Regulus Therapeutics begins RG-012 Phase I clinical study for treatment of Alport syndrome

Regulus Therapeutics Inc. (NASDAQ: 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 of disease study called ATHENA, will provide the clinical basis for the design of a proof-of-concept study to monitor the therapeutic effect of RG-012 in Alport syndrome patients."

Seng Cheng, Ph.D., Head of Research and Early Development of Rare Diseases at Genzyme, a Sanofi company, added, "MicroRNA therapeutics represent a novel and exciting new approach for the treatment of rare diseases and the progress made by Regulus represents a significant milestone for patients with Alport syndrome, a devastating and debilitating disease. There are few therapeutic treatment options for patients with Alport syndrome and we believe that RG-012 entering the clinic represents an opportunity to make a significant step forward. With the initiation of this study, Genzyme is excited to continue its leadership in rare diseases and commitment to innovation for diseases with unmet medical need."

In addition to the Phase I clinical study, Regulus is currently enrolling Alport syndrome patients in a natural history of disease study called ATHENA. With this study, which has thirteen clinical sites worldwide, Regulus aims to learn more 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. In addition, Regulus expects that many patients enrolled in ATHENA will be eligible to enroll in a Phase II proof-of-concept study, which Regulus expects to initiate under its 'Clinical Map Initiative' following the conclusion of the Phase I clinical study announced today.

Regulus is responsible for advancing RG-012 to proof-of-concept. At that stage of development, Sanofi has an exclusive option exercisable after proof-of-concept to assume worldwide development and commercialization of RG-012 subject to a co-promote right in the United States by Regulus.