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Krystal Biotech
Krystal Biotech (KRYS) investor relations material
Krystal Biotech 44th Annual J.P. Morgan Healthcare Conference summary
Complete event summary combining all related documents: earnings call transcript, report, and slide presentation.44th Annual J.P. Morgan Healthcare Conference summary12 Jan, 2026
2025 performance and financial highlights
Achieved all 2025 objectives, including first approvals and strong global launch for Sephience, with $823 million in revenue, surpassing guidance.
Sephience generated $112 million in five and a half months, with over 940 patients on therapy and high refill rates.
Closed 2025 with over $1.94 billion in cash, effective expense management, and a 6% decrease in OpEx guidance for 2026.
Product revenue guidance for 2026 is $700–$800 million, reflecting 19%–36% growth from 2025 product revenue.
Potential to reach cash flow breakeven in 2026, positioning for sustainable profitability.
Sephience launch and market opportunity
Sephience’s dual mechanism of action enables benefit across all PKU patient segments, including severe forms.
Clinical studies show Sephience provides over 70% greater phenylalanine reduction than BH4 and supports diet liberalization.
High compliance, low discontinuation, and strong uptake in both therapy-naive and previously treated patients.
Addressable market is nearly the entire PKU population (17,000 in the U.S.), with early penetration into all key segments.
2026 will see expanded launches in Japan, Brazil, and other countries, leveraging global commercial teams.
Pipeline and R&D progress
PTC518 for Huntington’s disease showed positive phase II results, with phase III (INVEST Global HD) to enroll ~770 participants globally.
FDA supports potential accelerated approval for PTC518; next data update expected in first half of 2026.
Vatiquinone for Friedreich’s Ataxia demonstrated significant slowing of disease progression; ongoing discussions with FDA on next steps.
Advanced early-stage programs from RNA splicing and inflammation/ferroptosis platforms, including PTSeq engine and new targets.
Strategic partnerships and internal development expected from expanded splicing platform capabilities.
How do EU reimbursement nuances impact VYJUVEK?
What criteria guide rare vs. expanded pipeline?
How does platform tech accelerate pipeline CMC?
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Next Krystal Biotech earnings date
Q4 202513 Feb, 2026
Krystal Biotech
Next Krystal Biotech earnings date
Q4 202513 Feb, 2026
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Frequently asked questions
Krystal Biotech Inc., known as Krystal Biotech, is a biotechnology firm engaged in the development and commercialization of genetic medicines targeting rare and serious diseases. The company focuses on creating treatments using gene therapy approaches, with a significant emphasis on dermatological conditions. Its product pipeline includes innovative therapies such as bercolagene telserpavec (B-VEC) for dystrophic epidermolysis bullosa, a severe skin disorder, and other treatments targeting conditions like autosomal recessive congenital ichthyosis and aesthetic skin conditions. Utilizing its proprietary skin TARgeted Delivery (STAR-D) platform, Krystal Biotech aims to advance treatments for various skin diseases, leveraging gene therapy's potential to offer long-term solutions. The company is headquartered in Pittsburgh, Pennsylvania, and its shares are listed on the NASDAQ under the ticker symbol KRYS.
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