{
  "abstract": "Background POD1UM-201, an open-label, single-arm, phase II multiregional study, evaluated efficacy and tolerability of retifanlimab, a humanized monoclonal antibody targeting programmed cell death protein-1 (PD-1) in chemotherapy-naive patients with recurrent locally advanced or metastatic Merkel cell carcinoma (MCC).Methods Patients were enrolled across 34 sites in the USA, Canada, and Europe. Eligible patients were ≥18 years with confirmed recurrent advanced locoregional or metastatic MCC not amenable to surgery or radiation therapy, had not received previous systemic treatment, had measurable disease, and Eastern Cooperative Oncology Group performance status 0–1. Retifanlimab 500 mg was administered intravenously every 4 weeks (Q4W) for up to 2 years. The primary endpoint was objective response rate (ORR). Key secondary endpoints included duration of response (DOR), disease control rate (DCR), progression-free survival (PFS), overall survival (OS), and safety.Results A total of 101 patients were enrolled between February 12, 2019, and June 16, 2021, with a median duration of follow-up for response of 22.2 (range: 1.1–55.3) months. ORR was 54.5% (95% CI: 44.2% to 64.4%; n=55), including 18 patients (17.8%) with complete responses (CRs) and 37 (36.6%) with partial responses (PRs). DCR was 60.4% (95% CI: 50.2% to 70.0%; n=61). Median DOR was not reached (95% CI: 14.0 months to not estimable (NE)) in patients who achieved CR and was 25.3 months (95% CI: 14.2 months to NE) in those with PR. With a median follow-up of 9.3 (range: 0.0–57.1) months, the estimated median PFS was 16.0 months (95% CI: 9.0 months to 32.2). Median OS was not reached with 63% of patients alive at 3 years. The safety profile was representative of the PD-(ligand)1 inhibitor class, with grade 3 immune-related adverse events occurring in 11 patients (10.9%).Conclusions Retifanlimab 500 mg Q4W led to frequent and durable responses in chemotherapy-naive patients with advanced MCC with an acceptable safety profile. Retifanlimab represents a new immunotherapy option for patients with locally advanced or metastatic MCC.Trial registration number NCT03599713; EudraCT 2018-001627-39",
  "authors": [
    {
      "affiliations": [
        "Department of Oncology, Candiolo Cancer Institute, FPO - IRCCS, Candiolo, Italy"
      ],
      "name": "Giovanni Grignani"
    },
    {
      "affiliations": [
        "Department of Soft Tissue/Bone Sarcoma and Melanoma, The Maria Sklodowska-Curie National Research Institute of Oncology in Warsaw, Warsaw, Poland"
      ],
      "name": "Piotr Rutkowski"
    },
    {
      "affiliations": [
        "Université Paris Cité, AP-HP Dermato-oncology, CIC, Cancer Institute AP-HP. Nord-Paris Cité, INSERM U1342 – Equipe 1 – CNRS EMR8000, Saint Louis Hospital, Paris, France"
      ],
      "name": "Céleste Lebbé"
    },
    {
      "affiliations": [
        "Unit Melanoma and Rare Tumors, IRCCS Istituto Tumori Giovanni Paolo II, Bari, Italy"
      ],
      "name": "Michele Guida"
    },
    {
      "affiliations": [
        "APHM, Service de Dermatologie et de Cancérologie Cutanée, Aix-Marseille University, Marseille, France"
      ],
      "name": "Caroline Gaudy-Marqueste"
    },
    {
      "affiliations": [
        "Medical Oncology 2, IRCCS Ospedale Policlinico San Martino, Genoa, Italy",
        "Department of Surgical Sciences and Integrated Diagnostics (DISC), University of Genoa, Genoa, Italy"
      ],
      "name": "Francesco Spagnolo"
    },
    {
      "affiliations": [
        "UPMC Hillman Cancer Center, Pittsburgh, Pennsylvania, USA"
      ],
      "name": "Melissa Burgess"
    },
    {
      "affiliations": [
        "Oncologia Medica 1, Fondazione IRCCS Istituto Nazionale Tumori, Milan, Italy"
      ],
      "name": "Federica Morano"
    },
    {
      "affiliations": [
        "Department of Dermatology, Centre Hospitalier Universitaire de Nice, Université Côte d’Azur, Nice, France"
      ],
      "name": "Henri Montaudié"
    },
    {
      "affiliations": [
        "Department of Oncology and Haematology, Division of Oncology, University Hospital of Modena, Modena, Italy"
      ],
      "name": "Roberta Depenni"
    },
    {
      "affiliations": [
        "Division of Gastrointestinal Medical Oncology and Neuroendocrine Tumors, European Institute of Oncology (IEO), IRCCS, Milan, Italy"
      ],
      "name": "Francesca Spada"
    },
    {
      "affiliations": [
        "Translational Science and Therapeutics Division, Fred Hutchinson Cancer Center and Department of Laboratory Medicine and Pathology, University of Washington, Seattle, Washington, USA"
      ],
      "name": "Cecilia C S Yeung"
    },
    {
      "affiliations": [
        "Incyte Corporation, Wilmington, Delaware, USA"
      ],
      "name": "Jennifer Pulini"
    },
    {
      "affiliations": [
        "Incyte Corporation, Wilmington, Delaware, USA"
      ],
      "name": "Mark Cornfeld"
    },
    {
      "affiliations": [
        "Incyte Corporation, Wilmington, Delaware, USA"
      ],
      "name": "Chuan Tian"
    },
    {
      "affiliations": [
        "Division of Medical Oncology, Department of Medicine, University of Washington and Fred Hutchinson Cancer Center, Seattle, Washington, USA"
      ],
      "name": "Shailender Bhatia"
    }
  ],
  "title": "Phase II study of retifanlimab in patients with recurrent locally advanced or metastatic Merkel cell carcinoma (POD1UM-201)",
  "uid": "f7096fdb-d793-5010-b16b-7d86576543ff"
}