Krystal Biotech, Inc. · NASDAQ:KRYS
Krystal Biotech — Technical Presentation (October 2020)
22 slides from Krystal Biotech's technical presentation, Oct 1, 2020
Slide 1: Topical gene therapy - A new direction in the management of dystrophic epidermolysis bullosa
Topical gene therapy - A new direction in the management of dystrophic epidermolysis bullosa Suma Krishnan – Chief Operating Officer, Krystal Biotech Peter Marinkovich – Assoc Professor of Dermatology, Head of Bullous Disease Clinic, Stanford October 1st, 2020 Exhibit 99.1
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: There are four types of Epidermolysis Bullosa (EB)
There are four types of Epidermolysis Bullosa (EB)1,2 Type Point of tissue separation Gene mutations Proteins involved in pathogenesis EB simplex (EBS) Epidermis KRT5 KRT14 Desmoplakin Plakophilin 1 Plakoglobin Transglutaminase 5 Keratin 5/14 Plectin Exophilin 5 Junctional EB (JEB) Lamina lucida LAM
Slide 4: Mutations in the COL7A1 gene cause DEB
Normal skin Affected skin Mutations in the COL7A1 gene cause DEB1,2 DEB is a rare genetic disease caused by a mutation in the COL7A1 gene that codes for Type VII collagen (COL7) COL7 plays an important role in strengthening and stabilizing the skin The diagnosed prevalence of DEB is estimated to be
Slide 5: There are two forms of DEB with overlapping severity
Autosomal Dominant DEB – DDEB Reduced COL7 Symptoms begin at birth Severe cases of DDEB can be as debilitating as RDEB There are two forms of DEB with overlapping severity1,2 Images taken from: Shinkuma S 20151 and Fine JD 20102 1. Shinkuma S. Clin Cosmet Investig Dermatol 2015; 8:275–284. 2. Fine J
Slide 6: There is currently no treatment to address the underlying cause of DEB
There is currently no treatment to address the underlying cause of DEB1 There are currently no approved treatments to address skin manifestations of patients with DEB and care is currently restricted to: Wound care – by meticulous bandaging of the skin Pain management Management of infections Preven
Slide 7: About Krystal Biotech, Inc.
About Krystal Biotech, Inc. Founded in 2016 to find an innovative way to treat patients with Dystrophic Epidermolysis Bullosa Investigational treatment (presently known as B-VEC) is designed to be: “Off-the-shelf” topical gel to deliver the missing or mutated COL7A1 gene to the skin wounds Modified
Slide 8: Krystal's in vivo approach vs. other ex vivo approaches
Krystal’s in vivo approach vs. other ex vivo approaches1,2 Transporting to manufacturing facility Viral gene integration GMP graft production Transport back to clinical site Ex vivo skin correction Graft placement under general anesthesia in operating room with 1 week hospitalization Skin harvesting
Slide 9: Krystal: Topical gel gene therapy
Krystal: Topical gel gene therapy In vivo gene therapy Vector Modified herpes simplex virus 1 (HSV-1) Skin cells targeted Fibroblast and keratinocytes Time taken Immediate use Administration method Topical Need for hospitalization? No – out-patient or home application Need for anaesthesia? No B-VEC
Slide 10: As new genetic based therapies emerge, early genetic diagnosis will become important
As new genetic based therapies emerge, early genetic diagnosis will become important1 1. Has C et al. Br J Dermatol 2020; 182:574–592. 2. Feinstein JA. JAMA Dermatol 2019; 155:196–203. 3. DEBRA International. Laboratory diagnosis for people living with epidermolysis bullosa (EB). Available at: https
Slide 11: B-VEC for treatment of dystrophic epidermolysis bullosa (DEB)
For treatment of dystrophic epidermolysis bullosa (DEB) B-VEC Ph 1/2 Data (previously KB103)* USAN & INN: beremagene geperpavec * RMAT designation PRIME eligibility Fast track designation granted Orphan Drug Designation in US and EU Rare Pediatric Disease Designation in US Eligible for Priority Revi
Slide 12: Topical B-VEC was evaluated in a Phase 1/2 study at Stanford
Topical B-VEC was evaluated in a Phase 1/2 study at Stanford1 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.
Slide 13: In Phase 1/2, Topical B-VEC was safe; COL7 expression and molecular correction observed
In Phase 1/2, Topical B-VEC was safe; COL7 expression and molecular correction observed No treatment-related serious AEs were reported; AEs deemed possibly related were mild (n=7) or moderate (n=1) and self limiting No immune response or blistering observed around the sites of administration followi
Slide 14: Statistically significant reduction in wound area achieved in Weeks 8, 10 and 12
Statistically significant reduction in wound area achieved in Weeks 8,10 and 12 Median change in wound area across Phase 1/2 study (efficacy observed in both recurring and chronic wounds) B-VEC is an investigational therapy being studied in clinical trials Median change from Baseline in wound area 1
Slide 15: B-VEC: Sustained closure in a recurring wound in Ph 1/2
B-VEC: Sustained closure in a recurring wound in Ph 1/2 B-VEC is an investigational therapy being studied in clinical trials
Slide 16: B-VEC: wound healing observed in a large chronic wound treated in Ph 1/2
B-VEC: wound healing observed in a large chronic wound treated in Ph 1/2 Large chronic wound (>60 cm2) present for >5 years covering the left side of patient’s torso B-VEC is an investigational therapy being studied in clinical trials
Slide 17: B-VEC: Long term durability of healing observed in chronic wounds
Before Following B-VEC treatment B-VEC: Long term durability of healing observed in chronic wounds Illustrative Durability: ~ 18 months (chronic wounds) Long term durability observed anecdotally on a majority of chronic wounds treated in Phase 1/2 clinical study B-VEC is an investigational therapy b
Slide 18: GEM-3: B-VEC Phase III trial information
Gem-3: B-vec phase iii TRIAL INFORMATION B-VEC is an investigational therapy being studied in clinical trials
Slide 19: The Pivotal GEM-3 study is currently enrolling
The Pivotal GEM-3 study is currently enrolling1 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-day safety follow u
Slide 20: Key inclusion/exclusion criteria
Key inclusion/exclusion criteria1 B-VEC is an investigational therapy being studied in clinical trials Clinical diagnosis of DEB Confirmation of DEB diagnosis (DDEB or RDEB) by genetic testing including COL7A1 Age ≥6 months and older At least two cutaneous wounds (recurrent or chronic) that are: Loc
Slide 21: Phase design
Phase 3 design 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 closure The fixed dose per wound is dependent on the size o
Slide 22: Enroll in our Phase III multicenter B-VEC clinical trial
Enroll in our Phase III multicenter B-VEC clinical trial1 Brittani Agostini Clinical Operations Manager bagostini@krystalbio.com 412-586-5830 Suma Krishnan Founder and Chief Operations Officer skrishnan@krystalbio.com 415-310-7747 Phase 3 Clinical Trial Site Locations United states, California Stanf
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