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Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), a leading RNAi therapeutics company, today reported its consolidated financial results for the fourth quarter and full year 2012, and company highlights.
“2012 was a remarkable year of clinical achievement for Alnylam and for the advancement of RNAi therapeutics. Importantly, we believe that data from our ALN-TTR02 program showing up to a 94% knockdown of a disease-causing protein and data from our PCSK9 program showing up to a 50% decrease in LDL cholesterol served as unambiguous proof points that RNAi works in man. Moreover, we have executed on a clear product strategy we call ‘Alnylam 5x15,’ where we are advancing RNAi therapeutics toward genetically defined targets for diseases with high unmet need and where we intend to directly commercialize certain core programs from this effort in major parts of the world,” said John Maraganore, Ph.D., Chief Executive Officer of Alnylam. “Specifically, in this past quarter and recent period we continued to execute on our pipeline efforts by enrolling patients in our Phase II trial for ALN-TTR02 and obtaining approval to initiate a Phase I study with ALN-TTRsc, a subcutaneously administered RNAi therapeutic for the treatment of ATTR. In addition, we presented key pre-clinical proof-of-concept data for several other programs, including ALN-AT3 for hemophilia, ALN-TMP for hemoglobinopathies, and ALN-AAT for liver disease associated with alpha-1 antitrypsin deficiency. We have also advanced a new program, ALN-AS1 for the treatment of acute intermittent porphyria, an ultra-rare orphan disease. These important accomplishments give us great confidence in the continued execution of our ‘Alnylam 5x15’ product strategy, where we believe we can make a difference in the lives of patients and deliver value to our shareholders.”
“In addition to the scientific and clinical progress we have made in the past quarter, we have also made important progress in our business and corporate development efforts. Over the past year, our new collaborations have resulted in over $75 million in realized funding,” said Barry Greene, President and Chief Operating Officer of Alnylam. “Recently, we formed a strategic alliance with Genzyme to advance ALN-TTR in Japan and the broader Asian-Pacific region, and earlier this week we announced a worldwide strategic alliance with The Medicines Company to advance ALN-PCS for the treatment of hypercholesterolemia. Both Genzyme and The Medicines Company are excellent partners for Alnylam with shared commitments to develop innovative medicines. Related to our partnered program efforts, Cubist has elected not to opt-in to continued development of our respiratory syncytial virus program; we plan to seek another partner to advance the program into a Phase III trial. In sum, we believe our corporate development progress enables our continued focus and execution on our ‘Alnylam 5x15’ product development and commercialization strategy with retention of value in our core programs in major markets.”