Regulus Therapeutics announced that the FDA has granted Orphan Drug designation to RG-012, a single stranded, chemically modified oligonucleotide that binds to and inhibits the function of microRNA-21 for the treatment of Alport syndrome.
RG-012 has demonstrated potent inhibition of miR-21 in vitro and in vivo, a decrease in the rate of progression of renal fibrosis, an increase in the lifespan of mice by up to 50%, and a favorable pharmacokinetic profile that supports the potential for a once per week dosing regimen.
Regulus plans to initiate a natural history of disease study to gather further information about the progression of Alport syndrome. In addition, Regulus also expects to initiate a Phase 1 clinical study of RG-012 for the treatment of Alport syndrome in the first half of 2015. Currently there are no approved treatments for Alport syndrome.
For more information visit RegulusRx.com.