Alnylam and INEX Accelerate Collaboration on Systemic Delivery of RNAi Therapeutics; Positive Results for Systemic RNAi Support Advancement to Next Phase of Collaboration - RNAi

Alnylam and INEX Accelerate Collaboration on Systemic Delivery of RNAi Therapeutics; Positive Results for Systemic RNAi Support Advancement to Next Phase of Collaboration
July 17, 2008
Alnylam
CAMBRIDGE, Mass. & VANCOUVER, British Columbia--(BUSINESS WIRE)--July 17, 2006--Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), a leading RNAi therapeutics company, and Inex Pharmaceuticals Corporation (TSX: IEX), announced today that based on progress made to date, the companies are accelerating their collaboration focused on the systemic delivery of RNAi therapeutics. Alnylam and INEX formed this collaboration in March 2006 to explore use of INEX's liposomal delivery technology for the systemic delivery of Alnylam's RNAi therapeutic products.
"Very rapidly, our scientific teams have made tremendous progress in achieving systemic delivery of Alnylam siRNAs formulated in INEX liposomes," said John Maraganore, Ph.D., President and Chief Executive Officer of Alnylam. "Given the results to date, we are confident that we will be able to achieve potent and specific gene silencing with systemically delivered siRNAs, and we are thrilled to move forward into the next, expanded phase of our collaboration with INEX, whom we view as having leading intellectual property and capabilities in the area of lipid-based drug delivery systems."
"We are enthusiastic to be working with Alnylam on solutions for the systemic delivery of RNAi therapeutics," said Timothy M. Ruane, President and Chief Executive Officer of INEX. "It is very gratifying to see such promising results after only a few months of working with Alnylam and we are pleased to enter the next phase of our collaboration ahead of schedule."
In March 2006, Alnylam and INEX formed an exclusive collaboration to evaluate the systemic delivery of RNAi therapeutic products using liposomes. The results to date encourage both parties to believe that Alnylam's RNAi therapeutics can be efficiently encapsulated in INEX's liposomes and effectively delivered to disease sites in animal models to carry out the desired effect of silencing the gene target.
As part of the collaboration, Alnylam has the option to take worldwide exclusive licenses to use INEX's technology for RNAi therapeutics directed to specific gene targets. Any exclusive license agreement would include upfront license fees, future milestone payments and royalties on product sales. Due to the rapid progress made so far, INEX and Alnylam have decided to move into the next phase of the collaboration, which includes looking at additional gene targets. In conjunction with this decision, Alnylam will make certain payments to INEX totaling approximately $1.9 million, $1.0 million of which relates to an option to take a broad exclusive license to INEX's technology. The remaining payments are for license option fees for certain named targets and support for the expanded research program.
About RNA Interference (RNAi)
RNA interference, or RNAi, is a naturally occurring mechanism within cells for selectively silencing and regulating specific genes. Since many diseases are caused by the inappropriate activity of specific genes, the ability to silence genes selectively through RNAi could provide a new way to treat a wide range of human diseases. RNAi is induced by small, double-stranded RNA molecules. One method to activate RNAi is with chemically synthesized small interfering RNAs, or siRNAs, which are double-stranded RNAs that are targeted to a specific disease-associated gene. The siRNA molecules are used by the natural RNAi machinery in cells to cause highly targeted gene silencing.
About Alnylam
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 building a pipeline of RNAi therapeutics; its lead program is in Phase 1 human clinical trials for the treatment of respiratory syncytial virus (RSV) infection, which is the leading cause of hospitalization in infants in the U.S. 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 Merck, Medtronic, and Novartis. The company, founded in 2002, maintains global headquarters in Cambridge, Massachusetts, and has an additional operating unit in Kulmbach, Germany. Alnylam is honored to be the "emerging/mid-cap" company recipient of the 2006 James D. Watson Helix Award, the biotechnology industry's award for outstanding achievement. For more information, visit www.alnylam.com.
About INEX
INEX is a Canadian biopharmaceutical company developing and commercializing proprietary drugs and drug delivery systems to improve the treatment of cancer. Further information about Inex and this news release can be found at www.inexpharm.com.
Alnylam Forward-Looking Statements
Various statements in this release concerning Alnylam's future expectations, plans, and prospects, including with respect to the development and systemic delivery of RNAi therapeutics, 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; Alnylam's ability to collaborate successfully with INEX and other collaborators; obtaining, maintaining, and protecting intellectual property utilized by Alnylam's products; 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; the successful development of Alnylam's product candidates, all of which are in early stages of development; obtaining regulatory approval for products; competition from others using technology similar to Alnylam's and others developing products for similar uses; Alnylam's dependence on INEX and other collaborators; and Alnylam's short operating history; as well as those risks more fully discussed in the "Risk Factors" section of Alnylam's most recent 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.