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RGX-314 Dose 1

Phase 2

Diabetic Macular Edema | Gene therapy | Other |REGENXBIO Inc.|Last Updated: Apr 24, 2025

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
RandomizedACTIVE_CONTROLLEDBiomarker
Total Trials1
Total Enrollment18
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT06942520Phase 2 Trial of RGX-314 in Adults With Center Involved - Diabetic Macular Edema (CI - DME)PHASE2 RECRUITING 18Mar 18, 2025Dec 1, 2027Apr 24, 20251 United States
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Study Endpoints
Primary Endpoints
Mean change from baseline in BCVA in the study eye at Week 52
52 Weeks

To evaluate the effect of RGX-314 on BCVA at Week 52

Secondary Endpoints
Mean change from baseline in BCVA in the study eye over time
52 Weeks
Proportion of participants with BCVA of 20/40 or better (ETDRS ≥ 69 letters) in the study eye over time
52 Weeks
Mean change from baseline in central subfield thickness (CST) in the study at week 52
52 Weeks
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Study Design & Arms
AllocationRANDOMIZED
MaskingNONE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
RGX-314 Treatment Arm (Dose 1)EXPERIMENTALGenetic: RGX-314 Dose 1 \- AAV8 vector containing a transgene for anti-VEGF fab (Dose 1)
Treatment Arm (Dose 2)EXPERIMENTALGenetic: RGX-314 Dose 2 -AAV8 vector containing a transgene for anti-VEGF fab (Dose 2)
Aflibercept Treatment ArmACTIVE_COMPARATORBiological: Aflibercept IVT * Commercially available Active Comparator * Other Names: Eylea
Interventions
NameTypeDescription
RGX-314 Dose 1GENETICAAV8 vector containing a transgene for anti-VEGF fab (Dose 1)
RGX-314 Dose 2GENETICAAV8 vector containing a transgene for anti-VEGF fab (Dose 2)
Aflibercept (2.0 mg)BIOLOGICALCommercially available Active Comparator
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Eligibility Criteria
Age Range25 Years — 89 Years
SexALL
Healthy VolunteersNo
Study Sites1

Inclusion Criteria: * Type I or Type II diabetes mellitus with center involving diabetic macular edema with previous treatment * HbA1c ≤12% * BCVA in study eye 78 to 25 ETDRS letters (\~20/32 to 20/320) at screening * Decreased visual acuity attributable primarily due to CI - DME * Demonstrate clin...

Countries:United States
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Recent Changes (Last 90 Days)
LOWMay 26, 2026NCT06942520primaryCompletionDate: changed
LOWMay 24, 2026NCT06942520studyFirstPostDate: changed