Strong Q4 and Full-Year Revenue
Q4 net revenue from VYJEVEC was $107.1M; year-to-date net revenue was $389.1M, representing ~34% growth versus full year 2024. Q4 revenue rose nearly 10% versus the prior quarter and ~18% versus prior-year Q4. Total net VYJEVEC revenue since launch exceeds $730M.
High Gross Margins and Profitability
Reported gross margin was 94% for the quarter and full year (guidance 90–95% expected going forward). Net income was $51.4M in Q4 ($1.77 basic / $1.70 diluted) and $204.8M for the full year ($7.08 basic / $6.84 diluted), aided by a one-time noncash tax benefit.
Strong Cash Position
Year-end combined cash and investments totaled $955.9M, providing substantial runway to support global launches and pipeline milestones.
Commercial Momentum and Global Launch Progress
US commercial momentum: added >50 new prescribers in Q4 and >500 unique prescribers since launch; reimbursement approval acceleration for three consecutive quarters with >60 approvals since launch. Distributor agreements now cover >20 countries with a goal to expand to >40 in 2026; expanded distributor network to include Israel.
Early International Adoption
Launches in Germany, France (AP2 early access), and Japan progressed with estimated >90 patients prescribed across Germany, France and Japan combined; Japan pricing negotiated and local distribution model enabling home delivery implemented.
Pipeline Breakthroughs — KB407 (CF)
KB407 showed successful delivery and expression of full-length wild-type CFTR in lung biopsies. Observed transduction rates ranged 29%–42% across biopsied patients (including modulator-ineligible), with apical CFTR expression and durability out to at least 96 hours. Repeat-dosing study planning with expectation to start repeat dosing in H1.
Registrational Program Updates and Home Dosing Enablement
Protocol updates: KB801 (EMERALD-1) up-sized to ~60 patients and dosing modified to allow once-daily home or caregiver administration; KB803 amended to allow three-times-weekly home administration. Over half of clinical sites activated and the company expects data readouts for these programs before year-end.
Regulatory Designations and Recognition
FDA granted Fast Track designation for KB111 and RMAT designation for KB707, supporting accelerated pathways. VYJEVEC received the Prix Galien in France for innovation and clinical impact.