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Alnylam Completes Enrollment in Phase IIb Study of ALN-RSV01, an RNAi Therapeutic for the Treatment of Respiratory Syncytial Virus (RSV) Infection

– On Track to Report Data from Completed Trial in Mid-2012 –

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Nov. 29, 2011-- Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), a leading RNAi therapeutics company, announced today that it has completed patient enrollment in its Phase IIb study with ALN-RSV01, an RNAi therapeutic for the treatment of respiratory syncytial virus (RSV) infection. The Phase IIb trial was designed as a multi-center, randomized, double-blind, placebo-controlled study in RSV-infected lung transplant patients. The global study is being conducted at 33 lung transplant centers in six countries around the world. Results are expected to be reported mid-2012.

“ALN-RSV01 represents Alnylam’s most advanced partner-based program and is aimed at the treatment of RSV infection in high-risk adult populations. In an earlier small Phase IIa study, we showed promising clinical activity in RSV-infected lung transplant patients that we have aimed to reproduce in this current Phase IIb study. We look forward to reporting on data from this trial in mid-2012, and then evaluating next steps for this program together with our partners Cubist and Kyowa Hakko Kirin,” said Akshay K. Vaishnaw, M.D., Ph.D., Senior Vice President and Chief Medical Officer of Alnylam. “In the meanwhile, Alnylam remains focused on its core ‘5x15’ strategy advancing RNAi therapeutics for the treatment of genetically defined targets and diseases, exemplified by our recent positive results in our ALN-TTR01 clinical study.”

The Phase IIb study was designed to repeat and extend findings from a previous small Phase IIa study of ALN-RSV01 in RSV-infected lung transplant patients (Zamora et al., Am. J. Respir. Crit. Care Med., Feb 2011, Vol 183, No 4: pp 531-538), where the drug was shown to significantly decrease the incidence of bronchiolitis obliterans syndrome (BOS), a life-threatening complication of RSV infection, at 90 days in ALN-RSV01 recipients compared to placebo (6.3% vs.50%; ALN-RSV01 N=16, placeboN=8; p=0.027). In the current Phase IIb study, the primary endpoint was designed as a reduction in the incidence of new or progressive BOS at 180 days. The trial enrolled approximately 90 patients who were randomized in a 1:1 ratio of drug to placebo. All patients received standard of care, and those receiving ALN-RSV01 had drug administered as a 0.6 mg/kg dose by inhalation using an investigational eFlow Nebulizer System (PARI Pharma) once daily for five days. A planned interim analysis was recently performed by an Independent Study Biostatistical Committee to determine whether an increase in sample size up to a maximum of 120 patients was warranted. Alnylam was informed that the study should be completed with the current sample size; because the study remains blinded to all parties, the interim analysis results cannot be interpreted to suggest either a positive or negative outcome.

The RSV program is partnered with Cubist Pharmaceuticals in North America and the rest of the world outside of Asia, where the program is partnered with Kyowa Hakko Kirin Co., Ltd. These partners maintain opt-in rights for the development of ALN-RSV01.

About RSV

Respiratory syncytial virus (RSV) is a highly contagious virus that causes infections in both the upper and lower respiratory tract. RSV infection typically results in cold-like symptoms but can lead to more serious respiratory illnesses such as croup, pneumonia, bronchiolitis, and in extreme cases, death. In the pediatric and adult populations, it account for more than 300,000 hospitalizations per year in the U.S. RSV infection in lung transplant patients represents an important unmet medical need; the condition is associated with significant morbidity, including the development of acute lung transplant rejection in up to 20% of infected patients. Lung transplant patients infected with RSV are also at risk for an increase in frequency of chronic allograft dysfunction, which manifests as bronchiolitis obliterans syndrome (BOS), a life-threatening complication representing an irreversible disease of the transplanted lung resulting in approximately 50% mortality within three to five years of onset. RSV infects nearly every child at least once by the age of two years and is a major cause of hospitalization due to respiratory infection in children and people with compromised immune systems, and others. In addition, RSV infection in infants has been linked to the development of childhood asthma. As a result, there is a significant need for novel therapeutics to treat patients who become infected with RSV.

About RNA Interference (RNAi)

RNAi (RNA interference) is a revolution in biology, representing a breakthrough in understanding how genes are turned on and off in cells, and a completely new approach to drug discovery and development. Its discovery has been heralded as “a major scientific breakthrough that happens once every decade or so,” and represents one of the most promising and rapidly advancing frontiers in biology and drug discovery today which was awarded the 2006 Nobel Prize for Physiology or Medicine. RNAi is a natural process of gene silencing that occurs in organisms ranging from plants to mammals. By harnessing the natural biological process of RNAi occurring in our cells, the creation of a major new class of medicines, known as RNAi therapeutics, is on the horizon. Small interfering RNAs (siRNAs), the molecules that mediate RNAi and comprise Alnylam’s RNAi therapeutic platform, target the cause of diseases by potently silencing specific mRNAs, thereby preventing disease-causing proteins from being made. RNAi therapeutics have the potential to treat disease and help patients in a fundamentally new way.

About Alnylam Pharmaceuticals

Alnylam is a biopharmaceutical company developing novel therapeutics based on RNA interference, or RNAi. The company is leading the translation of RNAi as a new class of innovative medicines with a core focus on RNAi therapeutics for the treatment of genetically defined diseases, including ALN-TTR for the treatment of transthyretin-mediated amyloidosis (ATTR), ALN-PCS for the treatment of severe hypercholesterolemia, ALN-HPN for the treatment of refractory anemia, and ALN-APC for the treatment of hemophilia. As part of its “Alnylam 5x15TM” strategy, the company expects to have five RNAi therapeutic products for genetically defined diseases in advanced stages of clinical development by the end of 2015. Alnylam has additional partner-based programs in clinical or development stages, including ALN-RSV01 for the treatment of respiratory syncytial virus (RSV) infection, ALN-VSP for the treatment of liver cancers, and ALN-HTT for the treatment of Huntington’s disease. The company’s leadership position on RNAi therapeutics and intellectual property have enabled it to form major alliances with leading companies including Merck, Medtronic, Novartis, Biogen Idec, Roche, Takeda, Kyowa Hakko Kirin, and Cubist. In addition, Alnylam and Isis co-founded Regulus Therapeutics Inc., a company focused on discovery, development, and commercialization of microRNA therapeutics; Regulus has formed partnerships with GlaxoSmithKline and Sanofi. Alnylam has also formed Alnylam Biotherapeutics, a division of the company focused on the development of RNAi technologies for application in biologics manufacturing, including recombinant proteins and monoclonal antibodies. Alnylam’s VaxiRNA™ platform applies RNAi technology to improve the manufacturing processes for vaccines; GlaxoSmithKline is a collaborator in this effort. Alnylam scientists and collaborators have published their research on RNAi therapeutics in over 100 peer-reviewed papers, including many in the world’s top scientific journals such as Nature, Nature Medicine, Nature Biotechnology, and Cell. Founded in 2002, Alnylam maintains headquarters in Cambridge, Massachusetts. For more information, please visit www.alnylam.com.

Alnylam Forward-Looking Statements

Various statements in this release concerning Alnylam’s future expectations, plans and prospects, including without limitation, statements regarding Alnylam's views with respect to the potential for RNAi therapeutics, including ALN-RSV01, its plan to disclose data from its ALN-RSV01 clinical trial, its expectations regarding the results from the interim analysis of the Phase IIb study, and Alnylam’s expectations regarding its “Alnylam 5x15” product strategy, constitute forward-looking statements for the purposes of the safe harbor provisions under The Private Securities Litigation Reform Act of 1995. Actual results may differ materially from those indicated by these forward-looking statements as a result of various important factors, including, without limitation, Alnylam’s ability to discover and develop novel drug candidates, successfully demonstrate the efficacy and safety of its drug candidates, including ALN-RSV01, in human clinical trials, the clinical results for its product candidates, which may not support further development of product candidates,and Alnylam’s ability to establish and maintain strategic business alliances, and new business initiatives, as well as those risks more fully discussed in the “Risk Factors” section of its most recent quarterly report on Form 10-Q on file with the Securities and Exchange Commission. In addition, any forward-looking statements represent Alnylam’s views only as of today and should not be relied upon as representing its views as of any subsequent date. Alnylam does not assume any obligation to update any forward-looking statements.

Source: Alnylam Pharmaceuticals

Alnylam Pharmaceuticals, Inc.
Cynthia Clayton, 617-551-8207
Senior Director, Investor Relations and
Corporate Communications
Amanda Sellers, 202-955-6222 x2597

"Safe Harbor" Statement under the Private Securities Litigation Reform Act of 1995: Statements in this press release regarding Alnylam Pharmaceuticals's business which are not historical facts are "forward-looking statements" that involve risks and uncertainties. For a discussion of such risks and uncertainties, which could cause actual results to differ from those contained in the forward-looking statements, see "Risk Factors" in the Company's Annual Report or Form 10-K for the most recently ended fiscal year.