Fortress Biotech Announces Initiation of Phase 3 Studies of CAEL-101 in AL Amyloidosis
- Caelum Biosciences, a company founded by Fortress, is collaborating with Alexion Pharmaceuticals on the studies
- 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 percent at 37 months) and durable organ response
NEW YORK, Sept. 14, 2020 (GLOBE NEWSWIRE) -- Fortress Biotech, Inc. (Nasdaq: FBIO) (“Fortress”), an innovative revenue-generating company focused on acquiring, developing and commercializing or monetizing promising biopharmaceutical products and product candidates cost-effectively, today announced that Caelum Biosciences, Inc. (“Caelum”), in collaboration with Alexion Pharmaceuticals, Inc. (“Alexion”), 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.
About the CARES Phase 3 Clinical Program
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.
The primary study objectives are overall survival and the safety and tolerability of CAEL-101. Key secondary objectives will assess functional improvement in the six-minute walk test (6MWT), quality of life measures (Kansas City Cardiomyopathy Questionnaire Overall Score & Short Form 36 version 2 Physical Component Score) and cardiac improvement (Global Longitudinal Strain (GLS)).
Phase 2 Study Results
Phase 1a/1b Long-Term Follow-Up Results Presented at International Symposium on Amyloidosis 2020
About AL Amyloidosis
AL amyloidosis is a rare disease but is the most common form of amyloidosis. There are approximately 22,000 patients across the United States, France, Germany, Italy, Spain and the United Kingdom. AL amyloidosis has a one-year mortality rate of 47 percent, 76 percent of which is caused by cardiac amyloidosis.
About Caelum Biosciences
About Fortress Biotech
Michael Spector, President & Chief Executive Officer
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