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AstraZeneca and Ionis sign $3.6bn deal for eplontersen

The liver-targeted antisense therapy is in phase 3 trials for the treatment of transthyretin amyloidosis, a systemic, progressive and fatal condition


AstraZeneca (AZ) has teamed up once more with California-based Ionis Pharmaceuticals, signing a global development and commercialisation agreement for its antisense therapy eplontersen.

This is the third deal between the companies. In 2018, AZ signed strategic collaborations covering ION532, in apolipoprotein L1-associated chronic kidney disease and ION839, for non-alcoholic steatohepatitis (NASH). However, this is the first time AZ has invested in a late-stage treatment.

Eplontersen is designed to reduce the production of transthyretin (TTR) protein to treat amyloid transthyretin (ATTR), a systemic, progressive and fatal disease. It is currently in global phase 3 trials for ATTR cardiomyopathy (ATTR-CM) and ATTR polyneuropathy (ATTR-PN).

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

Up to half a million people are believed to have ATTR-CM and up to 40,000 people have ATTR-PN worldwide.

“Eplontersen has the potential to halt the progression of TTR-mediated amyloidosis irrespective of whether it’s caused by genetic mutations or ageing,” said Mene Pangalos, head of biopharmaceuticals R&D at AZ. “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.”

It is expected that the first indication for the therapy will be hereditary ATTR-PN, which could be filed with the Food and Drug Administration (FDA) by the end of 2022.

The AZ deal bolsters Ionis' commercial organisation as it prepares for multiple product launches. It currently has three marketed products – Spinraza (nusinersen) for spinal muscular atrophy, Tegsedi (inotersen) for ATTR and Waylivra (volanesorsen) for familial chylomicronaemia syndrome – as well as ‘a premier late-stage pipeline’.

"We believe that bringing together Ionis' industry-leading experience in RNA-targeted therapeutics and deep knowledge of the TTR amyloidosis market with AstraZeneca's global scale and leadership in cardiovascular drug development and commercialization will enable faster and deeper market penetration for the benefit of patients," said Ionis CEO, Brett  Monia.

However, if approved, eplontersen will not have the ATTR market to itself and must compete with Pfizer’s Vyndaqel/Vyndamax (tafamidis) and Alnylam’s RNAi therapy Onpattro (patisiran).

The deal will see AZ will pay Ionis $200m upfront then conditional payments up to $485m following regulatory approvals as well as up to $2.9bn in sales-related milestones plus royalties in the range of low double-digit to mid-twenties percentage depending on the region.

Article by
Hugh Gosling

8th December 2021

From: Sales



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