Pfizer Company‘s experimental drug for a common, life-threatening condition A drug that helps cancer patients lose their appetite and lose weight showed promising results in a mid-term trial, the drugmaker said Saturday.
Patients with the condition, known as cancer cachexia, who took Pfizer’s treatment saw improvements in weight, muscle mass, quality of life and physical function, the drugmaker said. The results could pave the way for the drug, a monoclonal antibody called ponsegromab, to become the first treatment approved in the United States specifically for cancer cachexia.
The condition affects about 9 million people worldwide, and 80 percent of cancer patients with the disease are expected to die within a year of diagnosis, according to the company.
Patients with cancer cachexia do not eat enough food to meet their body’s energy needs, causing significant loss of fat and muscle, leaving them weak, tired, and in some cases unable to perform daily activities. According to the definition of National Cancer Institute.
Symptoms of the condition can reduce the effectiveness of cancer treatment and reduce survival rates, Pfizer said.
“We’re going to see ponsegromab fit into the treatment of cancer patients, really addressing the unmet need in cachexia, and through that, improving their health, their ability to care for themselves, and we hope also their ability to tolerate more treatments,” Charlotte Allerton, Pfizer’s director of discovery and early development, told CNBC in an interview.
Pfizer has not disclosed the estimated revenue opportunity for the drug, which is likely to be approved for a variety of uses.
The company presented data on Saturday at European Medical Oncology Congress 2024a cancer research conference held in Barcelona, Spain. The results were also published in the New England Journal of Medicine.
The phase two trial followed 187 people with non-small cell lung cancer, pancreatic cancer, or colorectal cancer who had high levels of a key cachexia-inducing factor called growth differentiation factor 15, or GDF-15. This is a protein that binds to a specific receptor in the brain and affects appetite, according to Allerton.
After 12 weeks, patients taking the highest dose of ponsegromab — 400 milligrams — saw a 5.6 percent increase in weight compared to those taking a placebo. Patients taking 200 milligrams or 100 milligrams of the drug saw weight increases of about 3.5 percent and 2 percent, respectively, compared to the placebo group.
A group of experts defined a weight gain of more than 5% as “a clinically meaningful difference in cancer patients with cachexia,” Allerton said. She added that the drug’s effects on other health measures, such as increased appetite and physical activity, “are really what gives us the encouragement.”
Pfizer said it did not observe any significant side effects with the drug. The company said treatment-related side effects occurred in 8.9% of people taking the placebo and 7.7% of people taking 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 lead to an application for approval. Pfizer is also studying ponsegromab in a phase 2 trial in heart failure patients who may also have cachexia.
Pfizer’s drug works by reducing GDF-15 levels. Pfizer believes this may improve appetite and help patients maintain and gain weight.
“For most of us, GDF-15 levels in our body tissues are low when we are healthy, but we actually see this upregulation of GDF-15 in many more chronic disease states, and in this case, cancer,” says Allerton.
