Krystal Biotech to Present Additional Data on B-VEC from the GEM-3 Phase 3 Study at the Society for Investigative Dermatology Annual Meeting

Rhea-AI Summary

Krystal Biotech, Inc. (NASDAQ: KRYS) unveiled new data from the phase 3 GEM-3 clinical trial on the investigational gene therapy B-VEC for dystrophic epidermolysis bullosa (DEB) at the Society for Investigative Dermatology Annual Meeting. The therapy aims to treat DEB by delivering copies of the COL7A1 gene to enhance collagen production. B-VEC has received orphan drug designation and fast track status from the FDA, indicating its potential for significant market impact. The findings contribute to Krystal's ongoing efforts to innovate in gene therapy for rare skin diseases.

Positive

• Presentation of phase 3 GEM-3 trial results for B-VEC, indicating continued progress in the development of gene therapy for DEB.
• B-VEC has received orphan drug designation and fast track status from both the FDA and EMA, underscoring its potential in the market.

Negative

• None.

PITTSBURGH, May 19, 2022 (GLOBE NEWSWIRE) -- Krystal Biotech, Inc. (the “Company”) (NASDAQ: KRYS), the leader in redosable gene therapy, is pleased to present new data entitled “GEM-3: phase 3 safety and immunogenicity results of beremagene geperpavec (B-VEC), an investigational, topical gene therapy for dystrophic epidermolysis bullosa (DEB)” at the Society for Investigative Dermatology 2022 Annual Meeting, taking place May 18-21 in Portland, Oregon.

Krystal Presentation

GEM-3: phase 3 safety and immunogenicity results of beremagene geperpavec (B-VEC), an investigational, topical gene therapy for dystrophic epidermolysis bullosa (DEB)
M. Peter Marinkovich, MD, FAAD
Poster Session 1
Date & Time: Thursday, May 19, 4:30-6:30pm PDT

ePoster Presentation: Session 2, Genetic Disease, Gene Regulation, and Gene Therapy
Date & Time: Friday, May 20, 5:54-6:00pm PDT

The poster and ePoster will be available to conference attendees. To register for the conference, please visit SID 2022 Annual Meeting | Society for Investigative Dermatology. The Company will be present at Booth 218 to educate about DEB and the mechanism of the disease. Following the presentation, materials will be available to view online on the Investor section of the Company’s website.

About Dystrophic Epidermolysis Bullosa (DEB)
DEB is a rare and severe disease that affects the skin and mucosal tissues. It is caused by one or more mutations in a gene called COL7A1, which is responsible for the production of the protein type VII collagen (COL7) that forms anchoring fibrils that bind the dermis (inner layer of the skin) to the epidermis (outer layer of the skin). The lack of functional anchoring fibrils in DEB patients leads to extremely fragile skin that blisters and tears from minor friction or trauma. DEB patients suffer from open wounds, which leads to skin infections, fibrosis which can cause 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.

About B-VEC
B-VEC is an investigational non-invasive, topical, redosable 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 the template to make normal COL7 protein, thereby addressing the fundamental disease-causing mechanism.

The U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) have each granted B-VEC an orphan drug designation for the treatment of DEB. The FDA has also granted B-VEC fast track designation and rare pediatric designation for the treatment of DEB. In addition, in 2019, the FDA granted Regenerative Medicine Advanced Therapy (“RMAT”) to B-VEC for the treatment of DEB and the EMA granted PRIority MEdicines ("PRIME") eligibility for B-VEC to treat DEB.

About Krystal Biotech, Inc.
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 develop life-changing medicines for patients with serious diseases, including rare diseases in skin, lung, and other areas. For more information please visit http://www.krystalbio.com, and follow @KrystalBiotech on LinkedIn and Twitter.

CONTACTS:
Investors and Media:
Meg Dodge
Krystal Biotech
mdodge@krystalbio.com

Source: Krystal Biotech, Inc.

FAQ

What are the results of the GEM-3 trial for KRYS?

The GEM-3 trial results presented at the Society for Investigative Dermatology Annual Meeting focus on the safety and immunogenicity of B-VEC for treating DEB.

What is the purpose of the B-VEC gene therapy?

B-VEC is designed to deliver copies of the COL7A1 gene to improve collagen production in patients suffering from dystrophic epidermolysis bullosa.

What designations has B-VEC received from the FDA?

B-VEC has received orphan drug designation, fast track designation, and rare pediatric designation from the FDA.

When was the GEM-3 trial data presented by KRYS?

The GEM-3 trial data was presented on May 19, 2022, at the Society for Investigative Dermatology Annual Meeting.

How does B-VEC address dystrophic epidermolysis bullosa?

B-VEC addresses DEB by delivering a gene therapy that enables the production of type VII collagen, targeting the molecular cause of the disease.