Zevra Therapeutics (ZVRA) 12th Annual Cantor Fitzgerald Global Healthcare Conference summary

Company overview and strategic priorities

• Focused on rare diseases with commercial products for Niemann-Pick disease type C (NPC) and OLPRUVA for urea cycle disorders in the U.S., plus global expansion plans for NPC.

• Advancing a phase III program for vascular Ehlers-Danlos syndrome (VEDS) in the U.S.

• Well-capitalized with $260 million in cash and no debt, enabling investment in core priorities.

• Emphasizes three strategic pillars: U.S. business, global expansion, and pipeline development.

• Strong intellectual property protection extends to 2041, supporting long-term growth.

Market opportunity and patient identification

• U.S. NPC market estimated between 350 and 900 patients, with 184 patient enrollment forms as of Q2.

• Significant learning that 50% of new NPC patients are adults, expanding the perceived market.

• Disease awareness, genetic testing support, and physician education are key to identifying new patients.

• European experience shows that approved therapies drive increased diagnosis and market growth.

• Expanded access and compassionate use programs globally support patient identification and treatment.

Commercial execution and growth

• Focused on centers of excellence in the U.S. with a flexible sales force strategy.

• Investments in disease awareness and patient support are driving increased diagnosis and therapy uptake.

• High patient retention and low discontinuation rates observed in long-term data.

• Willingness to refine commercial strategies based on real-world learnings and patient demographics.

• Confident in further market penetration as more adults are diagnosed and treated.