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AstraZeneca Acquires Caelum Biosciences For Rare Disease Antibody
Researchers are currently studying the antibody combined with standard of care therapy for rare disease AL amyloidosis in a Phase 3 clinical program.
AstraZeneca’s Alexion will fully acquire the remaining equity in Caelum Biosciences for the company’s first-in-class fibril-reactive monoclonal antibody (mAb) to treat rare disease light chain (AL) amyloidosis.
Researchers are studying the antibody, CAEL-101, in the Cardiac Amyloid Reaching for Extended Survival (CARES) Phase 3 clinical program combined with standard-of-care (SoC) therapy for AL amyloidosis.
Additionally, two parallel Phase 3 trials in patients with Mayo stage Illa disease and Mayo stage Illb 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. 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,” Marc Dunoyer, chief executive officer of Alexion, said in the announcement.
The FDA previously granted CAEL-101 Orphan Drug Designation as a therapy for patients with AL amyloidosis and a radio-imaging agent in AL amyloidosis.
AL amyloidosis is a rare disease that causes amyloid proteins to build up in organs throughout the body, including the heart and kidneys. This buildup causes significant organ damage and failure that may be fatal.
Nearly 20,000 people across the US, UK, France, Germany, Italy, and Spain live with AL amyloidosis. Although there is no cure for patients with AL amyloidosis, patients can go into remission with drug therapy.
In January 2019, Alexion and Caelum Biosciences announced a collaboration to develop CAEL-101.
Under the terms of the agreement, Alexion acquired a minority equity interest in Caelum and an exclusive option to acquire the remaining equity in the company based on Phase 2 data.
And the companies will collaborate on the design of the ongoing development program for CAEL-101. Caelum will be responsible for conducting the development program through the end of Phase 2 and for manufacturing CAEL-101.
In a Phase 1a/1b study, CAEL-101 demonstrated improved organ function, including cardiac and renal function, in 27 patients with relapsed and refractory AL amyloidosis who had not previously had an organ response to standard of care therapy.
