Oct 27, 2026 earnings call — Quarterly results: RUCONEST recovery and Joenja expansion
Total revenue rose 21% to $117.6 million, with Joenja up 58% and operating profit up 89%.
Revenue $0.1B (est $0.1B) · EPS $0.30 (est $0.21) · Next-quarter revenue outlook $0.4B
Key quotes
"Importantly, we have stabilized underlying US demand with volume returning to prior year levels."
"On September 11, as Kenneth mentioned, the FDA approved our new drug application for the 40 and 50 milligram twice daily dose for children."
"The data showed that treatment with leniolusib resulted in improvements across a number of objective measures consistent with the PI3K-delta mechanism, including spleen volume, lymph node size, lung gas exchange, and liver stiffness."
Analyst Q&A 5
RUCONEST uptake, CEO search, FALCON enrollment
Q (Benjamin Jackson · Jefferies): Asked about the 43 RUCONEST patient additions in 3Q and how that is tracking into Q4. Also asked about the CEO search (internal vs external profile) and why FALCON recruitment is slower—patient ID or competing trials?
A: Management said Q3 is seasonally softer and a single quarter is not a reliable long-term indicator. They highlighted >90% patient retention and disproportionate retention of high-burden (high-vial) patients, and expect momentum to continue into Q4. On the CEO search the board is conducting a formal search and interim co-CEOs remain in place; on FALCON the main bottleneck was site activation and that is now largely behind them with strong recent site activity.
"However, we do not necessarily view a single quarter as a reliable indicator of the long term demand trend." — Leverne, Marsh (CEO)
Joenja launch, CVID study, cash coverage
Q (Sushila Hernandez · Van Lanschott Kempen): Asked for country breakdown and growth triggers for the 30 patients on paid Joenja therapy in launch markets (Japan, Germany, UK). Asked what to expect from the CVID study and what would be a meaningful spleen volume reduction. Asked whether current cash covers prelaunch costs or more financing is needed.
A: They said 30 patients are in launch markets plus 45 in non-launch named-patient use, and they will not disclose country level yet because Japan and Germany launches are early; growth drivers include UK pediatric expansion and physician education in Germany and Japan. For CVID they are looking for a direction of benefit across lymphoproliferation, cytopenias and end-organ measures (including spleen volume decreases seen previously), clinician- and patient-reported measures, and safety/tolerability to set dose. Management said they do not need additional financing; the cash position at end-September plus projected cash flows covers current pipeline and prelaunch costs.
"The cash position at the end of September, together with the future cash flows that we are projecting are covering, let's say, all our current pipeline costs." — Kenneth Lynard EMBA (CEO)
Integrating Joenja into PID and CVID
Q (Joseph Pantginis · H. C. Wainwright): Asked how Joenja would be integrated if PID and CVID indications are approved, and whether that is easier given the current physician base and which specialties could be expanded.
A: They said the same immunologists who treat APDS also treat CVID and genetic PIDs, so the physician base overlaps. Commercial, medical affairs, market access and patient services built for the APDS launch in the U.S. are transferable to CVID/PID; the plan is to call on the same physicians, engage payers, and expand patient services.
"All of those capabilities will be transferable to CDID." — Leverne, Marsh (CEO)
Specialty pharmacy inventory and Q4 impact
Q (Simon Scholes · First Berlin): Asked whether other specialty pharmacies might also build inventory in Q4, given one large pharmacy built early in Q3.
A: They expect specialty pharmacies to increase inventory before year-end and noted one pulled inventory into Q3. They anticipate seasonal adjustments across the rest of the channel in Q4 while demand has stabilized, and that Q4 revenue will also reflect price increases and gross-to-net adjustments.
"We are expecting some seasonal adjustment from the remainder of the channel, including this including the specialty pharmacy that just bought in." — Leverne, Marsh (CEO)
RUCONEST outlook and CVID readout success
Q (Fanyi Zhong · OpCo): Asked if the softer Q4 RUCONEST revenue vs 3Q implies a similar pattern next year (weaker H1, stronger H2). Also asked what would define a successful November CVID readout for leniolumab/leniolisib.
A: For RUCONEST they are at the midpoint of guidance and are forecasting a 3% annual decline; they said Q3 showed stabilization but Q4 will be affected by inventory adjustments and they expect a similar pattern of impact if a new oral competitor launches. For CVID they said the study enriches for immune-dysregulation endotype and they will look for consistent direction of benefit in lymphoproliferation (including spleen and lymph node reductions) and other organ manifestations, plus safety and tolerability.
"So, for RUCONEST, we are at midpoint of guidance, forecasting a 3% annual decline." — Leverne, Marsh (CEO)