Alnylam plans clinical trials for RNAi therapeutic

ALN-AT3, Alnylam Pharmaceuticals' ($ALNY) RNAi therapeutic in development for the treatment of hemophilia, has shown positive results in a preclinical study in nonhuman primates by increasing thrombin generation as much as fourfold. Thrombin is one of a number of factors involved in the blood clotting process. These results support the company's plans for clinical trials next year.

In the study, presented at the 54th American Society of Hematology annual meeting, ALN-AT3, dosed weekly just under the skin, knocked down the levels of the naturally occurring protein antithrombin in the blood, boosting the levels of thrombin.

ALN-AT3, which uses the company's GalNAc-siRNA delivery platform, should enter clinical trials next year, along with ALN-TTRsc, in development for the treatment of transthyretin-mediated amyloidosis (ATTR). The company's aim, through its "Alnylam 5x15" product strategy, is to have 5 programs in clinical development by the end of 2015.

In November, after a tussle that entered the courts, Alnylam and Tekmira Pharmaceuticals ($TKMR) shook hands and settled litigation in a $75 million deal over Tekmira's lipid nanoparticle (LNP) technology.

- read the press release
- see the abstract (pdf)