Sanofi-Aventis, Regulus Form Alliance to Develop microRNA Therapies
The alliance is valued at potentially over $750 million, and includes a $25 million upfront fee, among other milestones and research payments and potential royalties. Alnylam and Isis will each receive a payment of $1.8 million from Regulus. ALNY shares rose about 1% and ISIS jumped some 2%.
The alliance will initially focus on the therapeutic area of fibrosis. Regulus and Sanofi will collaborate on up to four microRNA targets, including Regulus' lead fibrosis program targeting microRNA-21. Sanofi is getting worldwide, exclusive licenses for the four targets, and opt-in rights in future microRNA programs.
"Master Maestros" of the Genome
microRNAs are a class of small RNA molecules discovered this decade that scientists call the "master maestros" of the genome. A single microRNA has the ability to orchestrate the expression of broad networks of genes instrumental in normal cell function. More than one-third of all human genes are believed to be regulated by microRNA, and when they are disregulated, they disrupt the normal function of cells and lead to disease.
Targeting disregulated microRNAs can re-normalize the genetic pathways and thus halt the disease process. This opens the possibility of a novel class of therapeutics and a unique approach to treating disease by modulating entire biological pathways. microRNA function has been shown to be significantly altered in many disease states, including cancer, heart failure and viral infections. Regulus has demonstrated that modulating microRNAs can effectively regulate disease pathways and produce therapeutically beneficial effects.
"microRNAs are believed to be extremely important in human development and physiology. Together with Regulus we will develop therapeutics which could potentially open a new paradigm in the treatment of major diseases and could offer an attractive new therapeutic approach for patients," Marc Cluzel, executive vice president, research & development at Sanofi-Aventis, said in a statement.
Regulus is looking into several treatment areas including hepatitis C infection, cardiovascular disease, fibrosis, oncology, immuno-inflammatory diseases, and metabolic diseases. In 2008, it also entered into a major alliance with GlaxoSmithKline (GSK) microRNA therapies for immuno-inflammatory diseases, which was expanded in 2010 to treatment of Hepatitis C Viral infection.