Alnylam Pharmaceuticals, Inc. is announcing the addition of a new program to the company’s genetic medicines pipeline, ALN-F12, an investigational RNAi therapeutic targeting F12 for the treatment of HAE. Pre-clinical data for ALN-F12 were presented at the American Academy of Allergy, Asthma & Immunology (AAAAI) Annual meeting, held March 4-7, 2016.
Pre-clinical data showed that administration of ALN-F12 resulted in dose-dependent reduction of vascular permeability in two different mouse models of bradykinin-driven vascular leakage, demonstrating that suppression of F12 mRNA has the potential to mitigate excess bradykinin stimulation. Further, in non-human primates, a single subcutaneous dose of ALN-F12 at 3 mg/kg resulted in potent and durable knockdown of serum FXII of greater than 85 percent, with knockdown of over 50 percent sustained out to three months following administration.
Alnylam believes these pre-clinical data demonstrate the potential of ALN-F12 as a promising approach for prophylaxis of HAE attacks.