Zevra Therapeutics (ZVRA) Canaccord Genuity 44th Annual Growth Conference & Private Company Showcase 2024 summary

Pipeline and regulatory progress

• Achieved a positive advisory committee meeting for arimoclomol, with a 97% likelihood of FDA approval based on historical data.

• OLPRUVA is commercially available for urea cycle disorders, with over 90% of target clinicians reached.

• Arimoclomol is positioned to be the first approved therapy for Niemann-Pick type C, with high patient and physician awareness.

• Plans to transition all U.S. expanded access program (EAP) patients to commercial product within 12 months of launch.

• European regulatory strategy includes leveraging new robust data to address prior CHMP concerns and ongoing engagement with EMA.

Commercialization and market access

• OLPRUVA launch was slower than anticipated due to market dynamics, but commercial team is now established for both products.

• Payers are receptive to arimoclomol's clinical value and open to a range of pricing, with final price dependent on label specifics.

• U.S. pricing will inform European pricing, with an expected 30% lower price in Europe, following ultra-orphan drug norms.

• France generates pre-commercial revenue under the ATU program, with about $2 million net per quarter from 30 patients.

• Commercial and medical affairs teams are focused on physician education and early diagnosis to close the gap between diagnosed and prevalent patients.

Labeling, payer dynamics, and patient transition

• Label negotiations are ongoing, aiming for a label reflecting the studied population: arimoclomol vs placebo with routine care.

• Payers are expected to allow arimoclomol to be added to existing routine care, including off-label miglustat.

• Transition from EAP to commercial product in the U.S. is targeted for completion within 12 months post-approval.

• Global EAP will continue in Europe until commercial approval is secured.