Clinical Trials

Date: 2017-05-04

Type of information: update on patient enrollment

phase: 1b


Company: Caelum Biosciences (USA - NY)

Product: CAEL-101

Action mechanism: monoclonal antibody. CAEL?101 is a fibril?reactive monoclonal antibody.

Disease: amyloid light chain amyloidosis

Therapeutic area: Rare diseases - Genetic diseases


Trial details: The Phase 1a/1b study is examining the tolerance, safety, pharmacokinetics and possible clinical benefit of CAEL-101, a chimeric fibril?reactive monoclonal antibody, in patients with AL amyloidosis. CAEL-101 is administered to patients through a single intravenous infusion in the Phase 1a, and one weekly infusion for four weeks during the Phase 1b. The first patient received the starting dose and, if tolerated, the following patients received progressively higher doses. When trial investigators reached the maximum tolerated dose without toxicity, investigators enrolled another six patients to receive the same dose. (NCT02245867)

Latest news: • On May 4, 2017, Fortress Biotech announced that study sponsor Columbia University has dosed the final patient in the Phase 1b clinical trial of CAEL-101, in development by Fortress subsidiary Caelum Biosciences, for the treatment of amyloid light chain (“AL”) amyloidosis. Caelum expects to readout preliminary Phase 1b data mid-2017, and full data by the end of the year. Interim Phase 1a/1b data presented by Columbia at the American Society of Hematology’s (ASH) 58th Annual Meeting demonstrated that CAEL?101 is safe and well-tolerated with no drug?related adverse events or dose?limiting toxicity up to 500mg/m2. A single infusion of CAEL?101, or one weekly infusion for four weeks, provided early and sustained organ response in cardiac, renal, gastrointestinal, skin and soft tissue. Interim clinical efficacy data showed that CAEL?101 promoted amyloid resolution in 67 percent of patients (63 percent in Phase 1a; 70 percent in Phase 1b).

Is general: Yes