Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) Morgan Stanley 21st Annual Global Healthcare Conference 2023 September 13, 2023 11:30 AM ET Company Participants Charlie Wagner - EVP and CFO Reshma Kewalramani - President and CEO Conference Call Participants Terence Flynn - Morgan Stanley Terence Flynn Well, thanks, everybody, for joining us. I'm Terence Flynn from the U.S. biopharma analyst here at Morgan Stanley and we're very pleased to be hosting Vertex for this session. Today from the company, we have ...

SECOND QUARTER 2023 FINANCIAL RESULTS AUGUST 1, 2023 ©2023 Vertex Pharmaceuticals Incorporated VERTEX 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, Executive Vice President and Chief Financial Officer ©2023 Vertex Pharmaceuticals Incorporated 2 SAFE HARBOR ...

Financial Data and Key Metrics Changes - Vertex Pharmaceuticals reported a 14% year-over-year revenue increase in Q2 2023, reaching $2.49 billion, driven by a 26% increase in revenue outside the U.S. and a 7% increase in the U.S. [45][46] - The company raised its full-year 2023 CF product revenue guidance to a range of $9.7 billion to $9.8 billion, an increase of $100 million to $150 million from the previous range [6][49] - Non-GAAP operating income for Q2 2023 was $1.15 billion, down from $1.19 billion in Q2 2022, while non-GAAP earnings per share grew by 8% to $3.89 [48] Business Line Data and Key Metrics Changes - The CF business saw a 14% global revenue growth, attributed to expanded patient reach and increased uptake in younger age groups [6][34] - The company is preparing for multiple near-term launches, including exa-cel for sickle cell disease and beta-thalassemia, VX-548 for acute pain, and the vanzacaftor triple combination therapy for CF [7][32] Market Data and Key Metrics Changes - The FDA has accepted Vertex's filings for exa-cel, with a PDUFA date of December 8, 2023, for sickle cell disease and March 30, 2024, for beta-thalassemia [15][18] - In the U.S., approximately 65% of patients with sickle cell disease or beta-thalassemia are covered by government programs, with ongoing discussions for access and reimbursement [39][41] Company Strategy and Development Direction - Vertex is focused on expanding its CFTR portfolio and investing in future commercial excellence, with a goal of launching five new products in five years [7][8] - The company is also advancing programs in eight disease areas, including type 1 diabetes and APOL1-mediated kidney disease, reflecting a commitment to innovation and addressing unmet medical needs [9][28] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the transformative potential of exa-cel and the upcoming launches, highlighting the significant market opportunities [44] - The company anticipates continued growth in its CF portfolio driven by approvals and reimbursement, particularly in younger patient populations [36][34] Other Important Information - Vertex's R&D expenses increased to $1.04 billion in Q2 2023, reflecting investments in clinical studies and pre-commercial activities for exa-cel and other anticipated launches [46][47] - The company ended Q2 2023 with $12.6 billion in cash and investments, indicating strong financial health [48] Q&A Session Summary Question: Discussion on exa-cel advisory committee - Management confirmed that an advisory committee will be held for exa-cel, with details to be provided closer to the date [55] Question: Strategies for increasing patient adoption of exa-cel post-approval - Management emphasized the importance of securing access and reimbursement as critical steps for a successful launch, acknowledging the multi-month process for patient treatment [57][59] Question: Next data readout for type 1 diabetes programs - Management indicated that data for the VX-880 program will be presented at the fall diabetes conference, while enrollment for the VX-264 program has just begun [63] Question: Commercial path for pain management - Management outlined plans to focus on institutions and ambulatory surgical centers for the launch of VX-548, given the concentration of prescriptions in these settings [65] Question: Expectations for improved conditioning regimen for exa-cel - Management discussed ongoing efforts to optimize the conditioning regimen, aiming for a gentler approach that minimizes side effects [71] Question: Efficacy results for AATD candidates - Management expects to share results from the VX-864 and VX-634 trials in 2024, focusing on both liver and lung manifestations of the disease [78]

Table of Contents UNITED STATES SECURITIES AND EXCHANGE COMMISSION WASHINGTON, D.C. 20549 ________________________________________________________ FORM 10-Q ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 FOR THE QUARTERLY PERIOD ENDED JUNE 30, 2023 or Registrant's telephone number, including area code (617) 341-6100 | ____________________________________________ | | | | --- | --- | --- | | Securities registered pursuant to Section 12(b) of the Act: | | | | Title of ...

Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) Goldman Sachs 44th Annual Global Healthcare Conference Call June 13, 2023 5:40 PM ET Company Participants Reshma Kewalramani - President and Chief Executive Officer Stuart Arbuckle - Chief Operating Officer Conference Call Participants Salveen Richter - Goldman Sachs Salveen Richter Great. Good afternoon, everyone. Thanks for joining us. I am Salveen Richter, I cover the biotechnology sector at Goldman Sachs. And we are really pleased to have Vertex team wit ...

Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) BofA Securities 2023 Health Care Conference May 9, 2023 12:20 PM ET Company Participants Charlie Wagner - EVP and CFO David Altshuler - CSO and EVP, Global Research Conference Call Participants Geoff Meacham - Bank of America Geoff Meacham Welcome to the first morning of the BofA Healthcare Conference. I'm Geoff Meacham. I'm the senior biopharma analyst here and we're thrilled to have Vertex Pharmaceuticals in the big room, hopefully no protesting this year. ...

Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) Q1 2023 Earnings Conference Call May 1, 2023 4:30 PM ET Company Participants Susie Lisa - CFA, Senior Vice President, Investor Relations Reshma Kewalramani - President and Chief Executive Officer Stuart Arbuckle - Executive Vice President and Chief Operating Officer Charlie Wagner - Executive Vice President and Chief Financial Officer Conference Call Participants Salveen Richter - Goldman Sachs David Risinger - SV8 Securities Mohit Bansal - Wells Fargo Geoff ...

FIRST QUARTER 2023 FINANCIAL RESULTS ©2023 Vertex Pharmaceuticals Incorporated VERTEX MAY 1, 2023 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, Executive Vice President and Chief Financial Officer ©2023 Vertex Pharmaceuticals Incorporated 2 SAFE HARBOR STAT ...

Table of Contents UNITED STATES SECURITIES AND EXCHANGE COMMISSION WASHINGTON, D.C. 20549 ________________________________________________________ FORM 10-Q ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 FOR THE QUARTERLY PERIOD ENDED MARCH 31, 2023 or ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 FOR THE TRANSITION PERIOD FROM TO Commission file number 000-19319 ____________________________________________ Vertex Pharma ...

UNITED STATES SECURITIES AND EXCHANGE COMMISSION WASHINGTON, D.C. 20549 FORM 10-K ☒ ANNUAL REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the Fiscal Year Ended December 31, 2022 or ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 FOR THE TRANSITION PERIOD FROM TO Commission file number 000-19319 Vertex Pharmaceuticals Incorporated Registrant's telephone number, including area code (617) 341-6100 Securities registered pursuant to Se ...