Alnylam Pharmaceuticals Inc (ALNY)

[MinnieM]

Alnylam’s ALN-VSP Abstract Published by American Society of Clinical Oncology (ASCO)

http://ih.advfn.com/p.php?pid=nmona&article=47741508&symbol=ALNY

Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), a leading RNAi therapeutics company, announced today that ASCO has published an abstract regarding its Phase I clinical trial with ALN-VSP, a systemically delivered RNAi therapeutic for the treatment of advanced solid tumors with liver involvement. The full results will be presented in a poster discussion session at the ASCO 2011 Annual Meeting being held June 3 – 7 in Chicago.

“We are encouraged by the data from our ALN-VSP program, as they represent key clinical results from our most advanced systemically delivered RNAi therapeutic product candidate. Several patients in the Phase I trial have advanced to an extension protocol, including one patient who has received drug for one full year – itself, a very important milestone for the advancement of RNAi therapeutics,” said Jared Gollob, M.D., Senior Director of Clinical Research at Alnylam. “Indeed, these data are not only important for the continued advancement of our ALN-VSP program, but they also continue to significantly increase our confidence in our entire platform of systemically delivered RNAi therapeutics, including ALN-TTR01 which is in a Phase I study for transthyretin-mediated amyloidosis, as well as our second generation LNP-based programs. We look forward to sharing additional safety and tolerability data, as well as data pertaining to biological and clinical activity, in a poster discussion presentation at ASCO next month.”

The poster, titled “Phase I dose-escalation study of ALN-VSP02, a novel RNAi therapeutic for solid tumors with liver involvement,” will be presented in the Developmental Therapeutics – Experimental Therapeutics poster discussion session being held on Saturday, June 4, 2011 from 2:00 – 6:00 p.m.

The interim results described in the abstract published today relate to data on the first 31 patients available as of December 2010. Additional results on all patients will be presented including safety and tolerability data – the primary objective of the study – and data related to the drug’s clinical activity. Recently, Alnylam announced that it has completed enrollment of new patients in this study. ALN-VSP was administered to 41 patients at doses ranging from 0.1 to 1.5 mg/kg, with multiple patients continuing to receive drug on study, including one patient who has now received therapy for one full year. Earlier this year, Alnylam presented interim clinical data from this study. Specifically, analysis of human tissue samples showed RNAi-mediated target mRNA cleavage, and thus proof of RNAi in man. In addition, measurement of ALN-VSP in human tissue biopsies showed pharmacologically relevant drug levels in both hepatic and extra-hepatic tumors.

About ALN-VSP

ALN-VSP is a systemically delivered RNAi therapeutic comprising two siRNAs designed to target two genes critical for the growth and development of cancer cells: vascular endothelial growth factor (VEGF) and kinesin spindle protein (KSP), also known as eglin 5 (Eg5). The Phase I trial is a multi-center, open label, dose escalation study in patients with advanced solid tumors with liver involvement who have failed to respond to or have progressed after standard treatment. The primary objective was to evaluate the safety, tolerability, and pharmacokinetics of intravenous ALN-VSP, including demonstration of the maximum tolerated dose. Other exploratory objectives include the assessment of tumor response through:

•Response Evaluation Criteria for Solid Tumors (RECIST), a set of published guidelines that define when cancer patients’ disease improves, stabilizes or progresses during treatment;
•change in tumor blood flow or vascular permeability as measured by DCE-MRI; and,
•analysis of pharmacodynamic effects of ALN-VSP on tumors as measured in patients electing to proceed with voluntary pre- and post-treatment biopsies.
ALN-VSP is Alnylam's first systemic RNAi program and represents the company's first clinical program in oncology. The drug is formulated using a first generation lipid nanoparticle developed by Tekmira Pharmaceuticals Corporation. The company expects to partner its ALN-VSP program prior to initiating a Phase II clinical study, with the goal of initiating this study in 2012.

About Liver Cancers

Cancer affecting the liver, known as either primary or secondary liver cancer, is associated with one of the poorest survival rates in oncology and represents a major unmet medical need affecting a large number of patients worldwide. Primary liver cancer, or hepatocellular carcinoma (HCC), is one of the most common cancers worldwide, with more than 600,000 people diagnosed each year. Secondary liver cancer, also known as metastatic liver cancer, is cancer that spreads to the liver from another part of the body due to other common cancers like colon, lung, or breast cancer. Worldwide, more than 500,000 people are diagnosed with secondary liver cancer each year.

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 leading the translation of RNAi as a new class of innovative medicines with a core focus on RNAi therapeutics for the treatment of genetically defined diseases, including ALN-TTR for the treatment of transthyretin-mediated amyloidosis (ATTR), ALN-PCS for the treatment of severe hypercholesterolemia, and ALN-HPN for the treatment of refractory anemia. As part of its “Alnylam 5x15TM” strategy, the company expects to have five RNAi therapeutic products for genetically defined diseases in advanced stages of clinical development by the end of 2015. Alnylam has additional partner-based programs in clinical or development stages, including ALN-RSV01 for the treatment of respiratory syncytial virus (RSV) infection, ALN-VSP for the treatment of liver cancers, and ALN-HTT for the treatment of Huntington’s disease. The company’s leadership position on RNAi therapeutics and intellectual property have enabled it to form major alliances with leading companies including Merck, Medtronic, Novartis, Biogen Idec, Roche, Takeda, Kyowa Hakko Kirin, and Cubist. In addition, Alnylam and Isis co-founded Regulus Therapeutics Inc., a company focused on discovery, development, and commercialization of microRNA therapeutics; Regulus has formed partnerships with GlaxoSmithKline and sanofi-aventis. Alnylam has also formed Alnylam Biotherapeutics, a division of the company focused on the development of RNAi technologies for application in biologics manufacturing, including recombinant proteins and monoclonal antibodies. Alnylam scientists and collaborators have published their research on RNAi therapeutics in over 100 peer-reviewed papers, including many in the world’s top scientific journals such as Nature, Nature Medicine, Nature Biotechnology, and Cell. 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, including without limitation, statements regarding Alnylam's views with respect to the potential for RNAi therapeutics, including ALN-VSP, its plan to disclose additional data from its ALN-VSP clinical trial, its plan to partner its ALN-VSP program prior to initiating a Phase II clinical study, its goal with respect to the timing of initiating such study, and Alnylam’s expectations regarding its “Alnylam 5x15” product strategy, 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, Alnylam’s ability to discover and develop novel drug candidates, successfully demonstrate the efficacy and safety of its drug candidates, including ALN-VSP, in human clinical trials and establish and maintain strategic business alliances, including a partnership for the continued development of ALN-VSP, and new business initiatives, 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.