Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) claimed its second early-stage win after announcing positive results in a small study of VX-147 on a genetically defined form of chronic kidney disease.

• The 16-patient, Phase 2 trial focused on patients with focal segmental glomerulosclerosis, a rare disease where kidneys cannot filter blood properly. 
• Over 13 weeks, VX-147, on top of standard of care, achieved a statistically significant reduction in protein level in the patients' urine of an average of 47.6%.
• Stat News' Adam Feuerstein tweeted, "In a medical first, Vertex $VRTX drug successfully targets the underlying cause of a genetic kidney disease."
• Protein urine levels, or proteinuria, are a widely accepted biomarker for kidney function and a potential predictor for a patient's long-term risk of going into kidney failure.
• The 47.6% reduction came from 13 patients, with Vertex excluding three patients who did not comply with trial protocols. 
• There were no serious adverse events, the company said. The most common side effects were headache, back pain, and nausea, which all occurred in over 15% of patients.
• Vertex now plans to move VX-174 into a larger pivotal trial for multiple types of APOL1-mediated kidney diseases in Q1 2022. 
• Price Action: VRTX shares up 9.03 at $203.83 during the market session on the last check Wednesday.