CAEL-101 is a chimeric fibril-reactive monoclonal antibody (mAb) currently being evaluated in a Phase 1a/1b study for the treatment of patients with AL amyloidosis (ClinicalTrials.gov Identifier: NCT02245867).
Interim data presented by Suzanne Lentzsch, MD at ASH’s 58th Annual Meeting in December 2016 demonstrated that CAEL-101 is safe and well-tolerated with no drug-related grade four or five adverse events or dose-limiting toxicity up to 500mg/m2. A single infusion of CAEL-101 once per week for four weeks exhibited early and sustained organ response in cardiac, renal, gastrointestinal, skin and soft tissue, independent of light-chain subtype.
Interim clinical efficacy data have shown that CAEL-101 has promoted amyloid resolution in 67 percent of patients (63 percent in Phase 1a; 70 percent in Phase 1b). Caelum expects to report full data from the Phase 1a/1b study in mid-2017, and to initiate a Phase 2 study in 2018.
Caelum Biosciences holds two Orphan Drug Designations for CAEL-101, one for use as a therapeutic agent for patients with AL amyloidosis and one for use as a radioimmaging agent in amyloidosis.