At Ionis, we are focused on bringing better futures to people living with serious diseases. As pioneers in RNA-targeted medicines, we combine deep scientific understanding with innovation and a sense of urgency to deliver life-changing medicines, guided by a commitment to operating responsibly and sustainably.
Building on our commitment, 2025 was a defining year for Ionis.
We strengthened our position as a commercial-stage biotechnology company with two major milestones. Our medicine for familial chylomicronemia syndrome delivered a strong first year, and the U.S. Food and Drug Administration approval of our hereditary angioedema treatment marked the first RNA-targeted therapy for this disease. Together, these achievements reflect both the strength of our science and our ability to deliver for patients.
We also advanced our pipeline in meaningful ways. We reported positive Phase 3 results in severe hypertriglyceridemia (sHTG), demonstrating significant reductions in triglycerides and acute pancreatitis events as well as positive Phase 3 results in Alexander disease (AxD), a rare, progressive and often fatal neurological condition with no approved disease-modifying therapies. These programs position us for expected additional FDA approvals and independent launches in 2026. At the same time, our mid-stage pipeline and our late-stage partnered pipeline continues to progress, with multiple late-stage readouts and regulatory milestones anticipated this year and next.
As we grow, our approach to corporate responsibility remains central to how we operate.
Our 2025 Corporate Responsibility Report: Building Momentum for Better Futures highlights progress across three core CR pillars:
- Innovating to improve the lives of people with serious diseases
- Empowering our employees and communities
- Operating responsibly and sustainably
We continue to innovate for patients. We are advancing new medicines and have earned multiple FDA Priority Review designations and Breakthrough Therapy designations across our pipeline. We also deepened our engagement with the patient communities globally, working with more than 100 advocacy organizations.
We are investing in our people and communities. Ionis was recognized as the #2 Top Employer by Science magazine, reflecting the strength of our culture and team. Our employees contributed more than 2,100 volunteer hours and supported 35 nonprofit organizations. We also marked the tenth anniversary of our Surf Away+ program, expanding adaptive experiences for people living with neurological diseases.
We’re sharpening our focus on operating sustainability. In 2025, we completed our first Climate Risk Assessment, enhancing our report aligned with the Task Force for Climate-related Disclosures (TCFD) and further incorporating its recommendations.
Looking ahead, we remain focused on advancing our pipeline, preparing for future product launches and continuing to grow as a responsible, sustainable company. The dedication of our employees continues to drive this progress and extend our impact for patients and communities.
Together, we are advancing RNA-targeted medicines and building a company that delivers a healthier future for patients, society and all Ionis stakeholders.