Regulus(US:RGLS) Prnewswireยท2025-01-29 12:00Core Insights - Regulus Therapeutics Inc. announced positive clinical and regulatory updates from its ADPKD program, including favorable topline results from an interim analysis of the fourth cohort of its Phase 1b Multiple Ascending Dose (MAD) study of farabursen (RGLS8429) [1][4] - The company successfully held an End-of-Phase 1 meeting with the FDA, achieving alignment on key components for a Phase 3 pivotal trial aimed at potential Accelerated Approval [1][4] Clinical Trial Details - The Phase 1b MAD study is a double-blind, placebo-controlled trial assessing the safety, tolerability, pharmacokinetics, and pharmacodynamics of farabursen in adult patients with ADPKD [2] - The fourth cohort involved 26 subjects receiving a fixed dose of 300 mg of farabursen every other week for three months, showing a mechanistic dose response and a reduction in height-adjusted total kidney volume (htTKV) growth rate [3][5] - Safety data from all subjects indicated that farabursen was well tolerated [3][9] Regulatory Progress - The End-of-Phase 1 meeting with the FDA confirmed the acceptability of the program's chemistry, manufacturing, and controls (CMC), non-clinical, and clinical pharmacology plans [4] - The trial design for the Phase 3 study includes a 12-month htTKV endpoint for potential Accelerated Approval and a 24-month eGFR endpoint for potential Full Approval [5][6] Biomarker and Efficacy Insights - Exploratory results indicated a notable impact on urinary polycystin levels and htTKV growth rate, consistent across different imaging classes and PKD1 mutations [5][6] - The data suggest a high probability of success in meeting or exceeding targeted htTKV efficacy thresholds [5] Company Overview - Regulus Therapeutics Inc. focuses on developing innovative medicines targeting microRNAs, with a strong pipeline and intellectual property in the microRNA field [10] - The company is headquartered in San Diego, CA, and is publicly traded on Nasdaq under the ticker RGLS [10]