Core Insights - Zevra Therapeutics, Inc. announced presentations on MIPLYFFA (arimoclomol) at the NNPDF Conference, highlighting its role as the first FDA-approved treatment for Niemann-Pick disease type C (NPC) [1][2] - MIPLYFFA is indicated for use in combination with miglustat for treating neurological manifestations of NPC in patients aged 2 years and older [7][6] Presentation and Research Findings - Dr. Barbara K. Burton presented an overview of MIPLYFFA, emphasizing its unique mechanism that improves lysosomal function and halts disease progression at 12 months in a pivotal study [2][3] - The pivotal trial demonstrated that arimoclomol combined with miglustat halted disease progression compared to placebo, confirmed by a 48-month open-label extension study [4][6] - Two posters were presented: one detailing long-term effectiveness and safety of arimoclomol, and another providing mechanistic evidence of arimoclomol's action through the CLEAR network [4][5] Drug Mechanism and Clinical Significance - MIPLYFFA enhances the activation of transcription factors TFEB and TFE3, leading to the upregulation of CLEAR genes and improved lysosomal function [6][17] - The drug has shown to reduce unesterified cholesterol in NPC fibroblasts, although the clinical significance of this finding remains to be fully understood [6] Company Overview - Zevra Therapeutics focuses on developing therapies for rare diseases with limited treatment options, aiming to create transformational therapies through data-driven strategies [18]

Company Overview - Zevra Therapeutics (ZVRA) shares increased by 5.1% to close at $9.41, with trading volume significantly higher than usual [1] - The stock has shown a 0.9% gain over the past four weeks [1] Product Performance - The rise in stock price is linked to positive investor sentiment regarding the initial uptake of Miplyffa (arimoclomol), which received FDA approval for Niemann-Pick disease type C in September 2024 [2] - Miplyffa generated sales of $17.1 million in Q1 2025 [2] Earnings Expectations - Zevra Therapeutics is projected to report quarterly earnings of $0.65 per share, reflecting a year-over-year increase of 235.4% [3] - Expected revenues for the upcoming quarter are $71.83 million, representing a 1514.2% increase from the same quarter last year [3] - The consensus EPS estimate has remained unchanged over the last 30 days, indicating a potential lack of momentum in earnings estimate revisions [4] Industry Context - Zevra Therapeutics is categorized under the Zacks Medical - Drugs industry, which includes other companies like Xeris Biopharma (XERS) [5] - Xeris Biopharma's stock closed 1.5% higher at $4.73, with a slight return of 0.2% over the past month [5] - Xeris Biopharma's consensus EPS estimate has also remained unchanged, with a year-over-year change of 70% [6]

June 2025 NasdaqGS: ZVRA 1 A Rare Approach to Therapeutics Cautionary Note Regarding Forward-Looking Statements Presentation may contain forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements relate to future events or our future financial performance. We generally identify forward-looking statements by terminology such as "may," "will," "would," "should," "expects," "plans," "anticipates," "could," "intends," "target," "projects," " ...

ISS, Glass Lewis, and Egan-Jones Unanimously Conclude Mangless Has Not Made a Compelling Case for Change Board and Management Urge Stockholders to Follow Proxy Advisors' Recommendation and Vote "FOR" Wendy L. Dixon, Ph.D. and Tamara A. Favorito on the WHITE Proxy Card and "WITHHOLD" for Mangless Nominees CELEBRATION, Fla., May 21, 2025 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) ("Zevra," or the "Company"), a commercial-stage company focused on providing therapies to people living with rar ...

Core Insights - Zevra Therapeutics reported Q1 2025 net revenue of $20.4 million, a significant increase from $3.4 million in Q1 2024, primarily driven by product net revenue of $17.2 million from MIPLYFFA [8][6][30] - The company completed the sale of its Pediatric Rare Disease Priority Review Voucher (PRV) for gross proceeds of $150 million, enhancing its financial position to support commercial launches and development programs [5][2] - Zevra aims to establish MIPLYFFA as a cornerstone treatment for Niemann-Pick disease type C (NPC) and maximize commercial opportunities with OLPRUVA [2][30] Financial Highlights - Total net revenue for Q1 2025 was $20.4 million, including $17.1 million from MIPLYFFA, $0.1 million from OLPRUVA, and $2.3 million in reimbursements from the French Expanded Access Program for arimoclomol [8][6] - Operating expenses for Q1 2025 were $22.8 million, with R&D expenses at $3.3 million, a decrease from the previous year, while SG&A expenses increased to $19.5 million [13][8] - The net loss for Q1 2025 was $3.1 million, or $0.06 per share, compared to a net loss of $16.6 million, or $0.40 per share in Q1 2024 [13][8] Commercial Highlights - MIPLYFFA had 13 new prescription enrollments in Q1 2025, totaling 122 prescriptions, with market access at 38% of covered lives [7] - OLPRUVA received 5 new patient enrollment forms in Q1 2025, bringing the total to 28, with market access increasing to 78% of covered lives [7] - The company plans to file the MIPLYFFA Marketing Authorization Application (MAA) with the European Medicines Agency in the second half of 2025 [7] Pipeline and Innovation Highlights - Zevra enrolled 5 additional patients in the Phase 3 DiSCOVER trial of celiprolol for Vascular Ehlers-Danlos Syndrome, bringing total enrollment to 32 [7] - The company out-licensed intellectual property related to a deprioritized pre-clinical prodrug, which may yield future regulatory milestones and royalties [7] Upcoming Events - Zevra will participate in the H.C. Wainwright 3rd Annual Bioconnect Investor Conference on May 20, 2025, and will host a conference call to discuss Q1 2025 results [9][10]

CELEBRATION, Fla., April 17, 2025 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NASDAQ:ZVRA) (Zevra, or the Company), a commercial-stage company focused on providing therapies for people living with rare disease, today announces the publication of "Mechanistic insights into arimoclomol mediated effects on lysosomal function in Niemann-pick type C disease" in Molecular Genetics and Metabolism. "This elucidation of MIPLYFFA's® highly differentiated mechanism of action marks a critical step in understanding it ...

XOMA Royalty sold the remaining Kinnate pipeline assets for a total of up to $270 million in upfront and milestone payments, plus royalties on commercial sales at rates ranging from low single digits to mid-teens Holders of Kinnate Contingent Value Rights (CVRs) will receive 85% of all related upfront, milestone, and royalty payments paid to XOMA Royalty prior to April 2, 2029 EMERYVILLE, Calif., April 14, 2025 (GLOBE NEWSWIRE) -- XOMA Royalty Corporation (NASDAQ:XOMA), the biotech royalty aggregator, annou ...

Core Viewpoint - Zevra Therapeutics is preparing for its 2025 Annual Meeting of Stockholders, where it has nominated two incumbent directors for re-election while facing a proxy contest initiated by a stockholder proposing alternative candidates. The company emphasizes its progress and strategic focus, urging stockholders to support its current board. Group 1: Company Developments - Zevra Therapeutics filed its preliminary proxy statement with the SEC for the 2025 Annual Meeting, indicating that stockholders do not need to take action at this time [2] - The Board has nominated Wendy L. Dixon, Ph.D., and Tamara A. Favorito for re-election, both of whom bring over 70 years of combined life sciences experience [3][4] - The company has launched its first two products since the last annual meeting and has a cash runway extending into 2029, bolstered by a $150 million sale of a Priority Review Voucher [7] Group 2: Proxy Contest and Board Dynamics - Stockholder Daniel J. Mangless is proposing the election of two candidates, which the Zevra Board believes could disrupt its strategic plan and recommends stockholders reject these efforts [5][8] - The Board asserts that it has made significant progress in transforming the company into a more efficient organization focused on creating value for patients and stockholders [6][9] - Zevra's management has engaged with Mr. Mangless to avoid a costly proxy contest, emphasizing the importance of maintaining board stability for continued execution of its strategic plan [8][9]

Zevra Therapeutics, Inc. (NASDAQ:ZVRA) Q4 2024 Earnings Conference Call March 11, 2025 4:30 PM ET Company Participants Nichol Ochsner - VP, IR and Corporate Communications Neil McFarlane - President and CEO LaDuane Clifton - CFO Joshua Schafer - Chief Commercial Officer and EVP, Business Development Adrian Quartel - Chief Medical Officer Conference Call Participants Kristen Kluska - Cantor Jason Butler - Citizens JMP Sumant Kulkarni - Canaccord Oren Livnat - H.C. Wainwright Samantha Corwin - William Blair E ...