Alnylam Pharmaceuticals, Inc. · NASDAQ:ALNY
Alnylam Pharmaceuticals — Q2 2020 Earnings Presentation
34 slides from Alnylam Pharmaceuticals's earnings presentation, Aug 6, 2020
Slide 1: Second Quarter 2020 Financial Results
Second Quarter 2020 Financial Results August 6, 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 • Barry Greene President Alnylam Clinical Pipeline • Akshay Vaishnaw, M.D., Ph.D. President of R&D Fi
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: COVID-19 Planning Framework
COVID-19 Planning Framework Anticipated Business Impacts Through 2020 PANDEMIC RECOVERY NEW NORMAL Q1 Q2 Q3 Q4 5
Slide 6: Alnylam Q2 Context
Alnylam Q2 Context Building a Top-Tier Biopharmaceutical Company $ Strong Commercial Productive Organic Secured Bridge Toward Progress Pipeline Self-Sustainability 6
Slide 7: Organizational Updates
Organizational Updates Barry Greene, President, COO Yvonne Greenstreet to Depart at End of Q3 Appointed President and COO as of Oct 1st 7
Slide 8: Marketed Products and Clinical Programs
LAlnylan. 3 STAs DEEEEL:,> 4 3 Marketed Products Sm 64 10 Clinical ProgramS =ommmm> 14 4 Late Stage Programs =m §=66
Slide 9: Commercial/Med Affairs Highlights
Barry Greene President Commercial/Med Affairs Highlights 9
Slide 10: ONPATTRO Launch Update: Q2 2020
ONPATTRO® Launch Update: Q2 2020 Strong Performance with Significant Growth $66.5M >1,050 U.S. Demand and Access ONPATTRO Global Q2 Patients Worldwide on Commercial 40% Demand from new writers1 Net Product Revenues ONPATTRO at end of Q2 2020 U.S. lives with confirmed access to 98% ONPATTRO, if presc
Slide 11: ONPATTRO Global Commercialization
ONPATTRO Global Commercialization Increasing Access and Value Recognition • Significant progress with global ONPATTRO UNITED STATES CEMEA LATIN AMERICA ASIA availability ‒ Recent launches in Spain and Italy ‒ Reimbursement achieved in France ‒ Access achieved in “big five” Western European markets,
Slide 12: Alnylam Act – hATTR Amyloidosis
Alnylam Act – hATTR Amyloidosis Third-Party Genetic Testing and Counseling Program Sponsored by Alnylam 3,083 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 13: GIVLAARI Launch Update: Q2 2020
GIVLAARI® Launch Update: Q2 2020 Strong Initial Demand in U.S. $11.0M >100 U.S. Demand and Access GIVLAARI Q2 Patients on Commercial GIVLAARI 76% New U.S. starts from new writers* Net Product Revenues at end of Q2 2020 U.S. lives with confirmed access to >75% GIVLAARI, if prescribed Patients on comm
Slide 14: GIVLAARI Global Commercialization
GIVLAARI Global Commercialization Ensuring GIVLAARI Availability Around the World UNITED STATES CEMEA LATIN AMERICA ASIA • Now approved in Brazil • Initial launch underway in Germany • Named patient sales in France and other countries • ASMR II granted by HAS in France • Working with physicians in m
Slide 15: Alnylam Act – Acute Hepatic Porphyria
Alnylam Act – Acute Hepatic Porphyria Third-Party Genetic Testing and Counseling Program Sponsored by Alnylam 578 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 independen
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 Heredtiary & 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: Givosiran Phase 3 Data Published in NEJM
Givosiran Phase 3 Data Published in The New England Journal of Medicine 19
Slide 20: Lumasiran NDA and MAA Accepted
Lumasiran NDA and MAA Accepted Robust Registrational Program to Evaluate Lumasiran Across all Ages and Full PH1 Disease Spectrum Double-blind, placebo- Single arm, open-label study Single arm, open-label study controlled trial in PH1 in PH1 patients less than 6 in PH1 patients with impaired patients
Slide 21: 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 22: 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 23: Financial Summary and Guidance
Jeff Poulton Chief Financial Officer Financial Summary and Guidance 23
Slide 24: Global ONPATTRO Performance
Global ONPATTRO Performance Revenue ($M) Highlights YoY % Growth QoQ % Growth $66.7M $66.5M ROW U.S. 14% (13%) U.S. 29.5 34.2 ROW 241% 16% $55.8M Global 74% (0%) 20.0 $46.1M • U.S. QoQ growth was negatively impacted during Q2 ‘20 by 12.5 the following: $38.2M – Reduction in patient demand due to red
Slide 25: Global GIVLAARI Performance
Global GIVLAARI Performance Revenue ($M) Highlights QoQ % Growth $11.0M U.S. 64% ROW U.S. ROW N/A 2.3 Global 109% 8.7 • >85 U.S. Start Forms1, >100 global patients on therapy since launch $5.3M • Good progress with VBAs, now have confirmed access 5.3 for over 75% of covered U.S. lives • Initial cont
Slide 26: Second Quarter 2020 Financial Summary
Second Quarter 2020 Financial Summary Financial Results ($ millions) Q2 2020 Q2 2019 Q1 2020 YoY % Change QoQ % Change ONPATTRO Net Product Revenues $66.5 $38.2 $66.7 74.0% (0.2%) GIVLAARI Net Product Revenues $11.0 - $5.3 N/A 108.5% Net Revenue from Collaborations $26.4 $6.5 $27.5 307.7% (4.0%) Tot
Slide 27: Updated 2020 Full-Year Guidance
Updated 2020 Full-Year Guidance Prior FY 2020 Guidance Updated FY 2020 Guidance2 ONPATTRO Net Product Revenues $270M - $300M $280M - $300M GIVLAARI Net Product Revenues No guidance provided Unchanged Net Revenues from Collaborations $100M - $150M Unchanged GAAP Combined R&D and SG&A Expenses $1,155M
Slide 28: 2020 Goals Update
Yvonne Greenstreet, MBChB, MBA Chief Operating Officer 2020 Goals Update 28
Slide 29: 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 30: Upcoming RNAi Roundtables
Upcoming RNAi Roundtables Lumasiran, in Development for the Treatment of Primary Hyperoxaluria Type 1 • Monday, August 10, 2:00 pm ET Patisiran and Vutrisiran, in Development for the Treatment of ATTR Amyloidosis • Date TBD Givosiran, for the Treatment of Acute Hepatic Porphyria • Monday, September
Slide 31: Q&A Session
Q2 2020 Financial Results Q&A Session 31
Slide 32: Challenge Accepted
To those who say “impossible, impractical, unrealistic,” we say: CHALLENGE ACCEPTED © 2020 Alnylam Pharmaceuticals, Inc. 32
Slide 33: Appendix
Q2 2020 Financial Results Appendix 33
Slide 34: Reconciliation of Selected GAAP Measures to Non-GAAP Measures
Alnylam Pharmaceuticals, Inc. Reconciliation of Selected GAAP Measures to Non-GAAP Measures (In thousands) 34 Please note that the figures presented may not sum exactly due to rounding
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