Zevra Therapeutics (ZVRA) H.C. Wainwright 26th Annual Global Investment Conference 2024 summary

Physician and patient community engagement

• High anticipation and support from advocacy groups and physicians for new therapy options in Niemann-Pick C, with many eager to onboard or trial arimoclimol upon approval.

• Physicians outside the current expanded access program (EAP) express interest in using arimoclimol once it is no longer experimental.

• Increased diagnosis rates are expected once a therapy is available, as awareness and education efforts intensify.

• There is a significant population of patients and physicians ready to participate beyond the current EAP.

• Substantial ex-US EAP presence, especially in Europe, with reimbursement through nominative ATU programs.

Market access and commercial strategy

• Olpruva launch focused on market access, commercial engagement, and patient activation, with 75% coverage and 90% of target base reached.

• Patient awareness remains a key area for investment to drive uptake in a mature UCD market.

• Significant synergy between arimoclimol and Olpruva, with overlapping call points at centers of excellence and mutual benefits for market penetration.

• Regular physician engagement through both products enhances opportunities for education and patient identification.

Regulatory and pipeline updates

• Efforts are underway to recatalyze the European application for arimoclimol, leveraging robust data and potential US FDA approval.

• End-of-phase II meeting for KP1077 in idiopathic hypersomnia is scheduled before the end of the quarter, aiming to inform a differentiated phase III program.

• Focus for KP1077 is on addressing unmet needs, particularly sleep inertia in idiopathic hypersomnia.