BOSTON--(BUSINESS WIRE)--Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced multiple program updates ahead of upcoming investor meetings in January, including the company’s scheduled webcast from the 42nd Annual J.P. Morgan Healthcare Conference on Monday, January 8, 2024 at 11:15 a.m. ET/8:15 a.m. PT. “ 2023 was marked by continued strong performance in the cystic fibrosis business and acceleration of our development stage pipeline, propelling the company into 2024 with tremendous momentum ...

Sometimes a company has a mixture of proven performance and upcoming catalysts that make the idea of buying it hand-over-fist practically irresistible. And that's exactly what Vertex Pharmaceuticals (VRTX 0.59%) is modeling right now.Thanks to its long-standing dominance in a key niche market, its newfound pursuit of powerful gene therapies, and its cash output, there's good reason to be excited about the stock. So here are three arguments for why it's worth buying now.1. Its base of recurring revenue is ro ...

Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) Q3 2023 Earnings Call Transcript November 6, 2023 4:30 PM ET Company Participants Susie Lisa - CFA, Senior Vice President of Investor Relations Reshma Kewalramani - President & Chief Executive Officer Stuart Arbuckle - Executive Vice President & Chief Operating Officer Charlie Wagner - Executive Vice President & Chief Financial Officer Conference Call Participants Geoff Meacham - Bank of America Robyn Karnauskas - Truist Securities Salveen Richter - Goldman ...

THIRD QUARTER 2023 FINANCIAL RESULTS ©2023 Vertex Pharmaceuticals Incorporated VERTEX On track to complete Ph3 bunionectomy and single arm safety and effectiveness trial by end of 2023 • Granted Fast Track and Breakthrough Therapy designations ©2023 Vertex Pharmaceuticals Incorporated Each stage can take several months | --- | --- | --- | |-------------------------------------------------------------------------------------|------------------------------------------------------------------------------------ ...

Financial Performance - In Q3 2023, the company's net product revenues increased to $2.5 billion, up from $2.3 billion in Q3 2022, primarily driven by the performance of TRIKAFTA in the U.S. and strong uptake in ex-U.S. markets[109] - Product revenues for Q3 2023 reached $2,483.5 million, a 6% increase from $2,334.3 million in Q3 2022, driven primarily by strong uptake of TRIKAFTA/KAFTRIO[142] - Net income for the nine months ended September 30, 2023, was $2,650.8 million, reflecting a 6% increase compared to $2,503.1 million for the same period in 2022[142] - U.S. product revenues for Q3 2023 were $1,554.2 million, a 7% increase from $1,455.6 million in Q3 2022[144] - Ex-U.S. product revenues for Q3 2023 were $929.3 million, a 6% increase from $878.7 million in Q3 2022[144] Research and Development - The total R&D, AIPR&D, and SG&A expenses rose to $1.1 billion in Q3 2023, compared to $920.8 million in Q3 2022, due to the progression of several product candidates in clinical development[109] - Research and development expenses for Q3 2023 totaled $810.0 million, a 26% increase from $645.0 million in Q3 2022[147] - Total research and development expenses incurred since January 2021 amount to approximately $8.6 billion, reflecting significant investment in product discovery and development[148] - The company continues to invest in research and development to balance risks and enhance its pipeline for future years[126] - The company expects to complete pivotal clinical trials for vanzacaftor/tezacaftor/deutivacaftor by the end of 2023 and share results in early 2024[119] - The company is advancing VX-548 for acute pain, with expectations to complete the pivotal program by late 2023 and share results in early 2024[119] - Inaxaplin for APOL1-mediated kidney disease has received Breakthrough Therapy designation from the FDA and is currently in a pivotal Phase 2/3 clinical trial[124] - The company is developing VX-880 as a potential treatment for type 1 diabetes, with proof-of-concept achieved in 2022 and enrollment completed in Part C of the clinical trial[124] - The company is pursuing multiple NaV1.8 and NaV1.7 inhibitors for pain relief, with ongoing clinical trials expected to yield results in late 2023[119] Cash and Liquidity - As of September 30, 2023, total cash, cash equivalents, and marketable securities increased to $13.6 billion from $10.9 billion as of December 31, 2022, mainly due to net product revenues and operating cash flows[109] - Cash, cash equivalents, and marketable securities totaled $13.6 billion as of September 30, 2023, a 25% increase from $10.9 billion as of December 31, 2022[163] - Total working capital was $11.1 billion as of September 30, 2023, reflecting an increase of $603.7 million from $10.5 billion as of December 31, 2022[164] - Net cash provided by operating activities was $3.3 billion for the nine months ended September 30, 2023, compared to $3.1 billion for the same period in 2022[166] - Cash used in investing activities was $2.3 billion for the nine months ended September 30, 2023, compared to $496.7 million for the same period in 2022[167] - Cash used in financing activities was $442.4 million for the nine months ended September 30, 2023, compared to $107.9 million for the same period in 2022[169] - The company expects cash flows from product sales, along with current cash and marketable securities, to be sufficient to fund operations for at least the next twelve months[171] - The company has a revolving credit facility allowing borrowing up to $500 million, with the potential to increase this capacity to $1 billion, which was undrawn as of September 30, 2023[172] Future Obligations and Capital Management - Significant future capital requirements include operating expenses for research and development, manufacturing, commercialization, and obligations related to leases and royalties[174] - The company has $2.7 billion remaining authorization under its Share Repurchase Program as of September 30, 2023[175] - The company may raise additional capital through various financing methods, including public offerings and collaborative agreements[173] - Future obligations may include milestone and royalty payments contingent on the success of development and regulatory targets[174] - The company is committed to managing its capital structure and will consider all financing opportunities to strengthen its long-term liquidity profile[173] - The adequacy of available funds to meet future operating and capital requirements will depend on various factors, including future product sales and business development activities[171] Regulatory Updates - The FDA has assigned action dates of December 8, 2023, for SCD and March 30, 2024, for TDT regarding the regulatory submissions for exa-cel, which are currently under review[108] - The company made an upfront payment of $225.1 million to Entrada as part of a strategic collaboration and licensing agreement in February 2023[135] - In March 2023, the company entered into a license agreement with CRISPR, making a $100.0 million upfront payment and a subsequent $70.0 million payment for a research milestone[136] Operating Costs - Total operating costs and expenses for Q3 2023 were $1,445.4 million, a 20% increase from $1,207.6 million in Q3 2022[145] - Selling, general and administrative expenses increased by 7% to $263.8 million for the three months ended September 30, 2023, and by 13% to $767.5 million for the nine months ended September 30, 2023[155] - Acquired in-process research and development expenses for the nine months ended September 30, 2023, were $509.3 million, a significant increase of 448% from $92.9 million in the same period of 2022[137] - Acquired in-process research and development expenses rose by 78% to $51.7 million for the three months ended September 30, 2023, and by 448% to $509.3 million for the nine months ended September 30, 2023[154]

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 ...