Vertex Pharmaceuticals Incorporated · NASDAQ:VRTX
Vertex Pharmaceuticals — Q3 2022 Earnings Presentation
18 slides from Vertex Pharmaceuticals's earnings presentation, Oct 27, 2022
Slide 1: Third Quarter 2022 Financial Results
THIRD QUARTER 2022 FINANCIAL RESULTS OCTOBER 27, 2022 1 ©2022 Vertex Pharmaceuticals Incorporated
Slide 2: Agenda
AGENDA Introduction Susie Lisa, CFA, Senior Vice President, Investor Relations CEO Perspective and Pipeline Update Reshma Kewalramani, M.D., Chief Executive Officer and President Commercial Update Stuart Arbuckle, Executive Vice President and Chief Operating Officer Financial Results Charlie Wagner,
Slide 3: Safe Harbor Statement & Non-GAAP Financial Measures
SAFE HARBOR STATEMENT & NON-GAAP FINANCIAL MEASURES This presentation contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, the information provided regarding future financial performance and operations, fis
Slide 4: Strategic Inflection Point for Vertex
STRATEGIC INFLECTION POINT FOR VERTEX Unique and differentiated R&D strategy delivers success Expanding CF leadership Accelerating broad and deep Preparing for near-term Strong financial profile • Treating more patients with our R&D pipeline commercial opportunities • On track for 8th consecutive ye
Slide 5: Vertex Is Advancing a Broad and Deep Clinical Pipeline Across Multiple Modalities
VERTEX IS ADVANCING A BROAD AND DEEP CLINICAL PIPELINE ACROSS MULTIPLE MODALITIES RESEARCH PHASE 1 PHASE 2 PHASE 3 APPROVED KALYDECO ORKAMBI SYMDEKO/SYMKEVI TRIKAFTA/KAFTRIO Cystic Fibrosis vanzacaftor/tezacaftor/deutivacaftor Additional Small Molecules mRNA Therapeutics CRISPR/Cas9 Exa-cel (CTX001,
Slide 6: Vanzacaftor/Tezacaftor/Deutivacaftor Regimen May Further Enhance Patient Benefit in CF
VANZACAFTOR/TEZACAFTOR/DEUTIVACAFTOR REGIMEN MAY FURTHER ENHANCE PATIENT BENEFIT IN CYSTIC FIBROSIS Phase 3 enrollment now projected to complete before year end 2022 Two Phase 3 global, randomized, double-blind, active- Next-in-Class, Once-Daily, Oral Triple controlled trials underway (N=950 total):
Slide 7: Exa-cel: Compelling Efficacy in Patients with SCD and TDT
EXA-CEL: COMPELLING EFFICACY IN PATIENTS WITH SCD AND TDT; GLOBAL REGULATORY SUBMISSIONS TO BEGIN BY YEAR END 2022 Initiate U.S. BLA submission for rolling review in Nov 2022; targeted completion Q1:23; EU & UK submissions targeted YE 2022 31 of 31 SCD patients were VOC-free Pre-Study RBC 42 of 44 T
Slide 8: VX-548: Initiated Pivotal Development for Acute Pain in Q3
VX-548: INITIATED PIVOTAL DEVELOPMENT FOR ACUTE PAIN IN Q3 Near-term significant commercial opportunity targeting moderate to severe acute pain in the U.S. Two Randomized, Double-Blind, Placebo Controlled Trials n=1,000 Novel, Selective NaV1.8 Inhibitor for VX-548 Pain Bunionectomy Safety Placebo Su
Slide 9: Inaxaplin Is in Pivotal Development for AMKD
INAXAPLIN IS IN PIVOTAL DEVELOPMENT; POTENTIAL FIRST MEDICINE TO TREAT THE UNDERLYING CAUSE OF APOL1-MEDIATED KIDNEY DISEASE (AMKD) Program targets broad AMKD label; interim analysis at 48 weeks may provide path to accelerated U.S. approval Phase 2 (n=66) Phase 3 (n=400) Enrolling patients Small Mol
Slide 10: Advancing Potentially Curative Treatments for Type 1 Diabetes
ADVANCING POTENTIALLY CURATIVE TREATMENTS ~2.5 MILLION PEOPLE LIVING WITH TYPE 1 DIABETES IN U.S. AND E.U. ALONE VX-880 Cells + device Hypoimmune cells Stem cell-derived islets Stem cell-derived islets Stem cell-derived islets with standard immunosuppression with encapsulation with hypoimmune gene e
Slide 11: Continued Growth of Our CFTR Modulators Around the World
CONTINUED GROWTH OF OUR CFTR MODULATORS AROUND THE WORLD Global filings for TRIKAFTA in patients 2-5 years of age expected by year end 2022 UNITED STATES EUROPE and OTHER MARKETS • Steady performance with high persistence and compliance rates • Rapid uptake of KAFTRIO/TRIKAFTA in markets with recent
Slide 12: Significant Progress with Exa-cel Launch Preparation Activities
SIGNIFICANT PROGRESS WITH EXA-CEL LAUNCH PREPARATION ACTIVITIES ~24 States with ~90% of SCD/TDT Patients 4 Countries in Europe with ~75% of SCD/TDT Patients Commercial Readiness Efforts • Initial launch will focus on ~32,000 patients with severe forms of SCD and TDT in the U.S. and Europe/UK • U.S.:
Slide 13: Acute Pain: Large Market, Concentrated Prescribing, Unmet Needs
ACUTE PAIN: LARGE MARKET, CONCENTRATED PRESCRIBING, UNMET NEEDS CREATES SIGNIFICANT OPPORTUNITY FOR VX-548 ~35M In-Patient Visits Hospital Discharge Rx. Prescriptions 185M Visits ~150M Out-Patient & Emergency Office Visits to Hospitals Annually Department Visits ~500K Treatment >1B Treatment Days Dr
Slide 14: Q3 2022 Financial Highlights
Q3 2022 FINANCIAL HIGHLIGHTS ($ in millions except where noted or per share data and percentages) Q3 21 FY 21 Q3 22 Total CF product revenues $1.98B $7.57B $2.33B TRIKAFTA/KAFTRIO $1.56B $5.70B $2.01B SYMDEKO/SYMKEVI 81 420 38 ORKAMBI 185 772 146 KALYDECO 162 684 139 Combined non-GAAP R&D, acquired
Slide 15: Raising Full Year 2022 Revenue Guidance to $8.8 to 8.9 Billion
RAISING FULL YEAR 2022 REVENUE GUIDANCE TO $8.8 TO 8.9 BILLION, REPRESENTING 17% GROWTH AT THE MIDPOINT Current Previous FY 2022 Guidance FY 2022 Guidance FY 2022 Commentary Strong uptake of KAFTRIO/ TRIKAFTA in markets with Total CF Product Revenues $8.8 - $8.9B $8.6 - $8.8B recent reimbursement ag
Slide 16: Recent Advances Position Vertex for Multiple Milestones in the Months Ahead
RECENT ADVANCES POSITION VERTEX FOR MULTIPLE MILESTONES IN THE MONTHS AHEAD Recent Highlights Anticipated Key Milestones Completed Phase 3 study of TRIKAFTA in patients with CF ages 2 to 5 Submit global regulatory filings by YE 2022 More than 250 sites active in vanzacaftor/tezacaftor/deutivacaftor
Slide 17: Third Quarter 2022 Financial Results
THIRD QUARTER 2022 FINANCIAL RESULTS OCTOBER 27, 2022 17 ©2022 Vertex Pharmaceuticals Incorporated
Slide 18: Appendix: GAAP to Non-GAAP Financial Information
APPENDIX GAAP TO NON-GAAP FINANCIAL INFORMATION ($ in millions except as noted, per share data and percentages) Q3 21 FY 21 Q3 22 Combined R&D, Acquired IPR&D and SG&A GAAP 692 3.89B 921 Non-GAAP 588 3.44B 758 Operating income GAAP 1.05B 2.78B 1.13B Non-GAAP 1.16B 3.23B 1.29B Operating Margin %: GAA
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