Vertex Pharmaceuticals of Boston announced that its Phase 2b AMPLIFIED trial of the oral agent inaxaplin met its primary efficacy endpoints in patients with APOL1-mediated kidney disease (AMKD). The study evaluated a 45 mg once‑daily dose added to standard care.
The trial enrolled 41 participants across two cohorts. In the first group of 23 patients with modest proteinuria, the urine albumin‑to‑creatinine ratio fell by 42.7 % and the urine protein‑to‑creatinine ratio declined by 44.7 % after 13 weeks. The second cohort, comprising 18 patients who also had type‑2 diabetes, showed reductions of 17.3 % in albumin‑to‑creatinine and 25.4 % in protein‑to‑creatinine ratios over the same period.
Safety data were described as generally favorable. No serious adverse events were linked to inaxaplin, and all reported events were mild or moderate. Headache was the most frequent complaint, affecting 7.3 % of participants. Five subjects experienced isolated, asymptomatic elevations in liver enzymes that resolved without intervention.
Vertex noted that the Phase 2/3 AMPLITUDE trial has completed enrollment and that an interim analysis is slated for early 2027 once the cohort reaches 48 weeks of treatment. The company indicated that positive outcomes could support an accelerated approval pathway in the United States.
AMKD, a condition driven by two APOL1 gene variants, predominantly affects individuals of African ancestry, with an estimated 150,000 patients in the United States and Europe. No therapies are currently approved for this form of kidney disease, underscoring the potential significance of Vertex's findings.
