Pfizer Inc. experimental medication, ponsegromab, has shown promise in the treatment of cancer cachexia, a potentially debilitating condition marked by severe appetite loss and wasting associated with some cancers. In a mid-stage clinical trial, Pfizer said that patients with the treatment showed improved body weight, muscle mass, quality of life, and physical functions. Results might make ponsegromab, a monoclonal antibody, the first medication approved specifically to treat cancer cachexia patients in the United States.
There are approximately 9 million patients affected with cancer cachexia, and from these, it is estimated that approximately 80% of cancer patients will succumb to death one year after diagnosis. The condition results in loss of body weight and muscle mass, which the individuals gain through reduced food intake. The degree of weakness, fatigue, and weakening inability to carry out the normal daily tasks notably emerge with no treatments approved up to now.
Speaking about the findings of the trial, Head of Discovery and Early Development for Pfizer Charlotte Allerton said: “Ponsegromab addresses a huge unmet need in cachexia, as it improves well-being and possibly even ability to tolerate further cancer treatments.” She added that the effect the drug had on gaining weight along with other parameters of well-being such as appetite and level of physical activity gives strong encouragement for further development of the drug.”.
The results of the phase two trial were presented at the European Society for Medical Oncology 2024 Congress in Barcelona, Spain, and published in The New England Journal of Medicine. In the study, 187 patients with non-small cell lung cancer, pancreatic cancer, and colorectal cancer were enrolled–all whose levels of a protein called growth differentiation factor 15 had been found to be elevated, and has ties with appetite. An increase in the body weight of patients by 5.6% was recorded at the end of 12 weeks for patients who were given the highest dose of ponsegromab at 400 mg. Increases of 3.5 and 2% were found in the body weight of patients treated with 200 and 100 mg doses, respectively, as compared to those who were given the placebo.
The no significant side effects reported, treatment-emergent adverse events involved 7.7% of patients, while treatment-emergent adverse events were noted in 8.9% of the placebo group, Pfizer said. This company is preparing to initiate phase III late-stage clinical trials in 2025 for regulatory approval and is still assessing this drug in curing cachexia in patients with heart failure.
This positive outcome is an important step toward the treatment of a disabling disease that significantly impacts the quality of life of most cancer patients.
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