Retifanlimab Evidence: Trial Results and Peer-Reviewed Publications

Hipa.ai Research · Source: PubMed & ClinicalTrials.gov / AACT · Last updated:

The clinical evidence base for Retifanlimab comprises 22 peer-reviewed publications across 15 journals, 4 pivotal-trial primary-outcome rows reported to ClinicalTrials.gov, spanning indications including Carcinoma, Merkel Cell, Breast Neoplasms, Anus Neoplasms, and Carcinoma, Non-Small-Cell Lung. Most recent publication: Discovery of Dual A2A/A2B Adenosine Receptor Antagonist and Clinical Candidate INCB106385., J Med Chem, 2026.

Top peer-reviewed publications

Curated set of pivotal-trial result papers and recent publications in high-tier journals.

  1. A phase II study of retifanlimab (INCMGA00012) in patients with squamous carcinoma of the anal canal who have progressed following platinum-based chemotherapy (POD1UM-202).
    Rao S, Anandappa G, Capdevila J, et al. · ESMO Open · 2022
    PubMed: PMID 35816951 · NCT03597295
  2. FIGHT-101, a first-in-human study of potent and selective FGFR 1-3 inhibitor pemigatinib in pan-cancer patients with FGF/FGFR alterations and advanced malignancies.
    Subbiah V, Iannotti NO, Gutierrez M, et al. · Ann Oncol · 2022
    PubMed: PMID 35176457 · NCT02393248 · Breast Neoplasms
  3. Retifanlimab with carboplatin and paclitaxel for locally recurrent or metastatic squamous cell carcinoma of the anal canal (POD1UM-303/InterAACT-2): a global, phase 3 randomised controlled trial.
    Rao S, Samalin-Scalzi E, Evesque L, et al. · Lancet · 2025
    PubMed: PMID 40517007 · NCT04472429 · Anus Neoplasms
  4. Discovery of Dual A2A/A2B Adenosine Receptor Antagonist and Clinical Candidate INCB106385.
    Qi C, McCammant MS, Li Y, et al. · J Med Chem · 2026
    PubMed: PMID 42201333 · NCT04989387 · Squamous Cell Carcinoma of Head and Neck
  5. Brigatinib in NF2-Related Schwannomatosis with Progressive Tumors.
    Plotkin SR, Yohay KH, Nghiemphu PL, et al. · N Engl J Med · 2024
    PubMed: PMID 38904277 · NCT04374305 (INTUITT-NF2) · Ependymoma
  6. Retifanlimab versus placebo in combination with platinum-based chemotherapy in patients with first-line non-squamous or squamous metastatic non-small-cell lung cancer (POD1UM-304): a phase 3, multiregional, placebo-controlled, double-blind, randomised study.
    Lu S, Vynnychenko O, Kulyaba Y, et al. · Lancet Respir Med · 2025
    PubMed: PMID 40983066 · NCT04205812 (POD1UM-304) · Carcinoma, Non-Small-Cell Lung
  7. Phase II study of retifanlimab in patients with recurrent locally advanced or metastatic Merkel cell carcinoma (POD1UM-201).
    Grignani G, Rutkowski P, Lebbé C, et al. · J Immunother Cancer · 2025
    PubMed: PMID 40796223 · NCT03599713 · Carcinoma, Merkel Cell
  8. Next-Generation Immune Checkpoints and Tumor Microenvironment Modulation in Cancer Immunotherapy.
    Bautista J, Echeverría CE, Maldonado-Noboa I, et al. · J Immunol Res · 2026
    PubMed: PMID 41811823 · NCT04082364 (MAHOGANY) · Stomach Neoplasms

Primary-outcome results across pivotal trials

Per-arm reported values from Phase 2/3 and Phase 3 trials with results posted to ClinicalTrials.gov.

TrialIndicationPrimary endpointArmValue
NCT04082364
MAHOGANY
Stomach NeoplasmsNumber of Participants With Adverse Events of Margetuximab Plus Retifanlimab in Cohort A, as Assessed by CTCAE v5.0
Throughout the study, an average of 11 months.
Chemotherapy-free Arm47 Participants
Chemotherapy-free Arm38 Participants
Chemotherapy-free Arm25 Participants
Chemotherapy-free Arm12 Participants
Chemotherapy-free Arm8 Participants
NCT04082364
MAHOGANY
Stomach NeoplasmsObjective Response Rate (ORR) for Non-microsatellite Instability-high (Non-MSI-H) Participants (Cohort A) Using Investigator-assessed Radiology Reviews
Throughout the study, an average of 11 months.
Chemotherapy-free Arm52.1 percentage of participants with CR or PR
NCT04205812
POD1UM-304
Carcinoma, Non-Small-Cell LungOverall Survival
up to 39.1 months
Placebo + Chemotherapy13.4 months
Retifanlimab + Chemotherapy18.1 months
NCT04472429Anus NeoplasmsProgression-free Survival (PFS)
up to 33.9 months
Placebo + Chemotherapy7.4 months
Retifanlimab 500 mg Q4W + Chemotherapy9.3 months

Publications by year

2005–2026: 22 publications.

2005
1
2008
1
2011
1
2012
1
2017
1
2018
1
2020
1
2021
3
2022
6
2024
1
2025
3
2026
2

Publications by indication

Carcinoma, Merkel Cell (10)

  • Phase II study of retifanlimab in patients with recurrent locally advanced or metastatic Merkel cell carcinoma (POD1UM-201).
    J Immunother Cancer · 2025 · PMID 40796223 · NCT03599713
  • Neoadjuvant Nivolumab for Patients With Resectable Merkel Cell Carcinoma in the CheckMate 358 Trial.
    J Clin Oncol · 2021 · PMID 32324435 · NCT05594290
  • Avelumab in patients with previously treated metastatic Merkel cell carcinoma: long-term data and biomarker analyses from the single-arm phase 2 JAVELIN Merkel 200 trial.
    J Immunother Cancer · 2021 · PMID 32414862 · NCT05594290
  • Neoadjuvant immunotherapy leads to pathological responses in MMR-proficient and MMR-deficient early-stage colon cancers.
    Nat Med · 2020 · PMID 32251400 · NCT05594290
  • Analysis of Prognostic Factors from 9387 Merkel Cell Carcinoma Cases Forms the Basis for the New 8th Edition AJCC Staging System.
    Ann Surg Oncol · 2018 · PMID 27198511 · NCT05594290

Breast Neoplasms (2)

  • FIGHT-101, a first-in-human study of potent and selective FGFR 1-3 inhibitor pemigatinib in pan-cancer patients with FGF/FGFR alterations and advanced malignancies.
    Ann Oncol · 2022 · PMID 35176457 · NCT02393248
  • Evaluation of the clinical cardiac safety of pemigatinib, a fibroblast growth factor receptor inhibitor, in participants with advanced malignancies.
    Pharmacol Res Perspect · 2022 · PMID 34951522 · NCT02393248

Anus Neoplasms (2)

  • Retifanlimab with carboplatin and paclitaxel for locally recurrent or metastatic squamous cell carcinoma of the anal canal (POD1UM-303/InterAACT-2): a global, phase 3 randomised controlled trial.
    Lancet · 2025 · PMID 40517007 · NCT04472429
  • POD1UM-303/InterAACT 2: A phase III, global, randomized, double-blind study of retifanlimab or placebo plus carboplatin-paclitaxel in patients with locally advanced or metastatic squamous cell anal carcinoma.
    Front Oncol · 2022 · PMID 36091159 · NCT04472429

Carcinoma, Non-Small-Cell Lung (2)

  • Retifanlimab versus placebo in combination with platinum-based chemotherapy in patients with first-line non-squamous or squamous metastatic non-small-cell lung cancer (POD1UM-304): a phase 3, multiregional, placebo-controlled, double-blind, randomised study.
    Lancet Respir Med · 2025 · PMID 40983066 · NCT04205812
  • Dual checkpoint targeting of B7-H3 and PD-1 with enoblituzumab and pembrolizumab in advanced solid tumors: interim results from a multicenter phase I/II trial.
    J Immunother Cancer · 2022 · PMID 35414591 · NCT02475213

Stomach Neoplasms (2)

  • Next-Generation Immune Checkpoints and Tumor Microenvironment Modulation in Cancer Immunotherapy.
    J Immunol Res · 2026 · PMID 41811823 · NCT04082364
  • MAHOGANY: margetuximab combination in HER2+ unresectable/metastatic gastric/gastroesophageal junction adenocarcinoma.
    Future Oncol · 2021 · PMID 33263418 · NCT04082364

Squamous Cell Carcinoma of Head and Neck (1)

  • Discovery of Dual A2A/A2B Adenosine Receptor Antagonist and Clinical Candidate INCB106385.
    J Med Chem · 2026 · PMID 42201333 · NCT04989387

Publications by journal

Trial-results highlights

In studies evaluating retifanlimab for stomach neoplasms, the MAHOGANY trial (NCT04082364) reported on participants receiving margetuximab plus retifanlimab in Cohort A (chemotherapy-free arm). The number of participants with adverse events, as assessed by CTCAE v5.0 over an average of 11 months, included:

  • 47 participants
  • 38 participants
  • 25 participants
  • 12 participants
  • 8 participants
Also in NCT04082364, the objective response rate for non-microsatellite instability-high participants (Cohort A) using investigator-assessed radiology reviews, throughout the study (an average of 11 months), in the chemotherapy-free arm was 52.1 percentage of participants with complete response or partial response.

For other indications, retifanlimab was also studied in non-small-cell lung carcinoma and anus neoplasms. In the POD1UM-304 trial (NCT04205812) for carcinoma, non-small-cell lung, overall survival was assessed for up to 39.1 months. The median overall survival was 13.4 months in the placebo plus chemotherapy arm and 18.1 months in the retifanlimab plus chemotherapy arm. For anus neoplasms, a separate study (NCT04472429) assessed progression-free survival for up to 33.9 months. The median progression-free survival was 7.4 months in the placebo plus chemotherapy arm and 9.3 months in the retifanlimab 500 mg Q4W plus chemotherapy arm.

All values presented are sourced from primary registry reporting. Individual clinical papers should be consulted for clinical decisions.

All Retifanlimab publications (22)

2026 (2 papers)

  1. Discovery of Dual A2A/A2B Adenosine Receptor Antagonist and Clinical Candidate INCB106385.
    Qi C, McCammant MS, Li Y, et al. · J Med Chem · 2026 · Derived
    PubMed: PMID 42201333 · NCT04989387 · Squamous Cell Carcinoma of Head and Neck
  2. Next-Generation Immune Checkpoints and Tumor Microenvironment Modulation in Cancer Immunotherapy.
    Bautista J, Echeverría CE, Maldonado-Noboa I, et al. · J Immunol Res · 2026 · Derived
    PubMed: PMID 41811823 · NCT04082364 (MAHOGANY) · Stomach Neoplasms

2025 (3 papers)

  1. Retifanlimab with carboplatin and paclitaxel for locally recurrent or metastatic squamous cell carcinoma of the anal canal (POD1UM-303/InterAACT-2): a global, phase 3 randomised controlled trial.
    Rao S, Samalin-Scalzi E, Evesque L, et al. · Lancet · 2025 · Derived
    PubMed: PMID 40517007 · NCT04472429 · Anus Neoplasms
  2. Retifanlimab versus placebo in combination with platinum-based chemotherapy in patients with first-line non-squamous or squamous metastatic non-small-cell lung cancer (POD1UM-304): a phase 3, multiregional, placebo-controlled, double-blind, randomised study.
    Lu S, Vynnychenko O, Kulyaba Y, et al. · Lancet Respir Med · 2025 · Derived
    PubMed: PMID 40983066 · NCT04205812 (POD1UM-304) · Carcinoma, Non-Small-Cell Lung
  3. Phase II study of retifanlimab in patients with recurrent locally advanced or metastatic Merkel cell carcinoma (POD1UM-201).
    Grignani G, Rutkowski P, Lebbé C, et al. · J Immunother Cancer · 2025 · Derived
    PubMed: PMID 40796223 · NCT03599713 · Carcinoma, Merkel Cell

2024 (1 paper)

  1. Brigatinib in NF2-Related Schwannomatosis with Progressive Tumors.
    Plotkin SR, Yohay KH, Nghiemphu PL, et al. · N Engl J Med · 2024 · Derived
    PubMed: PMID 38904277 · NCT04374305 (INTUITT-NF2) · Ependymoma

2022 (6 papers)

  1. A phase II study of retifanlimab (INCMGA00012) in patients with squamous carcinoma of the anal canal who have progressed following platinum-based chemotherapy (POD1UM-202).
    Rao S, Anandappa G, Capdevila J, et al. · ESMO Open · 2022 · Trial result
    PubMed: PMID 35816951 · NCT03597295
  2. FIGHT-101, a first-in-human study of potent and selective FGFR 1-3 inhibitor pemigatinib in pan-cancer patients with FGF/FGFR alterations and advanced malignancies.
    Subbiah V, Iannotti NO, Gutierrez M, et al. · Ann Oncol · 2022 · Derived
    PubMed: PMID 35176457 · NCT02393248 · Breast Neoplasms
  3. Elraglusib (9-ING-41), a selective small-molecule inhibitor of glycogen synthase kinase-3 beta, reduces expression of immune checkpoint molecules PD-1, TIGIT and LAG-3 and enhances CD8+ T cell cytolytic killing of melanoma cells.
    Shaw G, Cavalcante L, Giles FJ, et al. · J Hematol Oncol · 2022 · Derived
    PubMed: PMID 36104795 · NCT05239182 (RiLEY)
  4. POD1UM-303/InterAACT 2: A phase III, global, randomized, double-blind study of retifanlimab or placebo plus carboplatin-paclitaxel in patients with locally advanced or metastatic squamous cell anal carcinoma.
    Rao S, Jones M, Bowman J, et al. · Front Oncol · 2022 · Derived
    PubMed: PMID 36091159 · NCT04472429 · Anus Neoplasms
  5. Evaluation of the clinical cardiac safety of pemigatinib, a fibroblast growth factor receptor inhibitor, in participants with advanced malignancies.
    Gong X, Ji T, Liu X, et al. · Pharmacol Res Perspect · 2022 · Derived
    PubMed: PMID 34951522 · NCT02393248 · Breast Neoplasms
  6. Dual checkpoint targeting of B7-H3 and PD-1 with enoblituzumab and pembrolizumab in advanced solid tumors: interim results from a multicenter phase I/II trial.
    Aggarwal C, Prawira A, Antonia S, et al. · J Immunother Cancer · 2022 · Derived
    PubMed: PMID 35414591 · NCT02475213 · Carcinoma, Non-Small-Cell Lung

2021 (3 papers)

  1. MAHOGANY: margetuximab combination in HER2+ unresectable/metastatic gastric/gastroesophageal junction adenocarcinoma.
    Catenacci DV, Rosales M, Chung HC, et al. · Future Oncol · 2021 · Derived
    PubMed: PMID 33263418 · NCT04082364 (MAHOGANY) · Stomach Neoplasms
  2. Neoadjuvant Nivolumab for Patients With Resectable Merkel Cell Carcinoma in the CheckMate 358 Trial.
    Topalian SL, Bhatia S, Amin A, et al. · J Clin Oncol · 2021 · Background
    PubMed: PMID 32324435 · NCT05594290 (MERCURY) · Carcinoma, Merkel Cell
  3. Avelumab in patients with previously treated metastatic Merkel cell carcinoma: long-term data and biomarker analyses from the single-arm phase 2 JAVELIN Merkel 200 trial.
    D'Angelo SP, Bhatia S, Brohl AS, et al. · J Immunother Cancer · 2021 · Background
    PubMed: PMID 32414862 · NCT05594290 (MERCURY) · Carcinoma, Merkel Cell

2020 (1 paper)

  1. Neoadjuvant immunotherapy leads to pathological responses in MMR-proficient and MMR-deficient early-stage colon cancers.
    Chalabi M, Fanchi LF, Dijkstra KK, et al. · Nat Med · 2020 · Background
    PubMed: PMID 32251400 · NCT05594290 (MERCURY) · Carcinoma, Merkel Cell

2018 (1 paper)

  1. Analysis of Prognostic Factors from 9387 Merkel Cell Carcinoma Cases Forms the Basis for the New 8th Edition AJCC Staging System.
    Harms KL, Healy MA, Nghiem P, et al. · Ann Surg Oncol · 2018 · Background
    PubMed: PMID 27198511 · NCT05594290 (MERCURY) · Carcinoma, Merkel Cell

2017 (1 paper)

  1. Prospects for combining targeted and conventional cancer therapy with immunotherapy.
    Gotwals P, Cameron S, Cipolletta D, et al. · Nat Rev Cancer · 2017 · Background
    PubMed: PMID 28338065 · NCT05594290 (MERCURY) · Carcinoma, Merkel Cell

2012 (1 paper)

  1. Recurrence after complete resection and selective use of adjuvant therapy for stage I through III Merkel cell carcinoma.
    Fields RC, Busam KJ, Chou JF, et al. · Cancer · 2012 · Background
    PubMed: PMID 22072529 · NCT05594290 (MERCURY) · Carcinoma, Merkel Cell

2011 (1 paper)

  1. Merkel cell carcinoma.
    Becker JC · Ann Oncol · 2011 · Background
    PubMed: PMID 20943647 · NCT05594290 (MERCURY) · Carcinoma, Merkel Cell

2008 (1 paper)

  1. Clinical characteristics of Merkel cell carcinoma at diagnosis in 195 patients: the AEIOU features.
    Heath M, Jaimes N, Lemos B, et al. · J Am Acad Dermatol · 2008 · Background
    PubMed: PMID 18280333 · NCT05594290 (MERCURY) · Carcinoma, Merkel Cell

2005 (1 paper)

  1. Merkel cell carcinoma: changing incidence trends.
    Hodgson NC · J Surg Oncol · 2005 · Background
    PubMed: PMID 15611998 · NCT05594290 (MERCURY) · Carcinoma, Merkel Cell

Sources and methodology

This page summarizes published evidence for general reference and does not constitute medical advice. For clinical decisions, consult the linked primary publications and your healthcare provider. Data sourced from PubMed and the ClinicalTrials.gov / AACT database maintained by the Clinical Trials Transformation Initiative (CTTI).