Takeaways:
- Inaxaplin (VX-147) by Vertex Pharmaceuticals enters phase 3 of a global trial, comparing a once-daily, 45 mg oral dose to placebo for APOL1-mediated kidney disease (AMKD).
- The AMPLITUDE trial expands to include adolescents aged 10 to 17 years and aims to evaluate inaxaplin's impact on kidney function and proteinuria.
- Phase 2a proof-of-concept data showed a significant reduction in urine protein to creatinine ratio, indicating potential efficacy in treating AMKD.
- The trial's primary outcomes include assessing changes in urine protein to creatinine ratio and estimated glomerular filtration rate (eGFR) slope.
- Inaxaplin has received Breakthrough Therapy Designation from the FDA, reflecting its potential as a disease-modifying therapy for AMKD.
Inaxaplin (VX-147; Vertex Pharmaceuticals) to treat APOL1-mediated kidney disease (AMKD) is moving into the phase 3 portion of a global phase 2/3 clinical trial that will compare the once-daily, 45 mg oral dose to placebo, in addition to standard of care, Vertex Pharmaceuticals announced in a press release.1
The AMPLITUDE trial (NCT05312879) was created to assess the impact of inaxaplin on kidney function and proteinuria for those living with proteinuric kidney disease mediated by 2 variants in the APOL1 gene (AMKD). The trail will now include adolescents with AMKD aged 10 to 17 years.1,2
“AMKD is a rapidly progressing condition and often remains silent until the disease reaches an advanced stage," said Glenn M. Chertow, MD, MPH, professor of medicine, Stanford University School of Medicine, chair, Vertex’s APOL1 Program Steering Committee, in a press release.1
"We have no approved disease-specific therapies for this truly devastating condition, and inaxaplin has the potential to transform the care of AMKD and significantly improve the lives of patients. The kidney community is strongly encouraged by inaxaplin’s potential, which energizes those of us caring for patients with AMKD,” added Chertow.1
Reported in The New England Journal of Medicine in March 2023 was phase 2a proof-of-concept data that demonstrated a statistically significant and clinically meaningful mean reduction in urine protein to creatinine ratio of 47.6% at 13 weeks of treatment compared to baseline (primary outcome).1,3
The data provided the first clinical evidence that an oral small molecule APOL1 inhibitor could decrease proteinuria in patients with AMKD.3
“Inaxaplin, a first-in-class molecule that addresses the underlying cause of APOL1-mediated kidney disease, has already shown impressive results in the Phase 2a proof-of-concept study," stated Carmen Bozic, MD, chief medical officer, executive vice president, Global Medicines Development and Medical Affairs, Vertex.1
