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The first drug to hit the hormone behind cancer wasting: patients on the highest dose gained nearly 3 kg more than placebo in 12 weeks and reported better appetite and more activity.
187 patients with cachexia (weight loss at least 5%, or 2% with BMI under 20) and GDF-15 at least 1,500 pg/mL were randomised. The primary endpoint, change in body weight at 12 weeks, showed placebo-adjusted gains of 1.22 kg (100 mg), 1.92 kg (200 mg) and 2.81 kg (400 mg). At 400 mg there were also improvements in appetite and cachexia symptoms, physical activity measured by accelerometer, and lumbar skeletal muscle index, and no increase in adverse events versus placebo. Follow-on work: a phase 2/3 programme in pancreatic cancer cachexia (NCT06989437, recruiting) and planned lung cancer studies, with functional and survival endpoints. The trial is the proof of concept that cachexia is a drug-treatable disease; it did not test survival, and 12 weeks is short.
Numbers are from the trial as recorded here; see the source links in the table below. This is orientation, not medical advice: ask your team how closely the trial population matches you.
187 enrolled.
| Endpoint | Arm | n | Value | HR (95% CI) | p | Source |
|---|---|---|---|---|---|---|
| Change in body weight at 12 weeks (placebo-adjusted)primary | Ponsegromab 400 mg | - | 2.81 kg | - | - | link |
| Ponsegromab 200 mg | - | 1.92 kg | ||||
| Ponsegromab 100 mg | - | 1.22 kg | ||||
| Placebo | - | 0 kg |
Shares Combine the new anti-wasting antibody with exercise and protein, Cachexia-directed therapy (GDF-15 blockade), Cancer cachexia, Body composition (lean mass, fat mass, visceral fat).
Shares Cachexia-directed therapy (GDF-15 blockade), Cancer cachexia, Body composition (lean mass, fat mass, visceral fat), Cachexia pharmacotherapy: GDF-15 blockade, anamorelin, olanzapine.
Shares Combine the new anti-wasting antibody with exercise and protein, Cachexia-directed therapy (GDF-15 blockade), Body composition (lean mass, fat mass, visceral fat), Cancer cachexia.
Shares A Study to Learn About the Medicine Ponsegromab in Adults With Cancer of the Pancreas Which Has Spread and Caused Significant Body Weight Loss and Fatigue, Ponsegromab for the Treatment of Cancer Cachexia, Pfizer (incl. Seagen), Pancreatic ductal adenocarcinoma.
Shares Cachexia pharmacotherapy: GDF-15 blockade, anamorelin, olanzapine, Cachexia, toxicity and the limits of the patient, Supportive care and survivorship roadmap: making treatment bearable → proving it extends life → caring for tens of millions afterwards, Pfizer (incl. Seagen).
Shares Cachexia-directed therapy (GDF-15 blockade), Cancer cachexia, Cachexia, toxicity and the limits of the patient, Diet, exercise and lifestyle roadmap: causes established → interventions that disappointed → exercise proven as treatment.
Shares Cachexia-directed therapy (GDF-15 blockade), Cancer cachexia, Colorectal cancer, Non-small-cell lung cancer.
Shares Ponsegromab for the Treatment of Cancer Cachexia, Cachexia, toxicity and the limits of the patient, Supportive care and survivorship roadmap: making treatment bearable → proving it extends life → caring for tens of millions afterwards, Pfizer (incl. Seagen).