AstraZeneca pushes deeper into rare diseases with $3.6bn deal for liver drug

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Rugmaker AstraZeneca today delved deeper into the lucrative but high-risk rare diseases market, signing a deal worth up to $3.6 billion for the rights to a promising treatment for an incurable liver condition.

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The FTSE 100’s most valuable company — which surprised investors last year with its $39 billion purchase of rare disease specialist Alexion — has signed a new deal with California-based biotech Ionis.

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Astra will pay Ionis $200 million in advance and pay more than $485 million when the drug, called eplontersen, receives regulatory approval.

It is also committed to milestone payments of up to $2.9 billion based on future sales.

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The companies will work together to develop and sell a drug that targets ATTR, a chronic genetic condition that causes nerve damage or heart failure.

Patients with the most severe form of the disease live on average only four years after diagnosis.

Mene Pangalos, head of Astra’s drug discovery team, said: “Thanks to its precise liver-targeting properties, it has the potential to be a best-in-class treatment for patients suffering from this devastating disease and who currently have limited options. “

Ionis was founded in 1989 by Stanley Crookes, a former research chief at GSK, to develop anti-sense therapy, where drugs are used to silence faulty genes by destroying mRNA that binds to the cell’s DNA and proteins. Acts as a messenger between construction machinery.

Astra’s share price has pushed this year to make the Pascal Soriot-led Cambridge-based company the most valuable on the FTSE 100 with a market cap of around £130 billion.

While the COVID-19 vaccine, developed and manufactured with the University of Oxford, has put it at the frontline of the fight against the pandemic, it has not been without disappointment – from delayed delivery to rare side effects and varying reports on its efficacy.

Investors in the company see greater value in a potential pipeline of complex drugs for rare diseases that prove more difficult for rivals to develop.

Astra shares fell 1.8% to 8221.0p as investors digested its latest major investment. Shares of Ionis rose 4% to $27.50 on the Nasdaq in pre-market trading.

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