Zevra Therapeutics (ZVRA) Morgan Stanley 24th Annual Global Healthcare Conference summary

Mission and strategic focus

• Aims to redefine possibilities for people with rare diseases by developing and delivering innovative therapies, focusing on ultra-orphan conditions with very low prevalence rates.

• Operates with a three-pronged strategy: U.S. commercial growth, global expansion, and advancing a pipeline asset for vascular Ehlers-Danlos syndrome.

• Seeks to leverage learnings from each patient journey to improve diagnosis, treatment, and patient support.

• Aspires to become a leading rare disease company and a preferred partner for assets that fit its infrastructure.

Commercial progress and patient impact

• Achieved significant U.S. market penetration for Niemann-Pick disease type C, enrolling 184 patients out of an estimated 350 diagnosed, with about half being adults.

• Generated approximately $30 million in quarterly revenue less than two years post-launch, driven by robust patient-finding and support programs.

• Published long-term data demonstrating disease progression halting and reinforced by updated clinical guidelines supporting early and combination therapy.

• Maintains high patient persistency and low discontinuation rates, supported by comprehensive patient services and ongoing feedback.

Global expansion and regulatory strategy

• Pursuing European approval for its lead therapy, supported by one of the largest NPC datasets and four-year extension data.

• Currently in the EMA re-examination process, with a global expanded access program enrolling 132 patients, mainly in Europe.

• Confident in continued international demand, leveraging U.S. label for named patient reimbursement in ex-U.S. markets.

• Will finalize European go-to-market strategy post-EMA decision.