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HPV DNA Plasmids Therapeutic Vaccine VGX-3100

Phase 2

Anal Intraepithelial Neoplasia | Monoclonal antibody | Infectious Disease |Inovio Pharmaceuticals, Inc.|Last Updated: Apr 29, 2026

Success Probability
Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
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Market & Valuation
rNPV $3.2B
Market Size $9.4B
Revenue Basis $1.6B
Competitors 6
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Trial Design
UNCONTROLLEDDMCBiomarker
Total Trials1
Total Enrollment44
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT03603808VGX-3100 and Electroporation in Treating Patients With HIV-Positive High-Grade Anal LesionsPHASE2 COMPLETED 44Sep 21, 2018Jun 17, 2025Apr 29, 20268 United States, Puerto Rico
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Study Endpoints
Primary Endpoints
Overall Response Rate at 48 Weeks
At 48 weeks

Defined as histopathological regression of human papillomavirus (HPV)-16 and/or 18-positive anal high grade squamous intraepithelial neoplasia (HSIL) to low grade squamous intraepithelial neoplasia (LSIL) \[anal intraepithelial neoplasia (AIN)1\] or normal.

Secondary Endpoints
Safety and Tolerability of Treatment as Assessed by Incidence of Adverse Events
Up to 72 weeks
Complete Response Rate
At 48 weeks
Viral Clearance Histological Specimen
At 48 weeks
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Study Design & Arms
AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
Treatment (VGX-3100, electroporation)EXPERIMENTALPatients receive HPV DNA plasmids therapeutic vaccine VGX-3100 IM and then undergo electroporation over 10 seconds for 4 doses in week 0, 4, 12, and 24 in the absence of disease progression or unacceptable toxicity.
Interventions
NameTypeDescription
ElectroporationDEVICEUndergo electroporation
HPV DNA Plasmids Therapeutic Vaccine VGX-3100BIOLOGICALGiven IM
Laboratory Biomarker AnalysisOTHERCorrelative studies
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Eligibility Criteria
Age Range18 Years — N/A
SexALL
Healthy VolunteersNo
Study Sites8

Inclusion Criteria: * Biopsy-proven intra-anal or per-HSIL at baseline (anal intraepithelial neoplasia \[AIN\]2 with a positive p16 stain, PAIN2-3, AIN2-3, or PAIN3/AIN3) * At least one focus of HSIL must be large enough to be monitored for response, i.e., not completely removed after the screening...

Countries:United StatesPuerto Rico
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