Alnylam Pharmaceuticals, Inc. · NASDAQ:ALNY
Alnylam Pharmaceuticals — Q1 2023 Earnings Presentation
29 slides from Alnylam Pharmaceuticals's earnings presentation, May 4, 2023
Slide 1: First Quarter 2023 Financial Results
Nathan (USA) Diagnosed with AHP First Quarter 2023 Financial Results May 4, 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 • Akshay Vaishnaw, M.D., Ph.D. President Finan
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: 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 5 hATTR amyloidosis
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 Update: Q1 2023
TTR Update: Q1 2023 $204M >3,160 Total TTR Total TTR patients Global Q1 2023 worldwide at end of Net Product Revenues Q1 2023 >3,160 >2,975 >2,580 Q1 Total TTR Highlights Patients on Therapy >2,400 ~640 ~1,110 >2,200 ~180 YoY % Growth QoQ % Growth ~2,400 ~2,335 U.S. 75% 5% ~2,050 ROW 28% 9% Global 4
Slide 9: Ultra Rare Update: Q1 2023
Ultra Rare Update: Q1 2023 $72M >850 Total Ultra Rare Total Ultra Rare Global Q1 2023 patients worldwide Net Product Revenues at end of Q1 2023 Q1 Ultra Rare Highlights >850 >800 YoY % Growth QoQ % Growth Patients on Therapy >715 >615 ~300 ~280 >550 ~240 GIVLAARI 36% 2% ~200 ~160 OXLUMO 66% 1% ~520
Slide 10: Alnylam Pipeline
Akshay Vaishnaw, M.D., Ph.D. President Alnylam Pipeline 10
Slide 11: 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 12: Preclinical Data Demonstrate Extensive, Potent, and Durable APP Reduction
Preclinical Data Demonstrate Extensive, Potent, and Durable APP Reduction Non-Human Primate Data of APP-targeting siRNAs Single Intrathecal Dose of APP siRNA Single Intrathecal Dose of APP siRNA Supports Distributes Throughout Spine and Brain Biannual or Less Frequent Regimen APP mRNA CSF sAPPα and
Slide 13: ALN-APP Phase 1 Overview
ALN-APP Phase 1 Overview Randomized, Double-Blind Study in Patients with Early-Onset Alzheimer’s Disease (EOAD) Part A: Single Ascending Dose Part B: Multiple Dose (Ongoing) (expected to begin 2023) • Population: Patients with Early Onset Alzheimer’s Disease • Primary Objective: Safety and tolerabil
Slide 14: ALN-APP Phase 1 Study in EOAD: Safety Summary
ALN-APP Phase 1 Study in EOAD Safety Summary from Interim Analysis of Part A Twenty patients with early-onset Alzheimer’s disease enrolled in 3 single-dose cohorts in Part A of Phase 1 study Single doses of ALN-APP well tolerated to date • All adverse events (AEs) mild or moderate in severity • Avai
Slide 15: ALN-APP Phase 1 Study in EOAD – Interim Results
ALN-APP Phase 1 Study in EOAD – Interim Results Robust and Sustained Reduction in sAPP and sAPP in CSF at the Highest Dose Tested Median % Change from Median % Change from Baseline (sAPP) Baseline (sAPPβ) Median % Change from Baseline in CSF Soluble Amyloid Beta Median % Change from Baseline in C
Slide 16: Patisiran Key Study Results; sNDA Accepted by FDA
Key Study Results sNDA Accepted by FDA; 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
Slide 17: Vutrisiran Phase 3 Study in ATTR Amyloidosis with Cardiomyopathy
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 18: ALN-TTRsc04
ALN-TTRsc04 About ALN-TTRsc04 • Potential for annual subcutaneous dosing regimen ALN-TTRsc04 with potent and reversible effects based on preclinical data An Investigational RNAi Therapeutic for Potential – Targeting >90% serum TTR reduction Treatment of ATTR Amyloidosis • No third-party royalty obli
Slide 19: Zilebesiran: Potential Novel Treatment for Patients with Hypertension
Zilebesiran: Potential Novel Treatment for Patients with Hypertension Phase 1 Data Support Development of 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) (
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: Q1 2023 Financial Summary
Q1 2023 Financial Summary Q1 Reported Q1 CER Financial Results ($ millions) Q1 2023 Q1 2022 Growth % Growth %2 Net Product Revenues $276 $187 48% 52% Net Revenues from Collaborations $36 $26 41% Royalty Revenues $7 $0 - Total Revenues $319 $213 50% 53% Cost of Goods Sold and Collaborations $55 $36 5
Slide 23: 2023 Reiterated Full Year Guidance
2023 Reiterated Full Year Guidance1 Guidance Key Assumptions Net Product Revenue: • Assumes U.S. sNDA approval of patisiran ONPATTRO, AMVUTTRA, GIVLAARI, $1,200M to $1,285M for ATTR amyloidosis with CM by the PDUFA date on October 8th, 2023 OXLUMO Net Product Revenue Growth vs. 2022 34% to 44% • Use
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
Q1 2023 Financial Results Q&A Session 25
Slide 26: Thank You!
| || Thank You! “LAlnylam
Slide 27: Appendix
Q1 2023 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 March 31, 2023 March 31, 2022 Reconciliation of GAAP to Non-GAAP research and development: GAAP research and development $ 230,569 $ 169,893 Less: Stock-based compensation ex
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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