Kidney Program Updates at the American Society of Nephrology Kidney Week 2025 November 8, 2025 ©2025 Vertex Pharmaceuticals Incorporated ©2025 Vertex Pharmaceuticals Incorporated Presentation intended for the investment community Agenda Welcome Susie Lisa, CFA, Senior Vice President, Investor Relations, Vertex Pharmaceuticals Kidney portfolio Reshma Kewalramani, M.D., President and Chief Executive Officer, Vertex Pharmaceuticals RUBY-3 late breaking data recap James A. Tumlin, M.D., Professor of Medicine in ...

Third Quarter 2025 Financial Results and Recent Corporate Updates November 2025 Forward-Looking Statements This presentation contains forward-looking statements, including statements relating to our strategy and plans; potential of and expectations for our business, commercial products, and pipeline programs; our goals, objectives, and priorities and our expectations under our growth strategy (including our expectations regarding our commercial products and launches, clinical stage products, revenue growth ...

Financial Performance - Q3 2025 total revenues reached $3.08 billion, representing an 11% year-over-year growth[5,45] - TRIKAFTA/KAFTRIO contributed $2.65 billion in Q3 2025[45] - ALYFTREK generated $247 million in revenue during Q3 2025[45] - CASGEVY revenues were $17 million in Q3 2025 and are expected to exceed $100 million for the full year 2025[45,32] - JOURNAVX revenues were $20 million in Q3 2025[45] - Non-GAAP operating income was $1.38 billion with a 45% margin in Q3 2025[45] - Non-GAAP net income was $1.24 billion, or $4.80 per diluted share in Q3 2025[45] - The company refined its 2025 revenue guidance to $11.9-12.0 billion and non-GAAP OpEx to $5.0-5.1 billion, while lowering the tax rate guidance to 17%-18%[8] Pipeline and Approvals - ALYFTREK is now approved in the U S, U K, European Union, Canada, New Zealand, and Switzerland for patients aged 6 and older[9,26] - TRIKAFTA pivotal study in 12 to <24-month-old children completed, showing a mean sweat chloride reduction of more than 70 mmol/L from baseline through week 24[8]

Group 1 - UBS sets a target price of HKD 33.5 for Zai Lab (09688) based on discounted cash flow (DCF) analysis, indicating 1x and 1.6x unadjusted/adjusted peak sales multiples [1] - UBS believes the market is overlooking Zai Lab's short-term growth potential, including the launch of VYVGART and new products, as well as the business development potential of DLL3ADC and ZL-1503 [1] - UBS expects VYVGART to continue expanding its indications and increasing sales, with several key candidate drugs anticipated to receive approval [1] Group 2 - UBS identifies VYVGART as a first-in-class FcRn drug with the potential to become a blockbuster in immunology, predicting peak sales in China to reach USD 1.8 billion [2] - Potential blockbuster candidates include Bemarituzumab for gastric cancer, Povetacicept for autoimmune diseases, VRDN-003 for thyroid eye disease, and KarXT for schizophrenia [2] - Zai Lab is expected to achieve profitability through revenue growth and operational efficiency improvements, with guidance for non-GAAP operational breakeven by Q4 2025 and for the full year 2026 [2] Group 3 - UBS believes that local manufacturing and reduced selling costs due to VYVGART's capacity expansion will improve overall gross margins [2] - The company plans to limit R&D spending growth by prioritizing high-value projects, while sales, general, and administrative expenses are expected to grow moderately [2]

Core Insights - Vertex Pharmaceuticals has announced significant advancements in its programs targeting immunoglobulin A Nephropathy (IgAN), APOL1-mediated kidney disease (AMKD), and autosomal dominant polycystic kidney disease (ADPKD) [1] - The updates indicate progress towards the company's goal of developing first-in-class or best-in-class therapies that address the underlying causes of these serious kidney diseases [1] Summary by Categories - **Company Developments** - Vertex Pharmaceuticals is making strides in its research and development efforts for kidney disease therapies [1] - The company aims to introduce innovative treatments that could potentially lead the market in addressing these conditions [1] - **Therapeutic Focus** - The advancements are specifically related to IgAN, AMKD, and ADPKD, highlighting the company's commitment to tackling significant health challenges in nephrology [1] - The focus on first-in-class or best-in-class therapies suggests a strategic approach to differentiate its products in a competitive landscape [1]