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Zevra Therapeutics Inc (ZVRA) is a biopharmaceutical innovator focused on rare diseases, combining advanced data analytics with targeted clinical development. This page provides investors and healthcare stakeholders with essential updates on Zevra's therapeutic advancements, regulatory milestones, and strategic collaborations.
Access timely press releases and curated news covering clinical trial progress, FDA communications, and partnership announcements. Our repository ensures efficient tracking of Zevra's patient-centric developments in complex therapeutic areas, including late-stage trial outcomes and commercialization strategies.
Key updates on: clinical program advancements, regulatory submissions, research partnerships, and therapy commercialization efforts. Bookmark this page for streamlined access to Zevra's evolving role in addressing unmet rare disease needs through science-driven solutions.
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.
Zevra Therapeutics (ZVRA) has published new research in Molecular Genetics and Metabolism detailing the mechanism of action of MIPLYFFA® in treating Niemann-Pick disease type C (NPC). The study reveals that arimoclomol enters cells and increases the movement of translation factors EB and E3 (TFEB & TFE3) from cytosol to nucleus.
This process triggers the upregulation of CLEAR genes, including NPC1, which is important for lysosomal function. The increased CLEAR gene expression leads to higher NPC1 protein levels in lysosomes, resulting in improved cholesterol trafficking. Animal studies demonstrated that this improved trafficking correlates with better neurological behaviors, specifically in rearing and gait.
Zevra Therapeutics (ZVRA) has completed the sale of its Rare Pediatric Disease Priority Review Voucher (PRV) for $150 million in gross proceeds. The PRV was granted by the FDA in September 2024 following the approval of MIPLYFFA™ (arimoclomol), the first U.S. approved treatment for Niemann-Pick type C, an ultra-rare neurodegenerative disease.
The company reported unaudited cash, cash equivalents and investments of $68.7 million as of March 31, 2025. After the PRV sale's net proceeds of $148.3 million, the total cash position would be $217.0 million. The non-dilutive capital will support strategic priorities including the commercial launches of MIPLYFFA and OLPRUVA®, ongoing Phase 3 trial for celiprolol, and future strategic investments.
Zevra Therapeutics (NASDAQ: ZVRA) has filed its preliminary proxy statement for the 2025 Annual Meeting, nominating two directors for re-election: Wendy L. Dixon and Tamara A. Favorito. The company faces a proxy contest from stockholder Daniel J. Mangless, who proposes two alternative candidates.
The current board comprises eight directors, with seven being independent and seven joining since 2023. The company reports significant progress, including:
- Launch of first two products
- Record net product revenue in Q4 2024
- Cash runway extending into 2029
- Recent sale of Priority Review Voucher for $150 million
The board recommends stockholders reject Mangless's nominees, including former CEO Travis C. Mickle, arguing that changes could disrupt the company's strategic execution. Stockholders are advised to wait for the definitive proxy materials with the WHITE proxy card before taking any action.
Zevra Therapeutics (ZVRA) reported FY 2024 financial results with net revenue of $23.6 million, including $12.0 million in Q4 2024. The company posted a net loss of $105.5 million ($2.28 per share) for 2024, compared to $46.0 million loss in 2023.
Key highlights include the successful commercial launch of MIPLYFFA following FDA approval in September 2024, with 109 prescription enrollment forms received by year-end. The company entered into an agreement to sell its Rare Pediatric Disease Priority Review Voucher (PRV) for $150 million.
Cash position stood at $75.5 million as of December 31, 2024. With current operating forecast and excluding PRV sale proceeds, Zevra projects cash runway into 2029. The Phase 3 DiSCOVER trial for celiprolol enrolled 27 total patients, and the company plans to file for EU marketing authorization for MIPLYFFA in H2 2025.
Zevra Therapeutics (ZVRA) has launched a new disease state awareness campaign called 'Learn NPC, Read Between the Signs' on Rare Disease Day, February 28, 2025. The initiative aims to improve early recognition and diagnosis of Niemann-Pick disease type C (NPC), an ultra-rare, progressive, and neurodegenerative lysosomal storage disorder.
The campaign, launched through www.LearnNPC.com, provides educational resources and testing information for healthcare professionals to better identify NPC, which is often challenging to diagnose due to its varying age of onset and diverse symptom presentation.
The National Niemann-Pick Disease Foundation (NNPDF) has expressed support for the campaign, highlighting its importance in increasing disease awareness among healthcare professionals. The initiative includes resources such as disease summaries, diagnostic challenges, symptom overviews, and testing options for suspected NPC cases.
Zevra Therapeutics (ZVRA) has entered into an agreement to sell its Rare Pediatric Disease Priority Review Voucher (PRV) for $150 million in gross proceeds. The transaction is expected to close within 30-45 days, subject to customary conditions.
The PRV was awarded to Zevra in September 2024 following FDA approval of MIPLYFFA™ (arimoclomol), a treatment for neurological manifestations of Niemann-Pick disease type C in patients 2 years and older. The non-dilutive capital will support the commercial launches of MIPLYFFA™ and OLPRUVA®, along with advancing the company's rare disease pipeline.
Zevra Therapeutics (ZVRA), a commercial-stage company specializing in rare disease therapies, has scheduled its fourth quarter and full year 2024 financial results announcement for Tuesday, March 11, 2025. The company will release its 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 investor relations website, with dial-in options available for both U.S. (800-245-3047) and international (+1-203-518-9765) participants using Conference ID: ZVRAQ424. A replay of the webcast will be available for 90 days following the presentation.
Zevra Therapeutics (NASDAQ: ZVRA), a commercial-stage rare disease therapy company, has announced its participation in two major investor conferences in March 2025. The company's executive leadership team will present at the TD Cowen 45th Annual Health Care Conference on March 5, 2025, at 10:30 a.m. ET, and the 37th Annual ROTH Conference on March 17, 2025, at 1:00 p.m. PT.
Management will be available for one-on-one meetings with registered attendees at both conferences. Interested parties can access live webcasts of the presentations through the 'Events & Presentations' section on Zevra's Investor Relations website at investors.zevra.com.
Zevra Therapeutics (ZVRA) announced its participation in the 21st Annual WORLDSymposium™, where it will receive a 2025 New Treatment Award for MIPLYFFA™. Eight abstracts related to MIPLYFFA (arimoclomol) have been accepted for poster presentation, with one selected for oral presentation by Dr. Eugen Mengel.
MIPLYFFA is approved for treating neurological manifestations of Niemann-Pick disease type C (NPC) in patients 2 years and older, used in combination with miglustat. The featured presentation will discuss efficacy results from a 12-month double-blind randomized trial using the re-scored 4-domain NPC Clinical Severity Score.
The poster presentations will cover various aspects including gene expression, real-world treatment data, regulatory journey, safety profiles, and long-term efficacy. E-posters will be available to registered attendees from February 4, 2025, through the WORLDSymposium mobile app, with on-demand access extending to March 14, 2025. The symposium takes place February 3-7, 2025, in San Diego, CA.
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