Alnylam Pharmaceuticals, Inc. · NASDAQ:ALNY
Alnylam Pharmaceuticals — Q4 2024 Earnings Presentation
29 slides from Alnylam Pharmaceuticals's earnings presentation, Feb 13, 2025
Slide 1: Fourth Quarter and Full Year 2024 Financial Results
Fourth Quarter and Full Year 2024 Financial Results February 13, 2025 1 © 2025 Alnylam Pharmaceuticals, Inc.
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 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
Yvonne Greenstreet, MBChB, MBA Chief Executive Officer Overview 4
Slide 5: 2024 Delivered Strong Progress Across the Business
2024 Delivered Strong Progress Across the Business Portfolio & Pipeline Financials & Culture Positive initial multi-dose Highly Positive HELIOS-B results with mivelsiran Combined net Phase 3 results product revenues: Initiated cAPPricorn-1 $1,646 million Phase 2 study in CAA (33% growth YoY) Global
Slide 6: 2025: A Landmark Year for Alnylam
2025: A Landmark Year for Alnylam Growth Strong TTR Through Financial Leadership Innovation Performance Best-in-Class Team + Award-Winning Culture 6
Slide 7: Strong Progress Against Ambitious Five-Year Goals
Strong 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 PR
Slide 8: Commercial Highlights
Tolga Tanguler Chief Commercial Officer Commercial Highlights 8
Slide 9: Commercial Portfolio – Continued Strong Performance in Q4 24
Commercial Portfolio – Continued Strong Performance in Q4 2024 TTR Franchise Rare Franchise Q4 2024 $451M 30% 7% Combined Net Product Revenue YoY growth1 vs. Q4’23 QoQ growth1 vs. Q3’24 Full Year 2024 $1,646M 33% Combined Net Product Revenue YoY growth1 vs. FY’23 9 1 With FX impact. For growth at CE
Slide 10: TTR Franchise Update: Q4 2024
TTR Franchise Update: Q4 2024 $343M Total TTR Global Q4 2024 Net Product Revenues Q4 TTR Franchise Highlights +35% +11% YoY % Growth QoQ % Growth $343M U.S. 42% 10% $307M $309M ROW 25% 12% $103M $264M Global 35% 11% $254M $82M $90M • U.S. TTR franchise YoY growth of +42% driven by: $65M $53M $36M –
Slide 11: 2025 TTR Franchise Guidance
2025 TTR Franchise Guidance $1,600 to $1,725 Million 2025 Guidance Total TTR Net Product Revenues (PN & CM*) (ONPATTRO, AMVUTTRA) 11 * Guidance assumes FDA approval of the sNDA for vutrisiran for the treatment of adults with ATTR amyloidosis with cardiomyopathy by the March 23, 2025 PDUFA target act
Slide 12: Disrupting an Expanding Growth Market
Disrupting an Expanding Growth Market Highly Differentiated AMVUTTRA Value Significant Unmet Patient Need Proposition RAPID KNOCKDOWN of TTR works upstream, at the source 36% RRR in all-cause mortality in ~80% contemporary treatment setting ATTR-CM patients undiagnosed globally1 Early & consistent p
Slide 13: Approaching Launch with Durable Success in Mind
Approaching Launch with Durable Success in Mind Pre-Launch Preparation Post Approval (Ongoing) (Late Mar – Dec’25) Optimizing access pathways at ~170 Securing health systems formulary approval priority health systems (label contingent) Bolstering a broad ecosystem of Finalizing VBAs for ATTR-CM alte
Slide 14: Communicating Progress
Communicating Progress Q3’25 Q2’25 Earnings Earnings 2025 Guidance Q1’25 Customer Utilization Earnings Early Launch PDUFA, $1,600 to Indicators US Launch Initial Customer $1,725 Million Final Label Feedback & Launch Readiness Indicators Total TTR Net Product Revenues 14
Slide 15: Rare Franchise Update: Q4 2024
Rare Franchise Update: Q4 2024 $108M Total Rare Global Q4 2024 Net Product Revenues Q4 Rare Franchise Highlights +18% -3% YoY % Growth QoQ % Growth $111M $108M $103M GIVLAARI 9% -9% $101M $25M OXLUMO 33% 8% $92M $25M $25M Total Rare 18% -3% $29M $22M $15M • GIVLAARI YoY growth of +9% driven by: $19M
Slide 16: Pipeline
Pushkal Garg, M.D. Chief Medical Officer Pipeline 16
Slide 17: Vutrisiran Regulatory Status
Vutrisiran Regulatory Status On Track Toward Early 2025 Launch Pursuing Rapid Ex-U.S. Launches ✓ Submitted sNDA within 90 days ✓ Parallel filings achieved in all of topline results major regions, including Europe and Japan ✓ sNDA under Priority Review ✓ Priority Review granted in Japan ✓ PDUFA date
Slide 18: Nucresiran (ALN-TTRsc04) Offers Potential for Best-in-Class Profile
Nucresiran (ALN-TTRsc04) Offers Potential for Best-in-Class Profile Continued Innovation to Secure Franchise Leadership Emerging Profile Encouraging Phase 1 Data1 20 Mean Serum TTR Knockdown 0 ✓ >95% TTR Knockdown (percent, ± SEM) -20 ✓ Biannual or annual dosing -40 to optimize patient experience -6
Slide 19: Robust and High-Value Pipeline of RNAi Therapeutics
Robust and High-Value Pipeline of RNAi Therapeutics PHASE 1 PHASE 2 PHASE 3 APPROVED ONPATTRO® (patisiran) hATTR Amyloidosis with Polyneuropathy AMVUTTRA® (vutrisiran) hATTR Amyloidosis with Polyneuropathy TTR Vutrisiran ATTR Amyloidosis with Cardiomyopathy Nucresiran (ALN-TTRsc04) ATTR Amyloidosis
Slide 20: R&D Day
R&D Day February 25, 2025 9:00 am ET This event will be webcast 20
Slide 21: Financial Summary and Upcoming Milestones
Jeff Poulton Chief Financial Officer Financial Summary and 21 Upcoming Milestones
Slide 22: Q4 and Full Year 2024 Financial Summary
Q4 and Full Year 2024 Financial Summary Q4 Reported Q4 CER FY24 Reported FY24 CER Financial Results ($ millions) Q4 2024 Q4 2023 FY 2024 FY 2023 Growth % Growth %2 Growth % Growth %2 Net Product Revenues $451 $346 30% 29% $1,646 $1,241 33% 33% Net Revenues from Collaborations $107 $76 $510 $546 Roya
Slide 23: 2025 Full-Year Guidance
2025 Full-Year Guidance Item FY 2025 Guidance Key Assumptions • AMVUTTRA-CM approval and launch in Total TTR Product Revenues (PN & CM) U.S. by FDA PDUFA date (March 23) (ONPATTRO, AMVUTTRA) $1,600 to $1,725 million • AMVUTTRA-CM approval and launch in Germany & Japan 2H 2025 Total Rare Product Reve
Slide 24: Alnylam 2025 Goals
Alnylam 2025 Goals Combined Net Product Revenue Guidance $2,050M – $2,250M 2025 PDUFA date U.S. FDA Approval VUTRISIRAN ATTR Amyloidosis March 23, 2025 Additional Global Approvals (Japan, EU) Q2, Q3 NUCRESIRAN* ATTR Amyloidosis Initiate Phase 3 Study in ATTR-CM H1 (ALN-TTRsc04) KARDIA-3 Phase 2 Resu
Slide 25: Q&A Session
Q4 and Full Year 2024 Financial Results Q&A Session 25
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Slide 27: Appendix
Q4 and Full Year 2024 Financial Results Appendix 27
Slide 28: Reconciliation of Selected GAAP Measures to Non-GAAP Measures
Alnylam Pharmaceuticals, Inc. Reconciliation of Selected GAAP Measures to Non-GAAP Measures (In thousands) Three Months Ended Twelve Months Ended December 31, December 31, 2024 2023 2024 2023 Reconciliation of GAAP to Non-GAAP research and development: GAAP Research and development $ 300,169 $ 272,1
Slide 29: Reconciliation of Product Revenue and Growth at Constant Currency
Alnylam Pharmaceuticals, Inc. Reconciliation of Product Revenue and Growth at Constant Currency December 31, 2024 Three Months Twelve Months Ended Ended Total TTR net product revenue growth, as reported 35 % 34 % Add: Impact of foreign currency translation (1) — Total TTR net product revenue growth
Alnylam Pharmaceuticals — Q4 2024 Earnings Presentation on 6ix