Strong Revenue Growth and Profitability
Q4 net revenue of $91.7M, representing sequential growth of nearly 40% from $66.0M in Q3; full-year 2025 net revenue of $190.4M (note: <9 months of commercial sales). Achieved profitability with full-year net income of $20.9M and Q4 net income of ~$43.4M compared to a net loss in the prior-year periods.
Robust Cash Generation and Balance Sheet
Generated $48.7M of cash from operating activities in Q4 and ended the year with $506.1M of cash, cash equivalents and marketable securities (after a $100M accelerated share repurchase), providing strong capital to support U.S. launch, EU activities and new indications.
Successful Commercial Launch Metrics
Since launch through Dec 31, 2025: 1,250 patient start forms (≈12.5% of the U.S. addressable market) and 859 active patients on treatment (up from 764 at end of Q3, a ~12.4% increase quarter-over-quarter). Added 136 new prescribers in Q4 to reach 630 unique prescribers.
Broad Payer Coverage
Secured coverage across commercial, Medicaid and Medicare channels covering over 180 million lives at the end of Q4, and reimbursed claims from ~45 state Medicaid programs through Q4, supporting rapid access and reauthorization success.
Real-World Safety and Adherence
Real-world safety profile mirrors clinical expectations; launch-to-date discontinuation related to adverse events ~12% and total discontinuation ~15%, consistent with management’s long-term expectation of 15%–20%. High adherence observed as patients settle into optimal doses.
Pipeline Expansion Plans — EU and GSD1
EMA MAA validated; Day 120 questions answered and Day 180 questions expected by end of Feb; decision anticipated mid-2026. Pursuing glycogen storage disease type 1 (GSD1) with orphan designation in U.S. and EU, IND planned in H1 2026 and trial initiation later in 2026.