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Pharma & Biotech

AstraZeneca to buy rare disease group Caelum Biosciences for up to US$500m

Caleum is investigating a drug to treat light chain amyloidosis, a fatal, rare disease in which abnormal proteins build up in organs throughout the body

AstraZeneca PLC (LSE:AZN) said its Alexion arm exercised an option to buy all remaining equity in Caelum Biosciences to gain control of CAEL-101, a potential treatment for light chain (AL) amyloidosis.

AL amyloidosis is a rare disease in which abnormal proteins build up in organs throughout the body, including the heart and kidneys, causing significant organ damage and failure that can cause death.

READ: AstraZeneca: Where now for the shares and the vaccines business? And why no good deed goes unpunished

Around 20,000 people across the US, France, Germany, Italy, Spain and the UK live with AL amyloidosis classified as Mayo stage IIIa or IIIb disease.

CAEL-101 is at the last stage of clinical trials, evaluated in combination with standard-of-care, with two parallel studies ongoing.

Alexion first bought a minority stake in 2019 and is now exercising the option to acquire the rest for US$150mln, plus extra payments of up to US$350mln based on milestones.

"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’s chief executive Marc Dunoyer said.

“CAEL-101 has the potential to be the first therapy to target and remove amyloid deposits from organ tissues, improve organ function, and, ultimately, lead to longer lives for these patients."

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