Pfizer Presents Positive Data from Phase 2 Study of Ponsegromab in Patients with Cancer Cachexia

Pfizer Inc. (NYSE: PFE) today announced its Phase 2 study of ponsegromab, a monoclonal antibody directed against growth differentiation factor-15 (GDF-15), met its primary endpoint of change from baseline in body weight compared to placebo in people with cancer cachexia and elevated levels of GDF-15. Cachexia is a common, life-threatening wasting condition that can significantly impact quality of life in affected patients with cancer.^ii,iii Results will be presented today as a late-breaking Proffered Paper Presentation (LBA82) at the European Society for Medical Oncology (ESMO) 2024 Congress and have simultaneously been published in The New England Journal of Medicine (NEJM).

“Cachexia is a common condition in cancer patients, associated with weight loss, functional decline, and ultimately poor outcomes. Despite the number of people suffering from cachexia, there are no available options for us to help treat patients,” said Jeffrey Crawford, M.D., George Barth Geller Professor for Research at Duke Cancer Institute, and principal investigator. “This study showed us those who received ponsegromab had improvement in body weight, muscle mass, quality of life, and physical function. These findings offer hope that a breakthrough targeted treatment is potentially on the horizon for our patients.”

The Phase 2 study included 187 participants with non-small cell lung cancer, pancreatic cancer, or colorectal cancer. Ponsegromab demonstrated significant and robust increases in body weight after 12 weeks across all doses: 2.02% (95% confidence interval (CI), -0.97 to 5.01%) in the 100 mg treatment group, 3.48% (95% CI, 0.54 to 6.42%) in the 200 mg group, and 5.61% (95% CI, 2.56 to 8.67%) in the 400 mg group, compared to placebo. Improvements across multiple domains of the cachexia phenotype were observed in the 400 mg group including in measures of appetite and cachexia symptoms, physical activity and skeletal muscle index. No clinically significant adverse trends were noted with ponsegromab administration. Treatment related adverse events occurred in 8.9% of patients taking placebo and 7.7% of patients taking ponsegromab.ⁱ

“Discovered and developed in-house at Pfizer, ponsegromab represents our ability to translate deep scientific expertise into patient benefit,” said Charlotte Allerton, Head of Discovery and Early Development, Pfizer. “These results provide strong evidence that we have unlocked a mechanism to interrupt a critical driver of cachexia, GDF-15, which has the potential to impact patients with cancer cachexia and other life-threatening conditions. We look forward to advancing this program as part of our broader cardiometabolic portfolio to address weight management across the spectrum of patient need.”