Together, Caelum Biosciences and Alexion Pharmaceuticals presented new Phase 2 safety and tolerability data for CAEL-101, a monoclonal antibody currently under clinical development for the treatment of AL amyloidosis.
In a press release from Alexion, the two companies reported their latest findings through two e-posters presented at the latest European Hematology Association (EHA) Congress 2021. With the latest safety and tolerability data, the profile of CAEL-101 is strengthened and its dose selection in the ongoing Phase 3 study is supported.
Results of Clinical Trials With CAEL-101
The data provided by Caelum and Alexion suggests possible cardiac disease improvement and renal response. In the first e-poster, CAEL-101 administered in combination with cyclophosphamide-bortezomib-dexamethasone (CyBorD), a three-drug combination used for patients with myeloma, plus daratumumab, also a monoclonal antibody used against myeloma, was found to be "generally safe and well-tolerated" for the first four weeks of administration in the trials.
In the second e-poster, Caelum and Alexion present long-term evidence that CAEL-101, together with CyBorD, was generally well-tolerated for a treatment period of 49 weeks. Additionally, exploratory clinical biomarker data from the trials suggest improvements in cardiac disease and renal response from patients having these medical conditions at baseline.
"AL amyloidosis is a relentless disease that is particularly devastating when it impacts the heart, with some of these patients facing a median survival of less than one year following diagnosis, says Caelum President and CEO Michael Spector in a statement. "Current treatments for AL amyloidosis are designed to prevent or suppress the formation of new amyloids, but they do not address the existing amyloid buildup in the involved organs like the heart and kidneys, which can result in continued organ damage and can ultimately be fatal."
As noted in a September 2020 announcement, also from Alexion and Caelum, the Phase 2 study regarding CAEL-101 administered together with CyBorD met its primary objectives, previously supporting the safety and tolerability of the exploratory biomarker.
The Phase 2 trial is a multicenter, open-label, dose-selection study designed to assess the safety and tolerability of CAEL-101 together with the standard of care (SoC) therapy for patients with Amyloid light-chain (AL) amyloidosis, as well as identifying the recommended dose to be used for its Phase 3 studies.
About CAEL-101
CAEL-101 is a monoclonal antibody (mAb), or a man-made protein that behave like natural human antibodies in the immune system. This is designed to improve organ function by reducing amyloid deposits for patients suffering from AL amyloidosis. CAEL-101 attaches itself to misfolded light-chain proteins.
In its earlier Phase 1a/1b, CAEL-101 demonstrated improvement in organ function, including cardiac and renal response, in patients with both relapsed and refractory AL amyloidosis. CAEL-101 is already recognized with the Orphan Drug Designation from the US Food and Drug Administration, as well as the European Medicine Agency, as a potential treatment for AL amyloidosis.
According to Cedars-Sinai Medical Group, AL amyloidosis is a rare disease caused by the accumulation of light-chain proteins. Usually, this protein is created by a malignant, or pre-malignant, growth of identical lymphocytes that continue synthesizing these proteins.
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