Krystal Biotech has reported that the GEM-3 clinical trial of its experimental gene therapy, beremagene geperpavec (B-VEC), for treating dystrophic epidermolysis bullosa (dystrophic EB) met the primary goal.

Presently called VYJUVEK, B-VEC is a non-invasive topical gene therapy that can offer two copies of the COL7A1 gene when applied to DEB wounds.

It can potentially treat DEB at the molecular level by offering a template to the skin cells of the patient to produce normal COL7 protein to address the basic disease-causing process.

The double-blind, randomised, intra-patient, placebo-controlled trial analysed the safety and efficacy of VYJUVEK to treat dystrophic EB.

A total of 31 subjects aged one to 44 years were enrolled at three study centres.

In the trial, a primary wound pair was detected in each patient of which one was treated with weekly topical VYJUVEK while the other received a placebo.

The trial’s primary goal met statistical significance at six-month timepoints with subjects experiencing full wound healing on treatment with topical VYJUVEK versus placebo.

Findings showed that 67% of wounds in the VYJUVEK arm had complete wound healing at the six-month timepoints as against 22% of wounds treated with placebo.

At the three-month timepoints, 71% of wounds that received VYJUVEK treatment met the secondary goal of investigator evaluated full wound healing versus 20% for the placebo arm.

Furthermore, VYJUVEK was found to be well-tolerated without any treatment-linked serious side effects events or discontinuations noted in the trial.

VYJUVEK’s immunogenicity profile was in line with the previous GEM-1/2 trial where no significant variation in anti-HSV-1 or anti-COL7 antibodies were reported.

Krystal Biotech founder and chief operating officer Suma Krishnan said: “Dystrophic epidermolysis bullosa is referred to as ‘the worst disease you’ve never heard of’, because of the incredibly devastating reality that patients with this genetic condition face, and we are thrilled to announce positive results from our pivotal GEM-3 trial of VYJUVEK, which showed that this topical gene therapy led to durable wound healing in dystrophic EB wounds.

“With these results in hand, we look forward to advancing discussions with regulatory authorities and will work quickly to bring this potential first-ever treatment to patients with dystrophic EB and their families who are in desperate need.”

Based on the latest data, the company plans to submit a Biologics License Application (BLA) to the US Food and Drug Administration (FDA) in the first half of next year.

A rare and severe monogenic ailment, dystrophic EB impacts the skin and mucosal tissues.

In March 2018, Krystal filed an IND application with the FDA to commence a Phase I/II trial of its gene therapy for dystrophic EB.

Cell & Gene Therapy Coverage on Clinical Trials Arena supported by Cytiva.

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