Pfizer's Cancer Cachexia Study Meets Primary Endpoint, Moving Towards Late-Stage Trials
Pfizer's Phase 2 study of ponsegromab met its primary endpoint for body weight increase in patients with cancer cachexia. Registration-enabling studies are set to begin in 2025.
Pfizer Inc. announced on September 14, 2024, positive outcomes from its Phase 2 study evaluating ponsegromab for the treatment of cancer cachexia. The drug, a monoclonal antibody targeting growth differentiation factor 15 (GDF-15), successfully met its primary endpoint of increasing patient body weight compared to placebo.
The study involved 187 participants with non-small cell lung cancer, pancreatic cancer, or colorectal cancer who had elevated GDF-15 levels. At the highest dose tested, 400 mg, patients experienced a mean increase in body weight of 5.6% over 12 weeks. Improvements were also observed in appetite, cachexia symptoms, physical activity, and muscle mass. The treatment was generally considered safe and well-tolerated across all dose levels.
Cancer cachexia is a common and life-threatening condition characterized by significant weight loss and functional decline in cancer patients, severely impacting their quality of life. Dr. Jeffrey Crawford, principal investigator and researcher at Duke Cancer Institute, noted that the findings offer hope for a breakthrough targeted therapy.
Based on these encouraging Phase 2 results, Pfizer is planning to initiate registration-enabling studies in 2025. The study data was simultaneously published in The New England Journal of Medicine. Ponsegromab is also under investigation for heart failure patients with elevated GDF-15 concentrations.