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OTX-TKI

Phase 3

Neovascular Age-related Macular Degeneration | Small molecule | Ophthalmology |Ocular Therapeutix, Inc.|Last Updated: Jun 17, 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
RandomizedDouble-BlindACTIVE_CONTROLLEDDMCBiomarker
Total Trials3
Total Enrollment1,198
FDA Designations
No designations recorded
Clinical Trials (3)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT06495918Study to Evaluate the Efficacy and Safety of Intravitreal OTX-TKI (Axitinib Implant) in Subjects With Neovascular Age- Related Macular DegenerationPHASE3 ACTIVE NOT_RECRUITING 825Nov 27, 2024Jan 8, 2027Jun 17, 202597 United States, Argentina +2
NCT06223958Study to Evaluate the Efficacy and Safety of Intravitreal OTX-TKI (Ocular Therapeutix) (Axitinib Implant) in Subjects With Neovascular Age-Related Macular DegenerationPHASE3 ACTIVE NOT_RECRUITING 344Jan 29, 2024Sep 27, 2027Apr 18, 2025108 United States, Argentina +1
NCT03630315CLN-0046: Treatment of AMD Subjects With OTX-TKIPHASE1 COMPLETED 29Feb 18, 2019Jan 10, 2024Jul 10, 20246 Australia
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Study Endpoints
Primary Endpoints
Best Corrected Visual Acuity (BCVA)
Week 48

Mean change in Best Corrected Visual Acuity (BCVA) from baseline at Week 48

Incidence of treatment emergent adverse events for each subject
9 months

All adverse events from screening through end of study will be captured

Secondary Endpoints
Determine the Maximum Tolerated Dose of the OTX-TKI injection
9 months
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Study Design & Arms
AllocationRANDOMIZED
MaskingQUADRUPLE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
OTX-TKI Re-doseEXPERIMENTAL -
Aflibercept 2mg on labelACTIVE_COMPARATOR -
Aflibercept 8mg high doseOTHER -
OTX-TKI (axitinib implant)EXPERIMENTAL -
ControlOTHER -
Cohort 1 (Low Dose)EXPERIMENTALSubjects will receive a low dose of OTX-TKI
Cohort 2 (Middle Dose)EXPERIMENTALSubjects will receive a middle dose of OTX-TKI.
Cohort 3 (High Dose)EXPERIMENTALSubjects will receive a high dose of OTX-TKI.
Cohort 3 (Anti-VEGF)EXPERIMENTALSubjects will receive OTX-TKI plus a single anti-VEGF injection
Cohort 4 (High Dose)EXPERIMENTALSubjects will receive a high dose of OTX-TKI.
Cohort 4 (Anti-VEGF)EXPERIMENTALSubjects will receive OTX-TKI plus a single anti-VEGF injection
Interventions
NameTypeDescription
OTX-TKIDRUGIntravitreal Injection of OTX-TKI
AfliberceptDRUGIntravitreal Injection of 2mg of aflibercept
OTX-TKI (axitinib implant)DRUGIntravitreal Injection of OTX-TKI (axitinib implant)
Anti-VEGFDRUGStandard of care therapy used to block vascular endothelial growth factor
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Eligibility Criteria
Age Range50 Years — N/A
SexALL
Healthy VolunteersNo
Study Sites97

Inclusion Criteria: * Treatment naïve juxtafoveal and/or SFNV secondary to Neovascular Age- Related Macular Degeneration (nAMD) with leakage involving the fovea in the study eye prior to Screening, or have a diagnosis of Neovascular Age- Related Macular Degeneration (nAMD) in study eye within 3 mon...

Countries:United StatesArgentinaAustraliaPuerto Rico
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Recent Changes (Last 90 Days)
LOWMay 26, 2026NCT06223958primaryCompletionDate: changed
LOWMay 26, 2026NCT06495918primaryCompletionDate: changed
LOWMay 24, 2026NCT06223958studyFirstPostDate: changed
LOWMay 24, 2026NCT06495918studyFirstPostDate: changed