A sign hangs in front of the world headquarters of Vertex Pharmaceuticals in Boston.
Brian Snyder | Reuters
Vertex Pharmaceuticals said its experimental drug for a rare kidney condition succeeded in a Phase 3 trial, a crucial step in the company’s path to diversify beyond its main drugs for cystic fibrosis.
The Boston-based drugmaker on Monday said its drug for immunoglobulin A nephropathy, povetacicept, decreased levels of a marker of the autoimmune condition by 52% in a late-stage trial. That passed the bar analysts had set for Vertex’s drug to compete with a recently approved treatment from Japan-based Otsuka and another in the pipeline from U.S.-based biotech Vera Therapeutics. Shares of Vertex 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 sees the three together generating more than $10 billion in revenue a year. That could rival Vertex’s cystic fibrosis franchise, which brought in more than $11 billion in sales last year.
“You don’t really have to look too hard to connect the dots and say this is pretty meaningful white space they could be growing into,” Gould said.
Vertex transformed the treatment for cystic fibrosis 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 conditions. The company in recent years expanded into blood disorders with the approval of its gene-editing treatment Casgevy and acute pain with its drug Journavx. Neither has so far been a runaway success, leaving Vertex to search 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 condition known as IgAN that prevents the kidneys from functioning properly, sometimes leading to patients needing dialysis or even transplant. Vertex also plans to test the drug for several other kidney conditions.
The company expects to complete its application with the U.S. Food and Drug Administration for povetacicept in IgAN by the end of this month, with approval possible later this year with the use of a voucher for priority review.