Ionis Pharmaceuticals (IONS) investor relations material

Introduction and purpose

• ZANVASTRO (zilganersen) received FDA approval as the first and only disease-modifying treatment for Alexander disease (AxD) in both pediatric and adult patients.

• Alexander disease is an ultra-rare, progressive, and often fatal neurological disorder with significant unmet medical need and limited prior treatment options.

Details of approval or decision

• FDA approved ZANVASTRO for children and adults with a broad label nearly three weeks ahead of schedule, based on positive pivotal study data showing statistically significant and clinically meaningful benefit on motor function.

• Approval included a Rare Pediatric Disease Priority Review Voucher, offering the opportunity to accelerate future FDA reviews.

• ZANVASTRO will be available in the U.S. within weeks of approval.

• Regulatory submissions in Europe and Japan are planned for next year or 2027.

Impact on industry and stakeholders

• ZANVASTRO marks the first independent neurology launch from the company's pipeline, expanding commercial capabilities and establishing a foundation for future launches in rare neurological diseases.

• Strong partnership with the Alexander disease patient community and strategies to improve diagnosis and access.

• Patient and caregiver support services will be offered, including education and insurance assistance.

• Commercial access outside the U.S. is being pursued, with Recordati as a partner.