Core Insights - Krystal Biotech, Inc. (NASDAQ:KRYS) is recognized as a leading gene-editing stock, with a Buy rating reaffirmed by Evercore ISI and a price target set at $218 [1][2]. Financial Performance - In Q3 2025, Krystal reported an EPS of $2.66 per diluted share, significantly exceeding the anticipated $1.09 [2]. - Revenue for the quarter reached $97.8 million from the VYJUVEK product, contributing to a total revenue of over $623 million since its US launch [2]. - The net income for the quarter was $79.4 million, attributed to manufacturing efficiencies and a one-time non-cash tax benefit [2]. - Gross margin improved to 96%, up from 93% in the previous quarter, due to optimizations in US product manufacturing [2]. - The company ended the quarter with over $864 million in cash and investments [2]. Operational Developments - Management highlighted ongoing expansion efforts into Germany, France, and Japan, along with partnerships in the Middle East, Turkey, and Central and Eastern Europe [3]. - The company is focused on increasing patient access for the treatment of Dystrophic Epidermolysis Bullosa (DEB) and expanding its global market presence [3]. Company Overview - Krystal Biotech, Inc. is a biotechnology company that develops and commercializes redosable gene therapies for rare and serious genetic diseases [4]. - Its first approved product, VYJUVEK, is the only FDA-approved redosable gene therapy for DEB, a severe skin disorder [4].

Core Insights - The article emphasizes the importance of enabling Javascript and cookies in browsers to prevent access issues [1] Group 1 - The article suggests that users may face blocks if they have ad-blockers enabled, indicating a need to disable them for proper access [1]

Third Quarter 2025 Financial and Operating Results November 3, 2025 © Copyright 2025 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. launch of VYJUVEK, includ ...

Financial Performance - The company reported $97.8 million in net product revenue for VYJUVEK in Q3 2025, an increase from $83.8 million in Q3 2024, reflecting a growth of approximately 16.7% year-over-year [3][23][37] - Gross margin for the quarter was 96%, indicating strong profitability from product sales [3] - Net income for Q3 2025 was $79.4 million, or $2.74 per common share (basic), compared to $27.2 million, or $0.95 per common share (basic) in Q3 2024, representing a significant increase [23][36] Product Development and Market Expansion - VYJUVEK was launched in Germany in Q3 2025, with approximately 20 patients prescribed the therapy across over 10 centers [3] - The product was also launched in France and Japan in Q4 2025, following successful pricing negotiations [3][4] - The FDA approved a label update for VYJUVEK, expanding the eligible patient population to include DEB patients from birth and allowing for home application by patients or caregivers [3][24] Pipeline and Future Prospects - The company is advancing its pipeline with multiple near-term readouts, including interim results for cystic fibrosis expected in Q4 2025 [2][6] - The company is preparing regulatory filings for the UK and Switzerland and is initiating pricing discussions in other key Western European markets [3][4] - The company continues to enroll in clinical trials for KB407 and KB408, with interim data readouts expected in late 2025 and early 2026 [6][11] Financial Guidance - The company provided guidance for FY 2025, estimating non-GAAP combined R&D and SG&A expenses to be between $145 million and $155 million [20]

Core Viewpoint - Krystal Biotech, Inc. is set to report its third quarter 2025 financial results on November 3, 2025, and will host a conference call 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 the discovery, development, and commercialization of 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 United States, Europe, and Japan for the treatment of dystrophic epidermolysis bullosa [3]. - The company is advancing a robust pipeline of investigational genetic medicines across various fields including respiratory, oncology, dermatology, ophthalmology, and aesthetics [3].

Core Viewpoint - Krystal Biotech, Inc. is advancing the development of KB707, a redosable immunotherapy targeting non-small cell lung cancer (NSCLC), with promising early efficacy data leading to an End of Phase 2 meeting with the FDA to discuss registration pathways [1][2][3] Group 1: Development Plans and Efficacy - The company has observed a 36% objective response rate in heavily pre-treated NSCLC patients as of April 15, 2025, with no Grade 4 or 5 adverse events reported, indicating a favorable safety profile [3] - Enrollment is ongoing in the KYANITE-1 study, a Phase 1/2 trial evaluating inhaled KB707 in patients with locally advanced or metastatic lung tumors [4] - The company has paused enrollment in the OPAL-1 study, which evaluates intratumoral KB707, to prioritize inhaled KB707, while continuing to monitor patients in OPAL-1 [5] Group 2: Company Overview - Krystal Biotech is a commercial-stage biotechnology company focused on genetic medicines for diseases with high unmet medical needs, with its first product, VYJUVEK, being the first redosable gene therapy approved in multiple regions [6]

Financial Performance & VYJUVEK Launch - VYJUVEK revenue since launch exceeds $525 million[8] - Second quarter 2025 product revenue reached $96 million, compared to $70.3 million in the second quarter of 2024[43] - Gross margin in the second quarter was 93%[10, 43] - Gross to net in the second quarter was 17%[10] VYJUVEK Expansion & Approvals - VYJUVEK received approval in Japan for DEB patients from birth, with launch expected in the fourth quarter of 2025[11, 14] - Over 575 reimbursement approvals in the U S[10] - U S compliance to weekly therapy while on drug at 82% as of end of 2Q 2025[10] - European launch is on track for later this quarter, with over 575 identified DEB patients in Germany and France[15, 16] Pipeline Development - 36% objective response rate was observed with KB707 monotherapy in advanced NSCLC patients[22] - 40% AAT positive cells after KB408[26] - Functional AAT delivery to the lungs was confirmed in the third patient dosed with KB408, showing a 79.3% reduction in free neutrophil elastase in ELF after a single dose[25, 26] Financial Guidance - Non-GAAP R&D and SG&A expense guidance for the full year 2025 is $150 million to $175 million[44]

Financial Performance - The company reported $96.0 million in net product revenue for the second quarter of 2025, a significant increase from $70.3 million in the same quarter of 2024, representing a growth of approximately 36% [20][38] - For the six months ended June 30, 2025, net product revenue totaled $184.2 million, compared to $115.5 million for the same period in 2024, indicating a year-over-year increase of about 61% [18][39] - The gross margin for the second quarter was 93%, reflecting strong profitability [6] - The company ended the quarter with a robust balance sheet, holding $820.8 million in cash and investments [20][37] Product Developments - VYJUVEK, the company's gene therapy product, has been approved in Japan for the treatment of dystrophic epidermolysis bullosa (DEB) patients from birth, with plans to launch before the end of 2025 [6][23] - The company has secured over 575 reimbursement approvals for VYJUVEK in the U.S., maintaining strong access nationwide [6] - VYJUVEK is also set for its first European launch in Germany in the third quarter of 2025 and in France in the fourth quarter of 2025 [6][34] Clinical Trials and Pipeline - The company is actively enrolling patients in various clinical trials, including KB407 for cystic fibrosis and KB408 for alpha-1 antitrypsin deficiency lung disease, with interim data expected before year-end [5][7] - The company has initiated a Phase 3 study for KB803, aimed at treating and preventing corneal abrasions in DEB patients, with ongoing enrollment [13] - In oncology, the company continues to evaluate inhaled KB707 in patients with solid tumors, with promising safety and efficacy data reported [11][19] Management Commentary - The CEO highlighted the company's global expansion plans following VYJUVEK's approvals in Europe and Japan, which are expected to enhance access for DEB patients worldwide [2] - The company is approaching key readouts in both lung and eye treatments, which could have transformative implications for its platform and patient care [2]

Core Insights - Krystal Biotech, Inc. will report its second quarter 2025 financial results on August 4, 2025, before U.S. market opens [1] - A conference call and webcast will be held on the same day at 8:30 am ET to discuss the financial results and provide a business update [1] Company Overview - Krystal Biotech, Inc. is a commercial-stage biotechnology company focused on genetic medicines for diseases with high unmet medical needs [3] - The company's first commercial product, VYJUVEK, is the first-ever redosable gene therapy approved in the U.S., Europe, and Japan for dystrophic epidermolysis bullosa [3] - The company is advancing a pipeline of investigational genetic medicines across various fields including respiratory, oncology, dermatology, ophthalmology, and aesthetics [3]

Neurotrophic Keratitis (NK) Market & KB801 Opportunity - The number of patients in the U S with at least one NK claim has increased by over 115% from 31,000 in 2020 to an estimated 68,000 in 2024[9, 10] - In 2023, U S Medicaid and Medicare spent over $540 million on Oxervate, the only FDA-approved therapy for NK[12] - An estimated over 410,000 days of Oxervate therapy were reimbursed in the U S in 2024[12] - KB801 is designed to address the shortcomings of Oxervate, aiming for superior NGF exposure with significantly reduced dosing frequency[11, 21] KB801 Preclinical Data - KB801 transduces primary human corneal epithelial cells in vitro, leading to dose-dependent expression and secretion of mature NGF[25, 26, 29] - In vitro studies confirmed functionality of secreted NGF using a growth factor starved cell proliferation assay[25, 30] - Topical administration of KB801 to wounded murine corneas was well-tolerated and resulted in localized NGF expression[25] - Head-to-head mouse PK studies demonstrated a clear durability advantage with KB801 compared to recombinant NGF, even against intensive recombinant dosing[43, 47] KB801 Clinical Development - The EMERALD-1 study is a Phase 1/2 double-masked, 2:1 randomized, placebo-controlled study in patients with moderate-to-severe NK, with the first patient dosed in July 2025[51, 53, 1] - Krystal is pursuing Platform Technology Designation with the FDA, potentially leading to expedited development of KB801[56, 57]