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EB-101

Phase 3

Epidermolysis Bullosa | Monoclonal antibody | Dermatology |Abeona Therapeutics Inc.|Last Updated: Dec 5, 2022

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
UNCONTROLLEDDMCBiomarker
Total Trials1
Total Enrollment11
FDA Designations
BREAKTHROUGH_THERAPYRMATORPHAN_DRUGRARE_PEDIATRIC_DISEASE
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT04227106Phase 3, Open-label Clinical Trial of EB-101 for the Treatment of Recessive Dystrophic Epidermolysis Bullosa (RDEB)PHASE3 COMPLETED 11Jan 10, 2020Oct 18, 2022Dec 5, 20222 United States
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Study Endpoints
Primary Endpoints
Wound Healing
24 weeks post-treatment

Proportion of RDEB wound sites with ≥50% healing from Baseline in treated versus untreated wounds

Pain Reduction
24 weeks post-treatment

Associated with wound dressing change assessed by the mean differences in scores of the Wong-Baker FACES scale between treated and untreated wounds

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Study Design & Arms
AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
EB-101EXPERIMENTALOne-time surgical application of EB-101 on up to 6 chronic, RDEB wounds
Interventions
NameTypeDescription
EB-101BIOLOGICALautologous RDEB keratinocytes isolated from skin biopsies and transduced with a recombinant retrovirus containing a full-length COL7A1 expression cassette for C7
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Eligibility Criteria
Age Range6 Years — N/A
SexALL
Healthy VolunteersNo
Study Sites2

Inclusion Criteria: * Clinical diagnosis of RDEB; * Age 6 years or older, willing and able to give consent/assent; * If under the age of 18, guardian(s) is/are willing and able to give consent; * Positive expression of the non-collagenous region 1 of the type 7 collagen protein (NC1+) in the skin; ...

Countries:United States
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