Krystal Completes Phase 3 Trial of B-VEC, Topical Gene Therapy for DEB

Margarida Maia, PhD avatar

by Margarida Maia, PhD |

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Topline data should be available toward the end of this year for a Phase 3 clinical trial testing B-VEC (beremagene geperpavec), a topical gene therapy for treating skin wounds in patients with dystrophic epidermolysis bullosa (DEB).

Krystal Biotech, the therapy’s developer, announced that the last of the 31 participants in the study had completed the 26-week (six-month) dosing period and 30-day safety follow-up visit.

“We want to express our gratitude to the trial participants, their families, the clinical investigators and their coordinators for their participation and commitment that enabled us to complete this study in a timely manner during such a challenging year,” Suma Krishnan, chief operating officer of Krystal, said in a press release.

“We remain on track to announce topline data from the trial this quarter, and if positive, we will work expeditiously to complete regulatory filings with the FDA [U.S. Food and Drug Administration] and EMA [European  Medicines Agency] with the goal of bringing a topical, genetically corrective therapy to patients living with DEB,” Krishnan added.

B-VEC is designed to deliver a healthy copy of the COL7A1 gene to cells in the body using a harmless virus as a carrier. Mutations in this gene are the cause of DEB, as they lead to faulty production of collagen type VII, known as COL7, a protein that helps hold skin layers together.

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The healthy gene copy delivered with this therapy would allow cells to make enough COL7 protein to protect the skin from blistering and help close skin wounds. Such skin blisters and wounds are two hallmark symptoms of DEB.

The Phase 3 clinical trial, called GEM-3 (NCT04491604), opened in the U.S. in 2020 and was fully enrolled earlier this year. Its goal is to determine how effective and safe is B-VEC against a topical placebo on one or more skin wound pairs in patients with DEB.

Of note, the primary endpoint is complete would healing. One wound per each pair gets B-VEC while the other gets the placebo.

The 31 patients enrolled in GEM-3 ranged in age from 1 to 44, with 61% (19 patients) being 18 years or younger. A total of 29 patients completed the study and did not miss any of the weekly visits during the treatment period nor the safety follow-up visit, which was scheduled 30 days after the last treatment.

Those who returned for the safety follow-up visit were eligible to roll over into an open-label extension study (NCT04917874). That study is primarily looking at how safe and well-tolerated B-VEC is when given weekly over a period of 78 weeks (about 1.5 years).

The open-label extension study also can enroll patients who were unable to enter the Phase 3 GEM-3 clinical trial but met all enrollment criteria. The six study sites are located in five U.S. states. More information can be found here.