Gene therapy phase 3 clinical results achieved key results, 67% of patients' skin wounds healed completely

On November 29, 2021, Krystal Biotech announced that a pivotal phase 3 clinical trial of Vyjuvek (beremagene geperpavec, B-VEC), a topical topical gene therapy under investigation, has achieved positive top-line results

DEB is a rare and serious single-gene chronic genetic disease.

Different from the current standard treatment, Vyjuvek aims to treat DEB at the molecular level by providing a template for the production of normal COL7 protein in the skin cells of patients

▲ Mechanism of action of Vyjuvek (picture source: Krystal Biotech official website)

31 DEB patients were enrolled in this phase 3 clinical trial.

At 6 months of treatment, compared with placebo (22%), 67% of patients in the Vyjuvek group had wounds healed completely (p<0.

At 3 months of treatment, compared with placebo (20%), 71% of patients in the Vyjuvek group had their wounds healed completely (p<0.

Vyjuvek is well tolerated, and no serious adverse events related to the drug have been reported or the drug has been discontinued due to treatment

▲Efficacy results of Vyjuvek's Phase 3 clinical trial (picture source: Krystal official website)

Reference materials:

[1] Krystal Biotech Announces Positive Topline Results from GEM-3 Pivotal Trial of VYJUVEK™ in Patients with Dystrophic Epidermolysis Bullosa.

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