Trial Outcomes & Findings for A Study of INCMGA00012 in Metastatic Merkel Cell Carcinoma (POD1UM-201) (NCT NCT03599713)

Last Updated: 2025-08-05

Results Overview

ORR was defined as the percentage of participants with a confirmed overall response of complete response (CR) or partial response (PR), per Response Evaluation Criteria in Solid Tumors (RECIST) version 1.1 (v1.1), as determined by Independent Central Radiographic Review (ICR), at any post-Baseline visit until the first progressive disease (PD) or new anti-cancer therapy. CR: disappearance of all target and non-target lesions and no appearance of any new lesions. Any pathological lymph nodes (whether target or non-target) must have a reduction in the short axis to \<10 millimeters (mm). PR: complete disappearance or at least a 30% decrease in the sum of the diameters of target lesions, taking as a reference the baseline sum diameters, no new lesions, and no progression of non-target lesions.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

107 participants

Primary outcome timeframe

up to 26.8 months

Results posted on

2025-08-05

Participant Flow

Participants were enrolled and treated at 34 study centers in Italy, France, the United States, Poland, Canada, Switzerland, Hungary, the Czech Republic, Germany, Spain, and the United Kingdom.

Participant milestones

Participant milestones
Measure	Chemotherapy: Naïve Participants with recurrent, advanced locoregional disease or distant metastatic disease who did not receive any prior chemotherapy received retifanlimab 500 milligrams (mg), administered by intravenous (IV) infusion over 60 minutes on Day 1 of each 28-day cycle (Q4W).	Chemotherapy: Refractory Participants with disease not responding to prior chemotherapy received retifanlimab 500 mg, administered by IV infusion over 60 minutes on Day 1 Q4W.
Overall Study STARTED	101	6
Overall Study Safety Evaluable Population	101	6
Overall Study Enrolled Population	101	6
Overall Study Full Analysis Set	65	6
Overall Study COMPLETED	0	0
Overall Study NOT COMPLETED	101	6

Reasons for withdrawal

Reasons for withdrawal
Measure	Chemotherapy: Naïve Participants with recurrent, advanced locoregional disease or distant metastatic disease who did not receive any prior chemotherapy received retifanlimab 500 milligrams (mg), administered by intravenous (IV) infusion over 60 minutes on Day 1 of each 28-day cycle (Q4W).	Chemotherapy: Refractory Participants with disease not responding to prior chemotherapy received retifanlimab 500 mg, administered by IV infusion over 60 minutes on Day 1 Q4W.
Overall Study Death	38	3
Overall Study Withdrawal by Subject	17	0
Overall Study Lost to Follow-up	4	0
Overall Study Discontinued Due to End of Study	42	3

Baseline Characteristics

A Study of INCMGA00012 in Metastatic Merkel Cell Carcinoma (POD1UM-201)

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Chemotherapy: Naïve n=101 Participants Participants with recurrent, advanced locoregional disease or distant metastatic disease who did not receive any prior chemotherapy received retifanlimab 500 milligrams (mg), administered by intravenous (IV) infusion over 60 minutes on Day 1 of each 28-day cycle (Q4W).	Chemotherapy: Refractory n=6 Participants Participants with disease not responding to prior chemotherapy received retifanlimab 500 mg, administered by IV infusion over 60 minutes on Day 1 Q4W.	Total n=107 Participants Total of all reporting groups
Age, Continuous	71.1 years STANDARD_DEVIATION 10.44 • n=5 Participants	63.8 years STANDARD_DEVIATION 10.46 • n=7 Participants	70.7 years STANDARD_DEVIATION 10.52 • n=5 Participants
Sex: Female, Male Female	33 Participants n=5 Participants	1 Participants n=7 Participants	34 Participants n=5 Participants
Sex: Female, Male Male	68 Participants n=5 Participants	5 Participants n=7 Participants	73 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	1 Participants n=5 Participants	0 Participants n=7 Participants	1 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	75 Participants n=5 Participants	6 Participants n=7 Participants	81 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	25 Participants n=5 Participants	0 Participants n=7 Participants	25 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Asian	1 Participants n=5 Participants	0 Participants n=7 Participants	1 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) White	78 Participants n=5 Participants	6 Participants n=7 Participants	84 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	22 Participants n=5 Participants	0 Participants n=7 Participants	22 Participants n=5 Participants

PRIMARY outcome

Timeframe: up to 26.8 months

Population: Full Analysis Set (FAS): all participants enrolled in the study who received at least 1 dose of study drug as of 15 October 2020 (selected to allow for at least 60 chemotherapy-naïve participants to be followed for at least 6 months after first response assessment). Analysis was based on the chemotherapy-naïve subset of the FAS. Confidence intervals (CIs) were calculated based on the exact method for binomial distributions.

Outcome measures

Outcome measures
Measure	Chemotherapy: Naïve n=65 Participants Participants with recurrent, advanced locoregional disease or distant metastatic disease who did not receive any prior chemotherapy received retifanlimab 500 milligrams (mg), administered by intravenous (IV) infusion over 60 minutes on Day 1 of each 28-day cycle (Q4W).	Chemotherapy: Refractory Participants with disease not responding to prior chemotherapy received retifanlimab 500 mg, administered by IV infusion over 60 minutes on Day 1 Q4W.
Objective Response Rate (ORR)	52.3 percentage of participants Interval 39.5 to 64.9	—

SECONDARY outcome

Timeframe: up to 55.3 months

Population: Safety Evaluable Population: all enrolled participants who received at least 1 dose of study drug. Analysis was based on the chemotherapy-naïve subset of the population. Only those participants who had confirmed CR or PR prior to PD or start of new anticancer therapy were assessed. The 95% CI was calculated using the Brookmeyer and Crowley's method and Klein and Moeschberger's method with log-log transformation.

DOR was defined as the time from an initial objective response (CR or PR) per RECIST v1.1 until PD, or death due to any cause, as determined by ICR. CR: disappearance of all target and non-target lesions and no appearance of any new lesions. Any pathological lymph nodes (whether target or non-target) must have a reduction in the short axis to \<10 mm. PR: complete disappearance or at least a 30% decrease in the sum of the diameters of target lesions, taking as a reference the baseline sum diameters, no new lesions, and no progression of non-target lesions. PD: progression of a target or non-target lesion or presence of a new lesion. A Kaplan-Meier estimate (estimated median) of the distribution function is reported.

Outcome measures

Outcome measures
Measure	Chemotherapy: Naïve n=55 Participants Participants with recurrent, advanced locoregional disease or distant metastatic disease who did not receive any prior chemotherapy received retifanlimab 500 milligrams (mg), administered by intravenous (IV) infusion over 60 minutes on Day 1 of each 28-day cycle (Q4W).	Chemotherapy: Refractory Participants with disease not responding to prior chemotherapy received retifanlimab 500 mg, administered by IV infusion over 60 minutes on Day 1 Q4W.
Duration of Response (DOR)	NA months Interval 22.87 to The median and the upper limit of the confidence interval were not estimable because too few participants had disease progression or died.	—

SECONDARY outcome

Timeframe: up to 57.1 months

Population: Safety Evaluable Population. Analysis was based on the chemotherapy-naïve subset of the population. CIs were calculated based on the exact method for binomial distributions.

DCR was defined as the percentage of participants with a confirmed overall response (CR and PR) or stable disease (SD) (non-CR/non-PD) lasting at least 6 months from the start of treatment, until the first PD or new anti-cancer therapy, per RECIST v1.1 as determined by ICR. CR: disappearance of all target and non-target lesions and no appearance of any new lesions. Any pathological lymph nodes (whether target or non-target) must have a reduction in the short axis to \<10 mm. PR: complete disappearance or at least a 30% decrease in the sum of the diameters of target lesions, taking as a reference the baseline sum diameters, no new lesions, and no progression of non-target lesions. PD: progression of a target or non-target lesion or presence of a new lesion. SD: no change in target lesions to qualify for CR, PR, or PD.

Outcome measures

Outcome measures
Measure	Chemotherapy: Naïve n=101 Participants Participants with recurrent, advanced locoregional disease or distant metastatic disease who did not receive any prior chemotherapy received retifanlimab 500 milligrams (mg), administered by intravenous (IV) infusion over 60 minutes on Day 1 of each 28-day cycle (Q4W).	Chemotherapy: Refractory Participants with disease not responding to prior chemotherapy received retifanlimab 500 mg, administered by IV infusion over 60 minutes on Day 1 Q4W.
Disease Control Rate (DCR)	60.4 percentage of participants Interval 50.2 to 70.0	—

SECONDARY outcome

Timeframe: up to 57.1 months

Population: Safety Evaluable Population. Analysis was based on the chemotherapy-naïve subset of the population. Median PFS time was estimated using the Kaplan-Meier method. The CI for median PFS time was calculated using the method of Brookmeyer and Crowley.

According to RESIST v1.1, PFS was defined the time from the start of therapy until disease progression, or death due to any cause, as determined by ICR. Evaluation of target lesions: PD: ≥20% increase in the sum of diameters of target lesions, taking as reference the smallest sum on study. The sum must also demonstrate an absolute increase of at least 5 mm. (Note: the appearance of one or more new lesions is also considered PD). Evaluation of non-target lesions: PD: Unequivocal progression of existing non-target lesions. (Note: the appearance of one or more new lesions is also considered PD).

Outcome measures

Outcome measures
Measure	Chemotherapy: Naïve n=101 Participants Participants with recurrent, advanced locoregional disease or distant metastatic disease who did not receive any prior chemotherapy received retifanlimab 500 milligrams (mg), administered by intravenous (IV) infusion over 60 minutes on Day 1 of each 28-day cycle (Q4W).	Chemotherapy: Refractory Participants with disease not responding to prior chemotherapy received retifanlimab 500 mg, administered by IV infusion over 60 minutes on Day 1 Q4W.
Progression-free Survival (PFS)	16.03 months Interval 9.03 to 32.23	—

SECONDARY outcome

Timeframe: up to 60.4 months

Population: Safety Evaluable Population. Analysis was based on the chemotherapy-naïve subset of the population. Median overall survival time was estimated using the Kaplan-Meier method. CI for median overall survival time was calculated using the method of Brookmeyer and Crowley.

Overall survival was defined as the time in months between the first dose date (Day 1) and the date of death due to any cause.

Outcome measures

Outcome measures
Measure	Chemotherapy: Naïve n=101 Participants Participants with recurrent, advanced locoregional disease or distant metastatic disease who did not receive any prior chemotherapy received retifanlimab 500 milligrams (mg), administered by intravenous (IV) infusion over 60 minutes on Day 1 of each 28-day cycle (Q4W).	Chemotherapy: Refractory Participants with disease not responding to prior chemotherapy received retifanlimab 500 mg, administered by IV infusion over 60 minutes on Day 1 Q4W.
Overall Survival	NA months Interval 45.24 to The median and the upper limit of the confidence interval were not estimable because too few participants died.	—

SECONDARY outcome

Timeframe: up to 846 days (up to approximately 2.3 years)

Population: Safety Evaluable Population

An adverse event (AE) is any untoward medical occurrence associated with the use of a drug in humans, whether or not considered drug related. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease (new or exacerbated) temporally associated with the use of study treatment. A TEAE was defined as either an AE reported for the first time or a worsening of a pre-existing event after the first dose of study drug until 90 days after the last dose of study drug. An AE with onset on/after starting a new anticancer therapy was not summarized as a TEAE.

Outcome measures

Outcome measures
Measure	Chemotherapy: Naïve n=101 Participants Participants with recurrent, advanced locoregional disease or distant metastatic disease who did not receive any prior chemotherapy received retifanlimab 500 milligrams (mg), administered by intravenous (IV) infusion over 60 minutes on Day 1 of each 28-day cycle (Q4W).	Chemotherapy: Refractory n=6 Participants Participants with disease not responding to prior chemotherapy received retifanlimab 500 mg, administered by IV infusion over 60 minutes on Day 1 Q4W.
Number of Participants With Any Treatment-emergent Adverse Event (TEAE)	92 Participants	5 Participants

SECONDARY outcome

Timeframe: preinfusion, 10 minutes postinfusion (± 10 minutes), and 4 hours postinfusion (± 10 minutes) on Day 1 of Cycle 1

Population: Pharmacokinetic (PK) Evaluable Population: all participants who received at least 1 dose of study drug and have provided a Baseline and at least 1 post-dose PK sample

Cmax was defined as the maximum observed plasma concentration.

Outcome measures

Outcome measures
Measure	Chemotherapy: Naïve n=102 Participants Participants with recurrent, advanced locoregional disease or distant metastatic disease who did not receive any prior chemotherapy received retifanlimab 500 milligrams (mg), administered by intravenous (IV) infusion over 60 minutes on Day 1 of each 28-day cycle (Q4W).	Chemotherapy: Refractory Participants with disease not responding to prior chemotherapy received retifanlimab 500 mg, administered by IV infusion over 60 minutes on Day 1 Q4W.
First-dose Cmax of Retifanlimab	144 micrograms per milliliter (μg/mL) Standard Deviation 32.6	—

SECONDARY outcome

Timeframe: preinfusion, 10 minutes postinfusion (± 10 minutes), and 4 hours postinfusion (± 10 minutes) on Day 1 of Cycle 1

Population: PK Evaluable Population

Cmin was defined as the minimum observed plasma concentration over the dose interval.

Outcome measures

Outcome measures
Measure	Chemotherapy: Naïve n=102 Participants Participants with recurrent, advanced locoregional disease or distant metastatic disease who did not receive any prior chemotherapy received retifanlimab 500 milligrams (mg), administered by intravenous (IV) infusion over 60 minutes on Day 1 of each 28-day cycle (Q4W).	Chemotherapy: Refractory Participants with disease not responding to prior chemotherapy received retifanlimab 500 mg, administered by IV infusion over 60 minutes on Day 1 Q4W.
First-dose Cmin of Retifanlimab	20.5 μg/mL Standard Deviation 7.23	—

SECONDARY outcome

Timeframe: preinfusion, 10 minutes postinfusion (± 10 minutes), and 4 hours postinfusion (± 10 minutes) on Day 1 of Cycle 1

Population: PK Evaluable Population

AUC0-t was defined as the area under the plasma concentration-time curve from time zero to time t.

Outcome measures

Outcome measures
Measure	Chemotherapy: Naïve n=102 Participants Participants with recurrent, advanced locoregional disease or distant metastatic disease who did not receive any prior chemotherapy received retifanlimab 500 milligrams (mg), administered by intravenous (IV) infusion over 60 minutes on Day 1 of each 28-day cycle (Q4W).	Chemotherapy: Refractory Participants with disease not responding to prior chemotherapy received retifanlimab 500 mg, administered by IV infusion over 60 minutes on Day 1 Q4W.
First-dose AUC0-t of Retifanlimab	1770 day*mg/L Standard Deviation 549	—

Adverse Events

Chemotherapy: Naïve

Serious events: 26 serious events

Other events: 83 other events

Deaths: 39 deaths

Chemotherapy: Refractory

Serious events: 2 serious events

Other events: 5 other events

Deaths: 3 deaths

Total

Serious events: 28 serious events

Other events: 88 other events

Deaths: 42 deaths

Serious adverse events

Other adverse events

Additional Information

Study Director

Incyte Corporation

Phone: 1-855-463-3463

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee Following the first publication, the Institution and/or Principal Investigator may publish data or results from the Study, provided, however, that the Institution and/or Principal Investigator submits the proposed publication to the Sponsor for review at least sixty (60) days prior to the date of the proposed publication. Sponsor may remove from the proposed publication any information that is considered confidential and/or proprietary other than Study data and results.
Publication restrictions are in place

Restriction type: OTHER