FRANKFURT, Germany and BOULDER, Colo., July 28, 2020 (GLOBE NEWSWIRE) -- Goethe University, University Hospital Frankfurt and miRagen Therapeutics, Inc. (NASDAQ:MGEN), a clinical-stage biopharmaceutical company focused on the discovery and development of RNA-targeted therapies, today announced that preliminary results from a Phase 1 trial detailing the pharmacodynamic activity of MRG-110 has been published in the peer-reviewed journal Nucleic Acid Therapeutics.
“We are pleased to have these Phase 1 mechanism of action results for MRG-110 published in Nucleic Acid Therapeutics,” said William S. Marshall, Ph.D., President and CEO of miRagen Therapeutics, Inc. “These data show that a single systemic dose of MRG-110 reduced detectable miR-92a levels in the peripheral blood of humans and led to the regulation of several well established miR-92a target genes.”
“The preliminary characterization of MRG-110 pharmacodynamic activity in the peripheral blood of humans provides important insight into the development of potential blood borne mechanistic biomarkers for miR-92a inhibition,” added Prof. Dr. Stefanie Dimmeler, Director of the Institute for Cardiovascular Regeneration at the Center for Molecular Medicine, Goethe University Frankfurt. “We believe these data provide the basis for further development of readily monitorable biomarkers that can be employed in future clinical trials.”
The article, titled “Efficiency and Target Derepression of Anti-miR-92a: Results of a First in Human Study,” reports mechanistic results from a single ascending dose, double-blind, placebo-controlled, randomized Phase 1 clinical trial. The trial was a study where MRG-110 was administered systemically by intravenous dosing. miR-92a levels were measured in whole blood, circulating endothelial cells, and circulating extracellular vesicles before and after MRG-110 administration. miR-92a levels were found to be significantly reduced in all three of the sample types tested. Importantly, two miR-92a target genes were derepressed after treatment, indicating the intended mechanism of action of MRG-110 in normal healthy volunteers. These data may identify a strategy for measuring pharmacodynamic activity of MRG-110 in peripheral blood in future clinical trials. This would allow for monitoring the intended mechanism of action in blood samples during the conduct of clinical trials in patients, thereby providing an enhanced degree of confidence that the product candidate is affecting the biological pathway that is intended.
Work reported in the article was the result of a collaboration between Goethe University, University Hospital Frankfurt, miRagen Therapeutics and Les Laboratories Servier. Financial support for the studies was also provided by the German Centre for Cardiovascular Research (DZHK).