Krystal Biotech (KRYS) 44th Annual J.P. Morgan Healthcare Conference summary

2025 performance and financial highlights

• Achieved $823 million in revenue, surpassing guidance, with $588 million from product sales and $112 million from Sephience since launch.

• Closed the year with over $1.94 billion in cash, maintaining strong expense management and moving toward cash flow breakeven.

• Provided 2026 revenue guidance of $700–$800 million, reflecting 19%–36% growth in product revenue, excluding Evrysdi royalties.

• Operating expenses for 2026 are guided at $680–$720 million, about 6% lower than the prior year midpoint.

• Company is positioned to potentially reach cash flow breakeven in 2026, a key milestone for future profitability.

Sephience launch and market opportunity

• Sephience launched in the U.S., Europe, Japan, and other countries, with over 940 patients on therapy and $112 million in revenue in five and a half months.

• Demonstrates broad uptake across all age groups and disease severities, with high refill rates and low discontinuation.

• Dual mechanism of action enables benefit for the full spectrum of PKU patients, including those with classical PKU.

• Clinical studies show Sephience provides over 70% greater phenylalanine reduction than BH4 and enables diet liberalization.

• Global expansion planned for 2026, including launches in Japan, Brazil, and additional countries, leveraging early access programs.

Pipeline and R&D progress

• PTC518 for Huntington’s disease showed positive phase II results, with durable, dose-dependent lowering of huntingtin protein and favorable safety.

• Phase III INVEST Global HD study to enroll ~770 participants globally, with Novartis funding and conducting the trial.

• FDA supports potential accelerated approval pathway for PTC518, with next data update expected in the first half of 2026.

• Vatiquinone for Friedreich’s Ataxia demonstrated significant slowing of disease progression; discussions with FDA on next steps are ongoing.

• Early-stage R&D programs advancing in RNA splicing and inflammation/ferroptosis, with proprietary PTSeq platform accelerating discovery.