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AstraZeneca’s acquisition of Caelum Biosciences gives them access to CAEL-101, a promising light chain amyloidosis treatment.
AstraZeneca’s Alexion, a group within AstraZeneca focused on rare diseases, announced on Sept. 29, 2021, that it had exercised its option to acquire all remaining equity in Caelum Biosciences. This acquisition gives them rights to CAEL-101, a drug candidate fibril-reactive monoclonal antibody (mAb) for the treatment of light chain (AL) amyloidosis.
AL amyloidosis is a rare disease caused by defective plasma cells in one’s bone marrow, causing misfolded amyloid proteins to build up in organs such as the heart and kidneys. This causes significant organ damage and failure, which can be fatal. According to a company press release, approximately 20,000 people in the United States, France, Germany, Italy, Spain, and the United Kingdom live with AL amyloidosis classified as Mayo stage IIIa or IIIb disease.
CAEL-101 is a mAb designed to improve organ function by reducing or eliminating amyloid deposits in the tissues and organs of patients with AL amyloidosis. It is designed to bind to misfolded light chain proteins and amyloid and has shown binding to both kappa and lambda subtypes. It received orphan drug designation from both FDA and the European Commission.
CAEL-101 is currently being evaluated in a Phase III clinical program in combination with standard-of-care therapy in patients with AL amyloidosis. Two parallel studies for patients with Mayo stage IIIa disease (n=270) and Mayo stage IIIb disease (n=110) 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 Dunover, CEO, Alexion, in the press release. “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.