Alnylam joins GSK in donating intellectual property to patent pool for neglected tropical diseases

Issued: Wednesday 8 July 2009, Cambridge, Massachusetts and London UK

- First company to join the pool since creation in March 2009
- Contribution triples number of patents in the pool
- RNAi technology could lead to new targets and treatments

GlaxoSmithKline (GSK) and Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY) announced today that Alnylam will contribute more than 1500 issued or pending patents on its RNA interference (RNAi) technology patent estate to the patent pool established by GSK earlier this year.  

Alnylam is the first company to add its patents to the approximately 800 patent filings GSK provided to populate the pool in March.  The company’s RNAi platform provides an innovative approach to drug discovery and development through “gene silencing,” a technology that targets the cause of diseases by potently silencing specific messenger RNAs (mRNAs), thereby preventing disease-causing proteins from being made.

“We are delighted that Alnylam will join GSK in this important programme by adding their unique RNAi technology to the patent pool,” said Andrew Witty, Chief Executive Officer of GSK. “The key objective of the pool is to make it easier for researchers across the world to access intellectual property that may be useful in the search for new medicines to treat neglected tropical diseases.  The more companies, academic institutions and foundations that join the pool, the more effective it will be.  Alnylam’s announcement today is therefore a welcome and significant step forward.”

The patent pool was formed to aid in the discovery and development of new medicines for the treatment of 16 neglected tropical diseases (NTD), as defined by the U.S. Food and Drug Administration, in the world’s Least Developed Countries.  By adopting a more flexible approach to intellectual property, the patent pool will facilitate access to compounds and technologies for organisations that want to conduct research on treatments for these neglected diseases.

“We are committed to the innovation of medicines for patients, so we cannot ignore the potential of our technology to make a difference in the discovery of important new medicines for neglected diseases that afflict millions of people each year,” said John Maraganore, Ph.D., Chief Executive Officer of Alnylam. “We are very proud to be joining GSK in this unique and bold vision of social responsibility for some of the world’s poorest nations.”

Through today’s contribution, Alnylam is providing RNAi intellectual property, technology and know-how on a royalty-free, non-profit basis in the Least Developed Countries via licensing agreements with qualified third parties.  Such organisations will be engaged in research efforts focused on discovery of new medicines for NTD and their distribution to Least Developed Countries.

In the near term, Alnylam RNAi technology is expected to help validate novel drug targets for the discovery and development of treatments for the targeted NTD in least developed countries. For example, the technology has already helped to identify new targets for malaria treatments. ^{[1], [2],[3]}  In the future, RNAi therapeutics may themselves be developed and used directly in the treatment of more neglected tropical diseases.

About the patent pool

The diseases targeted by the pool are the 16 diseases identified by the FDA for its own Neglected Tropical Diseases initiative. These are tuberculosis, malaria, blinding trachoma, buruli ulcer, cholera, dengue/dengue haemorrhagic fever, racunculiasis, fascioliasis, human African trypanosomiasis, leishmaniasis, leprosy, lymphatic filariasis, onchocerciasis, schistosomiasis, soil transmitted helminthiasis and yaws. The geographic focus of the pool will be the world’s Least Developed Countries as identified by the United Nations and includes much of western and central Africa as well as several countries in Southeast Asia. 

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 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 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 programme is in Phase II human clinical trials for the treatment of respiratory syncytial virus (RSV) infection and is partnered with Cubist and Kyowa Hakko Kirin.  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 programmes 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 commercialisation of microRNA-based therapeutics.  Founded in 2002, Alnylam maintains headquarters in Cambridge, Massachusetts.  For more information, please visit www.alnylam.com.

Alnylam forward-looking statement

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 whether Alnylam technology will be utilised by third parties to develop drugs for neglected tropical diseases in the world’s poorest nations 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.

About GlaxoSmithKline – one of the world’s leading research-based pharmaceutical and healthcare companies – is committed to improving the quality of human life by enabling people to do more, feel better and live longer.  For further information please visit www.gsk.com.

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