Alnylam Pharmaceuticals, Inc. · NASDAQ:ALNY

Alnylam Pharmaceuticals — Q3 2026 Earnings Presentation

33 slides from Alnylam Pharmaceuticals's earnings presentation, Oct 29, 2026

  1. Slide 1: Third Quarter 2026 Financial Results

    Third Quarter 2026 Financial Results October 29, 2026 © 2026 Alnylam Pharmaceuticals, Inc. 1

  2. Slide 2: Agenda

    Agenda Josh Brodsky 01 Welcome Vice President, Investor Relations Yvonne Greenstreet, M.D. 02 Overview Chief Executive Officer Tolga Tanguler 03 Commercial Highlights Chief Commercialization Officer Pushkal Garg, M.D. 04 R&D and Pipeline Perspective Chief Research & Development Officer Jeff Poulton

  3. Slide 3: Alnylam Forward-Looking Statements

    Alnylam Forward-Looking Statements This presentation contains forward-looking statements. Forward looking statements include statements regarding Alnylam’s expectations, beliefs, goals, plans or prospects including, without limitation, statements regarding: the potential for continued growth of AMVU

  4. Slide 4: Overview

    Overview Yvonne Greenstreet, M.D. Chief Executive Officer 4

  5. Slide 5: Q3 2026 Results Summary

    Q3 2026 Results Summary Financial Performance TTR Leadership Pipeline Progress Q3 Total Net Presented Promising Initial Phase 1 $1,292M Product $1,150M Q3 Total TTR Results for Brezosiran (ALN-HTT02)1 (+52% YoY) (+59% YoY) Net Revenues Revenues • Mean reduction of CSF HTT of 60-64% • Placebo-control

  6. Slide 6: Alnylam 2030

    Alnylam 2030 Accelerating Innovation. Scaling Impact. Achieve Global TTR Leadership BUILD A DURABLE TTR FRANCHISE • Lead TTR market in revenue by 2030 and cumulatively across 5-year period • Launch best-in-class, next-gen silencer, nucresiran, in PN by 2028 and CM by 2030 Grow Through Sustainable In

  7. Slide 7: Commercial Highlights

    Commercial Highlights Tolga Tanguler Chief Commercialization Officer 7

  8. Slide 8: Commercial Performance in Q3 2026

    Commercial Performance in Q3 2026 Q3 2026 Overall Portfolio $1.29B +52% +10% YoY growth1 QoQ growth1 Combined Net Product Revenues vs. Q3’25 vs. Q2’26 TTR Franchise Rare Franchise 1 With FX impact. For growth at CER = constant exchange rate – see the Financial Summary slide for more information. 8

  9. Slide 9: ATTR-CM Launch Continues to Fuel Franchise Growth

    ATTR-CM Launch Continues to Fuel Franchise Growth $1.15B Total TTR Global Q3 2026 Net Product Revenues Q3 2026 TTR Franchise Highlights QoQ % Growth YoY % Growth +$120M +59% +12% Global TTR U.S. 12% 69% vs. Q2 ROW 10% 28% $1,150M +$20M Global 12% 59% $1,030M $222M ROW TTR vs. Q2 $910M $203M $10M $85

  10. Slide 10: Expanding AMVUTTRA Utilization & Reach in a Growing U.S. TTR Market

    Expanding AMVUTTRA Utilization & Reach in a Growing U.S. TTR Market Category Prescriber AMVUTTRA Total • [+70% TTR TRx] Growth Expansion Rx Share in ATTR (TRx) 37% 2,000 24% +XX +XX +XX ~ > ~ ATTR-CM Category TRx volume New, unique AMV prescribers TRx share (total prescriptions, growth Q3’26 vs Q3’2

  11. Slide 11: Committed to Patient Access

    Committed to Patient Access Options Exist to Mitigate Access Risks Resulting From Potential 340B Reimbursement Changes Potential CMS Proposal Alnylam Approach Provider Responses • Proposed rule change would reduce • Continue traditional 340B • Options to address potential risks hospital outpatient r

  12. Slide 12: Committed to Patient Access: Broad and Favorable Access

    Committed to Patient Access Today, patients ~99% ~90% have broad and favorable access to of covered lives of patients can access AMVUTTRA have access; large treatment within ~10 majority in 1L miles of home Most pay $0 in out-of-pocket costs 12

  13. Slide 13: R&D and Pipeline Perspective

    R&D and Pipeline Perspective Pushkal Garg, M.D. Chief Research & Development Officer 13

  14. Slide 14: Strongly Positioned for Durable TTR Leadership

    Strongly Positioned for Durable TTR Leadership AM V U T T R A N U C R E S I R AN HELIOS-B Data Support AMVUTTRA as the Learnings from CARDIO-TTRansform First-Line Treatment of Choice in ATTR-CM Strengthen Our Conviction in TRITON-CM Consistency in effect across endpoints and subgroups TRITON-CM comb

  15. Slide 15: Key Learnings from CARDIO-TTRansform

    Key Learnings from CARDIO-TTRansform Less More Advanced Suboptimal TTR Knockdown Patient Population Endpoint Selection 15

  16. Slide 16: TRITON-CM: Optimized for Success & Broad Patient Impact

    TRITON-CM: Optimized for Success & Broad Patient Impact Enriched for patient population most likely to benefit • BNP limits specified in inclusion/exclusion criteria Event-driven outcomes study • Primary composite of all-cause mortality and recurrent cardiovascular events Enrollment expanded from N

  17. Slide 17: Industry Leading Pipeline of RNAi Therapeutics

    Industry Leading Pipeline of RNAi Therapeutics PHASE 1 PHASE 2 PHASE 3 Nucresiran ATTR Amyloidosis with Cardiomyopathy TTR Nucresiran hATTR Amyloidosis with Polyneuropathy Zilebesiran2 Hypertension CARDIOVASCULAR Zilebesiran REVERSIR2 Hypertension ALN-4324 (GRB14) Type 2 Diabetes Mellitus Rapirosira

  18. Slide 18: Huntington's Disease Robs Generations of Patients of Body and Mind

    Huntington’s Disease (HD) Robs Generations of Patients of Body and Mind Progressive, Fatal Monogenic Neurodegenerative Disease Movement: chorea, incoordination, falls Thinking: executive dysfunction, memory loss Mood: depression, apathy, personality change, suicidality Woody Guthrie Nancy Wexler & p

  19. Slide 19: One Gene, Two Potential Culprits

    One Gene, Two Potential Culprits One gene, two proteins The mutant gene gives rise to full-length mutant HTT (67 exons) and a shorter, toxic isoform, HTT1a (exon 1 only)² HTT1a mRNA (Exon 1) HTT mRNA (Exon 1-67) Both likely contribute to disease Full-length mutant HTT and the shorter HTT1a isoform i

  20. Slide 20: Robust, Dose-Dependent CSF mHTT Reduction Demonstrated with SMC Assay

    Robust, Dose-Dependent CSF mHTT Reduction Demonstrated with SMC Assay Up to 64% Mean Reduction at Month 3 and up to 80% Individual Reduction in CSF mHTT After a Single IT Dose of Brezosiran Placebo (N=12) Participants with Brezosiran Dose 1 (N=5) measurement below LLoQ, n (%) Brezosiran Dose 2 (N=4)

  21. Slide 21: Encouraging Safety & Tolerability Profile

    Encouraging Safety & Tolerability Profile CSF WBC CSF NfL – Fold Change Placebo range Placebo (N=12) Brezosiran Dose 2 (N=4) Brezosiran Dose 4 (N=9) Brezosiran Dose 1 (N=5) Brezosiran Dose 3 (N=9) Brezosiran Dose 5 (N=9) CSF WBC dashed line denotes typical reference values. CSF NfL shaded placebo ra

  22. Slide 22: Advancing Brezosiran to Pivotal Trial, Pending Regulatory Alignment

    Advancing Brezosiran to Pivotal Trial, Pending Regulatory Alignment Phase 1 Open-Label Extension Study Continuing ONGOING ONGOING Phase 1b study Phase 1b open-label extension study (adults with HD) (adults with HD) Single ascending dose study to characterize All participants eligible; repeat dosing

  23. Slide 23: 2026 Pipeline Goals to Drive Our Next Phase of Growth

    2026 Pipeline Goals to Drive Our Next Phase of Growth Advance TRITON-CM Phase 3 Trial Ongoing Nucresiran ATTR Amyloidosis Advance TRITON-PN Phase 3 Trial Ongoing Zilebesiran Hypertension Advance ZENITH Phase 3 Trial Ongoing Cerebral Amyloid Complete Enrollment of cAPPricorn-1 Phase 2 Trial H1 Mivels

  24. Slide 24: Financial Summary

    Financial Summary Jeff Poulton Chief Financial Officer 25

  25. Slide 25: Q3 2026 Financial Summary

    Q3 2026 Financial Summary Q3 2026 vs Q3 2026 vs ($ in millions except where noted as percentages) Q3 2025 Q3 2026 Q3 2025 Q3 2025 (Reported) (CER2) Total Net Product Revenues $851 $1,292 52% 53% Net Revenues from Collaborations & Royalties 398 166 -58% Collaboration Revenue 352 71 -80% Royalty Reven

  26. Slide 26: 2026 Reiterated Full Year Guidance

    2026 Reiterated Full Year Guidance Item FY 2026 Guidance Total Net Product Revenues1 $4,700 to $5,100 million • Total Rare Net Product Revenues (GIVLAARI, OXLUMO) $500 to $600 million • Total TTR Net Product Revenues (AMVUTTRA, ONPATTRO) $4,200 to $4,500 million Net Product Revenues Growth vs. 2025

  27. Slide 27: Q&A Session

    Q&A Session Q3 2026 Financial Results 28

  28. Slide 28: Poised for Continued Growth

    Poised for Continued Growth Commercial Pipeline Progress Toward Execution Momentum Alnylam 2030 29

  29. Slide 29: Silence disease & Amplify life

    Silence disease@@aa, & Amplify life Wri fl 15 \! “YAlnylam

  30. Slide 30: Appendix

    Appendix Q3 2026 Financial Results 31

  31. Slide 31: Q3 2026: Sustained Rare Franchise Performance

    Q3 2026: Sustained Rare Franchise Performance $142M Total Rare Global Q3 2026 Net Product Revenues Q3 2026 Rare Franchise Highlights QoQ % Growth YoY % Growth +12% $142M $142M GIVLAARI 0% 21% $136M $126M $126M $34M $34M OXLUMO 1% (1%) $32M $32M $34M TOTAL Rare 0% 12% $19M $18M $18M $21M $17M $30M $3

  32. Slide 32: Reconciliation of Selected GAAP Measures to Non-GAAP Measures

    Alnylam Pharmaceuticals, Inc. Reconciliation of Selected GAAP Measures to Non-GAAP Measures (In thousands) Please note that the figures presented above may not sum exactly due to rounding 33

  33. Slide 33: Reconciliation of Product Revenue and Growth at Constant Currency

    Alnylam Pharmaceuticals, Inc. Reconciliation of Product Revenue and Growth at Constant Currency Constant Currency = Constant Exchange Rate, or CER 34

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