Alnylam Announces Tuschl II Key Patent Claims Upheld in Invalidation Trial in Japan

Alnylam Announces Tuschl II Key Patent Claims Upheld in Invalidation Trial in Japan

Alnylam Pharmaceuticals, Inc.

(Nasdaq: ALNY), a leading RNAi therapeutics company, announced today that the Japanese Patent Office upheld key claims in the Tuschl II patent (JP4095895) in an invalidation trial held in Japan. The invalidation request was made by Bio Think-Tank, a Japanese company.

“We are extremely pleased with the outcome of the invalidation trial which resulted in key claims from the Tuschl II ’895 patent being upheld in Japan. This affirms our belief in the validity of these claims and the groundbreaking nature of the Tuschl II invention forming the basis of this critical patent family,” said Laurence Reid, Ph.D., Senior Vice President, Chief Business Officer at Alnylam. “Tuschl II remains a valuable patent in our overall broad estate of patents important for the development and commercialization of RNAi therapeutics, and a patent family to which we are the exclusive licensee. We look forward to the continued advancement of the Tuschl II patent family worldwide in conjunction with many other Alnylam-held patents and pending patent applications.”

“We are gratified that the Japanese Patent Office has upheld these claims from Tuschl II,” said Dr. Joern Erselius, Managing Director, Max Planck Innovation GmbH. “The successful examination of the Tuschl II patent series highlights that this invention is seminal for RNAi, demonstrating the importance of the work performed by Professor Tuschl and other co-inventors at the Max Planck Institute.”

The Tuschl II patent series provides broad exclusivity for small interfering RNA (siRNA), the molecules that mediate RNAi, in the world’s top pharmaceutical markets, which include the U.S. (US 7,056,704 and US 7,078,196), the European Union (EP 1407044), China (CN100523215), and Japan (JP4095895 and JP4494392). Specifically, the Japanese Tuschl II ’895 patent broadly covers compositions, methods, and uses of siRNA that are important for the development and commercialization of RNAi therapeutics. The Tuschl II patent family is exclusively licensed to Alnylam for RNAi therapeutics on a worldwide basis through an agreement with Max Planck Innovation GmbH, the licensing agent for the Max Planck Society.

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 RNA (siRNA), 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.

Alnylam is a biopharmaceutical company developing novel therapeutics based on RNA interference, or RNAi. The company is leading the translation of RNAi as a new class of innovative medicines with a core focus on RNAi therapeutics for the treatment of genetically defined diseases, including ALN-TTR for the treatment of transthyretin-mediated amyloidosis (ATTR), ALN-AT3 for the treatment of hemophilia, ALN-PCS for the treatment of severe hypercholesterolemia, ALN-HPN for the treatment of refractory anemia, and ALN-TMP for the treatment of hemoglobinopathies. As part of its “Alnylam 5x15” strategy, the company expects to have five RNAi therapeutic products for genetically defined diseases in clinical development, including programs in advanced stages, on its own or with a partner by the end of 2015. Alnylam has additional partnered programs in clinical or development stages, including ALN-RSV01 for the treatment of respiratory syncytial virus (RSV) infection, ALN-VSP for the treatment of liver cancers, and ALN-HTT for the treatment of Huntington’s disease. The company’s leadership position on RNAi therapeutics and intellectual property have enabled it to form major alliances with leading companies including Merck, Medtronic, Novartis, Biogen Idec, Roche, Takeda, Kyowa Hakko Kirin, Cubist, Ascletis, and Monsanto. In addition, Alnylam and Isis co-founded Regulus Therapeutics Inc., a company focused on discovery, development, and commercialization of microRNA therapeutics; Regulus has formed partnerships with GlaxoSmithKline, Sanofi, AstraZeneca and Biogen Idec. Alnylam has also formed Alnylam Biotherapeutics, a division of the company focused on the development of RNAi technologies for applications in biologics manufacturing, including recombinant proteins and monoclonal antibodies. Alnylam’s VaxiRNA™ platform applies RNAi technology to improve the manufacturing processes for vaccines; GlaxoSmithKline is a collaborator in this effort. Alnylam scientists and collaborators have published their research on RNAi therapeutics in over 100 peer-reviewed papers, including many in the world’s top scientific journals such as , , , and . Founded in 2002, Alnylam maintains headquarters in Cambridge, Massachusetts. For more information, please visit .

Various statements in this release concerning Alnylam’s future expectations, plans and prospects, including, without limitation, statements regarding Alnylam’s expectations with respect to its “Alnylam 5x15” product strategy, Alnylam’s views with regard to the strength, enforceability, and validity of its intellectual property estate, 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, Alnylam’s ability to enforce its patents against infringers and defend its patent portfolio against challenges from third parties, as well as those risks more fully discussed in the “Risk Factors” 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 Alnylam’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.