B-VEC Eyedrops Improve Vision in Ocular DEB Case Study

Beremagene geperpavec (B-VEC), in an eyedrop formulation, was well-tolerated and yielded vision improvements in 1 patient with ocular complications in dystrophic epidermolysis bullosa (DEB), according to the finding of a recently published case study.¹

“DEB is a devastating disease and patients with ocular complications have no corrective treatment options leaving them at risk of severe vision loss,” investigator Alfonso L. Sabater, MD, PhD, an associate professor of clinical ophthalmology at the Bascom Palmer Eye Institute of University of Miami Miller School of Medicine, said in a statement.² “We are encouraged by the improvements observed in the patient following B-VEC administration as an eyedrop directly to the affected eye and believe this data is supportive of further investigation in DEB patients with ocular complications. If approved, this approach could drastically benefit these patients.”

B-VEC was approved in a gel formulation under the name Vyjuvek for treating skin wounds in patients with DEB in May 2023.³ B-VEC is a topical herpes simplex virus type 1-based gene therapy that delivers COL7A1 to restore type 7 collagen protein, which is impacted by mutations in the COL7A1 gene in patients with DEB.

WATCH NOW: Raj Chovatiya, MD, PhD, on Investigating HSV-1 Vectors in Collagen-Based Disorders

The patient received 1 drop of undiluted B-VEC biologic suspension (5 × 10⁹ plaque-forming units per mL) and suturing of an amniotic membrane immediately after surgical symblepharon lysis with pannus removal in his right eye. A bandage contact lens (Kontur Kontact Lens) was placed on top of the amniotic membrane, and then ophthalmic B-VEC was administered 3 times per week for the first 2 weeks and then once weekly along with topical prednisolone and moxifloxacin.

One month later, the bandage contact lens was removed, and investigators observed a corneal epithelial defect and peripheral neovascularization. The patient then received topical insulin drops (1 U per mL) 4 times daily and used until full epithelial healing was observed at 3 months after surgery, at which point he received eye drops consisting of allogeneic (derived from the patient’s mother) plasma rich in growth factors 4 times daily, and B-VEC applications in a monthly cadence. The patient received a total of 19 applications of B-VEC.¹

“We are definitely very interested in treating additional patients that may have this condition and we have been discussing about that. But also treating other conditions in the eye that maybe have a genetic component, or even without that, patients that have a very severe condition and are not responding to the standard treatments that we have nowadays available in the market, where maybe collagen could play a role,” Sabater previously told CGTLive^® about potential future research with B-VEC.

The patient had 1 serious adverse event of complications from gastrointestinal surgery unrelated to B-VEC treatment. With the B-VEC treatment and other adjuvant treatments, the patient’s visual acuity in his right eye improved from hand motion (logMAR, 3.00) before surgery to 20/25 (logMAR, 0.10) without correction 8 months after the surgery.¹

“We are excited by this initial data suggesting additional applications of our proprietary HSV-1-based gene therapy platform to treat ocular diseases, and we are working with the FDA to get B-VEC approved for the treatment of DEB patients with lesions in the eye,” Suma Krishnan, President, Research & Development, Krystal Biotech, added.²

REFERENCES

1. Vetencourt AT, Sayed-Ahmed I, Gomez J, et al. Ocular Gene Therapy in a Patient with Dystrophic Epidermolysis Bullosa. New Eng J Med. 2024 (390)6: 530-535. doi: 10.1056/NEJMoa2301244
2. Krystal Biotech Announces Publication in the New England Journal of Medicine on the Application of B-VEC to Treat Ocular Complications in Patient with Dystrophic Epidermolysis Bullosa. News release. Krystal Biotech. February 8, 2024. https://finance.yahoo.com/news/krystal-biotech-announces-publication-england-120000461.html
3. Krystal Biotech Receives FDA Approval for the First-Ever Redosable Gene Therapy, VYJUVEK™ (beremagene geperpavec-svdt) for the Treatment of Dystrophic Epidermolysis Bullosa. News release. Krystal Biotech. May 19, 2023. Accessed May 19, 2023. https://ir.krystalbio.com/news-releases/news-release-details/krystal-biotech-receives-fda-approval-first-ever-redosable-gene