A sign hangs in front of Vertex Pharmaceuticals' global headquarters in Boston.
Brian Snyder | Reuters
Vertex Pharmaceuticals said its experimental drug for a rare kidney disease was successful in a Phase 3 trial, a crucial step in the company's path to diversify beyond its core cystic fibrosis drugs.
The Boston-based drugmaker said Monday that its immunoglobulin A nephropathy drug, povetacicept, decreased levels of a marker of the autoimmune disease by 52% in a late-stage trial. This surpassed the bar that analysts had set for Vertex's drug to compete with a recently approved treatment from Japan-based Otsuka and another in the pipeline from a U.S.-based biotech company. Vera Therapeutics. Vertex shares rose more than 9% on Tuesday.
Vertex's successful trial is an important first step in unlocking a new franchise in kidney disease, said Cantor Fitzgerald analyst Carter Gould. Vertex is developing two drugs behind povetacicept, and Gould predicts that the three together will generate more than $10 billion in revenue a year. That could rival Vertex's cystic fibrosis franchise, which generated more than $11 billion in sales last year.
“You really don't have to look too hard to connect the dots and say this is a pretty significant white space that they could be growing into,” Gould said.
Vertex transformed cystic fibrosis treatment with a portfolio of drugs for the inherited lung disorder, but the company has repeatedly faced questions about whether it could replicate that success in other diseases. In recent years, the company has expanded into blood disorders with the approval of its gene-editing treatment Casgevy and into acute pain with its drug Journavx. So far, neither has had much success, forcing Vertex to look for other opportunities to expand.
In 2024, Vertex paid nearly $5 billion to acquire Alpine Immune Sciences and its lead program, povetacicept. The drug could treat a rare autoimmune disease known as IgAN that prevents the kidneys from working properly, sometimes leading to patients needing dialysis or even a transplant. Vertex also plans to test the drug for other kidney conditions.
The company expects to complete its application to the US Food and Drug Administration for povetacicept in IgAN later this month, with approval possible later this year with the use of a priority review voucher.
