Vertex Pharmaceuticals (VRTX) investor relations material

Strategic diversification and pipeline expansion

• Expanded beyond cystic fibrosis into multiple therapeutic areas, maintaining strong CF growth while diversifying revenue and patient base.

• Ongoing launches in sickle cell disease, beta thalassemia, and acute pain have established strong commercial and clinical foundations.

• Targeting over $500 million in combined revenue from new products by 2026, with JOURNAVX prescriptions expected to more than triple year-over-year.

• Renal franchise aims to rival CF in size, with three phase III programs and a phase II in polycystic kidney disease underway.

• R&D strategy focuses on serious diseases with clear biology, validated markers, and efficient clinical/regulatory pathways.

Povetacicept phase III data and regulatory progress

• Phase III interim analysis showed a 52% reduction in UPCR at 36 weeks, with secondary endpoints showing 77% reduction in Gd-IgA1 and 85% hematuria resolution.

• 42% of patients achieved KDIGO guideline targets for UPCR, with high rates of background therapy and real-world patient characteristics.

• Safety profile was favorable, with most adverse events mild or moderate, no serious adverse events or discontinuations due to infection.

• Rolling submission for regulatory approval to be completed by end of month, using a priority review voucher for expedited FDA review.

• Additional indications in primary membranous nephropathy and myasthenia gravis are in phase II/III or planned for phase II initiation.

Commercial outlook and market opportunities

• IgAN market has 330,000 diagnosed patients in US/EU with significant unmet need; commercial infrastructure and payer engagement are well underway.

• PMN and myasthenia gravis represent additional opportunities, with differentiated mechanisms and ongoing clinical development.

• Inaxaplin for APOL1-mediated kidney disease is in pivotal trials, with interim results expected late this year or early next; endpoints include proteinuria reduction and eGFR stabilization.

• ALYFTREK is driving CF franchise growth, with strong uptake, especially in younger patients and those with rare mutations, and ongoing expansion in Europe.

• JOURNAVX momentum supported by expanded sales force, direct-to-consumer marketing, and positive phase IV data showing high opioid-free rates post-treatment.