Pfizer reports positive trial results for experimental drug targeting deadly condition causing appetite and weight loss in cancer patients.

• The midstage trial of Pfizer's experimental drug for a common, life-threatening condition that causes cancer patients to lose their appetite and weight showed positive results.
• Individuals with cancer cachexia who received Pfizer's treatment experienced enhancements in body weight, muscle mass, quality of life, and physical function.
• The development of results could lead to the approval of ponsegromab, a monoclonal antibody, as the first treatment specifically for cancer cachexia.

The experimental drug of the drugmaker for a common, life-threatening condition that causes cancer patients to lose their appetite and weight showed positive results in a midstage trial, the drugmaker said Saturday.

The monoclonal antibody called ponsegromab, developed by Pfizer, has shown improvements in body weight, muscle mass, quality of life, and physical function for patients with cancer cachexia, according to the drugmaker. These results could lead to the drug becoming the first treatment in the U.S. specifically approved for cancer cachexia.

Approximately 9 million people worldwide are affected by the condition, and 80% of cancer patients with it are predicted to die within one year of diagnosis, according to the company.

Cancer patients who suffer from cachexia do not consume enough food to meet their body's energy requirements, resulting in substantial weight loss of fat and muscle, leaving them weak, tired, and sometimes unable to perform routine tasks. According to the National Cancer Institute, cachexia is defined as a 5% or greater decrease in body weight over the past six months in cancer patients, accompanied by symptoms such as fatigue.

Pfizer stated that the symptoms of the condition can reduce the effectiveness of cancer treatments and lower survival rates.

Charlotte Allerton, Pfizer's head of discovery and early development, stated in an interview with CNBC that ponsegromab could be a suitable treatment for cancer patients, addressing the unmet need in cachexia and improving their wellness, ability to care for themselves, and tolerance for more treatment.

The estimated revenue opportunity of the drug, which could potentially be approved for different uses, has not been disclosed by Pfizer.

The European Society for Medical Oncology 2024 Congress, held in Barcelona, Spain, saw the company present the data on Saturday. The findings were also published in The New England Journal of Medicine.

The phase two trial involved 187 individuals with non-small cell lung cancer, pancreatic cancer, or colorectal cancer and high levels of GDF-15, a protein that binds to a specific receptor in the brain and affects appetite, as stated by Allerton.

Patients who received the highest dose of ponsegromab, 400 milligrams, experienced a 5.6% increase in weight compared to those who received a placebo. Those who took a 200-milligram or 100-milligram dose of the drug saw a roughly 3.5% and 2% increase in body weight, respectively, compared with the placebo group.

Allerton stated that a group of experts defines a weight gain of more than 5% in cancer patients with cachexia as a "clinically significant difference." She emphasized that the drug's impact on other aspects of wellness, such as increased appetite and physical activity, is what provides the most optimism.

Pfizer stated that they did not observe any significant side effects with the drug. The company reported that treatment-related side effects occurred in 8.9% of people taking a placebo and 7.7% of those who took Pfizer's treatment.

Pfizer is currently discussing late-stage development plans for the drug with regulators and aims to commence studies in 2025 to file for approval. Additionally, the company is conducting a phase two trial of ponsegromab in patients with heart failure who also suffer from cachexia.

Pfizer's drug reduces GDF-15 levels to improve appetite and enable weight gain.

GDF-15 levels in our tissues are typically low when we're healthy, but this upregulation of GDF-15 is commonly observed in chronic conditions, including cancer.