Regulus begins ATHENA study to monitor changes in renal function in Alport syndrome patients

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Regulus Therapeutics Inc. (NASDAQ:RGLS), a biopharmaceutical company leading the discovery and development of innovative medicines targeting microRNAs, announced today it has initiated its ATHENA natural history of disease study in patients with Alport syndrome, a life-threatening genetic kidney disease with no approved therapy. The ATHENA study is designed to characterize the natural decline of renal function markers such as Glomerular Filtration Rate (“GFR”), creatinine, proteinuria and β-2 microglobulin, in Alport syndrome patients over time. Over the course of two years, Regulus aims to enroll up to 120 Alport syndrome patients who are 16 years and older with a GFR between 30-75ml/min at planned clinical sites in the United States, Australia, Canada and Europe.

The data collected from the ATHENA study will provide much needed information about the changes in renal function over time in Alport syndrome patients, which will inform future clinical development plans of Regulus’ RG-012, a single stranded, chemically modified oligonucleotide that binds to and inhibits the function of microRNA-21 (“miR-21”), currently in development to treat renal dysfunction in Alport syndrome patients.

“Orphan diseases like Alport syndrome are poorly or incompletely understood and we believe that conducting a natural history study is an essential element to facilitate efficient clinical development. In the ATHENA study, we anticipate that prospectively measuring changes in GFR and other renal biomarkers should 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 patients with Alport syndrome,” said Paul Grint, M.D., Chief Medical Officer of Regulus. “The data from ATHENA will be important to the Alport syndrome patient community and Regulus’ future success in treating the disease with RG-012, a key program under our ‘Clinical Map Initiative’.”



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