What happened

Shares of Vertex Pharmaceuticals (NASDAQ:VRTX) were jumping 8.2% as of 12:01 p.m. ET on Wednesday. The nice gain came after the big biotech announced positive results from a phase 2 proof-of-concept study evaluating VX-147 in treating patients with APOL1-mediated focal segmental glomerulosclerosis (FSGS).

FSGS is a kidney disease caused by variants of the APOL1 gene. Individuals with FSGS can require dialysis or kidney transplants. Some eventually die of the disease, which currently has no approved therapies targeting the underlying cause.

Two scientists sitting in front of monitors.

Image source: Getty Images.

So what

Vertex reported that participants in the phase 2 study who were treated with VX-147 had "statistically significant, substantial, and clinically meaningful mean reduction in proteinuria [proteins in the urine] of 47.6%" at 13 weeks after dosing compared to their baseline levels. The company also stated that the experimental drug was well tolerated with no serious adverse events related to it and no treatment discontinuations due to adverse events.

These positive results provided a big boost for Vertex when it was much needed. Prior to today, the biotech stock had fallen nearly 21% year to date. The decline was primarily due to Vertex's disappointing results earlier this year for its alpha-1 antitrypsin deficiency (AATD) program. Investors now have reason to be more confident about Vertex's prospects beyond cystic fibrosis (CF), an indication where the company has achieved tremendous success.

Now what

Vertex now plans to advance VX-147 into pivotal development in the first quarter of 2022. The company's clinical studies will target APOL1-mediated kidney disease, including (but not limited to) FSGS.

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