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APX3330

Phase 2

Diabetic Retinopathy | Small molecule | Ophthalmology |Opus Genetics, Inc.|Last Updated: Feb 27, 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
RandomizedDouble-BlindPLACEBO_CONTROLLEDDMCBiomarker
Total Trials1
Total Enrollment103
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT04692688Study of the Safety and Efficacy of APX3330 in Diabetic RetinopathyPHASE2 COMPLETED 103Apr 8, 2021Jan 25, 2023Feb 27, 202324 United States
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Study Endpoints
Primary Endpoints
Percent of Subjects with an improvement in Diabetic Retinopathy Severity Score (DRSS)
24 Weeks

Percent of subjects with a ≥ 2-step improvement in DRSS in the study eye

Secondary Endpoints
Percent of Subjects with change in Diabetic Retinopathy Severity Scale (DRSS) Scores
Up to 24 Weeks
Mean Change in Diabetic Retinopathy Severity Scale (DRSS) Score
24 Weeks
Percent of Subjects without DR/DME Disease Progression
24 Weeks
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Study Design & Arms
AllocationRANDOMIZED
MaskingQUADRUPLE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
APX3330EXPERIMENTALFive 120 mg tablets will be taken by mouth as follows: 3 tablets every morning and 2 tablets every evening.
PlaceboPLACEBO_COMPARATORFive 120 mg tablets will be taken by mouth as follows: 3 tablets every morning and 2 tablets every evening.
Interventions
NameTypeDescription
APX3330DRUGAPX3330, a small-molecule oral tablet, is a Ref-1 inhibitor that can potentially reduce proinflammatory and hypoxic signaling that contributes to several eye diseases.
PlaceboDRUGPlacebo tablets are identical to APX3330 tablets except for the absence of the active pharmaceutical ingredient.
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Eligibility Criteria
Age Range18 Years — N/A
SexALL
Healthy VolunteersYes
Study Sites24

Inclusion Criteria: 1. Males or non-pregnant females ≥ 18 years of age 2. At least one eye with DR graded at least moderately severe to severe NPDR or mild PDR (corresponding to DRSS 47, 53, or 61) 3. BCVA assessed by ETDRS protocol letters score of ≥ 60 letters (Snellen equivalent ≥ 20/63) 4. Body...

Countries:United States
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