Regulus Therapeutics Announces Positive Clinical and Regulatory Updates from its Autosomal Dominant Polycystic Kidney Disease (ADPKD) Program for Farabursen (RGLS8429)
Topline data from an interim analysis of the fourth cohort of its Phase 1b Multiple-Ascending Dose (MAD) clinical trial showed continued mechanistic dose response
Exploratory results of imaging-based biomarkers continued to show reduction in height-adjusted total kidney volume (htTKV) growth rate
Successful End-of-Phase 1 meeting with the U.S. Food and Drug Administration (FDA) with agreement on key components of a Phase 3 single pivotal trial for potential Accelerated Approval
Excerpt from the Press Release:
SAN DIEGO, Jan. 29, 2025 /PRNewswire/ — 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 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 (MAD) study of farabursen (RGLS8429) 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 (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 (PC1 and PC2), 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.
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