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RGX-314

Phase 2

Neovascular Age-related Macular Degeneration | Gene therapy | Ophthalmology |AbbVie Inc.|Last Updated: Sep 5, 2024

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
CONTROLLEDDMCBiomarker
Total Trials2
Total Enrollment925
FDA Designations
No designations recorded
Clinical Trials (2)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT04832724RGX-314 Gene Therapy Pharmacodynamic Study for Neovascular Age-related Macular Degeneration (nAMD)PHASE2 COMPLETED 60 — — Feb 22, 2021Mar 18, 2024Sep 5, 202413 United States
NCT03999801Long-term Follow-Up Study of RGX-314 and Fellow Eye SubstudyPHASE2 ENROLLING BY_INVITATION 865 — — May 31, 2019Dec 1, 2028Jun 17, 20249 United States
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Study Endpoints
Primary Endpoints
RGX-314 target protein concentration in aqueous humor
At Week 24
Incidence of ocular adverse events and any serious adverse events in the study eye
5 years inclusive of parent study
Fellow eye substudy: Incidence of ocular adverse events and any serious adverse events in the fellow eye
54 weeks
Secondary Endpoints
Incidence and severity of ocular Adverse Events (AEs) and overall AEs
Through Week 48
Changes in Best Corrected Visual Acuity (BCVA)
At Week 24 and 48
Changes in Central Retinal Thickness (CRT)
At Week 24 and 48
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Study Design & Arms
AllocationNON_RANDOMIZED
MaskingNONE
ModelSEQUENTIAL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
Commercial Formulation Dose 1EXPERIMENTALDose 1 of RGX-314
Clinical Formulation Dose 1EXPERIMENTALDose 1 of RGX-314
Commercial Formulation Dose 2EXPERIMENTALDose 2 of RGX-314
Clinical Formulation Dose 2EXPERIMENTALDose 2 of RGX-314
Main Observational StudyNO_INTERVENTIONAll subjects that previously received RGX-314 in a subretinal administration parent study are enrolled into this arm.
RGX-314 Fellow Eye Treatment SubstudyEXPERIMENTALRGX-314 Fellow Eye Treatment
Interventions
NameTypeDescription
RGX-314GENETICRGX-314 is a recombinant adeno-associated virus gene therapy vector carrying a coding sequence for a soluble anti-VEGF protein
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Eligibility Criteria
Age Range50 Years — 89 Years
SexALL
Healthy VolunteersNo
Study Sites13

Inclusion Criteria: 1. Males or females, aged ≥ 50 years and ≤ 89 years. 2. An Early Treatment Diabetic Retinopathy Study (ETDRS) BCVA-letter score between ≤ 78 and ≥ 40 in the study eye at Screening. 3. Diagnosis of choroidal neovascularization (CNV) secondary to age-related macular degeneration i...

Countries:United States
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Recent Changes (Last 90 Days)
LOWMay 26, 2026NCT03999801primaryCompletionDate: changed
LOWMay 24, 2026NCT03999801studyFirstPostDate: changed