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AU-011

Phase 2

Eye Cancer | Small molecule | Oncology |Aura Biosciences, Inc.|Last Updated: Aug 6, 2026

Development status

Highest phase Phase 2
Registered trials 4 across 1 sponsor since Jun 2020

Target and mechanism

Molecular targetheparan sulfate proteoglycans
ModalitySmall molecule

Success Probability

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Market & Valuation

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Trial Design

NO_TREATMENT_CONTROLLEDDMC
Total Trials1
Total Enrollment24

FDA Designations

No designations recorded

Clinical trial landscape

AU-011 · 3 trials · 11 indications

Phase 2 2Phase 1 1
NCT06643884Suprachoroidal Administration in Subjects With Metastases to the ChoroidEye Cancer
RECRUITING24 Analytics
NCT04417530Phase 2 Trial to Evaluate Belzupacap Sarotalocan (AU-011) Via Suprachoroidal Administration in Subjects With Primary Indeterminate Lesions and Small Choroidal MelanomaUveal Melanoma
COMPLETED22 Analytics
PHASE2RECRUITING
Suprachoroidal Administration in Subjects With Metastases to the Choroid
Eye CancerUnlock trial analytics
PHASE2COMPLETED
Phase 2 Trial to Evaluate Belzupacap Sarotalocan (AU-011) Via Suprachoroidal Administration in Subjects With Primary Indeterminate Lesions and Small Choroidal Melanoma
Uveal MelanomaUnlock trial analytics

Study Endpoints

Primary Endpoints

Change from baseline in choroidal tumor thickness on B-scan ultrasonography (B-scan) 4 weeks after completion of treatment.
4 weeks after completion of treatment

B-Scan Ultrasonography

Change from baseline in choroidal tumor largest basal diameter (LBD) on fundus photos 4 weeks after completion of treatment.
4 weeks after completion of treatment

Fundus photos

Treatment related AEs and treatment related serious adverse events (SAEs).
52 weeks

Adverse Events

Safety of AU-011: Incidences of SAEs and DLTs
up to 12 months

Incidence and severity of treatment-related adverse events \[time frame 12 months\], serious adverse events \[time frame 12 months\], and incidence of dose-limiting toxicities \[time frame 14 days\]

Secondary Endpoints

Within-subject difference of historical tumor thickness growth rate and post-treatment growth rate over 52 weeks.
52 weeks
Time to reach tumor progression
52 weeks
Complete response (CR) rate
at the 3-month time point and at TURBT
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Study Design & Arms

AllocationNON_RANDOMIZED
MaskingNONE
ModelSEQUENTIAL
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
80 micrograms of bel-sarEXPERIMENTAL1 Cycle AU-011 with a dose of 80 micrograms
140 micrograms of bel-sarEXPERIMENTAL1 Cycle AU-011 with a dose of 140 micrograms
200 micrograms of bel-sar with one cycleEXPERIMENTAL1 Cycle AU-011 with a dose of 200 micrograms
200 micrograms of bel-sar with two cyclesEXPERIMENTAL2 Cycles AU-011 with a dose of 200 micrograms
ObservationalNO_INTERVENTIONSubjects that meet trial inclusion, but do not meet criteria for enrollment into the bel-sar treatment cohorts, may be enrolled in this cohort. The observational cohort will not receive bel-sar treatment.
40 micrograms of bel-sar with one cycleEXPERIMENTAL1 Cycle AU-011 with a dose of 40 micrograms
40 micrograms of bel-sar with two cyclesEXPERIMENTAL2 Cycles AU-011 with a dose of 40 micrograms
Cohort 1 AU-011& LaserEXPERIMENTALLow dose of AU-011 + 1 laser application
Cohort 2 AU-011 & LaserEXPERIMENTALMedium dose of AU-011 + 1 laser application
Cohort 3 AU-011 (belzupacap sarotalocan) & LaserEXPERIMENTALMedium dose of AU-011 + 2 laser applications
Cohort 4 AU-011 & LaserEXPERIMENTALHighest tolerated dose of AU-011 / laser applications from Cohorts 1 to 3 administered weekly for 2 treatments
Cohort 5 AU-011 & LaserEXPERIMENTALAU-011/laser applications from Cohorts 1 to 3 administered weekly for 3 treatments. Up to 2 cycles of this regimen may be administered and subjects have the option of receiving a third cycle of treatment.
Cohort 6 AU-011 & LaserEXPERIMENTALHigh dose of AU-011/laser applications administered weekly for 3 treatments and up to 3 cycles of treatment.
Focal injections of AU-011 prior to TURBT (1b)EXPERIMENTALIntratumoral (50 μg) and intramural (50 μg) injection of AU-011 prior to the standard of care (TURBT) in patients with NMIBC.
Focal injections of AU-011 with laser application before TURBT (4a)EXPERIMENTALIntratumoral (50 μg) and intramural (50 μg) injection of AU-011 with laser application before standard of care (TURBT) in patients with NMIBC.
Focal injection of AU-011 with laser application before TURBT (4b)EXPERIMENTALFocal injection of AU-011 (100 μg) with laser application before standard of care (TURBT) in patients with NMIBC.
Focal injection of AU-011 with laser application before TURBT (4c)EXPERIMENTALFocal injection of AU-011 (200 μg) with laser application before standard of care (TURBT) in patients with NMIBC.
Focal injection of AU-011 and laser application with option for TURBT (4d)EXPERIMENTALParticipants with intermediate-risk NMIBC will receive multiple treatment cycles of focal AU-011 (200 ug per lesion) with laser treatment and option for TURBT in patients with intermediate risk NMIBC. Participants will be evaluated for safety and response to AU-011 up to 12 months from initiation of treatment.
Focal injection of AU-011 and laser application with option for TURBT (4e)EXPERIMENTALParticipants with intermediate-risk NMIBC will receive multiple treatment cycles of focal AU-011 (400 µg per lesion) followed by laser activation, with the option for TURBT. Participants will be evaluated for safety and response to AU-011 for up to 12 months from initiation of treatment. Enrollment will occur in parallel with Cohort 4g, with randomized (1:1) assignment.
Focal injection of AU-011 and laser application with mandatory TURBT (4f)EXPERIMENTALParticipants with high-risk NMIBC will receive multiple treatment cycles of focal AU-011 (400 µg per lesion) followed by laser activation prior to mandatory TURBT. Participants will be evaluated for safety and response to AU-011 for up to 12 months from initiation of treatment.
Focal injection of AU-011 and laser application with optional TURBT (4g)EXPERIMENTALParticipants with intermediate-risk NMIBC will receive multiple treatment cycles of focal AU-011 (400 µg per lesion) followed by laser activation with the option of TURBT. Participants will be evaluated for safety and response to AU-011 for up to 12 months from initiation of treatment. Enrollment will occur in parallel with Cohort 4e, with randomized (1:1) assignment.
Focal injection of AU-011 and laser application with option for TURBT (4h)EXPERIMENTALParticipants with intermediate-risk NMIBC will receive multiple treatment cycles of focal AU-011 (800 ug per lesion) with laser treatment and option for TURBT. Participants will be evaluated for safety and response to AU-011 up to 12 months from initiation of treatment. This arm is optional and will be run at the discretion of the sponsor.
Focal injection of AU-011 and laser application with mandatory TURBT (4i)EXPERIMENTALParticipants with high-risk NMIBC will receive multiple treatment cycles of focal AU-011 (800 µg per lesion) before mandatory TURBT. Participants will be evaluated for safety and response to AU-011 up to 12 months from initiation of treatment. This arm is optional and will be run at the discretion of the sponsor.

Interventions

NameTypeDescription
AU-011DRUGAU-011 Via Suprachoroidal Administration with laser treatment
SCS MicroinjectorDEVICESuprachoroidal Injection Device
LaserDEVICELaser Administration
Suprachoroidal MicroinjectorDEVICESuprachoroidal Injection Device
PDT LaserDEVICELaser Administration
AU-011 in Combination with Medical Laser AdminstrationCOMBINATION_PRODUCTAU-011 Intratumorally and Intramurally
AU-011 in Combination with Medical Laser AdministrationCOMBINATION_PRODUCTAU-011 Intratumorally
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Eligibility Criteria

Age Range18 Years to N/A
SexALL
Healthy VolunteersNo
Study Sites10

Inclusion Criteria: * Have a clinical diagnosis of Metastases to the Choroid, from a histopathologically or cytologically confirmed tumor. * Have at least one Metastases to the Choroid in the study eye Exclusion Criteria: * Active ocular infection or disease. * Must not have evidence of a primary...

Countries:United StatesAustralia
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Competitive Landscape -Anal Cancer 6 trials (matched to "Eye Cancer")

CompanyTickerTrialsLead PhaseDrugs
Incyte CorporationINCY1PHASE2Retifanlimab
Pfizer Inc.PFE1PHASE1Avelumab
Iovance Biotherapeutics IncIOVA2PHASE2Aldesleukin
Oncolytics Biotech Inc.ONCY1PHASE1Pelareorep
TScan Therapeutics, Inc.TCRX1PHASE1TSC-204-A0201, TSC-204-C0702, TSC-200-A0201, TSC-203-A0201, TSC-204-A0101
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Recent Changes (Last 90 Days)

MEDIUMAug 6, 2026NCT05483868Status: RECRUITING → ACTIVE_NOT_RECRUITING
MEDIUMAug 6, 2026NCT05483868Status: RECRUITING → ACTIVE_NOT_RECRUITING

Frequently asked questions about AU-011

What is AU-011 used for?

AU-011, also known as belzupacap sarotalocan, is an investigational small molecule being studied for eye cancer, non-muscle-invasive bladder cancer, and uveal melanoma. It is being developed by Aura Biosciences, Inc. (NASDAQ: AURA) and is currently in Phase 2 clinical development for these oncology indications.

What does AU-011 target?

AU-011 targets heparan sulfate proteoglycans, which are molecules found on the surface of cells. By binding to these proteoglycans, the drug is designed to deliver a therapeutic effect specifically to cancer cells. This mechanism is being investigated in clinical trials for eye cancer and bladder cancer.

Who makes AU-011?

AU-011 is being developed by Aura Biosciences, Inc., a biopharmaceutical company traded on NASDAQ under the ticker symbol AURA. The company is conducting clinical trials to evaluate the drug's safety and efficacy in patients with various cancers, including uveal melanoma and non-muscle-invasive bladder cancer.

What phase is AU-011 in?

AU-011 is in Phase 2 clinical development. It is an investigational drug and has not been approved by the FDA. Clinical trials are ongoing to assess its use in eye cancer and non-muscle-invasive bladder cancer, with a completed Phase 2 trial in uveal melanoma.

What clinical trials is AU-011 in?

AU-011 is being studied in several clinical trials. NCT06643884 is a Phase 2 trial for metastases to the choroid, NCT05483868 is a Phase 1 trial for non-muscle-invasive bladder cancer, and NCT04417530 is a completed Phase 2 trial for primary indeterminate lesions and small choroidal melanoma.

Is AU-011 the same as belzupacap sarotalocan?

Yes, AU-011 is also known as belzupacap sarotalocan. Both names refer to the same investigational drug being developed by Aura Biosciences. In clinical trial records, the drug is often listed under its alternative name, belzupacap sarotalocan, while AU-011 is the code name used in research.