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Regulus Therapeutics Advances Orphan Disease Portfolio For Alport Syndrome

Regulus Therapeutics Inc. (RGLS) said it has advanced its orphan disease portfolio with the nomination of its second microRNA candidate for clinical development, RG-012, an anti-miR targeting microRNA-21 ("miR-21") to treat Alport Syndrome, a life-threatening, genetic kidney disease with no approved therapy.

RG-012 has a very favorable preclinical profile to date which Regulus believes supports clinical studies in man. RG-012 is a potent inhibitor of miR-21 in both in vitro and in vivo preclinical models.

The company said in a statement, "Subcutaneous administration of RG-012 has significantly reduced the rate of renal fibrosis and improved the lifespan of the mice up to 50% in a mouse model of Alport Syndrome, which we believe is a good surrogate for the human disease."

Regulus believes that these preclinical survival results may translate to a similar increased lifespan in human patients. Furthermore, RG-012 has been well tolerated to date with a favorable pharmacokinetic profile that supports the potential for a once/week dosing regimen.

Neil W. Gibson, Ph.D., Regulus' Chief Scientific Officer noted, "We believe that RG-012 may become a transformative treatment for patients with Alport Syndrome, a life-threatening, genetic disease with significant unmet medical need. We are currently performing additional preclinical studies and finalizing development plans for RG-012 and expect to enter clinical development in the first half of 2015."

Regulus is responsible for advancing RG-012 to proof-of-concept. At that stage of development, Regulus' strategic alliance partner, Sanofi, has an exclusive option exercisable after proof-of-concept to assume all costs, responsibilities and obligations for further development and commercialization of RG-012.

Sanofi would reimburse Regulus for a significant portion of its preclinical and clinical development costs and would pay Regulus an option exercise fee, provided Sanofi chooses to exercise its option on RG-012. Regulus is eligible to receive development and commercialization milestone payments and would have an option to co-promote in the U.S. or receive royalty payments in the mid 10% - 20% range.

Alport Syndrome is a genetic condition caused by mutations in the COL4A3, COL4A4, and COL4A5 genes that is characterized by kidney disease, hearing loss, and eye abnormalities.

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