Alexion and Caelum Biosciences announce start of phase III studies of CAEL 101 in AL amyloidosis.

Alexion Pharmaceuticals, Inc. and Caelum Biosciences announced the initiation of the Cardiac Amyloid Reaching for Extended Survival (CARES) Phase III clinical program to evaluate CAEL 101, a first-in-class amyloid fibril targeted therapy, in combination with standard-of-care (SoC) therapy in AL amyloidosis. The CARES clinical program includes two parallel Phase III studies – one in patients with Mayo stage IIIa disease and one in patients with Mayo stage IIIb disease – and will collectively enroll approximately 370 patients globally. Enrollment is underway in both studies. The primary objective of the clinical program is to assess overall survival. “In AL amyloidosis, misfolded amyloid proteins can build up in many organs throughout the body, including the heart and kidneys, causing significant damage to these organs and impairing their function. While current treatments address the bone marrow disorder that creates the misfolded amyloid proteins, there are no approved therapies for the significant organ damage the disease causes,” said John Orloff, M.D., Executive Vice President and Head of Research and Development at Alexion. “CAEL 101 has the potential to be the first treatment to target and remove the amyloid deposits from these organs. Data from Phase 1 studies suggest that this treatment approach may improve organ function and long-term survival. We look forward to investigating this further in the Phase III clinical program.”.