Regulus announces clinical and regulatory updates from its ADPKD program

Regulus (RGLS) Therapeutics announced positive clinical and regulatory updates from its ADPKD program. The update includes positive topline results from an interim analysis of the fourth cohort of its Phase 1b Multiple Ascending Dose, or MAD, study of farabursen for the treatment of ADPKD and an overview on its recent successful End-of Phase 1 meeting with the FDA. The Phase 1b MAD study is a double-blind, placebo-controlled trial evaluating the safety, tolerability, pharmacokinetics and pharmacodynamics, or PK/PD, of farabursen in adult patients with ADPKD. The study is evaluating farabursen treatment across three different weight-based dose levels and one fixed dose level, including measuring changes in urinary polycystins 1 and 2, htTKV, and overall kidney function. PC1 and PC2 are the protein products of the PKD1 and PKD2 genes and their levels have been shown to inversely correlate with disease severity. In the fourth cohort, 26 subjects received a fixed dose of 300 mg of farabursen every other week for three months. An interim analysis of efficacy data from the first 14 subjects of the cohort demonstrated continued evidence of a mechanistic dose response based on urinary PC1 and PC2 levels as well as a notable reduction in htTKV growth rate. In addition, review of complete safety data from all 26 subjects demonstrated farabursen was well tolerated.