Company: ZVRA
Filing Date: 2025-05-08
Form Type: DEFA14A
Source: 0001193125-25-115961
Chunk: 5

Company: ZEVRA THERAPEUTICS, INC.
Filing Date: 2025-05-08
Form: DEFA14A
Chunk 5
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. patients have already been prescribed, and the coverage approvals are rapidly being approved. This is a testament to the work Zevra has been doing in the NPC community for some time. “ ” “ March 2025 We expect the launch to continue its strong positive trend in 1H25 as the EAP and early de novo patients continue to work through the funnel and secure paid drug from the company. With the sale of the $150MM PRV and a strong early launch execution, ZVRA has sufficient cash to fund operations into 2029 and beyond (PRV not included in guidance), per management. While the U.S. launch remains the key investor focus, the company continues to execute on expansion opportunities, with EMA filing anticipated in 2H25. ” May 2025 We believe management continues to execute strongly on its strategic vision and most importantly in the near term, on the success of the MIPLYFFA launch, which we expect to continue to drive shareholder value. We continue to view the cash position as a demonstration of corporate strength, with pro forma cash following the closing of the PRV sale last month. ” “

2 Advancing our Mission of Becoming a Leading Rare Disease Company

Onset at Any Age Makes NPC particularly difficult to diagnose Heterogeneous Rate of progression, always fatal i. Miglustat is not approved in the U.S. for the treatment of NPC ii. carbamylphosphate synthetase (CPS), ornithine transcarbamylase (OTC), or argininosuccinic acid synthetase (AS) Transforming Patients' Lives Through our Commercial Products Zevra’s market access team works to increase awareness and educate potential patients, physicians, and treatment teams; we aim to expand knowledge about, and increase adoption of, our life saving drugs Advancing Treatment for NPC, an Ultra-Rare, Heterogeneous and Fatal Disease MIPLYFFA ~900 Addressable patients in the U.S. living with NPC; only 300-350 are diagnosed ~1,100 Addressable patients living with NPC in Europe NPC is a neurodegenerative, progressive, and fatal lysosomal storage disorder caused by progressive lipid build up; NPC leads to cell death and ultimately organ dysfunction in the spleen, liver, and brain ~80% Patients in our Phase 2/3 clinical trial who received MIPLYFFA in combination with miglustat, disease progression was halted through 12 months of treatmenti Fulfilling the Unmet Need of