Fortress Biotech Announces Initiation of Phase 3 Studies of CAEL-101 in AL Amyloidosis

Fortress Biotech announced Caelum Biosciences, in collaboration with Alexion Pharmaceuticals, has initiated 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.

In 2019, Caelum, a company founded by Fortress, entered into a collaboration agreement with Alexion under which Alexion acquired a minority equity interest in Caelum and an exclusive option to acquire the remaining equity in the company based on Phase 3 CAEL-101 data. Fortress maintains a 40 percent ownership in Caelum’s issued and outstanding stock and is eligible to receive approximately 43 percent of the proceeds from an Alexion acquisition option exercise.

“We are pleased that CAEL-101 has progressed into Phase 3 development,” said Lindsay A. Rosenwald, M.D., Chairman, President and Chief Executive Officer of Fortress. “CAEL-101 has the potential to fill the urgent need for new treatment options for patients with AL amyloidosis, as the data thus far suggest it may improve organ function and overall survival. The partnership between Caelum and Alexion represents an exciting opportunity to help patients and create value for our shareholders. We look forward to the continued late-stage development of this important therapy.”

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.

AL amyloidosis, also called primary amyloidosis, is a rare disease that occurs when an abnormal protein called amyloid builds up in organs and interferes with their normal function. It affects many organs, including the heart and kidneys, causes significant damage and impairs organ function. Median survival in patients with AL amyloidosis that has affected the heart is less than a year following diagnosis.