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Krystal Biotech (KRYS) Earnings Call Presentation
2025-07-09 14:39
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]
KALA BIO (KALA) 2025 Conference Transcript
2025-06-05 18:25
Summary of KALA BIO (KALA) 2025 Conference Call Company Overview - KALA BIO is a leader in the emerging field of mesenchymal stem cell secretomes, focusing on rare ophthalmic diseases [1] Industry and Market Opportunity - The ongoing pivotal trial for KALA's lead product, KPI O One Two, targets persistent corneal epithelial defects (PCED), a rare and vision-threatening condition with significant unmet needs [2] - The only approved product in this space, Oxervate, had sales of approximately $820 million in 2023 and is speculated to have exceeded $1 billion in sales [2] - KALA's product aims to cover a broader indication for PCED, addressing not only the one-third of patients treated by Oxervate but also the additional two-thirds with other etiologies [2][12] Product Development and Mechanism of Action - KPI O One Two utilizes a multifactorial mechanism of action, which includes wound healing, tissue repair, anti-inflammatory, and neuroprotective effects [6] - The product is derived from human bone marrow-derived mesenchymal stem cells, manufactured under GMP conditions to produce a variety of biomolecules [5] - The formulation is designed for easy patient administration, contrasting with Oxervate's complex dosing regimen [14] Clinical Trials and Results - KALA has received orphan drug and fast track designations from the FDA for KPI O One Two [7] - A phase one b clinical trial showed that 6 out of 8 patients experienced complete healing of their lesions, with significant pain reduction observed [24][28] - The pivotal trial is designed to demonstrate efficacy through complete healing of lesions at 8 weeks, with top-line data expected by the end of Q3 [36][38] Competitive Advantages - KALA's product is positioned to address a significant market gap, as Oxervate is only indicated for neurotrophic keratitis, leaving two-thirds of PCED patients without approved therapies [12][44] - The product's simpler dosing and administration method is expected to improve patient compliance and experience [14][15] Regulatory and Development Strategy - KALA has engaged with the FDA, receiving positive feedback on their clinical trial design and potency assay program [33][30] - The current study involves a multicenter randomized trial with a target of 90 patients, with plans to expand enrollment to ensure robust data [39][41] Key Takeaways - KALA BIO is targeting a significant unmet need in the ophthalmic market with its innovative product KPI O One Two, which has shown promising early clinical results and is positioned to capture a large patient population [2][24][44] - The company is well-prepared for regulatory engagement and has a clear path forward for its clinical development program [30][33]