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Alnylam Initiates Phase II Trial to Evaluate Safety and Tolerability of ALN-RSV01 in Adult Lung Transplant Patients Naturally Infected with Respiratory Syncytial Virus Infection

Business Wire, April 9, 2008

- Secondary Objectives Include Measurements of Anti-viral Activity -

CAMBRIDGE, Mass. -- Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), a leading RNAi therapeutics company, announced today it has initiated a Phase II clinical trial to assess the safety and tolerability of aerosolized ALN-RSV01 versus placebo in adult lung transplant patients naturally infected with RSV. As a secondary objective, this trial will be the first to evaluate the anti-viral activity of ALN-RSV01 in a naturally acquired RSV lower respiratory tract infection.

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"There is a significant need for novel therapeutics to effectively treat patients with RSV infection, a prevalent infection in immune-compromised adult populations such as lung transplant patients, and the leading cause of pediatric hospitalization in the U.S. today," said Akshay Vaishnaw, M.D., Ph.D., Vice President, Clinical Research at Alnylam. "While our primary focus is on developing an RNAi therapeutic for pediatric RSV populations, we see a clear need for new treatments in certain adult patient settings and recognize the importance that comprehensive safety and exploratory efficacy bring to our overall ALN-RSV01 development program."

RSV infection in lung transplant patients represents a significant unmet medical need. The condition is associated with significant morbidity - up to 15 to 20 percent of infected patients develop acute or chronic lung rejection. These patients are also at risk for an increase in frequency of bronchiolitis obliterans syndrome, a manifestation of chronic rejection that is associated with a high five-year mortality rate.

"I am encouraged by the potential for ALN-RSV01 based on its safety, tolerability, and anti-viral activity to date, and I look forward to working with Alnylam in developing this RNAi therapeutic for the treatment of RSV infection in lung transplant patients," said Martin Zamora, M.D., Professor of Medicine, and Medical Director, Lung Transplant Program, University of Colorado Health Sciences Center, and Principal Investigator for the trial in the U.S. "These patients have very limited treatment options and pulmonologists are in need for an RSV therapy in this critical disease area. Indeed, morbidities associated with RSV infection in this setting are significant due to the potential for acute and chronic lung rejection and other complications."

The Phase II trial is a multi-center, multi-national, randomized, double-blind, placebo-controlled study. The primary objective of the study is to assess the safety and tolerability of aerosolized ALN-RSV01 versus placebo in lung transplant patients naturally infected with RSV. Secondary objectives include evaluating the anti-viral activity and pharmacokinetics of ALN-RSV01. Methods for measuring anti-viral activity include nasal swabs to determine level of viral shedding, as well as bronchoalveolar lavage (BAL) sampling in certain patients as determined necessary by the attending physician. BAL is a minimally invasive medical procedure that allows measurement of infection in the lung.

The trial is expected to enroll 21 patients who will be randomized in a two to one, drug to placebo ratio. All patients will receive standard of care, and those receiving ALN-RSV01 will have drug administered as a 0.6 mg/kg dose by inhalation via nebulizer once daily for three days. Results from previous pre-clinical and clinical studies have demonstrated that ALN-RSV01 has a favorable safety profile as well as robust anti-viral activity. Alnylam recently completed its Phase II GEMINI study in experimentally infected adult volunteers where, compared to placebo, intranasally administered ALN-RSV01 demonstrated statistically significant anti-viral efficacy with a 38 percent relative reduction in RSV infection rate and a 95 percent increase in the number of infection-free subjects. In addition and in other studies, ALN-RSV01 was shown to be safe and well tolerated when administered by inhalation via nebulizer in a multi-dose regimen in adult volunteers.

About Respiratory Syncytial Virus (RSV)

RSV is a highly contagious virus that causes infections in both the upper and lower respiratory tract. 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. 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. RSV infection in the pediatric and adult populations account for more than 300,000 hospitalizations per year in the U.S. 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. 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.

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Alnylam Initiates Phase II Trial to Evaluate Safety and Tolerability of ALN-RSV01 in Adult Lung Transplant Patients Naturally Infected with Respiratory Syncytial Virus Infection

Business Wire, April 9, 2008

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