Alnylam Pharmaceuticals, Inc. · NASDAQ:ALNY
Alnylam Pharmaceuticals — Q3 2025 Earnings Presentation
30 slides from Alnylam Pharmaceuticals's earnings presentation, Oct 30, 2025
Slide 1: Third Quarter 2025 Financial Results
Third Quarter 2025 Financial Results October 30, 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: TTR Leadership Drove Q3 Results and Increase to FY25 Guidance
TTR Leadership Drove Q3 Results and Increase to FY25 Guidance TTR Leadership Growth Through Innovation Strong Financial Performance Q3 Total Net $724M Q3 Total TTR Hypertension Initiated ZENITH $851M Product zilebesiran Phase 3 Trial Net Revenues (+103% YoY) Revenues +135% YoY Alzheimer’s/ $543M Ini
Slide 7: Tremendous Progress on Alnylam P5x25 Goals
Tremendous Progress on 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; 10+ in late stages; 4+ INDs per year PERFORMANCE: ≥40% revenue CAGR through YE 2025 PROFITABI
Slide 8: Commercial Highlights
Commercial Highlights Tolga Tanguler Chief Commercial Officer 8
Slide 9: Robust Performance in Q3 2025
Robust Performance in Q3 2025 Strong Growth Achieved Across Franchises and Regions Q3 2025 Overall Portfolio $851M +103% +27% YoY growth1 QoQ growth1 Combined Net Product Revenues vs. Q3’24 vs. Q2’25 TTR Franchise Rare Franchise 9 1 With FX impact. For growth at CER = constant exchange rate – see th
Slide 10: Q3 2025: Sustained Rare Franchise Performance
Q3 2025: Sustained Rare Franchise Performance $127M Total Rare Global Q3 2025 Net Product Revenues Q3 2025 Rare Franchise Highlights QoQ % Growth YoY % Growth +14% GIVLAARI -9% 4% $128M $127M OXLUMO 13% 31% $111M $108M $109M $31M $32M TOTAL Rare -1% 14% $25M $25M $28M $16M Revenues ($M) $21M $15M •
Slide 11: AMVUTTRA ATTR-CM Launch Momentum Continues in Q3 2025
AMVUTTRA ATTR-CM Launch Momentum Continues in Q3 2025 $724M Total TTR Global Q3 2025 Net Product Revenues Q3 2025 TTR Franchise Highlights QoQ % Growth YoY % Growth +135% U.S. 42% 194% $724M ROW 13% 46% $155M Global 33% 135% $544M $27M • U.S. Q3’25 vs. Q2’25 (QoQ) +42% growth highlights: Revenues ($
Slide 12: AMVUTTRA ATTR-CM Launch Momentum Continues in Q3 2025
AMVUTTRA ATTR-CM Launch Momentum Continues in Q3 2025 Total TTR Growth in U.S. Driven by Estimated ~$300 Million in ATTR-CM Revenue PN Only PN+CM AMVUTTRA ONPATTRO +$159M $543M Average quarterly growth Q1’24 – Q1‘25 +$170M $383M Growth +10% driven +$14M +$18M +$10M $530M by CM +$24M $185M $203M $214
Slide 13: ATTR-CM U.S. Launch Momentum Continues
ATTR-CM U.S. Launch Momentum Continues ✓ Nearly all priority health systems ✓ Large majority of U.S. patients using AMVUTTRA in ATTR-CM covered for AMVUTTRA in 1L ✓ ~90% of patients can receive ✓ Most patients pay $0 in out-of- AMVUTTRA within ~10 miles pocket costs of home Health Access & System Af
Slide 14: Strong Launch Progress Expected to Drive Sustainable Growth
Strong Launch Progress Expected to Drive Sustainable Growth ✓ Well positioned for TTR leadership ✓ Growing & underserved category ✓ Adding global markets in 2026+ 14
Slide 15: Pipeline
Pipeline Pushkal Garg, M.D. Chief Research & Development Officer 15
Slide 16: HELIOS-B Data Continue to Support AMVUTTRA's Clinically Differentiated Profile
HELIOS-B Data Continue to Support AMVUTTRA’s Clinically Differentiated Profile Long-term vutrisiran treatment significantly Evidence of fewer gastrointestinal events in reduced the risk of ACM and CV events1 ATTR-CM patients treated with vutrisiran5 Time-to-First Event 37% Risk Reduction (nominal p=
Slide 17: Nucresiran TRITON Phase 3 Program
Nucresiran TRITON Phase 3 Program Next-generation silencer with potential for greater TTR knockdown, improved efficacy, and biannual dosing Targeting Launch ~2030 • Randomized, double-blind, event-driven outcomes study of nucresiran versus placebo (N ~ 1200) • Primary endpoint of all-cause mortality
Slide 18: Zilebesiran: Targeting Continuous Blood Pressure Control
Zilebesiran: Targeting Continuous Blood Pressure Control to Reduce Cardiovascular & Renal Risk Reduce SBP throughout Avoid exaggerated BP variability Placebo-Adjusted Change from Baseline to the 24-Hour Period over 24-Hour and long-term Month 3 in Office Systolic BP1 24-Hour BP BP Level Variability
Slide 19: Phase 3 Cardiovascular Outcomes Trial Design
Phase 3 Cardiovascular Outcomes Trial Design Randomized, Double-Blind, Event-Driven Study in High CV Risk Patients with Uncontrolled Hypertension Study Population • Adult patients with uncontrolled hypertension and established CV disease or at high risk • Office systolic BP ≥140 mmHg on stable treat
Slide 20: ALN-6400: Potential Universal Hemostatic Agent
ALN-6400: Potential Universal Hemostatic Agent Therapeutic Hypothesis: Lowering Plasminogen to Reduce Bleeding Hereditary Hemorrhagic Telangiectasia (HHT): Initial Clinical Proof-of-Mechanism1 Second most common inherited bleeding disorder Healthy Volunteer Dosed with Placebo 90% live with recurrent
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 AMVUTTRA® (vutrisiran) ATTR Amyloidosis with Cardiomyopathy and hATTR Amyloidosis with Polyneuropathy TTR Nucresiran ATTR Amyloidosis with Cardiomyopathy N
Slide 22: Financial Summary and Upcoming Milestones
Financial Summary and Upcoming Milestones Jeff Poulton Chief Financial Officer 22
Slide 23: Q3 2025 Financial Summary
Q3 2025 Financial Summary Q3 2025 vs Q3 2025 vs ($ in millions except where noted as percentages) Q3 2024 Q3 2025 Q3 2024 Q3 2024 (Reported) (CER2) Total Net Product Revenues $420 $851 103% 99% Net Revenues from Collaborations & Royalties 81 398 393% Collaboration Revenue 57 352 513% Royalty Revenue
Slide 24: 2025 Full-Year Guidance
2025 Full-Year Guidance Item Prior FY 2025 Guidance Updated FY 2025 Guidance Total Net Product Revenues1 $2,650 to $2,800 million $2,950 to $3,050 million • Total Rare Net Product Revenues (GIVLAARI, OXLUMO) $475 to $525 million Reiterate FY Guidance • Total TTR Net Product Revenues (PN & CM) (AMVUT
Slide 25: Alnylam 2025 Goals
Alnylam 2025 Goals Combined Net Product Revenue Guidance $2,950M – $3,050M 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 Q3 2025 Financial Results 26
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Slide 28: Appendix
Appendix Q3 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 Constant Currency = Constant Exchange Rate, or CER
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