Pfizer’s promising experimental drug, ponsegromab, has shown positive results in a midstage trial for cancer cachexia, a common and life-threatening condition that causes cancer patients to lose weight and appetite. The drug, a monoclonal antibody, demonstrated improvements in body weight, muscle mass, quality of life, and physical function in patients with cancer cachexia, potentially making it the first approved treatment for the condition in the U.S.
According to Pfizer, around 9 million people worldwide suffer from cancer cachexia, with 80% of patients expected to die within a year of diagnosis. The debilitating symptoms of the condition include significant fat and muscle loss, weakness, fatigue, and diminished ability to perform daily activities, making cancer treatments less effective and lowering survival rates.
Charlotte Allerton, Pfizer’s head of discovery and early development, emphasized the drug’s potential to address the unmet needs of cancer patients with cachexia, improving their overall wellness and treatment tolerance. The company presented the promising trial data at the European Society for Medical Oncology 2024 Congress in Barcelona, Spain, and published the results in The New England Journal of Medicine.
In the phase two trial involving 187 patients with non-small cell lung, pancreatic, or colorectal cancer and high levels of growth differentiation factor 15 (GDF-15), a key driver of cachexia, ponsegromab demonstrated significant weight gain benefits. Patients who received the highest dose of 400 milligrams saw a 5.6% increase in body weight compared to the placebo group.
Pfizer is currently discussing late-stage development plans with regulators and aims to initiate studies in 2025 for potential approval. The company is also exploring the drug’s efficacy in patients with heart failure, who may also experience cachexia.
The drug works by reducing GDF-15 levels, which Pfizer believes can enhance appetite and help patients maintain or gain weight. Allerton highlighted the importance of reducing GDF-15 in chronic conditions like cancer to improve overall health outcomes.
As Pfizer progresses with the development of ponsegromab, experts anticipate potential implications for cancer treatment and patient care, offering hope for a groundbreaking therapy in the fight against cancer cachexia.
