Vertex Updates on Kidney Programs at ASN Kidney Week

Vertex Pharmaceuticals Incorporated recently revealed promising progress in its research on various kidney diseases, including IgA nephropathy (IgAN), primary membranous nephropathy (pMN), and APOL1-mediated kidney disease (AMKD). The data presented showcases Vertex's potential transformative therapies and includes new findings on povetacicept, a dual inhibitor of the BAFF and APRIL pathways, specifically in IgAN and pMN. These updates were shared at the American Society of Nephrology’s (ASN) Kidney Week Congress held from October 23-27 in San Diego, California.

Carmen Bozic, M.D., the Executive Vice President of Global Medicines Development and Medical Affairs and Chief Medical Officer at Vertex, expressed satisfaction with the expansion of the company's innovative renal medicine pipeline. She emphasized the significant progress in their programs, which now cover AMKD, IgAN, pMN, and polycystic kidney disease. The data presented at the ASN congress further solidifies povetacicept as a potential leading therapy and demonstrates its versatility. Additionally, progress continues in developing treatments for AMKD and autosomal dominant polycystic kidney disease (ADPKD).

In the case of IgAN, Vertex provided data on 54 patients who received either 80 mg or 240 mg of povetacicept subcutaneously every four weeks. The treatment with 80 mg led to a meaningful reduction in proteinuria, with a 66% decrease in the urine protein to creatinine ratio (UPCR) at 48 weeks. This was accompanied by stable renal function, as measured by the estimated glomerular filtration rate (eGFR), and 63% of participants achieved clinical remission by week 48. The treatment also resulted in a significant decrease in anti-PLA2R1 autoantibodies, a marker associated with disease activity, by 87% at week 20.

Povetacicept was well-tolerated in patients with pMN, where it demonstrated a mean UPCR reduction of 62% from baseline at 24 weeks. The treatment's adverse effects were mild to moderate, with no serious adverse events linked to the drug.

Vertex also discussed their ongoing work with inaxaplin, an oral small molecule inhibitor of APOL1, aimed at treating AMKD. The Phase 3 portion of the global Phase 2/3 pivotal AMPLITUDE clinical trial for inaxaplin is currently enrolling and dosing participants.

IgAN is a chronic kidney disease characterized by the deposition of immune complexes in the renal glomerular mesangium, leading to kidney injury and fibrosis. It affects approximately 130,000 people in the U.S. alone and can progress to end-stage renal disease. Vertex's global Phase 3 RAINIER trial of povetacicept aims to assess its efficacy further in treating IgAN.

Primary membranous nephropathy (pMN) is another serious, life-threatening kidney disease that affects around 60,000 people in the U.S. It involves the immune system damaging the glomeruli, resulting in a progressive loss of kidney function. Vertex's RUBY-3 study, an ongoing Phase 1/2 trial, is investigating povetacicept in autoimmune glomerulonephritis, including IgAN and pMN.

AMKD is a genetic kidney disease caused by variants of the APOL1 gene, affecting about 100,000 individuals in the U.S. and Europe. These gene variants cause toxic effects on kidney cells, leading to kidney failure, which is often treated with dialysis or kidney transplant. Vertex's investigational drug, inaxaplin, is a potential first-in-class therapy targeting the underlying cause of AMKD.

Autosomal dominant polycystic kidney disease (ADPKD) is the most common inherited kidney disease, affecting approximately 250,000 people diagnosed in the U.S. and Europe. This genetic disorder leads to the formation of numerous fluid-filled cysts in the kidneys, causing progressive kidney enlargement and functional decline. Vertex is developing potential treatments to address this condition.

Vertex continues to advance its clinical and research programs in various serious diseases, with a robust pipeline of investigational therapies. Founded in 1989 and headquartered in Boston, Vertex is recognized for its commitment to scientific innovation and transformative medicines.