Inaxaplin Enters Phase 3 in APOL1 Kidney Disease Trial

Vertex Pharmaceuticals has progressed its drug candidate inaxaplin (VX-147) into the Phase 3 stage of a pivotal global clinical trial for APOL1-mediated kidney disease (AMKD). The trial will compare a daily 45 mg oral dose against a placebo, in addition to standard care. This advancement follows the Phase 2a proof-of-concept data that showed a significant reduction in urine protein levels in patients treated with inaxaplin. The study has also been expanded to include adolescents aged 10 to 17, broadening the potential patient base.

Inaxaplin is a novel, oral small molecule APOL1 inhibitor that targets the root cause of AMKD. The Phase 2a study provided initial clinical evidence that this type of inhibitor can effectively reduce proteinuria in individuals with AMKD. The drug's advancement to Phase 3 and the inclusion of younger patients is seen as a significant step towards offering a new treatment option for those affected by this condition.

AMKD is a rapidly progressing form of chronic kidney disease linked to two variants in the APOL1 gene. It often goes unnoticed until it reaches an advanced stage, and currently, there are no approved therapies specifically for AMKD. Inaxaplin's potential to transform care for this condition and improve the quality of life for patients is highly anticipated by the medical community.

The Phase 3 trial's primary goal is to measure the impact of inaxaplin on kidney function, specifically the estimated glomerular filtration rate (eGFR) slope, in comparison to placebo. A secondary endpoint will assess time to a composite clinical outcome, which includes a sustained decline in eGFR, onset of end-stage kidney disease, or death. The study is set to have a pre-planned interim analysis at Week 48, which could be instrumental for seeking accelerated approval in the U.S.

Inaxaplin has received Rare Pediatric Disease Designation (RPD) and Breakthrough Therapy Designation (BTD) from the FDA for APOL1-mediated focal segmental glomerulosclerosis (FSGS), and Priority Medicines (PRIME) and Orphan Drug designations from the European Medicines Agency (EMA) for AMKD.

Vertex Pharmaceuticals, a global biotechnology company, is dedicated to developing transformative medicines for serious diseases. The company has a history of innovation, with approved treatments for cystic fibrosis, sickle cell disease, and transfusion-dependent beta thalassemia, and continues to advance research in various serious conditions, including AMKD.

AMKD, caused by APOL1 gene variants, affects approximately 100,000 people in the U.S. and Europe who have proteinuric kidney disease. The inherited variants can lead to kidney cell damage and proteinuria, significantly impairing kidney function and potentially leading to dialysis, transplant, or death.

Vertex Pharmaceuticals is headquartered in Boston with international headquarters in London, and has a global presence with research and development sites and commercial offices across multiple regions.