Total Q1 Revenue and Guidance Reaffirmed
Q1 2026 revenue of $72.4M (down 8% YoY) with full-year 2026 revenue guidance reaffirmed at $405M–$425M, implying ~8%–13% growth vs. 2025.
Strong Joenja Growth
Joenja revenue grew 34% YoY to $14.1M globally. U.S. paid patients reached 127 (up 25% YoY), net adds accelerated to 7 patients in the quarter and U.S. fill rate remained high at 85%.
RUCONEST Franchise Durability and New Prescribers
RUCONEST added ~50 new patient enrollments and 23 new prescribers in Q1, and management reports the overwhelming majority of RUCONEST patients have remained on therapy nine months after a new oral competitor launch.
Regulatory and Geographic Progress for Joenja
Meaningful regulatory progress: resubmission of pediatric sNDA (40 mg & 50 mg) in April with FDA decision expected in ~6 months; CHMP positive opinion obtained and Germany launch planned this year; Japan launch anticipated in August.
Pipeline Catalysts and Clinical Momentum
Two Phase II readouts for leniolisib in higher-prevalence PIDs are expected later this year; napazimone registrational study enrollment expected to complete in 2026 with readout in 2027; expanded access CVID data (n=6) show consistent clinical improvement and biomarker modulation.
Positive Operating Cash Flow and Strong Cost Discipline
Positive operating cash flow of $2.0M in Q1; total operating expenses down 9% YoY (flat after adjusting for one-time 2025 acquisition costs); company expects operating expenses $330M–$335M for 2026 and cites $9M benefit from prior 20% G&A headcount reduction.
Healthy Balance Sheet to Support Pipeline
Total cash and marketable securities of $171.8M at quarter-end and management expects available cash plus future operating cash flows to fully support planned pipeline and prelaunch activities.