Alnylam Pharmaceuticals, Inc. · NASDAQ:ALNY
Alnylam Pharmaceuticals — Q3 2020 Earnings Presentation
36 slides from Alnylam Pharmaceuticals's earnings presentation, Nov 5, 2020
Slide 1: Third Quarter 2020 Financial Results
Third Quarter 2020 Financial Results November 5, 2020 © 2020 Alnylam Pharmaceuticals, Inc. 1
Slide 2: Agenda
Agenda Welcome • Christine Lindenboom Senior Vice President, Investor Relations & Corporate Communications Overview • John Maraganore, Ph.D. Chief Executive Officer Commercial/Med Affairs Highlights • Andy Orth Senior Vice President, Head of U.S. Business Alnylam Clinical Pipeline • Akshay Vaishnaw,
Slide 3: Forward Looking Statements & Non-GAAP Financial Measures
Alnylam Forward Looking Statements & Non-GAAP Financial Measures 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. There are a number of important factors that could cause act
Slide 4: Overview
John Maraganore, Ph.D. Chief Executive Officer Overview 4
Slide 5: Alnylam Q3 Context
Alnylam Q3 Context Building a Top-Tier Biopharmaceutical Company $ Strong Commercial Productive Organic Moving Toward Progress Pipeline Self-Sustainability 5
Slide 6: Numbers represent expectations as of 11/5/20
* 4 4 12 6 * Numbers represent 6 expectations as of 11/5/20
Slide 7: Alnylam R&D Day
SAVE THE DATE Alnylam R&D Day December 15-16, 2020 Virtual Event 7
Slide 8: Commercial/Med Affairs Highlights
Andy Orth Senior Vice President, Head of U.S. Business Commercial/Med Affairs Highlights 8
Slide 9: ONPATTRO Launch Update: Q3 2020
ONPATTRO® Launch Update: Q3 2020 Strong Performance with Significant Growth $82.5M >1,150 Q3 U.S. Highlights Notable demand increase and strong ONPATTRO Global Q3 Patients Worldwide on Commercial compliance Net Product Revenues ONPATTRO at end of Q3 2020 Cardiologists make up more than half of new p
Slide 10: ONPATTRO Global Commercialization
ONPATTRO Global Commercialization Increasing Access and Value Recognition • Significant progress with global ONPATTRO UNITED STATES CEMEA LATIN AMERICA ASIA availability ‒ Recent launch in Portugal ‒ Conclusion of price negotiations in France ‒ Completion of initial access agreement in Canada ‒ Achi
Slide 11: Alnylam Act – hATTR Amyloidosis
Alnylam Act – hATTR Amyloidosis Third-Party Genetic Testing and Counseling Program Sponsored by Alnylam 3,364 Reduce barriers to genetic testing and counseling to help people make participating more informed decisions about their health HCP accounts Tests and services are performed by independent th
Slide 12: ONPATTRO Wins 2020 Prix Galien USA Award
PRIX GALIEN ONPATTRO® Wins 2020 Prix Galien USA Award Best Biotechnology Product 12
Slide 13: GIVLAARI Launch Update: Q3 2020
GIVLAARI® Launch Update: Q3 2020 Strong Initial Performance $16.7M >150 U.S. Demand and Access GIVLAARI Q3 Patients on Commercial GIVLAARI 10 Value-Based Agreements (VBAs) finalized Net Product Revenues at end of Q3 2020 U.S. lives with confirmed access to >90% GIVLAARI, if prescribed Patients world
Slide 14: GIVLAARI Global Commercialization
GIVLAARI Global Commercialization Ensuring GIVLAARI Availability Around the World UNITED STATES CEMEA LATIN AMERICA ASIA • Successful ongoing launch in Germany • Named patient sales in France and other countries • Strong start for market access in CEMEA • ASMR II granted by HAS in France • “Consider
Slide 15: Third RNAi therapeutic receives positive CHMP opinion
The third RNAi therapeutic has received POSITIVE CHMP OPINION* 15 * If approved by the European Commission, lumasiran will be marketed in Europe under the brand name OXLUMO
Slide 16: Alnylam Clinical Pipeline
Akshay Vaishnaw, M.D., Ph.D. President of R&D Alnylam Clinical Pipeline 16
Slide 17: Patisiran Phase 3 Study
Patisiran Phase 3 Study Randomized, Double-Blind, Placebo-Controlled Study in ATTR Amyloidosis Patients with Cardiomyopathy N ~ 300 Primary Endpoint Patient Population Patisiran • Change in 6-MWT at 12 months IV q3w† 1:1 RANDOMIZATION • ATTR amyloidosis; wild-type or 0.3 mg/kg any TTR mutation Key S
Slide 18: Vutrisiran Phase 3 Program
Vutrisiran Phase 3 Program Robust Registrational Program to Evaluate Vutrisiran in Hereditary & Wild-Type ATTR Amyloidosis Randomized, double-blind, placebo- Randomized, open-label study in controlled outcomes study in hereditary hereditary ATTR amyloidosis patients and wild-type ATTR amyloidosis pa
Slide 19: Opportunity for q6M Vutrisiran Dosing Regimen
Opportunity for q6M Vutrisiran Dosing Regimen Modeling Supports Potential Biannual 50mg Dosing Regimen in Addition to Quarterly 25mg Dosing Regimen Phase 1 Study – Healthy Volunteers Pharmacodynamic Modeling • Mean max TTR reduction of >80% after single dose of • After repeat dosing, ~90% peak TTR r
Slide 20: Givosiran Phase 1/2 OLE Study
Givosiran Phase 1/2 OLE Study Maintenance and Potential Enhancement of Clinical Activity with Continuous Monthly Dosing Annualized Attack Rate1 30 25 Givosiran-treated2 Placebo cross-over3 Mean (SEM) AAR Mean (SEM) AAR 25 20 20 15 20.2 16.2 15 16.7 10 −95% −98% −97% 10 5 7.4 5 0.5 0.7 0 0 Run-in Tre
Slide 21: Lumasiran Phase 3 Study (Interim Results)
Lumasiran Phase 3 Study (Interim Results) Efficacy Results and Safety Profile in Pediatric Patients Similar to Those Observed in ILLUMINATE-A Rapid and sustained reduction in spot urinary Primary Endpoint Result oxalate:creatinine ratio across all weight groups 0 -71% Percent Change from Baseline in
Slide 22: Renal Stone Events
Renal Stone Events1 Lower Renal Stone Event Rates Were Seen After 6–12 Months of Treatment2 Lumasiran/Lumasiran Placebo/Lumasiran • In the Lumasiran/Lumasiran group, renal 6.0 6.0 Historical3 stone event rates decreased with Placebo lumasiran treatment through Month 12 5.0 5.0 Lumasiran Renal Stone
Slide 23: Exploratory Endpoints: Renal Stones and Nephrocalcinosis
Exploratory Endpoints: Renal Stones and Nephrocalcinosis No Change in Renal Stone Event Rates after Lumasiran Treatment; Improved Nephrocalcinosis in 8/18 Patients Renal stone events Change from Baseline to Month 6 in Nephrocalcinosis 12 Reported History Lumasiran Treated (N=18) (12 Months Prior Per
Slide 24: Late Stage Partnered Program Opportunities
Late Stage Partnered Program Opportunities INCLISIRAN FITUSIRAN Hypercholesterolemia Hemophilia A or B, with and without inhibitors Adults WW with high LDL-C; ASCVD Patients WW with hemophilia A or B, with 40% leading cause of death WW ~200K and without inhibitors Patients in key markets with ASCVD
Slide 25: Early Stage Clinical Development and 2020 IND Pipeline
Alnylam Early Stage Clinical Development and 2020 IND Pipeline Focused in 4 Strategic Therapeutic Areas (STArs): Genetic Medicines Cardio-Metabolic Diseases HUMAN BREAKTHROUGH 2020 IND EARLY STAGE COMMERCIAL POC1 DESIGNATION CANDIDATES (Phase 1-Phase 2) RIGHTS Infectious Diseases CNS/Ocular Diseases
Slide 26: Financial Summary and Guidance
Jeff Poulton Chief Financial Officer Financial Summary and Guidance 26
Slide 27: Global ONPATTRO Performance
Global ONPATTRO Performance Revenue ($M) Highlights YoY % Growth QoQ % Growth ROW U.S. 16% 21% $82.5M U.S. ROW 249% 27% 43.5 Global 79% 24% $66.7M $66.5M 29.5 34.2 • U.S. QoQ growth was favorably impacted by the following: $55.8M – Increase in patient demand due to addition of new patients on therap
Slide 28: Global GIVLAARI Performance
Global GIVLAARI Performance Revenue ($M) Highlights QoQ % Growth ROW $16.7M U.S. 40% U.S. 4.6 ROW 96% Global 52% $11.0M 12.1 • >150 global patients on therapy since launch 2.3 • Continued progress with VBAs in U.S. with 10 signed and confirmed access for over 90% of covered lives 8.7 • Continued pro
Slide 29: Third Quarter 2020 Financial Summary
Third Quarter 2020 Financial Summary Financial Results ($ millions) Q3 2020 Q3 2019 Q2 2020 YoY % Change QoQ % Change ONPATTRO Net Product Revenues $82.5 $46.1 $66.5 79.1% 24.0% GIVLAARI Net Product Revenues $16.7 - $11.0 N/A 51.8% Net Revenues from Collaborations $26.6 $24.0 $26.4 11.1% 0.8% Total
Slide 30: Updated 2020 Full-Year Guidance
Updated 2020 Full-Year Guidance Prior FY 2020 Guidance Updated FY 2020 Guidance2 ONPATTRO Net Product Revenues $280M - $300M $295M - $310M GIVLAARI Net Product Revenues No guidance provided Unchanged Net Revenues from Collaborations $100M - $150M Unchanged GAAP Combined R&D and SG&A Expenses $1,130M
Slide 31: 2020 Goals Update
Yvonne Greenstreet, MBChB, MBA President and Chief Operating Officer 2020 Goals Update 31
Slide 32: Alnylam 2020 Goals
Alnylam 2020 Goals 2020* *Early is Q1-Q2, Mid is Q2-Q3, and Late is Q3-Q4 Early Mid Late Global Commercial Execution Brazil Approval Additional Country Launches (ATTR Amyloidosis) APOLLO-B Enrollment EMA Approval Global Commercial Execution Additional ENVISION Results (Acute Hepatic Porphyria) Addit
Slide 33: Q&A Session
Q3 2020 Financial Results Q&A Session 33
Slide 34: Challenge Accepted
To those who say “impossible, impractical, unrealistic,” we say: CHALLENGE ACCEPTED © 2020 Alnylam Pharmaceuticals, Inc. 34
Slide 35: Appendix
Q3 2020 Financial Results Appendix 35
Slide 36: Reconciliation of Selected GAAP Measures to Non-GAAP Measures
Alnylam Pharmaceuticals, Inc. Reconciliation of Selected GAAP Measures to Non-GAAP Measures (In thousands, except per share amounts) 36 Please note that the figures presented may not sum exactly due to rounding
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