- Treatment with B-VEC demonstrated robust functional expression of COL7 followed by its assembly into basement membrane-associated anchor fibrils
- B-VEC treatment improved durable wound closure in patients with recessive dystrophic epidermolysis bullosa (RDEB) compared to placebo with minimal adverse effects
PITTSBURGH, March 28, 2022 (GLOBE NEWSWIRE) — Crystal Biotech, Inc.(“Krystal”) (NASDAQ: KRYS), the leader in repeatable gene therapies for rare diseases, today announced results from the Phase 1 and 2 study of topical beremagene geperpavec (B-VEC) for the treatment dystrophic epidermolysis bullosa (DEB) have been published in natural medicine.
The publication provides a comprehensive analysis of data from the Phase 1 and 2 study showing that repeated topical applications of B-VEC were associated with durable wound closure, expression of cutaneous collagen type VII (COL7) across the length and assembly of anchor fibrils with minimal reported adverse events.
“In this first-ever clinical trial of a repeatable topical gene therapy, we are delighted to see that these data show the potential of B-VEC to address the underlying cause of the disease and to delineate B-VEC as a drug easily. administered and well tolerated,” said Suma Krishnan, President, Research and Development, Krystal Biotech, Inc. “For so many years, all we were able to offer DEB patients was palliative care, so now it is rewarding to have a potential remedial treatment option for this deserving group of patients.We are grateful to the trial participants who made this study possible.
The study was led by senior author Mr. Peter Marinkovich, MD, director of the Blistering Disease Clinic at Stanford Health Care and associate professor of dermatology at Stanford University School of Medicine.
DEB is a rare and serious disease that affects the skin and mucosal tissues. It is caused by one or more mutations in COL7A1 gene, which is responsible for producing the COL7 protein which forms anchoring fibrils that link the dermis (inner layer of skin) to the epidermis (outer layer of skin). The lack of functional anchoring fibrils in DEB patients leads to extremely fragile skin that blisters and tears following minor friction or trauma. Patients with DEB suffer from open wounds leading to skin infections, fibrosis which can lead to fusion of fingers and toes and ultimately an increased risk of developing an aggressive form of squamous cell carcinoma which in severe cases can be fatal.
In the phase 1 and 2 study, matched wounds were evaluated in nine RDEB patients receiving topical B-VEC or placebo repeatedly for 12 weeks. Primary and secondary mechanistic and clinical endpoints were met. No grade 2 or higher adverse events related to B-VECs, vector shedding, or systemic drug exposure were noted.
B-VEC is a non-invasive, topical, redistributable investigational gene therapy designed to deliver two copies of the COL7A1 gene when applied directly to DEB wounds. B-VEC was designed to treat DEB at the molecular level by providing the patient’s skin cells with the template to make the normal COL7 protein, thus addressing the fundamental mechanism behind the disease.
The FDA and EMA have each granted Orphan Drug Designation to B-VEC for the treatment of DEB, and the FDA has granted Fast Track Designation to B-VEC and Rare Pediatric Designation for the treatment of DEB. Additionally, in 2019, the FDA granted B-VEC Regenerative Medicine Advanced Therapy (RMAT) for the treatment of DEB and the EMA granted PRIority MEdicines (“PRIME”) eligibility for B-VEC for process the DEB.
About Crystal Biotech
Krystal Biotech, Inc. (NASDAQ:KRYS) is a pivotal-stage gene therapy company leveraging its proprietary redosable gene therapy platform and in-house manufacturing capabilities to
potentially bring life-changing treatment options to patients with serious diseases, including rare diseases of the skin, lungs and other areas. For more information, please visit http://www.krystalbio.comand follow @KrystalBiotech on LinkedIn and Twitter.
Any statements contained in this press release regarding Krystal Biotech, Inc.’s future expectations, plans and prospects, including statements about the clinical utility of B-VEC and other statements containing the words “anticipate”, “believe “, “estimate”, “expect”, “intend”, “may”, “plan”, “predict”, “project”, “target”, “potential”, “probable”, “will “, “should”, “could”, “should”, “continue” and similar expressions constitute forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Actual results may differ materially from those indicated by these forward-looking statements. due to various important factors, including: uncertainties associated with regulatory review of clinical trials and marketing authorization applications, the availability or commercial potential of product candidates, including B-VEC, the sufficiency re cash sources and need for additional financing and any other important factors as set forth under “Risk Factors” in Krystal’s annual and quarterly reports filed with the United States Securities and Exchange Commission. In addition, the forward-looking statements included in this press release represent Krystal’s views as of the date of this release. Krystal anticipates that subsequent events and developments will cause her views to change. However, although Krystal may choose to update these forward-looking statements at some time in the future, it expressly disclaims any obligation to do so. These forward-looking statements should not be taken to represent the views of Krystal as of any date subsequent to the date of this release.
Source: Krystal Biotech, Inc.