Welcome to our dedicated page for Zevra Therapeutics news (Ticker: ZVRA), a resource for investors and traders seeking the latest updates and insights on Zevra Therapeutics stock.
Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) is a commercial-stage rare disease company whose news flow centers on its therapies for serious, low-prevalence conditions and the corporate developments that support these programs. The company regularly issues updates describing progress with its approved products, clinical pipeline, regulatory interactions, and financial performance.
A major theme in Zevra’s news is the commercialization and clinical evidence for MIPLYFFA (arimoclomol), its approved therapy for Niemann-Pick disease type C (NPC). Press releases highlight pivotal and long-term data, presentations at scientific and patient-focused meetings, and steps to broaden access, such as a distribution agreement with Uniphar to enable reimbursed named patient supply for NPC patients in select territories outside Europe. These items provide insight into how Zevra is working to expand the reach of its NPC treatment.
News coverage also includes updates on OLPRUVA (sodium phenylbutyrate), Zevra’s approved treatment for certain urea cycle disorders, and on celiprolol, its investigational candidate for vascular Ehlers-Danlos syndrome. The company reports on clinical trial enrollment milestones, publication of new analyses, and recognition of its scientific work at conferences, giving investors and clinicians visibility into the evolution of its rare disease portfolio.
In addition, Zevra issues quarterly financial results and corporate updates, detailing revenue contributions from its commercial products, operating expenses, and capital resources. Governance and organizational news, such as board appointments, executive transitions, and equity inducement grants under its employment inducement award plan, are disclosed through press releases and Form 8-K filings. For anyone tracking ZVRA, this news stream offers a consolidated view of the company’s therapeutic progress, regulatory status, and corporate trajectory in the rare disease space.
Zevra Therapeutics (NASDAQ: ZVRA) announced the presentation of data validating the swallow domain's significance in the Niemann-Pick Disease Type C Clinical Severity Scale (NPCCSS) at the 53rd Child Neurology Society Annual Meeting. The study, presented by Dr. Elizabeth Berry-Kravis, involved 12 NPC and swallow experts evaluating scoring categories and instructions.
The findings confirmed that NPCCSS swallow response categories can be consistently interpreted, with score changes reflecting actual changes in patient swallow function. The revised scoring algorithm improves linearity by re-ranking dysphagia categories and creating a separate category for tube-feeding. The R4DNPCCSS endpoint, which includes this revised swallow domain along with ambulation, fine motor skills, and speech domains, showed a favorable treatment difference of -1.51 (P=0.0413) for arimoclomol compared to placebo in the Phase 2/3 study.
Zevra Therapeutics (NASDAQ: ZVRA) has announced its participation in the upcoming Guggenheim Securities Healthcare Innovation Conference. The company's President and CEO, Neil F. McFarlane, will engage in a fireside chat on Wednesday, November 13, 2024, at 4 p.m. ET. The event will be held virtually, and management will be available for one-on-one meetings with registered attendees. Interested parties can access the live webcast through the 'Events & Presentations' section on Zevra's investor relations website at investors.zevra.com.
Zevra Therapeutics (ZVRA) announced it will release its third quarter 2024 financial results and provide a corporate update on November 12, 2024, after market close. The company will host a conference call at 4:30 p.m. ET the same day. The update will include progress on the commercial launches of MIPLYFFA™ and OLPRUVA®, along with updates on clinical stage programs. A webcast replay will be available for 90 days on the company's investor relations website.
Zevra Therapeutics (NASDAQ: ZVRA), a rare disease therapeutics company, has announced its participation in a Rare Disease Panel at the 2024 Maxim Healthcare Virtual Summit. The event, presented by Maxim Group , is scheduled for Thursday, October 17, 2024, at 2 p.m. ET. Neil F. McFarlane, President and CEO of Zevra, will represent the company at the panel.
The conference will be streamed live on M-Vest, with investors able to register and watch the discussion through the M-Vest page. For those unable to attend live, an archived version of the presentation will be made available in the 'Events & Presentations' section of Zevra's Investor Relations website at investors.zevra.com.
The National Urea Cycle Disorders Foundation (NUCDF) and Zevra Therapeutics (NasdaqGS: ZVRA) have launched the Check Ammonia Campaign in October to improve the diagnosis of urea cycle disorders (UCDs). The campaign aims to highlight the critical importance of prompt and accurate blood ammonia testing, which is essential for detecting elevated ammonia levels (hyperammonemia), a classic sign of UCDs.
The campaign website, checkammonia.com, offers resources on recognizing and testing for hyperammonemia. Quick diagnosis is important as toxic ammonia levels can rise rapidly in affected individuals, potentially leading to coma and death if left untreated. Newborns with severe UCDs typically become critically ill within 36-48 hours after birth, and it's estimated that up to 20% of sudden infant death syndrome (SIDS) cases may be due to undiagnosed inborn errors of metabolism like UCDs.
Zevra Therapeutics (NASDAQ: ZVRA) announced that top-line data from its Phase 2 clinical trial of KP1077 for idiopathic hypersomnia (IH) will be presented at Sleep Europe 2024. The trial (NCT05668754) was a placebo-controlled, double-blind, randomized withdrawal study evaluating the safety and tolerability of KP1077 (serdexmethylphenidate, or SDX) in IH patients. Dr. Rene Braeckman, Zevra's Senior VP of Clinical Development, will present the findings, which demonstrate clinically meaningful benefits for key IH symptoms. The data will provide important information for designing a Phase 3 study. Presentations will include an oral session on September 27 and a poster session on September 26 at the 27th Congress of the European Sleep Research Society in Seville, Spain.
Zevra Therapeutics announced FDA approval of MIPLYFFA™ (arimoclomol) as the first treatment for Niemann-Pick disease type C (NPC), an ultra-rare neurodegenerative disease. MIPLYFFA is indicated for use with miglustat to treat neurological manifestations in patients 2 years and older. The approval is based on a 12-month trial showing MIPLYFFA halted disease progression. Zevra received a rare pediatric disease priority review voucher and launched AmplifyAssist™, a patient support program.
MIPLYFFA will be commercially available in 8-12 weeks. The drug is administered orally three times daily, with dosage ranging from 47-124 mg based on body weight. Zevra estimates 900 people in the U.S. have NPC, with about one-third diagnosed. The approval marks a significant milestone for NPC patients, offering the first FDA-approved treatment option for this progressive and fatal disease.
Zevra Therapeutics (NASDAQ: ZVRA) presented new data for arimoclomol and OLPRUVA at the SSIEM 2024 Annual Symposium. Four posters focused on arimoclomol's efficacy and safety for treating Niemann-Pick disease type C (NPC). Key findings include:
1. Statistically significant treatment effect using the 4DNPCCSS scale
2. Long-term safety and efficacy in a 48-month open-label extension trial
3. Stable disease progression in real-world settings
4. Consistent safety profile in expanded access program
One poster on OLPRUVA showed increased drug exposure under fasting conditions, potentially allowing for lower effective dosages when taken without food.
Zevra Therapeutics (NASDAQ: ZVRA), a rare disease therapeutics company, has announced its participation in two upcoming investor conferences in September 2024. Neil F. McFarlane, President and CEO of Zevra, will present at:
1. The H.C. Wainwright 26th Annual Global Investment Conference in New York on September 9, 2024, at 2:00 p.m. ET.
2. The Cantor Annual Healthcare Conference in New York on September 17, 2024, at 2:30 p.m. ET.
Management will also be available for one-on-one meetings with registered attendees at both conferences. Live webcasts of the presentations will be accessible through the 'Events & Presentations' section of Zevra's investor website at investors.zevra.com.
Zevra Therapeutics (NASDAQ: ZVRA) reported Q2 2024 financial results and corporate highlights. Key points include:
1. FDA advisory committee voted favorably on arimoclomol's effectiveness for NPC treatment; PDUFA date set for September 21, 2024.
2. Pro forma cash position of $113.8 million as of June 30, 2024, including recent public offering.
3. Q2 2024 net revenue of $4.4 million, down from $8.5 million in Q2 2023.
4. Net loss of $19.9 million ($0.48 per share) in Q2 2024, compared to $2.6 million ($0.08 per share) in Q2 2023.
5. Cash runway extended into Q1 2027, excluding potential arimoclomol revenue.
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