公司财务报告安排 - 公司将于2025年8月11日上午8点（美国东部时间）举行电话会议和网络直播，报告截至2025年6月30日第一季度财务结果并提供业务更新 [1] - 可通过注册链接电话接入会议，演示文稿和网络直播详情将在公司网站投资者板块“活动与演示”中提供，会议结束后可在网站查看存档网络直播 [2] 公司简介 - 公司是一家生物制药公司，旨在加速重要药物开发和商业化，改善患者生活 [3] - 公司管线包括针对FcRn的全人单克隆抗体IMVT - 1402和batoclimab、TYK2和JAK1强效小分子抑制剂brepocitinib、吸入式sGC激活剂mosliciguat等 [3] - 公司通过创建子公司开发和商业化药物及技术，还孵化与生物制药业务互补的发现阶段公司和健康科技初创企业 [3] 公司联系方式 - 投资者联系Keyur Parekh，邮箱keyur.parekh@roivant.com [7] - 媒体联系Stephanie Lee，邮箱stephanie.lee@roivant.com [8]

MINNEAPOLIS, July 28, 2025 (GLOBE NEWSWIRE) -- Celcuity Inc. (Nasdaq: CELC) (the “Company”), a clinical-stage biotechnology company pursuing development of targeted therapies for oncology, today announced proposed underwritten public offerings of $150,000,000 aggregate principal amount of its convertible senior notes due 2031 (the “Convertible Notes” and such offering, the “Convertible Notes Offering”) and $75,000,000 of shares of its common stock (the “Common Stock” and, such offering, the “Common Stock Of ...

MET-097i (fully biased once-monthly GLP-1 RA) VESPER-1 and interim VESPER-3 data release expected in September 2025; Phase 3 initiation on track for late 2025 MET-233i (once-monthly amylin analog) delivered class-leading Phase 1 results including 8.4% five-week weight loss and 19-day half-life; 12-week monotherapy data expected in late 2025 MET-233i + MET-097i 12-week co-administration data expected by year-end 2025 or early 2026 Oral peptide programs on track; four-week data for selected lead (MET-097o or ...

CAMBRIDGE, Mass., July 28, 2025 (GLOBE NEWSWIRE) -- Fulcrum Therapeutics, Inc.® (Fulcrum) (Nasdaq: FULC), a clinical-stage biopharmaceutical company focused on developing small molecules to improve the lives of patients with genetically defined rare diseases, will host a conference call and webcast on Tuesday, July 29, 2025 beginning at 8:00 a.m. ET to present topline results from the 12 mg dose cohort of the Phase 1b PIONEER trial of pociredir in sickle cell disease. Members of Fulcrum management will be j ...

公司评级与投资机会 - HC Wainwright对Ultragenyx Pharmaceuticals Inc RARE启动覆盖 给予买入评级 目标价80美元[1] - 分析师认为当前股价水平代表有吸引力的投资机会 公司股价年内下跌35% 同期XBI指数仅下跌2.7%[1] - 分析师指出这是生物制药行业中最具多样化的罕见病专注公司之一的有趣买入机会[1] 股价压力原因 - 股价主要受压于投资者对UX143治疗成骨不全症的Orbit试验中期结果失望[2] - 7月公布的Orbit研究第二阶段中期分析未达到提前终止研究所需的p<0.01阈值[3] 产品管线进展 - Orbit研究的最终分析预计将在年底完成 与原始计划一致[3] - 分析师预计最终结果将呈阳性 支持明年在美国提交setrusumab的监管申请[4] - 公司现有商业产品组合本身应支持每股35-40美元的公平价值 不包括管线价值[4] 核心产品与监管动态 - 旗舰产品Crysvita系列预计可实现超过30亿美元的年销售峰值[5] - 7月FDA就UX111治疗Sanfilippo综合征A型的BLA发出完全回应函 要求提供CMC相关补充信息[5][6] - 预计公司可在今年晚些时候重新提交BLA 潜在批准时间为2026年中期[7] 市场表现 - RARE股票在发布时上涨0.93% 报27.59美元[7]

公司动态 - Tevogen Bio Holdings Inc 对白宫发布的《赢得竞赛：美国人工智能行动计划》表示欢迎 该计划强调在医疗AI领域建立特定领域标准、监管沙盒和利益相关者会议以增强信任、治理和生产力 [1] - 公司近期开发了PredicTcell™的alpha版本 这是一个与微软和Databricks合作开发的专有机器学习模型 该模型可缩短免疫疗法研究中的靶点分析时间数周 并可能节省数十亿美元的药物开发成本 [2] - 公司创始人兼CEO Ryan Saadi表示 AI有望显著降低为患者提供救命疗法的成本和时间 公司致力于开发可扩展、符合伦理的解决方案 以扩大所有美国人获得负担得起的先进疗法的机会 [3] 技术进展 - PredicTcell™模型体现了《AI行动计划》对快速科学AI创新和医疗行业AI技术应用改进的呼吁 [2] - 该技术专注于免疫疗法研究领域 通过机器学习优化靶点分析流程 直接响应了政府对医疗AI优先事项的规划 [2] 战略合作 - 公司与微软(Nasdaq: MSFT)和Databricks建立了技术合作关系 共同开发PredicTcell™机器学习模型 [2] - 合作模式符合国家AI战略方向 通过产学研结合推动医疗AI创新 [2][3]

- Roland Kolbeck, Ph.D. Appointed as Chief Scientific Officer - - Joe Monahan, Ph.D. will Continue to Support Aclaris as Special Scientific Advisor to the Chief Executive Officer through March 2026 as Part of Planned Retirement - WAYNE, Pa., July 28, 2025 (GLOBE NEWSWIRE) -- Aclaris Therapeutics, Inc. (NASDAQ: ACRS), a clinical-stage biopharmaceutical company focused on developing novel product candidates for immuno-inflammatory diseases, today announced that Roland Kolbeck, Ph.D. has been appointed as Chie ...

NEW YORK, July 28, 2025 (GLOBE NEWSWIRE) -- Leading securities law firm Bleichmar Fonti & Auld LLP announces that a lawsuit has been filed against Sarepta Therapeutics, Inc. (NASDAQ: SRPT) and certain of the Company’s senior executives for potential violations of the federal securities laws. If you invested in Sarepta, you are encouraged to obtain additional information by visiting: https://www.bfalaw.com/cases-investigations/sarepta-therapeutics-inc-class-action. Investors have until August 25, 2025, to as ...

ROCKVILLE, Md., July 28, 2025 (GLOBE NEWSWIRE) -- Supernus Pharmaceuticals, Inc., a Delaware corporation (NASDAQ: SUPN) ("Supernus", and the "Company"), today announced that the required waiting period under the Hart-Scott-Rodino Antitrust Improvements Act of 1976, as amended ("HSR Act"), with respect to Supernus’ proposed acquisition of Sage Therapeutics, Inc., a Delaware corporation (NASDAQ: SAGE) ("Sage"), expired at 11:59 p.m. Eastern Time on July 25, 2025. On June 13, 2025, Supernus and Sage, entered i ...

公司公告 - Syndax Pharmaceuticals将于2025年8月4日公布2025年第二季度财务业绩并提供业务更新 [1] - 公司管理层将在美东时间2025年8月4日下午4:30举行电话会议和网络直播 [1] 投资者参与方式 - 网络直播和演示文稿可通过公司官网投资者关系页面的"活动与演示"栏目获取 [2] - 电话会议接入信息：国内号码800-590-8290 国际号码240-690-8800 会议ID Syndax2Q25 [2] - 直播网址为https://sndx-2q25.open-exchange-net 会议录音将在结束后24小时内上线并保留90天 [2] 公司业务概况 - Syndax Pharmaceuticals是一家商业化阶段的生物制药公司 专注于创新癌症疗法开发 [3] - 核心管线包括FDA批准的menin抑制剂Revuforj(revumenib)和CSF-1受体单抗Niktimvo(axatilimab-csfr) [3] - 公司正在通过多项临床试验全面开发管线潜力 [3] 联系方式 - 投资者关系联系人Sharon Klahre 邮箱sklahre@syndax-com 电话781-684-9827 [4]