Fortress Biotech Inc. (NASDAQ:FBIO) has announced that Caelum Biosciences Inc., in partnership with Alexion Pharmaceuticals Inc., has commenced the Cardiac Amyloid Reaching for Extended Survival (CARES) Phase 3 clinical program evaluating CAEL-101 in combination with AL amyloidosis standard-of-care therapy. CAEL-101 is a first-in-class amyloid fibril targeted therapy.
Alexion is a collaboration agreement with Caelum
Last year Fortress-founded Caelum signed a collaboration agreement with Alexion through which Alexion acquired a minority stake in Caelum. Also, Alexion receives an exclusive option to acquire the rest of its equity-based on Phase 3 CAEL-101 data. Fortress still has a 40% stake in Caelum’s outstanding and issued stock, and it is eligible to get around 43% of the proceeds following the acquisition option of Alexion.
Chief Executive Officer and President of Fortress Lindsay Rosenwald said they were delighted to progress CAEL-101 into third phase development. She explained that CAEL-101 has the potential of filling the urgent need for a treatment alternative for patients with AL amyloidosis. So far, data suggest that CAEL-101 could enhance overall survival and organ function. Lindsay said that the Caelum and Alexion partnership represents a huge opportunity to help patients and create value for shareholders. She said that they are looking forward to continuing the late-stage development of the therapy.
Phase 3 study to enroll 370 patients
The CARES program comprises two Phase 3 parallel studies, which will collectively enroll around 370 patients worldwide. The first study will be in Mayo stage IIIa disease patients while the other will be on patients having Mayo stage IIIb disease. Currently, the company is conducting enrollment in both studies, with the program’s primary endpoint being an evaluation of overall survival.
AL amyloidosis, called primary amyloidosis, is a rare disease that usually occurs following the build-up of an abnormal protein called amyloid in organs, affecting normal functioning. Several organs might be affected, including kidneys, and heart causing a lot of damage and impairing organ function. Following diagnosis, median survival in AL amyloidosis patients with affected heart is usually below a year.