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Alnylam, Medtronic, and CHDI Foundation Form Collaboration to Advance RNAi Therapeutics for the Treatment of Huntington's Disease
- Commitment from CHDI to Fund up to 50% of Development Efforts toward IND Filing to Advance ALN-HTT, a Novel Drug-Device Combination -


Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), Medtronic Inc. (NYSE:MDT), and CHDI Foundation, Inc., announced today that they have formed a collaboration to advance ALN-HTT, a novel drug-device combination for the treatment of Huntington's disease. ALN-HTT consists of an RNAi therapeutic targeting huntingtin, the gene responsible for Huntington's disease, that is being developed for delivery to the central nervous system (CNS) using an implantable infusion system developed by Medtronic. CHDI is a not-for-profit virtual biotech company that is exclusively dedicated to rapidly discovering and developing therapies that slow the progression of Huntington's disease.

"Alnylam and Medtronic have shown leadership and encouraging progress in developing a novel drug-device combination for the treatment of Huntington's disease," said Robi Blumenstein, President of CHDI Management. "Their collaborative approach demonstrates a combined commitment to tackle this devastating disease and this program is closely aligned with CHDI's mission. We welcome the opportunity to accelerate this program and look forward to continued advancement toward clinical testing in patients."

Alnylam and Medtronic have worked collaboratively to advance ALN-HTT for the treatment of Huntington's disease. Under this new collaboration, CHDI has agreed to initially fund up to 50% of the investigational new drug (IND) application-enabling activities. The agreement between Alnylam and Medtronic will remain as a 50-50 partnership in the United States. Medtronic will commercialize the therapy consisting of the RNAi compound and delivery device. In the United States, Alnylam has the opportunity to invest in clinical development through product launch in return for a proportional share of the profits. In Europe, Medtronic is solely responsible for development and commercialization, and Alnylam is eligible to receive milestones and royalties.

"We are very pleased to add CHDI as a partner in our Huntington's disease program; they bring a tremendous amount of disease area expertise that will contribute to advancing ALN-HTT towards the clinic with Medtronic," said Dinah Sah, Ph.D., Vice President, Research, at Alnylam. "Our pre-clinical data provide a strong rationale for this program, and we remain encouraged by the potential that ALN-HTT may offer to patients."

Over the course of the existing collaboration, pre-clinical data have been generated supporting the continued development of ALN-HTT for the treatment of Huntington's disease, including:

  • demonstration that an siRNA targeting the huntingtin gene achieves sufficient distribution for coverage of brain regions affected in Huntington's disease;
  • data showing that direct delivery to the CNS results in robust silencing of the huntingtin gene mRNA which was achieved at substantial distances from the infusion site, an important step towards translating this delivery approach from pre-clinical models to the larger human brain; and,
  • results showing that ALN-HTT is well tolerated following continuous direct CNS administration over a period of approximately one month.1

"The ALN-HTT program represents an exciting opportunity to combine innovative medicines with our drug delivery technology in an area of extreme unmet medical need," said Gregory Stewart, Ph.D., Director of CNS Drug Therapy R&D in the Neuromodulation Business at Medtronic. "With no effective disease-modifying therapies available currently for patients afflicted with Huntington's disease, the collaboration between Alnylam and Medtronic, and now support from CHDI, will work to develop a novel treatment strategy for this devastating neurodegenerative disease."

About Huntington's Disease

Huntington's disease is an autosomal dominant neurodegenerative genetic disorder that causes motor, cognitive, and behavioral dysfunction. It is estimated that 120,000 people in the U.S. have the genetic mutation that causes Huntington's disease and will experience symptoms during a normal lifetime. The average lifespan for patients after onset of motor dysfunction is approximately 10 to 20 years. There are currently no disease-modifying therapies available to slow the progression of Huntington's disease.

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 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 for the treatment of a wide range of disease areas, including respiratory syncytial virus (RSV), liver cancers, TTR-mediated amyloidosis (ATTR), hypercholesterolemia, and Huntington's disease. In addition, Alnylam formed Alnylam Biotherapeutics, a division of the company focused on the development of RNAi technologies for application in manufacturing processes for biotherapeutic products, including recombinant proteins and monoclonal antibodies. 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. Alnylam and Isis are majority 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.

About Medtronic

Medtronic, Inc. (www.medtronic.com), headquartered in Minneapolis, is the global leader in medical technology - alleviating pain, restoring health and extending life for millions of people around the world.

Any forward-looking statements are subject to risks and uncertainties such as those described in Medtronic's periodic reports on file with the Securities and Exchange Commission. Actual results may differ materially from anticipated results.

About CHDI Foundation, Inc.

CHDI Foundation, Inc. is a privately funded, not-for-profit, virtual biotech company that is exclusively dedicated to rapidly discovering and developing therapies that slow the progression of Huntington's disease. CHDI's scientists work closely with a network of more than 600 researchers in academic and industrial laboratories around the world in the pursuit of these novel therapies, providing project management to ensure that their common goals remain in focus. This helps bridge the translational gap that often exists between academic and industrial research pursuits that adds costly delays to therapy development. In its role as a collaborative enabler, CHDI seeks to bring the right partners together to identify and address critical scientific issues and move drug candidates to clinical evaluation as rapidly as possible. CHDI's activities extend from exploratory biology to the identification and validation of therapeutic targets, and from drug discovery and development to clinical studies and trials. More information about CHDI can be found at www.chdifoundation.org.

Alnylam Forward-Looking Statement

Various statements in this release concerning Alnylam's future expectations, plans and prospects, including without limitation, expectations regarding the development of effective and efficient approaches for the development of a combination RNAi therapeutic and device for the treatment of Huntington's disease and expectations regarding the potential funding for such a program from CHDI Foundation, Inc., 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 but not limited to: Alnylam's ability to discover and develop novel drug candidates, such as ALN-HTT, a combination drug-device for the treatment of Huntington's disease, and successfully demonstrate efficacy and safety of ALN-HTT in pre-clinical IND-enabling studies; results from pre-clinical testing of a product candidate, including ALN-HTT, that may not predict the results that will be obtained in subsequent pre-clinical or human clinical trials; and Alnylam's dependence on collaborators for funding, development, manufacture, marketing, sales and/or distribution of products; 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.

1 2009 World Congress on Huntington's Disease; September 12-15, 2009; Vancouver, British Columbia

SOURCE: Alnylam Pharmaceuticals, Inc.

Cynthia Clayton (Investors), 617-551-8207
Amanda Sellers (Media), 202-955-6222 x2597
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"Safe Harbor" Statement under the Private Securities Litigation Reform Act of 1995: Statements in this press release regarding Alnylam Pharmaceuticals's business which are not historical facts are "forward-looking statements" that involve risks and uncertainties. For a discussion of such risks and uncertainties, which could cause actual results to differ from those contained in the forward-looking statements, see "Risk Factors" in the Company's Annual Report or Form 10-K for the most recently ended fiscal year.