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EB-101

Phase 3

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

Success Probability

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Market & Valuation

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Trial Design

UNCONTROLLEDDMC
Total Trials1
Total Enrollment11

FDA Designations

BREAKTHROUGH_THERAPYRMATORPHAN_DRUGRARE_PEDIATRIC_DISEASE

Clinical trial landscape

EB-101 · 1 trial · 2 indications

Phase 3 1
NCT04227106Phase 3, Open-label Clinical Trial of EB-101 for the Treatment of Recessive Dystrophic Epidermolysis Bullosa (RDEB)Epidermolysis Bullosa
COMPLETED11 Analytics
PHASE3COMPLETED
Phase 3, Open-label Clinical Trial of EB-101 for the Treatment of Recessive Dystrophic Epidermolysis Bullosa (RDEB)
Epidermolysis BullosaUnlock trial analytics

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 to 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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Frequently asked questions about EB-101

What is EB-101 used for?

EB-101 is an investigational therapy being developed for the treatment of recessive dystrophic epidermolysis bullosa (RDEB), a form of epidermolysis bullosa. It is a monoclonal antibody designed to address this rare genetic skin disorder. The drug is currently in clinical development and has not been approved by the FDA.

What does EB-101 target?

EB-101 is a monoclonal antibody, but its specific molecular target has not been disclosed in available information. The therapy is being studied for its potential to treat recessive dystrophic epidermolysis bullosa, a condition caused by defects in collagen production. Further details about its mechanism of action are not publicly available.

Who makes EB-101?

EB-101 is being developed by Abeona Therapeutics Inc., a biopharmaceutical company listed on the NASDAQ under the ticker symbol ABEO. The company is focused on developing gene and cell therapies for rare diseases, and EB-101 is one of its lead investigational candidates.

What phase is EB-101 in?

EB-101 is in Phase 3 clinical development. It has completed a Phase 3 trial, but it remains investigational and has not received FDA approval. The drug has been granted breakthrough therapy designation, regenerative medicine advanced therapy (RMAT) designation, orphan drug designation, and rare pediatric disease designation by the FDA.

What clinical trials is EB-101 in?

EB-101 has been studied in one clinical trial, identified as NCT04227106. This was a Phase 3, open-label, uncontrolled trial that enrolled 11 participants with recessive dystrophic epidermolysis bullosa. The trial was conducted in the United States and has been completed. No active trials are currently listed for EB-101.

Is EB-101 the same as other treatments for epidermolysis bullosa?

EB-101 is a distinct investigational therapy developed by Abeona Therapeutics. It is not known to be the same as any other approved or investigational treatment for epidermolysis bullosa. Its unique formulation and mechanism differentiate it from other potential therapies, though specific comparisons are not available in public data.