ZVRA Touches New High - Can This Zebra Keep Galloping?

(RTTNews) - Zevra Therapeutics Inc. (ZVRA), which emerged as a commercial-stage company in 2024, is continuing its transformation into a patient-focused leader in rare disease therapeutics. The company's shares touched a 52-week high of $10.03 last week.

The lead product of the company is MIPLYFFA (arimoclomol), an orally delivered treatment for Niemann-Pick disease type C, an ultra-rare, progressive, and neurodegenerative disorder. Approved by the U.S. regulatory agency in September 2024, the drug was commercially launched the following month. The company is expected to seek European Medicines Agency's approval for MIPLYFFA during the second half of this year.

In addition to MIPLYFFA, Zevra also markets OLPRUVA in the U.S., an oral suspension, indicated for the treatment of certain patients living with urea cycle disorders. Olpruva, approved by the FDA in December 2022, was launched in the market in January 2024. Olpruva came under Zevra's fold, following the acquisition of Acer Therapeutics Inc. in November 2023.

The company also has a partnered product, Azstarys, from which it derives royalties and other reimbursements under a license agreement with Commave Therapeutics SA, an affiliate of Gurnet Point Capital. Azstarys is an FDA-approved, once-daily product for the treatment of attention deficit hyperactivity disorder in patients aged six years and older.

The most advanced drug candidate in the pipeline is Celiprololm, which is being developed for the treatment of Vascular Ehlers-Danlos Syndrome, a severe autosomal dominant genetic connective tissue disorder characterized by the risk of dissection and rupture of the arteries, gastrointestinal tract, and uterus.

Celiprololm is under a phase III trial, dubbed DiSCOVER.

Also in the pipeline is KP1077, a potential treatment for idiopathic hypersomnia, which is characterised by brain fog, excessive daytime sleepiness, and sleep inertia. KP1077 is a phase III-ready compound for which the company is seeking strategic alternatives.

A Review of Q1, 2025

For the first quarter ended March 31, 2025, Zevra reported total net revenue of $20.4 million, a significant increase from $3.4 million in the same period of 2024. This growth was primarily driven by $17.1 million in net revenue from MIPLYFFA. Additional revenue contributions included $0.1 million from OLPRUVA, $2.3 million in net reimbursements from the French early access program (EAP) for arimoclomol, and $0.9 million in royalties and other reimbursements related to the AZSTARYS license agreement.

Net loss narrowed to $3.1 million, or $0.06 per share, for Q1 2025, from $16.6 million, or $0.40 per share, in Q1 2024.

The company's cash, cash equivalents, and investments were $68.7 million as of Mar. 31, 2025. On Apr.7, 2025, the company closed the sale of its Rare Pediatric Disease Priority Review Voucher (PRV) for net cash proceeds of $148 million.

The Path Forward

Looking ahead, Zevra is focused on positioning MIPLYFFA as the cornerstone treatment of Niemann-Pick disease type C globally, while also working to fully realize the commercial potential of OLPRUVA. At the same time, the company continues to advance its development pipeline to support long-term growth.

Formerly known as KemPharm Inc., the company made its debut on the NASDAQ Global Market under the ticker symbol "KMPH" on April 16, 2015, priced at $11 per share. The company changed its name to Zevra Therapeutics, Inc. and ticker symbol to "ZVRA" on March 1, 2023.

Zevra in Greek means zebra. Since the zebra is the official symbol of rare disease, the company rebranded as Zevra Therapeutics to reflect its mission of bringing life-changing therapeutics to the rare disease community.

ZVRA has traded in a range of $4.27 to $10.03 in the last 1 year. The stock closed Thursday's (July 3, 2025) trading at $9.74, up 3.51%.