By Colin Kellaher

 

Vertex Pharmaceuticals Inc. on Wednesday said it plans to advance its VX-147 drug candidate into pivotal development in the first quarter after positive results from a Phase 2 proof-of-concept study in a severe genetic kidney disorder.

The Boston drug maker said treatment of patients with APOL1-mediated focal segmental glomerulosclerosis with VX-147 on top of standard of care achieved a statistically significant and clinically meaningful 47.6% mean reduction in the urine protein to creatinine ratio, adding that VX-147 was well tolerated.

Vertex said the study results provide the first clinical evidence and proof of concept that an oral small molecule APOL1 inhibitor can decrease proteinuria in patients with APOL1-mediated kidney disease.

Vertex said VX-147 holds the potential to be a first-in-class therapy for the more than 100,000 patients in the U.S. and Europe living with proteinuric kidney disease mediated by variants in the APOL1 gene, including, but not limited to, focal segmental glomerulosclerosis.

Shares of Vertex, which closed Tuesday at $186.94, rose nearly 7% in premarket trading Wednesday.

 

Write to Colin Kellaher at colin.kellaher@wsj.com

 

(END) Dow Jones Newswires

December 01, 2021 08:08 ET (13:08 GMT)

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