For 2025, the Company anticipates: 

• Total revenues between US$325 million and US$340 million (9% to 14% growth), with quarterly fluctuations expected.
• Total operating expenses not to exceed the prior year pre-Abliva impact and we expect US$30 million in Abliva-related operating expenses, including research and development and non-recurring transaction and integration expenses.
• Significant progress finding additional APDS patients in the U.S., supported by VUS validation efforts and subsequently converting patients to paid Joenja® (leniolisib) therapy.
• Increasing ex-U.S. revenues for leniolisib - driven by funded access programs and commercial availability in the U.K.
• Progress towards additional regulatory approvals for leniolisib for APDS patients 12 years of age or older, and submitting regulatory filings in Japan and for pediatric label expansion in key global markets.
• Advancing the two ongoing Phase II clinical trials in PIDs with immune dysregulation to significantly expand the long-term commercial potential of leniolisib.
• Advancing the ongoing pivotal FALCON clinical study for KL1333 in mitochondrial DNA-driven primary mitochondrial diseases.
• Continued focus on potential acquisitions and in-licensing of clinical stage opportunities in rare diseases.

No further specific financial guidance for 2025 is provided.