Zevra Therapeutics (ZVRA) investor relations material

Company overview and portfolio

• Commercial-stage rare disease company with two approved products for urea cycle disorders and Niemann-Pick disease type C (NPC).

• Late-stage development program in vascular Ehlers-Danlos syndrome, currently enrolling phase III patients.

• Holds royalties and milestones from an ADHD program commercialized by another party.

• Maintains a robust balance sheet with approximately $230 million in capital.

Niemann-Pick disease type C (NPC) launch and market dynamics

• Product for NPC approved about a year ago; 137 prescription enrollment forms received, covering ~40% of diagnosed US patients.

• Ongoing efforts to expand diagnosis and awareness, including genetic testing and outreach to community physicians.

• Patient mix is about 50% adults and 50% children, mirroring the expanded access program (EAP).

• Increasing identification of newly diagnosed patients, suggesting the true market may be larger than initial estimates.

• High adherence and persistency rates observed, similar to EAP experience.

Physician and payer engagement

• Education efforts target both centers of excellence and community doctors to improve diagnosis and treatment rates.

• Market research indicates physicians prefer the product based on long-term data; payers are supportive due to robust evidence.

• Physicians and payers are open to multi-modality treatments for this heterogeneous disease.

• Strong data package, including five-year OLE data, supports payer reimbursement and physician adoption.