PfizerPfizer's experimental drug for a common, potentially fatal disease that causes cancer patients to lose appetite and weight showed positive results in a mid-stage trial, the drugmaker said Saturday.
Patients with the condition, called cancer cachexia, who took Pfizer’s treatment experienced improvements in body 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 U.S. specifically for cancer cachexia.
The disease affects about 9 million people worldwide and 80% 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, leading to significant loss of fat and muscle and leaving them weak, fatigued and in some cases unable to perform daily activities. Cancer cachexia 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.
Symptoms of the disease can make cancer treatments less effective and contribute to lower survival rates, Pfizer said.
“We would see ponsegromab fitting into the treatment of cancer patients, really addressing that unmet need for cachexia and through that improving their well-being, their ability to care for themselves and also hopefully their ability to tolerate further treatment,” Charlotte Allerton, Pfizer's head of discovery and early development, told CNBC in an interview.
Pfizer has not disclosed the estimated revenue opportunity for the drug, which could potentially be approved for different uses.
The company presented the data on Saturday at the European Society for Medical Oncology Congress 2024, a 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 and high levels of a key cachexia factor called growth differentiation factor 15, or GDF-15. This is a protein that binds to a certain receptor in the brain and has an impact on appetite, Allerton said.
After 12 weeks, patients taking the highest dose of ponsegromab (400 milligrams) experienced a 5.6 percent weight gain compared with those given a placebo. Patients taking a 200-milligram or 100-milligram dose of the drug experienced a body weight gain of about 3.5 percent and 2 percent, respectively, compared with the placebo group.
Allerton said a working group of experts defines a weight gain of more than 5% as a “clinically significant difference in patients with cancer and cachexia.” He added that the drug’s effect on other measures of well-being, such as increased appetite and physical activity, is “what really gives us encouragement.”
Pfizer said it did not see any significant side effects from the drug. Treatment-related side effects occurred in 8.9% of people taking a placebo and 7.7% of those taking Pfizer's treatment, 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 can be used to apply for approval. Pfizer is also studying ponsegromab in a phase two trial in patients with heart failure, who can also suffer from cachexia.
Pfizer's drug works by reducing levels of GDF-15. Pfizer believes this may improve appetite and allow patients to maintain and gain weight.
“Most of us have low levels of GDF-15 in our tissues when we're healthy, but we're really seeing this upregulation of GDF-15 in more of these chronic conditions and, in this case, cancer,” Allerton said.
