Pfizer’s cancer cachexia drug shows positive midstage trial results

Pfizer‘s experimental drug for a common, life-threatening condition that causes cancer patients to lose their appetite and weight showed positive ends in a midstage trial, the drugmaker said Saturday. 

Patients with the condition, called cancer cachexia, who took Pfizer’s treatment saw improvements in body weight, muscle mass, quality of life and physical function, in line with the drugmaker. The outcomes could pave the way in which for the drug, a monoclonal antibody called ponsegromab, to grow to be the primary treatment approved within the U.S. specifically for cancer cachexia. 

The condition affects about 9 million people worldwide, and 80% of cancer patients affected by it are expected to die inside one yr of diagnosis, in line with the corporate.

Patients with cancer cachexia don’t eat enough food to fulfill their body’s energy needs, causing significant fat and muscle loss and leaving them weak, fatigued and, in some cases, unable to perform every day activities. Cancer cachexia is currently defined as a lack of 5% or more body weight over the past six months in cancer patients, together with symptoms corresponding to fatigue, in line with the National Cancer Institute.

The symptoms of the condition could make cancer treatments less effective and contribute to lower survival rates, Pfizer said. 

“We’d see ponsegromab fitting into the treatment of cancer patients, really addressing that unmet need in cachexia, and thru that, improving their wellness, their ability to take care of themselves, and we might also hope their ability to tolerate more treatment,” Charlotte Allerton, Pfizer’s head of discovery and early development, told CNBC in an interview. 

Pfizer has not disclosed the estimated revenue opportunity of the drug, which could potentially be approved for various uses.

The corporate presented the info Saturday on the European Society for Medical Oncology 2024 Congress, a cancer research conference held in Barcelona, Spain. The outcomes were also published in The Recent England Journal of Medicine. 

The phase two trial followed 187 individuals 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 within the brain and has an impact on appetite, in line with Allerton. 

After 12 weeks, patients who took the very best dose of ponsegromab — 400 milligrams — saw a 5.6% increase in weight compared with those that received a placebo. Patients who took a 200-milligram or 100-milligram dose of the drug saw a roughly 3.5% and a pair of% increase in body weight, respectively, compared with the placebo group. 

Allerton said a piece group of experts defines a weight gain of greater than 5% as a “clinically meaningful difference in cancer patients with cachexia.” She added that the drug’s effect on other measures of wellness, corresponding to increased appetite and physical activity, is “really what offers us the encouragement.” 

Pfizer said it didn’t observe any significant unwanted side effects with the drug. Treatment-related unwanted side effects occurred in 8.9% of individuals taking a placebo and seven.7% of those that took Pfizer’s treatment, the corporate said. 

The corporate said it’s discussing late-stage development plans for the drug with regulators, and goals to begin studies in 2025 that might be used to file for approval. Pfizer can be studying ponsegromab in a phase two trial in patients with heart failure, who may also suffer from cachexia.

Pfizer’s drug works by reducing the degrees of GDF-15. Pfizer believes this will improve appetite and enable patients to take care of and gain weight. 

“For many of us, we’ve got low levels of GDF-15 in our tissues after we’re healthy, but we actually do see this up regulation of GDF-15 in additional of those chronic conditions, and on this case, cancer,” Allerton said.