Zevra Reports First Quarter 2025 Financial Results and Corporate Update

Q1 2025 net revenue of $20.4 million, driven by product net revenue of $17.2 million

Completed sale of PRV for gross proceeds of $150 million, positioning balance sheet to drive the Company's commercial launches and development programs

Company to host conference call and webcast today, May 13, 2025, at 4:30 p.m. ET

CELEBRATION, Fla., May 13, 2025 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company), a commercial-stage company focused on providing therapies for people living with rare disease, today reported its financial results for the first quarter ended Mar. 31, 2025.

"We are continuing Zevra's transformation into a patient-centric, commercial-stage rare disease therapeutics company," said Neil F. McFarlane, Zevra's President and Chief Executive Officer. "With our bolstered cash balance, we are well-positioned to support our strategic priorities, including establishing MIPLYFFA$(R)$ as the cornerstone of treatment for NPC globally, maximizing our commercial opportunity with OLPRUVA(R) , and supporting our growth through our development pipeline."

Commercial Highlights

   -- All active patients in the U.S. Expanded Access Program (EAP) for 
      arimoclomol, have been enrolled to receive MIPLYFFA. The EAP has since 
      closed, including locking the database and deactivating study sites. 
   -- MIPLYFFA: 13 new prescription enrollment forms during Q1 2025, marking 
      122 total through the end of the quarter. In line with expectations at 
      this stage of launch, market access is 38% of covered lives. 
   -- OLPRUVA: 5 new patient enrollment forms were received in Q1 2025, 
      bringing the total from initial product availability in July 2023, and 
      including Zevra's promotion of OLPRUVA since late January 2024, to 28. 
      Market access has increased to 78% of covered lives. 

Pipeline and Innovation Highlights

   -- Filing of the MIPLYFFA MAA with the European Medicines Agency remains on 
      track for the second half of 2025 as reinforced by recent conversations 
      with European authorities. 
   -- Enrolled 5 additional patients in the Phase 3 DiSCOVER trial of 
      celiprolol for the treatment of Vascular Ehlers-Danlos Syndrome, bringing 
      the total number of enrolled patients to 32. 
   -- The company out-licensed IP related to a pre-clinical prodrug that had 
      been deprioritized. Zevra is entitled to receive a nominal upfront cash 
      payment, potential future regulatory milestones, and mid-single-digit 
      royalties on net sales. 

Corporate Highlights and Upcoming Milestones

   -- On April 7, 2025, Zevra announced closing of the sale of its Pediatric 
      Rare Disease Priority Review Voucher ("PRV") to an undisclosed buyer for 
      gross proceeds of $150 million. 

Q1 2025 Financial Highlights

   -- Revenue, Net: $20.4 million for Q1 2025, which includes $17.1 million of 
      MIPLYFFA net revenue, $0.1 million of OLPRUVA net revenue, $2.3 million 
      in net reimbursements from the French EAP for arimoclomol, and $0.9 
      million in royalties and other reimbursements under the AZSTARYS(R) 
      license agreement. This was an increase in total net revenue of $17.0 
      million compared to $3.4 million in Q1 2024. 
   -- Cost of Goods Sold: $3.0 million for Q1 2025, which includes $1.6 million 
      of non-cash intangible asset amortization. 
   -- Operating Expenses: $22.8 million for Q1 2025, which includes non-cash 
      stock compensation expense of $3.1 million. 
 
          -- R&D expense was $3.3 million for Q1 2025, which was a decrease of 
             $9.0 million compared to Q1 2024 due primarily to a decrease in 
             third-party costs upon completion of the KP1077 Phase 2 trial, 
             combined with a decrease in personnel-related costs. 
 
          -- SG&A expense was $19.5 million for Q1 2025, which was an increase 
             of $9.6 million compared to Q1 2024. Period-over-period increase 
             was primarily related to an increase in personnel-related costs, 
             professional fees, and other expenses associated with our 
             commercial, medical and launch activities. 
   -- Net Loss: ($3.1) million, or ($0.06) per basic and diluted share for Q1 
      2025, compared to a net loss of ($16.6) million, or ($0.40) per basic and 
      diluted share in Q1 2024. 
   -- Cash Position: Cash, cash equivalents and investments were $68.7 million 
      as of Mar. 31, 2025. Combined with the net proceeds of $148.3 million 
      from the sale of the PRV received just after the end of the quarter on 
      Apr. 1, 2025, cash, cash equivalents and investments would be $217.0 
      million. Based on the Company's current operating forecast and adding the 
      non-dilutive capital from the PRV sale, we have ample resources and 
      financial flexibility to execute on our strategic priorities independent 
      from the capital markets. 
   -- Common and Fully Diluted Shares O/S: As of Mar. 31, 2025, total shares of 
      common stock outstanding were 54,679,363, and fully diluted common shares 
      were 67,897,631, which included 7,734,741 issuable from outstanding 
      awards under equity incentive plans, and 5,483,527 shares issuable upon 
      exercise of warrants. 

Upcoming Events

   -- Zevra will participate in a fireside chat at the H.C. Wainwright 3rd 
      Annual Bioconnect Investor Conference on Tuesday, May 20, 2025, at 12:30 
      p.m. ET. Management will be available for one-on-one meetings with 
      registered attendees. 

Conference Call Information

Zevra will host a conference call and audio webcast today at 4:30 p.m. ET to discuss its corporate update and financial results for the first quarter 2025.

The audio webcast will be accessible via the Investor Relations section of the Company's website, http://investors.zevra.com/. An archive of the audio webcast will be available for ninety (90) days beginning at approximately 5:30 p.m. ET on May 13, 2025.

Additionally, interested participants and investors may access the conference call by dialing either:

   -- (800) 245-3047 (United States) 
   -- +1 (203) 518-9765 (International) 
   -- Conference ID: ZVRAQ125 

About MIPLYFFA(R) (arimoclomol)

MIPLYFFA (arimoclomol) is Zevra's approved therapy for the treatment of Niemann-Pick disease type C (NPC). Approved by the U.S. Food and Drug Administration on Sep. 20, 2024, MIPLYFFA (arimoclomol) increases the activation of the transcription factors EB (TFEB) and E3 (TFE3) resulting in the upregulation of coordinated lysosomal expression and regulation (CLEAR) genes. MIPLYFFA has also been shown to reduce unesterified cholesterol in the lysosomes of human NPC fibroblasts. The clinical significance of these findings is not fully understood. In the pivotal phase 3 trial, MIPLYFFA halted disease progression compared to placebo over the one-year duration of the trial when measured by the only validated disease progression measurement tool, the NPC Clinical Severity Scale. MIPLYFFA has also received Orphan Medicinal Product designation by the European Medicines Agency (EMA) for the treatment of NPC.

INDICATIONS AND USAGE

MIPLYFFA is indicated for use in combination with miglustat for the treatment of neurological manifestations of Niemann-Pick disease type C (NPC) in adult and pediatric patients 2 years of age and older.

IMPORTANT SAFETY INFORMATION

Hypersensitivity Reactions:

Hypersensitivity reactions such as urticaria and angioedema have been reported in patients treated with MIPLYFFA during Trial 1: two patients reported both urticaria and angioedema (6%) and one patient (3%) experienced urticaria alone within the first two months of treatment. Discontinue MIPLYFFA in patients who develop severe hypersensitivity reactions. If a mild or moderate hypersensitivity reaction occurs, stop MIPLYFFA and treat promptly. Monitor the patient until signs and symptoms resolve.

Embryofetal Toxicity:

MIPLYFFA may cause embryofetal harm when administered during pregnancy based on findings from animal reproduction studies. Advise pregnant females of the potential risk to the fetus and consider pregnancy planning and prevention for females of reproductive potential.

Increased Creatinine without Affecting Glomerular Function:

Across clinical trials of MIPLYFFA, mean increases in serum creatinine of 10% to 20% compared to baseline were reported. These increases occurred mostly in the first month of MIPLYFFA treatment and were not associated with changes in glomerular function.

During MIPLYFFA treatment, use alternative measures that are not based on creatinine to assess renal function. Increases in creatinine reversed upon MIPLYFFA discontinuation.

The most common adverse reactions in Trial 1 (>=15%) in MIPLYFFA-treated patients who also received miglustat were upper respiratory tract infection, diarrhea, and decreased weight.

Three (6%) of the MIPLYFFA-treated patients had the following adverse reactions that led to withdrawal from Trial 1: increased serum creatinine (one patient), and progressive urticaria and angioedema (two patients). Serious adverse reactions reported in MIPLYFFA-treated patients were hypersensitivity reactions including urticaria and angioedema.

To report SUSPECTED ADVERSE REACTIONS, contact Zevra Therapeutics, Inc. at toll-free phone 1-844-600-2237 or FDA at 1 800-FDA-1088 or www.fda.gov/medwatch.

Drug Interaction(s):

Arimoclomol is an inhibitor of the organic cationic transporter 2 (OCT2) transporter and may increase the exposure of drugs that are OCT2 substrates. When MIPLYFFA is used concomitantly with OCT2 substrates, monitor for adverse reactions and reduce the dosage of the OCT2 substrate.

Use in Females and Males of Reproductive Potential:

(MORE TO FOLLOW) Dow Jones Newswires

May 13, 2025 16:05 ET (20:05 GMT)