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BOSTON - The U.S. Food and Drug Administration has granted Breakthrough Therapy Designation to Vertex Pharmaceuticals’ (NASDAQ:VRTX) povetacicept for the treatment of IgA nephropathy, a serious kidney disease affecting approximately 300,000 people in the United States and Europe. The $96.56 billion biotechnology company, which InvestingPro analysis shows maintains strong financial health with a current ratio of 2.52, continues to expand its therapeutic portfolio beyond its core cystic fibrosis franchise.
The designation, which aims to expedite development of drugs that may demonstrate substantial improvement over existing treatments, was based on data from Vertex’s Phase 2 RUBY-3 clinical trial.
Povetacicept is currently being studied in the global Phase 3 RAINIER trial. Vertex remains on track to file for accelerated approval in the U.S. in the first half of 2026 if interim analysis data at 36 weeks proves positive.
The company also announced completion of enrollment for the interim analysis cohort of its AMPLITUDE global Phase 2/3 trial evaluating inaxaplin in APOL1-mediated kidney disease. If the 48-week interim data shows positive results, Vertex plans to seek accelerated approval in the U.S.
Additionally, Vertex has initiated AGLOW, a Phase 2 proof-of-concept study of VX-407 for autosomal dominant polycystic kidney disease. The 52-week single-arm study will evaluate the effect of VX-407 on kidney volume in patients with a subset of PKD1 gene variants, estimated to represent up to 10% of the overall ADPKD population.
"Today’s announcements mark significant milestones toward bringing the next wave of promising medicines to patients who are waiting," said Carmen Bozic, Executive Vice President and Chief Medical Officer at Vertex, according to the company’s press release.
These developments represent Vertex’s expansion into kidney disease treatments, building on its existing portfolio of therapies for cystic fibrosis, sickle cell disease, beta thalassemia and acute pain.
In other recent news, Vertex Pharmaceuticals has reported several key developments. The company delivered a slight revenue beat in its latest quarterly earnings report and maintained its previous guidance, although Canaccord Genuity noted the commercial side of the report was "uneventful." Meanwhile, Italy’s Medicines Agency has approved reimbursement for Vertex’s gene editing therapy CASGEVY, which targets transfusion-dependent beta thalassemia and severe sickle cell disease. This approval is significant given Italy’s large population affected by these conditions.
On the analyst front, Leerink Partners upgraded Vertex’s stock rating to Outperform, citing strong prospects for the company’s pain medication, Journavx. Goldman Sachs reiterated its Buy rating on Vertex, emphasizing the company’s diversification beyond its cystic fibrosis franchise. However, BofA Securities lowered its price target for Vertex, maintaining a Neutral rating due to a weaker fiscal year 2025 outlook and concerns about macroeconomic conditions. Additionally, Canaccord Genuity adjusted its price target to $411, pointing to mixed pipeline updates as a factor. These developments highlight the varied perspectives on Vertex’s future performance.
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