Vertex Pharmaceuticals said Wednesday that it plans to further develop a drug that could treat the underlying cause of a genetic kidney disease, following positive safety and efficacy results from a small clinical trial.
The drug, called VX-147, aims to interfere with the APOL1 gene, which in some patients causes the buildup of protein in urine and hinders the kidney’s ability to function properly. In an analysis of just over a dozen patients with focal segmental glomerulosclerosis, or FSGS, Vertex found its drug led to a 47.6 percent reduction in protein levels over 13 weeks, a result that it said was both statistically significant and clinically meaningful for patients.
The company said it expects to begin a pivotal trial of the drug in the first quarter of next year. It’s a milestone for the Boston biotech, which has been trying to diversify its business beyond cystic fibrosis treatments.
“It is a very exciting moment,” said David Altshuler the company’s chief scientific officer, in an interview.
Altshuler said the latest kidney disease data validates the company’s strategy, which is to develop therapies for diseases where there is already a deep understanding of the underlying cause — and to “not just do it once.” Vertex has four approved drugs on the market for cystic fibrosis, but its aim has been to apply the same prowess to other disease areas, such as kidney disease, sickle cell disease, and diabetes.
He said there are several reasons that the trial results are “unprecedented,” including that if approved, VX-147 would be the first treatment that addresses the underlying cause of this type of kidney disease, which afflicts about 100,000 people in the US and Europe.
Other kidney disease treatments available aim to quell symptoms of the disease, but they have been associated with resistance or severe side effects. Vertex said its oral pill was well tolerated among trial participants, with no serious side effects observed. Headache, back pain, and nausea were the most common side effects.
Vertex started its drug program in 2015, five years after Dr. Martin Pollak of Beth Israel Deaconess Medical Center discovered the gene abnormalities that were driving kidney disease. Altshuler said going from the discovery of a genetic disease cause to a proof-of-concept trial in 11 years is “quite quick.”
The next trial of VX-147 would pave the way for regulatory approval. Vertex said it is too early to share a potential timeline for commercial availability of the kidney disease drug.