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An experimental pill from Vertex Pharmaceuticals reduced levels of a damaging urine protein by nearly half in a small clinical trial — encouraging evidence that a novel medicine can arrest the underlying genetic cause of a chronic kidney disease that progresses rapidly to kidney failure.

Vertex called the mid-stage study results “unprecedented” and said it was working quickly to advance the drug, called VX-147, into a pivotal clinical trial, pending discussions with regulators.


Investors viewed VX-147 as a high-risk program, mainly because there wasn’t much known about the drug or its potential impact on genetic kidney disease. Given low expectations, the positive study outcome could ease doubts about Vertex’s research pipeline and buoy its lagging stock price.

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