DUET-201 / Scopus - LARVOL DELTA

Local CpG-Stat3 siRNA treatment improves antitumor effects of immune checkpoint inhibitors. (PubMed, Mol Ther Nucleic Acids) - "Furthermore, locally delivered CpG-Stat3 siRNA enhanced CD8+ T cell tumor infiltration and antitumor activity in a xenograft tumor model. Overall, our studies in both B cell lymphoma and melanoma mouse models demonstrate the potential of combinatory immunotherapy with CpG-Stat3 siRNA and checkpoint inhibitors as a therapeutic strategy for B cell lymphoma and melanoma."

Checkpoint inhibition • Journal • B Cell Lymphoma • Hematological Malignancies • Lymphoma • Melanoma • Non-Hodgkin’s Lymphoma • Oncology • Solid Tumor • STAT3 • TLR9

DUET-01: A first-in-human, phase 1/2 study of BOXR1030 in patients with advanced glypican-3-positive solid tumors. (ASCO 2024) - P1/2 | "Eligible subjects will undergo leukapheresis to obtain T cells for BOXR1030 manufacturing and will receive 3 days of lymphodepleting chemotherapy with fludarabine and cyclophosphamide within 5 days prior to BOXR1030 administration. Key secondary endpoints include efficacy parameters such as objective response rate, BOXR1030 T-cell expansion and persistence, and levels of inflammatory markers and cytokines. The first patient was treated with BOXR1030 in December 2022."

Clinical • Metastases • P1/2 data • Gastrointestinal Cancer • Hepatocellular Cancer • Liposarcoma • Lung Cancer • Merkel Cell Carcinoma • Non-melanoma Skin Cancer • Oncology • Sarcoma • Skin Cancer • Solid Tumor • Squamous Cell Carcinoma • GPC3

CpG-STAT3 siRNA CAS3/SS3 and Localized Radiation Therapy for the Treatment of Relapsed/Refractory B-Cell NHL (clinicaltrials.gov) - P1 | N=0 | Withdrawn | Sponsor: City of Hope Medical Center | N=18 ➔ 0 | Trial completion date: Dec 2024 ➔ Jan 2024 | Recruiting ➔ Withdrawn | Trial primary completion date: Dec 2024 ➔ Jan 2024

Enrollment change • Trial completion date • Trial primary completion date • Trial withdrawal • Diffuse Large B Cell Lymphoma • Follicular Lymphoma • Hematological Malignancies • Lymphoma • Mantle Cell Lymphoma • Marginal Zone Lymphoma • Non-Hodgkin’s Lymphoma • Oncology • Small Lymphocytic Lymphoma

Lipid Nanoparticle Formulations for the Systemic Delivery of CpG-STAT3 siRNA to Myeloid Immune Cell for Cancer Immunotherapy (ASGCT 2023) - "While studies are ongoing, our results so far suggest potential species-specific differences between LNP targeting properties and underscore the need for testing in the context of human immune system in vitro or in humanized mouse models. We believe that the new LNP2PG formulation will provide vehicle for targeting human hematologic malignancies and solid tumors which rely on the tolerogenic myeloid cells for immune evasion."

Immune cell • Acute Myelogenous Leukemia • Bladder Cancer • Brain Cancer • CNS Tumor • Colon Cancer • Colorectal Cancer • Gastrointestinal Cancer • Gene Therapies • Glioma • Hematological Disorders • Hematological Malignancies • Leukemia • Lymphoma • Melanoma • Non-Hodgkin’s Lymphoma • Oncology • Solid Tumor • Transplantation • IFNA1 • STAT3 • TLR9

CpG-STAT3 siRNA CAS3/SS3 and Localized Radiation Therapy for the Treatment of Relapsed/Refractory B-Cell NHL (clinicaltrials.gov) - P1 | N=18 | Recruiting | Sponsor: City of Hope Medical Center | Trial completion date: Jan 2024 ➔ Dec 2024 | Trial primary completion date: Jan 2024 ➔ Dec 2024

Combination therapy • Trial completion date • Trial primary completion date • Diffuse Large B Cell Lymphoma • Follicular Lymphoma • Hematological Malignancies • Lymphoma • Mantle Cell Lymphoma • Marginal Zone Lymphoma • Non-Hodgkin’s Lymphoma • Oncology • Small Lymphocytic Lymphoma • STAT3

CpG-STAT3 siRNA CAS3/SS3 and Localized Radiation Therapy for the Treatment of Relapsed/Refractory B-Cell NHL (clinicaltrials.gov) - P1 | N=18 | Recruiting | Sponsor: City of Hope Medical Center | Initiation date: May 2022 ➔ Aug 2022

Combination therapy • Trial initiation date • Diffuse Large B Cell Lymphoma • Follicular Lymphoma • Hematological Malignancies • Lymphoma • Mantle Cell Lymphoma • Marginal Zone Lymphoma • Non-Hodgkin’s Lymphoma • Oncology • Small Lymphocytic Lymphoma • STAT3

Lipid Nanoparticle Formulation Enhances Gene Silencing, Immunostimulatory Properties and In Vivo Efficacy of CpG-STAT3siRNA Against Human B Cell Lymphoma (ASGCT 2022) - "These studies indicated ~10-fold increase in the potency of our oligonucleotide in local administration resulting in lymphoma growth arrest. While further studies in syngeneic lymphoma models are ongoing, our data suggest that the new LNP-02 formulation of CpG-STAT3siRNA can provide an attractive new therapeutic option for the immunotherapy of B cell lymphoma and potentially certain other malignancies."

IO biomarker • Preclinical • Brain Cancer • Gene Therapies • Glioma • Hematological Malignancies • Lymphoma • Non-Hodgkin’s Lymphoma • Oncology • Solid Tumor • Transplantation • IFNA1 • STAT3

CpG-STAT3 siRNA CAS3/SS3 and Localized Radiation Therapy for the Treatment of Relapsed/Refractory B-Cell NHL (clinicaltrials.gov) - P1 | N=18 | Recruiting | Sponsor: City of Hope Medical Center | Trial completion date: Jul 2023 ➔ Jan 2024 | Trial primary completion date: Jul 2023 ➔ Jan 2024

Combination therapy • Trial completion date • Trial primary completion date • Diffuse Large B Cell Lymphoma • Follicular Lymphoma • Hematological Malignancies • Lymphoma • Mantle Cell Lymphoma • Marginal Zone Lymphoma • Non-Hodgkin’s Lymphoma • Oncology • Small Lymphocytic Lymphoma • STAT3

Scopus BioPharma’s Subsidiary — Duet Therapeutics — Announces Release of Preclinical Data Being Presented at 17th Annual Meeting of the Oligonucleotide Therapeutics Society (GlobeNewswire) - "Scopus BioPharma Inc...announced the release of preclinical data being presented at the 17th Annual Meeting of the Oligonucleotide Therapeutics Society...Data from two different studies benchmarked Duet’s proprietary bifunctional oligonucleotides, CpG-STAT3siRNA (DUET-01) and CpG-STAT3ASO (DUET-02), against Checkpoint Inhibitors in lymphoma and prostate tumor models in mice....Dr. Horsager is also presenting results of a study comparing Duet’s antisense-based bifunctional molecule, or DUET-02, to both PD-1 and CTLA-4 Checkpoint Inhibitors in a bone localized tumor model of metastatic prostate cancer...Duet expects....clinical Phase 1 trials beginning in Q1 2023 in the United States."

New P1 trial • Preclinical • Genito-urinary Cancer • Hematological Malignancies • Lymphoma • Oncology • Prostate Cancer

Scopus BioPharma's Subsidiary — Duet Therapeutics — to Present at the 17th Annual Meeting of the Oligonucleotide Therapeutics Society (GlobeNewswire) - "Dr. Horsager will present DUET-01: Bispecific Oligonucleotide Targeting TLR9 and STAT3 Signaling for B Cell Lymphoma Immunotherapy as part of Session VIII: Oligonucleotide Preclinical I on Wednesday, September 29, 2021....In addition, Dr. Horsager will present Bifunctional Oligonucleotides for Systemic Treatment of Immunologically Cold Solid Tumors as an Industry Talk available throughout the conference."

Clinical data • Diffuse Large B Cell Lymphoma • Hematological Malignancies • Non-Hodgkin’s Lymphoma • Oncology • Solid Tumor

Scopus BioPharma Launches Duet Therapeutics (GlobeNewswire) - "DUET-01 is in a Phase 1 clinical trial, as a monotherapy, for B-cell non-Hodgkin lymphoma. In 2022, Duet is targeting to file two INDs for DUET-02 in two separate indications: genitourinary and head & neck cancers. Duet is also evaluating combination therapies with checkpoint inhibitors."

IND • Trial status • Genito-urinary Cancer • Head and Neck Cancer • Hematological Malignancies • Non-Hodgkin’s Lymphoma • Oncology

CpG-STAT3 siRNA CAS3/SS3 and Localized Radiation Therapy for the Treatment of Relapsed/Refractory B-Cell NHL (clinicaltrials.gov) - P1; N=18; Recruiting; Sponsor: City of Hope Medical Center; Not yet recruiting ➔ Recruiting

Clinical • Combination therapy • Enrollment open • Diffuse Large B Cell Lymphoma • Follicular Lymphoma • Hematological Malignancies • Lymphoma • Mantle Cell Lymphoma • Marginal Zone Lymphoma • Non-Hodgkin’s Lymphoma • Oncology • Small Lymphocytic Lymphoma • STAT3

CpG-STAT3 siRNA CAS3/SS3 and Localized Radiation Therapy for the Treatment of Relapsed/Refractory B-Cell NHL (clinicaltrials.gov) - P1; N=18; Not yet recruiting; Sponsor: City of Hope Medical Center

New P1 trial • Diffuse Large B Cell Lymphoma • Follicular Lymphoma • Hematological Malignancies • Lymphoma • Mantle Cell Lymphoma • Marginal Zone Lymphoma • Non-Hodgkin’s Lymphoma • Oncology • Small Lymphocytic Lymphoma • STAT3

Scopus BioPharma Announces FDA Approval of IND Application for Lead Drug Candidate (GlobeNewswire) - "Scopus BioPharma Inc...announced the approval of an investigational new drug application ('IND') by the United States Food and Drug Administration ('FDA') for CpG-STAT3siRNA, the company’s distinctive immuno-oncology RNA therapy for the treatment of multiple cancers....A Phase 1 clinical trial for B-cell non-Hodgkin lymphoma will be initiated at City of Hope."

IND • Oncology