Pfizer Inc.’s experimental drug for cancer-related weight loss has shown promise in helping patients regain weight in a mid-stage study, offering hope for addressing the serious condition of muscle wasting.
This condition, known as cachexia, often affects cancer patients, causing metabolic and appetite changes. It leads to the loss of essential skeletal muscle and fat, which weakens the body and can sometimes reduce the effectiveness of cancer treatments.
Research suggests that cachexia is responsible for up to 30% of cancer deaths, and around 80% of advanced-stage cancer patients experience it. Currently, there are no approved treatments for cachexia.
Pfizer’s drug, ponsegromab, is a monoclonal antibody that inhibits GDF-15, a protein produced when the body is under extreme stress. Elevated levels of GDF-15 in cancer patients are believed to be linked to reduced appetite and nausea. Based on this, Pfizer’s researchers theorized that blocking the protein might help prevent cachexia.
The drug was developed internally at Pfizer, which has been focusing on cancer treatments as part of its growth strategy. Last year, Pfizer acquired Seagen Inc., a cancer drug company, for $43 billion. Investors have been looking for positive updates as sales from Pfizer’s COVID-19 vaccine decline.
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In a Phase 2 study, Pfizer enrolled 187 cancer patients with high GDF-15 levels to assess ponsegromab’s impact on body weight compared to a placebo. The trial included patients with non-small cell lung cancer, pancreatic cancer, or colorectal cancer, with about 75% of them in stage 4.
At the highest dose of 400 milligrams, patients regained approximately 5.6% of their body weight over 12 weeks, according to results published in the New England Journal of Medicine. These patients also experienced improved appetite, physical activity, and skeletal muscle health.
The findings will be presented at the European Society for Medical Oncology conference in Barcelona. Charlotte Allerton, Pfizer’s head of discovery and early development, noted that the company is optimistic ponsegromab will help patients gain enough weight to better tolerate treatments and maintain physical activity, ultimately improving their overall health.
Pfizer is currently in discussions with regulatory agencies and plans to begin registration-enabling studies by 2025. The company is also investigating ponsegromab’s potential in treating heart failure and other conditions related to elevated GDF-15 levels, such as chronic kidney disease.
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