Hemophilia

We reported new results from an exploratory analysis of our Phase 1 study with fitusiran, an investigational RNAi therapeutic, in patients with hemophilia A or B with or without inhibitors at the 10th Annual Congress of the European Association of Haemophilia and Allied Disorders (EAHAD) held February 1 – 3, 2017 in Paris, France. [spotlight-link icon="release" href="http://investors.alnylam.com/releasedetail.cfm?ReleaseID=1010213" type=" "]Read our press release[/spotlight-link] [spotlight-link icon="presentation" href="http://www.alnylam.com/web/assets/EAHAD-2017_Management-of-Bleed-Events.pdf" type="(450 KB PDF)"]View the bleed management presentation[/spotlight-link] [spotlight-link icon="presentation" href="http://www.alnylam.com/web/assets/EAHAD-2017_Stability.pdf" type="(690 KB PDF)"]View the stability study presentation[/spotlight-link]

We reported positive interim results from our Phase 1 study of fitusiran in patients with hemophilia with inhibitors as well as from our Phase 2 open-label extension (OLE) study in hemophilia patients without inhibitors at the 58th Annual Meeting of the American Society of Hematology (ASH), held December 3 – 6, 2016 in San Diego, California. [spotlight-link icon="release" href="http://investors.alnylam.com/releasedetail.cfm?ReleaseID=1002502" type=" "]Read our press release on hemophilia patients with inhibitors[/spotlight-link] [spotlight-link icon="presentation" href="http://www.alnylam.com/web/assets/ASH-2016_Fitusiran_INHIBITORS_03Dec2016_CAPELLA.pdf" type="(320 KB PDF)"]View the inhibitor presentation[/spotlight-link] [spotlight-link icon="release" href="http://investors.alnylam.com/releasedetail.cfm?ReleaseID=1002510" type=" "]Read our press release on fitusiran Phase 2 OLE [/spotlight-link] [spotlight-link icon="presentation" href="http://www.alnylam.com/web/assets/ASH-2016_Fitusiran_NONINHIBITORS_04Dec2016_CAPELLA2.pdf" type="(460 KB PDF)"]View the Phase 2 OLE presentation[/spotlight-link]

We reported new data from Parts C and D of our Phase 1 study with fitusiran, an investigational RNAi therapeutic targeting antithrombin (AT) for the treatment of hemophilia and rare bleeding disorders, at the World Federation of Hemophilia (WFH) World Congress, held July 24-28, 2016 in Orlando, Florida. [spotlight-link icon="release" href="http://investors.alnylam.com/releasedetail.cfm?ReleaseID=980988" type=" "]Read our press release[/spotlight-link] [spotlight-link icon="presentation" href="http://www.alnylam.com/web/assets/WFH-2016_Fitusiran_Ph-1_072516_2.pdf" type="(760 KB PDF)"]View the presentation[/spotlight-link]

We reported new data from our Phase 1 study with the newly named fitusiran (ALN-AT3), an investigational RNAi therapeutic targeting antithrombin (AT) for the treatment of hemophilia and rare bleeding disorders. Interim results – presented at the American Society of Hematology (ASH) 2015 Annual Meeting, held December 5 – 8, 2015 – showed that monthly, subcutaneous administration of fitusiran achieved potent and dose-dependent lowering of AT of up to 88% in patients with hemophilia. This AT lowering was associated with statistically significant increases in thrombin generation and an 85% reduction in estimated median annualized bleeding rates (ABR) in all evaluable cohorts. The observed bleeding rates are comparable to those reported for prophylactic intravenous infusions of replacement factors in patients with hemophilia. Fitusiran was found to be generally well tolerated to date, including no thromboembolic events or clinically significant increases in D-dimer, a biomarker of excessive clot formation. [spotlight-link icon="release" href="http://investors.alnylam.com/releasedetail.cfm?ReleaseID=945893" type=" "]Read our press release[/spotlight-link] [spotlight-link icon="presentation" href="http://www.alnylam.com/web/assets/ASH-2015_fitusiran_010715.pdf" type="(1.0 MB PDF)"]View the fitusiran Phase 1 presentation[/spotlight-link]

We presented positive interim clinical data from our ongoing Phase 1 study for ALN-AT3, a subcutaneously administered, investigational RNAi therapeutic targeting antithrombin (AT) for the treatment of hemophilia and rare bleeding disorders. Data were presented at the International Society on Thrombosis and Haemostasis (ISTH) 2015 Congress held June 20 – 25, 2015. New clinical results from 12 subjects with severe hemophilia show that subcutaneous administration of ALN-AT3 achieved potent and dose-dependent knockdown of AT of up to 86%. AT knockdown was highly durable, with effects lasting over two months after the last dose, supporting further evaluation of a once-monthly subcutaneous dose regimen. In addition, AT knockdown was associated with statistically significant increases in thrombin generation with a mean increase of up to 350% and marked improvements in whole blood clotting; these results demonstrate a re-balancing of hemostasis in severe hemophilia subjects. Furthermore, in an exploratory post-hoc analysis, a reduced frequency of bleeding was observed at higher AT knockdown levels with a maximum bleed-free interval of 114 days. Very importantly, ALN-AT3 continues to be generally well tolerated. [spotlight-link icon="release" href="http://investors.alnylam.com/releasedetail.cfm?ReleaseID=919144" type=" "]Read our press release[/spotlight-link] [spotlight-link icon="presentation" href="http://www.alnylam.com/web/assets/2015-ISTH-AT3-Phase1-Data.pdf" type="(614 KB PDF)"]View our Phase 1 data presentation[/spotlight-link]

We announced updated clinical results from the ongoing Phase 1 study of ALN-AT3, a subcutaneously administered, investigational RNAi therapeutic targeting antithrombin (AT) for the treatment of hemophilia and rare bleeding disorders (RBD).  New results were presented at the 2015 Goring Coagulation Conference in London.  Specifically, data were presented from the study’s second dose cohort in hemophilia subjects (n=3), where subcutaneous administration of ALN-AT3 resulted in an up to 70% knockdown of AT.  New results provide initial evidence for potential correction of the hemophilia phenotype associated with ALN-AT3 administration and AT knockdown.  Specifically, ALN-AT3 administration resulted in an increase in thrombin generation of up to 334% and a marked improvement in whole blood clotting.  In addition, the most advanced severe hemophilia A subject in the cohort has remained bleed free for 47 days without replacement factor prophylaxis as of the January 6, 2015 data cut-off date. In addition, ALN-AT3 administration remains generally well tolerated. [spotlight-link icon="release" href="http://investors.alnylam.com/releasedetail.cfm?ReleaseID=890625" type=" "] Read our press release[/spotlight-link] [spotlight-link icon="presentation" href="http://www.alnylam.com/web/assets/2015-Goring-meeting-Akinc_Jan2015.pdf" type="(1.6 MB PDF)"] View our presentation[/spotlight-link]

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 positive initial Phase 1 data for ALN-AT3, an investigational RNAi therapeutic targeting antithrombin (AT) for the treatment of hemophilia and rare bleeding disorders. Preliminary results, presented at the 56th Annual Meeting of the American Society of Hematology (ASH), showed that subcutaneous administration of ALN-AT3 given once weekly for three weeks at low doses of 15 (N=3) or 45 (N=1) micrograms/kg (mcg/kg) resulted in an up to 57% knockdown of AT in hemophilia subjects.  The effects of ALN-AT3 lasted for about 60 days after a single dose. ALN-AT3 was found to be well tolerated in both healthy volunteers and hemophilia subjects enrolled in the study.  These initial results show preliminary evidence for potency and durability of RNAi therapeutics at mcg/kg subcutaneous doses in human studies, and are the first clinical data to be reported for Alnylam’s Enhanced Stabilization Chemistry (ESC)-GalNAc conjugate technology. [spotlight-link icon="release" href="http://investors.alnylam.com/releasedetail.cfm?ReleaseID=886691" type=" "] Read our press release[/spotlight-link] [spotlight-link icon="presentation" href="http://www.alnylam.com/web/assets/ALN-AT3_Phase-1_ASH_120814.pdf" type="(1.1 MB PDF)"] View our ALN-AT3 Phase 1 presentation[/spotlight-link] [spotlight-link icon="presentation" href="http://www.alnylam.com/web/assets/ASH_RNA_Symposium_Dec2014.pdf" type="(2.0 MB PDF)"] View our pre-clinical presentation[/spotlight-link]

We presented positive top-line data from our Phase 1 clinical trial with ALN-AT3, an RNAi therapeutic targeting antithrombin (AT) in development for the treatment of hemophilia and rare bleeding disorders (RBD). In “Part A” of the Phase 1 study, we gave a single dose of drug to healthy volunteers to evaluate the drug’s safety and tolerability. Importantly, we had a dose escalation stopping rule at a maximum of 40% AT knockdown. Initial results, presented at the World Federation of Hemophilia (WFH) 2014 World Congress held May 11 – 15, 2014 in Melbourne, Australia, show that a single, low subcutaneous dose of ALN-AT3 at 0.03 mg/kg resulted in an up to 28-32% knockdown of AT at nadir relative to placebo (p <0.01 by ANOVA) and a temporally associated increase in peak thrombin generation (p <0.01). ALN-AT3 was found to be well tolerated with no significant adverse events reported.  With these results in hand, we have now proceeded to “Part B” of the Phase 1 study. [spotlight-link icon="release" href="http://investors.alnylam.com/releasedetail.cfm?ReleaseID=847452" type=" "] Read our press release[/spotlight-link] [spotlight-link icon="presentation" href="http://www.alnylam.com/web/assets/ALNY-HemophiliaProgram-WFH-May2014.pdf" type="(1.0 MB PDF)"] View our presentation [/spotlight-link]

We presented pre-clinical data from three programs within our “Alnylam 5x15” RNAi therapeutic pipeline: ALN-AT3 for the treatment of hemophilia and rare bleeding disorders (RBD), ALN-CC5 for the treatment of complement-mediated diseases, and ALN-TMP for the treatment of β-thalassemia and iron overload disorders. These data were presented at the 55th Annual Meeting of the American Society of Hematology (ASH) held December 7 – 10 in New Orleans. [spotlight-link icon="release" href="http://investors.alnylam.com/releasedetail.cfm?ReleaseID=812683" type=" "]Read our ALN-AT3 press release[/spotlight-link] [spotlight-link icon="release" href="http://investors.alnylam.com/releasedetail.cfm?ReleaseID=812394" type=" "]Read our ALN-CC5/ALN-TMP press release[/spotlight-link]

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