Vertex Pharmaceuticals (VRTX) investor relations material

Diversification and pipeline overview

• Continued growth in cystic fibrosis (CF) with global ALYFTREK launch and expansion to younger age groups; last 5% of CF patients still targeted for therapy.

• CASGEVY expanding globally, expected to become a multi-billion dollar asset; JOURNAVX launched as first non-opioid acute pain therapy in decades.

• Rare endocrine (Crinetics/PALSONIFY) and renal medicine now key pillars; six phase III and multiple phase II programs ongoing.

Renal franchise and pove program

• Pove for IgA nephropathy targets a large global population, with differentiation based on dual APRIL/BAFF inhibition, strong efficacy, and patient-friendly monthly auto-injector dosing.

• Largest renal sales force built to reach both centers of excellence and community settings.

• Regulatory strategy aligns with global standards, prioritizing two-year GFR data for worldwide approval, despite FDA's comfort with one-year GFR.

• Productive payer discussions with over 70% of payers, expecting reimbursement in line with current field standards.

• Pove phase III readout in membranous nephropathy expected next, with myasthenia gravis in phase II; broad label and indication expansion anticipated.

Inaxaplin and APOL1-mediated kidney disease

• Inaxaplin targets APOL1-mediated kidney disease (AMKD), a rapidly progressive condition affecting 100,000–150,000 in the U.S., with no approved therapies.

• Phase III AMPLITUDE study powered for one-year GFR endpoint; phase II AMPLIFY study readout expected in fall, exploring indication expansion to lower proteinuria and diabetic cohorts.

• Double-digit proteinuria improvement in modest proteinuria cohort seen as positive; regulatory path for expansion to be determined post-data.

• Genetic testing initiatives underway to improve diagnosis and patient identification, supporting future commercial rollout.

• Significant overlap in sales force for inaxaplin and pove, with pricing expected to align with current renal therapies.