Zevra Therapeutics closes $150 million sale of rare pediatric disease priority review voucher

Zevra Therapeutics, Inc. (Nasdaq: ZVRA) said on April 7, 2025 that it closed the sale of its Rare Pediatric Disease Priority Review Voucher (PRV) for gross proceeds of $150 million. The company reported the sale in a Form 8-K and a press release. Zevra calls the money non-dilutive capital. It is still a one-time sale of a voucher, not recurring product revenue.

Terms and origin of the voucher

According to the 8-K, Zevra completed the sale to the buyer on April 1, 2025, under an asset purchase agreement dated February 26, 2025, which the filing calls the PRV Transfer Agreement. Zevra received the voucher when the FDA approved MIPLYFFA (arimoclomol), to be used with miglustat, for the neurological manifestations of Niemann-Pick disease type C (NPC) in adults and children 2 years of age and older. The press release says the FDA granted the PRV in September 2024 and calls MIPLYFFA the first U.S. approved treatment for that ultra-rare neurodegenerative disease. That is the company's own description.

The cash figures are preliminary

The 8-K reports that as of March 31, 2025, cash, cash equivalents and investments were approximately $68.7 million. The press release puts net cash proceeds from the voucher sale at $148.3 million and says adding them would bring the total to $217.0 million. The filing says the March 31 figure is preliminary, unaudited and based on management estimates, and subject to completion of the company's financial closing procedures. Its independent registered public accounting firm has not reviewed the estimate.

Losses behind the balance sheet

The 10-K, filed March 12, 2025, shows the scale of the burn. The company reported a net loss of $105,511 thousand for 2024, up from $46,049 thousand for 2023. Net cash used in operating activities was $69,665 thousand in 2024, compared with $33,535 thousand a year earlier. The 10-K says the company has historically had significant negative net operating cash flows since inception, and that its ability to continue as a going concern may require additional financing. It also says the company may never achieve or maintain profitability. Against that record, the voucher proceeds are a one-time cash event.

How Zevra funded itself before

The 8-K calls the buyer only "the buyer" and does not name it. Before the sale, the 10-K says, Zevra raised equity in 2024. Under an underwriting agreement dated August 8, 2024 with Cantor Fitzgerald & Co. and William Blair & Company, L.L.C. as representatives of the underwriters, it sold 10,615,385 shares at $6.50 per share for net proceeds of approximately $64.5 million. It also set up a 2024 ATM Agreement with Citizens JMP Securities LLC for up to $75.0 million, with a 3.0% commission, and no shares had been sold under it by December 31, 2024.

What the money is for

R. LaDuane Clifton, Zevra's Chief Financial Officer, said in the release: "The $150 million in gross proceeds is non-dilutive capital that further enhances our financial flexibility to support our strategic priorities." The company lists those priorities as the commercial launches of MIPLYFFA and OLPRUVA, support for an ongoing Phase 3 trial of celiprolol, and a stronger balance sheet for future investments. The release does not say how much of the $148.3 million will go to each.

Business and pipeline

Zevra describes itself in the release as a commercial-stage company focused on therapies for people living with rare disease. The release names MIPLYFFA and OLPRUVA as products in commercial launch and a Phase 3 trial of celiprolol as an ongoing program.

Signatories

Timothy J. Sangiovanni, CPA, Senior Vice President, Finance and Corporate Controller, signed the 8-K. Neil F. McFarlane is President and Chief Executive Officer, and Clifton is Chief Financial Officer and Treasurer, according to the 10-K.

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