AstraZeneca agrees $3.585bn deal with Ionis Pharmaceuticals for Eplontersen

AstraZeneca has agreed to pay up to $3.585bn plus royalties to Ionis Pharmaceuticals under a global development and commercialisation agreement for an investigational medicine in trials to treat a progressive, fatal condition.

The Cambridge-headquartered biopharmaceutical firm will pay $200m upfront plus conditional payments of up to $485m following regulatory approvals for eplontersen, formerly known as IONIS-TTR-LRX.

It will also pay up to $2.9bn of sales-related milestones, based on sales thresholds between $500m and $6bn, plus royalties the low double-digit to mid-twenties percentage range depending on the region.

Eplontersen is in Phase III clinical trials for transthyretin amyloidosis, a systemic, progressive and fatal condition.

Mene Pangalos, executive vice president, 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 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.”

Eplontersen is designed to reduce the production of transthyretin (TTR protein) to treat both hereditary and non-hereditary forms of TTR amyloidosis (ATTR).

It is in trials for the treatment of both amyloid transthyretin cardiomyopathy (ATTR-CM) and amyloid transthyretin polyneuropathy (ATTR-PN).

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

It is estimated that 300,000 to 500,000 patients worldwide have ATTR-CM, but its prevalence is believed to be under-estimated due to a lack of awareness and the heterogeneity of symptoms.

Hereditary ATTR-PN is a debilitating disease leading to peripheral nerve damage with motor disability within five years of diagnosis and, without treatment, is typically fatal within a decade. It is thought to affect 10,000 to 40,000 patients worldwide.

The companies are expected to seek regulatory approval for Eplontersen to treat hereditary ATTR-PN first, with a potential new drug application with the US Food and Drug Administration by the end of 2022.

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.

Ionis, headquartered in California, will continue to manufacture and supply eplontersen for the existing clinical studies and process qualification. AstraZeneca will be responsible for commercial supply.

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