US-based Regulus Therapeutics has started a Phase I trial of RGLS4326 for the treatment of autosomal dominant polycystic kidney disease (ADPKD).
The first-in-human randomised, double-blind, placebo-controlled, single ascending dose study of RGLS4326 seeks to examine the safety, tolerability, pharmacokinetics, and pharmacodynamics of RGLS4326 administered subcutaneously in healthy volunteers.
The company has also completed dosing of the first cohort of healthy volunteers enrolled in the trial.
Regulus chief research and development officer Dr Timothy Wright said: “Based on the strong preclinical evidence for the role of miR-17 in animal models of PKD and in-vitro testing of human ADPKD cyst cell cultures, we believe that RGLS4326 has great potential as a disease-modifying therapy in ADPKD.
“This programme highlights the speed with which our scientists can progress from oligonucleotide library synthesis to human testing, which in the case of RGLS4326, was less than 24 months.”
Regulus added that RGLS4326 is a new oligonucleotide developed to inhibit miR-17 using a chemistry design to preferentially target the kidney.
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Preclinical studies of the drug have highlighted a reduction in kidney cyst formation, improved kidney weight/body weight ratio, decreased cyst cell proliferation, and preserved kidney function in mouse models of ADPKD.
ADPKD is caused by mutations in the PKD1 or PKD2 genes and is one of the most common human monogenetic disorders.
It is also a leading genetic cause of end-stage renal disease.