Regulus Therapeutics Inc. (Nasdaq: RGLS), a biopharmaceutical company focused on the discovery and development of innovative medicines targeting microRNAs (the “Company” or “Regulus”), today announced it has completed enrollment in the third cohort of patients in the Phase 1b MAD study of RGLS8429 for the treatment of ADPKD.
“The completion of our third and final randomized placebo-controlled cohort in the Phase 1b MAD study is an exciting step for Regulus, and we are happy to reach this stage of development for RGLS8429,” said Jay Hagan, CEO of Regulus. “Our team is motivated and gearing up for 2024 when we expect topline data from our second cohort in Q1 2024, topline data from our third cohort in mid-2024, and the initiation of the fourth cohort, an open label fixed dose, of RGLS8429.”
The Phase 1b MAD study is a double-blind, placebo-controlled trial evaluating the safety, tolerability, pharmacokinetics and pharmacodynamics (PK/PD) of RGLS8429 in adult patients with ADPKD. The study will evaluate RGLS8429 treatment across three different weight-based dose levels, including measuring changes in polycystins, height-adjusted total kidney volume (htTKV), cyst architecture, and overall kidney function. The third cohort is being dosed at 3 mg/kg of RGLS8429 or placebo every other week for three months. The protocol was amended to include a fourth cohort of subjects who will receive an open label fixed dose of RGLS8429 to compare biomarker and safety data to the weight-based dosing.