Alnylam Pharmaceuticals, Inc. · NASDAQ:ALNY
Alnylam Pharmaceuticals — Q3 2023 Earnings Presentation
27 slides from Alnylam Pharmaceuticals's earnings presentation, Nov 2, 2023
Slide 1: Third Quarter 2023 Financial Results
Nathan (USA) Diagnosed with AHP Third Quarter 2023 Financial Results November 2, 2023 © 2023 Alnylam Pharmaceuticals, Inc. 1
Slide 2: Agenda
Agenda Welcome • Christine Lindenboom Senior Vice President, Investor Relations & Corporate Communications Overview • Yvonne Greenstreet, MBChB, MBA Chief Executive Officer Commercial Highlights • Tolga Tanguler Chief Commercial Officer Alnylam Pipeline • Pushkal Garg, M.D. Chief Medical Officer Fin
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: R&D Day Save the Date
SAVE THE DATE R&D Day December 13, 2023 8:30 am ET (held virtually) Registration information forthcoming 5
Slide 6: Ambitious Five-Year Strategy to Drive Growth
Ambitious Five-Year Strategy to Drive Growth Patients: Over 0.5 million on Alnylam RNAi therapeutics globally Products: 6+ marketed products in rare and prevalent diseases Pipeline: Over 20 clinical programs, with 10+ in late stages and 4+ INDs per year Performance: ≥40% revenue CAGR through YE 2025
Slide 7: Commercial Highlights
Tolga Tanguler Chief Commercial Officer Commercial Highlights 7
Slide 8: TTR Franchise Update: Q3 2023
TTR Franchise Update: Q3 2023 $230M >3,790 Total TTR Total TTR patients Global Q3 2023 worldwide at end of Net Product Revenues Q3 2023 >3,790 Q3 TTR Franchise Highlights >3,490 Patients on Therapy >2,975 >3,160 YoY % Growth QoQ % Growth >2,580 ~1,995 ~640 ~1,110 ~1,670 ~180 U.S. 47% 11% ROW 22% -7%
Slide 9: Ultra Rare Franchise Update: Q3 2023
Ultra Rare Franchise Update: Q3 2023 $83M >1,000 Total Ultra Rare Total Ultra Rare Global Q3 2023 patients worldwide Net Product Revenues at end of Q3 2023 >1,000 Q3 Ultra Rare Franchise Highlights >920 >850 Patients on Therapy >800 >715 ~375 ~300 ~350 YoY % Growth QoQ % Growth ~280 ~240 GIVLAARI 19
Slide 10: Alnylam Pipeline
Pushkal Garg, M.D. Chief Medical Officer Alnylam Pipeline 10
Slide 11: APOLLO-B Phase 3 Study (Open-Label Extension)
APOLLO-B Phase 3 Study (Open-Label Extension) Treatment Effect of Patisiran Sustained at 24 Months 6-MWT KCCQ 11 Maurer, et al. HFSA 2023
Slide 12: Vutrisiran Phase 3 Study
Vutrisiran Phase 3 Study Randomized, Double-Blind Outcomes Study in ATTR Amyloidosis Patients with Cardiomyopathy Primary Endpoint N = 655 • Composite outcome of all-cause mortality and recurrent CV events (when Vutrisiran last patient reaches Month 30) Patient Population SC q3M 1:1 RANDOMIZATION •
Slide 13: Amyloid Precursor Protein (APP) Target for Alzheimer's Disease and CAA
Amyloid Precursor Protein (APP) Target for Alzheimer’s Disease and Cerebral Amyloid Angiopathy APP: One target, two distinct pathological processes Genetically validated target for AD and CAA Soluble biomarkers of target engagement (sAPPα and sAPPβ) in CSF Significant patient population with h
Slide 14: ALN-APP Phase 1 Results
ALN-APP Phase 1 Results Rapid and Sustained Reductions in CSF sAPPα and sAPPβ Median Percent Change from Baseline in CSF sAPPα Median Percent Change from Baseline in CSF sAPPβ • All AEs were mild or moderate in severity • No deaths, SUSARs, or treatment or study discontinuations occurred • One indiv
Slide 15: Robust Reductions in CSF Aβ42 and Aβ40 at Month 2
Robust Reductions in CSF Aβ42 and Aβ40 at Month 2 Median Change from Baseline in CSF Aβ42 Median Change from Baseline in CSF Aβ40 • CSF safety biomarkers, routine lab assessments, and preliminary data for the exploratory biomarker neurofilament light chain (NfL) all continued to show no concerning t
Slide 16: Zilebesiran Phase 2 KARDIA Program
Zilebesiran Phase 2 KARDIA Program Monotherapy Phase 2 Study (N = 394) Combination Phase 2 Study (N = 672) • Enrollment completed December 2022 • Enrollment completed June 2023 • Positive topline results announced Sept. 2023 • Topline results expected early 2024 • Full results to be presented at AHA
Slide 17: Alnylam Clinical Development Pipeline
Alnylam Clinical Development Pipeline Focused in 4 Strategic Therapeutic Areas (STArs): Genetic Medicines Cardio-Metabolic Diseases EARLY/MID-STAGE LATE STAGE REGISTRATION/ COMMERCIAL Infectious Diseases CNS/Ocular Diseases (IND/CTA Filed-Phase 2) (Phase 2-Phase 3) COMMERCIAL1 RIGHTS hATTR Amyloidos
Slide 18: Financial Summary and Upcoming Milestones
Jeff Poulton Chief Financial Officer Financial Summary and Upcoming Milestones 18
Slide 19: Q3 2023 Financial Summary
Q3 2023 Financial Summary Q3 Reported Q3 CER Financial Results ($ millions) Q3 2023 Q3 2022 Growth % Growth %2 Net Product Revenues $313 $232 35% 33% Net Revenues from Collaborations $427 $29 Royalty Revenues $10 $3 Total Revenues $751 $264 184% 182% Product Cost of Goods Sold $79 $37 Cost of Collab
Slide 20: 2023 Updated Full Year Guidance
2023 Updated Full Year Guidance1 Prior FY 2023 Guidance Updated FY 2023 Guidance Net Product Revenue: ONPATTRO, AMVUTTRA, GIVLAARI, $1,200M to $1,285M No change OXLUMO Net Product Revenue Growth vs. 2022 at reported Fx rates 34% to 44% No change Net Product Revenue Growth vs. 2022 at constant exchan
Slide 21: Alnylam 2023 Goals
Alnylam 2023 Goals Early Mid Late Combined Net Product Revenue Guidance $1,200M – $1,285M VUTRISIRAN ATTR Amyloidosis Biannual Dosing Regimen Data ALN-TTRsc04* ATTR Amyloidosis Phase 1 Topline Results Complete KARDIA-2 Enrollment ZILEBESIRAN* Hypertension KARDIA-1 Phase 2 Topline Results ALN-APP* Al
Slide 22: Q&A Session
Q3 2023 Financial Results Q&A Session 22
Slide 23: Thank You!
| || Thank You! “LAlnylam
Slide 24: Appendix
Q3 2023 Financial Results Appendix 24
Slide 25: 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 September 30, September 30, 2023 2022 Reconciliation of GAAP to Non-GAAP research and development: GAAP research and development $ 253,179 $ 245,371 Less: Stock-based compens
Slide 26: Reconciliation of Revenue and Growth at Constant Currency
Alnylam Pharmaceuticals, Inc. Reconciliation of Revenue and Growth at Constant Currency Three Months Ended September 30, 2023 Total TTR net product revenue growth, as reported 35% Add: Impact of foreign currency translation (1) Total TTR net product revenue growth at constant currency 34% Total Ultr
Slide 27: Summary of Accounting Conclusion for the Roche Transaction
Summary of Accounting Conclusion for the Roche Transaction Transaction Consideration P&L Impact Recognized in collaboration revenue when license agreement $310 million upfront payment signed (Q3 2023) Development milestones Recognized in collaboration revenue when achieved Commercial milestones Reco
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