Pfizer’s cancer cachexia drug shows positive results in mid-stage trial

PfizerThe experimental drug for a common, life-threatening condition that causes cancer patients to lose their appetite and weight showed positive results in a mid-stage trial, the drugmaker said Saturday.

Patients with the condition, called cancer cachexia, who received Pfizer’s treatment saw 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.

According to the company, approximately 9 million people worldwide suffer from the condition and 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, leading to significant fat and muscle loss 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 in the past six months in cancer patients, along with symptoms such as fatigue, according to the National Cancer Institute.

According to Pfizer, the condition’s symptoms can make cancer treatments less effective and reduce survival rates.

“We would like ponsegromab to play a role in the treatment of cancer patients, and really address the unmet need in cachexia, and improve their well-being and their ability to care for themselves, and we hope that they will be able to tolerate more treatments,” Charlotte Allerton, head of discovery and early development at Pfizer, told CNBC in an interview.

Pfizer did not disclose the drug’s estimated sales potential. The drug could potentially be approved for several uses.

The company presented the data Saturday at the European Society for Medical Oncology 2024 Congress, a cancer research conference in Barcelona, ​​Spain. The results were also published in The New England Journal of Medicine.

The phase two study 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. It’s a protein that binds to a certain receptor in the brain and influences appetite, Allerton said.

After 12 weeks, patients taking the highest dose of ponsegromab — 400 milligrams — saw a 5.6 percent gain in weight compared with those taking a placebo. Patients taking a 200-milligram or 100-milligram dose of the drug saw about a 3.5 percent and 2 percent gain in body weight, respectively, compared with the placebo group.

Allerton said an expert task force defined a weight gain of more than 5 percent as a “clinically meaningful difference in cancer patients with cachexia.” She added that the drug’s effect on other measures of well-being, such as increased appetite and physical activity, “is really what gives us the encouragement.”

Pfizer said it had not observed any significant side effects from the drug. Treatment-related side effects occurred in 8.9% of people taking a placebo and in 7.7% of those taking the Pfizer treatment, the company said.

The company said it is discussing plans for late-stage development of the drug with regulators and hopes to begin studies in 2025 that could be used to file for approval. Pfizer is also studying ponsegromab in a phase two trial in patients with heart failure, who can also have cachexia.

Pfizer’s drug works by lowering levels of GDF-15. Pfizer believes this could improve appetite and allow patients to maintain or even gain weight.

“Most of us have low levels of GDF-15 in our tissues when we’re healthy, but we see these elevated GDF-15 levels more often in chronic conditions, and in this case, cancer,” Allerton said.