Ponsegromab for the Treatment of Cancer Cachexia
Abstract
This phase 2 trial evaluated the efficacy of ponsegromab, a humanized monoclonal antibody targeting growth differentiation factor 15 (GDF-15), in patients with cancer cachexia and elevated GDF-15 levels (≥1500 pg/mL). The study involved 187 patients randomly assigned to receive ponsegromab (100 mg, 200 mg, or 400 mg) or placebo subcutaneously every 4 weeks for 12 weeks. The primary endpoint was change in body weight at 12 weeks. Results showed significant weight gain in all ponsegromab groups compared to placebo, with median differences of 1.22 kg (100 mg), 1.92 kg (200 mg), and 2.81 kg (400 mg). The 400-mg group also demonstrated improved appetite, reduced cachexia symptoms, and increased physical activity. Adverse events were similar across groups (70% ponsegromab vs. 80% placebo), supporting GDF-15 as a therapeutic target for cancer cachexia.