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Ponsegromab is a potent, highly selective, humanized monoclonal antibody designed to treat cachexia, particularly in patients with advanced cancer. Its mechanism of action involves binding to and inhibiting growth differentiation factor 15 (GDF-15), a cytokine implicated in the development of cachexia. By blocking GDF-15 from interacting with its receptor GFRAL in the brainstem, ponsegromab disrupts signaling pathways that lead to reduced appetite, altered metabolism, and muscle wasting. Clinical trials have shown that ponsegromab increases body weight, improves appetite and physical activity levels, and reduces the severity of cachexia symptoms compared to placebo. The drug is being developed by Pfizer and has demonstrated promising results in phase 1b and phase 2 studies for cancer cachexia; it is also under investigation for heart failure.
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