VERTEX PHARMACEUTICALS INC

VERTEX PHARMACEUTICALS INC is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases, with a focus on specialty markets. The company has approved medicines for cystic fibrosis ("CF"), sickle cell disease ("SCD"), transfusion dependent beta thalassemia ("TDT"), and acute pain. VERTEX PHARMACEUTICALS INC continues to innovate and advance next-generation clinical and research programs in these areas. Its mid- and late-stage clinical pipeline includes programs across a range of modalities in additional serious diseases, including IgA nephropathy, APOL1-mediated kidney disease, neuropathic pain, type 1 diabetes, primary membranous nephropathy, autosomal dominant polycystic kidney disease, and myotonic dystrophy type 1. The company is advancing five pivotal programs across multiple disease areas. For IgA Nephropathy, VERTEX PHARMACEUTICALS INC is developing povetacicept, a dual inhibitor of the B cell activating factor ("BAFF") and a proliferation-inducing ligand ("APRIL") pathways, to treat IgA nephropathy ("IgAN"). Enrollment in the IgAN Phase 3 clinical trial was completed, and the first module of the rolling Biologics Licensing Application ("BLA") for povetacicept in IgAN was submitted in the fourth quarter of 2025. For APOL1-Mediated Kidney Disease, the company is developing inaxaplin, a small molecule inhibitor of APOL1. Enrollment of the interim analysis cohort of the Phase 2/3 clinical trial has been completed. For Peripheral Neuropathic Pain, VERTEX PHARMACEUTICALS INC is developing suzetrigine, a selective non-opioid NaV1.8 pain signal inhibitor, for diabetic peripheral neuropathy ("DPN"). Suzertrigine is being evaluated for the treatment of DPN in two Phase 3 clinical trials.