miRagen Therapeutics, Inc, a clinical-stage biopharmaceutical company focused on the discovery and development of RNA-targeted therapies, announced today the initiation of a Phase 1 clinical trial of MRG-110 (S95010) by Servier. MRG-110 is a locked nucleic acid modified oligonucleotide inhibitor of microRNA-92. In preclinical models, MRG-110 appeared to accelerate the formation of new blood vessels, which resulted in improved vascularization and improved functional outcomes. MRG-110 is the third product candidate from miRagen to enter human clinical trials. miRagen has commercialization rights for MRG-110 in the United States and Japan and Servier has rights to commercialize MRG-110 in the rest of the world.
In this clinical trial, MRG-110 will be evaluated for safety and tolerability in a systemic dosing protocol that is intended to support potential further clinical trials that may lead to regulatory approval for the potential treatment of heart failure. Later this year, miRagen plans to sponsor a separate Phase 1 clinical trial in the United States assessing the local and systemic safety and tolerability of MRG-110 after intradermal injection. miRagen recently filed an investigational new drug application, or IND, for this study which it expects to initiate in the first half of this year. In the clinical trial, miRagen plans to also examine several exploratory endpoints that are intended to provide mechanistic proof of concept and biomarker validation to support a global development plan that may include use in patients with high risk for complications after surgical incisions or chronic wounds.
“This clinical development milestone is an important step in our long-standing collaboration with Servier. The aspiration of our collaboration is to address cardiovascular and other diseases that have been difficult to treat with traditional therapeutic approaches,” said miRagen President and CEO William S. Marshall, Ph.D. “Cardiovascular disease is the leading cause of death worldwide, and over a third of the adult U.S. population suffers from at least one form of the disease. We look forward to the next step of development of MRG-110 which should guide how this product candidate may potentially be used as an innovative therapy for patients in need.”
MRG-110 is designed to inhibit the activity of microRNA-92, which has been shown in miRagen’s preclinical studies, and reported in multiple peer reviewed scientific publications, to be a regulator of new blood vessel creation. The Phase 1 trial is designed to evaluate the safety, tolerability, and pharmacokinetics of MRG-110; and to potentially establish the recommended Phase 2 clinical trial dose for the treatment of patients with heart failure. The Phase 1 clinical trial is planned to enroll 49 male subjects, aged 18 to 45. In addition, the Phase 1 clinical trial results will be analyzed for biomarkers that may provide mechanistic proof of concept and support further potential clinical trials of MRG-110 in the treatment of cardiovascular disease and certain other conditions where vascular flow is compromised. Additional details on the ongoing Phase 1 clinical trial can be found at www.clinicaltrials.gov.
Under the terms of the collaboration, Servier will pay miRagen a €3.0 million milestone for the dosing of the first patient in the Phase 1 trial.