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Alnylam Pharmaceuticals Reports Third Quarter 2013 Financial Results

Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), a leading RNAi therapeutics company, today reported its consolidated financial results for the third quarter 2013, and company highlights.

“This was a quarter of remarkable progress at Alnylam. A key highlight was achievement of positive data from our Phase I clinical trial with ALN-TTRsc in development for the treatment of familial amyloidotic cardiomyopathy. In this study, we demonstrated knockdown of serum TTR of up to 94% and showed that ALN-TTRsc was generally safe and well tolerated, thereby confirming human translation of our proprietary GalNAc-siRNA conjugate delivery platform. As such, we believe these data are very important not only for our ALN-TTRsc program, but for the entirety of our ‘Alnylam 5x15’ pipeline which employs this now clinically validated delivery approach,” said John Maraganore, Ph.D., Chief Executive Officer of Alnylam. “In addition, we were very pleased to complete enrollment in our Phase II trial with patisiran – the recommended International Nonproprietary Name for ALN-TTR02 – in development for the treatment of familial amyloidotic polyneuropathy. We have begun rolling over eligible patients onto the open-label extension study, which will include a number of clinical endpoint measurements with initial data expected to be presented in 2014. We also remain on track to start a Phase III pivotal trial for patisiran in polyneuropathy patients by the end of 2013. In aggregate, we believe our recent advances highlight Alnylam’s unique opportunity for shareholder value creation with a modular and reproducible approach for development and, ultimately, commercialization of innovative medicines for genetically defined diseases.”

“In addition to the progress we made with our patisiran and ALN-TTRsc programs, we advanced multiple additional ‘Alnylam 5x15’ programs this quarter. Notably, we presented key pre-clinical proof-of-concept data with ALN-AT3, an RNAi therapeutic for the treatment of hemophilia, in which we demonstrated that subcutaneous administration of ALN-AT3 can fully normalize thrombin generation in a non-human primate hemophilia ‘inhibitor’ model. We also recently filed a Clinical Trial Application for ALN-AT3 and we expect to initiate a Phase I clinical trial in early 2014,” said Barry Greene, President and Chief Operating Officer of Alnylam. “In addition, we selected GalNAc-conjugate Development Candidates for two programs: ALN-AS1, an RNAi therapeutic targeting aminolevulinate synthase-1 for the treatment of hepatic porphyrias; and ALN-PCSsc, an RNAi therapeutic targeting PCSK9 in development for the treatment of hypercholesterolemia. Further, we made progress on additional ‘5x15’ programs including ALN-CC5, an RNAi therapeutic targeting complement component C5 for the treatment of complement-mediated diseases, and ALN-AAT, an RNAi therapeutic targeting alpha-1-antitrypsin (AAT) in development for the treatment of liver disease associated with AAT deficiency. All told, we’re very pleased with the progress we’re making in executing on our ‘Alnylam 5x15’ product strategy, bringing our innovative medicines to patients.”

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