Regulus Therapeutics Releases Early Data From Of Investigational For Rare Type Of Kidney Disease

Regulus Therapeutics Inc RGLS released topline results from the first cohort of patients in its Phase 1b Multiple-Ascending Dose (MAD) trial of RGLS8429 for Autosomal Dominant Polycystic Kidney Disease (ADPKD).

ADPKD is an inherited condition that causes tiny fluid-filled sacs called cysts to develop in the kidneys.

In the first cohort, 12 subjects were randomized 3:1 to receive either 1 mg/kg of RGLS8429 or placebo every other week for three months. 

Also Read: Sanofi Opts To Terminate Rare Disease Program With Regulus Therapeutics.

RGLS8429 was well tolerated by all 9 subjects receiving the active drug with no safety findings.

Increases in disease-related urinary biomarkers, including urinary polycystin 1 (PC1) and polycystin 2 (PC2), were observed. 

PC1 and PC2 are the protein products of the PKD1 and PKD2 genes, respectively, and have been shown to correlate with disease severity inversely.

Statistically significant increases in mean PC1 levels were observed at Day 85 and Day 86 (n=9) compared to baseline (36%-41%). 

Although not statistically significant, numerical increases in PC2 were observed during the treatment period.

Furthermore, the data suggests the opportunity to demonstrate increased urinary polycystin response at the higher doses being tested in subsequent cohorts in Phase 1b. Data from the second cohort, dosed at 2mg/kg, are anticipated in the first quarter of 2024.

Price Action: RGLS shares are down 10.90% at $1.47 on the last check Wednesday.