For 2026, the Company anticipates:
- Total revenues between US$405 million and US$425 million (8% to 13% growth), with quarterly fluctuations expected.
- Total operating expenses between US$330 million and US$335 million (6% to 8% growth), including US$60 million incremental R&D expenses to advance the pipeline and US$9 million structural G&A cost reductions based on the plan announced in October 2025.
- Continued RUCONEST® growth, and significant and accelerating Joenja® U.S. and ex-U.S. growth.
- Progress towards additional regulatory approvals and commercial launches for leniolisib for APDS patients 12 years of age or older and for pediatric label expansion in key global markets.
- Top-line data readouts for the two ongoing leniolisib Phase II clinical trials in PIDs with immune dysregulation to expand the asset's addressable patient population.
- Completion of enrollment in the pivotal FALCON clinical study for napazimone (KL1333) in mitochondrial DNA-driven primary mitochondrial diseases.
- Enhancing capital allocation to drive growth and build a leading global rare disease company.
- Continued focus on potential acquisitions and in-licensing of clinical stage opportunities in rare diseases. Financing, if required, would come via a combination of our strong balance sheet and access to capital markets.
No further specific financial guidance for 2026 is provided.
