Strong Full-Year and Quarterly Revenue
2025 net revenue of $106.5M with fourth-quarter net revenue of $34.1M. MIPLYFFA accounted for $87.4M (82% of full-year revenue) and $26.4M (77% of Q4 revenue), demonstrating commercial traction for the lead product.
Profitability Turnaround
Full-year 2025 net income of $83.2M ($1.40 basic, $1.35 diluted) compared to a net loss of $105.5M in 2024 — a swing of $188.7M. Q4 2025 net income was $12.2M versus a Q4 2024 net loss of $35.7M.
Commercial Momentum and Patient Starts
52 prescription enrollment forms in 2025 (24 in Q4) and 161 total enrollments since launch. Management estimates they have captured roughly 40%–50% of currently diagnosed U.S. patients, supporting confidence in longer-term uptake.
Expanded Access and International Footprint
Global expanded access program (EAP) had 113 patients enrolled at year-end 2025. New distribution agreements initiated shipments beyond Europe in Q4 and produced $5.6M of net EAP reimbursements in Q4 ($13M for the full year).
Regulatory Progress in Europe
Marketing Authorization Application submitted to the EMA (July 2025); received a 120-day list of questions at year-end and is prepared to respond within the 90-day clock-stop period, advancing EU approval pathway.
Strong U.S. Market Access
Covered Lives at 68% in the U.S. after the first full year on market, with management reporting strong reimbursement rates for remaining patients via market access and patient services efforts.
Healthy Balance Sheet
As of December 31, 2025, cash, cash equivalents and investments totaled $238.9M with total debt of approximately $61.9M, providing runway to execute commercial expansion and clinical programs.
Operating Expense Discipline & R&D Reduction
Full-year operating expenses decreased $6.6M year-over-year to $90.4M. R&D expense fell to $12.7M for the year, a decrease of $29.4M (≈69.8%) versus the prior year, reflecting lower personnel-related and third-party costs.
Clinical and Real-World Evidence Supporting MIPLYFFA
Company highlighted clinical differentiation: rapid onset of improvement in 12 weeks, halting progression at 12 months, and durable efficacy/safety >5 years. Presentations and EAP analyses demonstrated sustained benefit and strong adherence in multiyear follow-up.