Alnylam Pharmaceuticals Reports Second Quarter 2012 Financial Results

Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), a leading RNAi therapeutics company, today reported its consolidated financial results for the second quarter 2012, and company highlights.

“This was an exceptional quarter and recent period for Alnylam, where we reported positive clinical data from several RNAi therapeutic programs. Within our ‘Alnylam 5x15’ product efforts, we showed that ALN-TTR02 achieves rapid, dose-dependent, durable, and specific knockdown of serum transthyretin - the disease causing protein in transthyretin-mediated amyloidosis - after just a single dose. Specifically, we showed transthyretin knockdown of up to 94% with a nearly 80% level of suppression sustained at one month. We believe these data document an unprecedented level of clinical activity for RNAi therapeutics and serve as an important industry milestone in the advancement of this new class of medicines to patients,” said John Maraganore, Ph.D., Chief Executive Officer of Alnylam. “We also reported encouraging clinical data from our Phase I ALN-PCS trial showing statistically significant and durable knockdown of plasma PCSK9 of up to 84% and lowering of LDL cholesterol of up to 50%. Further, we presented key pre-clinical proof of concept data for ALN-AT3, an RNAi therapeutic targeting antithrombin for the treatment of hemophilia, which employs our conjugate delivery platform enabling subcutaneous dose administration. In aggregate, these important accomplishments give us great confidence in the continued execution of our ‘Alnylam 5x15’ product strategy focused on advancement of RNAi therapeutics toward genetically defined targets for diseases where there are limited treatment options for patients and caregivers.”

“In addition to the important advancements made with our ‘Alnylam 5x15’ programs, we also made notable progress with our partnered RNAi therapeutic programs. We reported top-line results from our Phase IIb trial with ALN-RSV01 - an inhaled RNAi therapeutic for the treatment of respiratory syncytial virus infection in lung transplant patients - which we believe provide important evidence for clinical efficacy toward new or progressive bronchiolitis obliterans syndrome, an irreversible and life-threatening complication of infection in these patients. We look forward to presenting additional data from our ALN-RSV01 study and discussing our results with U.S. and European regulatory authorities,” said Barry Greene, President and Chief Operating Officer of Alnylam. “In addition, we are very pleased to have formed a strategic collaboration with Ascletis, a Chinese biopharmaceutical company, for the continued advancement of ALN-VSP, our RNAi therapeutic program for the treatment of liver cancer that has completed a positive Phase I study. We look forward to working with Ascletis in their efforts to advance this medicine into Phase II trials.”

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