Vertex Pharmaceuticals to Advance VX-147 Kidney Drug After Phase 2 Study
02 December 2021 - 12:23AM
Dow Jones News
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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