Alnylam Announces Allowance in Europe of New Fundamental Patent Broadly Covering RNAi Therapeutics

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), a leading RNAi therapeutics company, announced today that the European Patent Office (EPO) has issued a notification of intent to grant for a patent (EP1309726) in the Tuschl I patent series. The new patent is the first to be granted in the European Union (EU) from the Tuschl I patent series, and includes 19 claims broadly covering the use of double stranded RNAs (dsRNAs) for RNAi. This new patent grant extends the scope and breadth of Alnylam’s fundamental intellectual property estate that comprises numerous issued or granted patents and a large number of pending patent applications that together broadly cover RNAi therapeutics, including small interfering RNAs, or siRNAs, the molecules that mediate RNAi.

“a major scientific breakthrough that happens once every decade or so”

“We are very pleased with the EPO’s decision to grant claims from Tuschl I, a patent we believe is clearly part of the patent landscape required for the development and commercialization of all RNAi therapeutics,” said Barry Greene, President and Chief Operating Officer of Alnylam Pharmaceuticals. “This is the first patent of the Tuschl I patent series to be granted in the EU, and expands the scope of our fundamental IP that also includes issued or granted patents from the Crooke, Kreutzer-Limmer, Tuschl II, and Kay & McCaffrey patent estates, amongst other patent applications pending. We have demonstrated a clear track record of leveraging our patent portfolio to enable the field in developing RNAi therapeutics, as evidenced by more than 25 licensing agreements which have yielded over $660 million in realized cash funding for Alnylam. With this new patent grant, and the continued expansion of our IP estate, we expect our business development execution to only continue in the future.”

The current grant of the Tuschl I patent (EP1309726) consists of 19 claims broadly covering RNAi methods, including methods of reducing the expression of a gene, including those of mammalian or viral origin, with dsRNAs between 21 and 23 nucleotides in length. In addition, the patent also includes claims covering methods of examining the function of a gene, as well as the use of both unmodified and chemically modified dsRNAs. Upon completion of certain formalities, the patent will be granted in approximately four months. Alnylam is the exclusive licensee of the ownership interests of the Max Planck Society, the Massachusetts Institute of Technology, and Whitehead Institute for Biomedical Research, in the Tuschl I patent series for RNAi therapeutics.

“It is gratifying to see the Tuschl I patent series advance through the EPO, as we believe this patent has critical relevance for the development RNAi therapeutics. Combined with the Tuschl II patent series, which is a distinct patent owned by Max Planck and exclusively licensed to Alnylam for therapeutic applications, the new Tuschl I patent family significantly strengthens Alnylam’s efforts as our licensee,” said Dr. Joern Erselius, Managing Director, Max Planck Innovation GmbH. “We are, of course, pleased to have Alnylam as a partner for the commercialization of this discovery; they have clearly demonstrated a commitment to pursuing the full potential of RNAi technology.”

About Alnylam Intellectual Property (IP)

Alnylam’s IP estate includes issued, allowed, or granted fundamental patents in many of the world’s major pharmaceutical markets that claim the broad structural and functional properties of RNAi therapeutic products. These include:

the Crooke Patents (U.S. Patent Nos. 5,898,031, 6,107,094, 7,432,249, 7,432,250 and EP 0928290) issued in over 12 countries and licensed exclusively from Isis Pharmaceuticals, Inc. to Alnylam for RNAi therapeutics, which cover compositions, methods, and uses of modified oligonucleotides to inactivate a target mRNA mediated by a double stranded RNase, such as “RISC,” which is the cellular enzyme complex that mediates RNAi;
the Kreutzer-Limmer I ’719 patent (EP 1550719), owned by Alnylam and where the company has received a notification of ‘intent to grant’, which covers siRNAs comprising 15-21 nucleotides in length stabilized by chemical linkages;
the Kreutzer-Limmer I ’235 patent (DE 10066235), granted in January 2008 and owned by Alnylam, which covers methods, uses, and medicaments of siRNAs, with a length between 15 and 49 nucleotides, expressed through a vector;
the Kreutzer-Limmer II ’061 patent (EP 1352061), granted in May 2006 and owned by Alnylam, which covers therapeutic compositions, methods, and uses of siRNA and derivatives directed toward over 125 disease targets;
the Tuschl II ’704 patent (U.S. Patent No. 7,056,704) issued in June 2006 and exclusively licensed to Alnylam from the Max Planck Society, which broadly covers methods of making siRNAs to silence any and all disease targets;
the Tuschl II ’196 patent (U.S. Patent No. 7,078,196) issued in July 2006 and exclusively licensed to Alnylam from the Max Planck Society, which broadly covers methods of making siRNAs with or without chemical modifications;
the Tuschl II ’044 patent (EP 1407044), granted in January 2008 and exclusively licensed to Alnylam from the Max Planck Society, which broadly covers compositions, methods, and uses of siRNAs;
the Tuschl II patent (JP 4 095 895) granted in May 2008 in Japan and exclusively licensed to Alnylam from the Max Planck Society, which broadly covers compositions, methods, uses, and systems of siRNAs;
the Tuschl I patent (EP1309726) which received an intent to grant in April 2009 in Europe and is exclusively licensed to Alnylam from the Max Planck Society, the Massachusetts Institute of Technology, and Whitehead Institute, which broadly covers methods for using dsRNAs for RNAi;
the Kay & McCaffrey patent (AU patent application no. 2002326410) granted in February 2009 in Australia and exclusively licensed to Alnylam from Stanford University, which broadly covers methods and composition for RNAi therapeutics including siRNAs and shRNAs; and,
many divisional and continuing patent applications pending of the aforementioned issued or granted patents and additional patent applications pending, including patents and patent applications covering inventions by Fire & Mello (U.S. Patent No. 6,506,559), Kreutzer & Limmer, Glover, Li & Kirby, Pachuk, Tuschl, Hannon, and Giordano.

In addition to fundamental patents, Alnylam is the exclusive licensee in the field of RNAi therapeutics for more than 150 issued chemistry patents owned or controlled by Isis Pharmaceuticals broadly covering chemical modifications, including motifs and patterns of modifications of oligonucleotides, including RNAi therapeutics. These patents include:

phosphorothioate and 2’-O-methyl modifications of oligonucleotides (Buhr, U.S. Patent No. 6,476,205);
2’-Ribose modifications of oligonucleotides (Cook, U.S. Patent Nos. 5,670,633; 6,005,087; 6,531,584; and 7,138,517);
chemical conjugates of oligonucleotides (Manoharan, U.S. Patent No. 6,153,737); and,
“overhang,” “blunt-end,” and nucleotide pairing design motifs (Woppmann et al., UK 2417727), which is owned by Alnylam.

In addition to fundamental and chemistry patents, Alnylam is also the exclusive licensee in the field of RNAi therapeutics for certain delivery patents, including those owned and controlled by Tekmira Pharmaceuticals, Inc. covering delivery of oligonucleotides, including RNAi therapeutics, with liposomal formulations. These patents include:

formulations of oligonucleotides, including siRNAs, in cationic liposomes (Wheeler, U.S. Patent Nos. 5,976,567 and 6,815,432; and Semple, U.S. Patent No. 6,858,225).

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. 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 and is partnered with Cubist and Kyowa Hakko. In addition, the company is developing RNAi therapeutics for the treatment of a wide range of disease areas, including liver cancers, hypercholesterolemia, Huntington’s disease, and TTR amyloidosis. 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. To reflect its outlook for key scientific, clinical, and business initiatives, Alnylam established “RNAi 2010” in January 2008 which includes the company’s plan to significantly expand the scope of delivery solutions for RNAi therapeutics, have four or more programs in clinical development, and to form four or more new major business collaborations, all by the end of 2010. 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 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 the company’s ability to successfully research and develop products and to obtain and maintain intellectual property necessary to practice in the field of RNAi therapeutics, as well as those risks more fully discussed in the “Risk Factors” section of its most recent annual report on Form 10-K 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.