Zevra Therapeutics (ZVRA) Guggenheim Securities Inaugural Healthcare Innovation Conference summary

Company overview and product portfolio

• Commercial-stage rare disease company with two marketed products and two late-stage development programs.

• MIPLYFFA approved for Niemann-Pick disease type C, launched September 20th.

• OLPRUVA, acquired from Acer Therapeutics, treats urea cycle disorders and launched at the end of January.

• KP1077 in phase 3 for idiopathic hypersomnia; celiprolol in phase 3 for vascular Ehlers-Danlos syndrome.

• Receives royalties from out-licensed AZSTARYS for ADHD.

Commercial launch strategy and execution

• Built a new commercial team focused on rare disease launches, leveraging OLPRUVA experience for MIPLYFFA.

• Emphasis on patient identification, support through reimbursement, and access to therapies.

• 90 patient enrollments for MIPLYFFA, with 69 from the expanded access program and 21 de novo.

• Transition from expanded access to commercial drug is ahead of schedule, aiming to close EAP by end of Q2.

• Specialty pharmacy Orsini manages distribution; revenue recognized upon shipment.

Market dynamics and patient access

• Estimated 900 NPC patients in the US, with 300-350 diagnosed and treated, mostly at 30-40 centers of excellence.

• 80-85% of diagnosed patients use miglustat, now labeled for co-administration with MIPLYFFA.

• Efforts underway to identify undiagnosed patients via awareness, publications, and newborn screening initiatives.

• Average annual price for MIPLYFFA is about $1 million, skewed toward higher dose patients.

• High persistence rates observed; discontinuations mainly due to disease progression or death.