Spotlight

We have reported positive initial data from our Phase 2 open-label extension (OLE) study with patisiran (ALN-TTR02), an RNAi therapeutic targeting transthyretin (TTR) in development for the treatment of TTR-mediated amyloidosis (ATTR).  Data were presented at the International Symposium on Amyloidosis, held April 27 – May 1, 2014.  Preliminary results showed that multiple doses of patisiran achieved sustained knockdown of serum TTR protein levels at the approximately 80% target level through 168 days.  Moreover, OLE results showed a favorable tolerability profile with up to eight doses administered.  These data provide the first clinical evidence of sustained RNAi-mediated TTR knockdown in FAP patients beyond two doses of patisiran.     [spotlight-link icon="release" href="http://investors.alnylam.com/releasedetail.cfm?ReleaseID=842968" type=" "] Read our press release[/spotlight-link] [spotlight-link icon="presentation" href="http://www.alnylam.com/web/assets/ALNY-Clinical-Update-Patisiran-Phase2-Trials-ISA2014.pdf" type="(0.8 MB PDF)"] View the patisiran Phase 2/OLE presentation [/spotlight-link] [spotlight-link icon="presentation" href="http://www.alnylam.com/web/assets/ALNY-FAP-NaturalHistoryStudy-ISA2014.pdf" type="(0.8 MB PDF)"] View the FAP Natural History Study 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]

We presented positive data from our Phase II clinical trial of patisiran (ALN-TTR02) for the treatment of transthyretin-mediated amyloidosis (ATTR) at the International Symposium on Familial Amyloidotic Polyneuropathy, November 10 – 13, 2013. Results showed that multiple doses of patisiran led to robust and statistically significant knockdown of serum TTR protein levels of up to 96%, with mean levels of TTR knockdown exceeding 85%.  Knockdown of TTR, the disease-causing protein in ATTR, was found to be rapid, dose dependent, and durable, and similar activity was observed toward both wild-type and mutant protein.   In addition, patisiran was found to be generally safe and well tolerated in this study. [spotlight-link icon="presentation" href="http://www.alnylam.com/web/assets/ALNY-ISFAP-ALN-TTRprogram-Nov2013.pdf" type="(0.9 MB PDF)"] View our presentation [/spotlight-link] [spotlight-link icon="release" href="http://investors.alnylam.com/releasedetail.cfm?ReleaseID=805999" type=" "] Read our press release[/spotlight-link]

We presented new pre-clinical data on ALN-AAT, an RNAi therapeutic targeting alpha-1 antitrypsin (AAT) in development for the treatment of liver disease associated with AAT deficiency. These data were presented at the 64th Annual Meeting of the American Association for the Study of Liver Diseases (AASLD, “The Liver Meeting”) held November 1 – 5, 2013. In a transgenic mouse model of Z-AAT protein over-expression, subcutaneous administration of a GalNAc-siRNA targeting AAT led to rapid, potent, dose-dependent, and durable knockdown of AAT of greater than 90%, as well as a significant reduction in fibrosis and the incidence of liver tumors. [spotlight-link icon="release" href="http://investors.alnylam.com/releasedetail.cfm?ReleaseID=803892" type=" "] Read our press release[/spotlight-link] [spotlight-link icon="presentation" href="http://www.alnylam.com/web/assets/ALNY-LiverMeeting-AATProgram-PosterXPanel-Nov2013.pdf" type="(1.3 MB PDF)"] View our poster [/spotlight-link]

We presented new pre-clinical data supporting the selection of the ALN-AS1 Development Candidate for the treatment of hepatic porphyrias, including acute intermittent porphyria (AIP). The new research, presented at the 9th Annual Meeting of the Oligonucleotide Therapeutics Society, held October 6 – 8, 2013 in Naples, Italy, showed that multi-dose administration of a GalNAc-siRNA targeting ALAS-1 led to rapid, dose-dependent, and long-lasting knockdown of the ALAS-1 mRNA in non-human primates, with an ED50 of approximately 1.25 mg/kg.  Further, in a rat model of AIP, ALN-AS1 administration at doses as low as 2.5 mg/kg resulted in a complete blunting of phenobarbital-induced over-production of PBG and ALA, the toxic heme intermediates in AIP. [spotlight-link icon="release" href="http://investors.alnylam.com/releasedetail.cfm?ReleaseID=795424" type=" "] Read our press release[/spotlight-link] [spotlight-link icon="presentation" href="http://www.alnylam.com/web/assets/ALNY-ALN-AS1update-OTS2013.pdf" type="(1.1 MB PDF)"] View our ALN-AS1 presentation [/spotlight-link] [spotlight-link icon="presentation" href="http://www.alnylam.com/web/assets/ALNY-ConjugateUpdate-OTS2013.pdf" type="(1.3 MB PDF)"] View our GalNAc-siRNA conjugate presentation [/spotlight-link]

We have reported positive clinical results from our Phase I trial of ALN-TTRsc, a subcutaneously administered RNAi therapeutic targeting transthyretin (TTR) for the treatment of TTR-mediated amyloidosis (ATTR). Data were presented at the Heart Failure Society of America 17th Annual Scientific Meeting being held September 22 – 25. These results demonstrated that ALN-TTRsc achieved robust, consistent, and statistically significant (p<0.01) knockdown of serum TTR protein levels of up to 94%. In addition, knockdown of TTR was found to be rapid, dose-dependent, and durable.  ALN-TTRsc was found to be generally safe and well tolerated in this study. [spotlight-link icon="presentation" href="http://www.alnylam.com/web/assets/ALN-TTRsc-PhaseI-Results-HFSA-Sept2013.pdf" type="(1.8 MB PDF)"] View our presentation [/spotlight-link] [spotlight-link icon="presentation" href="http://www.alnylam.com/web/assets/ALN-TTRsc-PhI-HFSA-Poster-Sep2013.pdf" type="(0.7 MB PDF)"] View our poster [/spotlight-link] [spotlight-link icon="release" href="http://investors.alnylam.com/releasedetail.cfm?ReleaseID=792320" type=" "] Read our press release[/spotlight-link]

We presented pre-clinical proof-of-concept data from our ALN-AS1 program for the treatment of acute intermittent porphyria (AIP) at the International Congress of Porphyrins and Porphyrias being held May 16 – 18, 2013. The research, presented by Alnylam scientists and collaborators at the Icahn School of Medicine at Mount Sinai in New York City, showed that administration of a GalNAc-conjugated siRNA targeting aminolevulinate synthase-1 (ALAS-1) blocked production of aminolevulinic acid (ALA) and porphobilinogen (PBG), the toxic precursors that cause disease symptoms and pathophysiology, in a mouse model of AIP. [spotlight-link icon="presentation" href="http://www.alnylam.com/web/assets/ALNY-Porphyria-AS1-Pres-May17-2013.pdf" type="(1.6 MB PDF)"] View our presentation [/spotlight-link] [spotlight-link icon="release" href="http://investors.alnylam.com/releasedetail.cfm?ReleaseID=765697" type=" "] Read our press release[/spotlight-link]

We initiated dosing in our Phase I clinical trial with ALN-TTRsc, an RNAi therapeutic targeting transthyretin (TTR) for the treatment of TTR-mediated amyloidosis (ATTR).  The Phase I trial of ALN-TTRsc is being conducted in the U.K. as a randomized, double-blind, placebo-controlled, single- and multi-dose, dose-escalation study, enrolling up to 40 healthy volunteer subjects.  The primary objective of the study is to evaluate the safety and tolerability of single and multiple doses of subcutaneously administered ALN-TTRsc. [spotlight-link icon="release" href="http://investors.alnylam.com/releasedetail.cfm?ReleaseID=749037" type=" "] Read our press release[/spotlight-link][spotlight-link icon="release" href="http://www.alnylam.com/our-pipeline/clinical-trials/" type=" "] Learn more about our ALN-TTR program[/spotlight-link]

We have published complete study results from our Phase I clinical trial with ALN-VSP, an RNAi therapeutic for the treatment of advanced solid tumors with liver involvement. Results from the study – the most comprehensive study of a systemically administered RNAi therapeutic to date – demonstrated proof of RNAi activity in man and evidence of anti-tumor activity, in addition to highlighting the safety and tolerability of multiple doses of ALN-VSP. [spotlight-link icon="release" href="http://cancerdiscovery.aacrjournals.org/content/early/2013/01/26/2159-8290.CD-12-0429"  type=" "] Read the abstract in Cancer Discovery[/spotlight-link][spotlight-link icon="release" href="http://investors.alnylam.com/releasedetail.cfm?ReleaseID=834148"  type=" "] Read our press release[/spotlight-link][spotlight-link icon="presentation" href="http://www.alnylam.com/capella/presentations/aln-vsp-data-presentation-asco-2011/" type="(2.3 MB PDF)"] View our ALN-VSP presentation at ASCO 2011 [/spotlight-link][spotlight-link icon="presentation" href="http://www.alnylam.com/capella/presentations/aln-vsp-asco2012/" type="(2.3 MB PDF)"] View our ALN-VSP presentation at ASCO 2012 [/spotlight-link]

Today we are presenting new data from our ALN-AT3 program for the treatment of hemophilia and other bleeding disorders at the 54th American Society of Hematology (ASH) Annual Meeting in Atlanta. ALN-AT3 comprises part of our “Alnylam 5x15™” strategy, which is aimed at bringing innovative medicines to patients, with a focus on RNAi therapeutics toward genetically defined targets for diseases with very high unmet medical need. [spotlight-link icon="presentation" href="http://www.alnylam.com/web/assets/ALNY-ASH-AT3posterXpanel-Dec2012-r.pdf" type="(2.3 MB PDF)"] View our poster [/spotlight-link] [spotlight-link icon="release" href="http://investors.alnylam.com/releasedetail.cfm?ReleaseID=734078"  type=" "] Read our press release[/spotlight-link]

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