Tuesday, Regulus Therapeutics Inc (NASDAQ:RGLS) released topline results from the second cohort of patients in its Phase 1b multiple-ascending dose (MAD) study of RGLS8429 for Autosomal Dominant Polycystic Kidney Disease (ADPKD).
The study evaluates RGLS8429 treatment across three different weight-based dose levels, including measuring changes in urinary polycystins 1 and 2 (PC1 and PC2), height-adjusted total kidney volume (htTKV), cyst architecture, and overall kidney function.
PC1 and PC2 are the protein products of the PKD1 and PKD2 genes and have been shown to correlate with disease severity inversely.
RGLS8429 was well tolerated, with no ...