AstraZeneca to fully acquire Caelum Biosciences, advancing mAb for rare disease

By Rachel Arthur

- Last updated on GMT

pic: getty/buchachon
pic: getty/buchachon

Related tags Astrazeneca Monoclonal antibodies

AstraZeneca’s Alexion has exercised its option to acquire all remaining equity in Caelum Biosciences for CAEL-101, a fibril-reactive monoclonal antibody (mAb) for the treatment of light chain (AL) amyloidosis.

The deal will allow AstraZeneca to advance and accelerate the ongoing Phase III clinical development of mAb.

Alexion (acquired by AstraZeneca this year for $39bn) first gained a minority equity interest and an exclusive option to acquire the remaining equity in Caelum back in 2019. Alexion will now pay Caelum the agreed option exercise price of approximately $150m, with the potential for additional payments of up to $350m upon achievement of regulatory and commercial milestones.  

Phase 3 trials 

AL amyloidosis is a rare disease in which misfolded amyloid proteins build up in organs throughout the body, including the heart and kidneys, causing significant organ damage and failure that may ultimately be fatal.

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 designed to improve organ function by reducing or eliminating amyloid deposits in the tissues and organs of patients. The antibody is designed to bind to misfolded light chain proteins and amyloid and shows binding to both kappa and lambda subtypes.

The mAb is currently being evaluated in a Phase III clinical programme in combination with standard-of-care (SoC) therapy in AL amyloidosis. Two parallel Phase III trials in patients with Mayo stage IIIa disease and in patients with Mayo stage IIIb disease respectively are ongoing.

“With a median survival time of less than 18 months following diagnosis, there is an urgent need for new treatments for this devastating disease,” ​said Marc Dunoyer, CEO, Alexion.

“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.”

CAEL-101 has received Orphan Drug Designation from both the US Food and Drug Administration (FDA) and the European Commission as a potential therapy for patients with AL amyloidosis. The US FDA also granted Fast Track Designation to CAEL-101 for AL amyloidosis in June 2021.

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