Alnylam and Collaborators Publish New Pre-clinical Results on RNAi Therapeutics for the Treatment of Huntington's Disease
"We are very encouraged by the findings published today and feel that they contribute to a growing body of work representing a strong rationale for the advancement of an RNAi therapeutic for the treatment of Huntington's disease," said Dinah Sah, Ph.D., Senior Director of Research at Alnylam. "We believe these results reflect the progress that Alnylam and our collaborators are making in developing this promising therapeutic modality for a major neurodegenerative disease."
The new pre-clinical study (DiFiglia et al. (October 16, 2007) Proc. Natl. Acad. Sci. USA, 10.1073/pnas.0708285104) showed that a single injection of an siRNA targeting huntingtin, the gene responsible for Huntington's disease, resulted in improved symptoms of disease in an animal model. These improved effects included reduction in neuronal pathology and an improvement in motor behavior. The RNAi therapeutic reduced expression of mutant huntingtin in the brain and sustained a benefit in motor behavior for at least one week. In preliminary studies, the RNAi therapeutic was found to be well tolerated in the brain after direct CNS administration.
"I am excited by these new data as there is a very significant need for novel therapeutics to treat patients with Huntington's disease. Indeed, patients afflicted with this genetic disease are otherwise destined to an irreversible deterioration of neuronal function," said Neil Aronin, M.D., Professor at the University of Massachusetts Medical School. "While more work is needed to advance this approach, the mechanism of action for RNAi therapeutics defines a promising therapeutic strategy that could slow or halt disease progression."
Alnylam's Huntington disease program is partnered with Medtronic, Inc. The collaboration is focused on developing a novel drug-device combination for the treatment of Huntington's disease, with a product expected to consist of an RNAi therapeutic targeting the huntingtin gene that is delivered by Medtronic's implantable infusion pump.
About RNA Interference (RNAi)
RNAi 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, and 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.
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 influenza, hypercholesterolemia, and liver cancers, amongst other diseases. 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, and Roche. The company, founded in 2002, maintains headquarters in Cambridge, Massachusetts. For more information, visit www.alnylam.com.
Alnylam Forward-Looking Statements
Various statements in this release concerning Alnylam's future expectations, plans and prospects, 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; our ability to attract and retain highly qualified employees; 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 Alnylam's 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 quarterly report on Form 10-Q 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
Posted: October 2007