Alnylam Pharmaceuticals, Inc. · NASDAQ:ALNY
Alnylam Pharmaceuticals — Q2 2023 Earnings Presentation
26 slides from Alnylam Pharmaceuticals's earnings presentation, Aug 3, 2023
Slide 1: Second Quarter 2023 Financial Results
Nathan (USA) Diagnosed with AHP Second Quarter 2023 Financial Results August 3, 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: 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 6: Commercial Highlights
Tolga Tanguler Chief Commercial Officer Commercial Highlights 6
Slide 7: TTR Franchise Update: Q2 2023
TTR Franchise Update: Q2 2023 $224M >3,490 Total TTR Total TTR patients Global Q2 2023 worldwide at end of Net Product Revenues Q2 2023 >3,490 Q2 TTR Franchise Highlights >3,160 >2,975 Patients on Therapy >2,400 >2,580 ~640 ~1,110 ~1,670 YoY % Growth QoQ % Growth ~180 U.S. 72% 12% ROW 23% 6% ~2,400
Slide 8: Strong U.S. TTR Commercial Execution
Strong U.S. TTR Commercial Execution 12 Months Post AMVUTTRA Launch U.S. Start Forms | Doubled “New Start U.S. Prescribers | Increasing share of Accelerating Growth for U.S. TTR-PN Form” Monthly Average Since Launch prescribers new to TTR, Balanced Franchise Growth Across Account Types New TTR Start
Slide 9: Ultra Rare Franchise Update: Q2 2023
Ultra Rare Franchise Update: Q2 2023 $82M >920 Total Ultra Rare Total Ultra Rare Global Q2 2023 patients worldwide Net Product Revenues at end of Q2 2023 >850 >920 Q2 Ultra Rare Franchise Highlights >800 Patients on Therapy >715 ~350 >615 ~300 YoY % Growth QoQ % Growth ~280 ~240 ~200 GIVLAARI 28% 21
Slide 10: Alnylam Pipeline
Pushkal Garg, M.D. Chief Medical Officer Alnylam Pipeline 10
Slide 11: TTR Franchise Phase 3 Program
TTR Franchise Phase 3 Program Randomized, Double-Blind, Placebo-Controlled Studies in ATTR Amyloidosis Patients with Cardiomyopathy patisiran vutrisiran N = 360 N = 655 hereditary & wild-type hereditary & wild-type 6-minute walk test mortality & cardiovascular events 12 months 30 months Results pres
Slide 12: 18-Month Results
18-Month Results Changes from Baseline in Functional Capacity (6-MWT) and Health Status and QOL (KCCQ-OS) • The majority of AEs were mild or moderate in severity • The most common related AE was infusion-related reactions (14.1% of patients) • The safety profile of patisiran was consistent with prev
Slide 13: 18-Month Results
18-Month Results Cardiac Biomarkers (NT-proBNP, Troponin I) and Composite Endpoints aVisits with complete data collection are presented. Baseline is defined as the last non-missing value available on or before the date of first dose of study drug in the DB period. All patients received patisiran aft
Slide 14: 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 15: 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 16: Zilebesiran Updates
Zilebesiran Updates Clinical Progress and Significant Collaboration Agreement Monotherapy Phase 2 Study (N = 394) • Enrollment completed December 2022; topline results expected mid-2023; full results to be presented at future medical conference Combination Phase 2 Study (N = 672) • Enrollment comple
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: Q2 2023 Financial Summary
Q2 2023 Financial Summary Q2 Reported Q2 CER Financial Results ($ millions) Q2 2023 Q2 2022 Growth % Growth %2 Net Product Revenues $306 $214 43% 44% Net Revenues from Collaborations $6 $9 Royalty Revenues $7 $2 Total Revenues $319 $225 42% 43% Product Cost of Goods Sold $75 $34 Cost of Collaboratio
Slide 20: 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 21: 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 22: Q&A Session
Q2 2023 Financial Results Q&A Session 22
Slide 23: Thank You!
| || Thank You! “LAlnylam
Slide 24: Appendix
Q2 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 June 30, June 30, 2023 2022 Reconciliation of GAAP to Non-GAAP research and development: GAAP research and development $ 248,526 $ 205,712 Less: Stock-based compensation expe
Slide 26: 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 26 Constant Currency = CER
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