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8 December, 2021 - 22:25 By Tony Quested

AstraZeneca partners with Ionis to fight rare liver conditions

AstraZeneca has teamed up with San Diego company Ionis to develop and commercialise medicine to fight a rare liver disease.

Eplontersen is a liver-targeted antisense therapy in Phase III development for the treatment of transthyretin amyloidosis – a systemic, progressive and fatal condition – and variants.

The companies will jointly develop and commercialise eplontersen in the US, while AstraZeneca will do the same in the rest of the world, except in Latin America.

ATTR-CM is a systemic, progressive and fatal condition that leads to progressive heart failure and death within four years from diagnosis.

It remains underdiagnosed and its prevalence is thought to be underestimated due to a lack of disease awareness and the heterogeneity of symptoms.

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.

Mene Pangalos, executive VP, BioPharmaceuticals R & D at AstraZeneca, said: “Eplontersen has the potential to halt the progression of TTR-mediated amyloidosis, irrespective of whether it’s caused by genetic mutations or ageing.

“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.”

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 Administration by the end of 2022.

Cambridge headquartered AstraZeneca will pay Ionis an upfront payment of $200 million and additional conditional payments of up to $485m following regulatory approvals. It will also pay up to $2.9 billion of sales-related milestones based on sales thresholds between $500m and $6bn, plus royalties in the range of low double-digit to mid-twenties percentage depending on the region. The transaction will be funded with cash.

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