Vertex Pharmaceuticals (VRTX) Barclays 28th Annual Global Healthcare Conference summary

Strategic diversification and pipeline expansion

• Diversified beyond cystic fibrosis with strong launches in sickle cell, beta thalassemia, and acute pain therapies, aiming for over $500 million in combined revenue from CASGEVY and JOURNAVX by 2026.

• Ongoing investment in R&D and focus on diseases with clear causal biology and validated markers, supporting continued growth and diversification.

• Next-generation cystic fibrosis therapies are in clinical development, with ALYFTREK uptake growing, especially among younger patients and those with rare mutations.

• Commercial infrastructure and patient outreach have been strengthened to support new launches and market expansion.

Renal franchise and pivotal clinical data

• Phase III data for povetacicept in IgAN showed a 52% reduction in UPCR at 36 weeks, 77% reduction in Gd-IgA1, and 85% hematuria resolution, with 42% of patients reaching KDIGO guideline targets.

• Study population reflected real-world patients with high rates of background therapy and long disease duration, enhancing relevance of results.

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

• Rolling submission for regulatory approval to be completed by end of month, ahead of expectations, with priority review anticipated.

Pipeline opportunities and future indications

• Povetacicept is being studied in primary membranous nephropathy (PMN) and will enter phase II for myasthenia gravis, with potential for additional indications.

• Inaxaplin, targeting 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.

• JOURNAVX is expanding into diabetic peripheral neuropathy, with phase III trials expected to read out in 2027, targeting a large unmet need.