AstraZeneca buys rare disease drugmaker Caelum in $500m deal
12:00, 29 September 2021

AstraZeneca subsidiary Alexion Pharmaceuticals is set to take full control of Caelum Biosciences in a deal worth up to $500m (£371.3m), as the company extends its focus on rare disease drugs.
It means New Jersey-based Caelum will become part of the Alexion division, which AstraZeneca bought for $39bn earlier this year.
Shares in AstraZeneca rose 3.40% on the London Stock Exchange in early trade on Wednesday after it made the announcement on its website.
Rare disease drug development
The latest acquisition gives the pharma giant access to another potentially lucrative rare disease drug, CAEL-101, that is undergoing late-stage trials and has a fast-track status for regulatory review in the US.
It aims to treat AL amyloidosis – a rare, life-threatening disease that damages the heart and kidneys, affecting an estimated 20,000 people across six western countries.
The disease causes abnormal proteins called amyloids to build up in human organs and disrupt their normal function. Caelum’s drug is a type of monoclonal antibody that binds to amyloid deposits to reduce or remove them and improve organ function.
“With a median survival time of less than 18 months following diagnosis, there is an urgent need for new treatments for this devastating disease,” Alexion chief executive Marc Dunoyer said in a statement.
Financial considerations
In 2019 Caelum and Alexion first entered into a collaboration whereby Alexion acquired a minority stake as well as an exclusive option to acquire the remaining equity in Caelum.
Alexion will pay $150m to buy the remaining stake it does not already own in Caelum and make future payments of up to $350m, depending on milestones achieved, AstraZeneca said.
Since buying Alexion, AstraZeneca has had mixed fortunes with rare disease drugs under development. It reported promising results from trials of a Wilson’s disease drug. However, it abandoned trials for an amyotrophic lateral sclerosis (ALS) drug after it failed to show efficacy.