New Programs Added to Infectious Disease Pipeline: ALN-HDV and ALN-PDL

We have expanded our infectious disease pipeline with two new RNAi therapeutic programs. First, we are advancing ALN-HDV, an RNAi therapeutic targeting the hepatitis delta viral (HDV) genome in development for the treatment of HDV infection. We are also advancing ALN-PDL, an RNAi therapeutic targeting hepatocyte-expressed programmed death ligand 1 (PD-L1) in development for the treatment of chronic liver infections.

Hepatic infectious diseases, such as HBV and HDV infection, are major global health problems, affecting approximately 400 million and 15 million people worldwide, respectively.  We believe that multiple opportunities exist for RNAi therapeutics to treat hepatic infectious diseases, and our initial focus on HBV will now be expanded to include programs for HDV and liver-specific immune checkpoint blockade by targeting hepatocyte-expressed PD-L1.  In both cases, our approach will employ our Enhanced Stabilization Chemistry (ESC)-GalNAc-conjugate technology, enabling subcutaneous dose administration with high potency and long durability, and a wide therapeutic index.

We are also continuing to advance ALN-HBV, an RNAi therapeutic targeting the hepatitis B viral (HBV) genome for the treatment of HBV infection. We remain on track to select a Development Candidate (DC) in late 2014 and plan to file an Investigational New Drug (IND) application or IND equivalent around year-end 2015.