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AstraZeneca, Ionis to collaborate on eplontersen
AstraZeneca, Ionis to collaborate on eplontersen.
About this update from Astrazeneca Plc
[{"type":"text","content":"\n \n \n \n RNS Number : 8023U\n AstraZeneca PLC\n 07 December 2021\n \n \n \n 7 December 2021 07:00 GMT\n \n \n AstraZeneca and Ionis sign deal to develop and commercialise eplontersen\n \n \n \n Liver-targeted antisense therapy in Phase III development for the treatment of transthyretin amyloidosis,\n \n a systemic, progressive and fatal condition\n \n \n \n \n \n AstraZeneca has entered into a \n new global development and commercialisation agreement with Ionis Pharmaceuticals, Inc. (Ionis) for eplontersen, formerly known as IONIS-TTR-LRX. Eplontersen is a ligand-conjugated antisense investigational medicine currently in Phase III clinical trials for amyloid t\n ransthyretin \n cardiomyopathy (ATTR-CM) and \n amyloid t\n ransthyretin \n polyneuropathy (ATTR-PN). It is designed to reduce the production of transthyretin (TTR protein) to treat both hereditary and non-hereditary forms of TTR amyloidosis (ATTR).\n \n \n \n \n \n The companies will jointly develop and commercialise eplontersen in the US, while AstraZeneca will develop and commercialise it in the rest of the world, except in Latin America.\n \n \n \n \n \n ATTR-CM is a systemic, progressive and fatal condition that leads to progressive heart failure and death within four years from diagnosis.1 It remains underdiagnosed and its prevalence is thought to be underestimated due to a lack of disease awareness and the heterogeneity of symptoms.2 Hereditary ATTR-PN is a debilitating disease that leads to peripheral nerve damage with motor disability within five years of diagnosis and, without treatment, is generally fatal within a decade.3\n \n \n \n \n \n Mene Pangalos, Executive Vice President, BioPharmaceuticals R&D, \n AstraZeneca, said: \"Eplontersen has the potential to halt the progression of TTR-mediated amyloidosis, irrespective of whether it's caused by genetic mutations or aging. Thanks to its precise liver-targeting properties, it also has the potential to be a best-in-class treatment for patients suffering from this devastating disease and who currently have limited options.\"\n \n \n \n \n \n Hereditary ATTR-PN is expected to be the first indication for which the companies will seek regulatory approval for eplontersen, with the potential to file a new drug application with the US Food and Drug Administr...