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]

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]

Core Viewpoint - Krystal Biotech, Inc. has initiated its Phase 1/2 clinical trial, EMERALD-1, for KB801, a redosable eye drop gene therapy aimed at treating neurotrophic keratitis (NK), a rare degenerative corneal disease [1][2][3] Company Overview - Krystal Biotech, Inc. is a global biotechnology company focused on developing genetic medicines for diseases with high unmet medical needs, with its first commercial product being VYJUVEK, the first redosable gene therapy approved by the FDA and EMA [8] Clinical Trial Details - The EMERALD-1 trial is a randomized, double-masked, multicenter, placebo-controlled study involving up to 27 adults with Stage 2 or Stage 3 NK, where participants will receive either KB801 or a placebo twice weekly for 8 weeks [3][4] - The primary objective of the trial is to evaluate the safety and tolerability of KB801, while the secondary objective focuses on the efficacy measured by the proportion of patients achieving complete durable healing of corneal epithelium at 8 weeks [4] Product Information - KB801 is designed to enable sustained expression of nerve growth factor (NGF) in the front of the eye, potentially reducing the treatment burden for NK patients compared to current therapies that require frequent administration [6] - The estimated prevalence of NK is between 10 to 50 cases per 100,000, with claims data indicating a significant increase in awareness and diagnosis rates in the U.S., from 31,000 patients in 2020 to approximately 68,000 in 2024, representing an increase of over 115% [2][6]

Company Participation - Krystal Biotech, Inc. will participate in the Goldman Sachs 46th Annual Global Healthcare Conference on June 10, 2025, in Miami [1] - Krish S. Krishnan, Chairman and CEO, will engage in a fireside chat at 2:00 pm ET and host investor meetings throughout the day [1] Webcast Information - A webcast of the presentation will be available starting at 2:00 pm ET on June 10, 2025, and will be posted on the Investors section of the Company's website [2] Company Overview - Krystal Biotech, Inc. is a fully integrated, commercial-stage global 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 and the first genetic medicine approved by the FDA and EMA for treating dystrophic epidermolysis bullosa [3] - The Company is advancing a robust pipeline of investigational genetic medicines in various fields including respiratory, oncology, dermatology, ophthalmology, and aesthetics [3]

Company Overview - Krystal Biotech, Inc. is a fully integrated, commercial-stage global biotechnology company focused on the discovery, development, and commercialization of genetic medicines to treat 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 by the FDA and EMA for the treatment of dystrophic epidermolysis bullosa [3] - Krystal Biotech is advancing a robust preclinical and clinical pipeline of investigational genetic medicines in various fields including respiratory, oncology, dermatology, ophthalmology, and aesthetics [3] Upcoming Events - The company will participate in the BofA Securities 2025 Health Care Conference on May 14, 2025, in Las Vegas [1] - Krish S. Krishnan, Chairman and CEO, will take part in a fireside chat scheduled at 11:20 am PT and will host investor meetings throughout the day [1] - A webcast of the presentation will be available starting at 11:20 am PT on May 14, 2025, and will be posted on the Investors section of the company's website [2]