Zevra Therapeutics Inc
NASDAQ:ZVRA
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Zevra Therapeutics Inc
NASDAQ:ZVRA
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Zevra Therapeutics Inc
Zevra Therapeutics, Inc. is a clinical-stage specialty pharmaceutical company, which engages in the discovery and development of proprietary prodrugs. The company is headquartered in Celebration, Florida and currently employs 36 full-time employees. The company went IPO on 2015-04-16. The firm is engaged in creating therapies for diseases with limited or no treatment options. The firm has a diverse portfolio of products and product candidates, which includes a combination of both a clinical-stage pipeline and commercial stage assets. The company employs its LAT platform technology to create a portfolio of approved products. Its product candidates include Arimoclomol, KP1077IH, KP1077N and AZSTARYS. Arimoclomol is an orally delivered, investigational product candidate being developed for Niemann-Pick disease type C (NPC). KP1077 is the Company's lead clinical development product candidate, which is being developed as a treatment for idiopathic hypersomnia (IH) and narcolepsy. KP1077 is comprised solely of serdexmethylphenidate (SDX), the Company's prodrug of d-methylphenidate (d-MPH). AZSTARYS is a prodrug for the treatment of ADHD in patients aged six years or older.
Zevra Therapeutics, Inc. is a clinical-stage specialty pharmaceutical company, which engages in the discovery and development of proprietary prodrugs. The company is headquartered in Celebration, Florida and currently employs 36 full-time employees. The company went IPO on 2015-04-16. The firm is engaged in creating therapies for diseases with limited or no treatment options. The firm has a diverse portfolio of products and product candidates, which includes a combination of both a clinical-stage pipeline and commercial stage assets. The company employs its LAT platform technology to create a portfolio of approved products. Its product candidates include Arimoclomol, KP1077IH, KP1077N and AZSTARYS. Arimoclomol is an orally delivered, investigational product candidate being developed for Niemann-Pick disease type C (NPC). KP1077 is the Company's lead clinical development product candidate, which is being developed as a treatment for idiopathic hypersomnia (IH) and narcolepsy. KP1077 is comprised solely of serdexmethylphenidate (SDX), the Company's prodrug of d-methylphenidate (d-MPH). AZSTARYS is a prodrug for the treatment of ADHD in patients aged six years or older.
Revenue: Zevra reported total net revenue of $34.1 million in Q4 2025 and $106.5 million for full-year 2025, driven primarily by MIPLYFFA (arimoclomol) which generated $26.4 million in Q4 and $87.4 million for the year.
Commercial traction: Strong launch momentum for MIPLYFFA with 52 prescription enrollment forms in 2025 (24 in Q4) and 161 total enrollments since launch; company estimates U.S. prevalence of ~900 patients with 300–350 currently diagnosed.
Patient ID strategy: Management highlighted a three‑pronged approach—disease awareness campaign, a bespoke AI predictive targeting model, and partnerships with genetic testing providers (expanded GeneDx collaboration)—as drivers of newly diagnosed patients.
International expansion: Global Expanded Access Program had 113 patients at year-end 2025; new distribution agreements extended named‑patient shipments beyond Europe with initial variability in ordering expected.
Regulatory update (EU): MAA submitted to EMA in July 2025; company received 120‑day list of questions and plans to submit responses within the 90‑day clock stop.
Pipeline progress: Phase III DISCOVER study for soliprelol (vascular connective tissue disorder) has 52 of planned 150 patients enrolled (8 in Q4) with 1 confirmed event; company engaged FDA in a Type C meeting to explore acceleration options.
Profitability & balance sheet: Net income of $12.2 million in Q4 and $83.2 million for 2025; cash and investments of $238.9 million and total debt of approximately $61.9 million as of Dec 31, 2025.
Execution risks: Management repeatedly flagged quarter‑to‑quarter lumpiness for ultra‑rare patient counts, variable gross‑to‑net dynamics, and multi‑year timelines for international named‑patient programs.
