The experimental infection model was designed to evaluate viral infectivity of RSV administered intranasally in healthy adult volunteers. Top-line data are summarized as follows:
-- A total of 36 subjects were exposed to RSV in 5 cohorts;
-- Escalating viral inoculums were used from log 3 to log 5 plaque forming units (PFU), a measure of viral inoculum concentration;
-- Clinical and virologic signs of infection, e.g., viral load and viral shedding, were documented;
-- 72 percent of subjects were infected with a mean incubation period of 3.2 days, duration of infection of 7 days, and viral load (by an area under the curve analysis) of 61.9 log 10 PFU-days; and,
-- No significant adverse events were observed.
"We are pleased with the progress we have made to date with the clinical development of ALN-RSV01, an RNAi therapeutic for the treatment of RSV," said Akshay Vaishnaw, M.D., Ph.D., Vice President, Clinical Research of Alnylam. "Experimental infection models have been used to develop many anti-viral treatments including those for flu and the common cold, and we believe that such a model is a significant advance for the development of novel anti-RSV therapies. Importantly, with an experimental infection model in hand, we now have the ability to demonstrate the anti-viral activity for RNAi therapeutics in a well-controlled Phase II human trial. We expect to have data with ALN-RSV01 from this trial in the second half of the year."
"Based on the Phase I studies to date, the encouraging pre-clinical data showing anti-viral activity, and the drug's novel mechanism of action, ALN-RSV01 may represent a breakthrough treatment option for patients infected with RSV," said John P. DeVincenzo, M.D., Associate Professor of Pediatrics and Infectious Diseases at the University of Tennessee Health Science Center. "Establishing a safe, reliable RSV infection in human volunteers, and being able to quantify the infection and the symptoms it produces is a significant accomplishment. In the months to come, I look forward to the initiation of a Phase II trial for ALN-RSV01, where we will have the opportunity to demonstrate anti-viral activity in man."
ALN-RSV01 is an RNAi therapeutic being developed for the treatment of RSV infection, a prevalent viral infectious disease in pediatric and several adult patient populations. ALN-RSV01 was found to be safe and well tolerated when administered intranasally in clinically relevant doses to human adult volunteers in a Phase I study where it demonstrated a safety profile comparable to placebo. A Phase I inhalation safety study with a nebulized formulation of ALN-RSV01 is currently enrolling subjects in the U.S.; data from this trial is now expected in the second half of 2007. ALN-RSV01 is an Alnylam proprietary program and as such, represents an important component of the company's balanced pipeline of proprietary and partnered RNAi therapeutics programs.
Phase II Experimental Infection Study Details
Alnylam will be conducting a randomized, placebo-controlled, double-blind Phase II experimental infection st udy to assess the safety and tolerability of intranasally delivered ALN-RSV01 versus placebo, administered to healthy adult volunteers experimentally inoculated with the RSV virus. The study, expected to enroll approximately 90 subjects, aims to determine the safety and anti-viral activity of ALN-RSV01 as measured toward clinical symptoms of RSV infection and RSV infection rate based on measures of viral load.
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 leading cause of hospitalization due to respiratory infection in children and in 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 population accounts for more than 125,000 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)
RNA interference (or RNAi) is a naturally occurring mechanism within cells for selectively silencing and regulating specific genes. The discovery of RNAi has been widely acknowledged as a major breakthrough in biology, and the technology was recognized for its potential broad impact in medicine with the award of the 2006 Nobel Prize for Physiology or Medicine. Since many diseases are caused by the inappropriate activity of specific genes, the ability to silence genes selectively through RNAi has accelerated the understanding of these genes and their related pathways. Additionally, RNAi could provide a new way to treat a wide range of human diseases. RNAi is induced by small , double-stranded RNA molecules. One method to activate RNAi is with chemically synthesized small interfering RNAs, or siRNAs, which are double-stranded RNAs that are targeted to a specific disease-associated gene. The siRNA molecules are used by the natural RNAi machinery in cells to cause targeted gene silencing.
Alnylam is a biopharmaceutical company developing novel therapeutics based on RNA interference, or RNAi. The company is applying its therapeutic expertise in RNAi to address significant medical needs, many of which cannot effectively be addressed with small molecules or antibodies, the current major classes of drugs. Alnylam is building a pipeline of RNAi therapeutics; its lead program is in Phase I human clinical trials for the treatment of respiratory syncytial virus (RSV) infection. RSV infects nearly every child at least once by the age of two and accounts for more than 125,000 hospitalizations annually in the U.S. pediatric population. RSV infection also poses a great risk to the elderly and other adults with compromised immune systems. The company's leadership position in fundamental patents, technology, and know-how relating to RNAi has enabled it to form major alliances with leading companies including Merck, Medtronic, Novartis, and Biogen Idec. The company, founded in 2002, maintains global headquarters in Cambridge, Massachusetts, and has an additional operating unit in Kulmbach, Germany. For more information, visit www.alnylam.com.
Alnylam Forward-Looking Statements
Various statements in this release concerning our future expectations, plans, and prospects, including without limitation, the commencement of clinical trials and studies and the availability of results of clinical trials and studies, 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 the se forward-looking statements as a result of various important factors, including risks related to: our approach to discover and develop novel drugs, which is unproven and may never lead to marketable products; our ability to fund and the results of further pre-clinical and clinical trials; obtaining, maintaining and protecting intellectual property utilized by our products; our ability to enforce our patents against infringers and to defend our patent portfolio against challenges from third parties; our ability to obtain additional funding to support our business activities; our dependence on third parties for development, manufacture, marketing, sales, and distribution of products; the successful development of our product candidates, all of which are in early stages of development; obtaining regulatory approval for products; competition from others using technology similar to ours and others developing products for similar uses; our dependence on collaborators; and our short operating history; as well as those risks more fully discussed in the "Risk Factors" section of our most recent report on Form 10-K on file with the Securities and Exchange Commission. In addition, any forward-looking statements represent our views only as of today and should not be relied upon as representing our views as of any subsequent date. We do not assume any obligation to update any forward-looking statements.
Alnylam Pharmaceuticals, Inc.
Cynthia Clayton, 617-551-8207
Kathryn Morris, 845-635-9828