Pfizer has announced positive results from a midstage clinical trial of its experimental drug, ponsegromab, intended to treat cancer cachexia, a serious condition characterised by weight loss and appetite decline in cancer patients.
The trial demonstrated that participants who received ponsegromab experienced significant improvements in body weight, muscle mass, quality of life, and physical function.
Cancer cachexia affects approximately 9 million people worldwide, with around 80% of cancer patients suffering from it expected to die within a year of diagnosis. This condition leads to insufficient food intake to meet the body’s energy needs, resulting in substantial fat and muscle loss, which can weaken patients and hinder their ability to perform daily activities.
The National Cancer Institute defines cancer cachexia as a loss of 5% or more of body weight over six months, accompanied by symptoms such as fatigue.
The phase two trial involved 187 participants diagnosed with non-small cell lung cancer, pancreatic cancer, or colorectal cancer, all exhibiting high levels of growth differentiation factor 15 (GDF-15), a protein linked to appetite regulation.
After 12 weeks of treatment, patients receiving the highest dose of ponsegromab (400 mg) showed a 5.6% increase in body weight compared to those on placebo. Patients receiving 200 mg and 100 mg doses experienced weight gains of approximately 3.5% and 2%, respectively.
Pfizer presented these findings at the European Society for Medical Oncology Congress and published them in The New England Journal of Medicine. The company is currently discussing plans for late-stage development with regulatory authorities and aims to initiate studies in 2025 that could lead to approval.
Ponsegromab functions by reducing GDF-15 levels, which is believed to enhance appetite and help patients maintain or gain weight. The drug was well-tolerated during the trial, with treatment-related side effects occurring in 7.7% of ponsegromab recipients compared to 8.9% among placebo participants.
In addition to its focus on cancer cachexia, Pfizer is also exploring ponsegromab’s effects on patients with heart failure who may experience similar cachexia symptoms. The ongoing research aims to address the unmet medical needs associated with this debilitating condition and improve the well-being of affected patients.
