Health & Fitness
Health and Fitness
Health and Fitness
Thu, September 30, 2010
Wed, September 29, 2010
Alnylam Publishes Results from Phase IIa Study of ALN-RSV01 in the Treatment of Respiratory Syncytial Virus Infection in Lung T
CAMBRIDGE, Mass.--([ BUSINESS WIRE ])--Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), a leading RNAi therapeutics company announced today that it has published results in the American Journal of Respiratory and Critical Care Medicine (Zamora et al., Am. J. Respir. Crit. Care Med., doi:10.1164/rccm.201003-0422OC, 2010) from its double-blind, placebo-controlled, randomized Phase IIa study of ALN-RSV01 in lung transplant patients naturally infected with respiratory syncytial virus (RSV). These data, presented previously at the BIT Life Sciencea™s 2nd Annual Summit of Antivirals held in Beijing, China in July 2009, documented for the first time safety and tolerability of inhaled ALN-RSV01 in a naturally infected patient population. The results also demonstrated statistically significant improvements in clinical symptom scores and reductions in the incidence of bronchiolitis obliterans syndrome (BOS), an irreversible and life-threatening complication of RSV infection in lung transplant patients resulting in approximately 50% mortality within three to five years of onset.
"a major scientific breakthrough that happens once every decade or so"
aOur Phase IIa study of ALN-RSV01 demonstrated safety and tolerability in a naturally infected patient population. The study also demonstrated statistically significant effects on certain clinical endpoints that warrant verification in a larger study such as our current Phase IIb study which is actively enrolling,a said Akshay Vaishnaw, M.D., Ph.D., Senior Vice President of Clinical Research. aWe are also pleased with the continued and growing track record of Alnylam publications regarding our clinical trial results, as these importantly demonstrate our translation of RNAi therapeutics to patients.a
The Phase IIa study was conducted at 11 sites in four countries, where 24 lung transplant patients with confirmed RSV infection were randomized to receive inhaled ALN-RSV01 or placebo once daily for three consecutive days. Overall, the study achieved its primary objective of demonstrating safety and tolerability of ALN-RSV01. Interpretation of anti-viral effects for ALN-RSV01 was confounded by an imbalance in certain baseline patient characteristics. There was a statistically significant improvement in clinical symptom scores in patients receiving ALN-RSV01. Further, although the study was not powered for efficacy due to the small sample size, data at 90 days showed preliminary evidence for improved recovery of lung function and a statistically significant reduction in the incidence of new or progressive BOS, as compared with placebo (p=0.02).
aRSV infection in lung transplant patients is a cause of significant morbidity and even mortality due to the potential for both acute and chronic lung rejection,a 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. aThe results from this Phase IIa study with ALN-RSV01 are encouraging and support the continued development of an RNAi therapeutic for the treatment of RSV infection, a major unmet medical need with no effective treatments. I am pleased to be involved in the ongoing Phase IIb in the adult lung transplant patient population and look forward to continuing enrollment in this trial.a
As a result of this study, Alnylam initiated earlier this year a multi-center, global, randomized, double blind, placebo-controlled Phase IIb trial for ALN-RSV01 in RSV-infected adult lung transplant patients. This new study aims to repeat and extend the results of the Phase IIa study. The primary endpoint is the incidence of new or progressive BOS. Secondary endpoints include assessments for safety and additional measurements of efficacy, including: anti-viral activity; recovery of lung function, as monitored by the proportion of patients with forced expiratory volume in the first second (FEV1); and, improvement in RSV symptoms as measured by mean cumulative daily total symptom score. The Phase IIb study is expected to enroll up to 76 patients.
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 aa major scientific breakthrough that happens once every decade or so,a 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 Alnylama™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 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 leading the translation of RNAi as a new class of innovative medicines with peer-reviewed research efforts published in the worlda™s top scientific journals including Nature, Nature Medicine, and Cell. The company is leveraging these capabilities to build a broad pipeline of RNAi therapeutics for the treatment of a wide range of disease areas, including respiratory syncytial virus (RSV), liver cancers, TTR-mediated amyloidosis (ATTR), hypercholesterolemia, and Huntingtona™s disease. In addition, Alnylam formed Alnylam Biotherapeutics, a division of the company focused on the development of RNAi technologies for application in manufacturing processes for biotherapeutic products, including recombinant proteins and monoclonal antibodies. The companya™s leadership position in fundamental patents, technology, and know-how relating to RNAi has enabled it to form major alliances with leading companies including Medtronic, Novartis, Biogen Idec, Roche, Takeda, Kyowa Hakko Kirin, and Cubist. Alnylam and Isis are joint owners of Regulus Therapeutics Inc., a company focused on the discovery, development, and commercialization of microRNA therapeutics. Founded in 2002, Alnylam maintains headquarters in Cambridge, Massachusetts. For more information, please visit [ www.alnylam.com ].
Alnylam Forward-Looking Statement
Various statements in this release concerning Alnylama™s future expectations, plans and prospects, including without limitation, its expectations regarding the development of effective and efficient approaches for RNAi therapeutics for the treatment of respiratory syncytial virus (RSV) infection, and the timing and scope of ongoing clinical trials for ALN-RSV01, 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, risks related to: the difficulty drawing conclusions from the small sample size of patients in the Phase IIa study described in this press release; the difficulty drawing conclusions in light of the confounding differences in certain baseline characteristics between those patients taking ALN-RSV01 and those patients taking placebo in the Phase IIa study; results from pre-clinical testing or early clinical trials of a product candidate, including ALN-RSV01, that may not predict the results that will be obtained in subsequent human clinical trials; Alnylama™s approach to discovering and developing novel drugs, which is unproven and may never lead to marketable products, including ALN-RSV01 or any successor product thereto; obtaining, maintaining, and protecting intellectual property; Alnylama™s ability to enforce its patents against infringers and to defend its patent portfolio against challenges from third parties; Alnylama™s ability to obtain additional funding to support its business activities; Alnylama™s dependence on third parties for development, manufacture, marketing, sales and distribution of certain products; obtaining regulatory approval for products in different jurisdictions; competition from others using technology similar to Alnylama™s and others developing products for similar uses; Alnylama™s dependence on collaborators; and Alnylama™s short operating history; as well as those risks more fully discussed in the aRisk Factorsa 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 Alnylama™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.
