ESMO: Pfizer drug helps cancer patients gain weight

Pfizer's anti-GDF-15 antibody ponsegromab was able to achieve weight gain in cancer patients with cachexia – a life-threatening metabolic syndrome caused by the underlying disease with no approved drug treatments.

Mid-stage data reported at the ESMO congress in Barcelona showed that ponsegromab was able to increase weight by 5.6% at the most effective dose, above the 5% threshold deemed clinically meaningful, and Pfizer is now thinking about starting a phase 3 programme for the drug next year.

Cachexia is a complicated metabolic disorder that results in loss of muscle and fat in the body, and is often accompanied by other changes like insulin resistance and anorexia. It seems to have an underlying inflammatory component, and can drastically reduce life expectancy if patients stop responding to treatment, which currently relies on nutritional support, appetite stimulants, anti-inflammatories, and physical rehabilitation.

Pfizer shared the results of its phase 2 trial of ponsegromab in patients at ESMO at the same time as the data was published in the New England Journal of Medicine. It involved 187 patients with cachexia associated with lung, pancreatic, or colorectal cancer.

After 12 weeks of treatment, patients on a 100mg dose of ponsegromab saw a 2.2% weight gain, rising to 3.48% with a 200mg dose and 4.6% with 400mg, with accompanying improvements in appetite and cachexia symptoms, physical activity, and muscle mass at the highest dose.

The drug was generally well-tolerated, with adverse events of any kind reported in 70% of the patients in the ponsegromab group and in 80% of those in the placebo group.

Pfizer has previously reported data showing that ponsegromab was able to reduce circulating levels of CDF-15 in the blood, which are known to be elevated in cachexia.

"These results provide strong evidence that we have unlocked a mechanism to interrupt a critical driver of cachexia, GDF-15, which has the potential to impact patients with cancer cachexia and other life-threatening conditions," said Charlotte Allerton, Pfizer's head of discovery and early development.

Ponsegromab is also being tested in a phase 2 study in patients with heart failure and elevated serum GDF-15 concentrations, said the company.

Cachexia can also occur with chronic kidney disease, HIV, and multiple sclerosis. It affects about 9 million people worldwide and, according to Pfizer, 80% of cancer patients who develop the condition die within one year of diagnosis and it is responsible for around a third of all cancer deaths.

Photo by Ries Bosch on Unsplash