- Regulus Therapeutics (NASDAQ:RGLS) presents additional data from the first cohort of patients in its Phase 1b clinical trial of RGLS4326 for the treatment of autosomal dominant polycystic kidney disease (ADPKD), as well as new preclinical data from relevant animal models of the disease.
- The Phase 1b 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.
- In addition, one patient saw their uNGAL levels drop into the normal range during the course of the study.
- RGLS4326 was well-tolerated with no serious adverse events.
- Preclinical study results:
- RGLS4326 treatment led to increased Pkd1 and Pkd2 gene expression (>100% increase) and increased levels of their encoded proteins PC1 and PC2 (~50% increase) in Pkd1(RC/-) cells in vitro.
- RGLS4326 treatment demonstrated improvement in key efficacy parameters including kidney-weight-to-body-weight ratio (~75% decrease), serum creatinine (~50% decrease) and BUN (~60% decrease) in Pkd1(F/RC) mice compared to control.
- The results were presented at PKD Connect Conference 2021.