Krystal Biotech, Inc. · NASDAQ:KRYS
Krystal Biotech — Investor Presentation (July 2021)
31 slides from Krystal Biotech's investor presentation, Jul 1, 2021
Slide 1: The Leader in Redosable Gene Therapy for Rare Disease
The Leader in Redosable Gene Therapy for Rare Disease July 2021 © 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: Krystal overview
Krystal overview Proprietary, engineered replication incompetent HSV-1 based platform Clinical data shows maintenance of safety and transgene expression after repeat dosing Positive external clinical and regulatory precedent with in vivo HSV-1 based therapy Differentiated viral vector platform enabl
Slide 4: Upcoming Milestones
Upcoming Milestones 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 data from cohort 1 of Phase 1 study in facial wrinkles and acne scars 1Q21 KB301 for aesthetic indications Provide update on stra
Slide 5: Our technology platform enables noninvasive, redosable gene therapy
Our technology platform enables noninvasive, redosable gene therapy © 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 Lundstrom, K. Viral Vectors in Gene Therapy. Diseases 2018, 6, 42. Generation Bio (GBIO) Prospectus. (2020, June 11). Retrieved September 4, 2020, https://www.sec.gov/Archives/edgar/data/1733294/000119312520167812/d924849d424b4.h
Slide 7: Clinically validated platform targeting skin; broad tropism of HSV-1
Clinically validated platform targeting skin; broad tropism of HSV-1 could unlock additional target tissues Clinical Experience In development Research / Platform Development Efforts Topical Intradermal Inhaled The current vector has been clinically validated to target skin cells. Preclinical explor
Slide 8: Therapeutic pipeline
Therapeutic pipeline †: 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 studie
Slide 9: Aesthetics pipeline
Aesthetics pipeline © Copyright 2021 Krystal Biotech, Inc. All rights reserved. All pipeline compounds are investigational, being evaluated in clinical or pre-clinical studies.
Slide 10: 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 11: 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 © Copyright 2021 Krystal Biotech, Inc. All rights reserved.
Slide 12: Dystrophic epidermolysis bullosa (DEB)
Dystrophic epidermolysis bullosa (DEB) DEBRA International, http://www.debra-international.org/epidermolysis-bullosa/causes-and-subtypes.html; http://www.debra-international.org/what-is-eb/causes-and-subtypes/deb.html Pfendner EG, Lucky AW. Dystrophic Epidermolysis Bullosa. 2006 Aug 21 [Updated 2015
Slide 13: Beremagene geperpavec (B-VEC) for DEB
Beremagene geperpavec (B-VEC) for DEB Blister Untreated DEB Patient Skin B-VEC-treated Skin 1 Keratinocytes Dermoepidermal Junction Fibroblasts Anchoring Fibrils (Collagen VII) B-VEC 2 3 Nucleus COL7 transcripts COL7 protein Keratinocyte (or Fibroblast) Cell 4 1 B-VEC enters the compromised skin of
Slide 14: Topical B-VEC was evaluated in a Phase 1/2 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. Each patient on-study for ~6 months; 3 months of on-site visits followed by 3-month at-home imaging period Study PI: Dr. Peter Marinko
Slide 15: Repeat doses of topical B-VEC were well tolerated; COL7 expression established
Repeat doses of topical B-VEC were well tolerated; COL7 expression and molecular correction established In the Ph1/2 trial, the number of repeat doses per wound ranged from 4 to 41; the PFU per wound ranged from 1e8 to 8e8 No treatment-related serious AEs were reported; AEs deemed possibly related w
Slide 16: B-VEC showed statistically significant benefit in wound healing relative to placebo
B-VEC showed statistically significant benefit in wound healing relative to placebo B-VEC is an investigational therapy being studied in clinical trials © Copyright 2021 Krystal Biotech, Inc. All rights reserved. 78.6% of B-VEC treated wounds vs. 0% of placebo treated wounds were completely closed a
Slide 17: The pivotal GEM-3 study is ongoing; top line data expected in 4Q21
The pivotal GEM-3 study is ongoing; top line data expected in 4Q21 GEM-3 (NCT04491604) is a randomized, double-blind, intra-patient comparison of wounds randomized to receive either topical B-VEC or placebo Each patient on-study for approximately 7 months: the 6-month dosing period followed by a 30-
Slide 18: 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 Screening 6-month Treatment Period 30-day Safety OLE Dosing: Primary wounds will be treated once weekly with a fixed dose until wound closure; should a wound re-open, weekly dosing will resume at the assigned dose until wound
Slide 19: Autosomal Recessive Congenital Ichthyosis associated with TGM1 mutations
Autosomal Recessive Congenital Icthyosis associated with TGM1 mutations Autosomal Recessive Congenital Ichthyosis (ARCI) Associated with TGM1 The most common form of ARCI is caused by an inactivating mutation in the TGM1 gene encoding the enzyme transglutaminase-1, a protein that is essential for th
Slide 20: KB105 for TGM1 associated ARCI
KB105 for TGM1 associated ARCI 3 TGM1 transcripts are generated, which allows the cell to produce functional TGM1 protein that localizes to the cell membrane 2 KB105 is transported into the nucleus of transduced cells and the vector genome is deposited (episomally) 1 KB105 enters permeabilized skin
Slide 21: KB105 is being evaluated in a Phase 1/2 study
KB105 is being evaluated in a Phase 1/2 study The Ph1/2 trial (NCT04047732) is an open label, intra-patient comparison of KB105 and placebo Each patient on-study for four to six months Study PI: Dr. Amy Paller (Northwestern University) Design Enrollment Key Endpoints 4 TGM1-ARCI subjects were enroll
Slide 22: Phase 1 data shows repeat dosing of KB105 well tolerated
Phase 1 data shows repeat dosing of KB105 to be well tolerated; molecular and phenotypic improvement evident Three adult patients were enrolled and circular skin areas of 20cm2 each were chosen to receive placebo (n=1 or 2 per patient) or 2.0x10^9 PFU of topical KB105 (n=3 per patient) at various do
Slide 23: Initial Phase 2 data at higher dose shows similar phenotypic improvement
Initial Phase 2 data at higher dose and larger treatment area shows similar phenotypic improvement with rapid TGM-1 turnover KB105 is an investigational therapy being studied in clinical trials © Copyright 2021 Krystal Biotech, Inc. All rights reserved. Based on Phase 1 results, the dose, dosing fre
Slide 24: Leveraging platform to target new tissues and larger indications
Leveraging platform to target new tissues and larger indications © Copyright 2021 Krystal Biotech, Inc. All rights reserved.
Slide 25: HSV-1 has potential beyond rare skin diseases
HSV-1 has potential beyond rare skin diseases B-VEC (COL7) for DEB KB105 (TGM1) for TGM1-ARCI KB104 (SPINK5) for Netherton’s Disease Additional preclinical candidates Wild-type HSV-1 Krystal’s Current Platform HSV-1 has natural affinity for epithelial cells, in addition to several other cell types,
Slide 26: KB407 for cystic fibrosis
KB407 for cystic fibrosis Gene therapy approaches have been tried and failed in their attempts to replace CFTR protein Viral (adenovirus and AAV) and non-viral (DNA plasmids and stabilized mRNA) approaches have been tested in more than 25 clinical trials enrolling >470 patients Past approaches suffe
Slide 27: 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 expression and functional correction in 2D airway epithelial cell culture KB407-mediated functional correction of CF phenotype in clinically relevant 3D organotypic system (HUB) *Van Mourik et a
Slide 28: KB301 for aesthetic indications
KB301 for aesthetic indications KB301 aims increase neocollagenesis, thereby correcting the underlying molecular defect of the aged phenotype Dermal collagen, composed primarily of types 1 and 3 collagen fibrils, represents >90% (dry weight) of human skin Declining levels of collagen are caused by r
Slide 29: Financials and Milestones
Financials and Milestones © Copyright 2021 Krystal Biotech, Inc. All rights reserved.
Slide 30: Krystal summary
Krystal summary Current Status and Milestones Rare Skin B-VEC: Pivotal GEM-3 trial ongoing with topline data expected 4Q21. Commercial planning in US and EU underway KB105: Phase 2 study ongoing; Initiation of dosing in next Phase 2 cohort in 2022 KB104: IND anticipated in 2H21 Jeune Aesthetics, Inc
Slide 31: The Leader in Redosable Gene Therapy for Rare Disease
The Leader in Redosable Gene Therapy for Rare Disease July 2021 © Copyright 2021 Krystal Biotech, Inc. All rights reserved.