Ampreloxetine hydrochloride

Ampreloxetine clinical development program to be featured in four presentations at the upcoming International Symposium on The Autonomic Nervous System Topline results from the ongoing Phase 3 CYPRESS study of ampreloxetine anticipated in Q1 2026 DUBLIN, Oct. 29, 2025 /PRNewswire/ -- Theravance Biopharma, Inc. (NASDAQ: TBPH) today announced that it will be participating at the 36th International Symposium on the Autonomic Nervous System, organized by the American Autonomic Society (AAS), taking place November 5-8, 2025, in Clearwater Beach, FL. The Company will have one platform presentation and three poster presentations on its clinical development program for ampreloxetine, a potential first-in-class norepinephrine reuptake inhibitor in development for the treatment of symptomatic neurogenic orthostatic hypotension (nOH) symptoms due to multiple system atrophy (MSA). "We are excited to have a strong presence at the upcoming International Symposium on the Autonomic Nervous System. Our platform presentation will highlight results from the previous REDWOOD trial, where we observed a durable symptomatic nOH benefit with improvement in activities of daily living in the pre-specified subgroup analysis in patients with MSA treated with ampreloxetine1," commented Lucy Norcliffe-Kaufmann, Ph.D., Theravance Biopharma's Executive Director of Clinical Science. "Additionally, we are pleased to share the rigorous methodologies we developed based on our previous trial experience to support enrollment and patient retention. By applying these insights, we were well positioned to successfully address the executional challenges associated with clinical studies in rare and severe neurodegenerative diseases." Presentations information and key findings: Platform presentation: Precision therapy with ampreloxetine for neurogenic orthostatic hypotension in multiple system atrophy Presenter: Lucy Norcliffe-Kaufmann, Ph.D.; Theravance Biopharma's Executive Director of Clinical Science and Associate Professor, New York University Langone Health Dysautonomia Center Session date and time: Saturday, November 8, 2025, at 8:30 am ET The following three posters will be presented at Poster Session II, taking place on Thursday, November 6, 2025, at 7:00-8:30 pm ET: Poster title: The impact of ampreloxetine on supine hypertension: an ambulatory blood pressure monitoring study Presenter: Lucy Norcliffe-Kaufmann, Ph.D.; Theravance Biopharma's Executive Director of Clinical Science and Associate Professor, New York University Langone Health Dysautonomia Center Poster number: 9 Poster title: Retention strategies in rare disease clinical trials: a case study of symptomatic treatment for multiple system atrophy Presenter: Molly Szpyhulsky, MBA, BSN, RN; Theravance Biopharma U.S. Poster number: 15 Poster title: Enrollment strategies in a phase III trial for MSA-related nOH: insights from global investigators Presenter: Molly Szpyhulsky, MBA, BSN, RN; Theravance Biopharma U.S. Poster number: 10 About Ampreloxetine Ampreloxetine, an investigational, once-daily, selective norepinephrine reuptake inhibitor in development for the treatment of symptomatic neurogenic orthostatic hypotension (nOH) in patients with multiple system atrophy (MSA). The unique benefits of ampreloxetine treatment reported in MSA patients from Study 0170 included an increase in norepinephrine levels, a favorable impact on blood pressure, clinically meaningful and durable symptom improvement, and no signal for worsening of supine hypertension. In the US, the Company has been granted an Orphan Drug Designation for ampreloxetine for the treatment of symptomatic nOH in patients with MSA and, if results from the ongoing Phase 3 CYPRESS study are supportive, plans to file an NDA for full approval in this indication. About the Phase 3 CYPRESS (Study 0197) Study The CYPRESS Study (NCT05696717) is a registrational Phase 3, multi-center, randomized withdrawal study to evaluate the efficacy and durability of ampreloxetine in participants with MSA and symptomatic nOH after 20 weeks of treatment; the primary endpoint of the study is change in the Orthostatic Hypotension Symptom Assessment (OHSA) composite score. The Study includes four periods: screening, open label (12-week period, participants will receive a single daily 10 mg dose of ampreloxetine), randomized withdrawal (eight-week period, double-blind, placebo-controlled, participants will receive a single daily 10 mg dose of placebo or ampreloxetine), and a long-term treatment extension. Secondary outcome measures include change from baseline in Orthostatic Hypotension Daily Activity Scale (OHDAS) item 1 (activities that require standing for a short time) and item 3 (activities that require walking for a short time). About the Phase 3 SEQUOIA (Study 0169) and REDWOOD (Study 0170) Studies Study 0169 (NCT03750552) was a Phase 3, 4-week, multi-center, randomized, double-blind, placebo-controlled, parallel-group study to evaluate the efficacy and safety of ampreloxetine compared to placebo in patients with symptomatic nOH (n=195). Patients from Study 0169 were eligible to enter into Study 0170 (NCT03829657), a Phase 3, multi-center, 22-week study comprising a 16-week open-label period and a 6-week double-blind, placebo-controlled, randomized withdrawal period to evaluate the sustained benefit in efficacy and safety of ampreloxetine in patients with symptomatic nOH. The primary endpoint for Study 0170 of treatment failure at week 6 was defined as a worsening of both Orthostatic Hypotension Symptom Assessment Scale (OHSA) question #1 and Patient Global Impression of Severity (PGI-S) scores by 1.0 point. After Study 0169 did not meet its primary endpoint, the Company took actions to close out the ongoing clinical program including Study 0170. The study was more than 80% enrolled (n=128/154 planned) despite stopping early. The primary endpoint was not statistically significant for the overall population of patients, which included patients with Parkinson's disease, pure autonomic failure and MSA (odds ratio=0.6; p-value=0.196). The pre-specified subgroup analysis by disease type suggests the benefit seen in patients receiving ampreloxetine was largely driven by MSA patients (n=40). An odds ratio of 0.28 (95% CI: 0.05, 1.22) was observed in MSA patients indicating a 72% reduction in the odds of treatment failure with ampreloxetine compared to placebo. The benefit to MSA patients was observed in multiple endpoints including OHSA composite, Orthostatic Hypotension Daily Activities Scale (OHDAS) composite, Orthostatic Hypotension Questionnaire (OHQ) composite and OHSA #1.1 About Multiple System Atrophy (MSA) and Symptomatic Neurogenic Orthostatic Hypotension (nOH) MSA is a progressive brain disorder that affects movement and balance and disrupts the function of the autonomic nervous system. The autonomic nervous system controls body functions that are mostly involuntary. One of the most frequent autonomic symptoms associated with MSA is a sudden drop in blood pressure upon standing (nOH).2 There are approximately 50,000 MSA patients in the US3 and 70-90% of MSA patients experience nOH symptoms.4 Despite available therapies, many MSA patients remain symptomatic with nOH.5 Neurogenic orthostatic hypotension (nOH) is a rare disorder defined as a fall in systolic blood pressure of ⩾20 mm Hg or diastolic blood pressure of ⩾10 mm Hg, within 3 minutes of standing. Severely affected patients are unable to stand for more than a few seconds because of their decrease in blood pressure, leading to cerebral hypoperfusion and syncope. A debilitating disorder, nOH results in a range of symptoms including dizziness, lightheadedness, fainting, fatigue, blurry vision, weakness, trouble concentrating, and head and neck pain. About Theravance Biopharma Theravance Biopharma, Inc.'s focus is to deliver Medicines that Make a Difference® in people's lives. In pursuit of its purpose, Theravance Biopharma leverages decades of expertise, which has led to the development of FDA-approved YUPELRI® (revefenacin) inhalation solution indicated for the maintenance treatment of patients with chronic obstructive pulmonary disease (COPD). Ampreloxetine, its late-stage investigational once-daily norepinephrine reuptake inhibitor in development for symptomatic neurogenic orthostatic hypotension (nOH) in patients with Multiple System Atrophy (MSA), has the potential to be a first in class therapy effective in treating a constellation of cardinal symptoms in MSA patients. The Company is committed to creating/driving shareholder value. For more information, please visit www.theravance.com. THERAVANCE BIOPHARMA®, THERAVANCE® and the Cross/Star logo are registered trademarks of the Theravance Biopharma group of companies (in the U.S. and certain other countries). YUPELRI® is a registered trademark of Viatris Specialty LLC. Trademarks, trade names or service marks of other companies appearing on this press release are the property of their respective owners. Forward-Looking Statements This press release will contain certain "forward-looking" statements as that term is defined in the Private Securities Litigation Reform Act of 1995 regarding, among other things, statements relating to goals, plans, objectives, expectations and future events. Theravance Biopharma, Inc. (the "Company") intends such forward-looking statements to be covered by the safe harbor provisions for forward-looking statements contained in Section 21E of the Securities Exchange Act of 1934, as amended, and the Private Securities Litigation Reform Act of 1995. Examples of such statements include statements relating to: the Company's expectations regarding its future profitability, expenses and uses of cash, the Company's goals, designs, strategies, plans and objectives, future growth of YUPELRI sales, future royalty payments, the ability to provide value to shareholders, the Company's regulatory strategies and timing of clinical studies, possible safety, efficacy or differentiation of our investigational therapy, the status of patent infringement litigation initiated by the Company and its partner against certain generic companies in federal district courts; contingent payments due to the Company from the sale of the Company's TRELEGY ELLIPTA royalty interests to Royalty Pharma, and expectations around the use of OHSA scores as endpoints for clinical trials. These statements are based on the current estimates and assumptions of the management of Theravance Biopharma as of the date of this press release and the conference call and are subject to risks, uncertainties, changes in circumstances, assumptions and other factors that may cause the actual results of Theravance Biopharma to be materially different from those reflected in the forward-looking statements. Important factors that could cause actual results to differ materially from those indicated by such forward-looking statements include, among others, risks related to: factors that could increase the Company's cash requirements or expenses beyond its expectations and any factors that could adversely affect its profitability, whether the milestone thresholds can be achieved, delays or difficulties in commencing, enrolling or completing clinical studies, the potential that results from clinical or non-clinical studies indicate the Company's product candidates or product are unsafe, ineffective or not differentiated, risks of decisions from regulatory authorities that are unfavorable to the Company, dependence on third parties to conduct clinical studies, delays or failure to achieve and maintain regulatory approvals for product candidates, risks of collaborating with or relying on third parties to discover, develop, manufacture and commercialize products, and risks associated with establishing and maintaining sales, marketing and distribution capabilities with appropriate technical expertise and supporting infrastructure, the ability of the Company to protect and to enforce its intellectual property rights, volatility and fluctuations in the trading price and volume of the Company's shares, and general economic and market conditions. Other risks affecting the Company are in the Company's Form 10-Q filed with the SEC on August 13, 2025, and other periodic reports filed with the SEC. In addition to the risks described above and in Theravance Biopharma's filings with the SEC, other unknown or unpredictable factors also could affect Theravance Biopharma's results. No forward-looking statements can be guaranteed, and actual results may differ materially from such statements. Given these uncertainties, you should not place undue reliance on these forward-looking statements. Theravance Biopharma assumes no obligation to update its forward-looking statements on account of new information, future events or otherwise, except as required by law. Contact: investor.relations@theravance.com 650-808-4045 1 Freeman R, et al. Precision therapy with ampreloxetine for neurogenic orthostatic hypotension in multiple system atrophy. MedRxiv. https://www.medrxiv.org/content/10.1101/2025.08.12.25332833v1. 2https://medlineplus.gov/genetics/condition/multiple-system-atrophy/ 3 UCSD Neurological Institute (25K-75K, with ~10K new cases per year); NIH National Institute of Neurological Disorders and Stroke (15K-50K). 4 Delveinsight MSA Market Forecast (2023); Symptoms associated with orthostatic hypotension in pure autonomic failure and multiple systems atrophy, CJ Mathias (1999). 5 Data on file. MSA Natural History Statistics, NYU September 2019. View original content to download multimedia:https://www.prnewswire.com/news-releases/theravance-biopharma-to-present-data-on-ampreloxetine-at-the-36th-international-symposium-on-the-autonomic-nervous-system-302597901.html SOURCE Theravance Biopharma, Inc.

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Therapeutics宣布完成1180万美元A轮融资，以推进其肿瘤学管线。该公司专注于开发下一代双特异性抗体药物偶联物（bsADC）及相关疗法，旨在提高癌症治疗的疗效、安全性和精准性。本轮融资由欧洲风险投资基金redalpine领投，新投资者LongeVC和Oxford Science Enterprises参与，现有投资者也贡献其中。Valink计划利用资金推进两个肿瘤项目进入临床前开发，包括针对结直肠癌的治疗。 国产双抗双载荷ADC出海 原文标题：国产双抗双载荷ADC出海（新闻来源：动脉网） 原文地址：https://www.vbdata.cn/intelDetail/817993 三星集团子公司Samsung Bioepis与拓济医药（Phrontine Biopharma）签署全球合作协议。双方将共同开发、生产和商业化两种ADC药物，包括处于临床前开发阶段的靶向HER3和EGFR的双特异性双药载ADC——TJ108（结合TOP1i和微管抑制剂作为细胞毒性药载），以及另一款待命名产品。此外，Samsung Bioepis获得一种拓扑异构酶1抑制剂（TOP1i）有效载荷的独家许可，用于自身ADC管线开发。作为合作回报，Phrontline将获得预付款及与开发和监管成就挂钩的额外里程碑付款，具体财务细节未披露。 产业动态 ESMO: ADC的“过山车”之旅仍在继续，新一代疗法正在登场 原文标题：ESMO: The ADC 'rollercoaster' rides on with new generation of therapies（新闻来源：Fiercebiotech） 原文地址：https://www.fiercebiotech.com/biotech/esmo-adc-rollercoaster-rides 在2025年ESMO大会上，ADC成为焦点。罗氏宣布与汉森制药达成14.5亿美元的ADC许可协议，进一步巩固其在ADC领域的地位。GSK展示了与汉森合作的ADC数据，强调新一代ADC在靶向精准性和临床活性上的进步。阿斯利康在大会上重点介绍了其与第一三共合作的ADC药物Enhertu，数据显示其在HER2阳性早期乳腺癌辅助治疗中显著降低疾病复发或死亡风险。尽管ADC在大会上备受关注，但Moderna等公司对其持保留态度，认为ADC只是化疗的另一种形式，且常伴随类似副作用。Moderna更看好其个性化癌症疫苗和其他mRNA癌症疗法的潜力。 从乳膏到生物制剂：适应新疗法的皮肤科试验 原文标题：From Creams to Biologics: Adapting Dermatology Trials to New Therapies（新闻来源：Pharmaceutical Technology） 原文地址：https://www.pharmaceutical-technology.com/sponsored/from-creams-to-biologics-adapting-dermatology-trials-to-new-therapies/ 全球皮肤科市场蓬勃发展，创新不断。从智能口服药物到基于基因治疗的罕见皮肤病修复和皮肤年轻化，治疗方法不断变化，临床试验也必须随之调整。GlobalData预计，到2030年，全球主要制药市场（美国、欧洲五大国和日本）的银屑病治疗销售额将从2020年的116亿美元增长到317亿美元。细胞和基因治疗（CGT）是皮肤科的前沿领域，预计销售额将从2024年的2.91亿美元增长到2030年的15亿美元。尽管资金、制造需求和有限的报销可能会影响其应用，但皮肤科研究正在不断拓展和深化。 从生物技术到董事会：早期肿瘤数据如何建立投资者信心 原文标题：Biotech to boardroom: How early oncology data builds investor confidence（新闻来源：Pharmaceutical Technology） 原文地址：https://www.pharmaceutical-technology.com/sponsored/biotech-to-boardroom-how-early-oncology-data-builds-investor-confidence/ 肿瘤药物开发复杂且成本高昂，临床数据至关重要。早期临床数据对塑造投资者信心和建立合作起决定性作用。与临床研究组织（CRO）合作可增强投资者信心。GlobalData数据显示，2025年二季度十大制药交易集中在早期试验阶段，显示投资者对强大基础科学和早期数据的需求。早期数据不仅能吸引资金，还能推动战略合作伙伴关系。CRO通过提供专业知识和运营基础设施，帮助生成可靠数据，增强科学依据，确保研究设计和数据完整性。在投资环境收紧的情况下，早期数据的质量和强度对成功至关重要。 罗氏，人类基因组测序，4小时，吉尼斯纪录 原文标题：罗氏，人类基因组测序，4小时，吉尼斯纪录（新闻来源：动脉网） 原文地址：https://www.vbdata.cn/intelDetail/818059 10月15日，罗氏联合Broad Clinical Labs与波士顿儿童医院在《新英格兰医学杂志》发表成果，以不到4小时完成人类基因组测序与分析，刷新吉尼斯纪录，提升NICU临床决策和救治效率。该成果显示，从基因组DNA生成变异平均耗时4小时4分钟。罗氏提出全新Sequencing by Expansion（SBX）技术，其核心是Xpandomer分子，通过纳米孔实现高精度、高通量测序。该技术克服传统局限，8小时内可完成可指导诊疗的报告，首次展示当日全基因组诊断路径，虽未直接用于临床，但有望带来范式转变，在新生儿、肿瘤、传染病等领域有巨大价值。 全球裁员365人！又一Biotech放弃细胞疗法 原文标题：全球裁员365人！又一Biotech放弃细胞疗法（新闻来源：新浪财经） 原文地址：https://finance.sina.com.cn/stock/med/2025-10-21/doc-infuryam4272283.shtml 生物技术公司加拉帕戈斯（Galapagos）于10月23日宣布，因出售细胞治疗业务尝试失败，将逐步关停该业务板块，涉及欧洲、中国及美国地区的365个工作岗位。自今年5月起，公司为该业务探索多种发展方案，收到多份非约束性报价，但经评估认为这些报价均不具备可行性。公司表示，当前逐步终止细胞治疗业务，将资金转而用于构建新疗法研发管线，是更合理战略选择。受此消息影响，Galapagos股价在公告发布后的早盘交易中下跌14%。 Theravance在关键数据公布之前启动了针对nOH的医生教育活动 原文标题：Theravance stands up nOH physician education campaign ahead of pivotal data（新闻来源：Fiercepharma） 原文地址：https://www.fiercepharma.com/marketing/theravance-stands-physician-education-campaign-ahead-pivotal-data Theravance Biopharma正在加强教育医生关于一种罕见疾病的努力，推出了“Power in the Periphery”宣传活动，以迎接即将公布的3期数据。该活动聚焦于与多系统萎缩（MSA）相关的神经源性直立性低血压（nOH）的驱动因素和症状。在美国，约50,000名MSA患者中，70%至90%有nOH症状，表现为站立后血压下降，可能导致头晕、晕厥和疲劳。此次宣传活动在本月的国际帕金森病和运动障碍大会上启动，并设有专门网站。该活动旨在为公司的安普罗克司汀（ampreloxetine）铺路，该药物旨在通过选择性抑制去甲肾上腺素的再摄取来提高站立血压并减少nOH症状。公司计划在2026年第一季度报告3期数据，并在数据积极的情况下寻求FDA批准。 多款知名进口药告别国内市场，个别集采品种注销 原文标题：多款知名进口药告别国内市场，个别集采品种注销（新闻来源：新浪财经） 原文地址：https://finance.sina.com.cn/roll/2025-10-21/doc-infusksn9714010.shtml 10月15日晚，国家药监局宣布注销费卡华瑞的氯雷他定片等80个药品的注册证书，这些药品后续将停止生产销售。此次注销均为企业“依申请注销”，多是商业选择而非因安全问题。在80个药品中，超过55%来自外资企业，包括一些知名进口药。近年来，随着国内医药产业的发展，特别是国家药品集采和谈判政策的推进，一些外资药企调整在华策略，部分药品选择注销。 前沿发现 视网膜植入装置恢复地理萎缩患者视力 原文标题：Joy as retinal implant restores sight in geographic atrophy（新闻来源：pharmaphorum） 原文地址：https://pharmaphorum.com/news/joy-retinal-implant-restores-sight-geographic-atrophy Science Corp开发的PRIMA 2x2毫米无线视网膜植入装置在临床试验中显示出可恢复地理萎缩（GA）患者视力的效果。GA是一种严重的干性年龄相关性黄斑变性，会导致中央视力丧失。该植入装置通过微型芯片替换丢失的视网膜细胞，将光转化为电信号。患者需佩戴特制的摄像眼镜和口袋电脑，后者利用人工智能算法解读数据并放大文本。试验中，32名患者中有81%在植入12个月后视力显著改善，84%能在家中用该装置阅读。该装置为GA患者提供了除药物和基因疗法外的新治疗选择，Science Corp已向欧盟申请监管批准，并在美国推进FDA审批流程。 研究揭示神经退行性疾病中蛋白斑块作为酶分解ATP 原文标题：Study reveals protein plaques in neurodegeneration function as enzymes, breaking down ATP（新闻来源：The Medical News） 原文地址：https://www.news-medical.net/news/20251021/Study-reveals-protein-plaques-in-neurodegeneration-function-as-enzymes-breaking-down-ATP.aspx 莱斯大学Pernilla Wittung-Stafshede领导的新研究揭示，与帕金森病相关的α-突触核蛋白形成的蛋白斑块不仅不是废物，还能像酶一样分解ATP，从而耗尽脑细胞能量。研究发表于10月16日的《Advanced Science》，发现ATP结合到斑块时，蛋白会重塑自身，将ATP困在一个小口袋中并促使其分解。这一发现改变了人们对蛋白斑块的理解，可能解释为何神经退行性疾病患者的脑细胞会出现能量短缺、DNA损伤等化学应激反应，最终导致细胞死亡。该研究为开发新疗法提供了可能，未来或许可通过锁定这些斑块的形状来减轻其有害影响。 MRI年龄时钟揭示每个器官衰老速度不同并预测疾病或长寿 原文标题：MRI age clocks reveal how each organ ages differently and predict who develops disease or lives longer（新闻来源：The Medical News） 原文地址：https://www.news-medical.net/news/20251019/MRI-age-clocks-reveal-how-each-organ-ages-differently-and-predict-who-develops-disease-or-lives-longer.aspx 一项发表在《自然医学》杂志上的新研究开发了七种基于磁共振成像（MRI）的生物年龄差距（MRIBAGs），涵盖大脑、心脏、肝脏、脾脏、肾脏、脂肪组织和胰腺。这些模型通过人工智能/机器学习（AI/ML）预测器官年龄与实际年龄的差异，揭示了不同器官衰老速度的差异。研究还测试了这些模型与分子相关性、遗传学、疾病预测、死亡率联系以及对阿尔茨海默病试验结果的相关性。结果显示，这些器官特异性时钟与非胰岛素依赖性糖尿病、高血压等常见疾病相关，能预测全因死亡率差异，并追踪阿尔茨海默病预防试验中的认知轨迹。这些发现为精准预防、早期干预和更智能的临床试验提供了新的视角，但需在多样化人群中进行纵向验证。 研究人员实现从体细胞生成卵子的概念验证 原文标题：Researchers achieve proof of concept for generating eggs from somatic cells（新闻来源：The Medical News） 原文地址：https://www.news-medical.net/news/20251017/Researchers-achieve-proof-of-concept-for-generating-eggs-from-somatic-cells.aspx 俄勒冈健康与科学大学的研究人员在《自然通讯》杂志上发表了一项概念验证研究，成功将皮肤细胞转化为能够产生早期人类胚胎的卵子，为治疗不孕症开辟了新途径。这一成果为体外配子发生（IVG）提供了潜在方法，有望帮助因高龄、癌症治疗或其他原因无法产生健康卵子的女性解决不孕问题。该技术被称为“mitomeiosis”，结合了有丝分裂和减数分裂两种细胞分裂过程。研究人员通过将皮肤细胞核移植到去核的卵子中，成功产生了82个功能性卵子，并通过体外受精（IVF）使其受精。尽管大多数胚胎未能发育到囊胚阶段且存在染色体异常，但仍有9%的胚胎发育到囊胚阶段。研究人员强调，这一成果目前仅是概念验证，距离临床应用还需至少十年的进一步研究。