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GEM103

Phase 1

Dry Age-related Macular Degeneration | Monoclonal antibody | Ophthalmology |Disc Medicine, Inc.|Last Updated: Feb 11, 2021

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
CONTROLLEDBiomarker
Total Trials1
Total Enrollment12
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT04246866First in Human Study to Evaluate the Safety and Tolerability of GEM103 in Geographic Atrophy Secondary to Dry Age Related Macular DegenerationPHASE1 COMPLETED 12Dec 19, 2019Oct 21, 2020Feb 11, 202112 United States
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Study Endpoints
Primary Endpoints
Incidence and severity of AEs/SAEs following drug administration
Up to 8 Weeks
Secondary Endpoints
GEM103 concentrations in ocular fluids
Up to 8 Weeks
Changes in complement factor levels compared to baseline after drug administration
Up to 8 Weeks
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Study Design & Arms
AllocationNON_RANDOMIZED
MaskingNONE
ModelSEQUENTIAL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
Dose 1EXPERIMENTALA single dose of GEM103 will be administered via intravitreal injection. This arm will be the lowest dose of GEM103
Dose 2EXPERIMENTALA single dose of GEM103 will be administered via intravitreal injection. This arm will be the medium dose of GEM103
Dose 3EXPERIMENTALA single dose of GEM103 will be administered via intravitreal injection. This arm will be a higher dose of GEM103
Dose 4EXPERIMENTALA single dose of GEM103 will be administered via intravitreal injection. This arm will be the highest (extension) dose of GEM103
Interventions
NameTypeDescription
GEM103BIOLOGICALGEM103
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Eligibility Criteria
Age Range50 Years — N/A
SexALL
Healthy VolunteersNo
Study Sites12

Inclusion Criteria: 1. At least 50 years old at the time of signed informed consent 2. Able to cooperate sufficiently for adequate ophthalmic visual function testing and anatomic assessment and provide informed consent prior to initiation of any study procedure 3. Best corrected visual acuity (BCVA...

Countries:United States
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