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Ponsegromab phase 2 in cancer cachexia

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.

Setting
NSCLC, pancreatic or colorectal cancer with cachexia and elevated serum GDF-15: ponsegromab 100, 200 or 400 mg subcutaneously every 4 weeks vs placebo for 12 weeks
Phase
Phase 2
Sponsor
Pfizer
Registry
Headline result
Placebo-adjusted weight gain 2.81 kg at 400 mg over 12 weeks; improved appetite and activity.
Reported
2024
Enrolled
187
Replication
Phase 2/3 in pancreatic cancer cachexia underway (NCT06989437).

Outcomes

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In plain words
What these results mean for people, not percentages
187 people took part
Change in body weight at 12 weeks (placebo-adjusted)primaryother endpoint
  • Ponsegromab 400 mg: 2.8 kg; Ponsegromab 200 mg: 1.9 kg; Ponsegromab 100 mg: 1.2 kg; Placebo: 0 kg.
  • This endpoint is not one of the standard survival or response measures; read it alongside the trial's primary result.
  • These results apply to the people the trial enrolled: NSCLC, pancreatic or colorectal cancer with cachexia and elevated serum GDF-15: ponsegromab 100, 200 or 400 mg subcutaneously every 4 weeks vs placebo for 12 weeks. People in a different situation may not see the same effect.

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 participants enrolled.

Change in body weight at 12 weeks (placebo-adjusted)primary
Ponsegromab 400 mg
2.81 mo
Ponsegromab 200 mg
1.92 mo
Ponsegromab 100 mg
1.22 mo
Placebo
0 mo
Source
EndpointArmnValueHR (95% CI)pSource
Change in body weight at 12 weeks (placebo-adjusted)primaryPonsegromab 400 mg2.81 kglink
Ponsegromab 200 mg1.92 kg
Ponsegromab 100 mg1.22 kg
Placebo0 kg
Replication
Phase 2/3 in pancreatic cancer cachexia underway (NCT06989437).

Connected

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