Financial Data and Key Metrics Changes - The company reported Q4 2025 net revenue of $107.1 million, marking a growth of almost 10% compared to the previous quarter and approximately 18% year-over-year [25][26] - Year-to-date net revenue for VYJUVEK reached $389.1 million, an increase of approximately 34% compared to full year 2024 revenue [25] - Gross margin for Q4 was 94%, down from 96% in Q3 2025 and 95% in Q4 2024, attributed to increased sales outside the U.S. which carry higher costs [26] Business Line Data and Key Metrics Changes - VYJUVEK continues to show strong performance with total net revenue since launch exceeding $730 million [6] - The company has signed distributor agreements covering more than 20 countries, aiming to expand to over 40 countries in 2026 [4] - The U.S. market saw over 660 reimbursement approvals, with more than 500 unique prescribers since launch [9] Market Data and Key Metrics Changes - In Europe, over 90 DEB patients have been prescribed VYJUVEK across Germany, France, and Japan, with sustained prescription growth in Germany [11][12] - The launch in Japan has been successful, with a unique distribution model enabling home delivery [13] - Pricing negotiations are ongoing in Germany and France, with expectations for agreements in the second half of 2026 and 2027 respectively [12][14] Company Strategy and Development Direction - The company is focused on expanding global access for VYJUVEK while advancing a disciplined pipeline of gene therapies [5] - Plans to initiate two additional registrational programs in cystic fibrosis and Hailey-Hailey disease later this year [5] - The company aims to build a durable commercial gene therapy business with strong capital allocation and operational improvements [5] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the ongoing demand for VYJUVEK, particularly in international markets, which are expected to drive revenue growth in 2026 [30] - The company is aware of the evolving utilization patterns among U.S. patients, shifting towards more intermittent treatment cycles [31] - Management remains optimistic about the potential of their pipeline programs, especially with recent FDA designations that could accelerate development timelines [33] Other Important Information - The company ended the year with $955.9 million in cash and investments, positioning it well for future commercial launches and pipeline milestones [29] - R&D expenses for Q4 were $14.8 million, while SG&A expenses were $41.4 million, reflecting increased headcount and marketing costs [27] Q&A Session Summary Question: Visibility into Q1 and 2026 revenue contribution from U.S. vs. ex-U.S. - Management indicated that while growth will predominantly come from ex-U.S. markets, demand in the U.S. is also accelerating, with expectations to break out revenue in Q1 [36] Question: Data supporting modified dosing regimens for ocular and NK - Management confirmed that the changes were based on patient feedback and the need for home administration, ensuring compliance and convenience [40] Question: Compliance rates in EU and Japan - Compliance in Europe has been consistent with the U.S., while Japan's two-week prescription requirement may affect long-term compliance [46] Question: Updates on pricing negotiations in Europe - Management expects to reach a pricing agreement in Germany in the second half of 2026, while negotiations in France may extend into 2027 [54] Question: Differentiating factors for dosing schedules in ocular studies - The decision for different dosing regimens was based on patient convenience and feedback, with no changes to the dose volume [70]

Fourth Quarter and Full Year 2025 Financial and Operating Results February 17, 2026 © Copyright 2026 Krystal Biotech, Inc. All rights reserved. Forward Looking Statements and Disclosures This presentation and our discussion contain forward-looking statements that involve substantial risks and uncertainties. Any statements about future expectations, plans, and prospects for Krystal Biotech, Inc. (together with its subsidiaries, the "Company"), including but not limited to, statements about our U.S., Germany, ...

Product Approval and Market Launch - VYJUVEK, the first redosable gene therapy, was approved by the FDA on May 19, 2023, for treating patients with dystrophic epidermolysis bullosa (DEB) aged six months and older[36]. - Since the launch of VYJUVEK in the United States, the company has reported $730.3 million in net product revenue[35]. - The company estimates over 3,000 DEB patients in the United States, with 1,200 identified at launch through claims analytics and pre-launch activities[30]. - VYJUVEK was launched in Germany in August 2025 and in France in October 2025, with over 1,000 DEB patients identified in these countries[31]. - The FDA granted a Rare Pediatric Disease Priority Review Voucher (PRV) for VYJUVEK, which was sold for $100 million in Q3 2023[36]. - The FDA approved a label update for VYJUVEK in September 2025, expanding the treatment population to include DEB patients from birth[37]. - VYJUVEK is the first corrective therapy for Dystrophic Epidermolysis Bullosa (DEB) approved worldwide, highlighting its unique market position[123]. Manufacturing and Technology - The company has established two in-house, commercial scale CGMP manufacturing facilities to support its gene therapy platform[1]. - The proprietary manufacturing process developed for VYJUVEK aims to reduce variability and costs associated with viral vector production[117]. - The production process for VYJUVEK includes a master virus seed stock (MVSS) and a complementing master cell bank (MCB), enabling cost-effective production within a six-week cycle[122]. - Significant investments have been made in developing a comprehensive manufacturing process for VYJUVEK, focusing on high-efficiency purification techniques[122]. - The ASTRA facility, completed in 2023, enhances manufacturing capacity and integrates the supply chain from raw materials to patient experience[116]. Clinical Trials and Research - The GEM-3 trial for topical B-VEC enrolled 31 participants, with positive results announced in November 2021 and full results published in December 2022[45]. - The open-label extension (OLE) study enrolled 47 participants, concluding in Q3 2023, with no new safety concerns noted and a continued favorable safety profile for B-VEC[47]. - The Japan OLE study was initiated in July 2023, with all four patients achieving complete wound closure at six months, mirroring the GEM-3 trial results[48]. - KB407, a gene therapy for cystic fibrosis, was dosed to the first patient in the Phase 1 CORAL-1 study in July 2023, with interim safety data showing mild to moderate adverse events[54]. - In Cohort 3 of the CORAL-1 study, KB407 demonstrated successful lung delivery and expression of wild-type CFTR protein, with transduction rates between 29.4% and 42.1%[56]. - The company is working on a repeat dosing study design for KB407, with plans to start enrollment in the potentially registrational CORAL-3 study in the first half of 2026[57]. - The company initiated the IOLITE Phase 3 study for KB803, enrolling approximately 16 patients, with top-line results expected in late 2026[70]. - The EMERALD-1 study for KB801 aims to assess the safety and efficacy of the treatment, with primary efficacy measured by the proportion of patients achieving complete corneal epithelium healing at eight weeks[75]. Intellectual Property and Patent Strategy - The technology platform, VYJUVEK, is primarily protected by composition of matter and methods of use patents, with key patents expiring in 2036[138][139]. - The company actively seeks patent protection for its product candidates and proprietary technologies, evaluating its patent strategy based on market factors[140]. - Krystal holds multiple patents for B-VEC, with expiration dates set for December 28, 2036, across various regions including the United States, Europe, Japan, and Australia[142][143]. - The company has patents for KB407 and KB408, focusing on the use of herpes virus vectors for treating cystic fibrosis and diseases affecting the airways, with expiration dates in 2040[144][145]. - The expiration of key patents in 2036 and 2040 will be critical for future revenue streams and market exclusivity[143][144]. - The company is aware of potential intellectual property claims from third parties that could impact the commercialization of its product candidates[136]. Regulatory Environment and Compliance - The FDA regulates biologic products, including gene therapy, under the FDCA and PHSA, requiring substantial time and financial resources for compliance and approval[154]. - The process for marketing a biologic product candidate in the U.S. involves preclinical tests, IND application submission, and clinical trials, which must comply with CGMP requirements[157]. - The FDA reviews Biologics License Applications (BLAs) within 60 days to determine completeness before an in-depth review begins[167]. - The FDA requires compliance with CGMP regulations for manufacturing processes before approving a BLA[171]. - Fast Track designation facilitates development and review of drugs for serious conditions, allowing for more frequent interactions with the FDA[175]. - Orphan Drug designation provides exclusivity for seven years for products treating rare diseases affecting fewer than 200,000 individuals in the U.S.[177]. Market Competition and Risks - The biotechnology and pharmaceutical industries are highly competitive, with competitors having greater financial resources and larger R&D organizations[122]. - The company faces significant risks in identifying and developing additional product candidates, which could impair its ability to expand and achieve strategic objectives[203]. - The Patient Protection and Affordable Care Act (ACA) includes provisions for biosimilars, which could lead to competition for VYJUVEK and other products[208]. - The company is aware of several competitors with greater financial and technical resources, which may limit its commercial opportunities[211]. - The expiration or successful challenge of the company's patent rights could lead to litigation and limit demand for its products[213]. Financial and Operational Considerations - The commercial success of VYJUVEK is critical for the company's near-term prospects and future growth[201]. - The company has invested substantial resources in VYJUVEK, and its future revenue generation is heavily reliant on the product's commercial success[201]. - The company has 295 full-time employees focused on various operational areas, including research, development, and commercialization of VYJUVEK[197]. - The company is committed to employee development and offers competitive compensation packages to attract and retain talent[198]. - Regulatory changes and healthcare reforms in the U.S. and abroad pose ongoing uncertainties for the pharmaceutical industry, impacting the company's operations[192].

Financial Performance - The company reported $107.1 million in net product revenue for the fourth quarter of 2025, an increase from $91.1 million in the same quarter of 2024, representing a growth of approximately 17.5% [6][19][36] - For the full year 2025, net product revenue totaled $389.1 million, up from $290.5 million in 2024, indicating a year-over-year increase of about 34% [19][38] - The gross margin for the fourth quarter and full year of 2025 was 94% [6] - The company ended the quarter with a strong balance sheet, holding $955.9 million in cash and investments [1][19] Product Development and Pipeline - VYJUVEK, the company's gene therapy product for dystrophic epidermolysis bullosa (DEB), has secured over 660 reimbursement approvals in the U.S. and has seen high patient demand in Germany, France, and Japan, with over 90 patients prescribed the therapy [6][22] - The FDA granted Regenerative Medicine Advanced Therapy (RMAT) designation to KB707 for advanced non-small cell lung cancer (NSCLC) and Fast Track Designation to KB111 for Hailey-Hailey disease (HHD) [1][12][13] - The company is advancing its clinical trials, including a Phase 1 study for KB407 in cystic fibrosis, which showed successful lung delivery and expression of the CFTR protein [5][14] Future Outlook - The company aims to launch multiple products and treat over 10,000 patients with rare diseases by the end of 2030, with ongoing discussions for pricing negotiations in Germany and France expected to continue into 2026 and 2027 [2][6] - The company is on track to expand its specialty distributor network to cover over 40 countries by the end of 2026 [6] - The company expects to report interim efficacy data for KB707 and plans to initiate a registrational study for KB111 in HHD in the second half of 2026 [13][14]

Core Insights - Krystal Biotech, Inc. is a clinical-stage biotechnology company focused on gene therapies for rare diseases, with its lead product B-VEC in Phase III trials for dystrophic epidermolysis bullosa [1] - The consensus price target for Krystal Biotech's stock has increased from $269.25 a year ago to $327 last month, indicating growing analyst optimism [2][5] - The company projects 2025 revenues between $388 million and $389 million, supported by a strong cash reserve of $955 million, which is crucial for advancing its pipeline [3][5] - The clinical pipeline, including positive interim results for KB407, presents multibillion-dollar opportunities, with analysts expressing confidence in the company's growth potential [4][5]

Core Insights - Krystal Biotech and ARS Pharmaceuticals are gaining attention due to their unique therapies with blockbuster potential [1][2] Krystal Biotech - Krystal Biotech has developed Vyjuvek, the first topical genetic therapy approved by the FDA for dystrophic epidermolysis bullosa, a rare skin disorder [2] - The company is working on expanding the labels for Vyjuvek while using its growing revenues to fund its pipeline [3] - Krystal's KB707 candidate is aimed at treating advanced or metastatic non-small cell lung cancer and has received a Regenerative Medicine Advanced Therapy designation from the FDA [6] - Preliminary sales for Vyjuvek are expected to reach $388 million to $389 million, indicating a 34% increase at the midpoint [7] - Analysts predict annual EPS for Krystal will be between $6.46 and $8.92, a 156% increase over the prior year at the midpoint [8] - The stock has increased over 10% year to date and nearly 80% over the past year, currently trading around $270 per share [8] ARS Pharmaceuticals - ARS Pharma focuses on preventing allergic reactions that can lead to anaphylaxis, with its product Neffy being the first non-injection epinephrine treatment approved by the FDA [2][9] - The company has faced challenges with the FDA regarding its advertising for Neffy, which was deemed misleading [11] - ARS Pharma's stock has decreased over 24% year to date and over the past 12 months, indicating a riskier investment compared to Krystal Biotech [12] - Revenue for ARS Pharma reached $51.8 million in the first nine months of 2025, a significant increase from $568,000 in the same period in 2024 [13] - The global market size for epinephrine is projected to be $2.48 billion in 2025, highlighting ARS Pharma's growth potential [13] - ARS Pharma is also looking to expand indications for Neffy and has a pipeline candidate, ARS-2, for treating chronic urticaria [14]

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Core Viewpoint - Krystal Biotech, Inc. will report its fourth quarter and full year 2025 financial results on February 17, 2026, before U.S. markets open, and will host a conference call and webcast to discuss these results and provide a business update [1]. Company Overview - Krystal Biotech, Inc. is a fully integrated, commercial-stage global biotechnology company focused on discovering, developing, and commercializing genetic medicines for diseases with high unmet medical needs [3]. - The company's first commercial product, VYJUVEK, is the first-ever redosable gene therapy and the first genetic medicine approved in the U.S., Europe, and Japan for treating dystrophic epidermolysis bullosa [3]. - Krystal Biotech is advancing a robust pipeline of investigational genetic medicines and is headquartered in Pittsburgh, Pennsylvania [3].

Core Insights - The FDA granted Regenerative Medicine Advanced Therapy (RMAT) designation to KB707, an immunotherapy for advanced or metastatic non-small cell lung cancer (NSCLC) developed by Krystal Biotech [1][2] Group 1: FDA Designation and Implications - The RMAT designation reflects the urgent need for new NSCLC therapies and promising early clinical evidence of KB707's efficacy [2] - This is the second RMAT designation for a Krystal program, which is expected to accelerate development and shorten the approval timeline [2] - The RMAT designation provides benefits similar to Fast Track and Breakthrough Therapy designations, including potential for rolling review and intensive FDA guidance [2] Group 2: Clinical Evidence and Ongoing Studies - Early clinical evidence supporting the RMAT designation comes from the ongoing KYANITE-1 study, showing meaningful antitumor activity and durable responses in heavily pre-treated advanced NSCLC patients [3] - Enrollment in the KYANITE-1 study is ongoing, with further details to be presented at upcoming scientific conferences [3] Group 3: Company Overview - Krystal Biotech is a global biotechnology company focused on developing genetic medicines for diseases with high unmet medical needs [4] - The company's first commercial product, VYJUVEK, is the first redosable gene therapy approved in the U.S., Europe, and Japan for dystrophic epidermolysis bullosa [4] - Krystal Biotech is advancing a robust pipeline of investigational genetic medicines [4]

Core Insights - Krystal Biotech, Inc. (NASDAQ:KRYS) is recognized as one of the top 10 growth stocks on NASDAQ for the next decade, with multiple analysts raising their price targets significantly, indicating strong future potential [1][3][5] Price Target Adjustments - Goldman Sachs raised its price target from $206 to $327, suggesting a 16.7% upside from current levels [1] - Clear Street increased its price target from $288 to $338, indicating a further 20.7% upside [3] - Bank of America adjusted its price target from $288 to $318, reflecting a 13.8% upside [5] Growth Programs - The company is focusing on short-term growth through three registrational programs: KB801, KB803, and KB407, with potential product launches expected around 2028 [1] - Ophthalmic programs are viewed as de-risked and scalable, leveraging the existing Vyjuvek commercial platform [2] Market Opportunities - KB407 is seen as a promising opportunity in cystic fibrosis, with long-term potential contingent on additional FEV1 data [2] - The neurotrophic keratitis market is a significant source of investor interest, particularly driven by KB801, which contributed to the upward revision of price targets [4] Company Overview - Krystal Biotech, Inc. is a commercial-stage biotechnology company focused on developing genetic medicines for diseases with high unmet medical needs, with products including VYJUVEK, KB105, KB104, KB407, and KB707 [6]