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]

Summary of Crystal Biotech's Conference Call on KB801 Company and Industry Overview - **Company**: Crystal Biotech - **Industry**: Biotechnology, specifically focusing on gene therapy for ophthalmic conditions, particularly neurotrophic keratitis (NK) Key Points and Arguments 1. **First Patient Dosed**: The first patient has been dosed in the EMERALD-one Phase 1/2 study evaluating KB801 for the treatment of neurotrophic keratitis [2][5][22] 2. **Current Treatment Landscape**: NK is a rare, degenerative disease with only one FDA-approved therapy, Oxervate, which requires intensive dosing (six times daily) and has limitations such as rapid protein clearance and associated eye pain [7][9][10] 3. **Market Opportunity**: The estimated number of U.S. patients with an NK claim in 2024 is projected to be 68,000, representing a 115% increase from 2020 [7] 4. **KB801 Advantages**: KB801 aims to provide a more consistent nerve growth factor (NGF) exposure with a reduced treatment burden, potentially dosed once or twice weekly compared to Oxervate's six times daily regimen [10][24] 5. **Clinical Program Design**: EMERALD-one is a double-masked, randomized, placebo-controlled study involving up to 27 adult subjects with moderate to severe NK [22][23] 6. **Safety and Efficacy Focus**: The primary focus of the study is on the safety and tolerability of KB801, with secondary endpoints assessing efficacy through corneal defect closure and corneal sensitivity [23][24] 7. **Regulatory Confidence**: The company expresses confidence in obtaining an accelerated pathway for approval due to the existing knowledge from Oxervate and the favorable dosing regimen of KB801 [24][40][41] 8. **Platform Technology**: The HSV-1 based gene delivery platform is highlighted as versatile, with applications in multiple tissues (skin, lung, eye) and the potential for various genetic therapies [5][12][28] 9. **Future Pipeline**: The company has a diversified pipeline with ongoing programs in oncology, aesthetics, and skin, alongside KB801 and KB803, which are both in clinical stages [27][28] Additional Important Content 1. **Preclinical Data**: Preclinical studies indicate that KB801 can effectively transduce corneal epithelial cells, leading to sustained NGF production without cytotoxicity [15][18][19] 2. **Market Dynamics**: The company is actively engaging with key opinion leaders (KOLs) and clinical sites to facilitate patient enrollment, indicating a strong demand for new treatments in this space [79] 3. **Patient Compliance Issues**: The challenges of patient adherence to the current treatment regimen (Oxervate) are emphasized, with many patients unable to maintain the required dosing frequency [94] 4. **Potential for Faster Wound Closure**: There is optimism that KB801 could lead to faster wound closure compared to existing therapies, although the exact duration of treatment will be evaluated based on clinical data [76][81] 5. **Regulatory Interactions**: The company has had positive interactions with the FDA, which has provided feedback on the study design and expressed understanding of the product's profile [100][101] This summary encapsulates the critical insights from the conference call, highlighting the strategic direction of Crystal Biotech and the potential impact of KB801 on the treatment of neurotrophic keratitis.

Summary of Krystal Biotech (KRYS) Conference Call Company Overview - **Company Name**: Krystal Biotech - **Industry**: Biotechnology - **Focus**: Development and commercialization of genetic medicines for monogenic diseases - **Location**: Pittsburgh, with all manufacturing and intellectual property based in the US [4][5] Core Points and Arguments Commercial Stage and Pipeline - Krystal Biotech launched its drug, **BIJUVAK**, for dystrophic epidermolysis bullosa (DEB) in the US about 18 months ago and received approval in Europe, with plans to launch in Europe and Japan later in 2025 [4][5] - The company has been cash flow positive for the last seven quarters and does not plan to seek financing in the next few years [5][6] - The focus for 2025 includes expanding into lung and eye tissues, with early entry into oncology [5] Market Trends and Patient Dynamics - In Q1, patient healing led to temporary pauses in treatment, which is expected to result in a return of patients over the next 60-120 days [8][9] - The company has 17 sales representatives covering 52 states, facing challenges in prescription pull-through due to patient dispersion [10][11] - Insurance changes in Q1 impacted revenue but are expected to normalize in Q2 [12][13] European and Japanese Market Launch - The EU approval allows broader patient self-administration compared to the US, with launches expected in Germany and France in Q3 2025 [19][20] - Pricing in the EU is anticipated to be around 50-75% of the US price, with a conservative accrual strategy [27][28] Pipeline Developments - Upcoming focus on cystic fibrosis (CF) and alpha-one antitrypsin deficiency treatments, with strong value propositions for patients with null mutations [34][35] - The company is preparing for a lung cancer announcement at ASCO, reinforcing confidence in its lung delivery capabilities [37][38] Ophthalmology Programs - The company is initiating a registrational study for KBM-803 targeting ocular complications of DEB, with a market opportunity for about 10-15% of the RDEB population having eye lesions [48][49] - Another program, KB801, targets neurotropic keratitis, expected to be a significant market opportunity but not anticipated for launch until 2026 [50][51] Financial Outlook - The company is confident in its financial position, with plans to manage the launch of BIJUVAK and advance other pipeline assets without needing additional funding [53][55] Other Important Insights - The company emphasizes the importance of patient comfort and convenience in drug administration, with a high percentage of patients preferring home administration [30][31] - The management is focused on strategic partnerships for larger indications to enhance market reach and financial stability [55] This summary encapsulates the key points discussed during the conference call, highlighting the company's strategic direction, market dynamics, and pipeline developments.

Krystal Biotech (KRYS) reported first-quarter 2025 earnings per share (EPS) of $1.20, which missed the Zacks Consensus Estimate of $1.38.  Nonetheless, the reported EPS was up from 3 cents posted in the year-ago quarter. (Find the latest EPS estimates and surprises on Zacks Earnings Calendar.)Revenues of $88.1 million surged 95% year over year but missed the Zacks Consensus Estimate of $95 million. Revenues came in solely from the sales of Vyjuvek.Shares of the company are trading down in response to the di ...

First Quarter 2025 Financial and Operating Results May 6, 2025 © Copyright 2025 Krystal Biotech, Inc. All rights reserved. Forward Looking Statements and Disclosures This presentation and the accompanying oral presentation 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 the Company's U.S. c ...

Krystal Biotech (KRYS) Q1 2025 Earnings Call May 06, 2025 08:30 AM ET Speaker0 Thank you for standing by, and welcome to the Crystal Biotech First Quarter twenty twenty five Earnings Call. At this time, all participants are on a listen only mode. After the speakers' presentations, there will be a question and answer session. As a reminder, today's conference is being recorded. I would now like to hand the conference over to your host, Stephane Paquette, Vice President of Corporate Development. Please begin. ...