Krystal Biotech, Inc. (KRYS) came out with quarterly earnings of $1.20 per share, missing the Zacks Consensus Estimate of $1.38 per share. This compares to earnings of $0.03 per share a year ago. These figures are adjusted for non-recurring items.This quarterly report represents an earnings surprise of -13.04%. A quarter ago, it was expected that this company would post earnings of $1.29 per share when it actually produced earnings of $1.52, delivering a surprise of 17.83%.Over the last four quarters, the c ...

Financial Data and Key Metrics Changes - The net product revenue for the first quarter of 2025 was $88.2 million, representing a 95% increase from the first quarter of 2024 [49] - Cost of goods sold was $5 million, up from $2.4 million in the prior year's first quarter, with gross margin remaining consistent at 94% [49] - Research and development expenses increased to $14.3 million from $11 million in the prior year, primarily due to personnel-related expenses and clinical development costs [49] - General and administrative expenses rose to $32.7 million from $26.1 million in the prior year, driven by personnel-related expenses and increased professional service expenses [49] - Net income for the quarter was $35.7 million, equating to $1.24 per basic share and $1.20 per diluted share [49] - The company ended Q1 with $765.3 million in total cash plus investments, indicating strong financial health for upcoming launches and clinical programs [49] Business Line Data and Key Metrics Changes - The U.S. patient access to VYJUVEC exceeded 540 patients with reimbursement approvals as of April 2025, maintaining strong nationwide commercial and Medicaid coverage [23] - The company observed a slowdown in reimbursement approvals in Q1, attributed to the pace of patient start forms received [32] - The treatment success of VYJUVEC is leading to meaningful treatment pauses and maintenance treatment, which is expected to drive long-term growth [25][26] Market Data and Key Metrics Changes - The company plans to launch VYJUVEC in Germany and France in Q3 2025, with expectations that the opportunity in the EU could exceed current market anticipations [10] - The regulatory review in Japan is progressing well, with approval expected in Q3 2025 [10] Company Strategy and Development Direction - The company aims to build a geographically diversified business to limit exposure to regulatory or trade dynamics in any single market [22] - The focus is on establishing VYJUVEC as a lifelong first-line therapy for wound management, with ongoing efforts to enhance patient experience [15][22] - The clinical pipeline includes upcoming readouts for CF, AATD, aesthetic skin conditions, and ocular lesions in DEB, with optimism surrounding molecular data updates [16][40] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the long-term growth potential driven by patient outcomes and the effectiveness of VYJUVEC therapy [52] - The company is insulated from external turbulence due to all commercial and pipeline products being manufactured in the U.S. [20] - Management acknowledged the challenges in patient start forms but remains optimistic about the total market opportunity and long-term profitability [59] Other Important Information - The company has had seven consecutive quarters of profitable EPS, indicating strong financial management [21] - The upcoming ASCO presentation will showcase efficacy and safety data from inhaled KB707, highlighting the company's commitment to innovative therapies [47] Q&A Session Summary Question: Impact of insurance changes on VYJUVEC sales in Q1 - Management acknowledged that insurance changes had an impact but noted it was less prominent than in the previous year due to existing contracts [57] Question: Slowing reimbursement approvals and addressable population - Management clarified that while reimbursement approvals have slowed, the total market opportunity remains robust, with approximately 1,200 identified patients in the U.S. [59] Question: Long-term compliance rates and patient restart experiences - Management indicated that compliance rates are ahead of expectations, with patients pausing and restarting treatment based on individual needs [65][66] Question: Trajectory of the German launch compared to the U.S. - Management expects the German launch to have a strong trajectory due to lower patient identification issues and existing demand [70] Question: Increase in salesforce headcount and its purpose - Management confirmed that the increase in salesforce is aimed at enhancing educational efforts in the community rather than responding to competitive pressures [74][76] Question: Utilization of VYJUVEC in light of competitor products - Management does not view the newly approved competitor product as a direct threat due to significant differences in safety and efficacy [103][104]

Financial Data and Key Metrics Changes - The net product revenue for the first quarter of 2025 was $88.2 million, representing a 95% increase from the first quarter of 2024 [46] - Cost of goods sold was $5 million, up from $2.4 million in the prior year's first quarter, with gross margin remaining consistent at 94% [46] - Research and development expenses increased to $14.3 million from $11 million, primarily due to higher personnel-related expenses and clinical development costs [46] - General and administrative expenses rose to $32.7 million from $26.1 million, driven by increased personnel-related expenses and charitable contributions [46] - Net income for the quarter was $35.7 million, translating to $1.24 per basic share and $1.20 per diluted share [46] - The company ended Q1 with $765.3 million in total cash and investments, indicating strong financial stability [46] Business Line Data and Key Metrics Changes - The U.S. patient access to VYJUVEC continued to grow, with reimbursement approvals exceeding 540 as of April 2025 [21] - The access landscape in the U.S. remains strong, with full nationwide commercial and Medicaid coverage [21] - Treatment success on VYJUVEC is leading to meaningful treatment pauses and maintenance treatment, which is expected to drive long-term growth [23][24] Market Data and Key Metrics Changes - The company plans to launch VYJUVEC in Germany and France in Q3 2025, with expectations that the opportunity in the EU could be larger than currently anticipated [7] - Regulatory review in Japan is progressing well, with approval expected in Q3 2025 [7] Company Strategy and Development Direction - The company is focused on establishing VYJUVEC as a lifelong first-line therapy for wound management and is enhancing patient experience through various support initiatives [12][24] - Upcoming clinical readouts for CF, AATD, aesthetic skin conditions, and ocular lesions are anticipated in 2025, with optimism surrounding the clinical pipeline [13][38] - The company aims to build a geographically diversified business to limit exposure to regulatory or trade dynamics in any single market [19] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the long-term growth potential driven by patient outcomes and the upcoming launches in Europe [49] - The company is insulated from external turbulence due to all commercial and pipeline products being manufactured in the U.S. [17] - Management acknowledged the unpredictability of patient pausing patterns but remains optimistic about long-term growth [11] Other Important Information - The company has had seven consecutive quarters of profitable EPS, indicating strong financial management [18] - The launch of VYJUVEC in the U.S. has been characterized by strong patient outcomes, which are expected to support long-term market adoption [50] Q&A Session Summary Question: Impact of insurance changes on VYJUVEC sales in Q1 - Management noted that while there was an impact from insurance changes, it was less prominent than in previous years due to an established JCORD [55] Question: Slowing new reimbursement approvals - Management clarified that the slowing approvals are due to longer interaction times with physicians as the company reaches deeper into communities [57] Question: Long-term compliance rate expectations - Management indicated that compliance is ahead of expectations, with a target of 50% compliance in the long term [62] Question: Trajectory of the German launch compared to the U.S. - Management expects the German launch to have a similar growth target of achieving 60% market share in two years, with existing demand already noted [66] Question: Increase in Salesforce headcount - Management confirmed that the increase in headcount is focused on enhancing field sales efforts to engage more effectively with healthcare providers [70] Question: Compliance tracking on a quarterly basis - Management stated that while compliance has been consistent since launch, quarterly behavior may vary due to patient-specific factors [80] Question: EU launch and patient start cadence - Management acknowledged that the time to secure the first appointment with a physician may be a gating factor but is actively addressing this issue [90] Question: Coexistence with competitor products - Management emphasized that they do not consider the newly approved competitor product as direct competition due to significant differences in safety and efficacy [96]

[Financial and Operating Highlights](index=1&type=section&id=Financial%20and%20Operating%20Highlights) [First Quarter 2025 Overview](index=1&type=section&id=First%20Quarter%202025%20Overview) Krystal Biotech reported strong Q1 2025 results, with VYJUVEK European approval, **$88.2 million** revenue, and pipeline advancements Q1 2025 Key Metrics | Metric | Value | | :--- | :--- | | VYJUVEK Q1 Revenue | $88.2 million | | VYJUVEK Revenue Since Launch | $429.4 million | | Cash and Investments (End of Q1) | $765.3 million | - VYJUVEK received approval from the European Commission for the treatment of Dystrophic Epidermolysis Bullosa (DEB) patients from birth[1](index=1&type=chunk)[3](index=3&type=chunk) - The company advanced its pipeline with a second ophthalmic program, KB801, entering clinical trials for neurotrophic keratitis[1](index=1&type=chunk)[2](index=2&type=chunk) - Multiple clinical readouts are anticipated in 2025 across programs for cystic fibrosis (CF), alpha-1 antitrypsin deficiency (AATD), ocular complications of DEB, and aesthetics[1](index=1&type=chunk) [Pipeline and Commercial Update](index=1&type=section&id=Pipeline%20and%20Commercial%20Update) [VYJUVEK® for Dystrophic Epidermolysis Bullosa (DEB)](index=1&type=section&id=VYJUVEK%C2%AE%20for%20Dystrophic%20Epidermolysis%20Bullosa%20%28DEB%29) VYJUVEK achieved significant Q1 2025 commercial and regulatory milestones, including **$88.2 million** revenue and European approval VYJUVEK Q1 2025 Performance | Metric | Value | | :--- | :--- | | Net Product Revenue | $88.2 million | | Gross Margin | 94% | | U.S. Reimbursement Approvals | >540 | | Patient Compliance Rate | 83% | - The European Commission approved VYJUVEK for DEB patients from birth; the first European launch is on track for Germany in mid-2025[3](index=3&type=chunk) - A decision on the Japan New Drug Application (JNDA) is anticipated from Japan's PMDA in the second half of 2025[3](index=3&type=chunk) [Respiratory Pipeline](index=2&type=section&id=Respiratory%20Pipeline) The company is advancing its respiratory pipeline with ongoing enrollment for KB407 (CF) and KB408 (AATD) studies - **KB407 (Cystic Fibrosis):** Enrollment continues in Cohort 3 of the CORAL-1 study, with an interim molecular data readout anticipated in mid-2025[6](index=6&type=chunk) - **KB408 (AATD):** Enrollment is ongoing for Cohorts 2 and 3 of the SERPENTINE-1 study. Molecular results for these new patients are expected later in 2025[6](index=6&type=chunk) [Ophthalmology Pipeline](index=2&type=section&id=Ophthalmology%20Pipeline) The ophthalmology pipeline expanded with KB801 IND clearance and anticipated first patient dosing for KB801 and KB803 in May 2025 - **KB803 (Ocular DEB):** The first patient is expected to be dosed in the registrational Phase 3 IOLITE study later in May 2025[8](index=8&type=chunk) - **KB801 (Neurotrophic Keratitis):** The FDA cleared the IND for this second ophthalmology program. KB801 is a gene therapy eye drop designed to deliver nerve growth factor (NGF)[8](index=8&type=chunk)[9](index=9&type=chunk) - The first patient in the Phase 1/2 EMERALD-1 study for KB801 is expected to be dosed later in May 2025[13](index=13&type=chunk) [Oncology Pipeline](index=3&type=section&id=Oncology%20Pipeline) The oncology pipeline is advancing with ongoing enrollment for KB707 in two Phase 1/2 studies and a clinical update expected in June 2025 - Enrollment is ongoing in two Phase 1/2 studies for KB707: KYANITE-1 (inhaled for lung tumors) and OPAL-1 (intratumoral for solid tumors)[11](index=11&type=chunk)[13](index=13&type=chunk) - A clinical update from the KYANITE-1 monotherapy cohort will be presented at the 2025 ASCO Annual Meeting in June[13](index=13&type=chunk) [Aesthetics Pipeline](index=3&type=section&id=Aesthetics%20Pipeline) Jeune Aesthetics is advancing its pipeline for wrinkles, with KB301 Phase 2 enrollment in Q4 2025 and KB304 top-line results in 2H 2025 - **KB301 (Décolleté Wrinkles):** A Phase 2 study is planned to begin enrollment in Q4 2025 after alignment with the FDA on a new clinical scale[14](index=14&type=chunk) - **KB304 (Wrinkles):** Enrollment is complete in the Phase 1 PEARL-2 study, with top-line results anticipated in 2H 2025[14](index=14&type=chunk) [Dermatology Pipeline](index=4&type=section&id=Dermatology%20Pipeline) The dermatology pipeline includes KB105 Phase 2 initiation in 2026 and preclinical data for Hailey-Hailey and Darier diseases in 2025 - **KB105 (Lamellar Ichthyosis):** The Phase 2 portion of the JADE-1 trial in pediatric patients is expected to start in 2026[16](index=16&type=chunk) - **Pipeline Expansion:** Preclinical data for early-stage candidates targeting Hailey-Hailey and Darier diseases will be presented at the SID 2025 Annual Meeting[16](index=16&type=chunk) [Financial Performance](index=4&type=section&id=Financial%20Performance) [Q1 2025 Financial Results](index=4&type=section&id=Q1%202025%20Financial%20Results) Krystal Biotech reported strong Q1 2025 financial results, with net product revenue of **$88.2 million** and net income of **$35.7 million** Q1 2025 vs Q1 2024 Financial Results (in millions, except per share data) | Metric | Q1 2025 | Q1 2024 | | :--- | :--- | :--- | | Product Revenue, net | $88.2 | $45.3 | | Cost of Goods Sold | $5.0 | $2.4 | | R&D Expenses | $14.3 | $11.0 | | SG&A Expenses | $32.7 | $26.1 | | Net Income | $35.7 | $0.9 | | Diluted EPS | $1.20 | $0.03 | | Cash & Investments | $765.3 | N/A | [FY 2025 Financial Guidance](index=5&type=section&id=FY%202025%20Financial%20Guidance) The company projects full-year 2025 non-GAAP combined R&D and SG&A expenses between **$150.0 million** and **$175.0 million** - Full-year 2025 non-GAAP combined R&D and SG&A expense is projected to be between **$150.0 million** and **$175.0 million**[18](index=18&type=chunk) - This non-GAAP guidance excludes stock-based compensation expense, which cannot be confidently estimated at this time[18](index=18&type=chunk)[31](index=31&type=chunk) [Consolidated Financial Statements](index=9&type=section&id=Consolidated%20Financial%20Statements) Condensed consolidated financial statements detail the company's financial position, including **$1.07 billion** in total assets and **$35.7 million** net income [Condensed Consolidated Balance Sheet](index=9&type=section&id=Condensed%20Consolidated%20Balance%20Sheet) Balance Sheet Data (in thousands) | Account | March 31, 2025 | December 31, 2024 | | :--- | :--- | :--- | | Cash and cash equivalents | $308,770 | $344,865 | | Short-term investments | $308,076 | $252,652 | | Total assets | $1,074,416 | $1,055,838 | | Total liabilities | $89,742 | $109,458 | | Total stockholders' equity | $984,674 | $946,380 | [Condensed Consolidated Statement of Operations](index=9&type=section&id=Condensed%20Consolidated%20Statement%20of%20Operations) Statement of Operations (in thousands, except per share data) | Account | Three Months Ended Mar 31, 2025 | Three Months Ended Mar 31, 2024 | | :--- | :--- | :--- | | Product revenue, net | $88,183 | $45,250 | | Total operating expenses | $52,006 | $51,934 | | Income from operations | $36,177 | $(6,684) | | Net income | $35,733 | $932 | | Net income per common share (Diluted) | $1.20 | $0.03 | [Corporate Information and Disclosures](index=5&type=section&id=Corporate%20Information%20and%20Disclosures) [About VYJUVEK and Safety Information](index=5&type=section&id=About%20VYJUVEK%20and%20Safety%20Information) VYJUVEK is a topical gene therapy for DEB, approved in the U.S. and Europe, with common side effects and specific handling precautions - VYJUVEK is a topical, redosable gene therapy designed to deliver two copies of the COL7A1 gene directly to DEB wounds[20](index=20&type=chunk) - The most common side effects observed (incidence >5%) were itching, chills, redness, rash, cough, and runny nose[22](index=22&type=chunk) - Precautions include application by a healthcare provider and careful handling of treated wounds and dressings for 24 hours to avoid accidental exposure[24](index=24&type=chunk)[25](index=25&type=chunk) [About Krystal Biotech and Jeune Aesthetics](index=6&type=section&id=About%20Krystal%20Biotech%20and%20Jeune%20Aesthetics) Krystal Biotech is a commercial-stage genetic medicines company, with Jeune Aesthetics as its subsidiary focusing on skin aging and damage - Krystal Biotech is a commercial-stage company focused on genetic medicines, headquartered in Pittsburgh, PA[27](index=27&type=chunk) - Jeune Aesthetics, a wholly-owned subsidiary, focuses on developing products to address the biology of aging and damaged skin using Krystal's gene delivery platform[28](index=28&type=chunk) [Forward-Looking Statements and Non-GAAP Measures](index=6&type=section&id=Forward-Looking%20Statements%20and%20Non-GAAP%20Measures) The report contains forward-looking statements subject to risks and includes non-GAAP financial guidance excluding stock-based compensation - The press release includes forward-looking statements concerning product launches, regulatory timelines, and clinical trial progress, which are subject to inherent risks[29](index=29&type=chunk)[30](index=30&type=chunk) - The company uses a non-GAAP measure for its R&D and SG&A expense guidance, which excludes stock-based compensation, to better reflect ongoing operational performance[31](index=31&type=chunk)

UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 _____________________________________________________ FORM 10-Q _____________________________________________________ (Mark One) ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended March 31, 2025 or ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from to Commission file number: 001-38210 _____________ ...

Second ophthalmic program in clinic - KB801 for the treatment of neurotrophic keratitis Upcoming clinical readouts in 2025 - CF, AATD, ocular complications of DEB, and aesthetics Strong balance sheet, ending the quarter with $765.3 million in cash and investments VYJUVEK approved in Europe for the treatment of DEB patients from birth $88.2 million in 1Q VYJUVEK revenue and $429.4 million since launch in Q3 2023 PITTSBURGH, May 06, 2025 (GLOBE NEWSWIRE) -- Krystal Biotech, Inc. (the "Company") (NASDAQ: KRYS) ...

Core Viewpoint - The European Commission has granted marketing authorization for VYJUVEK, a gene therapy for treating dystrophic epidermolysis bullosa (DEB) from birth, marking a significant milestone for Krystal Biotech and patients in need of this therapy [2][4][5]. Group 1: Product Approval and Features - VYJUVEK (beremagene geperpavec-svdt) is approved for patients with DEB who have mutations in the COL7A1 gene, aiming to provide wound healing through the delivery of functional copies of the gene [2][6]. - The therapy can be administered at home or in healthcare settings, with the option for patient or caregiver administration if deemed appropriate by healthcare professionals [2][3]. - This approval allows for the marketing of VYJUVEK across all EU member states, as well as Iceland, Norway, and Liechtenstein, with the first launch planned in Germany by mid-2025 [3][4]. Group 2: Clinical Evidence and Development - The approval was based on positive recommendations from the European Medicines Agency and comprehensive clinical data from Phase 1/2 GEM-1 and Phase 3 GEM-3 studies, demonstrating successful gene delivery and durable wound closure [4][5]. - VYJUVEK is the first corrective medicine approved in Europe for DEB, highlighting its significance in addressing a high unmet medical need [2][5][7]. Group 3: Company Overview and Future Plans - Krystal Biotech is a global biotechnology company focused on developing genetic medicines for diseases with high unmet medical needs, with VYJUVEK being its first commercial product [7][8]. - The company is also pursuing approval for VYJUVEK in Japan, with a decision expected in the second half of 2025 [5].

4月27日，副省长方伟赴镇江检查安全生产工作。省政府副秘书长徐华勤参加检查。市委常委、常 务副市长张克陪同检查。 在江苏金斯瑞生物科技有限公司，方伟深入企业生产和研发一线，详细了解企业安全管理体系建设 和安全生产责任制落实情况。得知企业建立了EHS基本安全准则，成立了安全生产委员会，配备专职安 全管理机构EHS部门，方伟给予肯定。他指出，生物科技行业技术密集、工艺复杂，安全风险点多、管 理要求高，企业必须树牢底线思维，强化安全管理，持续完善安全生产各项制度，提升本质安全水平， 切实将安全责任落实到每一个岗位、每一名员工。 随后，方伟来到镇江信缘康米山颐养院，实地检查消防控制室、消防设施设备运行状况以及疏散通 道设置情况，仔细询问火灾防控、应急预案制定、日常巡查制度和员工安全培训等工作落实情况。在认 真听取养老机构负责人介绍后，方伟指出，养老机构入住人员多为高龄老人，行动不便、应急反应能力 弱，消防安全责任重大。要深刻吸取近期各地火灾事故教训，进一步压实安全管理责任，全面开展风险 隐患排查整治，完善应急处置预案，加强员工技能培训和演练，做到早发现、早处置，确保遇到突发情 况能够及时组织有效应对，切实守护入住老人 ...

Core Insights - Krystal Biotech, Inc. will present multiple programs related to lung, eye, and skin at scientific conferences in May and June 2025 [1] Conference Presentations - The company will participate in the following conferences: - American Society of Gene & Cell Therapy (ASGCT) 28th Annual Meeting: Invited Oral Presentation [2] - 2025 American Society of Clinical Oncology (ASCO) Annual Meeting: Poster Presentation [2] - Association for Research in Vision and Ophthalmology (ARVO) 2025 Annual Meeting: Poster Presentation [2] - Society for Investigative Dermatology (SID) 2025 Annual Meeting: Poster Presentation [3] Company Overview - Krystal Biotech, Inc. is a global biotechnology company focused on genetic medicines for diseases with high unmet medical needs [4] - The company's first commercial product, VYJUVEK®, is the first-ever redosable gene therapy approved by the FDA for dystrophic epidermolysis bullosa [4] - The company is advancing a pipeline of investigational genetic medicines across various fields including respiratory, oncology, dermatology, ophthalmology, and aesthetics [4]

PITTSBURGH, April 17, 2025 (GLOBE NEWSWIRE) -- Krystal Biotech, Inc. (the “Company”) (NASDAQ: KRYS), a commercial-stage biotechnology company, today announced that it will report its first quarter 2025 financial results on Tuesday, May 6, 2025, prior to the open of U.S. markets. The Company’s management will also host a conference call and webcast at 8:30 am ET on Tuesday, May 6, 2025, to discuss the financial results and provide a business update. Investors and the general public can access the live webcas ...