Krystal Biotech, Inc. · NASDAQ:KRYS

Krystal Biotech — Technical Presentation (May 2022)

7 slides from Krystal Biotech's technical presentation, May 20, 2022

  1. Slide 1: GEM-3: Phase 3 Safety and Immunogenicity Results of B-VEC for DEB

    GEM-3: Phase 3 Safety and Immunogenicity Results of Beremagene Geperpavec (B-VEC), an Investigational, Topical Gene Therapy for Dystrophic Epidermolysis Bullosa (DEB) 1Stanford University, Redwood City, CA, USA; 2University of Miami, Miami, FL, USA; 3Mission Dermatology Center, Children’s Hospital o

  2. Slide 2: Disclosures

    Disclosures  M. Peter Marinkovich reports the following disclosures: – Krystal Biotech (Investigator), Abeona Therapeutics (Investigator), CastleCreek (Investigator), Phoenix Tissue Repair (Investigator), WINGS Therapeutics (Investigator)  This study was funded by Krystal Biotech, Inc. 2

  3. Slide 3: GEM-3: Phase 3 Study Evaluating Efficacy and Safety of B-VEC in DEB

    NCT04491604; †Maximum weekly dose (PFU/week) was based on patient's age and unit dose (PFU/wound) was determined based on wound area at baseline. B-VEC, beremagene geperpavec; COL7, type VII collagen; DEB, dystrophic epidermolysis bullosa; HSV-1, herpes simplex virus type 1; PFU, plaque-forming unit

  4. Slide 4: B-VEC was Generally Well Tolerated

    B-VEC was Generally Well Tolerated Total patients (n=31) Total number of AEs 45 Patients with ≥1 AE, n (%) 18 (58.1) Mild AE 15 (48.4) Moderate AE 3 (9.7) Severe AE 2 (6.5) Serious AE 3 (9.7) Drug-related AE 1 (3.2) AE leading to treatment discontinuation 0 (0) Death 0 (0) *AEs were coded using MedD

  5. Slide 5: Anti-HSV-1 and Anti-COL7 Antibody Results

    Anti-HSV-1 and Anti-COL7 Antibody Results  22 of 31 patients (71.0%) provided a serum sample at baseline due to the difficulty of blood draws owing to skin fragility – 19 of the 22 patients (86.4%) also had matched serum samples at 6 months  At baseline, 14 of the 22 patients (63.6%) were anti-HSV

  6. Slide 6: Treatment Response to B-VEC Not Associated with Anti-HSV-1 Serostatus or Anti-COL7 Seroconversion

    Treatment Response to B-VEC was Not Associated with Anti-HSV-1 Serostatus at Baseline or with Anti-COL7 Seroconversion Data in figures based on post hoc analyses using imputation; a responder was defined as meeting the primary endpoint of complete wound healing at 6 months. *Seroconversion defined a

  7. Slide 7: Conclusions

    Conclusions  B-VEC treatment demonstrated a durable and statistically significant improvement in complete wound healing at 3 and 6 months compared with placebo  B-VEC was generally well tolerated, with no treatment-related discontinuations  No clinically significant immunologic reactions were rep

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