Zevra Therapeutics (ZVRA) Canaccord Genuity's 46th Annual Growth Conference summary

Commercial performance and market expansion

• MIPLYFFA remains a core focus, with 184 distinct U.S. patient enrollments, representing both previously diagnosed and newly diagnosed Niemann-Pick disease type C patients.

• The addressable U.S. market is estimated between 350 and 900 patients, with ongoing efforts to expand beyond the currently diagnosed population.

• Adult patient enrollment has been a significant and unexpected growth area, with real-world data showing a 50/50 adult-to-child split.

• Integrated strategies include disease awareness, genetic testing, AI-driven patient identification, and publication of clinical guidelines.

• The average wholesale acquisition cost is approximately $85,000 per patient per month, consistent across the current patient mix.

Strategic initiatives and competitive landscape

• Geographic expansion is underway, with a European Medicines Agency re-examination process ongoing and strong demand for MIPLYFFA through expanded access programs in Europe.

• The company leverages learnings from the more mature European market to drive U.S. patient identification and treatment rates.

• Combination therapy with miglustat is emphasized, aligning with new clinical guidelines and distinguishing the product with randomized controlled data.

• The competitive landscape is evolving, with new entrants expected to complement rather than displace current therapies, especially for younger patient segments.

• Investment in disease awareness and physician education is ongoing, with spending regularly evaluated for discipline and growth potential.

Pipeline development and business development

• The celiprolol program for Vascular Ehlers-Danlos Syndrome is advancing, with ongoing FDA engagement to accelerate development and enrollment.

• The company is actively seeking complementary ultra-rare disease assets, focusing on late-stage or commercial products that fit existing infrastructure.

• Strong capital position with $260 million and no debt enables flexibility for both organic and inorganic growth strategies.

• Business development is milestone-driven, targeting assets that align with the current therapeutic and commercial focus.