Key Developments For Alnylam Pharmaceuticals, Inc.
Alnylam Pharmaceuticals, Inc. (ALNY.O) (Consolidated Issue listed on NASDAQ Global Market)
Alnylam Pharmaceuticals, Inc. Presents New Pre Clinical Data On ALN-VSP
Alnylam Pharmaceuticals, Inc. announced new pre clinical data from its ALN-VSP program presented at the AACR-NCI-EORTC Molecular Targets and Cancer Therapeutics International conference being held November 15 to 19, 2009 in Boston, Mass. ALN-VSP is an RNAi therapeutic currently in a Phase I clinical trial for the treatment of liver cancers, including hepatocellular carcinoma (HCC) and other solid tumors with liver involvement. The new data demonstrated robust anti-tumor activity in orthotopic liver tumor models comprised of both HCC- and colorectal cancer-derived cell lines. ALN-VSP was also shown to act on disseminated tumors outside of the liver. Further, in addition to anti-proliferative effects, studies showed that ALN-VSP exerts a potent anti-angiogenic effect on tumors. The Company Scientist presented the following data: ALN-VSP-mediated silencing of KSP in both HCC and colorectal carcinoma models, resulting in the accumulation in tumor cells of aberrant mitotic figures, also known as monoasters, a hallmark of KSP inhibition; monoaster formation in tumor cells within lymph node metastases derived from the orthotopic liver tumors, demonstrating the ability of LNPs in general, and ALN-VSP in particular, to achieve effective delivery in extra-hepatic tumor sites; marked anti-angiogenic effects resulting from ALN-VSP treatment, including reductions in both tumor microvessel density and intratumoral hemorrhage; and similar anti-angiogenic results.
Alnylam Pharmaceuticals, Inc. Announces NewPre Clinical Data On RNAi Therapeutics At Oligonucleotides Therapeutics Society (OTS) Meeting
Alnylam Pharmaceuticals, Inc. announced that it has presented new data related to its overall delivery research efforts including the rational design of Mimetic Lipoprotein Particles, or MLPs, a technology for the systemic delivery of small interfering RNAs, or siRNAs, the molecules that mediate RNAi. In addition, Alnylam scientists and collaborators presented additional new data on systemic delivery and provided an update on RNA activation (RNAa) technology. In a poster titled Lipophilic siRNA Delivery by Reconstituted Lipoprotein Particles In Vivo Tomoko Nakayama, Ph.D., Associate Director, Research at Alnylam, presented for the first time the rational design and characterization of MLPs as an RNAi delivery platform. MLPs were designed to mimic the physiologic properties of endogenous lipoprotein particles and were engineered using recombinant human apolipoprotein A1 (rh-apoA1) or recombinant human apolipoprotein E (rh-apoE), phosphatidylcholine, and a cholesterol-conjugated siRNA (chol-siRNA). The resulting MLPs had biophysical properties comparable to normal high density lipoprotein (HDL, or good cholesterol) particles including a mean diameter size of approximately 10 nm. The stoichiometry for siRNA:particle loading ratio was observed to be 1:1. The reported in vivo studies were performed in mice using MLPs with chol-siRNAs targeting apolipoprotein B (apoB), the major apolipoprotein involved in the metabolism of low-density lipoprotein (LDL, or bad) cholesterol.
Alnylam Pharmaceuticals, Inc. and Roche Holding Ltd. Advance RNAi Therapeutic Collaboration Phase Of 2007 Alliance
Alnylam Pharmaceuticals, Inc. announced that it has advanced to the RNAi therapeutic collaboration stage of its alliance with Roche Holding Ltd. formed in 2007. In this phase of the collaboration, the partners will jointly collaborate on the discovery and development of specific RNAi therapeutic products and each will contribute key delivery technologies in the new disease target-focused effort. New delivery technologies include Alnylam lipid nanoparticles and Roche Madison dynamic polyconjugate delivery technologies. Alnylam and Roche will co-develop and co-commercialize RNAi therapeutic products in the U.S. market and Alnylam is eligible to receive additional milestone and royalty payments for products developed in the rest of world.
Alnylam Pharmaceuticals, Inc. Announces New Pre Clinical Data On ALN-TTR
Alnylam Pharmaceuticals, Inc. announced that it has presented new pre-clinical research findings from its transthyretin (TTR)-mediated amyloidosis (ATTR) program at the 60th Annual Meeting of the American Association for the Study of Liver Diseases (The Liver Meeting). Alnylam is developing ALN-TTR, a systemically delivered RNAi therapeutic targeting the TTR gene for the treatment of ATTR, including familial amyloidotic cardiomyopathy (FAC) and familial amyloidotic polyneuropathy (FAP). There are more than 100 mutations that have been identified in the TTR gene. ALN-TTR targets a region of the gene common to wild-type and all known mutant forms of TTR, and therefore, has potential as a therapeutic for all patients with FAC and FAP. The new pre-clinical research findings presented at the meeting demonstrated dose-dependent ALN-TTR reduction of liver TTR messenger (mRNA) and serum TTR protein levels by greater than 80% in transgenic mice and non-human primates, with gene silencing effects found to be durable for more than three weeks following a single dose administration.
Alnylam Pharmaceuticals, Inc. and Isis Pharmaceuticals, Inc. Founded Regulus Therapeutics Inc. Announces U.S. Allowance Of Fundamental microRNA Therapeutics Patent Application
Regulus Therapeutics Inc., founded by Alnylam Pharmaceuticals, Inc. and Isis Pharmaceuticals, Inc. announced that the United States Patent and Trademark Office (USPTO) has allowed a patent application within the 'Esau' patent family (US Application Serial No. 10/909,125). The Esau patent estate, which is owned exclusively by Regulus, represents one of the earliest filings in the microRNA intellectual property landscape, and is fundamental to the discovery, development, and commercialization of microRNA therapeutics.
