CAMBRIDGE, Mass. & MADRID–(BUSINESS WIRE)–Nov. 16, 2011— Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), a leading RNAi therapeutics company, and Sylentis, S.A., a Spanish bio-pharmaceutical company devoted to the research and development of new therapies based on RNAi, announced today that Alnylam has granted Sylentis a non-exclusive option for a new target-specific InterfeRx™ license. This license would enable the discovery, development, and commercialization of a synthetic siRNA directed towards an undisclosed target for the treatment of glaucoma. Sylentis’ program is currently in a Phase I/II clinical trial. Upon Sylentis’ exercise of this option, Alnylam would receive upfront and milestone payments, as well as royalties on sales of products covered by the licensing agreement. Additional financial details were not disclosed. Sylentis is a wholly owned subsidiary of Grupo Zeltia (MC:ZEL).
“We are pleased to be granting Sylentis a new InterfeRx license, thereby enabling their efforts with access to Alnylam intellectual property which we believe is critical for the development and commercialization of RNAi therapeutics,” said Laurence Reid, Ph.D., Senior Vice President and Chief Business Officer of Alnylam. “The agreement with Sylentis represents continued progress with our InterfeRx program, an important part of our overall strategy to create value today by leveraging our intellectual property portfolio.”
“Sylentis is focused on designing, developing, and commercializing RNAi therapeutics to treat serious medical conditions such as glaucoma. This license from Alnylam is an important step for us to further advance our glaucoma program in human clinical studies,” said José María Fernández Sousa, President of Grupo Zeltia.
Alnylam created the InterfeRx licensing program to grant licenses under its intellectual property to biotechnology and pharmaceutical companies wishing to pursue RNAi therapeutics against specific targets outside Alnylam’s core strategic interests. To date, a total of six companies are InterfeRx licensees; the license grants cover fourteen active target programs including four in clinical development. In addition to Sylentis, Alnylam’s InterfeRx licensees include Calando Pharmaceuticals, Inc., Tekmira Pharmaceuticals Corporation, Quark Pharmaceuticals, Inc., GeneCare Research Institute Co., Ltd., and, under an option agreement, Benitec Ltd.
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 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-PCS for the treatment of severe hypercholesterolemia, ALN-HPN for the treatment of refractory anemia, and ALN-APC for the treatment of hemophilia. As part of its “Alnylam 5x15TM” strategy, the company expects to have five RNAi therapeutic products for genetically defined diseases in advanced stages of clinical development by the end of 2015. Alnylam has additional partner-based 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, and Cubist. 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 and Sanofi. Alnylam has also formed Alnylam Biotherapeutics, a division of the company focused on the development of RNAi technologies for application 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 Nature, Nature Medicine, Nature Biotechnology, and Cell. Founded in 2002, Alnylam maintains headquarters in Cambridge, Massachusetts. For more information, please visit www.alnylam.com.
Sylentis was founded in 2006 as a spin-off of Genomica (a subsidiary of Grupo Zeltia). In January 2011, Sylentis received authorisation from the Spanish Agency of Medicines and Medical Devices (AEMPS) as a drug manufacturing facility, strengthening its position as a pioneer in Spain in the search for new therapies based on RNAi. The company aims to design effective short interfering RNAs (siRNAs) using bioinformatic programs such as SIRFINDER®, which optimises the design of siRNAs with pharmacological potential. Sylentis initially focused on developing therapies to treat glaucoma/ocular hypertension and dry eye associated pain, conditions which are addressed by the company’s most advanced compounds at present. It also has other research lines whose compounds are in the R&D and preclinical phases. Sylentis, which is headquartered in Madrid (Spain), is a subsidiary of Grupo Zeltia (Spanish stock exchange: ZEL), which has been listed on the Spanish Stock Exchange since 1963 and on Spain’s Electronic Market since 1998.
Zeltia S.A. is a world-leading biopharmaceutical company specialised in the development of marine-based drugs for use in oncology and central nervous system diseases. Grupo Zeltia consists mainly of the following companies: PharmaMar, the world-leading biotechnology company in advancing cancer care through the discovery and development of innovative marine-derived medicines; Noscira, a biotech firm focused on discovering and developing new drugs against Alzheimer’s disease and other neurodegenerative diseases of the central nervous system; Genomica, Spain’s leading molecular diagnostics company; Sylentis, dedicated to researching therapeutic applications of gene silencing (RNAi); and a chemical division comprising Zelnova and Xylazel, two highly profitable companies that are leaders in their respective market segments. Please visitwww.zeltia.com
Alnylam Forward-Looking Statements
Various statements in this release concerning Alnylam’s future expectations, plans and prospects, including without limitation, Alnylam’s views with respect to its InterfeRx™ program and the importance and scope of its intellectual property rights, the potential receipt of milestone payments and royalties under its InterfeRx license to Sylentis, as well as Alnylam’s expectations with respect to its “Alnylam 5×15” product strategy, 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: Alnylam’s approach to discover and develop novel drugs, which is unproven and may never lead to marketable products; the pre-clinical and clinical results for its product candidates, which may not support further development of product candidates; actions of regulatory agencies, which may affect the initiation, timing and progress of clinical trials; obtaining, maintaining and protecting intellectual property; Alnylam’s ability to enforce its patents against infringers and to defend its patent portfolio against challenges from third parties; Alnylam’s ability to obtain additional funding to support its business activities; Alnylam’s dependence on third parties for development, manufacture, marketing, sales and distribution of products; obtaining regulatory approval for products; and Alnylam’s short operating history; 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.
Source: Alnylam Pharmaceuticals, Inc.
Alnylam Pharmaceuticals, Inc.
Cynthia Clayton, 617-551-8207
Senior Director, Investor Relations and
Amanda Sellers (Media), 202-955-6222 x2597
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