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AAV2-sFLT01

Phase 1

Macular Degeneration | Monoclonal antibody | Ophthalmology |Sanofi|Last Updated: Aug 22, 2018

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
CONTROLLEDDMCBiomarker
Total Trials1
Total Enrollment19
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT01024998Safety and Tolerability Study of AAV2-sFLT01 in Patients With Neovascular Age-Related Macular Degeneration (AMD)PHASE1 COMPLETED 19Jan 11, 2010Jul 1, 2018Aug 22, 20184 United States
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Study Endpoints
Primary Endpoints
Maximum tolerated dose of a single uniocular intravitreal injection of AAV2-sFLT01
Time of treatment through Week 52 (referred to as the "core" study)
Number of Treatment Emergent Adverse Events
Time of treatment through Week 52 (referred to as the "core" study)
Secondary Endpoints
Decreased retinal thickness
Time of treatment through Week 52 (referred to as the "core" study)
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Study Design & Arms
AllocationNON_RANDOMIZED
MaskingNONE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
2 x 10^8 vector genomes (vg) AAV2-sFLT01EXPERIMENTAL -
2 x 10^9 vector genomes (vg) AAV2-sFLT01EXPERIMENTAL -
6 x 10^9 vector genomes (vg) AAV2-sFLT01EXPERIMENTAL -
2 x 10^10 vector genomes (vg) AAV2-sFLT01EXPERIMENTAL -
Interventions
NameTypeDescription
AAV2-sFLT01BIOLOGICAL2 x 10\^8 vector genomes (vg) AAV2-sFLT01. Single intravitreal injection to a single eye, using a fixed volume of 100 μL.
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Eligibility Criteria
Age Range50 Years — N/A
SexALL
Healthy VolunteersNo
Study Sites4

Inclusion Criteria: * Choroidal neovascular membrane (CNV) secondary to AMD, as confirmed by the patient's medical history and a documented diagnosis of CNV. * Distance BCVA of 20/100 or worse in the study eye. * The fellow eye must have distance BCVA of 20/400 or better. * The study eye, i.e., the...

Countries:United States
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