Slide 1: Topline GEM-3 Trial Results Call
The Leader in Redosable Gene Therapy for Rare Disease Topline GEM-3 Trial Results Call © Copyright 2021 Krystal Biotech, Inc. All rights reserved. Exhibit 99.2
Slide 2: Forward looking statements
Forward looking statements This presentation contains forward-looking statements that involve substantial risks and uncertainties. Any statements in this presentation about future expectations, plans and prospects for Krystal Biotech, Inc. (the “Company”), including but not limited to statements abo
Slide 3: Call Agenda
© Copyright 2021 Krystal Biotech, Inc. All rights reserved. Call Agenda Introductory Comments Krish Krishnan – Chairman and CEO Dystrophic Epidermolysis Bullosa Background Dr. Peter Marinkovich GEM-3 Results and Next Steps Suma Krishnan – Founder and COO Commercial Preparations Andy Orth – Chief Com
Slide 4: Chairman and CEO Krish Krishnan
Chairman and CEO Krish Krishnan © Copyright 2021 Krystal Biotech, Inc. All rights reserved.
Slide 5: Novel viral vector platform positively differentiated from other viral vector technologies
Novel viral vector platform positively differentiated from other viral vector technologies Robust tropism to target cells of interest upon local administration Large payload capacity allows for delivery of two copies of large gene Immune evasive properties of proprietary vectors enables redosability
Slide 6: GEM-3 results provide significant platform validation
GEM-3 results provide significant platform validation Engineered HSV-1 Vector Platform GEM-3 Study Confirms safety and efficacy of VYJUVEKTM* (beremagene geperpavec) for dystrophic EB Most advanced clinical application of platform Validates therapeutic vector in dermatologic applications Skin pipeli
Slide 7: Dr. Peter Marinkovich, Stanford University
Associate Professor of Dermatology at Stanford University Blistering Disease Clinic Director Dr. Peter Marinkovich Disclosures: Principal investigator in GEM-3 trial © Copyright 2021 Krystal Biotech, Inc. All rights reserved.
Slide 8: Epidermolysis Bullosa is a group of rare diseases
Images courtesy of Dr. Peter Marinkovich © Copyright 2021 Krystal Biotech, Inc. All rights reserved. Epidermolysis Bullosa is a group of rare diseases associated with fragile skin, causing skin to blister easily Dystrophic EB One of four inherited forms of EB Dystrophic EB can be inherited dominantl
Slide 9: The dystrophic form of EB is caused by mutations in the COL7A1 gene
Images courtesy of Dr. Peter Marinkovich © Copyright 2021 Krystal Biotech, Inc. All rights reserved. The dystrophic form of EB is caused by mutations in the COL7A1 gene The location of the blisters (below the lamina densa) corresponds to level of the “anchoring fibrils” Anchoring fibrils are the mol
Slide 10: There are currently no approved corrective treatments for dystrophic EB
© Copyright 2021 Krystal Biotech, Inc. All rights reserved. There are currently no approved corrective treatments for dystrophic EB Current treatment options for dystrophic EB are largely palliative in nature, involving wound care regimens similar to the care provided to burn victims Blistered areas
Slide 11: B-VEC is an investigational, off-the-shelf, topical gene therapy
B-VEC is an investigational, off-the-shelf, topical gene therapy designed to correct the underlying molecular defect in DEB wounds Blister Untreated DEB Patient Skin B-VEC-treated Skin 1 Keratinocytes Dermoepidermal Junction Fibroblasts Anchoring Fibrils (Collagen VII) B-VEC 2 3 Nucleus COL7 transcr
Slide 12: Founder and COO Suma Krishnan
Founder and COO Suma Krishnan © Copyright 2021 Krystal Biotech, Inc. All rights reserved.
Slide 13: VYJUVEK represents important firsts
1 1 1 VYJUVEKTM represents important firsts VYJUVEK is an investigational therapy being studied in clinical trials © Copyright 2021 Krystal Biotech, Inc. All rights reserved. These data also position VYJUVEK to become the first-ever in vivo redosable gene replacement therapy VYJUVEK could be the fir
Slide 14: GEM-3 trial design and endpoints
Once-weekly treatment until wound closure Resumed if wound reopened Open-label Extension GEM-3 evaluated weekly VYJUVEKTM or Placebo in dystrophic EB patients 31 Patients VYJUVEK Placebo Primary Wound Pair Randomized, double-blind 6-month Treatment Period 30-day Safety Primary Efficacy Endpoints Com
Slide 15: Topline Ph3 safety data summary
Topline Ph3 safety data summary © Copyright 2021 Krystal Biotech, Inc. All rights reserved. Topical VYJUVEK was well tolerated with a safety profile consistent with prior studies No drug-related serious AEs or discontinuations due to treatment were reported Immunogenicity profile (as measured by ant
Slide 16: Topline Ph3 efficacy data summary
Topline Ph3 efficacy data summary © Copyright 2021 Krystal Biotech, Inc. All rights reserved. Met primary endpoint of complete wound healing at 6-month timepoints Response Rate 67% 22% Absolute Difference 45.8% 95% Confidence Interval 23.6%-68.0% p-value* <0.005 0.0155 *based on McNemar test - In an
Slide 17: Secondary wounds treated open-label
Once-weekly treatment until wound closure Resumed if wound reopened Open-label Extension Secondary wounds received open-label VYJUVEKTM throughout the study 31 Patients VYJUVEK Placebo Primary Wound Pair Randomized, double-blind 6-month Treatment Period 30-day Safety Genetically confirmed dystrophic
Slide 18: Secondary wound (Illustrative): large chronic back wound
Secondary wound (Illustrative) © Copyright 2021 Krystal Biotech, Inc. All rights reserved. End of Study Baseline Large, chronic back wound in 21 year old RDEB patient VYJUVEK is an investigational therapy being studied in clinical trials
Slide 19: Secondary wound (Illustrative): recurring foot wound
Secondary wound (Illustrative) © Copyright 2021 Krystal Biotech, Inc. All rights reserved. End of Study Baseline left foot Right foot left foot Right foot Recurring foot wound in 34 year old RDEB patient VYJUVEK is an investigational therapy being studied in clinical trials
Slide 20: Open-label extension ongoing
Once-weekly treatment until wound closure Resumed if wound reopened Open-label Extension Open-label extension ongoing 31 Patients VYJUVEK Placebo Primary Wound Pair Randomized, double-blind 6-month Treatment Period 30-day Safety Genetically confirmed dystrophic EB VYJUVEK Open label 6-month Treatmen
Slide 21: VYJUVEK regulatory next steps
Evaluating approaches in these markets Known patient populations in rest-of-world (i.e. China) Japan in progress Orphan Drug Designation PRIority MEdicines (PRIME) Designation Orphan Drug Designation Rare Pediatric Disease Designation Regenerative Medicine Advanced Therapy (RMAT) designation Fast Tr
Slide 22: CCO Andy Orth
CCO Andy Orth © Copyright 2021 Krystal Biotech, Inc. All rights reserved.
Slide 23: Dystrophic EB patient population and VYJUVEK opportunity
Dystrophic EB patient population and VYJUVEKTM opportunity © Copyright 2021 Krystal Biotech, Inc. All rights reserved. VYJUVEK is an investigational therapy being studied in clinical trials
Slide 24: Launch readiness / efforts
Launch readiness / efforts © Copyright 2021 Krystal Biotech, Inc. All rights reserved. Education + Awareness Patient and Caregiver facing Community Educational Liaisons in the field Health Care Professional and Patient focused Disease State Awareness programming underway Medical Affairs Key Opinion
Slide 25: Platform supported by in-house manufacturing capacity and expertise
Platform supported by in-house manufacturing capacity and expertise Established process conducted at Krystal’s end-to-end GMP facility (Ancoris) Maintains control of IP/trade secrets relating to manufacturing process Adheres to internal process and production schedules, avoiding use of high demand g
Slide 26: Chairman and CEO Krish Krishnan
Chairman and CEO Krish Krishnan © Copyright 2021 Krystal Biotech, Inc. All rights reserved.
Slide 27: Pipeline upcoming events
Pipeline upcoming events †: FDA Orphan Drug Designation; ¤: FDA Rare Pediatric Disease Designation; •: Fast-track Designation; Δ: FDA RMAT designation; ‡: EMA Orphan Drug Designation; §: EMA PRIME Designation. All pipeline compounds are investigational, being evaluated in clinical or pre-clinical st
Slide 28: Redosable gene delivery technology has broad potential
Routes of Administration Redosable gene delivery technology has broad potential Engineered HSV-1 Platform Vector can deliver variety of therapeutic modalities and be administered repeatedly Provides multiple potential pathways to address different tissue types, with options for route of administrati
Slide 29: Questions & Answers
Questions & Answers © Copyright 2021 Krystal Biotech, Inc. All rights reserved.