PfizerThe company’s experimental drug for a common, life-threatening condition that causes appetite and weight loss in cancer patients has shown positive results in mid-stage clinical trials, the drugmaker said Saturday.
Pfizer said patients with the condition, known as cancer cachexia, saw improvements in weight, muscle mass, quality of life and physical function after receiving its treatment — findings that could pave the way for the drug, a monoclonal antibody called ponsegromab, to be approved in the U.S. as the first treatment specifically for cancer cachexia.
The disease affects approximately nine million people worldwide, with 80% of cancer patients suffering from the disease expected to die within a year of diagnosis, according to the company.
Patients with cancer cachexia are unable to eat enough to meet the body’s energy needs, resulting in significant loss of fat and muscle mass, weakness, fatigue, and in some cases, inability to perform daily activities. According to the National Cancer Institute, cancer cachexia is currently defined as a condition in which a cancer patient has lost 5% or more of their body weight in the past six months, accompanied by symptoms such as fatigue.
Pfizer said symptoms of the disease can make cancer treatment less effective and decrease survival rates.
“We hope that ponsegromab can help treat cancer patients and really address the unmet need of cachexia, thereby improving their health, their ability to manage themselves and better tolerate treatment,” Charlotte Allerton, head of discovery and early development at Pfizer, said in an interview with CNBC.
Pfizer has not disclosed its estimated revenue opportunity, saying the drug could be approved for a variety of uses.
The company presented the data on Saturday at the European Society for Medical Oncology 2024 Congress, a cancer research congress in Barcelona, ​​Spain. The results were also published in the New England Journal of Medicine.
The phase 2 trial followed 187 patients with non-small cell lung, pancreatic or colorectal cancer who had high levels of growth differentiation factor 15 (GDF-15), a protein that binds to specific receptors in the brain and affects appetite, a major cause of cachexia, Allerton said.
After 12 weeks, patients who took the highest dose of ponsegromab, 400 milligrams, gained 5.6 percent more weight than those who took a placebo, while patients who took the 200 milligram or 100 milligram doses gained about 3.5 percent and 2 percent more weight, respectively, compared with the placebo group.
Allerton said an expert working group defined a weight gain of 5 percent or more as “a clinically meaningful change in cachectic cancer patients,” adding that the drug’s effects on other markers of health, such as increased appetite and physical activity, “are really encouraging to us.”
Pfizer said no serious side effects had been seen with the drug. Treatment-related side effects occurred in 8.9 percent of people who took a placebo and 7.7 percent of people receiving Pfizer’s treatment, the company said.
The company said it is in discussions with regulators about plans for the drug’s later-stage development, aiming to begin a regulatory-ready study in 2025. The company is also conducting a Phase 2 trial of ponsegromab in heart failure patients who may suffer from cachexia.
Pfizer’s drug works by lowering levels of GDF-15, which the company believes could increase appetite and enable patients to maintain or gain weight.
“Most healthy people have low levels of GDF-15 in their tissues, but in chronic disease, in this case cancer, we actually see elevated GDF-15,” Allerton said.