Vertex gets first big homegrown win outside CF with VX-147 kidney data

Vertex’s first significant clinical win for a homegrown program outside of cystic fibrosis in the past decade was good for a gain of $4.6 billion in market cap and positions the company to advance the first genetically targeted kidney disease therapy into pivotal testing.

Vertex Pharmaceuticals Inc. (NASDAQ:VRTX) said Wednesday that oral small molecule APOL1 inhibitor VX-147 reduced proteinuria 47.6% in 13 evaluable patients, meeting the primary endpoint of percent change from baseline in urine protein to creatinine ratio (UPCR) at week 13 in a Phase II study to treat APOL1-mediated focal segmental glomerulosclerosis (FSGS)...