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rAAV.sFlt-1

Phase 1

Macular Degeneration | Monoclonal antibody | Ophthalmology |Adverum Biotechnologies, Inc.|Last Updated: Sep 1, 2017

Success Probability
Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
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Market & Valuation
rNPV $3.2B
Market Size $9.4B
Revenue Basis $1.6B
Competitors 6
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Trial Design
RandomizedACTIVE_CONTROLLEDDMCBiomarker
Total Trials1
Total Enrollment40
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT01494805Safety and Efficacy Study of rAAV.sFlt-1 in Patients With Exudative Age-Related Macular DegenerationPHASE1 COMPLETED 40Dec 1, 2011Aug 1, 2017Sep 1, 20171 Australia
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Study Endpoints
Primary Endpoints
No sign of unresolved ophthalmic complications, toxicity or systemic complications as measured by laboratory tests from 1 month post injection
Primary endpoint at 1 month

1. Ocular examination: * Ocular inflammation * Intraocular pressure * Visual acuity * Retinal bleeding 2. Abnormal laboratory data

Secondary Endpoints
Maintenance or improvement of vision without the necessity of ranibizumab re-injections
Up to 3 years
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Study Design & Arms
AllocationRANDOMIZED
MaskingSINGLE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
Low Dose rAAV.sFlt-1EXPERIMENTAL -
High Dose rAAV.sFlt-1EXPERIMENTAL -
Control - ranibizumab onlyACTIVE_COMPARATOR -
Interventions
NameTypeDescription
rAAV.sFlt-1BIOLOGICAL1 x 10\^10 vector genomes (vg) rAAV.sFlt-1, delivered by subretinal injection
Control (ranibizumab alone)OTHERPatients will not receive rAAV.sFlt-1, but will be eligible for retreatment with ranibizumab (Lucentis).
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Eligibility Criteria
Age Range55 Years — N/A
SexALL
Healthy VolunteersNo
Study Sites1

Inclusion Criteria: * Age greater than or equal to 55 years; * Subfoveal CNV secondary to AMD and with best corrected visual acuity of 3/60 - 6/9 with 6/60 or better in the other eye; * Fluorescein angiogram of the study eye must show evidence of a leaking subfoveal choroidal neovascular lesion, or...

Countries:Australia
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