Krystal Biotech, Inc. · NASDAQ:KRYS
Krystal Biotech — Corporate Update (October 2018)
21 slides from Krystal Biotech's corporate update, Oct 15, 2018
Slide 1: KB103: Gem Study Interim Phase 1/2 Clinical Update
Gene Therapy for Dermatological Diseases KB103: Gem Study Interim Phase 1/2 Clinical Update October 15, 2018 Exhibit 99.2
Slide 2: Forward-Looking Statements
Forward-Looking Statements This webcast and the slides accompanying it regarding positive interim data from Krystal’s phase 1/2 trial evaluating KB103 in patients suffering from Dystrophic epidermolysis bullosa, or DEB, contains "forward-looking statements" regarding matters that are not historical
Slide 3: KB103 Phase I/II Interim Clinical Update
KB103 Phase I/II Interim Clinical Update
Slide 4: Dystrophic Epidermolysis Bullosa (DEB)
"Butterfly Children" is used to describe young DEB patients because their skin is as fragile as a butterfly’s wings Dystrophic Epidermolysis Bullosa (DEB) Dystrophic Epidermolysis Bullosa A rare, genetic connective tissue disease that causes skin to tear or blister from minor contact Caused by a mut
Slide 5: Krystal's Approach
Krystal’s Approach “Off-the-shelf”, repeat-administration therapy Wild type HSV-1 virus Modified HSV-1 with gene(s) of interest inserted Specific modifications using our proprietary STAR-D platform to modify wild type HSV-1 into a suitable vector to introduce genes and effectors to treat skin diseas
Slide 6: Phase 1/2 Trial Design
Phase 1/2 Trial Design 6 Key objectives: Demonstrate efficacy and safety of KB103 Primary Objectives: Expression of COL7, presence of anchoring fibrils, and safety Secondary Objectives: Change in wound area, duration of wound closure, time to wound closure Principal investigator: Dr. Peter Marinkovi
Slide 7: Patient Characteristics
Patient Characteristics Two patients have completed the study per Phase 1/2 protocol Characteristic Patient 01 Patient 02 Age, y 35 28 Sex Male Male Clinical and genetic diagnosis Recessive DEB Recessive DEB Type VII collagen expression By immunofluorescence of skin biopsy Trace NC1 detected NC2 und
Slide 8: KB103 Pharmacology
KB103 Pharmacology Detection of both NC1 and NC2 domains confirms expression of full length COL7 Molecular Therapy 2009 17, 6-7DOI: (10.1038/mt.2008.262)
Slide 9: KB103 Safety Update in Wounds with Topical Application
KB103 Safety Update in Wounds with Topical Application No treatment-related adverse events (serious or otherwise) were reported. No immune response or blistering observed around the sites of administration following first and repeat dose. Blood and urine samples collected throughout the study reveal
Slide 10: KB103 Efficacy Update in Wounds with Topical Application
KB103 Efficacy Update in Wounds with Topical Application Results to date on 2 patients met all primary efficacy (presence of functional COL7 expression as early as Day 2 of treatment, observation of NC1 and NC2 reactive anchoring fibrils and continued expression following repeat administration) and
Slide 11: Patient 01: COL7 Immunofluorescence
Patient 01: COL7 Immunofluorescence Full length COL7 (NC1 and NC2) was detected at week 4 and week 6 Baseline 4 weeks 6 weeks BMZ BMZ BMZ BMZ BMZ BMZ BMZ SC BMZ SC COL7 NC1 COL7 NC2 SC: stratum corneum; BMZ: basement membrane zone
Slide 12: Patient 02: COL7 Immunofluorescence
Patient 02: COL7 Immunofluorescence COL7 NC1 COL7 NC2 Baseline Week 4 Week 6 BMZ BMZ BMZ BMZ BMZ BMZ Full length COL7 (NC1 and NC2) was detected at week 4 and week 6 Week 9 BMZ BMZ
Slide 13: Patient 1: Anchoring Fibrils using Immunoelectron Microscopy
Patient 1: Anchoring Fibrils using Immunoelectron Microscopy NC1 and NC2 reactive anchoring fibrils were detected as early as Week 2, and throughout the study Baseline Week 12 NC1 NC2 Week 2
Slide 14: Patient 2: Anchoring Fibrils using Immunoelectron Microscopy
Patient 2: Anchoring Fibrils using Immunoelectron Microscopy NC1 and NC2 reactive anchoring fibrils were detected as early as Week 2, and throughout the study Baseline Week 9 NC1 NC2 Week 2
Slide 15: Patients 1 and 02: Vector DNA Copies by qPCR
Patients 01 and 02: Vector DNA Copies by qPCR Post-dose KB103 DNA copies Dosing on Days 0 and 28. Measurements at Days 2 and 30. The ability to re-administer KB103 was confirmed.
Slide 16: Wound Monitoring
Wound Monitoring KB103-treated wounds closed by week 2 and continued to remain closed to date Parameters Patient 01 Patient 02 Number of wounds treated KB103 Placebo KB103 Placebo1 1 1 1 1 Time to complete wound closure (weeks) 2.0 10 2 - Duration of wound closure (months) 4.52 2.5 3.52 - % wound cl
Slide 17: Patient 1: Wound Closure Images
Patient 1: Wound Closure Images Patient 1, KB103- and Placebo-Randomized Wounds Baseline 1 Month 2.5 Months 1.5 Months KB103 Placebo
Slide 18: Patient 2: Wound Closure Images
Patient 2: Wound Closure Images Patient 2, KB103- and Placebo-Randomized Wounds Baseline 1 Month 2 Months KB103 Placebo
Slide 19: Patients 1 and 2: Long-term Wound Closure Images
Patients 1 and 2: Long-term Wound Closure Images Post-Study, At-Home Wound Imaging of the KB103-Administered site Sites of KB103 Administration Baseline 4.5 months of closure 3.5 months of closure Baseline 3.5 months of closure Patient 01 Patient 02
Slide 20: KB103 Efficacy Update in Wounds with Topical Application
KB103 Efficacy Update in Wounds with Topical Application Results to date on 2 patients met all primary efficacy (presence of functional COL7 expression, observation of NC1 and NC2 reactive anchoring fibrils and continued expression following repeat administration) and safety endpoints (no adverse ev
Slide 21: Next Steps in KB103 Development
Next Steps in KB103 Development Complete Phase 1/2 trial in adults and pediatric patients by 1H 2019 Clinical data from two patients submitted to FDA FDA acknowledges molecular correction as evidenced by expression of COL7 and anchoring fibrils Amended protocol allows for: Removal of the intradermal