Vertex Pharmaceuticals (NASDAQ: VRTX) has encountered several clinical setbacks over the past 18 months, including for two products in its growing pain medicine franchise. Even with recent approvals outside its core therapeutic area of drugs that treat the underlying causes of cystic fibrosis (CF), given the slow commercial uptake of these newer products and recent clinical trial failures, some investors were starting to doubt the company’s ability to perform well outside its CF business.
However, the biotech just showed that it has what it takes to do so. Vertex’s shares recently jumped by about 8% on strong late-stage clinical trial results for a promising candidate. But is it too late to buy the company’s shares now? Let’s find out.
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Vertex Pharmaceuticals is developing povetacicept as a potential treatment for immunoglobulin A nephropathy (IgAN), a kidney disease caused by an abnormal buildup of IgA antibodies in the glomeruli, which are filters in the kidney, leading to kidney damage and protein in the urine. IgAN can even cause kidney failure and death. In a phase 3 study, povetacicept demonstrated a significant, clinically meaningful reduction in the urine protein-to-creatinine ratio (UPCR), a marker of kidney damage.
A lower UPCR indicates that povetacicept is working as intended. The medicine was also well tolerated, with most adverse events being mild or moderate. Vertex Pharmaceuticals plans to submit an application to the U.S. Food and Drug Administration for accelerated approval of povetacicept by the end of March.
Some existing treatments for IgAN, although effective at protecting the kidney, fail to address the underlying causes of the disease. That’s what povetacicept targets: it seeks to reduce IgA antibody production. That’s why it could be a highly successful medicine in a market with about 1.5 million patients globally. What’s more, Vertex Pharmaceuticals sees povetacicept as a potential pipeline-in-a-drug candidate and plans to target several other kidney-related diseases with it.
But there is more. Vertex Pharmaceuticals has other potential catalysts on the horizon. The company could release phase 3 data for inaxaplin, an investigational therapy for APOL-1 mediated kidney disease, this year. It could also request approval for zimislecel, a potential therapy for Type 1 diabetes. Meanwhile, Vertex’s core CF franchise is doing well. In 2025, the company’s revenue increased by 9% year over year to $12 billion.
