Healthcare Industry News:  ALN-VSP01 

Biopharmaceuticals Oncology

 News Release - April 17, 2007

Alnylam Announces Advancement of a New RNAi Therapeutic Development Program for the Treatment of Liver Cancer

Pre-clinical In Vivo Data Show RNAi Achieves Gene Silencing of Two Cancer Pathway Genes

Company Expects to File IND for ALN-VSP01 in 2008

CAMBRIDGE, Mass.--(HSMN NewsFeed)--Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY ), a leading RNAi therapeutics company, announced today at the American Association for Cancer Research (AACR) 2007 Annual Meeting that it will advance a systemically delivered RNAi therapeutic, ALN-VSP01, for the treatment of liver cancer and potentially other solid tumors as a new development program. ALN-VSP01 is an RNAi therapeutic that is designed to target vascular endothelial growth factor (VEGF) and kinesin spindle protein (KSP), two well-validated genes that are involved in distinct pathways of tumor pathology, cell proliferation and angiogenesis, in a wide variety of cancers. Alnylam expects to submit an investigational new drug (IND) application for this program in 2008.

"We believe that there is real potential to develop an RNAi therapeutic for the treatment of liver cancer based on our encouraging progress with systemic delivery of siRNAs toward liver-expressed target genes," said John Maraganore, Ph.D., President and Chief Executive Officer of Alnylam. "Primary liver cancer is the world's most common solid tumor type and is associated with one of the poorest survival rates in cancer. Our encouraging pre-clinical data demonstrate the ability of siRNAs to silence two well-validated genes critical for tumor proliferation and survival, and our strategy of using an RNAi therapeutic that targets these two distinct pathways is aimed at increasing the likelihood of achieving meaningful clinical benefit for patients."

Data announced during a symposium titled "RNAi Therapies" at AACR showed the ability of RNAi therapeutics to silence both VEGF and KSP expression in the liver and to stop cancer cell proliferation by targeting KSP. Specifically, the pre-clinical data showed:

  • Durable and rapid reduction in vivo of VEGF protein by more than 50 percent after a single injection of a VEGF-specific siRNA in a rodent model;
  • Silencing of KSP mRNA in vivo by greater than 50 percent after a single injection of a KSP-specific siRNA in a rodent model; and
  • Halting of human cancer cell proliferation in vitro when KSP was silenced by a target-specific siRNA.

Alnylam's RNAi therapeutic, ALN-VSP01, is comprised of two small interfering RNAs, or siRNAs, the molecules that mediate RNAi. Each siRNA targets a distinct and well-validated gene in the growth and proliferation of tumors: VEGF, a key mediator of tumor angiogenesis; and KSP, a protein required for cell division that, when inhibited, leads to cell arrest and cell death in dividing cells. ALN-VSP01 is formulated using Alnylam's novel liposomal formulation technology, which has been used to achieve delivery of siRNAs to cells of the liver and efficient silencing of genes expressed in that organ. Liposomal delivery can also be optimized for delivery to other organs and tumor types, such that ALN-VSP01 may ultimately have potential for a range of solid tumors. Alnylam's collaborator, Inex Pharmaceuticals Corporation, is manufacturing the lipid-based formulation as the company prepares for pre-IND toxicology studies with ALN-VSP01.

"Despite many advances in the treatment of cancer with targeted small molecules and antibody therapeutics, the clinical impact of these therapies is generally measured in only months of survival benefit. New modalities like RNAi therapeutics offer the potential to advance cancer therapy to a whole new level," said George D. Demetri, M.D., of Dana-Farber Cancer Institute and Harvard Medical School. "I'm encouraged by Alnylam's pre-clinical data demonstrating the ability to silence disease pathway genes with delivery of RNAi therapeutics, and believe that the application of this approach for the treatment of primary and secondary liver cancers holds considerable promise."

About Liver Cancer

Primary liver cancer is cancer that forms in the tissues of the liver. Worldwide, primary liver cancer is the most common solid organ tumor, with more than 600,000 people diagnosed each year. The incidence of primary liver cancer is increasing due to underlying liver disease such as hepatitis infection and alcoholism. The overall five-year relative survival rate from primary liver cancer is about 10 percent, making it the cancer with the second poorest five-year survival rate. Liver cancer may be treated with surgery, radiation therapy or chemotherapy. The liver is also a common site for metastasis in other types of cancer, also known as secondary liver cancer. More than 500,000 people worldwide are diagnosed with secondary liver cancer each year; up to 50 percent of liver metastases are of colorectal cancer origin, while the remainder metastasize from a wide variety of primary cancer sites including breast and kidney.

About RNA Interference (RNAi)

RNA interference (or RNAi) is a naturally occurring mechanism within cells for selectively silencing and regulating specific genes. The discovery of RNAi has been widely acknowledged as a major breakthrough in biology, and the technology was recognized for its potential broad impact in medicine with the award of the 2006 Nobel Prize for Physiology or Medicine. Since many diseases are caused by the inappropriate activity of specific genes, the ability to silence genes selectively through RNAi has accelerated the understanding of these genes and their related pathways. Additionally, 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 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 I human clinical trials for the treatment of respiratory syncytial virus (RSV) infection. RSV infects nearly every child at least once by the age of two and accounts for more than 100,000 hospitalizations annually in the U.S. pediatric population. RSV infection also poses a great risk to the elderly and other adults with compromised immune systems. 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, Novartis, and Biogen Idec. The company, founded in 2002, maintains global headquarters in Cambridge, Massachusetts, and has an additional operating unit in Kulmbach, Germany. For more information, visit

Alnylam Forward-Looking Statements

Various statements in this release concerning our future expectations, plans, and prospects, including statements concerning the timing of filing an IND for ALN-VSP01, statements concerning use of RNAi therapeutics for the treatment of liver and other cancers, statements concerning use of siRNAs to achieve therapeutic silencing of genes and statements concerning effective delivery of siRNAs, 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 risks related to: our approach to discover and develop novel drugs, which is unproven and may never lead to marketable products; our ability to fund and the results of further pre-clinical and clinical trials; obtaining, maintaining and protecting intellectual property utilized by our products; our ability to enforce our patents against infringers and to defend our patent portfolio against challenges from third parties; our ability to obtain additional funding to support our business activities; our dependence on third parties for development, manufacture, marketing, sales, and distribution of products; the successful development of our product candidates, all of which are in early stages of development; obtaining regulatory approval for products; competition from others using technology similar to ours and others developing products for similar uses; our dependence on collaborators; and our short operating history; as well as those risks more fully discussed in the "Risk Factors" section of our most recent report on Form 10-K on file with the Securities and Exchange Commission. In addition, any forward-looking statements represent our views only as of today and should not be relied upon as representing our views as of any subsequent date. We do not assume any obligation to update any forward-looking statements.

Source: Alnylam Pharmaceuticals

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