Pfizer reported positive results from a midstage trial of their experimental drug, ponsegromab, designed to treat cancer cachexia. Cancer cachexia is a life-threatening condition that causes cancer patients to lose their appetite and weight. The drug showed improvements in body weight, muscle mass, quality of life, and physical function in patients with cancer cachexia. This could potentially make ponsegromab the first treatment approved specifically for cancer cachexia in the U.S. The condition affects about 9 million people worldwide, with 80% of cancer patients suffering from it expected to die within a year of diagnosis.
Patients with cancer cachexia experience significant fat and muscle loss due to not eating enough food to meet their body’s energy needs. This can lead to weakness, fatigue, and difficulties in performing daily activities. The symptoms of cancer cachexia can make cancer treatments less effective and contribute to lower survival rates. Pfizer sees ponsegromab as addressing an unmet need in cachexia and potentially improving patients’ wellness, ability to care for themselves, and tolerance for more treatment. The estimated revenue opportunity for the drug has not been disclosed by Pfizer, which could potentially have uses beyond cancer cachexia.
Pfizer presented the results of the phase two trial of ponsegromab at the European Society for Medical Oncology 2024 Congress and published them in The New England Journal of Medicine. The trial involved 187 people with non-small cell lung cancer, pancreatic cancer, or colorectal cancer who had high levels of a key driver of cachexia called growth differentiation factor 15 (GDF-15). After 12 weeks, patients who took the highest dose of ponsegromab saw a 5.6% increase in weight compared to those who received a placebo. The drug was also well-tolerated, with no significant side effects observed.
Ponsegromab works by reducing levels of GDF-15, a protein that impacts appetite by binding to a specific receptor in the brain. Pfizer believes that by lowering GDF-15 levels, the drug can improve appetite and help patients maintain or gain weight. The company is in discussions with regulators regarding late-stage development plans for ponsegromab and aims to start studies in 2025 to support approval. In addition to cancer cachexia, Pfizer is also studying the drug in a phase two trial for patients with heart failure, another condition where cachexia can occur.
The potential approval of ponsegromab could be a significant milestone in the treatment of cancer cachexia, a condition that currently lacks specific approved treatments. By addressing the symptoms of cachexia and improving patients’ wellness, the drug has the potential to enhance cancer treatment outcomes and survival rates. Further studies and regulatory discussions will determine the drug’s path to approval, but the positive results from the phase two trial are encouraging for both patients and healthcare providers in the fight against cancer cachexia.
