AstraZeneca Signs Blockbuster Deal With Ionis for TTR Amyloidosis Treatment

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AstraZeneca struck a deal worth up to more than $3.5 billion with Ionis Pharmaceuticals for the rights to develop and commercialize their transthyretin (TTR) amyloidosis treatment.

AstraZeneca announced on Dec. 7, 2021, that it would pay $200 million to Ionis Pharmaceuticals for the rights to its investigational medicine, eplontersen. Per the terms of the deal, if certain regulatory and sales thresholds are met, AstraZeneca could pay an additional $3.385 billion to Ionis.

Eplontersen is a ligand-conjugated antisense investigational medicine currently in Phase III clinical trials for amyloid transthyretin cardiomyopathy and amyloid transthyretin polyneuropathy. It is designed to reduce the production of transthyretin (TTR) to treat both hereditary and non-hereditary forms of TTR amyloidosis (ATTR).

ATTR is a very rare form of amyloidosis caused by misfolding TTR proteins, resulting in interference to normal function in peripheral nerves, heart, gastrointestinal system, eyes, kidneys, central nervous system, thyroid, or bone marrow tissues. It is a progressive, systematic, and ultimately fatal disease; according to a company press release, it typically results in progressive heart failure within four years of diagnosis.


“Eplontersen has the potential to halt the progression of TTR-mediated amyloidosis, irrespective of whether it’s caused by genetic mutations or aging,” said Mene Pangalos, executive vice-president, BioPharmaceuticals R&D, AstraZeneca, in the press release. “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.”

Source: AstraZeneca