Health & Fitness
Health and Fitness
Health and Fitness
Fri, May 21, 2010
Thu, May 20, 2010 Alnylama?s ALN-VSP Abstract Published by American Society of Clinical Oncology (ASCO)
CAMBRIDGE, Mass.--([ BUSINESS WIRE ])--Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), a leading RNAi therapeutics company, announced today that ASCO has published its abstract pertaining to preliminary results from an ongoing Phase I trial with ALN-VSP, a systemically delivered RNAi therapeutic for the treatment of advanced solid tumors with liver involvement. The results will be presented in a poster at the ASCO 2010 Annual Meeting being held June 4 a" 8 in Chicago.
"a major scientific breakthrough that happens once every decade or so"
aWe are encouraged by the results we have seen with ALN-VSP in this Phase I trial to date,a said Akshay Vaishnaw, M.D., Ph.D., Senior Vice President, Clinical Research at Alnylam. aThis study is aimed at defining the maximum tolerated dose, which has not yet been reached. This trial is actively enrolling and we look forward to sharing additional safety and tolerability data, as well as data pertaining to biological and clinical activity, in a poster presentation during the ASCO meeting.a
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 designed to enroll approximately 55 patients with advanced solid tumors with liver involvement who have failed to respond to or have progressed after standard treatment. The trial is evaluating eight potential dose levels from 0.1 to 1.7 mg/kg. The primary objective is 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 patientsa™ disease improves, stabilizes or progresses during treatment;
- change in tumor blood flow or vascular permeability as measured by DCE-MRI;
- change in plasma biomarkers of angiogenesis; and,
- analysis of pharmacodynamic effects of ALN-VSP on tumors as measured in patients electing to proceed with voluntary pre- and post-treatment biopsies.
The interim results described in the abstract submitted to ASCO and published today ([ http://abstract.asco.org/AbstView_74_51118.html ]) relate to data available as of December, 2009. Additional interim results, including results from patients enrolled after this date, will be presented in a poster at the ASCO meeting, and include safety and tolerability data - the primary objective of the study - and data related to certain secondary objectives, such as pharmacodynamic activity measured through serial DCE-MRI and biomarkers of angiogenesis.
The poster, titled aInterim safety and pharmacodynamic results for ALN-VSP02, a novel RNAi therapeutic for solid tumors with liver involvement,a will be presented in the Developmental Therapeutics a" Experimental Therapeutics poster session being held on Monday, June 7 from 8:00 a.m. to 12:00 p.m. in S Hall A2.
ALN-VSP is Alnylama™s first systemic RNAi program and represents the companya™s first clinical program in oncology. The drug is formulated in a first generation lipid nanoparticle, known as stable nucleic acid-lipid particle (SNALP), developed by Tekmira Pharmaceuticals Corporation.
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 aa major scientific breakthrough that happens once every decade or so,a 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 Alnylama™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 worlda™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 Huntingtona™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 companya™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 joint owners of Regulus Therapeutics Inc., a company focused on the discovery, development, and commercialization 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 Alnylama™s future expectations, plans and prospects, including without limitation, Alnylama™s views with respect to the potential for RNAi therapeutics, including ALN-VSP, its expectations with respect to the timing and success of its clinical trial of ALN-VSP, and its plan to disclose additional data from that clinical trial, 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 Alnylama™s ability to discover and develop novel drug candidates, such as ALN-VSP for the treatment of liver cancers, successfully demonstrate the efficacy and safety of ALN-VSP and other drug candidates in human clinical trials, as well as those risks more fully discussed in the aRisk Factorsa 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 Alnylama™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.
