RARE Daily

AstraZeneca Acquires Caelum Biosciences to Advance AL Amyloidosis Program

September 29, 2021

AstraZeneca’s Alexion Rare Diseases exercised its option to acquire all remaining equity in Caelum Biosciences for CAEL-101, a potentially first-in-class treatment of light chain AL amyloidosis.

Photo: Marc Dunoyer, CEO of Alexion

Photo: Marc Dunoyer, CEO of Alexion

“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 of 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.”

In 2019, Caelum and Alexion first entered into a collaboration whereby Alexion acquired a minority equity interest and an exclusive option to acquire the remaining equity in Caelum. Alexion’s consolidation of Caelum reflects a non-controlling interest of $150 million. Upon closing the acquisition, which is expected to take place on October 5, 2021, Alexion will pay Caelum the agreed option exercise price of approximately $150 million, with the potential for additional payments of up to $350 million upon achievement of regulatory and commercial milestones. 

AL amyloidosis is a rare disease caused by defective plasma cells in the bone marrow that produce abnormal antibody (immunoglobulin) proteins. These abnormal proteins misfold and aggregate to form amyloids that may deposit in tissues and/or organs. Amyloid accumulation in organs, particularly in the heart and kidneys, can cause widespread and progressive organ damage and high mortality rates, with death most frequently occurring as a result of cardiac failure. Approximately 20,000 people across the United States, France, Germany, Italy, Spain and the United Kingdom live with AL amyloidosis, classified as Mayo stage 3a or 3b disease.

CAEL-101 is a potentially first-in-class monoclonal antibody designed to improve organ function by reducing or eliminating amyloid deposits in the tissues and organs of patients with AL amyloidosis. The antibody is designed to bind to misfolded light chain proteins and amyloid and shows binding to both kappa and lambda subtypes. The U.S. Food and Drug Administration and the European Commission both granted CAEL-101 Orphan Drug designation as a potential therapy for patients with AL amyloidosis. Additionally, the FDA granted Fast Track designation to CAEL-101 for AL amyloidosis in June 2021.

CAEL-101 is currently being evaluated in the Cardiac Amyloid Reaching for Extended Survival (CARES) phase 3 clinical program in combination with standard-of-care (SoC) therapy in AL amyloidosis patients who are newly diagnosed and naïve to SoC treatment. Two parallel phase 3 trials in patients with Mayo stage 3a disease and in patients with Mayo stage 3b disease respectively are ongoing.

One trial is enrolling approximately 270 patients with Mayo stage 3a disease and one trial is enrolling approximately 110 patients with Mayo stage 3b disease. The primary endpoint for both clinical trials is overall survival and enrolment is underway.

In each study, participants are being randomized in a 2:1 ratio to receive either CAEL-101 plus SoC or placebo plus SoC once weekly for four weeks. This will be followed by a maintenance dose administered every two weeks until the last patient enrolled completes at least 50 weeks of treatment. Patients will continue follow-up visits every 12 weeks and will subsequently be enrolled in an open-label extension study.

Author: Rare Daily Staff

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