Alnylam Pharmaceuticals, Inc. · NASDAQ:ALNY
Alnylam Pharmaceuticals — Q4 2022 Earnings Presentation
29 slides from Alnylam Pharmaceuticals's earnings presentation, Feb 23, 2023
Slide 1: Fourth Quarter and Full Year 2022 Financial Results
Nathan (USA) Diagnosed with AHP Fourth Quarter and Full Year 2022 Financial Results February 23, 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: 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) PDUFA date October 8th, 2023 CTA filings 3 • ALN-TTRsc04 • ALN-KHK • ALN-PNP Initiated first-in-human clini
Slide 6: 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 6 hATTR amyloidosis
Slide 7: 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 8: Commercial Highlights
Tolga Tanguler Chief Commercial Officer Commercial Highlights 8
Slide 9: TTR Franchise Update: Year End 2022
TTR Franchise Update: Year End 2022 $651M >2,975 Total TTR Global 2022 TTR patients Net Product Revenues at YE 2022 >2,975 >2,580 Q4 TTR Franchise Highlights Patients on Therapy >2,400 ~640 >2,200 ~180 >2,050 YoY % Growth QoQ % Growth ~2,400 ~2,335 U.S. 72% 12% ROW 11% 12% Global 38% 12% Q4 2021 Q1
Slide 10: AMVUTTRA (vutrisiran) Update: Year End 2022
AMVUTTRA® (vutrisiran) Update: Year End 2022 Encouraging Launch Performance Across U.S. and Ex-U.S. Markets U.S. Start Forms | Doubled “New Start U.S. Prescribers | Increasing share of Global Access Form” Monthly Average Since Launch prescribers new to TTR, Balanced and Reimbursement Growth Across A
Slide 11: GIVLAARI (givosiran) Update: Year End 2022
GIVLAARI® (givosiran) Update: Year End 2022 $173M >520 GIVLAARI Global 2022 Patients Worldwide on Commercial Q4 Highlights Net Product Revenues GIVLAARI at YE 2022 YoY % Growth QoQ % Growth ROW U.S. 3% 0% $47M U.S. $45M $46M ROW 52% 10% $41M Global 16% 3% $15M $16M $35M $16M $10M • U.S. QoQ flat gro
Slide 12: OXLUMO (lumasiran) Update: Year End 2022
OXLUMO® (lumasiran) Update: Year End 2022 $70M >280 OXLUMO Global 2022 Patients Worldwide on Commercial Q4 Highlights Net Product Revenues OXLUMO at YE 2022 YoY % Growth QoQ % Growth ROW U.S. 54% 38% U.S. $24M ROW 12% 50% $15M Global 24% 45% $19M $13M $16M • U.S. QoQ growth of +38% primarily impacte
Slide 13: Alnylam Pipeline
Pushkal Garg, M.D. Chief Medical Officer Alnylam Pipeline 13
Slide 14: Key Study Results: Patisiran in ATTR-CM
Key Study Results sNDA Accepted; Potential to Expand Label to Include ATTR Amyloidosis Patients with CM Patisiran demonstrated statistically significant and clinically meaningful improvements in functional capacity, health status and quality of life compared to placebo at month 12 Change from Baseli
Slide 15: Safety and Exploratory Endpoints Support Therapeutic Potential of Patisiran
Safety and Exploratory Endpoints Support Therapeutic Potential of Patisiran 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 16: Vutrisiran HELIOS-B 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 17: Vutrisiran Biannual Dosing Regimen
Vutrisiran Biannual Dosing Regimen Topline Results and Next Steps • Evaluated opportunity to develop 50 mg biannual vutrisiran dosing regimen in HELIOS-A Randomized Treatment Extension • Topline results: – Established non-inferiority of 50 mg biannual vs 25 mg quarterly, based on percent TTR reducti
Slide 18: Zilebesiran: Potential Novel Treatment for Patients with Hypertension
Zilebesiran: Potential Novel Treatment for Patients with Hypertension Phase 1 Data Support Compelling 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) (n=8) (n=6) (n=8
Slide 19: 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 designed to lower APP production at its Durable Effect in NHP CSF sAPPα and sAPPß Protein Knockdown source, upstream of pathogenic process (Single Intrathecal Dose in NHP) (% remaining r
Slide 20: 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 21: Financial Summary and Upcoming Milestones
Jeff Poulton Chief Financial Officer Financial Summary and Upcoming Milestones 21
Slide 22: Q4 and Full Year 2022 Financial Summary
Q4 and Full Year 2022 Financial Summary Q4 Reported Q4 CER FY22 Reported FY22 CER Financial Results ($ millions) Q4 2022 Q4 2021 FY 2022 FY 2021 Growth % Growth %3 Growth % Growth %3 Net Product Revenues $262 $199 32% 41% $894 $662 35% 43% Net Revenues from Collaborations $71 $60 18% $135 $181 -25%
Slide 23: 2023 Full Year Guidance
2023 Full Year Guidance1 Guidance Key Assumptions • Assumes U.S. sNDA approval of patisiran Net Product Revenue: $1,200M to $1,285M for ATTR amyloidosis with CM by the ONPATTRO, AMVUTTRA, GIVLAARI, OXLUMO PDUFA date on October 8th, 2023 Net Product Revenue Growth vs. 2022 at 34% to 44% • Uses Decemb
Slide 24: Alnylam 2023 Goals
Alnylam 2023 Goals Early Mid Late Combined Net Product Revenue Guidance $1,200M – $1,285M PATISIRAN ATTR Amyloidosis FDA Approval of sNDA VUTRISIRAN ATTR Amyloidosis Biannual Dosing Regimen Data ALN-TTRsc04* ATTR Amyloidosis Phase 1 Topline Results Complete KARDIA-2 Enrollment ZILEBESIRAN* Hypertens
Slide 25: Q&A Session
Q4 and Full Year 2022 Financial Results Q&A Session 25
Slide 26: Thank You!
| || Thank You! “LAlnylam
Slide 27: Appendix
Q4 and Full Year 2022 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) Please note that the figures presented may not sum exactly due to rounding ¹ Beginning in 2022, presentations of non-GAAP financial measures will not include adjustments for upfront payment on
Slide 29: Reconciliation of Revenue and Growth at Constant Currency
Alnylam Pharmaceuticals, Inc. Reconciliation of Revenue and Growth at Constant Currency Please note that the figures presented may not sum exactly due to rounding 29 Constant Currency = CER
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