Pfizer (NYSE: PFE) recently announced positive results from a midstage trial of their experimental drug, ponsegromab, designed to treat cancer cachexia, a life-threatening condition causing appetite and weight loss in cancer patients. The study showed that patients receiving ponsegromab experienced improvements in body weight, muscle mass, quality of life, and physical function. This breakthrough could lead to the first U.S. approval of a treatment specifically for cancer cachexia, a condition affecting millions globally and associated with high mortality rates within a year of diagnosis.
Cancer cachexia, characterized by insufficient food intake and significant fat and muscle loss, weakens patients and can hinder cancer treatment effectiveness. Pfizer’s ponsegromab targets GDF-15, a protein linked to appetite regulation, aiming to improve appetite and facilitate weight maintenance and gain.
The phase two trial involved 187 patients with various cancers and high GDF-15 levels. Those receiving the highest dose of ponsegromab showed a clinically meaningful weight increase, along with improvements in other wellness measures. The drug was well-tolerated with no significant side effects.
Pfizer is now in discussions with regulators regarding late-stage development plans and aims to initiate studies in 2025 for potential approval. The company is also exploring the drug’s potential in treating cachexia associated with heart failure.
This positive development offers hope for cancer patients struggling with cachexia, potentially improving their quality of life and treatment outcomes.