Plymouth Meeting, Pennsylvania, USA – May 21, 2026
INOVIO Pharmaceuticals and its China-based partner ApolloBio announced positive topline results from a pivotal Phase 3 clinical trial evaluating VGX-3100, an investigational DNA immunotherapy designed to treat HPV-16/18-associated cervical dysplasia. The study successfully met its predefined primary efficacy endpoint while demonstrating a favorable safety and tolerability profile, marking a significant advancement for non-surgical treatment approaches targeting HPV-related precancerous diseases. The findings also strengthen the commercial and regulatory potential of INOVIO’s DNA medicine platform in one of the world’s largest healthcare markets.
Phase 3 Trial Demonstrates Strong Efficacy and Safety
The pivotal study conducted by ApolloBio was a multicenter, randomized, double-blind, placebo-controlled registrational Phase 3 trial involving women with high-grade squamous intraepithelial lesions (HSIL) caused by persistent HPV-16 and HPV-18 infection. The primary endpoint measured the composite response rate at Week 36, including both regression of cervical lesions and clearance of HPV infection.
According to the companies, the trial successfully achieved its primary efficacy endpoint and demonstrated a generally favorable safety profile without any significant new safety concerns. The positive data are expected to support a future regulatory submission in China for VGX-3100, potentially positioning the therapy as one of the first non-surgical therapeutic options for cervical dysplasia in the country.
The study was led by the Cancer Hospital of the Chinese Academy of Medical Sciences, with participation from 22 major tertiary hospitals across China. Researchers emphasized that the clinical findings represent important evidence supporting the broader use of DNA immunotherapies in HPV-related diseases, an area where treatment options remain limited.
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VGX-3100 Could Reduce Need for Surgical Interventions
VGX-3100 is an innovative therapeutic DNA vaccine designed to stimulate a targeted immune response capable of eliminating persistent HPV infection while promoting regression of precancerous cervical lesions. Unlike preventive HPV vaccines, VGX-3100 is intended for patients who are already infected and have developed disease associated with high-risk HPV strains.
The therapy specifically targets HPV types 16 and 18, which collectively account for approximately 70% of cervical cancer cases worldwide. Researchers believe the immunotherapy could provide an important alternative to conventional surgical treatments such as LEEP and conization, procedures commonly used to remove abnormal cervical tissue but associated with risks including miscarriage and preterm birth.
By potentially eliminating or reducing the need for invasive surgical procedures, VGX-3100 may offer significant reproductive health benefits for women of childbearing age. Analysts believe the therapy could address a major unmet medical need in women’s health, particularly in countries with high HPV prevalence and increasing cervical cancer screening programs.
ApolloBio is also exploring VGX-3100 in additional HPV-related high-grade precancerous conditions, including anal, vulvar, and vaginal lesions, potentially expanding the therapy’s long-term commercial opportunity.
DNA Medicine Platform Gains Momentum in Oncology and Infectious Disease
The latest clinical success further validates INOVIO’s proprietary DNA medicines platform, which combines specially designed DNA plasmids with its investigational CELLECTRA delivery technology. The platform is engineered to instruct the body’s cells to produce targeted proteins capable of triggering disease-specific immune responses without the need for viral vectors or lipid nanoparticles.
Company executives highlighted the topline results as evidence of the growing potential of DNA immunotherapies in both oncology and infectious disease treatment. Industry experts noted that therapeutic vaccines capable of directly treating persistent viral infections and associated precancerous diseases represent an emerging area of precision medicine with significant global healthcare relevance.
Cervical cancer remains the fourth most common cancer in women globally, with approximately 660,000 new cases and 350,000 deaths reported worldwide in 2022. Persistent HPV infection continues to be the primary cause of cervical cancer and cervical dysplasia, underscoring the urgent need for innovative treatment strategies beyond surgery and preventive vaccination alone.
If approved in China, VGX-3100 could become a major milestone for therapeutic HPV immunotherapy and further strengthen INOVIO’s position in the rapidly evolving DNA medicines market.
Source: INOVIO press release
