Regulus Completes Enrollment for Phase 1b Trial of RGLS8429 in ADPKD

Regulus Therapeutics, a biopharmaceutical firm dedicated to developing novel treatments by targeting microRNAs, has achieved a significant milestone in its clinical research. The company has successfully completed the enrollment of patients for the third group in its Phase 1b Multiple-Ascending Dose (MAD) study. This study is examining the effects of RGLS8429, a new oligonucleotide therapy, on Autosomal Dominant Polycystic Kidney Disease (ADPKD).

ADPKD is a prevalent genetic disorder caused by mutations in either the PKD1 or PKD2 genes, leading to the formation of numerous cysts in the kidneys and potentially other organs. This condition is a significant cause of end-stage renal disease, affecting an estimated 4 to 7 million people worldwide, with around 160,000 diagnosed in the United States.

RGLS8429 is designed to inhibit miR-17, a microRNA implicated in the disease process, and has shown promising results in preclinical trials. The drug has demonstrated improvements in kidney function and size, and it has a favorable safety profile. The Phase 1b MAD study is a rigorous, double-blind, placebo-controlled trial that is assessing the drug's safety, tolerability, pharmacokinetics, and pharmacodynamics in adult ADPKD patients. The study is divided into three cohorts, each receiving different weight-based dosages of RGLS8429 or a placebo.

The third cohort is receiving a dosage of 3 mg/kg every other week for a duration of three months. Additionally, a fourth cohort has been introduced to receive a fixed dose of the drug, allowing for a comparison of biomarker and safety data with the weight-based dosing approach.

Jay Hagan, CEO of Regulus Therapeutics, expressed enthusiasm about the progress, stating that the team is eagerly preparing for the upcoming phases of the study. The company anticipates releasing preliminary data from the second cohort in the first quarter of 2024, with data from the third cohort expected to follow in mid-2024.

Regulus Therapeutics, headquartered in San Diego, California, has a robust pipeline of microRNA-targeting drugs and a strong intellectual property portfolio in this field. The company's commitment to innovation in drug discovery and development is evident in its pursuit of therapies for ADPKD and other diseases.