Regulus Initiates Phase I Clinical Study of RG-012, a microRNA Therapeutic in Development for the Treatment of Alport Syndrome

Regulus Therapeutics Inc. RGLS, a biopharmaceutical company leading the discovery and development of innovative medicines targeting microRNAs, announced today that dosing has begun in a first-in-human Phase I clinical study of RG-012, a single stranded, chemically modified oligonucleotide that binds to and inhibits the function of microRNA-21 ("miR-21").  RG-012 is being developed by Regulus in a strategic alliance with Genzyme, a Sanofi company, for the treatment of Alport syndrome, a life-threatening genetic kidney disease with no approved therapy.  The Phase I clinical study is being conducted in the United States as a randomized, double-blind, placebo-controlled, single ascending dose study to evaluate the safety, tolerability and pharmacokinetics of subcutaneous dosing of RG-012 in healthy volunteers. "Advancement of RG-012 into the clinic represents an important achievement under our 'Clinical Map Initiative' and further underscores our focus on discovering and developing novel microRNA therapies for orphan and rare diseases such as Alport syndrome," said Paul Grint, M.D., President and Chief Executive Officer of Regulus.  "We expect that the results from this first-in-human clinical study of RG-012, combined with our learnings from our natural history See full press release