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RGX-314

Phase 1

Neovascular Age-related Macular Degeneration | Gene therapy | Ophthalmology |REGENXBIO Inc.|Last Updated: May 16, 2023

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 Trials1
Total Enrollment42
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT03066258Safety and Tolerability of RGX-314 (Investigational Product) Gene Therapy for Neovascular AMD TrialPHASE1 COMPLETED 42Mar 29, 2017Jun 17, 2021May 16, 20238 United States
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Study Endpoints
Primary Endpoints
Safety (Participants With Ocular and Non-ocular AEs (Adverse Events) and SAEs (Serious Adverse Events))
26 weeks (24 weeks following RGX-314 administration)

Participants with ocular and non-ocular AEs and SAEs through 26 weeks (24 weeks following RGX-314 administration)

Secondary Endpoints
Safety (Participants With Ocular and Non-ocular AEs and SAEs)
106 weeks (104 weeks following RGX-314 administration)
Change From Baseline in BCVA (Best Corrected Visual Acuity)
106 weeks (104 weeks following RGX-314 administration)
Change From Baseline in CRT (Central Retinal Thickness)
106 weeks (104 weeks following RGX-314 administration)
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Study Design & Arms
AllocationNON_RANDOMIZED
MaskingNONE
ModelSEQUENTIAL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
Cohort 1EXPERIMENTAL3E9 GC (genome copies)/eye of RGX-314 (E means the exponential constant)
Cohort 2EXPERIMENTAL1E10 GC/eye of RGX-314
Cohort 3EXPERIMENTAL6E10 GC/eye of RGX-314
Cohort 4EXPERIMENTAL1.6E11 GC/eye of RGX-314
Cohort 5EXPERIMENTAL2.5E11 GC/eye of RGX-314
Interventions
NameTypeDescription
RGX-314GENETICRGX-314 is a recombinant adeno-associated virus (AAV) 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 Sites8

Inclusion Criteria: 1. Patients ≥ 50 and ≤ 89 years with a diagnosis of subfoveal CNV (Choroidal neovascularization) secondary to AMD in the study eye receiving prior intravitreal anti-VEGF therapy. 2. BCVA (Best Corrected Visual Acuity) between ≤20/63 and ≥20/400 (≤63 and ≥19 Early Treatment Diabe...

Countries:United States
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