Krystal Biotech announces data on topical application of B-VEC

Krystal Biotech announced that the Company presented new data on the compassionate use of topical beremagene geperpavec, B-VEC, to treat a patient with dystrophic epidermolysis bullosa, DEB, with recurrent cicatrizing conjunctivitis at the Association for Research in Vision and Ophthalmology, ARVO, 2023 Annual Meeting on April 23, 2023. The data presented describes the first application of B-VEC to treat ocular complications in a patient with DEB under a compassionate use program. B-VEC was well tolerated and associated with full corneal healing by 3 months as well as significant visual acuity improvement from hand motion to 20/40 at 7 months, the latest time point of the on-going treatment effect evaluation. No drug-related adverse events have been observed. Two non drug related, serious AEs were reported: 1) Prolonged hospitalization due to complications post-gastrointestinal surgery, and 2) Prolonged hospitalization due to complications post-esophageal dilation. B-VEC treatment was not interrupted during either event. Ocular complications are common in patients with DEB, with over half of the patients diagnosed with recessive DEB potentially affected. Typical ocular manifestations include corneal abrasion, as well as corneal scarring, pannus, eyelid ectropions and blisters.1,2 There are no specific FDA-approved treatment options for ocular manifestations of DEB.3