Biotech
Monday, September 16th, 2024 5:09 pm EDT
Key Points
- Pfizer’s experimental drug, ponsegromab, showed positive results in a phase two trial for treating cancer cachexia, a life-threatening condition that causes significant weight and muscle loss in cancer patients.
- Patients taking the drug experienced improvements in body weight, muscle mass, physical function, and quality of life, with the highest dose group seeing a 5.6% weight increase, potentially making ponsegromab the first FDA-approved treatment for cancer cachexia.
- The drug works by targeting and reducing the levels of GDF-15, a protein linked to appetite loss in cancer cachexia, and has demonstrated minimal side effects, prompting Pfizer to plan late-stage trials starting in 2025.
Pfizer has announced promising results from a mid-stage clinical trial for its experimental drug, ponsegromab, designed to treat cancer cachexia, a debilitating condition characterized by severe weight and muscle loss in cancer patients. This condition affects around 9 million people globally and is linked to poor outcomes, with 80% of cancer patients suffering from cachexia expected to die within a year. The symptoms of cachexia, which include weakness, fatigue, and a significant decline in physical function, often make cancer treatments less effective and worsen patient survival rates.
Ponsegromab, a monoclonal antibody, works by targeting and reducing levels of the protein growth differentiation factor 15 (GDF-15), a key driver of cachexia that affects appetite and energy regulation. High levels of GDF-15 are common in cancer patients suffering from cachexia, and Pfizer believes that lowering these levels can help improve appetite, muscle mass, and overall quality of life. The mid-stage phase two trial involved 187 patients with non-small cell lung cancer, pancreatic cancer, or colorectal cancer, all of whom exhibited high levels of GDF-15.
After 12 weeks, the trial’s results were encouraging. Patients who received the highest dose of ponsegromab, 400 milligrams, saw a 5.6% increase in body weight compared to those on a placebo. Lower doses of 200 milligrams and 100 milligrams resulted in weight gains of 3.5% and 2%, respectively. Pfizer highlighted that weight gain greater than 5% is considered a clinically meaningful improvement for cancer cachexia patients. Additionally, ponsegromab improved other health measures, such as appetite and physical activity levels, which are critical for cancer patients to maintain their strength and tolerate treatments better.
Importantly, Pfizer reported that the drug showed minimal side effects. In fact, treatment-related side effects were less frequent among patients taking ponsegromab (7.7%) compared to those taking a placebo (8.9%), further adding to the drug’s potential for widespread use. This positive safety profile is crucial as Pfizer advances discussions with regulatory bodies to move the drug into late-stage development, with plans to begin larger trials in 2025.
If successful, ponsegromab could become the first FDA-approved treatment for cancer cachexia, filling a significant unmet need in oncology. Beyond cancer, Pfizer is also exploring the drug’s potential in treating cachexia in other conditions like heart failure, where GDF-15 levels are also elevated.
Pfizer presented the results of this trial at the European Society for Medical Oncology 2024 Congress and published the findings in The New England Journal of Medicine. The success of ponsegromab represents a major step forward in addressing a condition that has long plagued cancer patients with limited treatment options, offering new hope for improving their quality of life and ability to fight their disease.
For the full original article on CNBC, please click here: https://www.cnbc.com/2024/09/14/pfizers-cancer-cachexia-drug-shows-positive-midstage-trial-results.html
