The poster will be presented at 3:00 PM Friday, I think.
Preclinical Evaluation and Results from the First Cohort of Phase 1b Clinical Trial of RGLS4326 for the Treatment of Patients with ADPKD
Autosomal dominant polycystic kidney disease (ADPKD) is caused by mutations in either PKD1 or PKD2 genes, which results in kidney cyst formation. Here we report the evaluation of RGLS4326, a novel investigational drug identified to inhibit a target called miR-17, and to slow down kidney cyst growth. We have shown that miR-17 is overproduced in kidneys of ADPKD patients and causes decreased levels of PC1 and PC2 (proteins encoded by PKD1 and PKD2). In studies in animals with PKD, RGLS4326 increased levels of PC1 and PC2, reduced kidney injury marker uNGAL, and inhibited kidney cyst growth. In an ongoing Phase 1b study in patients with ADPKD, four doses of subcutaneous (s.c.) injections every other week of RGLS4326 in the first cohort was well tolerated and showed increases in urinary PC1 and PC2 levels. The study is continuing to enroll ADPKD patients in additional cohorts to evaluate different doses of RGLS4326.