Krystal Biotech, Inc. · NASDAQ:KRYS

Krystal Biotech — Corporate Update (July 2025)

18 slides from Krystal Biotech's corporate update, Jul 9, 2025

  1. Slide 1: KB801 for Neurotrophic Keratitis First Patient Dosed Update

    © Copyright 2025 Krystal Biotech, Inc. All rights reserved. KB801 for Neurotrophic Keratitis First Patient Dosed Update July 2025

  2. Slide 2: Forward Looking Statements and Disclosures

    Krystal | 2 Forward Looking Statements and Disclosures This presentation and the accompanying oral presentation contain forward-looking statements that involve substantial risks and uncertainties. Any statements about future expectations, plans, and prospects for Krystal Biotech, Inc. (together with

  3. Slide 3: Agenda

    Krystal | 3 Agenda About Neurotrophic Keratitis Krish Krishnan; Chairman and CEO Krystal’s Platform for Front of Eye Suma Krishnan, MS, MBA; President, Research & Development Krystal’s KB801 Program  Preclinical Overview  Phase 1/2 Clinical Trial Design  Regulatory and Pipeline Outlook Trevor Par

  4. Slide 4: Neurotrophic Keratitis is a Degenerative, Vision-Threatening Disease

    Krystal | 4 Neurotrophic Keratitis is a Degenerative, Vision-Threatening Disease Sacchetti M, et al. Clin Ophthalmol. 2014. 8: 571-579; Bian Y, et al. Ophthalmology. 2022. 129: 1255-1262; Rama P, et al. Orphanet. Neurotrophic Keratopathy. 2017 [accessed Sept 28 2023]; Gablson EE et al. Invest Ophtha

  5. Slide 5: KB801 Designed to Address Shortcomings of Only FDA Approved Therapy

    Krystal | 5 • The only specific FDA approved therapy for NK is Oxervate® • First approved in 2018, Oxervate is an ophthalmic formulation of recombinant human nerve growth factor (cenegermin-bkbj) for topical application as an eye drop • Oxervate® targets underlying nerve defect and has been shown to

  6. Slide 6: Krystal's HSV-1 Platform Well Suited for Front of the Eye Applications

    Krystal | 6 Sabater A et al., Poster # 787 - C0388. at the 2023 Association for Research in Vision and Ophthalmology Annual Meeting; Vetencourt AT, et al. N Engl J Med. 2024;390:530-535 B-VEC, beremagene geperpavec; DEB, dystrophic epidermolysis bullosa; HSV-1, herpes simplex virus type 1 Rapid prot

  7. Slide 7: Ophthalmology Expansion Already Well Underway with KB803 Phase 3

    Krystal | 7 Ophthalmology Expansion Already Well Underway with KB803 Phase 3 KB803 for Ocular Complications of DEB  Repeat administration of B-VEC eye drops under compassionate use previously shown to be well tolerated and associated with full corneal healing as well as significant visual acuity im

  8. Slide 8: KB801 Preclinical Data Summary

    Krystal | 8B-VEC, beremagene geperpavec; HSV-1, herpes simplex virus 1; NGF, nerve growth factor KB801 Replication-incompetent HSV-1 vector containing functional human NGF 2 x NGF genes      Topical administration to wounded murine corneas was well tolerated and resulted in localized NGF expres

  9. Slide 9: KB801 Transduces Corneal Epithelial Cells In Vitro For Secretion of Mature NGF

    Krystal | 9 KB801 Transduces Corneal Epithelial Cells In Vitro For Secretion of Mature NGF Cartwright HN, et al. Poster #2467 at the 2025 Association for Research in Vision and Ophthalmology (ARVO) Annual Meeting; Krystal Biotech. Data on File. DNA, deoxyribonucleic acid; ELISA, enzyme linked immuno

  10. Slide 10: NGF Expression and Corneal Localization Confirmed in Pilot Mouse Study

    Krystal | 10 NGF Expression and Corneal Localization Confirmed in Pilot Mouse Study Cartwright HN, et al. Poster #2467 at the 2025 Association for Research in Vision and Ophthalmology (ARVO) Annual Meeting; Krystal Biotech. Data on File. * Assuming single eye drop of Oxervate® is 35 uL and contains

  11. Slide 11: No Evidence of Adverse Inflammation or Histological Abnormalities

    Krystal | 11 No Evidence of Adverse Inflammation or Histological Abnormalities Cartwright HN, et al. Poster #2467 at the 2025 Association for Research in Vision and Ophthalmology (ARVO) Annual Meeting; Krystal Biotech. Data on File. * Assuming single eye drop of Oxervate® is 35 uL and contains 700 n

  12. Slide 12: Clear Durability Advantage with KB801 in Head to Head Mouse PK Study

    Krystal | 12 Clear Durability Advantage with KB801 in Head to Head Mouse PK Study Head to Head PK Study #1 • Study conducted in BALB/c mice, 6-10 weeks of age, with eyes wounded using crosshatch technique • Test conditions, each administered as 3 µL eyedrop • KB801: 4.6 x 107 PFU • Dilution factor m

  13. Slide 13: Superior PK Profile Confirmed Even Against Intensive Recombinant Dosing

    Krystal | 13 Superior PK Profile Confirmed Even Against Intensive Recombinant Dosing Head to Head PK Study #2 • Study conducted in BALB/c mice, 6-10 weeks of age, with eyes wounded using crosshatch technique • Test conditions, each administered as 3 µL eyedrop • KB801: 4.6 x 107 PFU • Dilution facto

  14. Slide 14: EMERALD-1 Study Objectives and Key Enrollment Criteria

    Krystal | 14 Study Objectives • Evaluate safety and tolerability of twice-weekly KB801, as well as preliminary efficacy evaluation including: • Complete durable healing of the corneal epithelium at 8 weeks, defined as 0 mm fluorescein staining at 8 weeks and no residual staining at 10 weeks • Change

  15. Slide 15: Opportunity for Expedited Development on EMERALD-1 Success

    Krystal | 15 Opportunity for Expedited Development on EMERALD-1 Success CMC, chemistry, manufacturing, and controls Leveraging Prior Experience with Oxervate  Extensive clinical and commercial experience with Oxervate derisks safety of KB801 payload  Opportunity for efficient registrational study

  16. Slide 16: Broadening the Scope of HSV-1 Platform with an Active Clinical Pipeline

    Krystal | 16 Broadening the Scope of HSV-1 Platform with an Active Clinical Pipeline Respiratory  KB407 Phase 1 Cohort 3 in cystic fibrosis patients  KB408 Phase 1 in patients with alpha-1 antitrypsin deficiency lung disease Ophthalmology  KB803 Phase 3 in DEB patients with ocular complications 

  17. Slide 17: Clear Path to Delivering Long-Term Growth and Shareholder Value

    Krystal | 17  VYJUVEK franchise poised for multiple years of growth in U.S. and through global expansion  Potential for efficient, near-term launch of KB803 highly synergistic with VYJUVEK  Lung and eye are springboards targeting multiple blockbuster markets  Significant value unlock and multi-p

  18. Slide 18: Developing Genetic Medicines to Treat Diseases with High Unmet Medical Needs

    Developing Genetic Medicines to Treat Diseases with High Unmet Medical Needs © Copyright 2025 Krystal Biotech, Inc. All rights reserved.

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