Reaffirmed Full-Year Revenue Guidance
Q1 revenues were $72.4M (down 8% YoY) but management reaffirmed full-year 2026 revenue guidance of $405M–$425M, implying growth of ~8%–13% YoY and signaling confidence in near‑term recovery and product momentum.
Strong Joenja Commercial Momentum
Joenja revenue grew 34% YoY to $14.1M in Q1; U.S. paid patients reached 127 (+25% YoY) with a U.S. fill rate of 85%. Management expects further acceleration from upcoming launches in Germany and Japan and pediatric label expansion in the U.S.
RUCONEST Franchise Durability and New Prescriber/Enrollment Growth
RUCONEST remains a strategic cash engine despite headline declines: added ~50 new patient enrollments and 23 new prescribers in Q1; the majority of existing RUCONEST patients have remained on therapy nine months after the launch of a new oral competitor.
Pipeline and Regulatory Progress
Key clinical and regulatory catalysts ahead: resubmission of Joenja pediatric sNDA for highest doses in April with FDA decision expected in ~6 months; planned summer sNDA for lower doses; two Phase II readouts for leniolisib in higher‑prevalence PIDs expected in H2 2026; napazimone registrational study expected to complete enrollment in 2026 with readout in 2027.
Disciplined Cost Management and Positive Operating Cash Flow
Total operating expenses down 9% YoY (adjusted flat excluding nonrecurring 2025 acquisition costs). Q1 operating cash flow was positive at $2M. Cash and marketable securities totaled $171.8M. Company expects gross margin ~90% and reiterated Opex guidance of $330M–$335M including $60M incremental R&D.