Zevra Therapeutics Inc Stock Price, News & Analysis

Zevra Therapeutics (NASDAQ:ZVRA) has submitted a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for arimoclomol, targeting the treatment of Niemann-Pick Disease Type C (NPC). The drug, marketed as MIPLYFFA® in the U.S., is the only treatment directly addressing NPC's underlying pathology by enhancing lipid clearance through increased gene expression.

The submission is supported by extensive clinical data from over 270 NPC patients, including Phase 2/3 trials, Open-Label Extension study, Expanded Access Programs, and a pediatric sub-study, representing 5-7 years of patient experience. Currently, 89 European patients are enrolled in the company's Expanded Access Program as of Q2.

Zevra Therapeutics (NASDAQ:ZVRA) announced the presentation of four posters at the 42nd Annual Meeting of the Southeastern Regional Genetics Group (SERGG) in Asheville, North Carolina. Three posters focus on MIPLYFFA® (arimoclomol) for Niemann-Pick disease type C (NPC) and one on OLPRUVA® (sodium phenylbutyrate) for urea cycle disorders.

The MIPLYFFA presentations highlight long-term efficacy data showing disease stabilization for up to 5 years in treated patients, real-world outcomes from the U.S. Expanded Access Program, and insights into the drug's mechanism of action. The OLPRUVA presentation demonstrates successful administration through gastrostomy tubes, expanding treatment options for UCD patients.

Zevra Therapeutics (NASDAQ:ZVRA) announced the publication of long-term efficacy data for MIPLYFFA® (arimoclomol) in treating Niemann-Pick disease type C (NPC) in the Molecular Genetics and Metabolism journal.

The open-label extension study demonstrated that MIPLYFFA maintains its ability to reduce disease progression for at least 5 years in NPC patients. The data encompasses safety and efficacy results from over 270 patients worldwide, with some patients receiving treatment for up to seven years. The results align with the previous Phase 2/3 trial showing MIPLYFFA's effectiveness in halting disease progression compared to placebo.

Zevra Therapeutics (NasdaqGS: ZVRA) announced that its FDA-approved treatment MIPLYFFA® (arimoclomol) will be featured in three presentations at the National Niemann Pick Disease Foundation Conference in July 2025. MIPLYFFA is the first FDA-approved treatment for Niemann-Pick disease type C (NPC), indicated for use with miglustat in patients 2 years and older.

The presentations include clinical data showing that MIPLYFFA, combined with miglustat, halted disease progression at 12 months in pivotal trials. Long-term data from a 48-month open-label extension confirmed the treatment's effectiveness and safety profile. Additional research demonstrated MIPLYFFA's mechanism of action through upregulation of CLEAR genes and improvement of lysosomal function.

Zevra Therapeutics (NASDAQ: ZVRA) announced the final results of its 2025 Annual Meeting of Stockholders, where shareholders overwhelmingly re-elected Wendy L. Dixon, Ph.D. and Tamara A. Favorito as Class I Directors to serve until 2028. The incumbent directors received approximately 74% of the votes, defeating potential opposition nominees Travis C. Mickle and Arthur C. Regan, who would have received only 26.45% and 23.97% respectively. The opposition nominations were ultimately disregarded due to failure to meet SEC and bylaw requirements. Stockholders also approved Ernst & Young LLP as the company's independent auditors for FY2025.

Zevra Therapeutics (ZVRA) reported strong Q1 2025 financial results with net revenue of $20.4 million, primarily driven by $17.1 million from MIPLYFFA sales. The company completed the sale of its Pediatric Rare Disease Priority Review Voucher for $150 million, significantly strengthening its balance sheet to $217 million in cash and equivalents.

Commercial progress includes 13 new MIPLYFFA prescriptions in Q1 (total 122) with 38% market access coverage, and 5 new OLPRUVA patients (total 28) with 78% market access. The company reported a reduced net loss of $3.1 million ($0.06 per share) compared to $16.6 million in Q1 2024. Operating expenses were $22.8 million, with R&D expenses decreasing by $9.0 million while SG&A increased by $9.6 million due to commercial activities.

Zevra Therapeutics (NASDAQ: ZVRA), a commercial-stage company focused on rare disease therapies, has scheduled its Q1 2025 financial results announcement for Tuesday, May 13, 2025. The company will release its corporate and financial results after market close, followed by a conference call and audio webcast at 4:30 p.m. ET.

Investors can access the webcast through Zevra's website investor relations section. For telephone participation, U.S. callers can dial (800) 245-3047, while international participants should use +1 (203) 518-9765, with Conference ID: ZVRAQ125. A replay will be available on the company's website for 90 days following the presentation.

Zevra Therapeutics (ZVRA), a commercial-stage rare disease therapeutics company, has announced its participation in the upcoming Citizens Life Science Conference in New York. The company's executive leadership team will engage in a fireside chat on Wednesday, May 7, 2025, at 11:30 a.m. ET. Management will also be available for one-on-one meetings with registered conference attendees. Investors can access the live webcast through the "Events & Presentations" section on Zevra's investor relations website at investors.zevra.com.

Zevra Therapeutics (ZVRA) has filed its definitive proxy statement for the 2025 Annual Meeting scheduled for May 29, urging stockholders to vote for two incumbent directors - Wendy L. Dixon and Tamara A. Favorito. The company highlights a 54.8% total stockholder return under its refreshed board and management team.

The Board strongly opposes attempts by Daniel J. Mangless, a 2.8% stockholder, to replace two directors. Mangless, who already has three nominees on the board from 2023, seeks to add two more nominees - Travis C. Mickle and Arthur C. Regan. The company argues this would give Mangless majority board control without presenting any clear strategic plans.

Under current leadership, Zevra has launched two rare disease therapies: MIPLYFFA® for Niemann-Pick disease type C, which earned a Priority Review Voucher sold for $150 million, and OLPRUVA® for urea cycle disorders. The company is executing a five-year strategic plan focused on commercial excellence, pipeline innovation, talent development, and corporate foundation strengthening.