Inaxaplin Shows Kidney Benefit In Additional Patient Groups As Vertex Completes Key Trial Enrollment
(RTTNews) - Vertex Pharmaceuticals Inc. (VRTX) reported positive Phase 2b results for Inaxaplin in additional groups of patients with APOL1-mediated kidney disease (AMKD), while also announcing completion of enrolment in its ongoing Phase 2/3 AMPLITUDE trial.
The findings support the continued development of the investigational therapy for a rare genetic kidney disease that can lead to progressive kidney damage.
APOL1-mediated kidney disease is caused by specific genetic variants that increase the risk of kidney disease and kidney failure. Patients often experience proteinuria, a condition in which excess protein leaks into the urine, serving as an important marker of kidney damage.
The Phase 2b AMPLIFIED study evaluated a once-daily 45 mg dose of Inaxaplin on top of standard treatment in 41 patients across two groups: those with AMKD and modest proteinuria and those with AMKD, type 2 diabetes, and proteinuria. The primary endpoint measured changes in urine albumin to creatinine ratio (UACR), a marker of kidney health.
In patients with AMKD and modest proteinuria, treatment with Inaxaplin reduced UACR by 42.7% at Week 13 compared with baseline. In patients with AMKD and type 2 diabetes, UCAR was reduced by 17.3% over the same period. Reductions were also observed in urine protein-to-creatinine ratio (UPCR), another measure of protein leakage in the urine.
Vertex said Inaxaplin was generally safe and well tolerated across both study cohorts. There were no serious adverse events related to treatment, and most side effects were mild to moderate in severity.
Inaxaplin is a first-in-class oral therapy designed to target the underlying cause of APOL1-mediated kidney disease. The program is part of Vertex's broader effort to develop treatments for serious kidney disorders.
Upcoming Milestones
Vertex has completed enrollement in the Phase 2/3 AMPLITUDE trial evaluating Inaxaplin in patients with more severe forms of APOL-1 mediated kidney disease. The company remains on track to report interim analysis results in early 2027, which, if positive, could support a potential accelerated approval filing in the United States.
VRTX has traded between $374.17 and $560.25 over the past year. The stock is currently trading at $525.18, up 2.88%.
