Recent Updates
Recently added Catalysts

OCU410ST

Phase 2

Stargardt Disease | Small molecule | Other |Ocugen, Inc.|Last Updated: Apr 6, 2026

Success Probability
Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
Premium
Market & Valuation
rNPV $3.2B
Market Size $9.4B
Revenue Basis $1.6B
Competitors 6
Premium
Trial Design
RandomizedNO_TREATMENT_CONTROLLEDDMCBiomarker
Total Trials1
Total Enrollment51
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT05956626A Phase 2/3 Trial to Assess the Efficacy and Safety of OCU410ST for Stargardt DiseasePHASE2 ACTIVE NOT_RECRUITING 51Aug 25, 2023Sep 28, 2026Apr 6, 202614 United States
Unlock Drug Trial Details
Study Endpoints
Primary Endpoints
Change from baseline in atrophic lesion size as measured by Fundus Auto Fluorescence
12 months (Screening to 12 months post OCU410ST administration)

Change in the area of atrophy will be evaluated from the baseline measurements, using FAF to assess the loss of retinal layers.

Safety (Participants With Ocular and Non-ocular AEs (Adverse Events) and SAEs (Serious Adverse Events))
12 months (Screening to 12 months post OCU410ST administration)

Safety will be determined by the number of ocular and non-ocular Study Drug-related adverse events (SDAE), treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs).

Secondary Endpoints
Change from baseline in Low Luminance Visual Acuity (LLVA)
12 months (Screening to 12 months post OCU410ST administration)
Change from baseline in Best Corrected Visual Acuity (BCVA)
12 months (Screening to 12 months post OCU410ST administration)
Unlock Study Endpoints
Study Design & Arms
AllocationRANDOMIZED
MaskingSINGLE
ModelSEQUENTIAL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
Phase 2/3 Randomized Treatment ArmEXPERIMENTALSubjects will receive a single subretinal injection of 200uL OCU410ST in concentration of 1.5 x 10E11vg/mL
Phase 2/3 Randomized Control ArmNO_INTERVENTIONSubjects will not receive any active study intervention
Interventions
NameTypeDescription
OCU410STDRUGSubretinal Administration of OCU410ST
Unlock Study Design Details
Eligibility Criteria
Age Range5 Years — N/A
SexALL
Healthy VolunteersNo
Study Sites14

Phase 2/3 Inclusion Criteria (applicable for both adult and pediatric subjects): 1. Males or females aged ≥5 years at the time of consent. 2. Subjects who have confirmed clinical and CLIA certified or equivalent genetic diagnosis of Stargardt disease (including ABCA4 related retinopathies). 3. Adul...

Countries:United States
Unlock Eligibility Criteria
Recent Changes (Last 90 Days)
MEDIUMMay 26, 2026NCT05956626Status: RECRUITING → ACTIVE_NOT_RECRUITING
LOWMay 24, 2026NCT05956626studyFirstPostDate: changed