PfizerAn experimental drug for a common, life-threatening condition that causes cancer patients to lose appetite and weight has shown positive results in a mid-stage trial, the drugmaker said Saturday.
Patients with a condition called cancer cachexia who received the Pfizer treatment saw improvements in body weight, muscle mass, quality of life and physical activity, according to the drug maker. The results could pave the way for the monoclonal antibody drug ponsegromab to become the first treatment approved specifically for cancer cachexia in the United States.
The disease affects approximately 9 million people worldwide and, according to the company, 80% of cancer patients are expected to die within a year of diagnosis.
Patients with cancer cachexia do not eat enough to meet their body’s energy needs, which causes significant fat and muscle loss, leaving them weak, tired, and in some cases unable to perform daily tasks. According to the National Cancer Institute, cancer cachexia is currently defined as a loss of 5% or more of body weight over the past six months, along with symptoms such as fatigue in cancer patients.
Symptoms of the condition can make cancer treatments less effective and lower survival rates, Pfizer said.
“We would see ponsegromab adapt to the treatment of cancer patients, address the unmet need in cachexia, and thereby improve their health, their ability to care for themselves, and hopefully their ability to tolerate more treatment,” Charlotte said. Allerton, Pfizer’s head of discovery and early development, told CNBC in an interview.
Pfizer did not disclose the estimated revenue potential of the drug, which could potentially be approved for a variety of uses.
The company presented the data on Saturday at the European Society of Medical Oncology Congress 2024, a cancer research conference in Barcelona, ​​Spain. The results were also published in The New England Journal of Medicine.
The second phase trial followed 187 people with small cell lung cancer, pancreatic or colorectal cancer and high levels of a key driver of cachexia called growth differentiation factor 15, or GDF-15. According to Allerton, it is a protein that binds to a specific receptor in the brain and affects appetite.
After 12 weeks, patients who received the highest dose of ponsegromab, 400 milligrams, saw a 5.6% increase in weight compared to those who received placebo. Patients who received doses of 200 milligrams or 100 milligrams of the drug had an increase in body weight of about 3.5% and 2%, respectively, compared to the placebo group.
Allerton said the expert task force defined weight gain of more than 5% as a “clinically significant difference in cancer patients with cachexia.” He added that the drug’s effects on other measures of health, such as increased appetite and physical activity, “are what really excites us.”
Pfizer said it has not observed any significant side effects with the drug. Treatment-related side effects occurred in 8.9% of placebo-treated patients and 7.7% of Pfizer-treated patients, the company said.
The company said it is discussing late-stage development plans for the drug with regulators and aims to begin studies in 2025 that could be used to apply for approval. Pfizer is also studying ponsegromab in phase II trials in heart failure patients with cachexia.
The Pfizer drug works by reducing levels of GDF-15. Pfizer believes it may improve appetite and allow patients to maintain and gain weight.
“For most of us, we have low levels of GDF-15 in our tissues when we’re healthy, but we’re really seeing upregulation of GDF-15 in these chronic conditions and in this case, cancer.” Allerton said.
