Core Insights - The company announced a positive interim clinical update from the highest dose cohort of the CORAL-1 study, confirming successful lung delivery and expression of wild-type CFTR protein in cystic fibrosis patients following inhaled administration of KB407 [1][2][13] - The CORAL-3 study design has been submitted to the FDA, with enrollment expected to start in the first half of 2026 [1][13] Clinical Study Details - KB407 is being evaluated in the CORAL-1 study, which is a multi-center Phase 1 study with three dose escalation cohorts [4] - As of January 6, 2026, seven patients have been dosed in the highest dose cohort, with successful bronchoscopies yielding biopsies suitable for molecular analysis [6][12] - The percentage of conducting airway cells transduced with KB407 ranged from 29.4% to 42.1% across six patients with successful bronchoscopies [7][9] Patient Demographics and Results - Among the four modulator ineligible patients, all biopsies were positive for CFTR or viral marker expression, indicating widespread dissemination of KB407 [9] - For modulator eligible patients, the percentage of conducting airway cells positive for viral markers ranged from 31.4% to 36.8% [11] Safety Profile - Inhaled KB407 was well tolerated in the highest dose cohort, with most adverse events being mild to moderate [12] - One serious adverse event of asthma exacerbation was reported but deemed procedure-related and not related to KB407 [12] Future Plans - The company is moving forward with the CORAL-3 study, designed to evaluate the safety and efficacy of repeat KB407 administration [13] - The company expects to align on the CORAL-3 study design with the FDA in the first quarter of 2026 and start enrollment in the second quarter of 2026 [13]

Core Insights - Krystal Biotech, Inc. will participate in the 8th Annual Evercore Healthcare Conference on December 3, 2025, in Miami, with CEO Krish S. Krishnan scheduled for a fireside chat at 10:00 am ET [1] - A webcast of the presentation will be available on the Company's website starting at the same time [2] Company Overview - Krystal Biotech, Inc. is a global biotechnology company focused on genetic medicines for diseases with high unmet medical needs, with its first product, VYJUVEK®, being the first-ever redosable gene therapy approved in the US, 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]

Company Participation - Krystal Biotech, Inc. will participate in the Cantor Global Healthcare Conference 2025 on September 4, 2025, in New York [1] - Krish S. Krishnan, Chairman and CEO, will engage in a fireside chat at 10:20 am ET and host investor meetings throughout the day [1] Webcast Information - A webcast of the presentation will be available starting at 10:20 am ET on September 4, 2025, and will be posted on 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 in the US, Europe, and Japan for 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]

Core Insights - Jeune Aesthetics, Inc. announced positive safety and efficacy results for KB304, a treatment aimed at restoring skin by delivering collagen and elastin, with significant improvements in skin attributes such as wrinkles and elasticity [1][7][9] Study Results - The PEARL-2 study was a randomized, double-blind, placebo-controlled Phase 1 trial involving 19 subjects, with 12 receiving KB304 and 7 receiving placebo [4][13] - At three months post-treatment, 100% of KB304 subjects reported at least a one-point improvement in wrinkles, compared to 28.6% in the placebo group [5] - Subject Satisfaction Questionnaire (SSQ) indicated that 81.8% of KB304 subjects reported improved satisfaction with their wrinkles' appearance, versus 14.3% for placebo [5] Additional Findings - Improvements were also noted in other skin attributes such as elasticity, crepiness, hydration, and radiance, with 100% of KB304 subjects showing at least a one-point improvement across these metrics at all assessment points [6] - The safety profile of KB304 was consistent with previous studies, with mild-to-moderate adverse events reported, primarily after the first dose, and no severe drug-related adverse events [8] Future Development - Based on the positive results, KB304 will progress to Phase 2 development for treating wrinkles in the décolleté, a site with no FDA-approved injectable products [9] - Jeune plans to meet with the FDA to discuss the Phase 2 study protocol, aiming to initiate the study in the first half of 2026 [9]

Core Viewpoint - Krystal Biotech, Inc. has initiated its Phase 3 clinical trial named "IOLITE" to evaluate KB803 for treating and preventing corneal abrasions in patients with dystrophic epidermolysis bullosa (DEB) [1][2] Group 1: Clinical Trial Details - IOLITE is a double-blind, multicenter, placebo-controlled study with a crossover design, focusing on KB803 administered as eye drops [4] - The trial will enroll approximately 16 subjects, with a primary endpoint measuring the change in average days per month with corneal abrasion symptoms while receiving KB803 versus placebo [5][6] - Patients must complete a 12-week run-in period in an ongoing natural history study before participating in IOLITE [4][5] Group 2: Patient Population and Disease Impact - Over 25% of DEB patients are believed to experience ocular complications, with more than 50% of patients suffering from the recessive form of DEB (RDEB) [2] - The estimated number of DEB patients in the U.S. exceeds 750, with over 2,000 affected globally [2] - Current management of DEB-related ocular complications is limited to supportive care and surgical interventions, with no corrective therapies available [2] Group 3: Previous Findings and Compassionate Use - A previous compassionate use case of KB803 demonstrated full corneal healing at three months and significant visual acuity improvement from hand motion to 20/25 by eight months [1][12] - The compassionate use case involved a patient with severe cicatrizing conjunctivitis secondary to DEB, treated with regular B-VEC administration [11][12] Group 4: Company Overview - Krystal Biotech, Inc. is a global biotechnology company focused on developing genetic medicines for diseases with high unmet medical needs [13] - The company’s first commercial product, VYJUVEK®, is the first-ever redosable gene therapy approved by the FDA and EMA for treating dystrophic epidermolysis bullosa [13]

VYJUVEK Commercial Performance and Expansion - VYJUVEK has generated over $429 million in revenue since its launch[15] - Q1 2025 net revenue reached $88.2 million, a 95% increase compared to Q1 2024[16, 18] - The gross margin for Q1 2025 was 94%, with a gross to net of 17%[18] - Over 540 reimbursement approvals have been secured as of April 2025, with 45% from commercial payers and 39% from Medicaid[24, 25] - 97% of covered lives under commercial and Medicaid plans have positive access[25] Clinical Development Programs - Four clinical readouts are expected in 2025 for KB407 (cystic fibrosis), KB408 (AATD lung disease), KB304 (aesthetic skin conditions), and KB803 (ocular DEB)[19] - A Phase 1/2 study evaluating KB801 for neurotrophic keratitis is underway, with the first patient expected to be dosed this month[43] - Initial KB408 molecular data showed 35% AAT positive cells at baseline increasing to 39% after KB408 in patient 07[37] Financial Stability - The company's cash and investments totaled $7653 million as of March 31, 2025[54] - Non-GAAP R&D and SG&A expense guidance for the full year 2025 is $150 million to $175 million[55]