Vertex Pharmaceuticals has reported positive data from its Phase IIb AMPLIFIED trial evaluating inaxaplin in individuals with APOL1-mediated kidney disease (AMKD), including those with modest proteinuria and people diagnosed with type 2 diabetes (T2D).
The AMPLIFIED study evaluated a 45mg once-daily dose of inaxaplin alongside optimised standard of care. It included 41 people in two cohorts.
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The first involved 23 people with AMKD and modest proteinuria, while the second included 18 people with AMKD, T2D and proteinuria.
After 13 weeks, urine albumin-to-creatinine ratio (UACR) in the modest-proteinuria group had fallen by 42.7% from baseline, with a 95% confidence interval of -58.3% to -21.1%. Urine protein to creatinine ratio (UPCR) decreased by 44.7%.
Most people in this cohort had not been diagnosed with focal segmental glomerulosclerosis (FSGS).
Vertex said the findings were consistent with results from an earlier Phase IIa study in people with AMKD and FSGS, which recorded reductions of 43.4% in UACR and 47.6% in UPCR at week 13.
In the T2D cohort, UACR fell by 17.3% from baseline at week 13. The 95% confidence interval ranged from -36.3% to 7.2%. UPCR decreased by 25.4%.
Inaxaplin was generally safe and well tolerated across both groups. No serious adverse events related to treatment were reported, and all adverse events were mild or moderate.
Headache was the most common adverse event, affecting 7.3% of participants. Five people experienced isolated, asymptomatic transaminase elevations, which resolved.
Vertex chief medical officer and global medicines development and medical affairs executive vice-president Carmen Bozic said: “These results add to the evidence base for inaxaplin and its potential as a first- and best-in-class treatment targeting the underlying cause of AMKD.
“The modest proteinuria cohort, which included patients with and without FSGS, delivered remarkable reductions in proteinuria consistent with the original Phase IIa study in patients with AMKD and FSGS.
“We are also excited about the treatment effect, though smaller in magnitude, in the type 2 diabetes cohort. We look forward to discussing the AMPLIFIED results with regulators in the context of the AMPLITUDE pivotal study.”
The company has completed full enrolment in the Phase II/III AMPLITUDE trial studying inaxaplin in a separate group with severe proteinuria and no other kidney disease-causing conditions.
Vertex expects to report data from a planned interim analysis in early 2027, once the relevant group has received 48 weeks of treatment. If positive, the findings could support a potential application for accelerated approval in the US.
AMKD is caused by two APOL1 gene variants and can lead to progressive kidney damage.
In June 2025, Vertex Pharmaceuticals reported data from the Phase I/II segment of the Phase I/II/III FORWARD-101 trial of the stem cell-derived, fully differentiated islet cell therapy, zimislecel (VX-880), for individuals with type 1 diabetes.
