Regulus's Polycystic Kidney Disease Candidate Shows Benefit At Interim Analysis

Regulus Therapeutics Inc RGLS has announced an incremental update from the first cohort of patients from its ongoing Phase 1b trial evaluating RGLS4326 in Autosomal Dominant Polycystic Kidney Disease (ADPKD).
ADPKD is a genetic disorder characterized by the growth of numerous cysts in the kidneys.
In the first cohort, nine patients were enrolled and received 1 mg/kg of RGLS4326 subcutaneously every other week for four doses.
The mean increase in polycystins 1 and 2 (lower protein levels associated with ADPKD) at the end of the study compared to baseline levels for all nine patients in the first cohort was 58% (p=.0004) 38%, respectively.
As disclosed earlier, treatment with RGLS4326 was generally well-tolerated with no serious adverse events reported. All reported adverse events were mild and generally transient.
These data will be presented at the Oppenheimer Rare & Orphan Disease Summit today.
Additional data from this first cohort will be presented at PKD Connect in June 2021, and an abstract will be submitted to the American Society of Nephrology annual meeting in November.
Data from the second cohort is expected in the third quarter.
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