Vertex Pharmaceuticals FY Conference Summary Company Overview - **Company**: Vertex Pharmaceuticals (NasdaqGS: VRTX) - **Industry**: Biotechnology, focusing on cystic fibrosis and expanding into renal and pain management therapies Key Points Strategic Diversification - Vertex has transitioned from a cystic fibrosis-focused company to a more diversified biotech firm, aiming to broaden its revenue base and patient demographics [2][3] - The company has launched ALYFTREK, its fifth commercialized medicine, and is developing a next-gen 3.0 family of therapies [2] Revenue Projections - Vertex expects combined revenue from CASGEVY (for sickle cell disease and beta thalassemia) and Journavx (for acute pain) to exceed $500 million in 2026 [3] - Journavx is projected to triple the number of prescriptions in 2026 compared to 2025, with revenue growth expected to outpace prescription growth [3] Renal Franchise Development - Vertex aims to establish a renal franchise comparable in size to its cystic fibrosis business, with three ongoing phase 3 programs and a phase 2 study for autosomal dominant polycystic kidney disease [4] - The company is committed to R&D in serious diseases with validated markers and efficient clinical pathways [5] Povetacicept Data Highlights - Povetacicept shows a 52% reduction in UPCR at 36 weeks, with significant secondary endpoint results: 77% reduction in Gd-IgA1 and 85% resolution of hematuria [7][8] - 42% of patients achieved KDIGO recommended guidelines of UPCR less than 0.5 grams per gram, indicating strong efficacy in a real-world patient population [9] Safety Profile - The safety profile of povetacicept is favorable, with most adverse events being mild or moderate and no serious adverse events related to the drug [19] - No discontinuations due to infections were reported, and common adverse events included upper respiratory tract infections and injection site reactions [19] Future Studies and Indications - Vertex is conducting phase 2/3 studies for primary membranous nephropathy and plans to initiate a phase 2 study for myasthenia gravis in the first half of the year [23][24] - The company sees potential for povetacicept in multiple conditions, with ongoing discussions about commercial opportunities in nephrology [26] Cystic Fibrosis Business Update - ALYFTREK is expected to see continued growth, with a focus on younger patients and rare mutations, particularly in Europe [35] - The company reported that 30% of patients aged 12+ and over 60% of patients aged 2-5 are achieving diagnostic levels, indicating strong treatment efficacy [37] Journavx Commercial Strategy - Vertex is doubling its sales force from 150 to 300 reps to enhance physician access and drive adoption of Journavx [44] - The company is increasing marketing investments, including direct-to-consumer advertising, to boost awareness and uptake [44] Upcoming Trials - Vertex is conducting two phase 3 trials for diabetic peripheral neuropathy, with enrollment expected to complete by year-end, setting the stage for results in 2027 [50] Conclusion - Vertex Pharmaceuticals is strategically diversifying its portfolio beyond cystic fibrosis, with promising developments in renal therapies and pain management. The company is focused on maintaining a strong safety profile while driving revenue growth through innovative therapies and expanding its commercial capabilities.

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 ...