miRagen Therapeutics Receives Orphan Drug Designation for MGN-4893 for the Treatment of Polycythemia Vera

Drug candidate targets microRNA-451, a recently discovered key regulator of blood cell development

BOULDER, Colo.--(BUSINESS WIRE)--miRagen Therapeutics, Inc., a privately-held biopharmaceutical company focused on improving patients’ lives by developing innovative microRNA (miRNA)-based therapeutics for cardiovascular and muscle disease, today announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to the company’s compound MGN-4893 for the treatment of polycythemia vera, a myeloproliferative disease characterized by an overabundance of blood cells and platelets in the body. MGN-4893 targets microRNA-451, which has been demonstrated in recent studies to play a crucial role in the regulation of blood cell development. miRagen is currently conducting pre-clinical studies and expects to initiate human clinical trials in 2012.

“We are very pleased that the FDA has concluded our drug candidate MGN-4893 for the treatment of polycythemia vera meets that standard and look forward to advancing the compound into human clinical trials.”

“One of the essential requirements in the orphan drug application process that companies must meet is the demonstration of promise,” said William S. Marshall, Ph.D., President and CEO of miRagen Therapeutic, Inc. “We are very pleased that the FDA has concluded our drug candidate MGN-4893 for the treatment of polycythemia vera meets that standard and look forward to advancing the compound into human clinical trials.”

The FDA grants orphan drug designation to drugs and drug candidates developed for the intended treatment of rare diseases or conditions, specifically those that affect less than 200,000 Americans. Orphan status provides certain incentives, including tax credits and a seven-year marketing exclusivity, should a sponsor be successful in receiving marketing approval.

A potentially fatal disorder, polycythemia vera affects fewer than 100,000 people in the United States and is implicated in the development of heart failure, thrombosis, heart attack and stroke.

About microRNAs: MicroRNAs have emerged as an important class of small RNAs encoded in the genome. They act to control the expression of sets of genes and entire pathways and are thus thought of as master regulators of gene expression. Recent studies have demonstrated that microRNAs are responsible for many diseases. Because they are single molecular entities that dictate the expression of fundamental regulatory pathways, microRNAs represent potential drug targets for controlling many biologic and disease processes.

About miRagen Therapeutics: miRagen Therapeutics, Inc., was founded in 2007 to develop innovative microRNA-based therapeutics for cardiovascular and muscle disease. Only recently discovered, microRNAs are short, single-stranded RNA molecules encoded in the genome that regulate gene expression and play a vital role in influencing cardiovascular and muscle disease. Cardiovascular disease is the leading cause of death globally and represents an enormous burden on global healthcare systems. Principally funded through venture capital investments, miRagen combines world recognized leadership in cardiovascular medicine with unprecedented in-house expertise in microRNA biology and chemistry. For more information, please visit www.miragentherapeutics.com.