Zevra Therapeutics, Inc.

What they do

Zevra markets MIPLYFFA in the United States for neurological manifestations of Niemann-Pick disease type C (NPC) in patients 2 years and older, in combination with miglustat, following FDA approval on September 20, 2024. It also sells OLPRUVA (sodium phenylbutyrate) for certain urea cycle disorders, acquired with Acer Therapeutics in November 2023. The company runs a global Expanded Access Program for arimoclomol and is developing celiprolol in a Phase 3 trial (DiSCOVER) for Vascular Ehlers-Danlos syndrome.

Revenue drivers

• MIPLYFFA (arimoclomol) — Lead commercial product for NPC; generated $30.2 million of the $39.7 million Q2 2026 net revenue and is the primary growth driver.
• Global Expanded Access Program (EAP) — Contributed $9.0 million of net reimbursements in Q2 2026, with 132 patients enrolled as of June 30, 2026.
• OLPRUVA (sodium phenylbutyrate) — Commercially available U.S. product for certain urea cycle disorders; small at $0.2 million of Q2 2026 net revenue.
• AZSTARYS license and other — Past-due royalties and reimbursements under the AZSTARYS license agreement contributed $0.3 million in Q2 2026; the underlying agreement was terminated in the March 2026 Commave settlement.

Recent performance

Q2 2026 net revenue was $39.7 million, up 53% from $25.9 million in Q2 2025, including $30.2 million from MIPLYFFA. Net income was $8.8 million, or $0.14 per basic and diluted share, versus $74.7 million in Q2 2025, which had included the $148.3 million PRV sale gain partly offset by a $58.7 million intangible impairment and $11.7 million inventory obsolescence charge. Operating expenses fell to $21.0 million from $24.2 million, with R&D up to $4.5 million and SG&A down to $16.6 million. The company received 14 MIPLYFFA prescription enrollment forms in Q2 2026, bringing the total to 184 since launch, with market access stable at 69% of covered lives. Reported cash was $260.2 million, and operating cash flow for the first half of 2026 was $23.2 million versus $(11.8) million a year earlier.

Strategy

Management is prioritizing commercialization and global expansion of MIPLYFFA while keeping OLPRUVA commercially available. It filed an MAA for arimoclomol in the EU in July 2025; after CHMP adopted a negative opinion in July 2026, the company requested re-examination. It is enrolling the Phase 3 DiSCOVER trial of celiprolol in VEDS and plans to expand the pipeline through inorganic growth. The March 2026 Commave settlement sold the SDX portfolio (AZSTARYS and KP1077) and terminated the AZSTARYS license agreement, sharpening focus on rare-disease assets.

Risks

• EU regulatory setback — CHMP adopted a negative opinion on the arimoclomol MAA in July 2026, and a re-examination is pending, so European approval and revenue are uncertain.
• Limited commercial infrastructure — The company states it has limited marketing and sales experience and depends on building or partnering for sales, marketing and distribution capabilities.
• Concentrated revenue — MIPLYFFA accounted for $30.2 million of $39.7 million Q2 2026 net revenue, so results hinge on a single product and its market access (69% of covered lives).
• Clinical and pipeline execution — The event-driven Phase 3 DiSCOVER trial of celiprolol had 66 patients enrolled and only three confirmed events as of Q2 2026, with timing dependent on event accrual.

Outlook

Management points to U.S. commercial execution of MIPLYFFA, continued global EAP enrollment, and pursuit of EU access via the re-examination request. It expects continued FDA engagement in the second half of 2026 to explore pathways to accelerate celiprolol development. The company notes it cannot guarantee continued positive operating cash flow despite recent positive quarters.