Phase 1b trial of ponsegromab in patients with cancer and cachexia

Cachexia is common in patients with advanced cancer and is associated with elevated serum growth differentiation factor 15 (GDF-15) concentration. A first-in-patient phase 1b trial assessed use of ponsegromab, a monoclonal antibody against GDF-15 in adults with advanced cancer, cachexia, and elevated GDF-15 serum concentration. Ten patients received ponsegromab 200 mg every 3 weeks for 12 weeks in addition to standard anti-cancer treatment. From day 1 through week 15 increases in body weight were observed at all time points during treatment and follow-up. A least-squares mean increase of 4.63 kg (SE 1.98) was observed at week 12, an increase of approximately 6.6% relative to baseline. Improvements in physical activity and quality of life, including appetite, were also observed. The authors conclude that ponsegromab was well tolerated and demonstrated preliminary evidence of efficacy.