Positive results from midstage trial of Pfizer’s cancer cachexia drug



Pfizer announced positive results from a midstage trial of its experimental drug for cancer cachexia, a condition that causes cancer patients to lose weight and appetite. The drug, ponsegromab, showed improvements in body weight, muscle mass, quality of life, and physical function in patients with cancer cachexia. This could lead to ponsegromab becoming the first approved treatment in the U.S. for this condition, which affects about 9 million people worldwide, with 80% of patients expected to die within a year of diagnosis. Cancer cachexia is characterized by significant weight loss, weakness, and fatigue, making cancer treatments less effective and decreasing survival rates.

Pfizer presented the trial data at a cancer research conference and published it in The New England Journal of Medicine. The trial included 187 people with non-small cell lung cancer, pancreatic cancer, or colorectal cancer and high levels of GDF-15, a key driver of cachexia. Patients who took ponsegromab saw weight increases of 5.6%, 3.5%, and 2% with doses of 400mg, 200mg, and 100mg, respectively, compared to a placebo. Pfizer is now discussing late-stage development plans with regulators and aims to start studies in 2025 for approval. The drug works by reducing levels of GDF-15, improving appetite, and enabling weight maintenance and gain in patients.

Pfizer believes that addressing the unmet need in cachexia with ponsegromab will improve wellness and tolerance to treatment in cancer patients. The drug had no significant side effects in the trial, with a low incidence of treatment-related side effects compared to placebo. Pfizer is also studying the use of ponsegromab in patients with heart failure, another condition associated with cachexia.



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