VNX-101-2 / Vironexis Biotherapeutics - LARVOL DELTA

Using Gene Therapy to Solve Challenges with CAR-T Cell Immunotherapy: Lead Selection and Preclinical Development of an Adeno-Associated Virus with Reduced Immunogenicity Exhibiting Efficient and Long-Term Expression of an Anti-CD19 T-Cell Engager (ASH 2024) - "GP101 purified after cell transfection using this construct was functionally equivalent to blinatumomab in a co-culture cell killing assay (both EC50s < 0.08 ng/mL). Collectively, these preclinical data support the efficacy and safety of VNX-101 as a potential AAV-based treatment for cancer. Based on our findings, we have received a "safe to proceed" from the FDA to conduct a Phase 1/2 clinical trial of VNX-101 in patients with relapsed or refractory B cell precursor ALL."

CAR T-Cell Therapy • Gene therapy • IO biomarker • Preclinical • Burkitt Lymphoma • Gene Therapies • Hematological Malignancies • Hepatology • Lymphoma • Oncology • AVEN

VNX-101-01: Gene Therapy for CD19-Positive Hematologic Malignancies (SENTRY-CD19) (clinicaltrials.gov) - P1/2 | N=32 | Recruiting | Sponsor: Vironexis Biotherapeutics Inc. | Trial primary completion date: Jun 2026 ➔ Jun 2027

Trial primary completion date • Acute Lymphocytic Leukemia • B Acute Lymphoblastic Leukemia • B Cell Lymphoma • Burkitt Lymphoma • Chronic Lymphocytic Leukemia • Diffuse Large B Cell Lymphoma • Follicular Lymphoma • Gene Therapies • Hematological Malignancies • High-grade B-cell lymphoma • Large B Cell Lymphoma • Leukemia • Lymphoma • Mantle Cell Lymphoma • Marginal Zone Lymphoma • Non-Hodgkin’s Lymphoma • Oncology • Primary Mediastinal Large B-Cell Lymphoma • Small Lymphocytic Lymphoma

Development of VNX-101, an adeno-associated virus with less immunogenicity and efficient long-term expression of a CD19 T cell engager. (PubMed, Mol Ther Methods Clin Dev) - "Collectively, these preclinical data support the efficacy and safety of VNX-101 as a potential AAV-based treatment for cancer. A phase 1/2 clinical trial of VNX-101 for relapsed or refractory B cell malignancies is under way."

Journal • Gene Therapies • Hematological Malignancies • Leukemia • Lymphoma • Oncology • CD19

Design of the First Clinical Trial of AAV Gene Therapy in Immuno-Oncology (SENTRY-ALL): A Single Dose CD3/CD19 Bi-Specific T-Cell Engager for the Treatment of CD19+ B-Cell Acute Lymphoblastic Leukemia (ASGCT 2025) - P1/2 | "VNX-101 is an investigational adeno-associated virus (AAV) gene therapy developed to express a secreted anti-CD19/anti-CD3 scFv diabody (termed GP101)...Prior blinatumomab and/or CD19 CAR T-cell treatment is allowed...This trial aims to provide the first clinical experience in the use of AAV gene therapy as a new paradigm for cancer immunotherapy. Disease Focus of Abstract:Cancer Hematologic"

Clinical • Gene therapy • Immuno-oncology • Acute Lymphocytic Leukemia • B Acute Lymphoblastic Leukemia • Gene Therapies • Hematological Malignancies • Leukemia • Lymphoma • Oncology

Development of an Adeno-Associated Virus Expressing a Secreted T Cell Engager for Long-Term B Cell Ablation with High Transgene Expression to Minimize Vector Dose (ASGCT 2025) - "Serum levels of GP101 were consistently above the lower threshold of the human therapeutic target range (300 pg/mL, based on clinical experience with blinatumomab) at doses of 3.0E10 vg/kg (females) and 1.0E10 vg/kg (males). Based on the mechanism of action, VNX-101 may also be applicable to B cell lymphoma, chronic lymphocytic leukemia, and autoimmune diseases. Disease Focus of Abstract:Cancer Hematologic"

IO biomarker • B Cell Lymphoma • Chronic Lymphocytic Leukemia • Gene Therapies • Hematological Disorders • Hematological Malignancies • Hepatology • Immunology • Leukemia • Lymphoma • Non-Hodgkin’s Lymphoma • Oncology

VNX-101-01: Gene Therapy for B-Cell Acute Lymphoblastic Leukemia (clinicaltrials.gov) - P1/2 | N=26 | Recruiting | Sponsor: Vironexis Biotherapeutics Inc. | Initiation date: Dec 2024 ➔ Mar 2025

Trial initiation date • Acute Lymphocytic Leukemia • B Acute Lymphoblastic Leukemia • Gene Therapies • Hematological Malignancies • Leukemia • Oncology

VNX-101-01: Gene Therapy for B-Cell Acute Lymphoblastic Leukemia (clinicaltrials.gov) - P1/2 | N=26 | Recruiting | Sponsor: Vironexis Biotherapeutics Inc. | Not yet recruiting ➔ Recruiting

Enrollment open • Gene therapy • Acute Lymphocytic Leukemia • B Acute Lymphoblastic Leukemia • Gene Therapies • Hematological Malignancies • Leukemia • Oncology

Vironexis Biotherapeutics Launches with FDA Clearance of IND Application for First-Ever Clinical Trial of an AAV-delivered Cancer Immunotherapy (GlobeNewswire) - "Vironexis Biotherapeutics...launched from stealth today, unveiling its TransJoin AAV Gene Therapy Platform and a pipeline of more than ten product candidates for blood-based cancers, solid tumor metastasis prevention, and a cancer vaccine. Vironexis’s $26 million seed financing was led by Drive Capital and Future Ventures, with participation from Moonshots Capital and Capital Factory. The company has received clearance of its Investigational New Drug (IND) application from the U.S. Food and Drug Administration (FDA) for VNX-101, its first gene therapy product candidate, for the treatment of CD19+ acute lymphoblastic leukemia. Vironexis anticipates initiating patient enrollment of a Phase 1/2 trial of VNX-101 in the fourth quarter of 2024, which will mark the first-ever clinical trial of an AAV-delivered cancer immunotherapy."

Financing • IND • Pipeline update • Trial status • Acute Lymphocytic Leukemia • Hematological Malignancies • Leukemia • Oncology • Solid Tumor

Gene Therapy for B-Cell Acute Lymphoblastic Leukemia (clinicaltrials.gov) - P1/2 | N=26 | Not yet recruiting | Sponsor: Vironexis Biotherapeutics Inc.

Gene therapy • New P1/2 trial • Acute Lymphocytic Leukemia • B Acute Lymphoblastic Leukemia • Gene Therapies • Hematological Malignancies • Leukemia • Oncology