Zevra Therapeutics (NASDAQ:ZVRA) announced today that it will begin trading under the new ticker symbol “ZVRA” on the Nasdaq Global Select Market as of March 1, 2023. The rare disease therapeutics company, formerly known as KemPharm, Inc., has changed its name to Zevra, which means zebra in Greek, the internationally recognized symbol for the rare disease community.

Zevra’s commitment to the rare disease community is reflected in its primary mission to provide life-changing treatments for individuals with rare conditions, their families, and caregivers. The company’s focus is on developing therapies for Niemann-Pick type C disease (NPC) and idiopathic hypersomnia (IH), two rare diseases with no approved treatments in the US.

Arimoclomol, Zevra’s first-in-class investigational product candidate, is an orally delivered drug for the treatment of NPC, a rare disease that primarily affects children and is often fatal. The US Food and Drug Administration (FDA) has granted arimoclomol orphan drug designation, Fast Track designation, and rare pediatric disease designation for the treatment of NPC. Zevra plans to submit a New Drug Application for arimoclomol to the FDA as early as Q3 2023.

Zevra is also developing KP1077, a product candidate for the treatment of IH, a rare sleep disorder. Pending the results from the ongoing Phase 2 trial, the company plans to conduct a pivotal Phase 3 study in IH, with the potential to study an expanded indication in narcolepsy.

Zevra expects several key milestones in 2023, including the planned resubmission of the NDA for arimoclomol to the FDA, interim and final data readouts for KP1077 in IH, and the potential achievement of one or more commercial sales milestones for AZSTARYS®, a partnered asset, during FY 2023.

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