Regulus reports positive phase 1b ADPKD trial results
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SAN DIEGO – Regulus (NASDAQ:RGLS) Therapeutics Inc. (NASDAQ:RGLS), a biopharmaceutical company, announced encouraging preliminary results from the second cohort of its Phase 1b clinical trial of RGLS8429, a treatment for Autosomal Dominant Polycystic Kidney Disease (ADPKD), on Monday.
The study demonstrated a dose response based on urinary biomarkers and exploratory imaging-based biomarkers, with the most significant reductions in kidney volume observed in patients with the highest increases in polycystins 1 and 2 (PC1 and PC2).
The Phase 1b trial is a double-blind, placebo-controlled study designed to assess the safety, tolerability, pharmacokinetics, and pharmacodynamics of RGLS8429. The medication is being evaluated at various weight-based dose levels, with the second cohort involving 14 patients receiving a 2 mg/kg dose of RGLS8429 or placebo every other week for three months. The drug was well-tolerated with no safety concerns noted.
Urinary measurements of PC1 and PC2 showed greater biological activity at the 2 mg/kg dose compared to both the 1 mg/kg dose and placebo, particularly after three months of treatment. Three patients with the highest increases in PC1 and PC2 experienced reductions in height-adjusted total kidney volume (htTKV) greater than 4%, along with a decrease in total kidney cyst volume (TKCV).
The company plans to expand the sample size for the fourth cohort to up to 30 patients to further examine the potential impact on cystic volume in patients with ADPKD. This decision follows the positive data from the second cohort, which suggests a potential effect on htTKV and TKCV that will be explored at higher doses in the upcoming cohorts. Topline data from the third cohort is anticipated in mid-2024, with the fourth cohort’s screening set to begin in the second quarter of 2024.
Alan Yu, M.B., B.Chir, from the University of Kansas Medical Center, highlighted the biomarker data indicating increased activity of RGLS8429 at the 2 mg/kg dose. Preston Klassen, M.D., President and Head of Research & Development at Regulus, expressed optimism about the drug’s potential efficacy in ADPKD.
Regulus also confirmed a constructive meeting with the FDA regarding an accelerated approval pathway based on a single Phase 2 study of RGLS8429 for ADPKD treatment. The company reported having approximately $23.8 million in cash and cash equivalents as of December 31, 2023.
The information for this article is based on a press release statement from Regulus Therapeutics Inc .
