Alnylam Awarded Continued Funding Under U.S. Government Contract to Develop RNAi Therapeutics for Biological Threats

Business Wire, August 6, 2008

Commitment for Continued Funding of $7.5 Million Supports Alnylam Biodefense[TM] Efforts and Broad Technology Development

CAMBRIDGE, Mass. -- Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), a leading RNAi therapeutics company, today announced that the National Institute of Allergy and Infectious Diseases (NIAID), a component of the National Institutes of Health (NIH), has committed to $7.5 million of continued funding related to the 2006 contract for the development of a broad spectrum RNAi anti-viral therapeutic against hemorrhagic fever virus, including Ebola virus. Ebola virus can cause a severe, often fatal infection, and poses a potential biological safety risk and bioterrorism threat.

"We are pleased with the continued support of the federal government for the development of an anti-viral RNAi therapeutic for Ebola," said Barry Greene, President and Chief Operating Officer. "To date, we have been granted more than $63 million in federal contracts for Alnylam Biodefense, and we are looking forward to continuing our work with the NIH to help strengthen our nation's capabilities to counter serious biological security threats."

"We are encouraged by the data we have seen with the Ebola program. These data have demonstrated potent and specific in vivo efficacy mediated by an RNAi mechanism," said Antonin de Fougerolles, Ph.D., Senior Director of Research at Alnylam. "This funding from the NIAID allows us to continue to develop our technology as we advance our pipeline programs."

In September 2006, Alnylam was awarded a federal contract (No. HHSN266200600012C) providing the company with up to $23 million in funding over a four-year period to develop small interfering RNAs (siRNAs), the molecules that mediate RNAi, as anti-viral drugs targeting Ebola virus. To date, the government has committed to paying Alnylam up to $14.2 million for the first two years of the contract. As a result of the continued progress of this program, the government has committed to fund an additional $7.5 million over year three of the contract.

At the RNAi Keystone Symposia in March 2008, Alnylam presented pre-clinical data from this program utilizing an optimized RNAi therapeutic formulated in a lipid particle for systemic delivery. These data showed that potent siRNAs with in vitro anti-viral activity were identified against all genes in the Ebola genome. A greater than 95 percent decrease in viral titer was seen when an RNAi therapeutic targeting one of these genes, VP35, was administered to mice infected with Ebola. Moreover, the VP35 siRNA, as compared with a control non-specific siRNA, protected both mice and guinea pigs from lethal Ebola infection. This work was done in collaboration with Tekmira Pharmaceuticals Corporation using their lipid particle delivery formulation technology.

As part of a public sector-private sector partnership with its Ebola program, Alnylam is working with the United States Army Medical Research Institute of Infectious Diseases (USAMRIID), an organization which is uniquely experienced in the handling, safety, and security requirements of specialized biological agents. Alnylam produces drug candidates which are then sent to USAMRIID for in vitro and in vivo testing against Ebola virus.

About Alnylam BioDefense[TM]

Alnylam Biodefense was established to build a robust platform for developing RNAi therapeutics targeting threats of bioterrorism. Funding for the company's Ebola virus, pandemic flu, and viral hemorrhagic fever programs represents an example of broad public health and federal interest in the potential of RNAi technology to treat and prevent disease from these, and other serious and life-threatening viruses. Government funding extends Alnylam's anti-viral pipeline, which includes programs for treatment of: respiratory syncytial virus (RSV) infection with ALN-RSV01, which is in Phase II human clinical trials; pandemic influenza with ALN-FLU01 in a partnership with Novartis; and JC virus in a partnership with Biogen Idec.

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. RNAi therapeutics target the cause of diseases by potently silencing specific messenger RNAs (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.