Zevra Therapeutics (ZVRA) H.C. Wainwright 4th Annual BioConnect Investor Conference summary

Strategic focus and organizational changes

• Implemented a strategic plan at the end of 2023 to focus on core areas: U.S. business, global regulatory expansion, and pipeline development.

• Relocated headquarters to Boston to access top commercial, regulatory, and development talent, supporting long-term growth.

• Streamlined operations by divesting non-core activities and leveraging facilities in Florida and Copenhagen.

U.S. and global product launch and growth drivers

• MIPLYFFA launch in the U.S. targets both diagnosed and undiagnosed Niemann-Pick C patients, with increasing confidence in reaching total addressable market.

• AI-driven patient identification leverages electronic health records and claims data to uncover undiagnosed cases, improving diagnosis and treatment rates.

• Expansion outside the U.S. includes named patient pre-commercial revenue and broadening global access.

Patient enrollment, diagnosis, and AI integration

• U.S. diagnosed patient pool estimated at 300–350, with total prevalence around 900; Europe shows higher maturity in diagnosis.

• AI models triangulate data to identify likely undiagnosed patients, enabling targeted physician outreach and earlier intervention.

• Continuous learning from patient journeys and global expert networks enhances AI effectiveness and supports earlier diagnosis.