Regulus' RG-012 receives orphan medicinal product designation in EU for treatment of Alport syndrome

Mar 25 2015

Regulus Therapeutics Inc. (NASDAQ: RGLS), a biopharmaceutical company leading the discovery and development of innovative medicines targeting microRNAs, announced today that the European Commission has granted orphan medicinal product designation for RG-012, a single stranded, chemically modified oligonucleotide that binds to and inhibits the function of microRNA-21 ("miR-21") for the treatment of Alport syndrome, a life-threatening genetic kidney disease with no approved therapy. In July 2014, the U.S. Food & Drug Administration granted orphan drug designation to RG-012 for the treatment of Alport syndrome.

"We are pleased to have received orphan medicinal product designation in the European Union for RG-012, a key microRNA therapeutic program under our 'Clinical Map Initiative'," said Paul Grint, M.D., Chief Medical Officer of Regulus. "Alport syndrome is a life threatening disease and patients have very limited treatment options because there is currently no approved therapy. We believe that RG-012 represents an opportunity to make a significant impact in the lives of patients with Alport syndrome and we look forward to advancing this program into the clinic."

Regulus is currently enrolling patients in a natural history of disease study called ATHENA to gather information about the changes in renal function over time in patients with Alport syndrome. Data from the ATHENA study will provide the clinical basis for the design of a Phase II study to monitor the therapeutic effect of RG-012 on the decline in renal function and time to end-stage renal disease in Alport syndrome patients. Under Regulus' 'Clinical Map Initiative', the company expects to initiate a Phase I study evaluating RG-012 in healthy volunteers in the first half of 2015 and a Phase II proof-of-concept study thereafter.

Orphan drug designation by the European Commission provides regulatory and financial incentives for companies to develop and market therapies that treat a life-threatening or chronically debilitating condition affecting no more than five in 10,000 persons in the European Union (EU), and where no satisfactory treatment is available. In addition to a 10-year period of marketing exclusivity in the EU after product approval, orphan drug designation provides incentives for companies seeking protocol assistance from the European Medicines Agency during the product development phase, and direct access to the centralized authorization procedure.