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OTX-TKI

Phase 3

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

Target and mechanism

Molecular targetVEGFR
ModalitySmall molecule

Also known as OTX-TKI (axitinib implant)

Success Probability

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Market & Valuation

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Trial Design

UNCONTROLLED
Total Trials1
Total Enrollment850

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

Frequently asked questions about OTX-TKI

What is OTX-TKI used for in neovascular age-related macular degeneration?

OTX-TKI is an investigational intravitreal axitinib implant being developed for the treatment of neovascular age-related macular degeneration (nAMD), a condition that causes abnormal blood vessel growth and leakage in the retina. It is designed to be injected into the eye to deliver sustained drug levels over time.

What does OTX-TKI target?

OTX-TKI contains axitinib, a small molecule that inhibits vascular endothelial growth factor (VEGF) receptors. By blocking these receptors, axitinib aims to reduce abnormal blood vessel growth and vascular permeability in the retina, which are key drivers of vision loss in neovascular age-related macular degeneration.

Who makes OTX-TKI?

OTX-TKI is being developed by Ocular Therapeutix, Inc., a biopharmaceutical company traded on NASDAQ under the ticker OCUL. The company is focused on ophthalmology and is advancing this intravitreal implant through clinical trials for neovascular age-related macular degeneration.

What phase is OTX-TKI in?

OTX-TKI is currently in Phase 3 clinical development for neovascular age-related macular degeneration. It is not yet approved by the FDA and remains investigational. Multiple Phase 3 trials are ongoing or planned to evaluate its efficacy and safety in patients with this condition.

What clinical trials is OTX-TKI in?

OTX-TKI has been studied in several trials. A Phase 1 study (NCT03630315) is completed. Two Phase 3 trials, NCT06223958 and NCT06495918, are active but not recruiting. A third Phase 3 trial, NCT07516132, is recruiting to evaluate long-term safety in participants who completed prior studies.

Is OTX-TKI the same as an axitinib implant?

Yes, OTX-TKI is also known as the axitinib implant. The drug name OTX-TKI refers to the intravitreal implant formulation of axitinib, a small molecule VEGF receptor inhibitor. This implant is designed for sustained release within the eye to treat neovascular age-related macular degeneration.