Zevra Therapeutics (ZVRA) H.C. Wainwright 28th Annual Global Investment Conference summary

Recent progress and strategic focus

• Transitioned to a commercial-stage organization focused on ultra-rare diseases in the U.S., with ongoing global expansion, particularly in Europe and other regions.

• MIPLYFFA (arimoclomol) launch in the U.S. has revealed a broader patient base, including adults, with 184 prescription enrollment forms by end of Q2 and a target market of 900 patients.

• Emphasis on increasing diagnosis rates and expanding the total addressable market through education, earlier testing, and peer-to-peer physician support.

• Updated clinical guidelines now recommend early genetic testing and combination therapy, supporting broader and earlier treatment.

• Investments in machine learning and data analysis are helping to identify undiagnosed patients, especially adults, and refine outreach strategies.

Commercial execution and patient access

• High refill rates and low discontinuation are achieved through dedicated patient services and ongoing education for physicians and payers.

• Internal metrics focus on newly diagnosed patients and connecting physicians with experts to improve treatment plans.

• Efforts to reach the full potential market include targeted outreach using EMR and claims data, and learning from patterns such as cataplexy codes in adult patients.

• The presence of additional therapies and updated guidelines is expected to increase awareness and diagnosis rates, benefiting the entire patient community.

European and global expansion

• Confidence in a positive outcome for the European re-examination is based on robust data and updated clinical guidelines.

• Ongoing engagement with European regulators and continued demand for expanded access in several countries.

• Strategic flexibility in Europe and other markets, with decisions on direct commercialization or partnerships pending regulatory outcomes.