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Fortress Biotech Forms New Subsidiary, Caelum Biosciences, Inc., To Develop Novel Treatment for Al Amyloidosis
Fortress Biotech, Inc. announced the formation of a new subsidiary company, Caelum Biosciences, Inc., to advance the development of CAEL‐ 101 (11‐1F4) for the treatment of amyloid light chain (“AL”) amyloidosis. Caelum has entered into an agreement with Columbia University to secure exclusive worldwide license rights to CAEL‐101, a chimeric fibril‐reactive monoclonal antibody (mAb) currently being evaluated in a Phase 1a/1b study. In addition, Fortress announced the appointment of Michael Spector as Caelum’s Chief Executive Officer.
AL amyloidosis is a rare systemic disorder caused by an abnormality of plasma cells in the bone marrow. Misfolded amyloid proteins produced by plasma cells cause buildup in and around tissues, nerves and organs, gradually affecting their function. This can cause progressive and widespread organ damage, and high mortality rates.
Alan Solomon, M.D., Professor of Medicine Emeritus and a former American Cancer Society Clinical Research Professor at the University of Tennessee Graduate School of Medicine, who pioneered early development of antibodies that work to break up pre‐existing amyloid in tissues and oversaw preclinical research on CAEL‐101, said: “Existing AL amyloidosis treatment using chemotherapy eliminates the plasma cells that produce the abnormal proteins, but most often the pathologic amyloid deposits in the body’s vital tissues remain or progress, resulting in organ failure and even death. For this reason, our research efforts were focused on helping the body’s immune system remove this deleterious material. Our “anti‐amyloid” monoclonal antibody 11‐1F4, now designated CAEL‐101, has demonstrated its ability to bind to amyloid and promote dissolution in mice bearing human AL amyloid tumors. Most importantly, CAEL‐101 injection has been shown to specifically bind to the amyloid and not to normal tissue in patients with AL amyloidosis. We hope that this anti‐amyloid immunotherapy, in combination with anti‐plasma cell chemotherapy, will benefit patients with this fatal disease.”
Dr. Lindsay A. Rosenwald, Fortress Biotech’s Chairman, President and Chief Executive Officer, said, “We are thrilled to enter this collaboration with Columbia to advance the development of CAEL‐101. The launch of Caelum, Fortress Biotech’s eighth subsidiary, underscores our mission of developing a broad pipeline of novel products across areas of unmet need.”
CAEL‐101 Phase 1a/1b Study Update
Data from an ongoing Phase 1a/1b study (ClinicalTrials.gov Identifier: NCT02245867) have demonstrated that CAEL‐101 is well tolerated and safe with no drug‐related grade four or five adverse events or dose‐limiting toxicity up to 500mg/m2. A single infusion of CAEL‐101 or one weekly infusion for four weeks have demonstrated early and sustained organ response in cardiac, renal, gastrointestinal, skin and soft tissue. Interim clinical efficacy data show CAEL‐101 promotes amyloid resolution in 67 percent of patients (63 percent in Phase 1a; 70 percent in Phase 1b). These data were presented by Columbia University on December 5, 2016 at the American Society of Hematology’s (ASH) 58th Annual Meeting.
Suzanne Lentzsch, M.D., Ph.D., Professor of Medicine at Columbia University Medical Center, College of Physicians and Surgeons of Columbia University and at New York Presbyterian Hospital, and the principle investigator on the Phase 1a/1b study, said, “These interim results demonstrate strong biomarker activity that may speak to CAEL‐101’s ability to safely promote amyloid elimination and the subsequent improvement of organ function. We look forward to working with Caelum on this promising program and expect to report full Phase 1a/1b data in the first half of 2017 and initiate a Phase 2 study in 2018.”
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