Regulus Therapeutics Inc. (RGLS) announced that it has nominated RG-012 for clinical development to treat patients suffering from alport syndrome, an orphan indication. The syndrome is a genetic kidney disorder. Currently there are no approved therapies to treat patients suffering from this life threatening rare disease which affects 1 in 50,000 newborns according to data released by the National Institutes of Health.
RG-102, an anti-miR targeting micro ribo nucleic acid or RNA -21, has performed well in pre-clinical studies and is expected to move into clinical development in the first half of next year following its nomination by the company. RG-102 is the second microRNA candidate to be nominated for clinical development by the company following RG-101 (chronic hepatitis C virus).
The biopharmaceutical company is looking to advance its “road to clinic” strategy through these nominations. Through the scheme, launched in Feb 2013, Regulus intends to develop its microRNA therapeutic pipeline in the direction of the clinic.
Regulus has an agreement with Sanofi (SNY) on RG-102. Regulus has agreements with many other companies to advance its pipeline. Regulus is leaving no stone unturned to develop its pipeline. In a move in that direction, the company appointed Martin Beaulieu as the head of its research and development wing – microRNA biomarkers – earlier in the month. The appointment of Beaulieu is expected to be beneficial to Regulus since he is vastly experienced in the fields of diagnostic assay development and biomarker research.
Regulus currently carries a Zacks Rank #3 (Hold). Some better-ranked biopharma stocks include Alexion Pharmaceuticals, Inc. (ALXN) and Gilead Sciences, Inc. (GILD). While Alexion carries a Zacks Rank #1 (Strong Buy), Gilead holds a Zacks Rank #2 (Buy).
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