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Alnylam Reports Phase I Clinical Safety Data for ALN-RSV01, an RNAi Therapeutic for the Treatment of RSV Infection; Results Reported at the 2006 Pediatric Academic Societies' Annual Meeting

Posted on: Sunday, 30 April 2006, 21:00 CDT

Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), a leading RNAi therapeutics company, announced today that the company's lead product candidate, ALN-RSV01, was found to be safe and well tolerated when administered intranasally in two Phase I clinical studies. ALN-RSV01 is being evaluated for the treatment of respiratory syncytial virus (RSV) infection and is the first RNAi therapeutic in human clinical development for an infectious disease.

"These Phase I study results are an important step forward for Alnylam and for the entire field of RNAi therapeutics. Data from these trials provide us with a significant clinical experience for ALN-RSV01 with 101 subjects enrolled in the two trials combined," said John Maraganore, Ph.D., President and Chief Executive Officer of Alnylam. "Treatment of RSV infection, the leading cause of pediatric hospitalization in the U.S. today and a prevalent infection in certain adult populations, represents a major unmet medical need in a large number of patients. We believe that ALN-RSV01 is a promising treatment option for these patients and, as an unpartnered clinical program, an important component of Alnylam's balanced pipeline of wholly owned and partnered RNAi therapeutics."

"As a clinician committed to the advancement of new therapies for the treatment of pediatric infectious diseases, I am very encouraged by the results of these first human studies with ALN-RSV01," said John P. DeVincenzo, M.D., Associate Professor of Pediatrics at the University of Tennessee Health Science Center. "Based on these Phase I studies, the encouraging pre-clinical data showing anti-viral activity at similar doses, and the drug's novel mechanism of action, ALN-RSV01 may represent a breakthrough treatment option for patients infected with RSV."

Alnylam conducted two Phase I trials with ALN-RSV01 to evaluate the safety, tolerability, and pharmacokinetics of ALN-RSV01 in healthy adult volunteers. Both trials were double-blind, placebo-controlled, and randomized. In total, 101 subjects were enrolled in the trials and 65 were exposed to ALN-RSV01. The subjects received single or multiple daily doses of ALN-RSV01 or saline placebo in a nasal spray. Results showed that ALN-RSV01 was safe when administered intranasally in relevant doses to human volunteers, and comparable to placebo with respect to any reported adverse events. All reported adverse events for both ALN-RSV01 and placebo subjects were mild, with no reported serious adverse events. Further, there was no evidence of laboratory or electrocardiographic abnormalities in subjects exposed to drug. Finally, as expected, there was no significant systemic exposure of ALN-RSV01 when administered intranasally.

"These data are an important milestone as we advance ALN-RSV01 into further clinical development. In the second half of this year we expect to initiate a Phase I study with an inhaled formulation of ALN-RSV01, and we are also actively exploring the initiation of an experimental infection, or 'viral challenge', study with our intranasal formulation in adult volunteers later in the year. We believe that these and other efforts will enable us to initiate a Phase II trial in naturally infected RSV patients in the first half of 2007," said Barry Greene, Chief Operating Officer of Alnylam. "Finally, as the industry's most advanced RNAi therapeutic for the treatment of infectious diseases, ALN-RSV01's development provides a critically important roadmap for our other pipeline efforts, including those with Novartis on pandemic influenza."

The data from the Phase I trials are being presented at the 2006 Pediatric Academic Societies' (PAS) Annual Meeting being held in San Francisco, April 29 - May 2. Presentation of the results are taking place in two sessions: the State of the Art Plenary Session #3810 titled "RNA Interference, Technological Development of siRNAs and Potential Treatments for Childhood Diseases" to be held in Moscone West, Room 3016-3018 on April 30, 2006 at 4:15 p.m. PT; and the PAS/PIDS platform session #4130 titled "Infectious Diseases II" on May 1, 2006 at 8:00 a.m. PT in Moscone West, Room 3001.

Trial Details

The trial conducted in the U.S. enrolled 34 healthy adult volunteers. Drug or placebo was administered intranasally in single ascending doses (1.5, 5, 15, 50, or 150 mg) across five cohorts. The trial conducted in Europe enrolled 67 healthy adult volunteers. Drug or placebo was administered intranasally in both single ascending doses (5, 25, or 150 mg) across three cohorts and in multiple ascending doses (5, 25, and 150 mg) daily for five consecutive days across three cohorts. Dose levels administered were comparable to active anti-viral dose levels observed in pre-clinical animal studies. In both studies, ALN-RSV01 was evaluated for safety, tolerability, and pharmacokinetics.

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 population accounts for more than 100,000 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. Since many diseases are caused by the inappropriate activity of specific genes, the ability to silence genes selectively through RNAi could provide a new way to treat a wide range of human diseases. RNAi is induced by small, double-stranded RNA molecules. RNAi can be activated by 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 highly targeted gene silencing. Alnylam's initial drug development programs are focused on Direct RNAi(TM) therapeutics which are siRNAs administered directly to diseased parts of the body. In parallel, the company is developing Systemic RNAi(TM) therapeutics that travel through the bloodstream to reach diseased parts of the body.

About Alnylam

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, which is the leading cause of hospitalization in infants in the U.S. 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, and Novartis. The company, founded in 2002, maintains global headquarters in Cambridge, Massachusetts, and has an additional operating unit in Kulmbach, Germany. Alnylam is honored to be the "emerging/mid-cap" company recipient of the 2006 James D. Watson Helix Award, the biotechnology industry's award for outstanding corporate achievement. For more information, please visit www.alnylam.com.

Alnylam Forward-Looking Statements

Various statements in this release concerning our future expectations, plans, and prospects, including statements with respect to clinical development plans for ALN-RSV01 and the potential for ALN-RSV01 to be a treatment option for RSV infection patients, 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 risks related to: our approach to discover and develop novel drugs, which is unproven and may never lead to marketable products; 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 our products; the successful development of products, 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.

Source: Business Wire

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