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beremagene geperpavec

Phase 3

Dystrophic Epidermolysis Bullosa | Monoclonal antibody | Dermatology |Krystal Biotech, Inc.|Last Updated: Apr 9, 2024

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
RandomizedDouble-BlindPLACEBO_CONTROLLEDDMCBiomarker
Total Trials3
Total Enrollment90
FDA Designations
No designations recorded
Clinical Trials (3)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT04917874A Long-term Treatment With B-VEC for Dystrophic Epidermolysis BullosaPHASE3 COMPLETED 47May 25, 2021Jul 31, 2023Apr 9, 20246 United States
NCT04491604Ph 3 Efficacy and Safety of B-VEC for the Treatment of DEBPHASE3 COMPLETED 31Aug 17, 2020Jan 14, 2022Feb 17, 20233 United States
NCT03536143A Phase I/II Study of KB103, a Topical HSV1-COL7, on DEB PatientsPHASE1 COMPLETED 12May 6, 2018Nov 1, 2019Jan 31, 20231 United States
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Study Endpoints
Primary Endpoints
To record safety outcomes of participants while on B-VEC.
Up to 112 weeks

Record reportable Adverse (AE) and Serious Adverse Events (SAEs) during the continued use of B-VEC to participants who have participated in and completed Krystal Biotech's Phase 3 Protocol (A Phase 3 Efficacy and Safety Study of Beremagene Geperpavec (B-VEC, previously KB103) for the Treatment of Dystrophic Epidermolysis Bullosa (DEB)), as an extension of use, upon study completion; as well as, for participants diagnosed with DEB who have not participated in the B-VEC Phase 3 trial.

Primary Wound With Complete Wound Healing (100% Wound Closure) on Weeks 22 and 24 or Weeks 24 and 26
26 weeks post-baseline

The primary wound was defined as a responder wound that met either of the following conditions: * Complete wound healing on Week 22 and Week 24, or * Complete wound healing on Week 24 and Week 26. For subjects with missing primary wound healing data, a multiple imputation approach (10 repliates) was used. The total numbers of primary wounds with complete healing for B-VEC and Placebo presented below were the average of those from the multiple imputation replicates, and therefore, they would not be whole numbers (integers).

Number of Subjects Reported at Least One Adverse Event, Safety Population
baseline to 12 weeks

Safety assessments included evaluation of medical and medication history, physical / skin examination, vital signs, adverse events, and laboratory evaluations. Due to the 'split-person' intrasubject design, the safety assessments were reported at subject level, but not per intervention.

Number of Adverse Events Reported, Safety Population
baseline to 12 weeks

Safety assessments included evaluation of medical and medication history, physical / skin examination, vital signs, adverse events, and laboratory evaluations. Due to the 'split-person' intrasubject design, the safety assessments were reported at subject level, but not per intervention.

Complete Wound Closure Responder, ITT Population
from baseline at Weeks 8, 10, and 12

One wound is a responder if the reduction from baseline in wound surface is ≥90%.

Time to Wound Closure Analysis, ITT Population
baseline to complete wound closure

Time to wound closure was defined as the time from the first treatment to Complete Wound Closure (≥90% reduction in wound surface area from baseline)

Duration of Wound Closure, ITT Population
Time from the complete closure to the first reopening of the same wound

Duration of wound closure

Secondary Endpoints
Primary Wound With Complete Wound Healing (100% Wound Closure) on Weeks 8 and 10 or Weeks 10 and 12
12 weeks post-baseline
Primary Wound Pain Severity (Visual Analog Scale (VAS)) Change for Ages 6 and Above Subjects at Weeks 22, 24, and 26.
26 weeks post-baseline
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Study Design & Arms
AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
B-VECEXPERIMENTALOpen label B-VEC topical treatment of DEB wounds.
PlaceboPLACEBO_COMPARATORMatching masked inactive topical gel
Topical beremagene geperpavecEXPERIMENTALHSV1-COL7A1 vector (KB103)
Interventions
NameTypeDescription
Open Label Topical Beremagene Geperpavec (B-VEC)BIOLOGICALTopical gel of non-integrating, replication-incompetent HSV-1 expressing the human collagen VII protein
Topical Beremagene GeperpavecBIOLOGICALTopical gel of non-integrating, replication-incompetent HSV-1 expressing the human collagen VII protein
PlaceboOTHERMatching masked inactive topical gel
Placebo gelBIOLOGICALPlacebo gel
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Eligibility Criteria
Age Range2 Months — N/A
SexALL
Healthy VolunteersNo
Study Sites6

Inclusion Criteria: * Willing and able to give consent/assent * Clinical diagnosis of epidermolysis bullosa * Confirmation of diagnosis (either DDEB or RDEB) by genetic testing including COL7A1. * Age: 2 months of age and older at the time of informed consent/assent * Women of childbearing age must...

Countries:United States
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