December 15, 2014
By Mark Terry, BioSpace.com Breaking News Staff
Alnylam Pharmaceuticals , headquartered in Cambridge, Mass., announced Friday its new pipeline growth strategy, which will split into three units. Built on Alnylam’s RNAi therapeutics technology, the three Strategic Therapeutic Areas (STArs) include Genetic Medicines, Cardio-metabolic Disease, and Hepatic Infectious Disease.
The company’s technology includes its Enhanced Stabilization Chemistry (ESC)-GalNAc conjugate technology. GalNAc-siRNA conjugates are Alnylam’s proprietary delivery platform that targets delivery of RNAi therapeutics to hepatocytes via uptake by the asialoglycoprotein receptor. The bottom line is the technology allows subcutaneous dosing with increased potency, greater durability, and a wide therapeutic index.
“Based on a wealth of growing pre-clinical and clinical data, we now believe that the breadth of Alnylam’s opportunities for RNAi therapeutics extends beyond the original framework of our ‘Alnylam 5x15’ strategy,” said company CEO John Maraganore in a statement. “Accordingly, we are excited to announce an evolution in our growth strategy with a focus on development and commercialization of RNAi therapeutics in three Strategic Therapeutic Areas (STArs): Genetic Medicines, Cardio-metabolic Disease, and Hepatic Infectious Disease.”
In the Genetics Medicine STAr, the company will focus on its pipeline of RNAi compounds for rare diseases. Included in this pipeline are patisiran and revusiran, investigation therapeutics for the treatment of transthyretin-mediated amyloidosis (ATTR). They are both currently in Phase 3 clinical trials dubbed APOLLO and ENDEAVOUR, respectively. The company also has ALN-AT3, a therapeutic for hemophilia and rare bleeding disorders, with positive initial Phase 1 data.
In Cardio-metabolic Disease, the company’s pipeline is focused on ALN-PCSsc, an investigation RNAi therapeutic that targets PCSK9 for the treatment of hypercholesterolemia. This is in collaboration with The Medicines Company. The compound recently entered a Phase 1 clinical study, with initial data expected mid-2015. The company has additional compounds in this category for the treatment of dyslipidemias, hypertriglyceridemia and hypertension.
In the Hepatic Infectious Disease STAr, Alnylam is pushing ALN-HBV for the treatment of hepatitis B virus (HBV) and ALN-HDV in the treatment of hepatitis D virus. The company plans to file an investigational new drug (IND) application, or IND equivalent, for the ALN-HBV in late 2015. Alnylam also plans to look for strategic partnerships for its Hepatic Infectious Disease programs.
“Across our three STArs,” said Maraganore in a statement, “we believe that we can address major unmet needs in a wide range of diseases with high-impact, differentiated medicines, and continue to build what we believe to be one of the most robust pipelines in biotech. By executing this strategy, we believe we have the potential to make a meaningful difference in the lives of patients, and maximize value for shareholders.”
