PfizerThe U.S. drugmaker said on Saturday its experimental drug to treat a common, life-threatening condition that causes cancer patients to lose their appetite and weight showed positive results in a mid-stage trial.
According to the drug company, patients with the condition, called “cancer wasting,” who took Pfizer’s treatment saw improvements in body weight, muscle mass, quality of life and physical function. The findings could pave the way for the drug, a monoclonal antibody called ponsegorumab, to become the first treatment approved in the United States specifically for cancer wasting.
The condition affects about 9 million people worldwide, and 80% of cancer patients with it are expected to die within one year of diagnosis, according to the company.
Cancer patients with wasting do not eat enough food to meet their bodies’ energy needs, causing significant loss of fat and muscle and leaving them weak, tired and in some cases unable to perform daily activities. Cancer wasting is currently defined as a loss of 5% or more of body weight over the past six months in cancer patients, along with symptoms such as fatigue, according to the National Cancer Institute.
Pfizer said symptoms of the condition may make cancer treatments less effective and contribute to lower survival rates.
“We see ponsegrumab as a good fit for cancer patients, really addressing that unmet need in wasting, and through that, improving their health and their ability to care for themselves, and hopefully their ability to tolerate more treatment,” Charlotte Allerton, Pfizer’s head of discovery and early development, told CNBC.
Pfizer did not disclose estimated revenue opportunities for the drug, which could potentially be approved for a variety of uses.
The company presented the data Saturday at the European Society for Medical Oncology 2024 Congress, a cancer research conference held in Barcelona, Spain. The results were also published in the New England Journal of Medicine.
The second phase of the study followed 187 people with non-small cell lung cancer, pancreatic cancer or colorectal cancer who had high levels of a key factor that causes wasting called growth differentiation factor 15, or GDF-15. It's a protein that binds to a specific receptor in the brain and has an effect on appetite, Allerton said.
After 12 weeks, patients who took the highest dose of ponsegrumab — 400 mg — gained 5.6% more weight than those who received a placebo. Patients who took the 200 mg or 100 mg dose of the drug gained about 3.5% and 2% more body weight, respectively, than the placebo group.
A working group of experts defines a weight gain of more than 5% as a “clinically meaningful difference in cancer patients who are lean,” Allerton said. The drug’s effect on other measures of health, such as increased appetite and physical activity, is “what really gives us encouragement,” she added.
Pfizer said it had not observed any major side effects from the drug. The company said treatment-related side effects occurred in 8.9% of people who took a placebo and 7.7% of those who took Pfizer's treatment.
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 file for approval. Pfizer is also studying bonsegorumab in a phase 2 trial in patients with heart failure, who may also be wasted.
Pfizer's drug works by lowering levels of GDF-15. Pfizer believes this will improve appetite and enable patients to maintain and gain weight.
“For most of us, we have low levels of GDF-15 in our tissues when we are healthy, but we are actually seeing upregulation of GDF-15 in more of these chronic conditions, in this case, cancer,” Allerton said.
