A sign is posted in front of Vertex Pharmaceuticals’ world headquarters in Boston.
brian snyder reuter
vertex pharmaceuticals The company said its investigational drug for a rare kidney disease has passed a successful Phase 3 trial. This is an important step for the company to diversify beyond its flagship drug for cystic fibrosis.
The Boston-based drug company announced Monday that its immunoglobulin A nephropathy drug pobetacicept reduced levels of markers of autoimmune conditions by 52% in a late-stage trial. This exceeded the threshold that analysts had set for Vertex’s drug to compete with a recently approved treatment from Japan-based Otsuka Pharmaceuticals and another treatment being developed by U.S.-based Biotech. bella therapeutics. Vertex stock rose more than 9% on Tuesday.
Cantor Fitzgerald analyst Carter Gould said Vertex’s successful trial is an important step in opening new territory in kidney disease. Vertex is developing the two drugs behind pobetacicept, and Gould expects the three to generate combined annual revenue of more than $10 billion. That could rival Vertex’s cystic fibrosis franchise, which brought in more than $11 billion in sales last year.
“You don’t have to look very hard to connect the dots. This is a very meaningful void where they can grow,” Gould said.
Vertex has transformed the treatment of cystic fibrosis with its portfolio of treatments for inherited lung diseases, but the company has repeatedly faced questions about whether its success can be replicated in other diseases. The company has recently expanded into blood disorders with the approval of its gene-editing drug Kasgevy, and acute pain treatment with its drug Jernavx. Neither has been a huge success so far, and Vertex is exploring other expansion opportunities.
In 2024, Vertex paid nearly $5 billion to acquire Alpine Immune Sciences and its flagship program, pobetacicept. The drug could potentially treat a rare autoimmune disease known as IgAN, which prevents the kidneys from functioning properly and sometimes requires patients to undergo dialysis or a transplant. Vertex also plans to test drugs for several other kidney diseases.
The company expects to complete its U.S. Food and Drug Administration application for pobetacicept in IgAN by the end of this month, with priority review vouchers allowing for approval later this year.
