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OPT-302

Phase 2

Neovascular Age-related Macular Degeneration | Monoclonal antibody | Ophthalmology |Opthea Limited|Last Updated: Apr 22, 2025

Success Probability

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Trial Design

RandomizedDouble-BlindSHAM_CONTROLLEDDMC
Total Trials1
Total Enrollment366

FDA Designations

No designations recorded

Clinical trial landscape

OPT-302 · 3 trials · 7 indications

Phase 2 1Phase 1 2
NCT03345082A Dose Ranging Study of OPT-302 With Ranibizumab in Neovascular (Wet) AMDNeovascular Age-related Macular Degeneration
COMPLETED366 Analytics
PHASE2COMPLETED
A Dose Ranging Study of OPT-302 With Ranibizumab in Neovascular (Wet) AMD
Neovascular Age-related Macular DegenerationUnlock trial analytics

Study Endpoints

Primary Endpoints

Mean change in Early Treatment Diabetic Retinopathy Study (ETDRS) best corrected visual acuity (BCVA)
Baseline to Week 24
Incidence of Treatment-Emergent Adverse Events (Safety and Tolerability)
Baseline to Week 12

Safety and Tolerability will be assessed using the National Cancer Institute Common Terminology Criteria for Adverse Events and CTC v4.0 (if available, otherwise protocol defined grading were used)

Phase 2a: Response Rate Defined as Proportion of Participants Receiving OPT-302 With Aflibercept Achieving at Least a 5-letter Gain in BCVA at Week 12
Baseline to Week 12

Change from baseline in Best Corrected Visual Acuity (BCVA) will be measured at Week 12 according to Early Treatment Diabetic Retinopathy Score (ETDRS) criteria

Safety (Adverse Events)
Up to 1 month after the last dose

Subject incidences of ophthalmic and systemic Adverse Events during study and follow-up period

Secondary Endpoints

Proportion of participants gaining 15 or more ETDRS BCVA letters
Baseline to Week 24
Area under the ETDRS BCVA over time curve
Baseline to Week 24
Change in central subfield thickness (CST) on spectral domain optical coherence tomography (SD-OCT)
Baseline to Week 24
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Study Design & Arms

AllocationRANDOMIZED
MaskingQUADRUPLE
ModelPARALLEL
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
0.5 mg ranibizumab with 2.0 mg OPT-302EXPERIMENTAL0.5 mg ranibizumab intravitreal injection (0.05 ml) followed by 2.0 mg OPT-302 intravitreal injection (0.05 ml)
0.5 mg ranibizumab with 0.5 mg OPT-302EXPERIMENTAL0.5 mg ranibizumab intravitreal injection (0.05 ml) followed by 0.5 mg OPT-302 intravitreal injection (0.05 ml)
0.5 mg ranibizumab with shamSHAM_COMPARATOR0.5 mg ranibizumab intravitreal injection (0.05 ml) followed by sham
Ph 1b: 2.0 mg aflibercept with 0.3 mg OPT-302EXPERIMENTAL2.0 mg aflibercept intravitreal injection (0.05 mL) followed by 0.3 mg OPT-302 intravitreal injection (0.05 mL)
Ph 1b: 2.0 mg aflibercept with 1.0 mg OPT-302EXPERIMENTAL2.0 mg aflibercept intravitreal injection (0.05 mL) followed by 1.0 mg OPT-302 intravitreal injection (0.05 mL)
Ph 1b: 2.0 mg aflibercept with 2.0 mg OPT-302EXPERIMENTAL2.0 mg aflibercept intravitreal injection (0.05 mL) followed by 2.0 mg OPT-302 intravitreal injection (0.05 mL)
Ph 2a: 2.0 mg aflibercept with 2.0 mg OPT-302EXPERIMENTAL2.0 mg aflibercept intravitreal injection (0.05 mL) followed by 2.0 mg OPT-302 intravitreal injection (0.05 mL)
Ph 2a: 2.0 mg aflibercept with shamSHAM_COMPARATOR2.0 mg aflibercept intravitreal injection (0.05 mL) followed by sham intravitreal injection
Part 1 Dose escalation - Cohort 1EXPERIMENTALDose Level 1 of OPT-302 in combination with Lucentis™
Part 1 Dose escalation - Cohort 2EXPERIMENTALDose Level 2 of OPT-302 in combination with Lucentis™
Part 1 Dose escalation - Cohort 3EXPERIMENTALDose Level 3 of OPT-302 in combination with Lucentis™
Part 1 Dose escalation - Cohort 4EXPERIMENTALDose Level 3 of OPT-302 monotherapy
Part 2 Dose expansion - Cohort 5EXPERIMENTALOPT-302 (at Maximum Tolerated Dose \[MTD\] or highest dose tested in Part 1) in combination with Lucentis™
Part 2 Dose expansion - Cohort 6EXPERIMENTALOPT-302 (at MTD or highest dose tested in Part 1) monotherapy

Interventions

NameTypeDescription
OPT-302BIOLOGICALIntravitreal injection
ranibizumabBIOLOGICALIntravitreal injection
sham intravitreal injectionOTHERSham (mock) intravitreal injection
AfliberceptBIOLOGICALIntravitreal injection
Lucentis™DRUGLucentis™ (0.5 mg) will be administered by intravitreal injection once every month for 3 months
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Eligibility Criteria

Age Range50 Years to N/A
SexALL
Healthy VolunteersNo
Study Sites108

Inclusion Criteria: * Active subfoveal choroidal neovascular (CNV) lesion or juxtafoveal lesion secondary to age-related macular AMD * An ETDRS BCVA score between 60 and 25 (inclusive) letters Exclusion Criteria: * Previous treatment for wet AMD or previous treatment for CNV due to other causes i...

Countries:United StatesCzechiaFranceHungaryIsraelItalyLatviaPolandSpainUnited KingdomAustralia
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Frequently asked questions about OPT-302

What is OPT-302?

OPT-302 is an investigational monoclonal antibody developed by Opthea Limited for retinal eye diseases. It has been studied in diabetic macular edema and neovascular age-related macular degeneration, where it is given in combination with standard anti-VEGF therapies such as aflibercept and ranibizumab. It remains in clinical development.

What is OPT-302 used for in diabetic macular edema?

OPT-302 has been studied in diabetic macular edema as an add-on to aflibercept in patients with persistent disease. A completed Phase 1 dose-ranging trial, NCT03397264, enrolled 153 patients across the United States, Australia, Israel and Latvia. OPT-302 is investigational and has not been approved for this indication.

Who makes OPT-302?

OPT-302 is developed by Opthea Limited, an ophthalmology-focused biopharmaceutical company whose shares trade under the ticker OPT. Opthea has run the clinical program for OPT-302, including dose-ranging studies in diabetic macular edema and neovascular age-related macular degeneration.

What phase is OPT-302 in?

OPT-302 is an investigational drug that has completed Phase 1 and Phase 2 clinical testing. Its clinical program includes a completed Phase 1 study in wet AMD, a completed Phase 1 dose-ranging study in diabetic macular edema, and a completed Phase 2 dose-ranging study in neovascular age-related macular degeneration. It has not been approved by the FDA.

What clinical trials is OPT-302 in?

Three completed trials have evaluated OPT-302: NCT02543229, a Phase 1 study of OPT-302 with or without Lucentis in wet AMD; NCT03397264, a Phase 1 dose-ranging study with aflibercept in persistent diabetic macular edema; and NCT03345082, a Phase 2 dose-ranging study with ranibizumab in neovascular age-related macular degeneration.

How does OPT-302 work?

OPT-302 is a monoclonal antibody designed to bind vascular endothelial growth factor C and D, blocking these VEGF family ligands. By inhibiting VEGF-C and VEGF-D alongside standard anti-VEGF-A therapy, it is intended to provide broader suppression of the angiogenic signaling that drives retinal vascular leakage and abnormal vessel growth in diseases such as diabetic macular edema and wet AMD.