Vertex claimed its second early-stage win of the fall Wednesday, announcing positive results in a small study on a genetically defined form of kidney disease.
The 16-patient, Phase II trial focused on patients with focal segmental glomerulosclerosis, a rare disease where kidneys are unable to filter blood properly. Over 13 weeks on an experimental pill, the level of protein in the patients’ urine fell by an average of 47.6%.
Protein urine levels, or proteinuria, is a widely accepted biomarker for kidney function and a potential predictor for a patient’s long-term risk of going into kidney failure.
“As a first result, I think it’s terrific,” said Jonathan Himmelfarb, director of the Kidney Research Institute at the University of Washington, who was not involved in the study. “If it’s sustainable and it reflects improvement in an injury process in the kidneys, it is potentially very significant.”
Vertex sees the study as a proof of concept for its efforts to tackle a larger set of kidney diseases driven by the gene APOL1. Variants of the gene, which plays a role in the immune system, can help boost protection against parasitic infections, including human African trypanosomiasis, also known as African sleeping sickness.
But patients who carry one or especially two variants are also at a higher risk of developing kidney disease. Vertex cites figures that 50% of African Americans carry at least one variant and 13% carry two. It’s one of several factors — alongside access to healthcare and other social determinants of health — that account for the wide disparity in kidney disease between white and Black Americans.
Vertex’s molecule, VX-147, is designed to inhibit APOL1 and slow kidney injury.
“It’s a very important target from a public health perspective,” Himmelfarb said.
The data, while early, will likely come as welcome news for analysts, many of whom have remained skeptical of the company’s ability to repeat the success of its cystic fibrosis franchise. In a note Tuesday, Jefferies analyst Michael Yee placed the bar for success at a 15% reduction in proteinuria, but “the higher the better.”
The 47.6% reduction came from 13 patients, with Vertex excluding 3 patients who it said did not comply with trial protocols. There were no serious adverse events, the company said. The most common side effects were headache, back pain and nausea, which all occurred in over 15% of patients.
It’s the second successful readout for Vertex this fall after landmark results on a potential cure for type 1 diabetes came in October, albeit for just one patient.
Vertex now plans to move VX-174 into a larger pivotal trial for multiple types of APOL1-mediated kidney diseases. The company did not disclose details for the study, but the FDA has said that, for certain kidney diseases, the agency will grant accelerated approval based on reduction of proteinuria, providing a clear path to market if early results bear out in a larger study.
The company estimates 100,000 people in the US and Europe are currently living with kidney disease potentially addressable by their molecule.
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