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Pharmaceutical giant Pfizer’s experimental drug for a common, life-threatening condition that causes weight and appetite loss in cancer patients has shown positive results in a trial. The drugmaker announced that those who suffer from cancer cachexia – a complex condition that results in muscle and fat loss and can be fatal, took Pfizer’s treatment and saw improvements in body weight, muscle mass, quality of life, and physical function.
The results could now pave the way for the drug – a monoclonal antibody known as ponsegromab, to become the first treatment approved in the US specifically for cancer cachexia.
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According to experts, cancer cachexia is a condition that affects about 9 million people worldwide, with over 80 per cent of cancer patients suffering from it. It happens when cancer affects your appetite making you lose skeletal muscle and fat. In cancer cachexia, a patient would not eat enough food to meet their body's energy needs. Most of the patients are expected to die within a year of diagnosis.
Pfizer says the symptoms of the condition can make cancer treatments less effective and contribute to lower survival rates.
“We would see ponsegromab fitting into the treatment of cancer patients, really addressing that unmet need in cachexia, and through that, improving their wellness, their ability to care for themselves, and we would also hope their ability to tolerate more treatment,” Charlotte Allerton, Pfizer’s head of discovery and early development, told CNBC in an interview.
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Pfizer has not disclosed the estimated revenue opportunity of the drug, which could potentially be approved for different uses.
The study
The phase two trial, results of which were presented at the research conference in Barcelona, followed nearly 200 people with non-small cell lung cancer, pancreatic cancer, and colorectal cancer along with high levels of a key driver of cachexia-growth differentiation factor 15, or GDF-15. It is a protein that binds to a certain receptor in the brain and has an impact on appetite.
After three months, patients who took the highest dose of ponsegromab – which is around 400 milligrams, saw a 5.6 per cent increase in weight compared with those who received a placebo. Patients who took a 200-milligram or 100-milligram dose of the drug saw a roughly 3.5 per cent and 2 per cent increase in body weight, compared with the placebo group.
The drug’s effect on other measures of wellness, such as increased appetite and physical activity, is “really what offers us encouragement,” said Allerton.
No major side effects reported
Pfizer said it has not observed any significant side effects with the drug. However, a few treatment-related ones did occur in 8.9 per cent of people taking a placebo and 7.7 per cent of those who took Pfizer’s treatment.
The company says it is discussing late-stage development plans for the drug with regulators and aims to start the second part of the study next year, after which they would file for approval. According to Pfizer, they are also studying ponsegromab in a phase two trial in patients with heart failure, who suffer from cachexia.