Vertex Pharmaceuticals (VRTX) investor relations material

Key catalysts and pipeline updates

• Three ongoing commercial launches: LifTrak for cystic fibrosis, Casgevy for sickle cell disease and beta thalassemia, and Jurnavix for acute pain, marking a new phase of revenue and commercialization diversification.

• Four ongoing phase 3 programs and a fifth to start soon, including diabetic peripheral neuropathy, immunoglobulin A nephropathy (IgAN), APOL1-mediated kidney disease, and primary membranous nephropathy.

• Major enrollment milestones for enoxaparin in AMKD and POVI in IgAN expected by year-end, with data for POVI in IgAN anticipated in the first half of 2026.

• Regulatory filings for POVI and Zomylacell in type 1 diabetes are possible in 2026.

• Ongoing dispute with Royalty Pharma over LifTrak royalties, expected to resolve by end of 2026.

Cystic fibrosis franchise and growth drivers

• Survival benefits from CFTR modulators are driving market growth, with approvals expanding to younger patients and new geographies.

• LifTrak targets rare mutations and patients who discontinued previous therapies, offering once-daily dosing and a favorable side effect profile.

• VX-522, an inhaled mRNA therapy, is in development for patients not producing CFTR protein.

• Majority of LifTrak prescriptions are switches from Trikafta, with additional liver monitoring required in the U.S.

• Next-generation VX-828 aims to bring more patients to carrier-level sweat chloride, with patient trials expected before year-end.

Pain franchise and Jurnavix launch

• Jurnavix achieved unrestricted access for 85 million U.S. lives by August, with ongoing negotiations to expand coverage.

• Jurnavix is positioned as a non-opioid with a clean side effect profile, used primarily at discharge for acute pain, with average script duration of 14 days.

• Two phase 3 studies in diabetic peripheral neuropathy (DPN) are underway, with enrollment expected to complete in 2026.

• Strategy for chronic pain label is evolving based on FDA feedback, with potential for combination therapies in the future.