VNX-202 gene therapy for HER2-positive solid tumors
A Phase 1/2, First-in-Human Study of VNX-202 Gene Therapy in Patients With HER2-Positive Cancer
PHASE1; PHASE2 · Vironexis Biotherapeutics Inc. · NCT07192432
This study will test a one-time IV gene therapy (VNX-202) that instructs the liver to produce a HER2-targeting protein to treat adults with HER2-positive solid tumors who have had prior therapy or are at high risk of relapse.
Quick facts
| Phase | PHASE1; PHASE2 |
|---|---|
| Study type | Interventional |
| Enrollment | 27 (estimated) |
| Ages | 18 Years and up |
| Sex | All |
| Sponsor | Vironexis Biotherapeutics Inc. (industry) |
| Locations | 5 sites (Los Angeles, California and 4 other locations) |
| Trial ID | NCT07192432 on ClinicalTrials.gov |
What this trial studies
This is a first-in-human Phase 1/2, open-label, dose-escalation and expansion study of VNX-202, an AAV vector designed to drive continuous systemic secretion of a secreted anti-HER2/anti-CD3 diabody called GP202. Part 1 (about 12 patients) uses escalating single IV doses to find the minimal dose that achieves target steady-state PK levels at 8 weeks without dose-limiting toxicities and to define the recommended Part 2 dose. Part 2 (about 15 patients) will enroll patients with earlier-stage, high relapse–risk HER2-positive disease to characterize safety and preliminary anti-tumor activity at the selected dose. Safety monitoring includes standard labs, hepatic assessments, and long-term follow-up for AAV-related events and immune-mediated toxicities.
Who should consider this trial
Good fit: Adults aged 18 or older with histologically or cytologically confirmed HER2-positive solid tumors, ECOG 0–1, acceptable organ and hematologic function, AAV capsid antibody ≤1:400, and either metastatic disease progressed after prior therapy (Part 1) or early-stage disease at high risk of relapse (Part 2).
Not a fit: Patients with active CNS or leptomeningeal disease, significant hepatotoxicity (AST/ALT >2x ULN), history of thrombotic microangiopathy or cardiomyopathy, pregnancy or lactation, high anti-AAV titers, or other exclusionary comorbidities are unlikely to benefit or be eligible for this approach.
Why it matters
Potential benefit: If successful, a single infusion could provide long-term, steady production of a HER2-targeting engager, reducing the need for repeated dosing and potentially improving sustained tumor control.
How similar studies have performed: While bispecific T-cell engagers and AAV-based antibody-expression approaches have shown activity in other settings, delivering a secreted anti-HER2/anti-CD3 diabody via AAV is a novel, first-in-human strategy.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: * Age: ≥18 years of age * Histologically or cytologically confirmed diagnosis of HER-2 positive solid tumor as defined in the protocol * Part 1: presence of advanced or metastatic disease that has progressed during or following previous treatment * Part 2: Early stage HER-2 positive cancers with high risk for relapse following completion of SOC or after neoadjuvant systemic treatment * AAV specified capsid total antibody ≤1:400 * ECOG performance status of 0 or 1 * Life expectancy ≥3 months * Protocol-specified ranges for renal, liver, cardiac and pulmonary function * Protocol-specified ranges for hematology parameters Exclusion Criteria: * Hepatoxicity (AST or ALT \> 2x upper limit of normal) * Known active CNS or leptomeningeal disease * History of thrombotic microangiopathy or cardiomyopathy, or evidence of sensory neuropathy * Pregnant or nursing (lactating) women * History of other malignancy within 5 years prior to screening as defined in protocol * History of hypersensitivity to corticosteroids or history of corticosteroid-related toxicity Concurrent anti-cancer treatment in another investigational trial
Where this trial is running
Los Angeles, California and 4 other locations
- Valkyrie Clinical Trials — Los Angeles, California, United States (RECRUITING)
- SCRI Denver DDU at HealthOne — Denver, Colorado, United States (RECRUITING)
- Memorial Sloan Kettering Cancer Center — New York, New York, United States (RECRUITING)
- Thomas Jefferson University, Sidney Kimmel Cancer Center — Philadelphia, Pennsylvania, United States (RECRUITING)
- SCRI Oncology Partners — Nashville, Tennessee, United States (RECRUITING)
Study contacts
- Study coordinator: Allen Reha
- Email: allen.reha@vironexis.com
- Phone: 908-938-6019
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
Conditions: HER2 Expressing Solid Tumors, HER2+ breast cancer, HER2+ gastric cancer, Other HER+ cancer, HER2-low cancer