Vertex Pharmaceuticals Inc. (VRTX): Phase 2 kidney data is a positive for the vertical

Goldman Sachs maintains a Buy rating on Vertex Pharmaceuticals (VRTX) with a 12-month price target of $653 (26.8% upside vs. $514.99). VRTX reported Phase 2b AMPLIFIED results for inaxaplin, an oral once-daily small molecule, in APOL1-mediated kidney disease (AMKD). Cohort 1 (modest proteinuria, n=22) showed UACR/UPCR reductions of 42.7%/44.7%, above management's ~20-40% bar and consistent with prior NEJM Phase 2 data. Cohort 2 (AMKD with type 2 diabetes, n=17) showed modest but additive reductions of 17.3% (UACR) and 25.4% (UPCR). Inaxaplin was generally safe and well tolerated, with no drug-related SAEs. Management noted the data could expand the addressable AMKD population by ~100K patients. The pivotal Phase 3 AMPLITUDE trial is fully enrolled, with 48-week interim data expected in early 2027, potentially supporting accelerated approval in the US. GS estimates peak WW sales of ~$2bn, with VRTX-estimated addressable population of ~150K in the US/EU (85% in the US).