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Posted: April 27, 2010

Alnylam Publishes Results from Phase II GEMINI Trial with ALN-RSV01

(Nanowerk News) Alnylam Pharmaceuticals, Inc., a leading RNAi therapeutics company, announced today that it has published results from its Phase II GEMINI study in the Proceedings of the National Academy of Sciences ("A randomized, double-blind, placebo-controlled study of an RNAi-based therapy directed against respiratory syncytial virus"). These data, previously presented at the International Symposium on Respiratory Viral Infections in Singapore in 2008, showed human proof of concept with an RNAi therapeutic in a randomized, double blind, placebo-controlled study. In this study, treatment with ALN-RSV01 showed statistically significant anti-viral activity, including a decreased RSV infection rate and an increase in the number of subjects who remained free of infection.
“The positive results generated from GEMINI established encouraging human proof of concept with an RNAi therapeutic,” said Akshay Vaishnaw, M.D., Ph.D., Senior Vice President, Clinical Research at Alnylam. “GEMINI also set the stage for us to advance ALN-RSV01 into the naturally RSV-infected adult lung transplant population where we are currently conducting a Phase IIb clinical study. We are encouraged by the cumulative data to date in this program and look forward to the program’s continued advancement.”
GEMINI was a double blind study, where 88 adult subjects experimentally infected with RSV were randomized to receive ALN-RSV01 or placebo. Administration of ALN-RSV01 resulted in statistically significant anti-viral activity with a 38% relative reduction in RSV infection rate (p<0.01) resulting in a 95% increase in the number of infection-free subjects as compared with placebo (p<0.01). ALN-RSV01 was also found to be safe and well tolerated.
“Results from the GEMINI study are notable, as they established anti-viral activity for an RNAi therapeutic in a major respiratory disease and provide a solid foundation for ongoing development of ALN-RSV01,” said John P. DeVincenzo, M.D., Professor of Pediatrics and Infectious Diseases and Professor of Molecular Sciences at the University of Tennessee Health Science Center and Principal Investigator for the GEMINI study. “I am enthusiastic about the continued development of RNAi therapeutics for the treatment of RSV infection, a major infectious disease in both adult and pediatric populations where there are no effective treatments used widely today.”
Earlier this year, Alnylam initiated a randomized, double blind, placebo-controlled Phase IIb trial of ALN-RSV01 in RSV-infected adult lung transplant patients. The primary endpoint of this study is the incidence of new or progressive bronchiolitis obliterans syndrome (BOS), a life-threatening complication of RSV infection in the transplanted lung resulting in approximately 50% mortality within three to five years of onset. The Phase IIb study is expected to enroll up to 76 patients.
The ALN-RSV program is partnered with Kyowa Hakko Kirin Co., Ltd. in Asia, and Cubist Pharmaceuticals, Inc. worldwide except Asia. In parallel with Alnylam's development of ALN-RSV01 for the treatment of RSV in lung transplant patients, Alnylam and Cubist are developing a second-generation compound, ALN-RSV02, which will be focused on the pediatric patient population. Cubist will take the lead in advancing ALN-RSV02 in the pediatric setting in continued collaboration and 50-50 funding with Alnylam, and Cubist retains an opt-in right for ALN-RSV01 in the adult transplant indication.
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. Small interfering RNAs (siRNAs), the molecules that mediate RNAi and comprise Alnylam’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 world’s top scientific journals including Nature, Nature Medicine, and Cell. The company is leveraging these capabilities to build a broad pipeline of RNAi therapeutics; its most advanced program is in Phase II human clinical trials for the treatment of respiratory syncytial virus (RSV) infection. In addition, the company is developing RNAi therapeutics for the treatment of a wide range of disease areas, including liver cancers, TTR-mediated amyloidosis (ATTR), hypercholesterolemia, and Huntington’s disease. 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 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-based therapeutics. Founded in 2002, Alnylam maintains headquarters in Cambridge, Massachusetts.
Source: Alnylam Pharmaceuticals (press release)
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