First-in-human phase I study of immunomodulatory E7046, an antagonist of PGE2-receptor E-type 4 (EP4), in patients with advanced cancers

Background E7046 is a highly selective, small-molecule antagonist of the E-type prostanoid receptor 4 (EP4) for prostaglandin E2, an immunosuppressive mediator of the tumor immune microenvironment. This first-in-human phase 1 study assessed the safety, tolerability, pharmacokinetics, pharmacodynamic...

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Main Authors: Aurélien Marabelle, Aung Naing, Funda Meric-Bernstam, Xingfeng Bao, Andrea Varga, David S Hong, Aparna Parikh, Geoffrey I Shapiro, Özlem Ataman, Larisa Reyderman, Terri A Binder, Min Ren, Mingjie Liu, Satish Dayal, Amy Y Siu, Pallavi Sachdev, Lucy Xu, Vijay Bhagawati-Prasad, Ilian Tchakov, Chean Eng Ooi
Format: Article
Language:English
Published: BMJ Publishing Group 2020-05-01
Series:Journal for ImmunoTherapy of Cancer
Online Access:https://jitc.bmj.com/content/8/1/e000222.full
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author Aurélien Marabelle
Aung Naing
Funda Meric-Bernstam
Xingfeng Bao
Andrea Varga
David S Hong
Aparna Parikh
Geoffrey I Shapiro
Özlem Ataman
Larisa Reyderman
Terri A Binder
Min Ren
Mingjie Liu
Satish Dayal
Amy Y Siu
Pallavi Sachdev
Lucy Xu
Vijay Bhagawati-Prasad
Ilian Tchakov
Chean Eng Ooi
author_facet Aurélien Marabelle
Aung Naing
Funda Meric-Bernstam
Xingfeng Bao
Andrea Varga
David S Hong
Aparna Parikh
Geoffrey I Shapiro
Özlem Ataman
Larisa Reyderman
Terri A Binder
Min Ren
Mingjie Liu
Satish Dayal
Amy Y Siu
Pallavi Sachdev
Lucy Xu
Vijay Bhagawati-Prasad
Ilian Tchakov
Chean Eng Ooi
author_sort Aurélien Marabelle
collection DOAJ
description Background E7046 is a highly selective, small-molecule antagonist of the E-type prostanoid receptor 4 (EP4) for prostaglandin E2, an immunosuppressive mediator of the tumor immune microenvironment. This first-in-human phase 1 study assessed the safety, tolerability, pharmacokinetics, pharmacodynamics, maximum tolerated dose (MTD) and recommended phase 2 dose of E7046.Methods This first-in-human study enrolled 30 patients with advanced tumors of cancer types associated with high levels of myeloid infiltrates. E7046 was administered orally once-daily in sequential escalating dose cohorts (125, 250, 500, and 750 mg) with ≥6 patients per cohort. Tumor assessments were performed every 6 weeks. Paired tumor biopsies and blood samples, before and on treatment, were collected for pharmacokinetic and pharmacodynamic characterization of the treatment.Results No dose-limiting toxicities were observed, and the MTD was not reached. E7046 had an elimination half-life (t1/2) of 12 hours, and drug exposure increased dose-dependently from 125 to 500 mg. Target modulation by E7046 was supported by changes in genes downstream of EP4 with concurrent enhanced antitumoral immune responses. A best response of stable disease (per irRECIST) was reported in 23% of patients treated with E7046 (n=30) (125 mg: n=2; 250 mg: n=2; 750 mg: n=3). Over half (4/7) of the patients with stable disease had treatment duration of 18 weeks or more, and three patients (3/15; 20%) achieved metabolic responses.Conclusions In this first-in-human study, E7046 administered orally once daily demonstrated manageable tolerability, immunomodulatory effects, and a best response of stable disease (≥18 weeks) in several heavily pretreated patients with advanced malignancies. The 250 and 500 mg doses are proposed for further development in the combination setting.Trial registration number NCT02540291.
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spelling doaj-art-e224332c09c046d89e9e666fa3f6b4e32025-08-20T02:49:52ZengBMJ Publishing GroupJournal for ImmunoTherapy of Cancer2051-14262020-05-018110.1136/jitc-2019-000222First-in-human phase I study of immunomodulatory E7046, an antagonist of PGE2-receptor E-type 4 (EP4), in patients with advanced cancersAurélien Marabelle0Aung Naing1Funda Meric-Bernstam2Xingfeng Bao3Andrea Varga4David S Hong5Aparna Parikh6Geoffrey I Shapiro7Özlem Ataman8Larisa Reyderman9Terri A Binder10Min Ren11Mingjie Liu12Satish Dayal13Amy Y Siu14Pallavi Sachdev15Lucy Xu16Vijay Bhagawati-Prasad17Ilian Tchakov18Chean Eng Ooi19Faculté de médecine, Université Paris-Saclay, Le Kremlin-Bicêtre, France14 Department of Investigational Cancer Therapeutics, Division of Cancer Medicine, The University of Texas MD Anderson Cancer Center, Houston, Texas, USA2The University of Texas MD Anderson Cancer Center, Houston, TX, USA11 H3 Biomedicine, Cambridge, Massachusetts, USAPalliative Care Unit, Gustave Roussy Institute, Villejuif, France12 Investigational Cancer Therapeutics, The University of Texas MD Anderson Cancer Center, Houston, Texas, USADepartment of Medicine, Division of Hematology and Oncology, Massachusetts General Hospital, Boston, Massachusetts, USA12Ludwig Center at Harvard, Harvard Medical School, Boston, MA, USA5 Formerly of Eisai, Hatfield, UK6 Eisai, Woodcliff Lake, New Jersey, USA6 Eisai, Woodcliff Lake, New Jersey, USA6 Eisai, Woodcliff Lake, New Jersey, USA7 Formerly of Eisai, Woodcliff Lake, New Jersey, USA8 Eisai, Andover, Massachusetts, USA9 Formerly of Eisai, Andover, Massachusetts, USA6 Eisai, Woodcliff Lake, New Jersey, USA6 Eisai, Woodcliff Lake, New Jersey, USA5 Formerly of Eisai, Hatfield, UK10 Eisai, Hatfield, UK6 Eisai, Woodcliff Lake, New Jersey, USABackground E7046 is a highly selective, small-molecule antagonist of the E-type prostanoid receptor 4 (EP4) for prostaglandin E2, an immunosuppressive mediator of the tumor immune microenvironment. This first-in-human phase 1 study assessed the safety, tolerability, pharmacokinetics, pharmacodynamics, maximum tolerated dose (MTD) and recommended phase 2 dose of E7046.Methods This first-in-human study enrolled 30 patients with advanced tumors of cancer types associated with high levels of myeloid infiltrates. E7046 was administered orally once-daily in sequential escalating dose cohorts (125, 250, 500, and 750 mg) with ≥6 patients per cohort. Tumor assessments were performed every 6 weeks. Paired tumor biopsies and blood samples, before and on treatment, were collected for pharmacokinetic and pharmacodynamic characterization of the treatment.Results No dose-limiting toxicities were observed, and the MTD was not reached. E7046 had an elimination half-life (t1/2) of 12 hours, and drug exposure increased dose-dependently from 125 to 500 mg. Target modulation by E7046 was supported by changes in genes downstream of EP4 with concurrent enhanced antitumoral immune responses. A best response of stable disease (per irRECIST) was reported in 23% of patients treated with E7046 (n=30) (125 mg: n=2; 250 mg: n=2; 750 mg: n=3). Over half (4/7) of the patients with stable disease had treatment duration of 18 weeks or more, and three patients (3/15; 20%) achieved metabolic responses.Conclusions In this first-in-human study, E7046 administered orally once daily demonstrated manageable tolerability, immunomodulatory effects, and a best response of stable disease (≥18 weeks) in several heavily pretreated patients with advanced malignancies. The 250 and 500 mg doses are proposed for further development in the combination setting.Trial registration number NCT02540291.https://jitc.bmj.com/content/8/1/e000222.full
spellingShingle Aurélien Marabelle
Aung Naing
Funda Meric-Bernstam
Xingfeng Bao
Andrea Varga
David S Hong
Aparna Parikh
Geoffrey I Shapiro
Özlem Ataman
Larisa Reyderman
Terri A Binder
Min Ren
Mingjie Liu
Satish Dayal
Amy Y Siu
Pallavi Sachdev
Lucy Xu
Vijay Bhagawati-Prasad
Ilian Tchakov
Chean Eng Ooi
First-in-human phase I study of immunomodulatory E7046, an antagonist of PGE2-receptor E-type 4 (EP4), in patients with advanced cancers
Journal for ImmunoTherapy of Cancer
title First-in-human phase I study of immunomodulatory E7046, an antagonist of PGE2-receptor E-type 4 (EP4), in patients with advanced cancers
title_full First-in-human phase I study of immunomodulatory E7046, an antagonist of PGE2-receptor E-type 4 (EP4), in patients with advanced cancers
title_fullStr First-in-human phase I study of immunomodulatory E7046, an antagonist of PGE2-receptor E-type 4 (EP4), in patients with advanced cancers
title_full_unstemmed First-in-human phase I study of immunomodulatory E7046, an antagonist of PGE2-receptor E-type 4 (EP4), in patients with advanced cancers
title_short First-in-human phase I study of immunomodulatory E7046, an antagonist of PGE2-receptor E-type 4 (EP4), in patients with advanced cancers
title_sort first in human phase i study of immunomodulatory e7046 an antagonist of pge2 receptor e type 4 ep4 in patients with advanced cancers
url https://jitc.bmj.com/content/8/1/e000222.full
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