Long-Term Survival and Biomarker Analysis Evaluating Neoadjuvant Plus Adjuvant Relatlimab (anti-LAG3) and Nivolumab (anti-PD1) in Patients With Resectable Melanoma

E Elizabeth M. Burton D Denái R. Milton (Department of Biostatistics, The University of Texas MD Anderson Cancer Center, Houston, TX) M Michael T. Tetzlaff K Khalida Wani M Merrick I. Ross M Michael A. Postow (From the Sandra and Edward Meyer Cancer Center (J.D.W.) and the Department of Medicine (J.D.W., M.A.P.), Weill Cornell Medicine, and Memorial Sloan Kettering Cancer Center (M.A.P.) — both in New York; Istituto Oncologico Veneto, IRCCS, Padua (V.C.-S.), European Institute of Oncology, IRCCS, Milan (P.Q.), Istituto Scientifico Romagnolo per lo Studio e la Cura dei Tumori, IRCCS, Meldola (M.G.), University of Siena and the Center for Immuno-Oncology, University Hospital of Siena, Siena (M.M.), and Istituto Nazionale Tumori IRCCS Fondazione Pascale, Naples (P.A.A.) — all in Italy; Maria Sklodowska-Curie National Institute of Oncology, Warsaw, Poland (P.R.); Texas Oncology–Baylor Charles A. Sammons Cancer Center, Dallas (C.L.C.); University Hospital Essen, the German Cancer Consortium, the National Center for Tumor Diseases–West, the Research Alliance Ruhr, Research Center One Health, and University Duisburg-Essen — all in Essen, Germany (D.S.); the College of Medicine, Swansea University, Swansea (J.W.), Brist...) R Rossana Lazcano (Department of Translational Molecular Pathology, The University of Texas MD Anderson Cancer Center, Houston, TX) I Isabella C. Glitza (Department of Melanoma Medical Oncology, The University of Texas MD Anderson Cancer Center, Houston, TX) M Michael K. Wong S Sapna P. Patel (Department of Melanoma Medical Oncology, The University of Texas MD Anderson Cancer Center, Houston, TX) A Adi Diab (The University of Texas MD Anderson Cancer Center) J Jeffrey E. Gershenwald J Jennifer L. McQuade (Department of Melanoma Medical Oncology, The University of Texas MD Anderson Cancer Center, Houston, TX) A Allison Betof Warner (Stanford University School of Medicine, Stanford University, Stanford, CA) V Victor G. Prieto (Department of Pathology, The University of Texas MD Anderson Cancer Center, Houston, TX) J Jeffrey E. Lee (Department of Surgical Oncology, The University of Texas MD Anderson Cancer Center, Houston, TX) R Ryan P. Goepfert S Sarah B. Fisher (Department of Surgical Oncology, The University of Texas MD Anderson Cancer Center, Houston, TX) A Addison Song (Department of Translational Molecular Pathology, The University of Texas MD Anderson Cancer Center, Houston, TX) J Jared Malke (Department of Surgical Oncology, The University of Texas MD Anderson Cancer Center, Houston, TX) J Julie M. Simon (Department of Surgical Oncology, The University of Texas MD Anderson Cancer Center, Houston, TX) C Charlotte Ariyan (Department of Surgical Oncology, Memorial Sloan Kettering Cancer Center, New York, NY) C Carlos A. Torres-Cabala (Department of Translational Molecular Pathology, The University of Texas MD Anderson Cancer Center, Houston, TX) M Michael A. Davies A Alexander Lazar J Jennifer A. Wargo H Hussein A. Tawbi (The University of Texas MD Anderson Cancer Center, Houston, TX) R Rodabe N. Amaria (Department of Melanoma Medical Oncology, The University of Texas MD Anderson Cancer Center, Houston, TX)

Abstract

Immune checkpoint blockade (ICB) has revolutionized outcomes for patients with melanoma across multiple disease settings. In patients with advanced, unresectable disease, the ICB combination of nivolumab (anti-PD1) and relatlimab (anti–LAG-3) has demonstrated improved clinical outcomes compared with nivolumab monotherapy. There exists an unmet need to identify biomarkers that predict response to this combination regimen and rational therapeutic strategies to overcome resistance. We previously reported the initial results of a phase II clinical trial (ClinicalTrials.gov identifier: NCT02519322 ) of neoadjuvant systemic treatment (NST) followed by adjuvant treatment with nivolumab and relatlimab, which achieved a major pathologic response (MPR; ≤10% viable tumor) rate of 63% in patients with stage III/IV, surgically resectable melanoma. Our updated clinical follow-up (median 47 months) for these patients demonstrates that at 4 years from the start of NST, 80% of patients remain event-free, including 95% of patients who achieved a MPR. Gene expression analysis of longitudinally collected biospecimens from the trial identifies baseline upregulation of several immune modulatory pathways associated with MPR; by contrast, increased B7-H3 expression was associated with resistance. This work demonstrates the long-term benefit of neoadjuvant nivolumab and relatlimab and identifies a potentially targetable predictor of resistance to this combination therapy.

Article Details

Volume / Issue Vol. 43, Issue 26
Published September 10, 2025
Pages 2856-2862
ISSN 0732-183X
Publisher Lippincott Williams & Wilkins

Journal Info

Journal of Clinical Oncology

Lippincott Williams & Wilkins

ISSN: 0732-183X Health Sciences

Authors (28)

E

Elizabeth M. Burton

D

Denái R. Milton

Department of Biostatistics, The University of Texas MD Anderson Cancer Center, Houston, TX

M

Michael T. Tetzlaff

K

Khalida Wani

M

Merrick I. Ross

M

Michael A. Postow

From the Sandra and Edward Meyer Cancer Center (J.D.W.) and the Department of Medicine (J.D.W., M.A.P.), Weill Cornell Medicine, and Memorial Sloan Kettering Cancer Center (M.A.P.) — both in New York; Istituto Oncologico Veneto, IRCCS, Padua (V.C.-S.), European Institute of Oncology, IRCCS, Milan (P.Q.), Istituto Scientifico Romagnolo per lo Studio e la Cura dei Tumori, IRCCS, Meldola (M.G.), University of Siena and the Center for Immuno-Oncology, University Hospital of Siena, Siena (M.M.), and Istituto Nazionale Tumori IRCCS Fondazione Pascale, Naples (P.A.A.) — all in Italy; Maria Sklodowska-Curie National Institute of Oncology, Warsaw, Poland (P.R.); Texas Oncology–Baylor Charles A. Sammons Cancer Center, Dallas (C.L.C.); University Hospital Essen, the German Cancer Consortium, the National Center for Tumor Diseases–West, the Research Alliance Ruhr, Research Center One Health, and University Duisburg-Essen — all in Essen, Germany (D.S.); the College of Medicine, Swansea University, Swansea (J.W.), Brist...

R

Rossana Lazcano

Department of Translational Molecular Pathology, The University of Texas MD Anderson Cancer Center, Houston, TX

I

Isabella C. Glitza

Department of Melanoma Medical Oncology, The University of Texas MD Anderson Cancer Center, Houston, TX

M

Michael K. Wong

S

Sapna P. Patel

Department of Melanoma Medical Oncology, The University of Texas MD Anderson Cancer Center, Houston, TX

A

Adi Diab

The University of Texas MD Anderson Cancer Center

J

Jeffrey E. Gershenwald

J

Jennifer L. McQuade

Department of Melanoma Medical Oncology, The University of Texas MD Anderson Cancer Center, Houston, TX

A

Allison Betof Warner

Stanford University School of Medicine, Stanford University, Stanford, CA

V

Victor G. Prieto

Department of Pathology, The University of Texas MD Anderson Cancer Center, Houston, TX

J

Jeffrey E. Lee

Department of Surgical Oncology, The University of Texas MD Anderson Cancer Center, Houston, TX

R

Ryan P. Goepfert

S

Sarah B. Fisher

Department of Surgical Oncology, The University of Texas MD Anderson Cancer Center, Houston, TX

A

Addison Song

Department of Translational Molecular Pathology, The University of Texas MD Anderson Cancer Center, Houston, TX

J

Jared Malke

Department of Surgical Oncology, The University of Texas MD Anderson Cancer Center, Houston, TX

J

Julie M. Simon

Department of Surgical Oncology, The University of Texas MD Anderson Cancer Center, Houston, TX

C

Charlotte Ariyan

Department of Surgical Oncology, Memorial Sloan Kettering Cancer Center, New York, NY

C

Carlos A. Torres-Cabala

Department of Translational Molecular Pathology, The University of Texas MD Anderson Cancer Center, Houston, TX

M

Michael A. Davies

A

Alexander Lazar

J

Jennifer A. Wargo

H

Hussein A. Tawbi

The University of Texas MD Anderson Cancer Center, Houston, TX

R

Rodabe N. Amaria

Department of Melanoma Medical Oncology, The University of Texas MD Anderson Cancer Center, Houston, TX