Topical Gene Therapy B-VEC Reaches Phase 3 Efficacy Endpoints for DEB Disease Reversal


New AAD 2022 data show the novel gene therapy from Krystal Biotech provided wound healing by 3 months in patients with dystrophic epidermolysis bullosa.

A topically-applied gene therapy has reported positive phase 3 outcomes and may soon become available for patients with a rare dermatologic condition.

In a late-breaking session at the American Academy of Dermatology (AAD) 2022 Annual Meeting this weekend, investigators reported that beremagen geperparvec (B-VEC) met the primary and secondary endpoints of the multi center, randomized, double-blind, placebo-controlled intra-patient GEM-3 trial sponsored by Krystal Biotech.

The findings, presented by primary investigator Peter Marinkovich, MD, associate professor of dermatology at Stanford University School of Medicine, showed a significantly greater rate of patients treated with B-VEC achieved complete wound healing from dystrophic epidermolysis bullosa (DEB) than placebo at both 3 and 6 months (P <.005).

While an open-label extension trial including GEM-3’s 31 participants is ongoing, investigators are now anticipating the novel topical gene therapy could reach the US market.

In an interview with HCPLive during AAD 2022, Marinkovich discussed the pharmacokinetic strategy of B-VEC as it treats the rare condition DEB—correcting molecular defects to promote wound healing, while more importantly retaining and even strengthening the affected cells.

As he explained, it’s the gene therapy’s simplistic application added with the newly established efficacy that gives it an opportunity to shortly begin reversing disease in eligible patients.

“It may be the case that more dermatologists will be seeing these patients, because the therapy is such an easy therapy to use,” Marinkovich said. “You use it at an outpatient basis, you can just use it in the clinic to treat these patients’ wounds. I think it’s really a cutting-edge type of therapy that, if everything goes well, may be approved in a year or so.”

Listen to Marinkovich’s full interview—including his perspective on the state of DEB therapies, the long-term extension trial of GEM-3, and other potential topical gene therapy uses in dermatology—in the video above.

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