Krystal Biotech, Inc. · NASDAQ:KRYS
Krystal Biotech — Corporate Update (March 2022)
27 slides from Krystal Biotech's corporate update, Mar 28, 2022
Slide 1: AAD GEM-3 Phase 3 Data Conference Call
AAD GEM-3 Phase 3 Data Conference Call © Copyright 2022 Krystal Biotech, Inc. All rights reserved. Exhibit 99.1
Slide 2: 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 about the development of the C
Slide 3: Agenda
Agenda 1 Introductory Comments Krish Krishnan – Chairman and CEO 2 DEB Background and GEM-3 Results (AAD Late-Breaking Presentation) Dr. Hubert Chen – SVP, Clinical Development 3 Market Opportunity and Commercial Preparations Andy Orth – Chief Commercial Officer 4 Closing and Q&A Krish Krishnan – Ch
Slide 4: Krystal Biotech: Bringing Transformative, Redosable Gene Therapies to Underserved Patient Populations
Krystal Biotech: Bringing Transformative, Redosable Gene Therapies to Underserved Patient Populations Cash position as of December 31, 2021 Leader in the science of redosable gene therapies – powered by proprietary HSV-1 vector technology Initial focus on rare dermatologic diseases established clini
Slide 5: Lead Program: B-VEC for DEB
Lead Program: B-VEC (beremagene geperpavec) for DEB A Topical Redosable Gene Therapy Designed to Treat Dystrophic EB © Copyright 2022 Krystal Biotech, Inc. All rights reserved. Topically applied B-VEC gel designed to induce local COL7 expression and molecular correction > 2,500 patients diagnosed ac
Slide 6: Dr. Hubert Chen, SVP, Clinical Development
Dr. Hubert Chen SVP, Clinical Development © Copyright 2022 Krystal Biotech, Inc. All rights reserved.
Slide 7: Dystrophic Epidermolysis Bullosa and B-VEC
Dystrophic Epidermolysis Bullosa and B-VEC Dystrophic epidermolysis bullosa (DEB) is a serious, ultra-rare genetic blistering disease caused by mutations in the COL7A1 gene which lead to skin fragility and wounds1-3 Patients with DEB are at increased risk for serious complications, including aggress
Slide 8: GEM-3 Study Design
GEM-3 Study Design B-VEC, beremagene geperpavec; DEB, dystrophic epidermolysis bullosa GEM-3 (NCT04491604) is a Phase 3, multicenter, randomized, double-blind, placebo-controlled intra-patient study evaluating the efficacy and safety of B-VEC in patients with DEB B-VEC Placebo Randomized, double-bli
Slide 9: Patient Disposition
Patient Disposition 31 patients were randomized and made up the intent-to-treat (ITT) population used for all primary and secondary efficacy analyses The safety population was the same as the ITT population and used for all safety analyses Three patients withdrew from the study for nondrug-related r
Slide 10: Baseline Demographics and Clinical Characteristics
Baseline Demographics and Clinical Characteristics *In a small number of patients, the pre-defined threshold values for wound area/size category fell in between the size of the two wounds B-VEC, beremagene geperpavec; DDEB, dominant dystrophic epidermolysis bullosa; RDEB, recessive dystrophic epider
Slide 11: Significantly Greater Complete Wound Healing with B-VEC Treatment
Significantly Greater Complete Wound Healing with B-VEC Treatment The proportion of primary wounds with complete wound healing was significantly greater with B-VEC than placebo at both 3- and 6-month timepoints (p <0.005) In the patient with DDEB, primary endpoint of complete wound healing at 6 mont
Slide 12: Primary Wound Pairs at Baseline and 6 Months
Primary Wound Pairs (15 – 30 cm2) at Baseline and 6 Months Baseline 6 Months Baseline 6 Months Patient A Knee Patient A Knee Patient B Lower abdomen Patient B Sternum Patient C Lower trapezius Patient C Upper paraspinal B-VEC, beremagene geperpavec 12 B-VEC Placebo
Slide 13: Treatment with B-VEC Demonstrated Durability of Response
Treatment with B-VEC Demonstrated Durability of Response 49.7% of B-VEC treated wounds compared to 7.1% of placebo treated wounds demonstrated durability of response, defined as wounds that met complete wound healing at both 3 months (key secondary endpoint) and 6 months (primary endpoint) Nearly ha
Slide 14: Consistent Evidence of a Treatment Response with B-VEC Across Subgroups
Consistent Evidence of a Treatment Response with B-VEC Across Subgroups Treatment response was in favor of B-VEC for all gender, age, and wound area/size subgroups, however the individual subgroups were not powered to demonstrate statistical significance Data as of database lock on 19Nov2021; data i
Slide 15: Pain and PRO Assessments Demonstrated Improvement
Pain and PRO Assessments Demonstrated Improvement Consistent with a Wound Healing Response Baseline VAS score of enrolled patients were approximately 2 to 3 on average A trend towards decreased pain in B-VEC treated versus placebo treated wounds was observed across Weeks 22, 24, and 26; improvement
Slide 16: B-VEC was Generally Well-Tolerated
B-VEC was Generally Well-Tolerated Total Patients (n=31) Total number of adverse events (AEs) 45 Patients with ≥ 1 AE, n (%) 18 (58.1) Serious AEs 3 (9.7) Severe AEs 2 (6.5) Drug-related AEs 1 (3.2) AE leading to treatment discontinuation 0 (0) Death 0 (0) Data as of database lock on 19Nov2021 AEs,
Slide 17: Andy Orth, Chief Commercial Officer
Andy Orth Chief Commercial Officer © Copyright 2022 Krystal Biotech, Inc. All rights reserved.
Slide 18: Dystrophic EB Patient Population and B-VEC Opportunity
Dystrophic EB Patient Population and B-VEC Opportunity B-VEC is an investigational therapy being studied in clinical trials Genetic prevalence suggests ~3,000 patients in the US Initial claims data mining points to >1,000 known patients diagnosed with DEB in the US and >2500 patients worldwide Payer
Slide 19: Preparing for U.S. Launch
Preparing for U.S. Launch Education 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 Leader engagement underway Payer education and engagement Initial Launch
Slide 20: Decode DEB genetic testing program
No-charge genetic testing program available to eligible U.S. residents who are suspected of having EB and have not yet been genetically confirmed Comprehensive testing panel to identify Dystrophic EB or conditions with similar phenotypes, including other EB types and some non-EB genetic blistering c
Slide 21: DEB Disease Awareness Update
DEB Disease Awareness Update DEBFacts Web Sites Launched early 2022 DEB Caused by COL7 Dysfunction Importance of accurately diagnosing DEB Patients Every Wound Matters/Has Consequences DDEB RDEB For internal use only >1,000 visits since launch. Media investment and other websites traffic drivers lau
Slide 22: Proactive Education at American Academy of Dermatology
Medical affairs booth focused on disease state and Krystal Biotech awareness, highlighting: Patient journey cases Decode DEB genetic testing program Mechanism of disease and mechanism of action animations/videos Pipeline and clinical trial information Engaging HCPs serving the dystrophic EB communit
Slide 23: Significantly Expanding In-house Manufacturing Capacity and Expertise
Existing ANCORIS Facility New ASTRA Facility Significantly Expanding In-house Manufacturing Capacity and Expertise ~10,000 sq ft GMP facility Process validation batches complete Designed to support B-VEC launch in U.S. and Europe+ Comfortably within biologics gross margin range ~150,000 sq ft GMP fa
Slide 24: Krish Krishnan, Chairman and CEO
Krish Krishnan Chairman and CEO © Copyright 2022 Krystal Biotech, Inc. All rights reserved.
Slide 25: Key Takeaways
Key Takeaways B-VEC full Phase 3 data is compelling Well-prepared to bring B-VEC to the DEB community Robust upcoming catalysts for B-VEC and pipeline Redosable gene delivery technology/platform has broad potential © Copyright 2022 Krystal Biotech, Inc. All rights reserved.
Slide 26: Upcoming Milestones
Upcoming Milestones Timing Program Event 2Q22 B-VEC for Dystrophic EB Present more detailed GEM-3 safety results at SID (May 18-21) 2Q22 B-VEC for Dystrophic EB File BLA with U.S. FDA 2Q22 KB407 for cystic fibrosis Initiate Phase 1 clinical trial in Australia 2H22 B-VEC for Dystrophic EB Fila MAA wi
Slide 27: Questions & Answers
Questions & Answers © Copyright 2022 Krystal Biotech, Inc. All rights reserved.