Pfizer‘s experimental drug for a common, life-threatening condition that causes cancer patients to lose appetite and weight showed positive results in a midstage trial, the drugmaker said Monday.
Patients with the condition, called cancer cachexia, who took Pfizer treatment saw improvements in body weight, muscle mass, quality of life and physical function, according to drugmakers. The results could pave the way for the drug, a monoclonal antibody called ponsegromab, to become the first treatment approved in the US specifically for cancer cachexia.
The condition affects about 9 million people worldwide, and 80% of cancer patients with the condition will die within a year of diagnosis, according to the company.
Patients with cancer cachexia do not eat enough food to meet the body’s energy needs, causing significant fat and muscle loss and making them weak, tired and, in some cases, unable to carry out their daily activities. Cancer cachexia is now defined as weight loss of 5% or more in the past six months in cancer patients, along with symptoms such as fatigue, according to the National Cancer Institute.
Symptoms of these conditions can make cancer treatments less effective and contribute to lower survival rates, Pfizer said.
“We will see ponsegromab fit into the treatment of cancer patients, correctly addressing the unmet need in cachexia, and thus, improving well-being, the ability to take care of themselves, and we also hope the ability to tolerate more treatment,” Charlotte Allerton, Pfizer’s head of discovery and early development, told CNBC in an interview.
Pfizer has not disclosed an estimated revenue potential for the drug, which could be approved for a variety of uses.
The company presented the data on Saturday at the Congress of the European Society for Medical Oncology 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 driver of cachexia called growth differentiation factor 15, or GDF-15. These are proteins that bind to certain receptors in the brain and have an effect on appetite, according to Allerton.
After 12 weeks, patients who took the highest dose of ponsegromab – 400 milligrams – saw a 5.6% increase in weight compared to those who received a placebo. Patients who took a 200-milligram or 100-milligram dose of the drug experienced an increase in body weight of approximately 3.5% and 2%, respectively, compared to the placebo group.
Allerton said the expert group defined a weight gain of more than 5% as a “clinically significant difference in cancer patients with cachexia.” He added that the drug’s effect on other health measures, such as increased appetite and physical activity, “is really encouraging.”
Pfizer says there have been no significant side effects with the drug. Treatment-related side effects occurred in 8.9% of people taking the placebo and 7.7% of those taking the Pfizer treatment, the company said.
The company said it is discussing late-stage development plans for the drug with regulators, and aims to start a study in 2025 that could be used to file for approval. Pfizer is also studying ponsegromab in a phase two trial in heart failure patients, who may also suffer from cachexia.
The Pfizer drug works by reducing GDF-15 levels. Pfizer believes this can increase 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 actually seeing this upregulation of GDF-15 in more of these chronic conditions, and in this case, cancer,” Allerton said.
