文章核心观点 - 公司是一家专注于罕见疾病治疗的公司 [3] - 公司正在开发创新性的治疗方法来解决罕见疾病领域的挑战 [3] - 公司将在Canaccord Genuity's 44th Annual Growth Conference上进行演讲 [1][2] 公司概况 - 公司名称为Zevra Therapeutics Inc [1] - 公司股票代码为NASDAQ:ZVRA [1] - 公司总裁兼首席执行官为Neil F. McFarlane [1] - 公司将提供一对一的投资者会议 [1] - 公司提供扩展访问计划以满足患者需求 [4] 公司发展战略 - 公司采用独特的数据驱动的开发和商业化策略 [3] - 公司正在克服复杂的药物开发挑战,为罕见疾病患者提供新的治疗方法 [3]

文章核心观点 - 公司是一家专注于罕见疾病治疗的公司 [3] - 公司正在开发针对罕见疾病的创新疗法 [3] - 公司将在Canaccord Genuity's 44th Annual Growth Conference上进行演讲 [1][2] 公司概况 - 公司名称为Zevra Therapeutics Inc [1] - 公司股票代码为ZVRA [1] - 公司总裁兼首席执行官为Neil F. McFarlane [1] - 公司提供扩展访问计划以帮助罕见疾病患者获得治疗 [4] 会议信息 - 公司将于2024年8月14日在波士顿参加Canaccord Genuity's 44th Annual Growth Conference [1][2] - 公司高管将在会议期间接受一对一投资者会议 [1] - 公司将通过其网站investors.zevra.com提供现场网络直播 [2]

文章核心观点 - 预计Zevra Therapeutics(ZVRA)在2024年6月结束的季度将出现同比收益下降,收入也将下降 [1] - 公司的实际业绩如何与预期相比将是影响其近期股价的重要因素 [2][3] 公司业绩分析 - 预计Zevra Therapeutics将在下一季度报告中公布每股亏损0.47美元,同比下降213.3% [4] - 预计收入为419万美元,同比下降50.5% [4] - 分析师对该季度的盈利预测在过去30天内下调了2.29% [5][6] 业绩预测 - 分析师对公司业绩的预测存在偏差,这可能会影响实际业绩 [7][8][9] - 公司的每股收益预测值低于共识预测值,预示分析师对公司业绩前景持悲观态度,这导致公司的收益意外预测值为负6.38% [12] - 公司目前的股票评级为4级,这使得预测其能否战胜共识预测值变得困难 [12][13] 历史业绩表现 - 公司在上一季度实际每股亏损0.40美元,超出预期0.49美元,实现了18.37%的正面业绩惊喜 [15] - 过去4个季度,公司2次战胜了共识每股收益预测 [15] 行业同行表现 - 同行Crinetics Pharmaceuticals, Inc.(CRNX)预计将在2024年6月季度报告每股亏损0.86美元,同比增长8.5%,收入预计为137万美元,同比增长38.4% [19][20] - Crinetics Pharmaceuticals的每股收益预测值低于共识预测值,加上其目前的股票评级为4级,这使得其能否战胜共识预测值变得困难 [20][21]

CELEBRATION, Fla., Aug. 02, 2024 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company), a rare disease therapeutics company, today announced that the U.S. Food and Drug Administration (FDA) Genetic Metabolic Diseases Advisory Committee (GeMDAC) voted favorably (11 yes, 5 no) that the data support that arimoclomol is effective in the treatment of patients with Niemann-Pick disease type C (NPC). "We are extremely pleased with the committee's recognition of the benefits of arimo ...

文章核心观点 - 美国食品药品监督管理局(FDA)Genetic Metabolic Diseases Advisory Committee(GeMDAC)投票支持arimoclomol治疗尼曼-皮克病C型(NPC)的有效性[1][2][3] - 公司对arimoclomol获批持乐观态度,认为临床数据支持其治疗NPC的疗效[2][3] - arimoclomol已获FDA授予孤儿药、快速通道、突破性疗法和罕见儿童疾病指定,欧洲药品管理局也授予其治疗NPC的孤儿药品地位[5] NPC疾病概述 - NPC是一种罕见、进行性和神经退行性的溶酶体贮积障碍性疾病,由NPC1或NPC2基因突变导致[4] - 患者会因身体和认知功能受限而失去独立生活能力,出现言语、认知、吞咽、行走和精细运动技能障碍[4] - 疾病进程不可逆转,可在几个月内致命,也可能需要多年才被诊断并逐步恶化[4] 公司概况 - 公司是一家专注于罕见疾病的生物制药公司,致力于利用科学、数据和患者需求开发创新疗法[6] - 公司采取独特的数据驱动开发和商业化策略,克服复杂的药物开发挑战,为罕见疾病患者带来新的治疗方案[6]

CELEBRATION, Fla., July 30, 2024 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company) a rare disease therapeutics company, today announced that it will host a conference call and audio webcast on Tuesday, August 13, 2024, at 4:30 p.m. ET, to review its corporate and financial results for the second quarter of 2024. The link to the audio webcast will be accessible via the Investor Relations section of the Company's website, https://investors.zevra.com/. To join the meeting by ...

CELEBRATION, Fla., July 30, 2024 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company) a rare disease therapeutics company, today announced that it will host a conference call and audio webcast on Tuesday, August 13, 2024, at 4:30 p.m. ET, to review its corporate and financial results for the second quarter of 2024. The link to the audio webcast will be accessible via the Investor Relations section of the Company's website, https://investors.zevra.com/. To join the meeting by ...

Zevra Therapeutics, Inc. (ZVRA) announced that the FDA has indicated that it will convene a meeting with the recently formed Genetic Metabolic Diseases Advisory Committee (“GeMDAC”) to review the company’s new drug application (NDA) for pipeline candidate arimoclomol.The NDA is seeking approval for arimoclomol, an orally delivered, first-in-class treatment for Niemann-Pick disease type C (“NPC”).NPC is an ultra-rare, progressive, and neurodegenerative lysosomal storage disorder characterized by an inability ...

Zevra Therapeutics (ZVRA) shares rallied 21.9% in the last trading session to close at $5.48. This move can be attributable to notable volume with a higher number of shares being traded than in a typical session. This compares to the stock's 3.2% loss over the past four weeks.The stock rallied after the FDA indicated that it would convene a meeting with the recently formed Genetic Metabolic Diseases Advisory Committee to review the company’s new drug application (NDA) for pipeline candidate arimoclomol. The ...

UNITED STATES SECURITIES AND EXCHANGE COMMISSION WASHINGTON, D.C. 20549 FORM 10-Q (Mark One) ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended March 31, 2024 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the Transition Period From to Commission File Number: 001-36913 Zevra Therapeutics, Inc. (Exact Name of Registrant as Specified in Its Charter) Delaware 20-5894398 (State or Other Juris ...