Vertex Pharmaceuticals (VRTX) investor relations material

Cystic fibrosis portfolio and launches

• Alyftrek, the latest CFTR modulator, is showing strong uptake, especially among new-to-medicine patients and those with additional mutations not covered by Trikafta.

• Alyftrek offers once-daily dosing and greater efficacy, with improved sweat chloride reduction and expanded mutation coverage.

• Emotional attachment to Trikafta slows switching, but Alyftrek's convenience and efficacy are driving steady transitions.

• U.S. launch is nearly a year in, with recent regulatory approvals and reimbursement secured in key European countries.

• Ex-U.S. launches are progressing well, with a larger pool of eligible patients and unchanged liver monitoring requirements.

Pipeline expansion and next-generation therapies

• VX-828, a next-gen CFTR modulator, is in patient cohorts, aiming to treat 95% of CF patients and achieve normal sweat chloride levels.

• VX-522, an mRNA-based therapy with Moderna, targets the 5% of CF patients who lack CFTR protein; the program has resumed after a pause and is dosing patients.

• Safety and efficacy data for VX-522 are expected next year.

Kidney disease and autoimmune pipeline

• Povetacicept (pove) for IgAN completed phase III enrollment in under 15 months, with breakthrough designation and rolling BLA submission planned.

• Priority review voucher will accelerate review, with first module submission by year-end and full submission in H1 2025.

• Pove is positioned as best-in-class due to dual APRIL/BAFF inhibition, high potency, and patient-friendly monthly autoinjector dosing.

• Pove is also in pivotal development for membranous nephropathy and is being considered for myasthenia gravis.

• Inaxaplin for AMKD has completed interim cohort enrollment, with readout expected next year.