Krystal Biotech, Inc. · NASDAQ:KRYS
Krystal Biotech — Investor Presentation (March 2021)
30 slides from Krystal Biotech's investor presentation, March 2021
Slide 1: The Leader in Redosable Gene Therapy for Rare Disease
The Leader in Redosable Gene Therapy for Rare Disease MARCH 2021 Krystal Bio | © Copyright 2021 Krystal Biotech, Inc. All rights reserved.
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: Krystal overview
Krystal overview A fully integrated, clinical stage gene therapy company powered by proprietary HSV-1 vector technology Differentiated viral vector platform enables in vivo, repeat dose gene therapies • Proprietary, engineered replication incompetent HSV-1 based platform • Clinical data shows mainte
Slide 4: Upcoming Milestones
Upcoming Milestones Progress across pipeline will yield pivotal data for B-VEC, a 4th clinical stage program and a new respiratory candidate in 2021 Timing Program Event 1Q21 B-VEC for DEB Complete enrollment in pivotal GEM-3 study in DEB 1Q21 KB301 for aesthetic indications Announce Initial safety
Slide 5: Our technology platform enables noninvasive, redosable gene therapy
Our technology platform enables noninvasive, redosable gene therapy Krystal Bio | © Copyright 2021 Krystal Biotech, Inc. All rights reserved.
Slide 6: HSV-1 is positively differentiated vs. other gene therapy technologies
HSV-1 is positively differentiated vs. other gene therapy technologies Wild-type Krystal’s Therapeutic Vector Platform Comparison HSV-1 Modified HSV-1 Vector Platform LV AAV HSV-1 LNP In vivo dosing? No Yes Yes Yes Baseline antibody No Yes No No exclusion criteria? (if ex vivo) Repeat-dose Yes HSV-1
Slide 7: Clinically validated platform targeting skin; broad tropism of HSV-1 could unlock additional target tissues
Clinically validated platform targeting skin; broad tropism of HSV-1 could unlock additional target tissues Formulation / Route Drug Product of Admin Therapeutic ✓ B-VEC ✓ KB105 Vector • KB104 • Early stage research Topical projects ✓ KB301 • KB104 • Early stage research Intradermal projects The cur
Slide 8: Current pipeline
Current pipeline Key Upcoming WW Rights Product Indication Discovery Preclinical Phase I/II Phase III Milestone Top line pivotal B-VEC†¤•Δ‡§ Dystrophic EB data in 4Q21 Rare Skin Ph2 data and next KB105†¤•‡ TGM1-deficient ARCI steps in 1H21 KB104¤ Netherton Syndrome File IND in 2H21 Rare Lung KB407 †
Slide 9: Platform supported by in-house manufacturing capacity and expertise
Platform supported by in-house manufacturing capacity and expertise Facility operational Established process conducted at Krystal’s end-to-end GMP facility Size: 7,500 sq. ft. (Ancoris) • Maintains control of IP/trade secrets relating to manufacturing process • Adheres to internal process and produc
Slide 10: Initial focus on rare skin diseases led to rapid clinical POC and pipeline
Initial focus on rare skin diseases led to rapid clinical POC and pipeline Krystal Bio | © Copyright 2021 Krystal Biotech, Inc. All rights reserved.
Slide 11: Dystrophic epidermolysis bullosa (DEB)
Dystrophic epidermolysis bullosa (DEB) “Butterfly Children” is often used to describe young DEB patients because their skin is as fragile as a butterfly’s wings Dystrophic Epidermolysis Bullosa • A rare, genetic skin disease that causes skin to tear or blister from minor contact • Mutations in the C
Slide 12: Beremagene geperpavec (B-VEC) for DEB
Beremagene geperpavec (B-VEC) for DEB Topically applied B-VEC gel is designed to induce local COL7 expression and molecular correction 1 B-VEC enters the compromised skin of B-VEC COL7 protein DEB patients and transduces both 1 2 3 4 keratinocytes and fibroblasts COL7 Anchoring Nucleus transcripts f
Slide 13: Topical B-VEC was evaluated in a Phase1/2 study
Topical B-VEC was evaluated in a Phase1/2 study • GEM1/2 (NCT03536143) was an intra-patient comparison of wounds randomized to receive either topical B-VEC or placebo. Design • Each patient on-study for ~6 months; 3 months of on-site visits followed by 3-month at-home imaging period • Study PI: Dr.
Slide 14: Repeat doses of topical B-VEC were well tolerated; COL7 expression and molecular correction established
Repeat doses of topical B-VEC were well tolerated; COL7 expression and molecular correction established Molecular correction established and Increasing doses and dosing frequency were correlated with wound healing well tolerated • Expression and correct localization of full-length COL7 was observed
Slide 15: B-VEC showed statistically significant benefit in wound healing relative to placebo
B-VEC showed statistically significant benefit in wound healing relative to placebo Percent change in wound area from (individual Percentage of wounds reaching 100% closure measurements and average) from Day 0 to 90¤ at weeks 8, 10 and 12¤ BVEC Placebo BVEC Average Placebo Average 90.0% 82.4% 180% 8
Slide 16: The pivotal GEM-3 study is enrolling; top line data expected in 2021
The pivotal GEM-3 study is enrolling; top line data expected in 2021 • GEM-3 (NCT04491604) is a randomized, double-blind, intra-patient comparison of wounds randomized to receive either topical B-VEC or placebo. Design • Each patient on-study for approximately 7 months: the 6-month dosing period fol
Slide 17: Phase 3 trial is well powered and inclusive of a broad patient population
Phase 3 trial is well powered and inclusive of a broad patient population The trial is designed to maximize chances of success while maintaining potential for a broad label, inclusive of chronic and recurring wounds of any size in RDEB or DDEB patients 30-day Screening 6-month Treatment Period OLE S
Slide 18: Autosomal Recessive Congenital Icthyosis associated with TGM1 mutations
Autosomal Recessive Congenital Icthyosis associated with TGM1 mutations Transglutaminase-1 deficiency is associated with increased mortality in the neonatal period and has a dramatic impact on quality of life Autosomal Recessive Congenital Ichthyosis (ARCI) Associated with TGM1 • The most common for
Slide 19: KB105 for TGM1 associated ARCI
KB105 for TGM1 associated ARCI Topically applied KB105 delivers multiple copies of the human transglutaminase 1 (“TGM1”) gene 5 1 IVL KB105 enters permeabilized skin TGM1 and transduces keratinocytes FLG (native TGM1-producing cells) SPRs KB105 1 2 3 4 2 KB105 is transported into the TGM1 nucleus of
Slide 20: KB105 is being evaluated in a Phase1/2 study
KB105 is being evaluated in a Phase1/2 study • The Ph1/2 trial (NCT04047732) is an open label, intra-patient comparison of KB105 and placebo Design • Each patient on-study for approximately six months; 3 months of on-site visits followed by 3-month at-home imaging period • Study PI: Dr. Amy Paller (
Slide 21: Initial data shows repeat dosing of KB105 to be well tolerated; molecular and phenotypic improvement evident
Initial data shows repeat dosing of KB105 to be well tolerated; molecular and phenotypic improvement evident KB105 Was Well Tolerated and Generated Phenotypic Improvement Evident After Topical Functional TGM1 protein and Microneedle Application • Repeat dosing with KB105 was well tolerated with no d
Slide 22: Leveraging platform to target new tissues and larger indications
Leveraging platform to target new tissues and larger indications Krystal Bio | © Copyright 2021 Krystal Biotech, Inc. All rights reserved.
Slide 23: HSV-1 has potential beyond rare skin diseases
HSV-1 has potential beyond rare skin diseases ✓ B-VEC (COL7A1) for DEB Rare Skin ✓ KB105 (TGM1) for TGM1-ARCI Krystal’s Current Platform • KB104 (SPINK5) for Netherton’s Disease Disease • Additional preclinical candidates Skin Wild-type HSV-1 ✓ KB301 (COL3) for Acne Scars and Aesthetics Wrinkles (Je
Slide 24: KB407 for cystic fibrosis
KB407 for cystic fibrosis Gene therapy approaches have been tried and failed in Our vector can be formulated and delivered via their attempts to replace CFTR protein nebulizer with no significant change in activity • Viral (adenovirus and AAV) and non-viral (DNA plasmids and stabilized • In vitro da
Slide 25: Preclinical data supports KB407 in CF and broader development in lung disease
Preclinical data supports KB407 in CF and broader development in lung disease Robust, dose-dependent CFTR KB407-mediated functional correction of Two repeat doses of KB407 in a nonhuman expression and functional correction CF phenotype in clinically relevant 3D primate were well tolerated and distri
Slide 26: KB301 for aesthetic indications
KB301 for aesthetic indications KB301 and other discovery programs in Aesthetics, are housed in our wholly owned subsidiary, Jeune Inc. Many Characteristics of Skin Aging are Due to Aberrant KB301 is currently being evaluated in a Phase 1 trial Collagen Homeostasis (NCT04540900) • Dermal collagen, c
Slide 27: Preclinical data supports KB301 development aesthetic indications
Preclinical data supports KB301 development aesthetic indications A dose dependent increase in COL3A1 observed in young and aged mice Similar dose dependent effect was observed in primary human dermal fibroblasts • COL3A1 DNA (A) and transcript levels (B) upon KB301 transduction of primary aged HDFs
Slide 28: Financials and Milestones
Financials and Milestones Krystal Bio | © Copyright 2021 Krystal Biotech, Inc. All rights reserved.
Slide 29: Krystal summary (updated)
Krystal summary (updated) A fully integrated, clinical stage gene therapy company powered by proprietary HSV-1 vector technology Current Status and Milestones Rare Skin • B-VEC: Pivotal GEM-3 trial currently enrolling with enrollment completion anticipated in 1Q21; topline data expected 4Q21. Commer
Slide 30: The Leader in Redosable Gene Therapy for Rare Disease
The Leader in Redosable Gene Therapy for Rare Disease MARCH 2021 Krystal Bio | © Copyright 2021 Krystal Biotech, Inc. All rights reserved.