Alnylam Pharmaceuticals, Inc. · NASDAQ:ALNY
Alnylam Pharmaceuticals — Investor Presentation (January 2023)
28 slides from Alnylam Pharmaceuticals's investor presentation, January 2023
Slide 1: Alnylam Pharmaceuticals
Nathan (USA) Diagnosed with AHP Alnylam Pharmaceuticals January 2023 © 2023 Alnylam Pharmaceuticals, Inc. 1
Slide 2: 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 3: Alnylam Poised to Become a Top-Tier Biotech
Alnylam Poised to Become a Top-Tier Biotech Pioneer in RNAi Therapeutics • 5 medicines approved in < 4 years • Robust clinical pipeline across rare, specialty, and prevalent diseases • Global footprint with strong commercial capabilities • Strong balance sheet, on path toward financial self- sustain
Slide 4: Notable Accomplishments in 2022
Notable Accomplishments in 2022 Combined net product revenues of Reported positive Phase 3 study results in FIFTH $894 million* patients with ATTR-CM; RNAi therapeutic approved (35% growth YoY) sNDA submitted Dec. 8, 2022 CTA filings 3 • ALN-TTRsc04 • ALN-KHK • ALN-PNP Initiated first-in-human clini
Slide 5: Robust Commercial Execution Driven by Strong AMVUTTRA Launch
Robust Commercial Execution Driven by Strong AMVUTTRA Launch 2022 Combined Net Product Revenues ($894M) Represent 35% YoY Growth (43% CER)* TTR Ultra-Rare Disease Franchise Portfolio Patients on Therapy Patients on Therapy >2,975 >800 >2,580 >690 >2,400 >620 >2,200 >2,050 >560 >490 Q4 2021 Q1 2022 Q
Slide 6: 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 7: High-Yield Productivity of Alnylam RNAi Therapeutics Platform
High-Yield Productivity of Alnylam RNAi Therapeutics Platform Comparison of Historical Metrics to Alnylam Portfolio over Past Decade1 Probability of Success (POS) by Phase Transition 100 90 86.7 87.5 81.8 80 69.2 70 Percent POS 63.7 62.0 60 50 44.5 38.6 40 35.2 30 27.4 20 10.3 10 5.7 0 % POS, Phase
Slide 8: 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 9: Multiple Drivers of Future Growth
Multiple Drivers of Future Growth TTR Franchise Leadership Expansion Beyond Rare Diseases Engine for Sustainable Innovation 9
Slide 10: Multiple Drivers of Future Growth
Multiple Drivers of Future Growth TTR Franchise Leadership Expansion Beyond Rare Diseases Engine for Sustainable Innovation Liana (Brazil) Diagnosed with 10 hATTR amyloidosis
Slide 11: ATTR Amyloidosis
ATTR Amyloidosis Rare, Progressively Debilitating, and Fatal Disease Description Caused by a misfolded TTR protein that accumulates GI: as amyloid deposits in multiple tissues including heart, Diarrhea Nausea nerves, and GI tract1 Vomiting CARDIAC: GU: Heart failure Proteinuria Hereditary ATTR (hATT
Slide 12: Building a Leading TTR Franchise
Building a Leading TTR Franchise Potential to Expand Benefit to Patients Globally for Many Years to Come Novel siRNA Conjugates^ Ocular & CNS hATTR Amyloidosis ALN-TTRsc04 ATTR Amyloidosis Patisiran TBD1 Phase 3 ATTR Amyloidosis with CM (incl. WT)‡ Stargardt Disease Biannual Dosing Vutrisiran Vutris
Slide 13: Patisiran sNDA Submitted with Positive Study Results
Patisiran sNDA Submitted with Positive Study Results If Approved, Broadens Commercial Opportunity to Full Spectrum of ATTR Amyloidosis Patients Patisiran demonstrated statistically significant and clinically meaningful improvements in functional capacity, health status and quality of life compared t
Slide 14: Safety and Exploratory Endpoints Support Patisiran's Therapeutic Potential
Safety and Exploratory Endpoints Support Patisiran’s Therapeutic Potential Biomarker Based Disease Progression at Month 12a,b • Majority of AEs were mild or moderate in severity • AEs ≥5% in the patisiran group observed 3% more commonly than in placebo included infusion-related Change from Baseline
Slide 15: 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 16: Significant Commercial Potential in ATTR Amyloidosis
Significant Commercial Potential in ATTR Amyloidosis* Initial Entry with Patisiran and Potential Expansion with Vutrisiran • Potential for a robust efficacy profile, including outcomes data • Potential best-in-class product profile • First RNAi therapeutic demonstrating impact across a spectrum of d
Slide 17: Multiple Drivers of Future Growth
Multiple Drivers of Future Growth TTR Franchise Leadership Expansion Beyond Rare Diseases Engine for Sustainable Innovation 17
Slide 18: RNAi Therapeutics Profile Supports Potential Beyond Rare Diseases
RNAi Therapeutics Profile Supports Potential Beyond Rare Diseases • Durability • Clamped pharmacology • Potential for improved adherence • Supportive safety profile ULTRA RARE RARE/SPECIALTY PREVALENT GIVLAARI® ONPATTRO®: hATTR w/ PN1 ALN-TTRsc04 Leqvio®4 ALN-HSD OXLUMO® AMVUTTRA® : hATTR w/ PN2 Fit
Slide 19: Uncontrolled Hypertension is a Global Health Crisis
Uncontrolled Hypertension is a Global Health Crisis Hypertension is Highly Prevalent and Carries Substantial Risk of CV Morbidity and Mortality Potential complications of uncontrolled hypertension Primary Hypertension1 High CV Risk with Hypertension2 in 7 Major Markets, 2020 in 7 Major Markets, 2020
Slide 20: Zilebesiran: Potential Novel Treatment for Patients with Hypertension
Zilebesiran: Potential Novel Treatment for Patients with Hypertension Compelling Phase 1 Data Support Transformative Product Profile Serum AGT Lowering Blood Pressure Reduction 40 Week 12 Week 24 Placebo 200 mg 400 mg 800 mg 200 mg 400 mg 800 mg 20 Baseline in 24-hour ABPM, mmHg (n=10) (n=2) (n=8) (
Slide 21: Additional Growth Opportunities via Partnered Programs
Additional Growth Opportunities via Partnered Programs ALN-HBV02 (VIR-2218) FITUSIRAN CEMDISIRAN/POZELIMAB Hepatitis B/D Virus Infection Hemophilia Complement-Mediated Diseases Potential for functional cure Innovative approach to hemophilia A Novel approach providing of chronic HBV infection and B,
Slide 22: Multiple Drivers of Future Growth
Multiple Drivers of Future Growth TTR Franchise Leadership Expansion Beyond Rare Diseases Engine for Sustainable Innovation 22
Slide 23: Two-Decade Track Record of Industry Leadership in RNAi
Two-Decade Track Record of Industry Leadership in RNAi Human Genetics Platform Designs Extrahepatic Delivery 12 months IKARIA™ Reversir™ GEMINI™ • Sourcing novel, genetically validated • IKARIA™ enables robust target • Novel conjugates with variety of ligands targets knockdown with annual dosing pot
Slide 24: Expanding Beyond Liver with First CNS Program in Clinic
Expanding Beyond Liver with First CNS Program in Clinic Multiple Patient Populations with High Unmet Need ALN-APP lowers APP production at its source, Durable Effect in NHP CSF sAPPα and sAPPß Protein Knockdown upstream of pathogenic process (Single Intrathecal Dose in NHP) (% remaining relative to
Slide 25: Alnylam 2023 Goals
Alnylam 2023 Goals Early Mid Late Combined Net Product Revenue Guidance to be Provided at Q4/YE 2022 Earnings PATISIRAN ATTR Amyloidosis FDA Approval of sNDA Biannual Dosing Regimen Data VUTRISIRAN ATTR Amyloidosis Submit sNDA for Biannual Dosing Regimen ALN-TTRsc04* ATTR Amyloidosis Phase 1 Topline
Slide 26: Alnylam 2023 Goals
Alnylam 2023 Goals Early Mid Late Combined Net Product Revenue Guidance to be Provided at Q4/YE 2022 Earnings PATISIRAN ATTR Amyloidosis FDA Approval of sNDA Biannual Dosing Regimen Data VUTRISIRAN ATTR Amyloidosis • Commercial execution across fourforproducts Submit sNDA Biannual Dosing Regimen ALN
Slide 27: Nurturing a Winning Culture
Nurturing a Winning Culture Commitment to People Diversity, Equity, & Inclusion Scientific Innovation Social Responsibility 27
Slide 28: Thank You!
| || Thank You! ‘Alnylam
Alnylam Pharmaceuticals — Investor Presentation (January 2023) on 6ix