Regulus Therapeutics (RGLS) said on Friday the preliminary results of a planned interim data analysis from the new mouse chronic toxicity study of RGLS4326 in development for the treatment of Autosomal Dominant Polycystic Kidney Disease, or ADPKD, found no adverse findings.
“The planned interim analysis of this study after 13 weeks of dosing has shown no adverse or other significant findings across the range of doses tested and is intended to support re-initiation of the Phase 1 MAD (Multiple Ascending Dose) study after consultation with FDA,” the company said. “RGLS4326 has also been generally well-tolerated in the Phase 1 Single Ascending Dose and MAD studies in human subjects to date.”
Regulus said the unexpected observations from the previously terminated study were likely a result of technical issues at the contract research organization. In July, it paused its early-stage trial of RGLS4326 in healthy volunteers due to unexpected observations in the 27-week mouse chronic toxicity study.
It plans to submit a comprehensive data package for RGLS4326 to FDA that will include the results from the planned 13-week interim analysis and results from additional investigations, adding that it expects to engage with FDA to discuss the resolution of the voluntary pause in human dosing and the plan to resume the Phase 1 MAD study.