Efficacy, safety, and biomarker analyses of bintrafusp alfa, a bifunctional fusion protein targeting TGF-β and PD-L1, in patients with advanced non-small cell lung cancer

Background Bintrafusp alfa, a first-in-class bifunctional fusion protein targeting transforming growth factor-β (TGF-β) and programmed cell death ligand 1, has demonstrated encouraging efficacy as second-line treatment in patients with non-small cell lung cancer (NSCLC) in a dose expansion cohort of...

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Main Authors: Arun Rajan, Renee N Donahue, Jeffrey Schlom, James L Gulley, Elizabeth Lamping, Osama Rahma, Houssein Abdul Sater, Jennifer L Marté, Beatriz Walter-Rodriguez, Yulia Vugmeyster, Yo-Ting Tsai, Shania Bailey, Wiem Lassoued, Richy Agajanian, Andrew Weisman, Rena Ito, Masashi Sato, Andreas Machl
Format: Article
Language:English
Published: BMJ Publishing Group 2024-03-01
Series:Journal for ImmunoTherapy of Cancer
Online Access:https://jitc.bmj.com/content/12/3/e008480.full
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author Arun Rajan
Renee N Donahue
Jeffrey Schlom
James L Gulley
Elizabeth Lamping
Osama Rahma
Houssein Abdul Sater
Jennifer L Marté
Beatriz Walter-Rodriguez
Yulia Vugmeyster
Yo-Ting Tsai
Shania Bailey
Wiem Lassoued
Richy Agajanian
Andrew Weisman
Rena Ito
Masashi Sato
Andreas Machl
author_facet Arun Rajan
Renee N Donahue
Jeffrey Schlom
James L Gulley
Elizabeth Lamping
Osama Rahma
Houssein Abdul Sater
Jennifer L Marté
Beatriz Walter-Rodriguez
Yulia Vugmeyster
Yo-Ting Tsai
Shania Bailey
Wiem Lassoued
Richy Agajanian
Andrew Weisman
Rena Ito
Masashi Sato
Andreas Machl
author_sort Arun Rajan
collection DOAJ
description Background Bintrafusp alfa, a first-in-class bifunctional fusion protein targeting transforming growth factor-β (TGF-β) and programmed cell death ligand 1, has demonstrated encouraging efficacy as second-line treatment in patients with non-small cell lung cancer (NSCLC) in a dose expansion cohort of the phase 1, open-label clinical trial (NCT02517398). Here, we report the safety, efficacy, and biomarker analysis of bintrafusp alfa in a second expansion cohort of the same trial (biomarker cohort).Methods Patients with stage IIIb/IV NSCLC who were either immune checkpoint inhibitor (ICI)-naïve (n=18) or ICI-experienced (n=23) were enrolled. The primary endpoint was the best overall response. Paired biopsies (n=9/41) and peripheral blood (n=14/41) pretreatment and on-treatment were studied to determine the immunological effects of treatment and for associations with clinical activity.Results Per independent review committee assessment, objective responses were observed in the ICI-naïve group (overall response rate, 27.8%). No new or unexpected safety signals were identified. Circulating TGF-β levels were reduced (>97%; p<0.001) 2 weeks after initiation of treatment with bintrafusp alfa and remained reduced up to 12 weeks. Increases in lymphocytes and tumor-associated macrophages (TAMs) were observed in on-treatment biospies, with an increase in the M2 (tumor trophic TAMs)/M1 (inflammatory TAMs) ratio associated with poor outcomes. Specific peripheral immune analytes at baseline and early changes after treatment were associated with clinical response.Conclusions Bintrafusp alfa was observed to have modest clinical activity and manageable safety, and was associated with notable immunologic changes involving modulation of the tumor immune microenvironment in patients with advanced NSCLC.
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spelling doaj-art-299f4d847daa42ea885a8c75c936bce72025-08-20T02:49:09ZengBMJ Publishing GroupJournal for ImmunoTherapy of Cancer2051-14262024-03-0112310.1136/jitc-2023-008480Efficacy, safety, and biomarker analyses of bintrafusp alfa, a bifunctional fusion protein targeting TGF-β and PD-L1, in patients with advanced non-small cell lung cancerArun Rajan0Renee N Donahue1Jeffrey Schlom2James L Gulley3Elizabeth Lamping4Osama Rahma5Houssein Abdul Sater6Jennifer L Marté7Beatriz Walter-Rodriguez8Yulia Vugmeyster9Yo-Ting Tsai10Shania Bailey11Wiem Lassoued12Richy Agajanian13Andrew Weisman14Rena Ito15Masashi Sato16Andreas Machl17Thoracic and Gastrointestinal Malignancies Branch, Center for Cancer Research, National Cancer Institute, National Institutes of Health, Bethesda, Maryland, USACenter for Immuno-Oncology, Center for Cancer Research, National Cancer Institute, Bethesda, Maryland, USACenter for Immuno-Oncology, Center for Cancer Research, National Cancer Institute, National Institutes of Health, Bethesda, Maryland, USACenter for Immuno-Oncology, Center for Cancer Research, National Cancer Institute, National Institutes of Health, Bethesda, Maryland, USAGenitourinary Malignancies Branch, Center for Cancer Research, National Cancer Institute, National Institutes of Health, Bethesda, Maryland, USA1 Harvard Medical School, Boston, Massachusetts, USACleveland Clinic, Stuart, Florida, USAGenitourinary Malignancies Branch, Center for Cancer Research, National Cancer Institute, National Institutes of Health, Bethesda, Maryland, USALaboratory of Pathology, National Cancer Institute, National Institutes of Health, Bethesda, Maryland, USAEMD Serono Research & Development Institute, Inc., an affiliate of Merck KGaA, Billerica, Massachusetts, USACenter for Immuno-Oncology, Center for Cancer Research, National Cancer Institute, National Institutes of Health, Bethesda, Maryland, USAUniversity of Maryland School of Medicine, Baltimore, Maryland, USACenter for Immuno-Oncology, Center for Cancer Research, National Cancer Institute, National Institutes of Health, Bethesda, Maryland, USAInnovative Clinical Research Institute, Whittier, California, USAAxle Informatics, Bethesda, Maryland, USAMerck Biopharma Co., Ltd; an affiliate of Merck KGaA, Tokyo, JapanMerck Biopharma Co., Ltd; an affiliate of Merck KGaA, Tokyo, JapanEMD Serono Research & Development Institute, Inc., an affiliate of Merck KGaA, Billerica, Massachusetts, USABackground Bintrafusp alfa, a first-in-class bifunctional fusion protein targeting transforming growth factor-β (TGF-β) and programmed cell death ligand 1, has demonstrated encouraging efficacy as second-line treatment in patients with non-small cell lung cancer (NSCLC) in a dose expansion cohort of the phase 1, open-label clinical trial (NCT02517398). Here, we report the safety, efficacy, and biomarker analysis of bintrafusp alfa in a second expansion cohort of the same trial (biomarker cohort).Methods Patients with stage IIIb/IV NSCLC who were either immune checkpoint inhibitor (ICI)-naïve (n=18) or ICI-experienced (n=23) were enrolled. The primary endpoint was the best overall response. Paired biopsies (n=9/41) and peripheral blood (n=14/41) pretreatment and on-treatment were studied to determine the immunological effects of treatment and for associations with clinical activity.Results Per independent review committee assessment, objective responses were observed in the ICI-naïve group (overall response rate, 27.8%). No new or unexpected safety signals were identified. Circulating TGF-β levels were reduced (>97%; p<0.001) 2 weeks after initiation of treatment with bintrafusp alfa and remained reduced up to 12 weeks. Increases in lymphocytes and tumor-associated macrophages (TAMs) were observed in on-treatment biospies, with an increase in the M2 (tumor trophic TAMs)/M1 (inflammatory TAMs) ratio associated with poor outcomes. Specific peripheral immune analytes at baseline and early changes after treatment were associated with clinical response.Conclusions Bintrafusp alfa was observed to have modest clinical activity and manageable safety, and was associated with notable immunologic changes involving modulation of the tumor immune microenvironment in patients with advanced NSCLC.https://jitc.bmj.com/content/12/3/e008480.full
spellingShingle Arun Rajan
Renee N Donahue
Jeffrey Schlom
James L Gulley
Elizabeth Lamping
Osama Rahma
Houssein Abdul Sater
Jennifer L Marté
Beatriz Walter-Rodriguez
Yulia Vugmeyster
Yo-Ting Tsai
Shania Bailey
Wiem Lassoued
Richy Agajanian
Andrew Weisman
Rena Ito
Masashi Sato
Andreas Machl
Efficacy, safety, and biomarker analyses of bintrafusp alfa, a bifunctional fusion protein targeting TGF-β and PD-L1, in patients with advanced non-small cell lung cancer
Journal for ImmunoTherapy of Cancer
title Efficacy, safety, and biomarker analyses of bintrafusp alfa, a bifunctional fusion protein targeting TGF-β and PD-L1, in patients with advanced non-small cell lung cancer
title_full Efficacy, safety, and biomarker analyses of bintrafusp alfa, a bifunctional fusion protein targeting TGF-β and PD-L1, in patients with advanced non-small cell lung cancer
title_fullStr Efficacy, safety, and biomarker analyses of bintrafusp alfa, a bifunctional fusion protein targeting TGF-β and PD-L1, in patients with advanced non-small cell lung cancer
title_full_unstemmed Efficacy, safety, and biomarker analyses of bintrafusp alfa, a bifunctional fusion protein targeting TGF-β and PD-L1, in patients with advanced non-small cell lung cancer
title_short Efficacy, safety, and biomarker analyses of bintrafusp alfa, a bifunctional fusion protein targeting TGF-β and PD-L1, in patients with advanced non-small cell lung cancer
title_sort efficacy safety and biomarker analyses of bintrafusp alfa a bifunctional fusion protein targeting tgf β and pd l1 in patients with advanced non small cell lung cancer
url https://jitc.bmj.com/content/12/3/e008480.full
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