Regulus Therapeutics Inc., a biopharmaceutical company leading the discovery and development of innovative medicines targeting microRNAs, announced that it has identified a third microRNA target in its alliance with GlaxoSmithKline (GSK), triggering a preclinical milestone payment from GSK. This selection represents another significant milestone for Regulus in its microRNA alliance with GSK, which is focused on the discovery and development of compounds targeting four different microRNAs in inflammation and HCV infection.

“GSK has been a committed and valuable partner, providing Regulus with their vast expertise in preclinical models and an understanding of clinical pathways and commercial opportunities,” said Neil W. Gibson, Ph.D., chief scientific officer of Regulus. “The alliance has benefitted from Regulus’ proprietary technology platform and leading knowledge base of microRNA biology.”

Under the 2008 alliance with GSK, Regulus has the potential to earn more than $600 million in payments across all programmes including license and milestone payments, and tiered royalties up to double digits on worldwide sales of products resulting from the alliance. Regulus is responsible for identifying compounds that target four microRNAs through to completion of proof of concept trials. GSK has an exclusive option to license the worldwide development and commercialization rights under each target microRNA.

The discovery of microRNA in humans during the last decade is potentially one of the most exciting scientific breakthroughs in recent history. microRNAs are small RNA molecules, typically 20 to 25 nucleotides in length, that do not encode proteins but instead regulate gene expression. More than 700 microRNAs have been identified in the human genome, and over one-third of all human genes are believed to be regulated by microRNAs. A single microRNA can regulate entire networks of genes. As such, these molecules are considered master regulators of the human genome. microRNAs have been shown to play an integral role in numerous biological processes, including the immune response, cell-cycle control, metabolism, viral replication, stem cell differentiation and human development. Most microRNAs are conserved across multiple species, indicating the evolutionary importance of these molecules as modulators of critical biological pathways. Indeed, microRNA expression, or function, has been shown to be significantly altered in many disease states, including cancer, heart failure and viral infections. Targeting microRNAs with anti-miRs, antisense oligonucleotide inhibitors of microRNAs, or miR-mimics, double-stranded oligonucleotides to replace microRNA function opens potential for a novel class of therapeutics and offers a unique approach to treating disease by modulating entire biological pathways.

Regulus Therapeutics is a biopharmaceutical company leading the discovery and development of innovative medicines targeting microRNAs. Regulus is using a mature therapeutic platform based on technology that has been developed over 20 years and tested in more than 5,000 humans.