Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
beremagene geperpavec · 3 trials · 4 indications
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.
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).
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.
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.
One wound is a responder if the reduction from baseline in wound surface is ≥90%.
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
| Arm | Type | Description |
|---|---|---|
| B-VEC | EXPERIMENTAL | Open label B-VEC topical treatment of DEB wounds. |
| Placebo | PLACEBO_COMPARATOR | Matching masked inactive topical gel |
| Topical beremagene geperpavec | EXPERIMENTAL | HSV1-COL7A1 vector (KB103) |
| Name | Type | Description |
|---|---|---|
| Open Label Topical Beremagene Geperpavec (B-VEC) | BIOLOGICAL | Topical gel of non-integrating, replication-incompetent HSV-1 expressing the human collagen VII protein |
| Topical Beremagene Geperpavec | BIOLOGICAL | Topical gel of non-integrating, replication-incompetent HSV-1 expressing the human collagen VII protein |
| Placebo | OTHER | Matching masked inactive topical gel |
| Placebo gel | BIOLOGICAL | Placebo gel |
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...
Beremagene geperpavec is an investigational therapy for Dystrophic Epidermolysis Bullosa (DEB), a rare genetic skin disorder. It is being developed as a topical treatment for this condition. The drug is currently in Phase 3 clinical development and has not been approved by the FDA.
Beremagene geperpavec is a topical gene therapy that delivers a functional copy of the COL7A1 gene to skin cells. It is designed to restore production of type VII collagen, which is deficient in patients with Dystrophic Epidermolysis Bullosa. The drug is administered topically to wounds.
Beremagene geperpavec is being developed by Krystal Biotech, Inc., a biopharmaceutical company traded on the NASDAQ under the ticker symbol KRYS. The company is focused on developing gene therapies for rare diseases, with beremagene geperpavec being one of its lead product candidates.
Beremagene geperpavec is in Phase 3 clinical development for Dystrophic Epidermolysis Bullosa. It has completed three clinical trials, including two Phase 3 studies and one Phase 1/2 study. The drug is investigational and has not received FDA approval.
Beremagene geperpavec has completed three clinical trials: NCT03536143, a Phase 1/2 study in 12 patients; NCT04491604, a Phase 3 efficacy and safety study in 31 patients; and NCT04917874, a Phase 3 long-term treatment study in 47 patients. All trials were conducted in the United States.
Yes, beremagene geperpavec is also known as B-VEC. Clinical trial records refer to the drug as B-VEC, including the Phase 3 studies NCT04491604 and NCT04917874. The drug is being developed by Krystal Biotech for Dystrophic Epidermolysis Bullosa.