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ponsegromab

Phase 2

Cachexia | Small molecule | Oncology |Pfizer, Inc.|Last Updated: Aug 24, 2026

Success Probability

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Trial Design

RandomizedDouble-BlindPLACEBO_CONTROLLEDDMC
Total Trials1
Total Enrollment982

FDA Designations

No designations recorded

Clinical trial landscape

ponsegromab · 4 trials · 9 indications

Phase 2 2Phase 1 2
NCT06989437A Study to Learn About the Medicine Ponsegromab in Adults With Cancer of the Pancreas Which Has Spread and Caused Significant Body Weight Loss and FatigueCachexia
RECRUITING982 Analytics
NCT05546476Study of the Efficacy and Safety of Ponsegromab in Patients With Cancer, Cachexia and Elevated GDF-15Non-small Cell Lung Cancer
COMPLETED187 Analytics
PHASE2RECRUITING
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
CachexiaUnlock trial analytics
PHASE2COMPLETED
Study of the Efficacy and Safety of Ponsegromab in Patients With Cancer, Cachexia and Elevated GDF-15
Non-small Cell Lung CancerUnlock trial analytics

Study Endpoints

Primary Endpoints

Percent change from baseline in body weight for ponsegromab compared to placebo
Baseline, Week 12
Change from baseline in Functional Assessment of Anorexia/Cachexia Therapy 5-item Anorexia Symptom Scale scores
Baseline, Week 12

Scale consists of five items, each rated 0-4. Total score ranges from 0 (minimum) to 20 (maximum). Higher scores are associated with a better outcome.

Part A: Change From Baseline in Body Weight at Week 12
Baseline, Week 12

Body weight was measured in kilograms using a calibrated weighing scale. Baseline was defined as the last average of the duplicate measurements prior to, or on Day 1. The average of the duplicate body weights collected at assessment time was considered. The posterior medians and 90 percent (%) credible intervals (5th and 95th percentiles of the relevant posterior distribution) were reported for each randomized dose (including placebo). 4-Parameter maximal effect (E max) model: change from baseline = E 0 + (E max \* dose\^Hill) / (ED 50\^Hill + dose\^Hill), where E0 is the placebo effect, E max is the maximum effect, ED 50 is the dose producing 50% of the maximum effect, and Hill is the slope parameter. Model utilized a Bayesian methodology with a robustified, informative meta-analytic predictive prior for the placebo change from baseline at week 12.

Percent change from baseline in Lumbar Skeletal Muscle Index
Baseline, Week 12

Assessed by computer tomography (CT) (or MRI \[magnetic resonance imaging\])

Area Under the Serum Concentration-Time Profile From Time 0 Extrapolated to Infinite Time (AUCinf) of Unbound and Total Ponsegromab
Pre-dose, 12, 24, 48, 72, 120, 168 hours after dose on Day 1, Days 10, 15, 29, 50, 71, 93, 106, and 127

AUCinf was defined as area under the serum concentration-time profile from time 0 extrapolated to infinite time.

Area Under the Serum Concentration-Time Profile From Time 0 to the Time of the Last Quantifiable Concentration (AUClast) of Unbound and Total Ponsegromab
Pre-dose, 12, 24, 48, 72, 120, 168 hours after dose on Day 1, Days 10, 15, 29, 50, 71, 93, 106, and 127

AUClast was defined as area under the serum concentration-time profile from time 0 to the time of the last quantifiable concentration.

Maximum Observed Serum Concentration (Cmax) of Unbound and Total Ponsegromab
Pre-dose, 12, 24, 48, 72, 120, 168 hours after dose on Day 1, Days 10, 15, 29, 50, 71, 93, 106, and 127

Cmax was defined as maximum observed concentration. Cmax was observed directly from data.

Time for Cmax (Tmax) of Unbound and Total Ponsegromab
Pre-dose, 12, 24, 48, 72, 120, 168 hours after dose on Day 1, Days 10, 15, 29, 50, 71, 93, 106, and 127

Tmax was defined as time for Cmax. Tmax was observed directly from data as time of first occurrence.

Terminal Half Life (t1/2) of Unbound and Total Ponsegromab
Pre-dose, 12, 24, 48, 72, 120, 168 hours after dose on Day 1, Days 10, 15, 29, 50, 71, 93, 106, and 127

t1/2 was defined as terminal half life. t1/2 was calculated as Loge(2)/kel, where kel was the terminal phase rate constant calculated by a linear regression of the log linear concentration time curve.

Secondary Endpoints

Change from baseline in non-sedentary physical activity time
Baseline, Week 12
Overall survival
Randomization through completion of Phase 3 of the study, an average of 1 year
Change from baseline in body weight (kg)
Baseline, Week 12 and up to Week 52
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Study Design & Arms

AllocationRANDOMIZED
MaskingQUADRUPLE
ModelPARALLEL
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
Double-Blind ponsegromab Treatment lower doseEXPERIMENTALponsegromab 200 mg subcutaneous injection every 4 weeks
Double-Blind ponsegromab Treatment higher doseEXPERIMENTALponsegromab 400 mg subcutaneous injection every 4 weeks
Double-Blind Placebo TreatmentPLACEBO_COMPARATORMatch placebo subcutaneous injection every 4 weeks
Double-Blind ponsegromab Treatment low dose followed by Open Label ponsegromab TreatmentEXPERIMENTALponsegromab low dose subcutaneous injection every 4 weeks
Double-Blind Placebo Treatment followed by Open-Label ponsegromab TreatmentPLACEBO_COMPARATORMatch placebo subcutaneous injection every 4 weeks
Double-Blind ponsegromab Treatment medium dose followed by Open Label ponsegromab TreatmentEXPERIMENTALponsegromab medium dose subcutaneous injection every 4 weeks
Double-Blind ponsegromab Treatment high dose followed by Open Label ponsegromab TreatmentEXPERIMENTALponsegromab high dose subcutaneous injection every 4 weeks
Double-Blind ponsegromab Treatment doseEXPERIMENTALPonsegromab 400 mg subcutaneous injection every 4 weeks
Ponsegromab low doseEXPERIMENTAL -
Ponsegromab high doseEXPERIMENTAL -

Interventions

NameTypeDescription
ponsegromabDRUGDouble-Blind ponsegromab Treatment
placeboDRUGDouble-Blind placebo Treatment
Placebo for ponsegromabDRUGDouble-Blind placebo Treatment followed by Open Label ponsegromab Treatment
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Eligibility Criteria

Age Range18 Years to N/A
SexALL
Healthy VolunteersNo
Study Sites220

Key inclusion Criteria: * Signed Informed Consent Document * Documented active diagnosis of metastatic pancreatic ductal adenocarcinoma * Cachexia defined by Fearon criteria of weight loss * Completed 1 x 28-day cycle of first-line systemic nab-paclitaxel and gemcitabine chemotherapy or 2 x 14-day ...

Countries:United StatesAustraliaBelgiumBrazilBulgariaCanadaChinaFranceGermanyIndiaIsraelItalyJapanMexicoPolandPuerto RicoSlovakiaSouth KoreaSpainTaiwanUnited KingdomHungary
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Recent Changes (Last 90 Days)

LOWAug 24, 2026NCT06989437lastUpdatePostDate: changed
LOWAug 24, 2026NCT06989437lastUpdatePostDate: changed
LOWJul 9, 2026NCT06989437lastUpdatePostDate: changed
LOWJul 9, 2026NCT06989437lastUpdatePostDate: changed
MEDIUMJul 4, 2026NCT05546476TRIAL_REMOVED: changed
MEDIUMJul 4, 2026NCT05546476TRIAL_REMOVED: changed
MEDIUMJul 4, 2026NCT05546476TRIAL_REMOVED: changed
LOWJul 2, 2026NCT06989437lastUpdatePostDate: changed
LOWJul 2, 2026NCT06989437lastUpdatePostDate: changed
LOWJul 2, 2026NCT06989437lastUpdatePostDate: changed
LOWJul 2, 2026NCT06989437lastUpdatePostDate: changed
LOWJun 23, 2026NCT07663630NEW_TRIAL: changed
LOWJun 23, 2026NCT06989437lastUpdatePostDate: changed
LOWJun 23, 2026NCT07663630NEW_TRIAL: changed
LOWJun 23, 2026NCT06989437lastUpdatePostDate: changed
LOWJun 10, 2026NCT06989437lastUpdatePostDate: changed
LOWJun 10, 2026NCT06989437lastUpdatePostDate: changed

Frequently asked questions about ponsegromab

What is Ponsegromab used for?

Ponsegromab is an investigational antibody being studied for cachexia, a condition of severe weight loss and muscle wasting, in patients with cancer. It is also being evaluated in non-small cell lung cancer (NSCLC) and metastatic pancreatic ductal adenocarcinoma, where it targets cancer-related weight loss and fatigue.

Who is developing Ponsegromab?

Ponsegromab is being developed by Pfizer, Inc., a biopharmaceutical company traded on the New York Stock Exchange under the ticker symbol PFE. The company is conducting clinical trials to evaluate the drug's safety and efficacy in cancer patients with cachexia.

What phase is Ponsegromab in?

Ponsegromab is in clinical development, with trials listed as Phase 1 and Phase 2. It is an investigational drug and has not been approved by regulatory authorities. Studies are ongoing or completed to assess its effects in healthy volunteers and in patients with cancer-related cachexia.

What clinical trials is Ponsegromab in?

Ponsegromab has been studied in several clinical trials. NCT05546476 evaluated its efficacy and safety in cancer patients with cachexia and elevated GDF-15. NCT05685264 examined how the drug is processed in healthy Chinese adults. NCT06989437 is recruiting patients with metastatic pancreatic cancer and weight loss, and NCT07663630 is planned for lung cancer-associated weight loss.

Is Ponsegromab the same as GDF-15 targeting therapy?

Ponsegromab is an antibody that targets GDF-15, a protein associated with cachexia. Clinical trials have specifically enrolled patients with elevated GDF-15 levels, indicating that the drug's mechanism is linked to this biomarker. However, it is not described as being the same as any other named therapy.

What is the enrollment status of Ponsegromab trials?

Ponsegromab trials have enrolled a total of 982 participants across studies. One trial, NCT05546476, is completed with 187 patients. NCT05685264 is also completed with 18 healthy volunteers. NCT06989437 is currently recruiting, and NCT07663630 is not yet recruiting, with planned enrollment of 80 patients.