Benzinga | Sep 14, 2020 08:07AM EDT

Alexion and Caelum Biosciences Announce Start Of Phase 3 Studies Of CAEL-101 In AL Amyloidosis

- Phase 2 study met primary objective, supporting initiation of two parallel Phase 3 studies that will enroll ~370 AL amyloidosis patients -

- Positive long-term Phase 1a/1b data presented at the International Symposium on Amyloidosis (ISA) 2020 demonstrate prolonged overall survival (78% at 37 months) and durable organ response -

Alexion Pharmaceuticals, Inc. (NASDAQ:ALXN) and Caelum Biosciences today announced the initiation of the Cardiac Amyloid Reaching for Extended Survival (CARES) Phase 3 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 3 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.

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"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 3 clinical program."

"AL amyloidosis is particularly devastating when it affects the heart, with median survival in these patients of less than one year following diagnosis," said Michael Spector, President and Chief Executive Officer of Caelum. "Long-term survival data from AL amyloidosis patients treated with CAEL-101 in the Phase 1a/1b study showed that 78 percent were still alive after a median follow-up time of more than three years. We recognize the urgent need for new treatments that address the organ damage caused by AL amyloidosis and are working together with the AL amyloidosis community and Alexion to advance the Phase 3 clinical program as quickly as possible."