M $361M driven by $147M CM launch $183M $198M $148M $169M $
Slide 13: ATTR-CM U.S. Launch Focused on 3 Key Enablers
ATTR-CM U.S. Launch Focused on 3 Key Enablers ✓ Large Majority of U.S. patients covered for AMVUTTRA in 1L ✓ Most patients pay $0 in out-of- pocket costs Health Access & System Affordability ✓ AMVUTTRA on Formulary Setup for majority of ~170 priority Health Systems Treatment CM patients on therapy C
Slide 14: ATTR-CM | Rapid Progress on Health System Setup
ATTR-CM | Rapid Progress on Health System Setup >2,000 AMVUTTRA on formulary Alternate Sites of for majority of ~170 priority Care enabled Health System Access & Affordability Health Systems Setup U.S. patients able to receive AMVUTTRA Treatment Choice ~90% within ~10 miles of home 14 Source: Data o
Slide 15: ATTR-CM | Patients Are Accessing AMVUTTRA in First Line
ATTR-CM | Patients Are Accessing AMVUTTRA in First Line Large majority of U.S. patients have first-line payer coverage (no step through required) Health Access & System Affordability Setup Most patients have paid Treatment Choice $0 Confirmed utilization out of pocket across all payer segments 15 So
Slide 16: ATTR-CM | HCPs & Patients Are Choosing AMVUTTRA
ATTR-CM | HCPs & Patients Are Choosing AMVUTTRA ~1,400 CM patients on therapy with Broad & balanced utilization AMVUTTRA in Q2 (i.e., New To Treatment & Stabilizer Health Access & System Progressor; Academic & Community) Affordability Setup Treatment Choice 3X increase in total AMVUTTRA prescribers
Slide 17: Strong Launch Progress Expected to Drive Sustainable Growth
Strong Launch Progress Expected to Drive Sustainable Growth ✓ Well positioned for TTR leadership ✓ Growing & underserved category ✓ Expanding impact with ROW ATTR-CM launches 17
Slide 18: Pipeline
Pipeline Pushkal Garg, M.D. Chief Research & Development Officer 18
Slide 19: Additional Data from HELIOS-B Continue to Drive Confidence in AMVUTTRA's Clinically Differentiated Profile
Additional Data from HELIOS-B Continue to Drive Confidence in AMVUTTRA’s Clinically Differentiated Profile Cardiac Structure Biomarkers1 Survival3 & Function2 Vutrisiran improved NT-proBNP Vutrisiran was associated with improvements in Vutrisiran reduced all-cause & and Troponin I levels vs. placebo
Slide 20: Nucresiran TRITON Phase Program
Nucresiran TRITON Phase 3 Program Next-generation silencer with potential for greater TTR knockdown, improved efficacy, and biannual dosing Targeting Launch CVO primary endpoint ~2030 (event driven) • Randomized, double-blind, event-driven outcomes study U.S. FDA • Initiated Fast Track Designation T
Slide 21: Mivelsiran: Durable Knockdown and Tolerability Observed >1 Year
Mivelsiran: Durable Knockdown and Tolerability Observed >1 Year Multiple Doses of Mivelsiran Provided Additional Reductions in CSF Aβ42 and Aβ40 Levels Mean (SD) Change from Baseline, % 0 Mean (SE) Change from Baseline, % Mean (SE) Change from Baseline, % Mivelsiran 50 mg (N=10) Mivelsiran 75 mg (N=
Slide 22: 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 with Ca
Slide 23: Financial Summary and Upcoming Milestones
Financial Summary and Upcoming Milestones Jeff Poulton Chief Financial Officer 23
Slide 24: Q2 2025 Financial Summary
Q2 2025 Financial Summary Q2 2025 vs Q2 2025 vs ($ in millions except where noted as percentages) Q2 2024 Q2 2025 Q2 2024 Q2 2024 (Reported) (CER2) Total Net Product Revenues $410 $672 64% 62% Net Revenues from Collaborations & Royalties 249 101 (59%) Collaboration Revenue 227 61 (73%) Royalty Reven
Slide 25: 2025 Full-Year Guidance Upgraded
2025 Full-Year Guidance Upgraded Item Prior FY 2025 Guidance Updated FY 2025 Guidance Total Net Product Revenues1 $2,050 to $2,250 million $2,650 to $2,800 million • Total Rare Net Product Revenues (GIVLAARI, OXLUMO) $450 to $525 million $475 to $525 million • Total TTR Net Product Revenues (PN & CM
Slide 26: 2025 Full-Year Guidance Upgraded
2025 Full-Year Guidance Upgraded Item Prior FY 2025 Guidance Updated FY 2025 Guidance Total Net Product Revenues1 $2,050 to $2,250 million $2,650 to $2,800 million • Total Rare Net Product Revenues (GIVLAARI, OXLUMO) TTR Franchise $450 to $525 Growth million $475 to $525 million • Total TTR Net Prod
Slide 27: Alnylam 2025 Goals
Alnylam 2025 Goals Combined Net Product Revenue Guidance $2,650M – $2,800M 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 28: Q&A Session
Q&A Session Q2 2025 Financial Results 28
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Slide 30: Appendix
Appendix Q2 2025 Financial Results 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 above may not sum exactly due to rounding
Slide 32: Reconciliation of Product Revenue and Growth at Constant Currency
Alnylam Pharmaceuticals, Inc. Reconciliation of Product Revenue and Growth at Constant Currency 32 Constant Currency = Constant Exchange Rate, or CER
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