Presentations

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]

On July 11, we hosted an R&D Day in New York City. Alnylam scientists and management reviewed progress with our Alnylam 5x15 pipeline for the development and commercialization of RNAi therapeutics. The event featured presentations by experts in the following disease areas: TTR-Mediated Amyloidosis, Hemophilia and Rare Bleeding Disorders, and Acute Intermittent Porphyria. [spotlight-link icon="podcast" href="http://investors.alnylam.com/eventdetail.cfm?eventid=131417" type=" "] Listen to the webcast replay[/spotlight-link] [spotlight-link icon="presentation" href="http://www.alnylam.com/web/assets/Alnylam-RDDayPres-Capella-July2013.pdf" type="(3.4 MB PDF)"] View the complete presentation[/spotlight-link] TTR-Mediated Amyloidosis [spotlight-link icon="podcast" href="http://www.alnylam.com/web/assets/RDday-2013/alnylam_podcast_RDDay-ATTRAmyloidosisOverview-July2013.mp3" type="(24.7 MP3)"] Listen to Dr. Philip Hawkins discuss TTR-Mediated Amyloidosis[/spotlight-link][spotlight-link icon="presentation" href="http://www.alnylam.com/web/assets/RDday-2013/Alnylam-RDDay-ATTRAmyloidosisOverview-July2013.pdf" type="(1.8 MB PDF)"] View the presentation[/spotlight-link][spotlight-link icon="podcast" href="http://www.alnylam.com/web/assets/RDday-2013/alnylam_podcast_RDDay-ALN-TTR-ProgramOverview-July2013.mp3" type="(25.4 MP3)"] Listen to the ALN-TTR program overview[/spotlight-link][spotlight-link icon="presentation" href="http://www.alnylam.com/web/assets/RDday-2013/Alnylam-RDDay-ALN-TTR-ProgramOverview-July2013.pdf" type="(0.6 MB PDF)"] View the presentation[/spotlight-link] Hemophilia and Rare Bleeding Disorders (RBD) [spotlight-link icon="podcast" href="http://www.alnylam.com/web/assets/RDday-2013/alnylam_podcast_RDDay-HemophiliaAndRDBsOverview-July2013.mp3" type="(28.6 MP3)"] Listen to Dr. Craig Kessler discuss Hemophilia and RBDs[/spotlight-link][spotlight-link icon="presentation" href="http://www.alnylam.com/web/assets/RDday-2013/Alnylam-RDDay-HemophiliaAndRDBsOverview-July2013.pdf" type="(0.8 MB PDF)"] View the presentation[/spotlight-link]

We presented new pre-clinical with ALN-AT3, an RNAi therapeutic targeting antithrombin (AT) for the treatment of hemophilia and rare bleeding disorders (RBD) at the XXIV Congress of the International Society of Thrombosis and Haemostasis (ISTH) being held June 29 – July 4. These data demonstrate that ALN-AT3 can normalize thrombin generation and improve hemostasis in hemophilia mice and fully correct thrombin generation in a non-human primate (NHP) hemophilia “inhibitor” model. [spotlight-link icon="presentation" href="http://www.alnylam.com/web/assets/ALN-AT3-preclin-ISTH-July2013.pdf" type="(900 KB PDF)"] View our presentation [/spotlight-link] [spotlight-link icon="release" href="http://investors.alnylam.com/releasedetail.cfm?ReleaseID=774899" type=" "] Read our press release[/spotlight-link]

We presented positive data from our Phase II clinical trial of ALN-TTR02 for the treatment of transthyretin-mediated amyloidosis (ATTR) at the Biennial Meeting of the Peripheral Nerve Society being held June 29 – July 3. Interim results show that multiple doses of ALN-TTR02 led to robust and statistically significant knockdown of serum TTR protein levels of up to 93%. 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, ALN-TTR02 was found to be generally safe and well tolerated in this study. [spotlight-link icon="presentation" href="http://www.alnylam.com/web/assets/ALN-TTR02-PhaseII-InterimResults-June2013.pdf" type="(0.7 MB PDF)"] View our presentation [/spotlight-link] [spotlight-link icon="presentation" href="http://www.alnylam.com/web/assets/ALN-TTR02-PhII-PNSposter-130630.pdf" type="(0.7 MB PDF)"] View our poster [/spotlight-link] [spotlight-link icon="release" href="http://investors.alnylam.com/releasedetail.cfm?ReleaseID=774605" type=" "] Read our press release[/spotlight-link] 

We have presented pre-clinical data with ALN-CC5, an RNAi therapeutic for the treatment of complement-mediated diseases, at the 6th International Conference on Complement Therapeutics being held June 18 – 23, 2013. The data showed that administration of a GalNAc-siRNA conjugate targeting the C5 component of complement resulted in potent, dose-dependent, and durable silencing of C5 liver mRNA, knockdown of C5 serum protein levels, and inhibition of complement-mediated hemolytic activity in rodents. [spotlight-link icon="presentation" href="http://www.alnylam.com/web/assets/Alnylam-ALN-CC5-ComplementTxConf-130620.pdf" type="(0.8 MB PDF)"] View our presentation [/spotlight-link] [spotlight-link icon="release" href="http://investors.alnylam.com/releasedetail.cfm?ReleaseID=772627" 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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