Spark of hope for potential disease modifier in nephrology

2 December 2021

Results from a small Phase II trial using a urine-based biomarker as a primary endpoint have been upheld by Vertex Pharmaceuticals (Nasdaq: VRTX) as a promising sign for its oral kidney disease candidate.

The data provide the first clinical validation of the mechanism for VX-147, a novel genetically targeted approach, in focal segmental glomerulosclerosis (FSGS).

In just 13 evaluable patients, a statistically-significant 47.6% reduction in proteinuria was observed after 13 weeks, compared to baseline.

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