VRTX · Earnings call · 2026Q3T · Full report

Product / Drug Pipeline Progress

VERTEX PHARMACEUTICALS INC / MA · 2026-11-02 · Importance 17 · Surprise 24

Management said the AMPLIFIED inaxaplin study’s cohort 1 comprised patients with modest proteinuria, defined as a 0.3 to 0.7 UPCR range. Most participants did not have FSGS, and management said the cohort’s response was therefore not driven by an FSGS subgroup. The response was observed among a population that was predominantly non-FSGS.

Key facts

• Vertex completed enrollment in the AETNA Phase 2 dose-ranging study of povetacicept in generalized myasthenia gravis and expected proof-of-concept data in the first half of 2027.
• Both Phase 3 studies of suzetrigine in painful diabetic peripheral neuropathy remained on track to complete enrollment by year-end 2026, with results expected by mid-2027.
• Vertex expected interim results from the GALILEO global Phase 1/2 VX-670 trial in DM1 before the end of 2026.
• Vertex targeted completion of enrollment in the adult CAH Phase 3 atumelnant study in Q1 2027, followed by 32 weeks of dosing.
• Atumelnant has orphan drug designation in the U.S., EU, and Japan, and its Phase 3 pivotal trial in congenital adrenal hyperplasia is underway.
• Atumelnant was in a Phase 2 dose-ranging study in pediatric CAH; Vertex aimed to advance the program rapidly into pivotal development after planned regulatory interactions.
• The adaptive global Phase 2/3 atumelnant study in ACTH-dependent Cushing syndrome was underway outside the U.S.; the program recently received U.S. orphan drug designation.
• Povetacicept's OLYMPUS Phase 2/3 pivotal trial in membranous nephropathy was ongoing, with Phase 3 enrollment and dosing well underway at 80 mg subcutaneously every four weeks; the study endpoint is at 104 weeks.