Alnylam Provides Key 2014 Goals For RNAi Therapeutics Pipeline

Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), a leading RNAi therapeutics company, announced today its key “Alnylam 5x15™” pipeline goals. The company also announced today that it now expects to exceed its original “Alnylam 5x15” guidance from 2011, where Alnylam had guided to have five genetic medicine programs in the clinic by the end of 2015. Alnylam now expects to end 2015 with six to seven genetic medicine programs in the clinic, including at least two programs in Phase 3 and five to six programs that will have achieved human proof-of-concept results supporting further development.

“2013 was a year of remarkable accomplishments, as we continued to lead the translation of RNAi therapeutics as a new class of medicines, and we believe this progress positions us well for continued execution on our ‘Alnylam 5x15’ pipeline product strategy. Moreover, 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 genetic medicines,” said John Maraganore, Ph.D., Chief Executive Officer of Alnylam. “As a result of the achievements of the past period, we now believe that we will exceed our original ‘Alnylam 5x15’ guidance as issued in 2011, when we stated we expected to have 5 programs in clinical development by the end of 2015. Indeed, we now expect to significantly exceed that guidance with six to seven programs in clinical development by the end of 2015, including at least two programs in Phase 3 and five to six programs that will have achieved human proof-of-concept results.”

“We fully expect that 2014 will be another exciting year for Alnylam, as we advance and broaden our pipeline of RNAi therapeutics in development. Specifically, we will be enrolling patients in our APOLLO Phase 3 trial of patisiran and also collecting key clinical endpoint data from our ongoing Phase 2 open-label extension study with results in late 2014. We also plan on starting our company’s second Phase 3 trial, in this case with ALN-TTRsc in the cardiac amyloidosis indication; an ongoing Phase 2 study of this drug will also read out data by year-end,” said Barry Greene, President and Chief Operating Officer of Alnylam. “In our hemophilia program, ALN-AT3 is positioned to start Phase 1 in the coming weeks, and we expect to report interim proof-of-concept data by end of the year. We also expect to advance our pipeline with 2 additional IND filings this year from a few of our development stage programs including ALN-CC5 for complement-mediated diseases, ALN-AS1 for hepatic porphyrias, and ALN-PCSsc for hypercholesterolemia. Of course, we intend to also continue to significantly invest in our genetic medicine discovery pipeline, where we see a large number of potentially attractive opportunities for innovative RNAi therapeutics. In sum, we fully expect 2014 to bring us closer to market with our Phase 3 trials, yield multiple data read outs that may provide expanded proof of concept, and broaden our opportunities with a significant growth in our clinical pipeline of potential high impact genetic medicines.”

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