First-in-human clinical outcomes with NG-350A, an anti-CD40 expressing tumor-selective vector designed to remodel immunosuppressive tumor microenvironments

Background Tumor-selective oncolytic viral vectors are promising anticancer therapeutics; however, challenges with dosing and potency in advanced/metastatic cancers have limited efficacy and usage. NG-350A is a next-generation blood-stable adenoviral vector engineered to express an agonist anti-clus...

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Main Authors: Aung Naing, Danny Khalil, Matthew Thomas, Tom Lillie, Oliver Rosen, Lee Rosen, D Ross Camidge, Rui-Ru Ji, Andrea Stacey
Format: Article
Language:English
Published: BMJ Publishing Group 2024-10-01
Series:Journal for ImmunoTherapy of Cancer
Online Access:https://jitc.bmj.com/content/12/10/e010016.full
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author Aung Naing
Danny Khalil
Matthew Thomas
Tom Lillie
Oliver Rosen
Lee Rosen
D Ross Camidge
Rui-Ru Ji
Andrea Stacey
author_facet Aung Naing
Danny Khalil
Matthew Thomas
Tom Lillie
Oliver Rosen
Lee Rosen
D Ross Camidge
Rui-Ru Ji
Andrea Stacey
author_sort Aung Naing
collection DOAJ
description Background Tumor-selective oncolytic viral vectors are promising anticancer therapeutics; however, challenges with dosing and potency in advanced/metastatic cancers have limited efficacy and usage. NG-350A is a next-generation blood-stable adenoviral vector engineered to express an agonist anti-cluster of differentiation (CD)40 antibody without affecting tumor-selectivity and oncolytic potency.Methods Intravenous and intratumoral (IT) administration of NG-350A was assessed in a phase Ia/Ib study in patients with metastatic/advanced epithelial tumors (NCT03852511). Dose-escalation was performed separately for intravenous (four dose levels available, each with infusions on Days 1, 3 and 5 of a 57-day treatment period) and IT (single injection on D1 only or injections on Days 1, 8, 15 and 22) administration. The primary objective was safety and tolerability; secondary objectives included determining a recommended dose, pharmacokinetics, and immunogenicity.Results In total, 25 heavily pretreated patients received NG-350A (16 with intravenous and 9 with IT administration). Intravenous and IT dosing were both well tolerated, with no evidence of transgene-related or off-target viral toxicity. Intravenous and IT dosing resulted in dose-dependent increases in systemic NG-350A Cmax. Despite both routes of administration inducing anti-virus antibodies, sustained persistence of NG-350A in blood samples was observed up to 7 weeks after the last dose, particularly with higher intravenous dose levels. Delivery of NG-350A to tumors was demonstrated in biopsy samples following both routes of administration; a dose-dependent pattern was seen with intravenous infusion, with four patients remaining positive for vector DNA in biopsies at Day 57. Transgene messenger RNA from replicating NG-350A was detected in 5/12 patients with intravenous treatment and 1/9 patients with IT injection, and sustained increases in inflammatory cytokines were observed following dosing, particularly with higher intravenous dose levels.Conclusions This phase 1a study provided initial proof-of-mechanism for NG-350A, with strong evidence of tumor delivery, viral replication and transgene expression—particularly after intravenous dosing. The lack of transgene-related or off-target viral toxicity was consistent with the highly selective delivery and replication of NG-350A, even after systemic delivery. The efficacy of intravenous-dosed NG-350A will now be evaluated in combination with pembrolizumab (NCT05165433), as well as with chemoradiotherapy (NCT06459869).Trial registration number NCT05165433, NCT06459869.
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spelling doaj-art-271f6fd5885648ffbf19df0742770c6d2025-08-20T02:14:07ZengBMJ Publishing GroupJournal for ImmunoTherapy of Cancer2051-14262024-10-01121010.1136/jitc-2024-010016First-in-human clinical outcomes with NG-350A, an anti-CD40 expressing tumor-selective vector designed to remodel immunosuppressive tumor microenvironmentsAung Naing0Danny Khalil1Matthew Thomas2Tom Lillie3Oliver Rosen4Lee Rosen5D Ross Camidge6Rui-Ru Ji7Andrea Stacey81 Investigational Cancer Therapeutics, The University of Texas MD Anderson Cancer Center, Houston, Texas, USADepartment of Medicine, Memorial Sloan Kettering Cancer Center, New York, New York, USAAkamis Bio Ltd, Abingdon, UKAkamis Bio Ltd, Abingdon, UKClinical, Akamis Bio Inc, Cambridge, Massachusetts, USA11UCLA Medical Center, Los Angeles, CA, USASchool of Medicine, University of Colorado Denver, Denver, Colorado, USAClinical, Akamis Bio Inc, Cambridge, Massachusetts, USAAkamis Bio Ltd, Abingdon, UKBackground Tumor-selective oncolytic viral vectors are promising anticancer therapeutics; however, challenges with dosing and potency in advanced/metastatic cancers have limited efficacy and usage. NG-350A is a next-generation blood-stable adenoviral vector engineered to express an agonist anti-cluster of differentiation (CD)40 antibody without affecting tumor-selectivity and oncolytic potency.Methods Intravenous and intratumoral (IT) administration of NG-350A was assessed in a phase Ia/Ib study in patients with metastatic/advanced epithelial tumors (NCT03852511). Dose-escalation was performed separately for intravenous (four dose levels available, each with infusions on Days 1, 3 and 5 of a 57-day treatment period) and IT (single injection on D1 only or injections on Days 1, 8, 15 and 22) administration. The primary objective was safety and tolerability; secondary objectives included determining a recommended dose, pharmacokinetics, and immunogenicity.Results In total, 25 heavily pretreated patients received NG-350A (16 with intravenous and 9 with IT administration). Intravenous and IT dosing were both well tolerated, with no evidence of transgene-related or off-target viral toxicity. Intravenous and IT dosing resulted in dose-dependent increases in systemic NG-350A Cmax. Despite both routes of administration inducing anti-virus antibodies, sustained persistence of NG-350A in blood samples was observed up to 7 weeks after the last dose, particularly with higher intravenous dose levels. Delivery of NG-350A to tumors was demonstrated in biopsy samples following both routes of administration; a dose-dependent pattern was seen with intravenous infusion, with four patients remaining positive for vector DNA in biopsies at Day 57. Transgene messenger RNA from replicating NG-350A was detected in 5/12 patients with intravenous treatment and 1/9 patients with IT injection, and sustained increases in inflammatory cytokines were observed following dosing, particularly with higher intravenous dose levels.Conclusions This phase 1a study provided initial proof-of-mechanism for NG-350A, with strong evidence of tumor delivery, viral replication and transgene expression—particularly after intravenous dosing. The lack of transgene-related or off-target viral toxicity was consistent with the highly selective delivery and replication of NG-350A, even after systemic delivery. The efficacy of intravenous-dosed NG-350A will now be evaluated in combination with pembrolizumab (NCT05165433), as well as with chemoradiotherapy (NCT06459869).Trial registration number NCT05165433, NCT06459869.https://jitc.bmj.com/content/12/10/e010016.full
spellingShingle Aung Naing
Danny Khalil
Matthew Thomas
Tom Lillie
Oliver Rosen
Lee Rosen
D Ross Camidge
Rui-Ru Ji
Andrea Stacey
First-in-human clinical outcomes with NG-350A, an anti-CD40 expressing tumor-selective vector designed to remodel immunosuppressive tumor microenvironments
Journal for ImmunoTherapy of Cancer
title First-in-human clinical outcomes with NG-350A, an anti-CD40 expressing tumor-selective vector designed to remodel immunosuppressive tumor microenvironments
title_full First-in-human clinical outcomes with NG-350A, an anti-CD40 expressing tumor-selective vector designed to remodel immunosuppressive tumor microenvironments
title_fullStr First-in-human clinical outcomes with NG-350A, an anti-CD40 expressing tumor-selective vector designed to remodel immunosuppressive tumor microenvironments
title_full_unstemmed First-in-human clinical outcomes with NG-350A, an anti-CD40 expressing tumor-selective vector designed to remodel immunosuppressive tumor microenvironments
title_short First-in-human clinical outcomes with NG-350A, an anti-CD40 expressing tumor-selective vector designed to remodel immunosuppressive tumor microenvironments
title_sort first in human clinical outcomes with ng 350a an anti cd40 expressing tumor selective vector designed to remodel immunosuppressive tumor microenvironments
url https://jitc.bmj.com/content/12/10/e010016.full
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