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OTX-TKI

Phase 3

Neovascular Age-related Macular Degeneration | Small molecule | Ophthalmology |Ocular Therapeutix, Inc.|Last Updated: Jul 1, 2026

Success Probability
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Market & Valuation
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Trial Design
RandomizedDouble-BlindACTIVE_CONTROLLEDDMC
Total Trials3
Total Enrollment1,198
FDA Designations
No designations recorded
Clinical trial landscape

OTX-TKI · 4 trials · 2 indications

Phase 3 3Phase 1 1
NCT07516132Study to Evaluate Long-Term Safety of Intravitreal OTX-TKI (Axitinib Implant) in Participants With Neovascular Age-Related Macular Degeneration Who Successfully Completed 2-Year OTX-TKI-2023-AMD-301 or OTX-TKI-2023-AMD-303 StudyNeovascular Age-Related Macular Degeneration (nAMD)
RECRUITING850 Analytics
NCT06495918Study to Evaluate the Efficacy and Safety of Intravitreal OTX-TKI (Axitinib Implant) in Subjects With Neovascular Age- Related Macular DegenerationNeovascular Age-related Macular Degeneration
ACTIVE NOT_RECRUITING825 Analytics
NCT06223958Study to Evaluate the Efficacy and Safety of Intravitreal OTX-TKI (Ocular Therapeutix) (Axitinib Implant) in Subjects With Neovascular Age-Related Macular DegenerationNeovascular Age-Related Macular Degeneration
ACTIVE NOT_RECRUITING344 Analytics
PHASE3RECRUITING
Study to Evaluate Long-Term Safety of Intravitreal OTX-TKI (Axitinib Implant) in Participants With Neovascular Age-Related Macular Degeneration Who Successfully Completed 2-Year OTX-TKI-2023-AMD-301 or OTX-TKI-2023-AMD-303 Study
Neovascular Age-Related Macular Degeneration (nAMD)Unlock trial analytics
PHASE3ACTIVE NOT_RECRUITING
Study to Evaluate the Efficacy and Safety of Intravitreal OTX-TKI (Axitinib Implant) in Subjects With Neovascular Age- Related Macular Degeneration
Neovascular Age-related Macular DegenerationUnlock trial analytics
PHASE3ACTIVE NOT_RECRUITING
Study to Evaluate the Efficacy and Safety of Intravitreal OTX-TKI (Ocular Therapeutix) (Axitinib Implant) in Subjects With Neovascular Age-Related Macular Degeneration
Neovascular Age-Related Macular DegenerationUnlock trial analytics
Study Endpoints
Primary Endpoints
Incidence and severity of treatment-emergent adverse events (TEAEs) from Baseline (Day 1) through Week 144
from Baseline (Day 1) through Week 144

Number of events

Proportion of participants with severe vision loss (defined as ≥ 30 Early Treatment Diabetic Retinopathy Study [ETDRS] letters loss) from Baseline (Day 1) through Week 144
from Baseline (Day 1) through Week 144

Percentage

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
AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
OTX-TKIEXPERIMENTAL -
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 to N/A
SexALL
Healthy VolunteersNo
Study Sites8

Inclusion Criteria: * Participants with a diagnosis of nAMD in the study eye who successfully completed Study OTX-TKI-2023-AMD-301 through Week 104 or Study OTX-TKI-2023-AMD-303 through Week 96 * Have adequate ocular media and adequate pupillary dilation in the study eye to permit good quality fund...

Countries:United StatesArgentinaAustraliaPuerto Rico
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Recent Changes (Last 90 Days)
LOWJul 2, 2026NCT07516132Status: NOT_YET_RECRUITING → RECRUITING
LOWJul 2, 2026NCT07516132Status: NOT_YET_RECRUITING → RECRUITING
LOWJul 2, 2026NCT07516132Status: NOT_YET_RECRUITING → RECRUITING
LOWJul 2, 2026NCT07516132Status: NOT_YET_RECRUITING → RECRUITING
LOWMay 26, 2026NCT07516132primaryCompletionDate: changed
LOWMay 26, 2026NCT06495918primaryCompletionDate: changed
LOWMay 26, 2026NCT06223958primaryCompletionDate: changed
LOWMay 24, 2026NCT07516132studyFirstPostDate: changed
LOWMay 24, 2026NCT06495918studyFirstPostDate: changed
LOWMay 24, 2026NCT06223958studyFirstPostDate: changed