–   Q3 2025 revenue of €143.1 million for YORVIPATH® and €50.7 million for SKYTROFA® –   Q3 2025 operating profit of €11.0 million –   TransCon® CNP (navepegritide) under FDA Priority Review for the treatment of children with achondroplasia with PDUFA date of November 30, 2025 –   Conference call today at 4:30 pm ET COPENHAGEN, Denmark, Nov. 12, 2025 (GLOBE NEWSWIRE) -- Ascendis Pharma A/S (Nasdaq: ASND) today announced financial results for the third quarter ended September 30, 2025, and provided a busine ...

Vor Biopharma (NasdaqGS:VOR) Conference November 12, 2025 02:30 PM ET Speaker2All right, good afternoon, everybody, and thank you once again for joining us for our first annual INI Summit. I'm Jørn from the TD Cowen Biotech team, and it's a great pleasure to have with us today the management from Vor Bio. We have Jean-Paul Kress, who is the CEO. We have Dallan Murray, who's Chief Commercial Officer, and we have Jeremy Sokolove, who's Chief Medical Officer. Somewhere in the background, weaving all his magic ...

SAN FRANCISCO, Nov. 12, 2025 (GLOBE NEWSWIRE) -- Telix Pharmaceuticals Limited (NASDAQ: TLX), a commercial-stage biopharmaceutical company focused on medical imaging and therapeutic radiopharmaceuticals, is now the target of a securities class action lawsuit, capping a tumultuous several months that saw the company's stock crater following revelations of a regulatory subpoena and an FDA rejection letter. National shareholders rights firm Hagens Berman is actively investigating the alleged legal claims and u ...

SAN FRANCISCO, Nov. 12, 2025 (GLOBE NEWSWIRE) -- Shareholder rights firm Hagens Berman is scrutinizing Icelandic biopharmaceutical company Alvotech (NASDAQ: ALVO) over the propriety of its disclosures regarding its lead drug candidate and its manufacturing operations, following a dramatic revision to the company’s full-year financial forecasts. The firm urges investors in Alvotech who suffered significant losses to submit your losses now. The firm also encourages persons with knowledge who may be able to as ...

Accessibility StatementSkip Navigation PHILADELPHIA, Nov. 12, 2025 /PRNewswire/ -- National plaintiffs' law firm Berger Montague PC announces that a class action lawsuit has been filed against Telix Pharmaceuticals Ltd. (NASDAQ: TLX) ("Telix" or the "Company") on behalf of investors who purchased Telix securities during the period of February 21, 2025 through August 28, 2025 (the "Class Period"). Investor Deadline: Investors who purchased Telix securities during the Class Period may, no later than Januar ...

AnaptysBio (NasdaqGS:ANAB) FY Conference November 12, 2025 11:30 AM ET Speaker0Hi. Good morning, everyone. Welcome to Guggenheim Healthcare Innovation Conference. My name is Yatin Suneja, one of the biotech analyst here. It is my pleasure to welcome our next presenting company, Anaptis Bio.From the company, we have president and CEO, Danielle Vega. Dan, thank you so much for joining us. Always pleasure to have you. Why don't you make some opening comments, you know, highlight some of the upcoming or near te ...

公司概况 * 涉及的上市公司为Rhythm Pharmaceuticals (NasdaqGM: RYTM) [1] * 公司专注于通过靶向MC4通路治疗遗传性和获得性肥胖症 [1] 核心产品与监管进展 * 核心产品setmelanotide是一种α-MSH类似物，旨在通过黑皮质素-4受体通路进行激素替代治疗 [25] * 美国食品药品监督管理局将setmelanotide用于下丘脑肥胖适应症的处方药使用者付费法案目标日期推迟了三个月至3月20日 [6][8] * 延期原因是公司提交了新的数据集，被FDA认定为重大修订，需要额外时间审查，但敏感性分析显示数据结果无差异 [8][9][10] 商业机会与市场策略 * 下丘脑肥胖在美国的患者估计数约为10，000人，高于早期估计的5，000-7，000人 [12][21] * 公司已通过理赔分析识别出约2，000名疑似和确诊患者 [24] * 商业准备方面，北美现场团队已全部招聘完毕并部署就位，三个月的延期提供了更多的准备时间 [11] * 国际市场上，欧洲和日本是重点，日本的患者流行病学估计为5，000-8，000人，与美国相当 [29][31] 临床数据与产品差异化 * 在下丘脑肥胖临床试验中，setmelanotide显示出约20%的体重减轻效果 [25] * 临床试验允许使用GLP-1药物的患者入组，数据显示约20%或更少的患者对GLP-1有反应，且反应温和（体重减轻10%或更少） [26] * 对GLP-1无反应或体重反弹的患者，在使用setmelanotide后表现出良好反应，提示其可能恢复患者对其他抗肥胖药物（包括GLP-1）的反应能力 [27] * 与普拉德-威利综合征相比，下丘脑肥胖患者的诊断率、就医动力和医疗系统可及性不同，预计市场渗透速度将快于Bardet-Biedl综合征但慢于普拉德-威利综合征 [16][19][20] 研发管线与下一代资产 * 针对普拉德-威利综合征的setmelanotide研究数据预计在圣诞节前公布，成功概率被评估为50/50 [35][37] * 下一代资产包括口服药物bivamelagon和每周注射一次的药物718（RM-718） [38] * RM-718是一种七氨基酸肽，比bivamelagon效力更强、特异性可能更高，目标是完成开放标签第一部分患者入组，并在2026年中期公布数据 [40][42] * setmelanotide在疗效上已被证明非常有效，下一代资产的主要优势在于改善hyperpigmentation等副作用 [44] 重要但可能被忽略的细节 * 公司有一项名为M&A的临床试验将于明年启动，旨在研究四个不同的基因 [3] * 下丘脑肥胖患者中，超过80%存在某种程度的垂体功能不全，这为识别患者提供了线索 [13] * 公司强调在国际市场拓展中，拥有合适的本地团队成员至关重要，而非仅仅依赖患者数量 [31]

– Recruitment ongoing for Phase 2 Seabreeze STAT studies for acute exacerbations in asthma and COPD; expect to report topline data from both studies in 1H26 – – New Drug Application for rademikibart for the treatment of atopic dermatitis submitted by Simcere, the Company’s exclusive licensee in Greater China, was accepted by China’s NMPA – – Completed termination of American Depositary Receipt program and directly listed our ordinary shares on Nasdaq – – Presented positive data at ERS 2025 supporting the po ...

公司人事任命 - Immix Biopharma任命Michael Grabow为首席商务官，负责领导NXC-201在美国的上市和商业化建设[1] - Michael Grabow在生物制药商业化领域拥有超过25年经验，曾担任Chimerix商业运营负责人，成功领导孤儿药MODEYSO®的市场进入策略和上市[2] - Michael Grabow职业生涯中直接参与推动超过10次成功的商业药物上市[2] 核心产品NXC-201 - NXC-201是一种空间优化的BCMA靶向CAR-T细胞疗法，具有过滤非特异性激活的"数字滤波器"[5] - NXC-201已获得美国FDA授予的再生医学先进疗法（RMAT）资格以及美国FDA和欧盟EMA授予的孤儿药资格（ODD）[5][7] - NXC-201采用"一次性"治疗方法，有望成为首个在复发/难治性AL淀粉样变性中获得批准的治疗方案[2] - NXC-201目前正在美国进行多中心注册性研究NEXICART-2（NCT06097832）[7] 疾病领域与市场前景 - AL淀粉样变性是一种毁灭性疾病，免疫系统持续产生有毒轻链，导致心脏、肾脏和肝脏堵塞，引起器官衰竭和死亡[3][7] - 美国复发/难治性AL淀粉样变性患者数量预计以每年12%的速度增长，2025年将达到约37,270名患者[3] - 淀粉样变性市场规模从2017年的36亿美元预计增长至2025年的60亿美元[4] 行业背景与高管观点 - Michael Grabow此前供职的Chimerix公司被Jazz Pharmaceuticals以9.35亿美元收购[1][2] - 公司首席执行官Ilya Rachman认为NXC-201在解决复发/难治性AL淀粉样变性的高度未满足医疗需求方面具有独特优势[2] - 公司首席财务官Gabriel Morris表示此次任命将加速NXC-201的部署[2]

WILMINGTON, Del., Nov. 12, 2025 (GLOBE NEWSWIRE) -- NRx Pharmaceuticals, Inc. (Nasdaq: NRXP) ("NRx Pharmaceuticals", the "Company"), a clinical-stage biopharmaceutical company, today announced that it will release its third quarter 2025 financial results before the market opens on Monday, November 17, 2025 via press release, which will be available on the Company's website at https://ir.nrxpharma.com/. The Company will host a conference call at 8:30am ET the same day. The call will provide a corporate and f ...