Regulus Therapeutics Presents Additional Data From Rare Kidney Disease Program

  • Regulus Therapeutics Inc RGLS announced the presentation of additional data from the first cohort from Phase 1b trial of RGLS4326 for autosomal dominant polycystic kidney disease (ADPKD), as well as new preclinical data. 
  • The data were shared at the PKD Connect Conference 2021.
  • The data demonstrate clinical proof of mechanism by showing target engagement in the kidneys through a statistically significant increase in urinary biomarkers PC1 and PC2, validating miR-17 as a target for ADPKD treatment. 
  • RGLS4326 was well-tolerated with no serious adverse events. 
  • The poster also describes new preclinical data showing treatment with RGLS4326 results in increased gene and polycystin levels in vitro. 
  • In addition, improvements in key disease markers were demonstrated in the Pkd1(F/RC) mouse model that harbors Pkd1 mutation equivalent to human ADPKD.
  • Price Action: RGLS shares are down 1.64% at $0.98 during the market session on the last check Friday.
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