PfizerThe New Zealand's experimental drug for a common, life-threatening disease that causes appetite and weight loss in cancer patients has shown positive results in a mid-stage trial, the drugmaker said on Saturday.
Patients with the condition, called cancer cachexia, who took Pfizer's drug showed improvements in body weight, muscle mass, quality of life and physical function, according to the drugmaker. The results 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 about nine million people worldwide and 80 percent of affected cancer patients are expected to die within a year of diagnosis, according to the company.
Patients with cancer cachexia do not eat enough to meet their body's energy needs, resulting in significant fat and muscle loss and leaving them weak, tired, and in some cases unable to perform daily activities. Cancer cachexia is currently defined as a loss of 5% or more body weight in cancer patients over the past six months, along with symptoms such as fatigue, according to the National Cancer Institute.
The disease's symptoms could affect the effectiveness of cancer treatments and contribute to lower survival rates, Pfizer said.
“We would see ponsegromab fit into the treatment of cancer patients and really address this unmet need in cachexia, improving their well-being and their ability to care for themselves. And we also hope that they tolerate the treatment better,” said Charlotte Allerton, head of discovery and early development at Pfizer, in an interview with CNBC.
Pfizer has not disclosed the estimated sales potential of the drug, which could potentially be approved for various uses.
The company presented the data on Saturday at the European Society for Medical Oncology 2024 Congress, a cancer research conference held in Barcelona, Spain. The results were also published in the New England Journal of Medicine.
The second phase trial involved 187 people with non-small cell lung cancer, pancreatic cancer or colon cancer who had high levels of a key factor in cachexia called growth differentiation factor 15, or GDF-15. Allerton said it is a protein that binds to a specific receptor in the brain and affects appetite.
After 12 weeks, patients who took the highest dose of ponsegromab – 400 milligrams – experienced a 5.6 percent weight gain compared to those who received a placebo. Patients who took a 200-milligram or 100-milligram dose of the drug experienced a weight gain of about 3.5 percent and 2 percent, respectively, compared to the placebo group.
Allerton said a working group of experts defined a weight gain of more than 5% as a “clinically meaningful difference in cancer patients with cachexia.” She added that the drug's effect on other health indicators, such as increased appetite and physical activity, “is what really gives us encouragement.”
Pfizer said it had not observed any significant side effects with the drug. Treatment-related side effects occurred in 8.9 percent of patients who took a placebo and 7.7 percent of patients who received Pfizer's drug, the company said.
The company said it is discussing plans with regulators for the drug's late-stage development and aims to begin trials in 2025 that could be used to file a regulatory application. Pfizer is also studying ponsegromab in a Phase 2 trial in patients with heart failure, who may also suffer from cachexia.
Pfizer's drug works by lowering GDF-15 levels, which Pfizer believes can increase appetite and help patients maintain and gain weight.
“Most of us have low levels of GDF-15 in our tissues when we are healthy. But in many of these chronic diseases, in this case cancer, we actually see an upregulation of GDF-15 levels,” Allerton said.
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