Zevra Therapeutics Inc.

Zevra Therapeutics, Inc. reported second-quarter 2026 financial results, posting GAAP earnings per share of $0.14, which beat analyst estimates by $0.03. Revenue came in at $39.7 million, a 53.4% increase year-over-year, exceeding expectations by $5.82 million. The company held cash, cash equivalents, and investments totaling $260.2 million as of June 30, 2026, and believes its available financial resources are sufficient to execute on strategic priorities without relying on capital markets. As of the same date, total shares of common stock outstanding were 59,341,906, with fully diluted common shares at 69,298,105, including shares issuable from outstanding equity awards and warrants.

Zevra Therapeutics announced the publication of encouraging Phase 2/3 data for MIPLYFFA in treating Niemann-Pick type C disease in pediatric patients two years or younger. The results, published in Molecular Genetics and Metabolism Reports, come from a sub study of five patients aged 12 to 24 months who received arimoclomol with concomitant miglustat over 36 months as part of an open-label extension trial. The data showed MIPLYFFA was generally well-tolerated with a good safety profile, while development assessments indicated variable delays suggesting underlying phenotypic heterogeneity. MIPLYFFA, which activates transcription factors to upregulate CLEAR genes and remove unesterified cholesterol from lysosomes, was approved by the FDA in September 2024 and has Orphan Medicinal Product designation from the EMA. A marketing authorization application has been filed with the EMA for arimoclomol as a treatment for Niemann-Pick disease.