Multinational phase 2 trial of ponsegromab for the treatment of cancer cachexia

Cachexia is a common complication of cancer and is associated with an increased risk of death. The level of growth differentiation factor 15 (GDF-15), a circulating cytokine, is elevated in cancer cachexia. A multinational phase 2 trial evaluated various levels of ponsegromab, a humanized monoclonal antibody inhibiting GDF-15, versus placebo among patients with cancer cachexia and elevaten GDF-15 levels. The median level of weight gain at 12 weeks was 2.81 kg (95% credible interval 1.55-4.08) in patients receiving ponsegromab 400 mg every 4 weeks for three doses. Improvements were also observed across measures of appetite and cachexia symptoms, along with physical activity. Adverse events of any cause were reported in 70% of patients in the ponsegromab group and 80% of patients in the placebo group. The authors conclude that among patients with cancer cachexia and elevated GDF-15 levels, the inhibition of GDF-15 with ponsegromab resulted in increased weight gain.