Zevra Therapeutics (ZVRA) TD Cowen 45th Annual Healthcare Conference summary

Strategic vision and infrastructure

• Focused on rare diseases with two commercial-stage products and an advanced clinical pipeline, including phase III and II programs.

• Four strategic pillars: commercial excellence, pipeline and innovation, talent and culture, and corporate foundation.

• Lean commercial infrastructure supports current and future product launches, with about a dozen sales and commercial staff and scalable back-office support.

• Strong financial position, bolstered by a recent $150 million sale of a pediatric priority review voucher.

Commercial products and market execution

• MIPLYFFA approved for Niemann-Pick disease type C, launched in late 2023, with orphan drug exclusivity and strong safety and efficacy data.

• OLPRUVA, acquired in 2023, targets urea cycle disorders with a differentiated, palatable, and convenient dosing profile.

• Both products leverage a shared commercial infrastructure, including patient support and reimbursement services.

• Early MIPLYFFA launch exceeded expectations, with rapid conversion of expanded access patients and new de novo enrollments.

Pipeline and development updates

• CELIPROLOL in phase III for Vascular Ehlers-Danlos syndrome, with ongoing enrollment and strong historical efficacy data.

• KP1077 (serdexmethylphenidate) showed positive phase II data for idiopathic hypersomnia; seeking strategic alternatives for commercialization due to lack of synergy with current infrastructure.

• Next major catalyst is the regulatory pathway for MIPLYFFA in Europe, with efforts underway to file an MAA and expand access.