Targeted apoptotic immune modulator for the treatment of metastatic EGFR-positive solid tumors

D Derrick Broka (TargImmune Therapeutics) S Shoshana Klein (Department of Biological Chemistry, Silberman Life Sciences Institute, The Hebrew University of Jerusalem, Edmond J. Safra Campus) A Alexei Shir (TargImmune Therapeutics) B Babette Schade (TargImmune Therapeutics) M Meera Saxena (TargImmune Therapeutics) A Athanasia Dasargyri (TargImmune Therapeutics) A Anita Jarzebinska (TargImmune Therapeutics) C Caroline De Feyter (TargImmune Therapeutics) D Davor Bajic (TargImmune Therapeutics) D David Colecchia (TargImmune Therapeutics) L Lucia D'Amico (TargImmune Therapeutics) E Eric Kitas (TargImmune Therapeutics) E Elad Hikri (Department of Otorhinolaryngology, Head and Neck Surgery, Rabin Medical Center) M Michal Jerzy Skowicki (Department of Chemistry, University of Basel) M Michal Jerzy Okoniewski (Scientific IT Services ID, ETH) L Laura Baldino (Human Oncology and Pathogenesis Program, Memorial Sloan Kettering Cancer Center) B Besnik Qeriqi (Molecular Pharmacology Program, Memorial Sloan-Kettering Cancer Center) E Elisa de Stanchina J Joerg Schreiber (Department of Biosystems Science and Engineering, Swiss Federal Institute of Technology) M Melanie Buchi (Department of Biomedicine, University of Basel and University Hospital) C Cornelia G. Palivan (Department of Chemistry, University of Basel) Y Yaakov Benenson (Department of Biosystems Science and Engineering, Swiss Federal Institute of Technology) A Alfred Zippelius D Doriano Fabbro (TargImmune Therapeutics) M Maurizio Scaltriti A Aviram Mizrachi (Department of Otorhinolaryngology, Head and Neck Surgery, Rabin Medical Center) A Alexander Levitzki (Department of Biological Chemistry, Silberman Life Sciences Institute, The Hebrew University of Jerusalem, Edmond J. Safra Campus) E Esteban Pombo-Villar (TargImmune Therapeutics) M Maya Zigler (TargImmune Therapeutics)

Abstract

Aberrant activation and overexpression of the epidermal growth factor receptor (EGFR) occurs in various solid cancers and often correlates with poor outcome. The clinical benefit from EGFR-targeted therapies is usually short-lived, with resistance being driven by tumor heterogeneity and an immunosuppressive tumor microenvironment (TME). To address these limitations, we developed Targeted Apoptotic Immune Modulators (TAIM), a nonviral nanoparticle platform for the targeted delivery of polyinosine:polycytosine (polyIC), to simultaneously induce tumor cell death and activate antitumor immunity. The first TAIM compound, TAR001, was designed as a systemic treatment against metastatic EGFR-positive solid cancers. Here, we present TAR001’s multifaceted mode of action. We demonstrate that TAR001 is selective toward EGFR-overexpressing cancers, provoking a pattern recognition response, apoptosis, cytokine secretion, and antitumor immunity. TAR001 modulates the TME, recruiting and activating both innate and adaptive immune cells. Systemic delivery of TAR001 markedly extends survival and inhibits tumor growth in multiple murine tumor models. TAR001 represents an innovative, safe, multimodal treatment approach with the potential to benefit patients with metastatic head and neck, non–small cell lung cancer, colorectal, renal, and triple-negative breast cancers. This unique modality utilizes a broad range of mechanisms to overcome the tumor’s ability to escape apoptosis and immune cell activation.

Article Details

Volume / Issue Vol. 122, Issue 22
Published June 03, 2025
ISSN 0027-8424
Publisher National Academy of Sciences

Authors (29)

D

Derrick Broka

TargImmune Therapeutics

S

Shoshana Klein

Department of Biological Chemistry, Silberman Life Sciences Institute, The Hebrew University of Jerusalem, Edmond J. Safra Campus

A

Alexei Shir

TargImmune Therapeutics

B

Babette Schade

TargImmune Therapeutics

M

Meera Saxena

TargImmune Therapeutics

A

Athanasia Dasargyri

TargImmune Therapeutics

A

Anita Jarzebinska

TargImmune Therapeutics

C

Caroline De Feyter

TargImmune Therapeutics

D

Davor Bajic

TargImmune Therapeutics

D

David Colecchia

TargImmune Therapeutics

L

Lucia D'Amico

TargImmune Therapeutics

E

Eric Kitas

TargImmune Therapeutics

E

Elad Hikri

Department of Otorhinolaryngology, Head and Neck Surgery, Rabin Medical Center

M

Michal Jerzy Skowicki

Department of Chemistry, University of Basel

M

Michal Jerzy Okoniewski

Scientific IT Services ID, ETH

L

Laura Baldino

Human Oncology and Pathogenesis Program, Memorial Sloan Kettering Cancer Center

B

Besnik Qeriqi

Molecular Pharmacology Program, Memorial Sloan-Kettering Cancer Center

E

Elisa de Stanchina

J

Joerg Schreiber

Department of Biosystems Science and Engineering, Swiss Federal Institute of Technology

M

Melanie Buchi

Department of Biomedicine, University of Basel and University Hospital

C

Cornelia G. Palivan

Department of Chemistry, University of Basel

Y

Yaakov Benenson

Department of Biosystems Science and Engineering, Swiss Federal Institute of Technology

A

Alfred Zippelius

D

Doriano Fabbro

TargImmune Therapeutics

M

Maurizio Scaltriti

A

Aviram Mizrachi

Department of Otorhinolaryngology, Head and Neck Surgery, Rabin Medical Center

A

Alexander Levitzki

Department of Biological Chemistry, Silberman Life Sciences Institute, The Hebrew University of Jerusalem, Edmond J. Safra Campus

E

Esteban Pombo-Villar

TargImmune Therapeutics

M

Maya Zigler

TargImmune Therapeutics