Vertex Pharmaceuticals (VRTX) investor relations material

Cystic fibrosis (CF) franchise growth and innovation

• Growth in CF driven by serial innovation, younger patient access, rare mutation coverage, geographic expansion, and price increases, with 2026 revenue guidance of $12.95–$13.1 billion including $500M+ from CASGEVY and JOURNAVX.

• ALYFTREK uptake is strong, especially in Europe due to favorable label and reimbursement, with 11 new country agreements in Q1 and surpassing $1B in cumulative sales.

• Ongoing filings for ALYFTREK (ages 2–5) and TRIKAFTA (ages 1–2) expected to drive further growth in younger age groups and new geographies in 2025–2027.

• Conversion from TRIKAFTA to ALYFTREK is steady in the U.S. and steeper in Europe, aided by label differences and rare mutation coverage.

• Next-generation CFTR modulators (VX-828, VX-581, VX-272) are in development, with data on VX-828 expected later this year; high bar set by ALYFTREK for future innovation.

JOURNAVX launch and revenue outlook

• JOURNAVX and CASGEVY expected to generate $500M+ in revenue in 2026, with JOURNAVX prescription volume and revenue projected to more than triple year-over-year.

• Access progress is strong, with 240 million covered lives and major PBM and Medicare contracts secured; further Medicare negotiations ongoing.

• Free drug program being rolled back as coverage expands, improving gross-to-net and revenue conversion.

• Q1 saw 350,000 prescriptions and $29M in revenue, with a meaningful sequential ramp in JOURNAVX revenues expected in Q2.

• Contracts are structured to minimize access barriers, focusing on long-term value and patient access.

Pipeline and povetacicept (Povi) development

• Povi, an engineered APRIL-BAFF fusion protein, shows strong efficacy and safety in IgAN, with 52% UPCR reduction, 77% Gd-IgA1 reduction, and 85% hematuria resolution.

• Povi's monthly auto-injector format and safety profile are highlighted as key differentiators; accelerated approval filing submitted, with potential approval by year-end.

• Large U.S. IgAN market (160,000 patients) seen as supporting multiple transformative therapies; early launch signs are promising.

• Povi also in development for PMN and GMG, with phase II/III trials ongoing; mechanism targets B-cell-mediated diseases with potential for chronic dosing.

• High correlation between proteinuria reduction and eGFR stabilization expected to be validated by upcoming competitor data.