Regulus and Alport Syndrome Foundation Raise Awareness of Life-Threatening, Genetic Kidney Disease

In Press Release by Cameron


March 25, 2014

Regulus Therapeutics Inc., a biopharmaceutical company leading the discovery and development of innovative medicines targeting microRNAs, and the Alport Syndrome Foundation (“ASF”), a non-profit corporation, announced today they are working together to raise awareness of Alport Syndrome Foundation, a life-threatening genetic kidney disease driven by genetic mutations with no approved therapy.

This month, Regulus will join the Alport Syndrome Foundation in observing National Kidney Month and the first ever Alport Syndrome Awareness Month.  As part of that effort, Regulus hosted an Alport Syndrome awareness day at Regulus’ corporate headquarters in San Diego, CA. Alport Syndrome patients Sharon Lagas and Ryan Landwehr were invited to provide Regulus employees with patient perspectives on the impacts of living with Alport Syndrome.  Additionally, Regulus will serve as a Spirit of Action sponsor of the Alport Syndrome Foundation 5K for Healthy Kidneys on Sunday, March 30th, 2014 in Tempe, Arizona.  For more information, please

“We are grateful for the support from Regulus and know that it will take patients working with companies like Regulus to advance the search for potential Alport Syndrome treatment options,” said Sharon Lagas, President of the Alport Syndrome Foundation.

“Partnerships are critical to advancing scientific research for orphan diseases such as Alport Syndrome,” said Kleanthis G. Xanthopoulos, Ph.D., President and CEO of Regulus. “We are hopeful that our relationship with the Alport Syndrome Foundation will advance our understanding of the disease and believe that ASF will serve as an invaluable resource to us as we develop RG-012, an anti-miR targeting microRNA-21 for the treatment of Alport Syndrome.  We are excited to initiate a natural history of disease study for RG-012 in the third quarter this year and expect to initiate a Phase I clinical study of RG-012 in the first half of 2015.”

Alport Syndrome is a genetic condition caused by mutations in the COL4A3, COL4A4 or COL4A5 genes which impacts the body’s ability to create a specific type of collagen highly expressed in the kidney and essential to normal kidney structure.  In the absence of this type of collagen, the kidneys are unable to effectively filter toxins and waste products, resulting in end-stage renal disease. Lack of this special type of collagen can also result in hearing loss or effects on vision. Alport Syndrome is considered an orphan disease with no approved therapy.

Regulus is currently developing an innovative new therapy for the treatment of Alport Syndrome.  The company recently nominated a microRNA candidate to enter clinical development, RG-012, an anti-miR targeting microRNA-21 (“miR-21”) for the treatment of Alport Syndrome. Preclinical studies have demonstrated that RG-012 is a potent inhibitor of miR-21 in both in vitro and in vivo models and that subcutaneous administration of RG-012 has significantly decreased the rate of renal fibrosis and increased the lifespan of the mice in the studies up to 50%.  RG-012 has been well tolerated to date with a favorable pharmacokinetic profile that supports the potential for a once/week dosing regimen.

See the full release here.