Results from a phase 3 trial for the medicine for Wilson’s disease proved three times more successful at removing copper deposits compared with standard treatments. The trial enrolled 214 patients, including treatment-naive participants and those who had been on alternative therapy for an average of 10 or more years. Wilson’s disease is a progressive genetic condition where the body’s ability to remove excess copper is compromised.
Damage from toxic copper build-up can lead to liver disease and psychiatric and neurological problems, such as personality changes, tremors and difficulty in walking, swallowing or talking.
The study met the main goal of improvement in the daily mean copper mobilised from tissues over a period of 48 weeks.
The experimental oral medicine which should be taken once a day could provide a less invasive option for patients who are usually given treatments that involve injecting a metal removing solution into the blood or zinc therapy.
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Marc Dunoyer, 68, former AstraZeneca chief financial officer who now heads Alexion, said: “Where existing treatments remove copper from the blood, these 48-week results demonstrate its significant impact in mobilising copper from tissues.
“As we advance this first innovation in Wilson disease treatment in more than 30 years, we will continue to follow these patients long term to further assess clinical impact on disease symptoms,”
Adam Barker, an analyst at Shore Capital said that Alexion had indicated previously that the medicine for Wilson’s disease had a peak sales opportunity of between $500 million and $1 billion.
The takeover of Alexion was completed last month and AstraZeneca plans to boost sales of Alexion’s rare diseases medicines globally.
This is especially the case in China, which is becoming an increasingly important market for AstraZeneca.
They are also targeting a longer-term opportunity to develop immune-based medicines for more common conditions.
Based in Cambridge, the firm has other therapy specialisms, including oncology, respiratory and cardiovascular diseases.
It has been thrust into the spotlight for its heroic work on producing a vaccine for COVID-19, which has helped save thousands of lives already.
Shares yesterday in AstraZeneca rose by 27p, or 0.3 percent, to £85.80, an increase of 17 percent so far this year.