Hyperlipidemia

On December 12, we hosted an R&D Day in New York City. Alnylam management and key opinion leaders discussed the latest progress as well as plans for the future development of our RNAi therapeutics pipeline. At this event, we announced our pipeline growth strategy for development and commercialization of RNAi therapeutics across three Strategic Therapeutic Areas (STArs): Genetic Medicines, Cardio-metabolic Disease, and Hepatic Infectious Disease. [spotlight-link icon="podcast" href="http://edge.media-server.com/m/p/njherf95" type=" "] Listen to the webcast replay[/spotlight-link] [spotlight-link icon="presentation" href="http://www.alnylam.com/web/assets/MASTER-RD-DAY-DECK_Capella.pdf" type="(12.3 MB PDF)"] View the complete presentation[/spotlight-link]

We presented pre-clinical data from our investigational RNAi therapeutic programs toward genetically validated targets in development for the treatment of cardiovascular metabolic diseases, including: ALN-PCSsc targeting PCSK9 for the treatment of hypercholesterolemia; ALN-AC3 targeting apolipoprotein C3 (apoC3) for the treatment of hypertriglyceridemia; and ALN-ANG targeting angiopoietin-like 3 (ANGPTL3) for the treatment of genetic forms of mixed hyperlipidemia and severe hypertriglyceridemia.  The data, presented at the American Heart Association (AHA) Scientific Sessions 2014, included new pre-clinical multi-dose data in non-human primates (NHPs) with over six months of dosing for ALN-PCSsc, showing robust and clamped knockdown of PCSK9 of up to 92% and reductions in LDL-C of up to 77% with a once-monthly subcutaneous dosing regimen.  These studies confirm the potential for a once-monthly, and possibly once-quarterly, low volume subcutaneous dose regimen, thus highlighting the emerging profile of our ESC-GalNAc conjugate delivery technology. We believe that ALN-PCSsc represents an innovative, differentiated, and well-validated approach for the treatment of hypercholesterolemia. [spotlight-link icon="release" href="http://investors.alnylam.com/releasedetail.cfm?ReleaseID=883469" type=" "] Read our press release[/spotlight-link] [spotlight-link icon="presentation" href="http://www.alnylam.com/web/assets/CardioMetabolic_AHA_Poster_111714.pdf" type="(1.1 MB PDF)"] View our poster[/spotlight-link]

We announced a new program, ALN-AGT, an RNAi therapeutic targeting angiotensinogen (AGT) in development for the treatment of hypertensive disorders of pregnancy (HDP), including preeclampsia. Pre-clinical data were presented at the American Heart Association’s High Blood Pressure Research 2014 Scientific Sessions, held September 9 – 12, 2014, in San Francisco. Data from experiments in an established rat model of preeclampsia demonstrated that an RNAi therapeutic targeting AGT ameliorates the clinical sequelae of preeclampsia and improves outcomes for the fetus.  This treatment approach has the potential for selective delivery to the pregnant mother without fetal drug exposure, as our study confirmed undetectable siRNA levels in the fetus. [spotlight-link icon="release" href="http://investors.alnylam.com/releasedetail.cfm?ReleaseID=870551" type=" "] Read our press release[/spotlight-link] [spotlight-link icon="presentation" href="http://www.alnylam.com/web/assets/ALN-AGT_HBPR_presentation_09112014.pdf" type="(1.3 MB PDF)"] View our presentation[/spotlight-link] [spotlight-link icon="presentation" href="http://www.alnylam.com/web/assets/ALN-AGT_HBPR_poster_09112014.pdf" type="(1.3 MB PDF)"] View our poster[/spotlight-link]

We have presented new pre-clinical data from our growing pipeline of RNAi therapeutics for the treatment of cardiovascular metabolic disease, including ALN-PCSsc targeting PCSK9 for the treatment of hypercholesterolemia, and a newly named program, ALN-AC3 targeting apolipoprotein C-III (apoCIII) for the treatment of hypertriglyceridemia.  The data were presented in an oral presentation at the Arteriosclerosis, Thrombosis and Vascular Biology (ATVB) 2014 Scientific Sessions held May 1-3, 2014 in Toronto, Ontario. [spotlight-link icon="release" href="http://investors.alnylam.com/releasedetail.cfm?ReleaseID=844592" type=" "] Read our press release[/spotlight-link] [spotlight-link icon="presentation" href="http://www.alnylam.com/web/assets/ALNY-CardioMetabolicPrograms-ATVB-May2014.pdf" type="(1.3 MB PDF)"] View our presentation [/spotlight-link]

We presented new pre-clinical data with RNAi therapeutic programs for cardiovascular disease: ALN-PCSsc, an RNAi therapeutic targeting PCSK9 for the treatment of hypercholesterolemia; and ALN-ANG, an RNAi therapeutic targeting ANGPTL3 for the treatment of genetic forms of mixed hyperlipidemia and severe hypertriglyceridemia. These data were presented at the American Heart Association (AHA) Scientific Sessions held November 16 – 20, 2013 in Dallas, Texas. The data showed that subcutaneous administration of ALN-PCSsc led to an up to 95% knockdown of plasma PCSK9 and an up to 67% reduction of low density lipoprotein cholesterol (LDL-C) – in the absence of statin co-administration. In addition, new results for ALN-ANG demonstrated a greater than 95% reduction of triglycerides and greater than 85% reduction of LDL-C in a rodent model of mixed hyperlipidemia. [spotlight-link icon="release" href="http://investors.alnylam.com/releasedetail.cfm?ReleaseID=807877" type=" "] Read our press release[/spotlight-link] [spotlight-link icon="presentation" href="http://www.alnylam.com/web/assets/ALNY-AHAposterByPanel-Nov2013.pdf" type="(1.1 MB PDF)"] View our poster [/spotlight-link]

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