MINNEAPOLIS—As the year comes to a close, a new collaboration is underway between Bio-Techne Corporation and Regulus Therapeutics in which Bio-Techne will support Regulus with biomarker analysis for the clinical development of RGLS4326 against autosomal dominant polycystic kidney disease (ADPKD). Financial details were not disclosed.
"Regulus is pleased to enter this collaboration with Bio-Techne," said Jay Hagan, CEO of Regulus. "We recently initiated the dosing of patients in our Phase 1b ADPKD study, and our ability to determine PC1 and PC2 levels via urinary exosomes is intended to advance our understanding of the potential differentiation of RGLS4326 as a disease-modifying treatment for ADPKD patients. We are excited to collaborate with Bio-Techne and employ their expert diagnostic capabilities towards generating this important clinical data."
According to the Genetic and Rare Disease Information Center, part of the National Center for Advancing Translational Sciences branch of the U.S. National Institutes of Health, ADPKD “is a genetic disorder characterized by the growth of numerous cysts in the kidneys. Symptoms vary in severity and age of onset, but usually develop between the ages of 30 and 40. ADPKD is a progressive disease and symptoms tend to get worse over time … ADPKD is most often caused by changes in the PKD1 and PKD2 genes, and less often by changes in the GANAB and DNAJB11 genes. It is inherited in a dominant pattern. Treatment for ADPKD involves managing the symptoms and slowing disease progression.”
RGLS4236 is a first-in-class, anti-miR-17 therapy being evaluated in a Phase 1b, open-label, multiple-dose study in up to three cohorts of patients with ADPKD to determine the compound's safety, pharmacokinetics and pharmacodynamic effects on the levels of polycystin 1 (PC1) and polycystin 2 (PC2). A mutation in the PKD genes cause ADPKD patients to present with low levels of PC1 and PC2, and this trial aims to determine whether different dose levels of RGLS4326 can increase PC1 and PC2 levels in patients. Regulus expects results from the first cohort, which will enroll up to nine patients, should be available by the end of the first quarter of 2021.
For this collaboration, Regulus is making use of two Bio-Techne product lines to support clinical patient testing: ProteinSimple and Exosome Diagnostics. Regulus hopes to develop high-performance biomarker assays to aid in assessing PC1 and PC2 levels using the fully automated Simple Western instrument as a read-out.
Bio-Techne's ProteinSimple brand covers a number of protein analysis solutions, which the company says offers “efficient and streamlined options for fully automated CE-Western blot, multiplexed ELISA workflow and tools to assess the identity, purity and heterogeneity profiles of therapeutic proteins.” Exosome Diagnostics specializes in the development of liquid biopsy-based diagnostics, isolating and analyzing exosomes to measure RNA, cell-free DNA and proteins in an effort to identify biomarkers, according to Bio-Techne.
"Our Exosome Diagnostics technology in combination with the Simple Western platform are powerful tools in biomarker discovery and assay development processes," Chuck Kummeth, president and CEO of Bio-Techne, commented in a statement. "Regulus adds to a large list of cutting-edge biopharmaceutical companies leveraging Bio-Techne's broad portfolio of life-science tools to enable scientific discoveries and drug discovery efforts."