United States First Tender

Ionis Pharmaceuticals Inc. the leader in antisense therapeutics today announced that data from the IONIS-TMPRSS6-LRx Phase 1 study TEGSEDI inotersen and other Ionis research and development programs will be presented at the 60th American Society of Hematology ASH Annual Meeting and Exposition in San Diego California December 1-4 2018.Ionis Pharmaceuticals PRNewsfotoIonis Pharmaceuticals Inc.The results to be reported at ASH demonstrate the potential for antisense therapeutics to address a broad range of diseases by potentially bringing transformational medicines to patients with severe diseases who have limited treatment options. IONIS-TMPRSS6-LRx an antisense therapy in development for people suffering with beta-thalassemia and other potential indications is one of several of Ionis advanced LIgand Conjugated Antisense or LICA drugs to demonstrate potent target reduction and a favorable safety and tolerability profile in humans.Additional data and topics covered in the platform presentations and posters will includeThe therapeutic potential of IONIS-TMPRSS6-LRx as demonstrated by modulation of important biomarkers including observed reductions in plasma iron levels in a Phase 1 study.The therapeutic potential of IONIS-TMPRSS6-LRx in combination with erythropoietic stimulating agents and iron restriction to correct anemia while improving splenomegaly and iron overload in an animal model of non-transfusion dependent beta-thalassemia.The use of antisense oligonucleotides to specifically modulate platelet levels as a potentially novel therapeutic approach for cancer and potentially other disorders. The long-term analyses of the NEURO-TTR open-label extension study and TEGSEDIs impact on the lives of patients living with polyneuropathy caused by hereditary transthyretin amyloidosis hATTR.