A Prospective Single-arm Exploratory Study on the Efficacy and Safety of Treprostinil Injection in the Treatment of Patients With Intermediate-risk Pulmonary Arterial Hypertension

Pulmonary Arterial Hypertension (PAH) is a serious condition that puts strain on the right side of the heart. While oral medications can help, many patients with intermediate-risk PAH may not see enough improvement, and their heart function can continue to decline.
This study aims to find out if adding an injectable medication, Treprostinil, to a patient's current oral PAH therapy can improve heart function and overall health. This is a single-arm study, which means all participants will receive the study treatment.
The main goal is to measure the change in the amount of blood the right side of the heart pumps with each beat (Right Ventricular Stroke Volume, or RVSV) after 3 months of treatment. This will be measured using a specialized heart scan called Cardiac Magnetic Resonance Imaging (CMR). Researchers will also assess changes in exercise ability (with a 6-minute walk test), blood markers, and patient symptoms.
Participants will be in the main part of the study for 3 months, with follow-up for a total of 24 months to monitor the long-term effects and safety of the treatment.

开始日期2025-10-31

申办/合作机构-

NCT06603285

/ Not yet recruitingN/A

A Multi-center, Prospective Study to Observe the Safety, Tolerability, and Efficacy of Remodulin® in Patients With Pulmonary Arterial Hypertension

This is a multi-center, single-arm, prospective study to enroll patients diagnosed as pulmonary arterial hypertension. The objective in this study is to observe the efficacy and safety of treprostinil in subjects with pulmonary arterial hypertension.

开始日期2025-10-16

申办/合作机构

Excelsior

NCT07112183

/ Recruiting临床4期IIT

An Open Label Study to Assess Efficacy of Oral Treprostinil Titrated to 3.0mg Three Times Daily or Highest Tolerable Dose in 30 Patients With Symptomatic Primary or Secondary Raynaud's Phenomenon Resistant to Standard Vasodilatory Therapy

Raynaud's phenomenon is a condition where the blood vessels in participants fingers and toes get too narrow when cold or stressed. This makes participants fingers and toes change colors - they might turn white, then blue, and finally red as blood flow returns. It can be painful and cause numbness or tingling.
When participants have Raynaud's, blood vessels react too strongly to cold or stress. Fingers and toes may turn white (blood moves away from the area), blue (lack of oxygen), or red and feel painful or tingly when warming up. These episodes usually last from a few minutes to several hours.
There are two types of Raynaud's. Primary Raynaud's (also called Raynaud's disease) itself and isn't connected to other health problems. It's the most common type and affects mostly women under 30. Secondary Raynaud's (also called Raynaud's phenomenon) is caused by other diseases like lupus, scleroderma, or rheumatoid arthritis. This type tends to be more serious and may cause painful sores on fingertips called digital ulcers.
For mild cases, staying warm might be enough. But if symptoms are severe, participants doctor might prescribe various medications including calcium channel blockers - blood pressure medicines that help open blood vessels, or other vasodilators - medicines that widen blood vessels. About 40% of people with scleroderma develop painful sores on their fingertips called digital ulcers. These happen when there isn't enough blood flow to heal small injuries.
For severe cases with digital ulcers, doctors might use prostacyclin therapy - medicines that mimic a natural substance that opens blood vessels. Oral treprostinil is a newer pill form of prostacyclin therapy that helps improve blood flow. The investigators are conducting a research study testing whether oral treprostinil - a pill that mimics prostacyclin (a natural blood vessel opener) - can help people with severe Raynaud's that doesn't respond to usual treatments. This represents hope for better treatment options for people with the most challenging cases of this condition.

开始日期2025-10-15

申办/合作机构

The Brigham & Women's Hospital, Inc.

100 项与曲前列尼尔相关的临床结果

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100 项与曲前列尼尔相关的转化医学

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100 项与曲前列尼尔相关的专利（医药）

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876

项与曲前列尼尔相关的文献（医药）

2026-08-01·CURRENT PROBLEMS IN CARDIOLOGY

Management of pulmonary arterial hypertension with intermediate-high-risk pulmonary embolism during pregnancy: Diagnostic challenges, catheter-directed thrombolysis, and intravenous treprostinil — A narrative review with an institutional case snapshot

Review

作者: Navarro-Vergara, Dulce-Iliana ; Torres-Rojas, María-Berenice ; Roldan-Valadez, Ernesto ; Hernandez-Villa, Lizbeth ; Cueto-Robledo, Guillermo ; Ruiz-Domínguez, Daniel ; García-Cesar, Marisol

BACKGROUND:

Maternal mortality from pregnancy-related pulmonary arterial hypertension (PAH) is estimated at 7-12%. The superimposition of pulmonary embolism (PE) and COVID-19 further compromises right-ventricular (RV) function. Pregnancy-compatible PAH pharmacotherapy and catheter-directed techniques show promise; however, their intersection remains undocumented.

METHODS:

We performed a narrative review of imaging diagnosis, risk stratification, catheter-directed reperfusion, PAH-targeted pharmacotherapy, and multidisciplinary peripartum management of PAH complicated by acute PE during pregnancy. MEDLINE/PubMed, Embase, Scopus, and the Cochrane Library were searched up to March 2026. We also describe an institutional case from the Hospital General de México Dr. Eduardo Liceaga (Mexico City, Mexico).

RESULTS:

Evidence supports echocardiography for initial assessment, CT pulmonary angiography for embolic/vascular evaluation, right-heart catheterization for hemodynamic determination, and catheter-directed thrombolysis for intermediate-high-risk PE when systemic thrombolysis is contraindicated. The institutional case describes a 17-year-old primigravida (24.6 weeks' gestation) with undiagnosed idiopathic PAH (mean pulmonary artery pressure 64 mmHg; pulmonary vascular resistance (PVR) 13.8 Wood units), intermediate-high-risk PE (thrombotic burden 37.5%), and COVID-19 pneumonia. Catheter-directed thrombolysis combined with sildenafil and intravenous treprostinil achieved substantial hemodynamic improvement (cardiac index 2.8 to 4.2 L/min/m²; PVR 13.8 to 6.7 Wood units). Elective cesarean at 37 weeks resulted in satisfactory maternal and neonatal outcomes.

CONCLUSIONS:

Early, multidisciplinary approaches integrating invasive hemodynamics, catheter-directed thrombolysis, and pregnancy-compatible PAH therapy can decompress RV function and allow safe pregnancy continuation. Prospective international registries and pregnancy-specific PERT pathways are urgently needed.

2026-06-01·INTERNAL MEDICINE

Pulmonary Hypertension Associated with Severe Interstitial Pneumonia Successfully Treated with Inhaled Treprostinil

Article

作者: Furusawa, Haruhiko ; Yamada, Takayuki ; Sonoda, Shiro ; Shirai, Tsuyoshi ; Otsuka, Mitsuki ; Tateishi, Tomoya ; Sawada, Atsushi ; Yasuda, Tomoka ; Aoki, Shotaro ; Miyazaki, Yasunari

Inhaled treprostinil is the first-choice drug for the treatment of Group 3 pulmonary hypertension (PH) associated with chronic lung disease. A 72-year-old man was diagnosed with interstitial pneumonia, and his respiratory symptoms had gradually worsened. Echocardiography revealed a maximum tricuspid regurgitation pressure gradient (TRPG) of 64 mmHg, and the patient was diagnosed with Group 3 PH. Subsequently, he was admitted to the hospital with acute heart failure. Inhaled treprostinil was prescribed, his B-type natriuretic peptide level and maximum TRPG subsequently improved, and his oxygen requirement decreased. Treprostinil inhalation may be effective in treating patients with severe respiratory symptoms of interstitial pneumonia.

2026-05-19·Zhonghua yi xue za zhi

[Progress in targeted therapy for chronic thromboembolic pulmonary hypertension].

Review

作者: Wan, J ; Hu, X H ; Zhang, M

Chronic thromboembolic pulmonary hypertension (CTEPH) is a pulmonary vascular disease characterized by organized thrombotic obstruction of the pulmonary arteries and subsequent pulmonary vascular remodeling. The therapeutic strategy has evolved from a solely surgical approach to a comprehensive system incorporating pulmonary endarterectomy, balloon pulmonary angioplasty and targeted drugs. Although riociguat remains the only drug formally approved for patients with inoperable or residual/recurrent pulmonary hypertension in China, accumulating evidence suggests that other targeted agents, such as treprostinil, may also show clinical benefits in CTEPH. Current evidence highlights the importance of multimodal therapy. This review aims to comprehensively summarize recent advances in targeted drugs for CTEPH patients and to examine the current applications and unresolved challenges associated with multimodal therapy strategies in this field.

366

项与曲前列尼尔相关的新闻（医药）

2026-06-11

·Business Wire

Corsair Pharma Announces Three Presentations of Phase 1 Clinical Data for TRX-248 Transdermal System at the 2026 Pulmonary Hypertension Association Conference

NEWARK, Calif.--(BUSINESS WIRE)--Corsair Pharma, Inc., a private biopharmaceutical company, today announced three presentations related to its clinical program at the Pulmonary Hypertension Association (PHA) meeting taking place June 11-14, 2026, in Dallas, Texas. Corsair is developing the TRX-248 Transdermal System, an investigational once-daily transdermal patch for the treatment of pulmonary arterial hypertension (PAH). Positive Phase 1 pharmacokinetic, tolerability, and dosing data from healthy volunteers were recently announced, and these presentations will provide additional details on the study results. Positive Phase 1 results demonstrate steady, continuous treprostinil exposure and support further clinical development. Prostacyclins are a standard-of-care drug class for the treatment of PAH. Corsair’s transdermal patch is designed to provide steady, continuous systemic exposure to treprostinil with once-daily application. The patch delivers an inactive prodrug, TRX-248, which crosses the skin and enters the bloodstream before conversion to active treprostinil in the liver. Corsair believes this approach may also have potential to treat other forms of pulmonary hypertension. Dr. Bobby Singh, President and Chief Operating Officer of Corsair, will present a poster titled “Pharmacokinetics and Safety of a Novel Once-daily Treprostinil Prodrug Transdermal System in Humans” during the Unopposed Research Poster Hall Session on Thursday, June 11 from 4:00 p.m. to 5:00 p.m. The Corsair TRX-248 Transdermal System will also be featured in the PHA Innovation Forum, which highlights novel development programs that may improve the diagnosis and management of patients with pulmonary hypertension. At the Innovation Forum, R. James White, MD, PhD, Professor of Medicine, Pharmacology & Physiology at the University of Rochester Medical Center will present Corsair’s Phase 1 data on June 11 from 5:00 p.m. to 6:30 p.m. In addition, a Corsair Product Theater presentation titled “Back to the Future: A Daily Treprostinil Prodrug Transdermal System Delivers Systemic Treprostinil with Modest Skin Irritation” will be held on Friday, June 12 from 10:30 a.m. to 11:30 a.m. Dr. White and Murali Chakinala, MD, Professor in the Division of Pulmonary and Critical Care Medicine at Washington University will discuss the benefits and current challenges of treprostinil administration modalities, relevant Phase 1 findings, next steps in development, and practical application of this product candidate in the management of PAH. About PAH and Treprostinil Pulmonary arterial hypertension (PAH) is a serious, progressive, and ultimately fatal disease that causes shortness of breath and markedly reduces quality of life. In the U.S., approximately 45,000 patients are currently taking treatments labeled for PAH. Current therapies clearly improve function and outcomes, but there remains a tremendous unmet medical need. Importantly, many patients are unwilling or unable to utilize prostanoids, one of the most effective therapies, and Corsair believes that the TRX-248 Transdermal System could provide an important new option for them. Treprostinil is commonly used in the treatment of PAH and exerts its pharmacological effects through vasodilation, reduced platelet aggregation, and inhibition of smooth muscle proliferation. Treprostinil is currently marketed by United Therapeutics and Liquidia in multiple formulations to alleviate symptoms, maintain or improve functional class, delay disease progression, and enhance quality of life in patients with PAH. The U.S. prostacyclin market for PAH is estimated at approximately $3.3 billion annually. About Corsair Pharma, Inc. Corsair Pharma is developing transformative solutions to improve the therapeutic profile of medications and provide superior treatment options for patients. The company is focused on the development of novel prodrugs of treprostinil to treat patients with Pulmonary Arterial hypertension (PAH) using a once-daily transdermal patch. The company intends to pursue a 505(b)(2) regulatory pathway, which is a streamlined process to develop new versions of approved drugs. For more information, visit www.corsairpharma.com.

临床1期上市批准

2026-06-09

·Drug科技与狠活

ATS 2026 国际会议

ATS 2026国际会议医药研发趋势深度报告 ━━━━━━━━━━━━━━━━━━━━━━━━━━━━ 聚焦：MNC管线进展| MNC押注公司| FIC/BIC分子| 差异化平台与技术第一章 ATS 2026 大会数据全景1.1 摘要总体分布本次ATS 2026大会共收录6,820篇摘要，按AI分类统计如下： AI分类篇数占比非干预性研究 2,657 39.0% 其他（含病例报告等） 2,843 41.7% 临床前研究 768 11.3% 遗传学 201 2.9% 临床结果 194 2.8% 二次研究 147 2.2% 其中临床结果类（194篇）是本报告的核心分析对象，涵盖了多项III期关键研究和II期概念验证数据。1.2 疾病领域分布 ATS 2026的疾病领域高度集中于呼吸系统疾病，前十大领域如下：疾病领域摘要数占比呼吸领域 1,521 22.3% 呼吸+罕见疾病 393 5.8% 感染领域 368 5.4% 肿瘤领域 285 4.2% 心脑血管领域 209 3.1% 心脑血管+罕见疾病 171 2.5% 肿瘤+罕见疾病 116 1.7% 神经领域 43 0.6%1.3 药物模态分布在药物形式（Modality）方面，小分子和单克隆抗体仍是两大主流： •小分子药物：775篇（11.4%），涵盖PDE4B、LPAR1、BTK、JAK1等激酶及其他酶类靶点 •单克隆抗体：213篇（3.1%），以抗IL-4Rα、抗TSLP、抗IL-5/IL-5Rα、抗IL-33/IL-33R为主导 •Fc融合蛋白/多肽/ADC：合计约120篇，代表了新一代生物制剂的差异化方向 •基因疗法/细胞疗法：共10篇，仍处于早期探索阶段 •非降解型分子胶：24篇（临床前），代表新兴的药物设计范式第二章 MNC药企临床管线全景本报告追踪了16家主要MNC在ATS 2026上的临床数据展示，共计267篇摘要与MNC相关（含64篇临床结果类摘要）。以下按MNC在ATS 2026上的量级和临床重要性逐一分析。2.1 AstraZeneca（77篇，含24篇临床结果）阿斯利康是ATS 2026上最活跃的MNC，临床数据覆盖哮喘、COPD、IPF、支气管扩张等多个治疗领域，核心看点集中于三大资产：Tezepelumab（特泽利尤单抗，抗TSLP） Tezepelumab是AZ呼吸管线中的旗舰产品，ATS 2026展示了多项重要数据： •SUNRISE III期研究：Tezepelumab显著降低OCS依赖性重度哮喘患者的OCS使用量 •COURSE 2a期：在血嗜酸性粒细胞升高的中重度COPD患者中，Tezepelumab降低COPD相关医疗资源利用率 •NAVIGATOR分析：基因表达调控与嗜酸性粒细胞炎症降低相关，验证了TSLP作为上游靶点的独特定位 •CASCADE功能性呼吸成像：Tezepelumab降低T2-high哮喘患者气道阻力 •超越黏液评分：Tezepelumab改善定量黏液栓塞指标，揭示TSLP通路的全新药效维度Tozorakimab（抗IL-33抗体）作为AZ在IL-33通路的布局，Tozorakimab是差异化竞争的关键资产： •COPD：改善中重度COPD患者定量黏液栓塞指标 •哮喘（FRONTIER-3）：抑制未控制早发性哮喘患者气道内T2和T1炎症通路，显示IL-33靶向可能覆盖更广泛的炎症表型其他关键管线 •AZD8965（口服精氨酸酶抑制剂）：I期数据显示安全性和靶点结合良好，针对肺纤维化开发 •AZD0292（Fc工程化双抗，靶向PcrV/Psl）：针对铜绿假单胞菌定植的支气管扩张症，I期数据 •Londamocitinib（吸入性JAK1抑制剂）：轻度哮喘受试者中验证两种吸入装置等效性 •本瑞利珠单抗（抗IL-5Rα）：HES III期NATRON研究结果+ 功能性呼吸成像数据AZ核心策略总结 AZ在呼吸领域构建了从上游（TSLP/IL-33）到下游（IL-5/IL-5Rα）的完整2型炎症通路布局，并通过口服精氨酸酶抑制剂、Fc工程化双抗等分子拓展至非2型炎症/感染领域。其研发战略呈现「全通路覆盖+给药方式创新」的双轮驱动特征。2.2 Sanofi/Regeneron（48篇，含17篇临床结果）赛诺菲-再生元联盟的核心资产度普利尤单抗（Dupilumab）在ATS 2026上继续释放COPD适应症的深度数据：Dupilumab（度普利尤单抗，抗IL-4Rα） •BOREAS & NOTUS事后分析：Dupilumab改善COPD咳嗽结局、BODE指数和呼吸困难评分（2型炎症人群） •因果中介分析：证实急性加重减少和肺功能改善是患者报告结局改善的主要中介因素 •患者层面数据汇总：扩大范围人群获益验证 •QUEST/TRAVERSE长期随访：Dupilumab使达到临床哮喘缓解的可能性翻倍，长期疗效持续 •EVEREST IV期（头对头Omalizumab）：在合并哮喘的重度CRSwNP伴嗅觉缺失患者中优效 •VESTIGE研究：降低未控制哮喘患者黏液负担Rilzabrutinib（口服BTK抑制剂）赛诺菲在呼吸领域的差异化管线： •II期汇总分析：Rilzabrutinib改善哮喘生活质量和哮喘控制，尤其是在有急性加重史的患者中 •BTK靶点在呼吸疾病的首次临床验证，开辟了非细胞因子通路的治疗新方向下一代免疫调节管线 •Amlitelimab（艾莫特利单抗，抗OX40L）：显著降低哮喘相关血液生物标志物（IgE、IL-13、IL-17A、IL-5、CCL17等），OX40L作为共刺激分子靶点具有上游调控优势 •SAR445399（IL-1RAcP拮抗剂）：皮肤激发模型转化数据支持呼吸疾病开发 •Efdoralprin Alfa（重组A1AT）：II期ElevAATe试验数据显示其对比血浆来源A1PI的差异化优势Sanofi核心策略总结赛诺菲围绕IL-4Rα建立了COPD+哮喘+CRSwNP等多适应症矩阵，同时通过BTK（rilzabrutinib）、OX40L（amlitelimab）、IL-1RAcP（SAR445399）三条差异化通路构建下一代免疫管线。这一策略使赛诺菲在「从2型到非2型、从抗体到小分子」的转型中保持先发优势。2.3 GSK（23篇，含5篇临床结果） GSK以超长效IL-5抗体Depemokimab（德莫奇单抗）为核心亮点：Depemokimab（德莫奇单抗，每半年一次抗IL-5） •SWIFT-1/-2 & AGILE整合分析：2年长期数据显示Depemokimab在2型哮喘中持续疗效，安全性与耐受性良好 •合并CRSwNP的哮喘亚组：长期患者报告结局一致改善 •青少年亚组分析：疗效与成人一致 •每半年一次给药频率有望成为重度哮喘管理的差异化优势，显著改善依从性2.4 Boehringer Ingelheim（26篇，含6篇临床结果）Nerandomilast（那米司特，PDE4B抑制剂）勃林格殷格翰的Nerandomilast是IPF/PPF领域最受关注的在研药物： •FIBRONEER-IPF：按确诊时间/GAP分期分层的亚组分析，验证全人群疗效 •FIBRONEER-ILD：PPF适应症按确诊时间和GAP分期的多个亚组分析 •长期生存获益预测模型：支持Nerandomilast的长期临床价值 •美国IPF/PPF真实世界患者数据分析：验证目标人群特征策略视角 Nerandomilast是FIBRONEER系列III期研究在ATS 2026上的集中展示窗口。若该药获批，将成为IPF领域自吡非尼酮和尼达尼布之后的首个新机制药物（PDE4B选择性抑制，区别于非选择性PDE4抑制剂），且覆盖IPF+PPF双适应症。2.5 Genentech/Roche（46篇，含5篇临床结果）Astegolimab（艾特利单抗，抗IL-33R/ST2）罗氏/基因泰克以Astegolimab为核心，推进IL-33通路在COPD中的应用： •ARNASA III期：Astegolimab在有频繁加重史的COPD患者中的随机、双盲、安慰剂对照试验——这是IL-33R通路在COPD中的首个III期关键数据 •药效学标志物：血清可溶性ST2和血液嗜酸性粒细胞作为Astegolimab的药效学标志物 •SGRQ-C生活质量评估：ALIENTO和ARNASA汇总分析2.6 其他MNC亮点BMS — Admilparant（BMS-986278，LPAR1拮抗剂） •ALOFT-IPF III期试验：基线特征数据公布，是IPF领域LPAR1靶向的首个III期研究 •与尼达尼布/吡非尼酮的DDI评估：无显著相互作用，支持联合用药Johnson & Johnson — 马昔腾坦（ETA/ETB拮抗剂） •UNISUS研究：PAH领域首个头对头优效性研究，CMR和PK/PD子研究设计创新Takeda — Oveporexton（TAK-861，OX2R激动剂） •Radiant Light III期：口服食欲素受体2激动剂治疗1型发作性睡病的有效性和安全性，FIC口服治疗方案Amgen — Fipaxalparant（LPAR1拮抗剂） •IIB期：IPF治疗的有效性、安全性和耐受性数据，与BMS-BMS-986278形成LPAR1双雄竞争格局第三章 MNC押注公司与战略合作方 ATS 2026数据中，我们识别出多家获得MNC战略关注或合作的创新Biotech公司，这些公司的靶点和平台代表了MNC对未来赛道的战略判断。3.1 重点押注公司分析公司核心资产/靶点治疗领域阶段关联MNC Insmed Inc. Brensocatib (DPP-1) 支气管扩张 III期 — Upstream Bio Verekitug (TSLPR) CRSwNP/哮喘 II期 TSLP通路对标AZ Vicore Pharma Buloxibutid (AT2R) IPF II期 — ReAlta Life Sciences Pegtarazimod (补体) AECOPD II期 — Enanta Pharma Zelicapavir (RSV) RSV感染 II期 — Edesa Biotech Paridiprubart (TLR4) ARDS/COVID-19 III期 — GEn1E Lifesciences GEn-1124 (p38α/MK2) 炎症性疾病 I期 — Altesa Biosciences Vapendavir (HRV) COPD/鼻病毒 IIa期 — Inhibikase Therapeutics IkT-001 (伊马替尼前药) PAH/纤维化 I期 — 普沐生物(Pulmongene) PMG1015 (抗AREG) IPF I期中国创新药企 Insilico Medicine AI驱动药物发现平台多领域临床前/I期 — Spark Biopharma SB17170 (HMGB1) IPF IIa期 —3.2 关键投融资信号从ATS 2026数据出发，结合行业公开信息，以下公司和靶点呈现出MNC战略押注的强烈信号： 1.TSLPR靶点（Upstream Bio的Verekitug）：与AZ的TSLP单抗形成直接竞争，但通过靶向TSLP受体可能实现差异化药理学。CRSwNP+哮喘双适应症开发策略值得关注。 2.DPP-1靶点（Insmed的Brensocatib）：ASPEN III期数据已释放，若获批将成为支气管扩张症首个靶向治疗药物，填补巨大未满足需求。 3.AT2R靶点（Vicore的Buloxibutid）：通过激活AT2受体促进肺泡上皮修复和纤维化消退，区别于所有现有IPF疗法的全新机制。 4.补体通路（ReAlta的Pegtarazimod）：AECOPD中降低炎症生物标志物和氧需求，开辟补体系统在COPD急性加重中的新应用。 5.AI制药（Insilico Medicine）：4篇摘要体现了AI驱动药物发现在呼吸领域的早期渗透，代表未来MNC合作/收购的潜在标的。第四章 FIC/BIC分子深度分析 ATS 2026临床结果数据中，我们按靶点新颖度和竞争格局，筛选出以下具有FIC/BIC潜力的分子。4.1 First-in-Class潜力分子分子靶点/机制适应症开发公司 FIC论证 Nerandomilast PDE4B选择性抑制 IPF/PPF Boehringer 首款PDE4B选择性抑制剂 Brensocatib DPP-1抑制剂支气管扩张 Insmed 首个针对支扩的靶向药 Buloxibutid AT2R激动剂 IPF Vicore 首个促修复机制的IPF药物 Oveporexton OX2R激动剂发作性睡病 Takeda 首个口服OX2R激动剂 AZD0292 PcrV/Psl双抗 PA支扩 AstraZeneca 首个Fc工程化PA靶向抗体 Paridiprubart TLR4抗体 ARDS Edesa Biotech 首个ARDS抗TLR4 III期 Amlitelimab OX40L抗体哮喘 Sanofi 首个OX40L靶向哮喘临床数据 Pegtarazimod 补体/炎症多靶点 AECOPD ReAlta 首个补体通路COPD急性加重4.2 Best-in-Class潜力分子分子靶点适应症竞品 BIC优势 Depemokimab IL-5 (半年一次) 重度哮喘/CRSwNP Mepolizumab等超长半衰期，每半年一次给药 Tezepelumab TSLP (最上游) 重度哮喘/COPD — 唯一获批的抗TSLP，T2/T2-low均有效 Dupilumab IL-4Rα COPD/哮喘/CRSwNP/AD — 唯一COPD获批的生物制剂 Admilparant LPAR1 IPF/PPF Fipaxalparant 首个进入III期的LPAR1拮抗剂 Tozorakimab IL-33 COPD/哮喘 Astegolimab 靶向IL-33配体vs受体差异化4.3 中国创新药企FIC/BIC分子 ATS 2026上有数家中国Biotech展示了具有全球竞争力的差异化分子： •普沐生物PMG1015（抗AREG单抗）：全球首个靶向双调蛋白（Amphiregulin）的IPF治疗抗体，靶点新颖度极高 •英矽智能AI平台：4篇摘要展示了AI在新靶点发现和分子设计方面的应用潜力，代表中国AI制药前沿 •先声药业：在呼吸领域持续布局，参与多项国际合作研究第五章差异化平台与技术5.1 药物递送与吸入技术 ATS 2026展示了多个差异化吸入药物和技术平台： •AZ新一代推进剂（HFA-152a替代HFA-134a）：布地奈德/格隆溴铵/福莫特罗三联吸入器的推进剂转换研究，降低碳足迹的同时验证PK/PD等效性 •GSK低碳沙丁胺醇MDI：HFA-152a推进剂的开发和安全耐受性评估 •吸入性西罗莫司（LAM-001）：OrphAI Therapeutics开发的2a期PAH治疗，吸入给药提高肺部靶向性和安全性 •吸入性JAK1抑制剂（Londamocitinib）：AZ的Turbuhaler/Genuair双装置等效性验证 •吸入性PDE3/4抑制剂（KIT2014）：Kither Biotech的首个人体试验，支气管扩张+抗炎双功能 •肺部限制性ALK5抑制剂（AGMB-447）：AgomAb Therapeutics开发，吸入给药减少系统性暴露的差异化策略5.2 新型抗体工程平台 MNC和Biotech在抗体工程方面的创新正在扩展生物制剂的边界： •Fc工程化：AZ的AZD0292采用Fc工程化设计，优化与FcRn的结合以实现延长半衰期或增强效应功能 •超长效抗体平台：GSK的Depemokimab实现每半年一次给药，通过抗体工程改造延长半衰期 •多特异性/Biparatopic抗体：赛诺菲和AZ的管线中多靶点抗体设计趋势明显（如PcrV/Psl双靶点） •非催化性信号调节：GEn-1124作为p38α:MK2双信号调节剂，以非催化性机制区别于传统激酶抑制剂5.3 创新给药与组合策略 ATS 2026中呈现的治疗组合策略和新型给药方式： •前药策略：IkT-001（伊马替尼前药）通过化学修饰改善药代动力学和安全性 •联合治疗：度普利尤单抗与局部糖皮质激素的EVEREST研究展示了生物制剂+标准治疗的新范式 •差异化给药途径：支气管动脉灌注PD-1抑制剂（中国创新方案）、紫杉醇涂层球囊气道应用等 •联合靶向策略：Nerandomilast+尼达尼布、Admilparant+尼达尼布/吡非尼酮的联合用药DDI评估第六章关键疾病领域趋势与竞争格局6.1 特发性肺纤维化（IPF）：新机制竞逐 IPF是ATS 2026竞争最为激烈的赛道之一，呈现「Nerandomilast vs Admilparant vs Fipaxalparant」三雄争霸+ 下一代机制蓄势待发的格局：药物靶点公司阶段差异化 Nerandomilast PDE4B Boehringer III期（FIBRONEER） IPF+PPF双适应症 Admilparant LPAR1 BMS III期（ALOFT）首个LPAR1 III期 Fipaxalparant LPAR1 Amgen IIB期 LPAR1赛道跟随者 Buloxibutid AT2R Vicore II期促修复机制，新范式 AZD8965 精氨酸酶 AstraZeneca I期口服新机制 PMG1015 AREG 普沐生物 I期中国FIC抗体关键洞察：IPF领域正从抗纤维化（尼达尼布/吡非尼酮）的一元化时代，走向PDE4B/LPAR1/AT2R/ARG等多机制并行的多元化格局。BMS和BI的III期数据将决定IPF标准治疗的演进方向。6.2 COPD：生物制剂时代来临 COPD治疗范式正在发生根本性转变，生物制剂从哮喘领域向COPD的大迁移是ATS 2026最重要的主题： •Dupilumab：唯一已获批COPD适应症的生物制剂（FDA 2024年批准），BOREAS/NOTUS事后分析持续强化证据 •Tezepelumab（TSLP）：COURSE研究显示在COPD中的初步疗效信号，适应症拓展潜力巨大 •Astegolimab（IL-33R）：ARNASA III期是COPD中IL-33通路的决定性验证 •Tozorakimab（IL-33）：黏液栓塞改善和双通路炎症抑制的差异化策略 •Pegtarazimod（补体）：AECOPD的全新机制探索6.3 哮喘：从控制到缓解哮喘治疗正在从症状控制向临床缓解（Clinical Remission）的目标演进： •Dupilumab QUEST/TRAVERSE：首次量化临床缓解率，使缓解可能性翻倍 •Tezepelumab SUNRISE：OCS减量/停药是重度哮喘的重要临床终点 •Rilzabrutinib（BTK）：口服小分子靶向非细胞因子通路，可能覆盖T2-low人群 •Amlitelimab（OX40L）：共刺激分子靶向，上游+广谱免疫调节6.4 肺动脉高压（PAH）：联合治疗与头对头竞争 •马昔腾坦UNISUS：PAH首个头对头优效性研究设计，有望重塑治疗格局 •吸入性西罗莫司（LAM-001）：PAH 2a期探索，mTOR通路新方向 •高剂量曲前列尼尔（Treprostinil）：ARTISAN中期分析，早期高强度治疗策略第七章趋势展望与战略建议7.1 ATS 2026十大关键趋势 6.COPD生物制剂时代全面加速：Dupilumab获批后，TSLP/IL-33/IL-33R多条通路竞逐，2026-2028将是COPD生物制剂的密集数据读出窗口 7.IPF新机制元年：PDE4B、LPAR1、AT2R三条新机制集中推进，IPF治疗有望在2027-2029年迎来范式转变 8.2型炎症通路从上游到下游全覆盖竞争：IL-4Rα → TSLP/TSLPR → IL-33/IL-33R → IL-5/IL-5Rα，MNC差异化策略清晰 9.口服小分子回归：BTK（rilzabrutinib）、JAK1（londamocitinib）、PDE4B（nerandomilast）、精氨酸酶（AZD8965）等口服小分子提供生物制剂之外的便利选择 10.非2型炎症通路进入临床验证：BTK、OX40L、补体、TLR4、HMGB1等靶点为T2-low患者提供了新选择 11.吸入给药技术创新和环保推进剂转型：HFA-152a替代HFA-134a成为行业趋势 12.中国创新药企国际化提速：普沐生物、英矽智能等通过高新颖度靶点切入全球竞争 13.支气管扩张症首次出现靶向治疗曙光：Brensocatib填补巨大空白 14.临床缓解（Remission）成为哮喘治疗新目标：从控制到缓解的范式升级 15.AI制药从概念走向临床：Insilico Medicine的多篇摘要标志着AI在呼吸药物研发中的实质性进展7.2 MNC竞争格局展望基于ATS 2026数据，我们对主要MNC在呼吸领域的竞争格局做出以下前瞻判断： MNC 当前优势中期管线(2-4年) 关键风险 AZ TSLP/IL-5Rα/IL-33全通路覆盖，吸入装置优势 Tozorakimab III期、AZD8965 II期 Tezepelumab COPD适应症拓展不确定性 Sanofi Dupilumab多适应症先发优势 Rilzabrutinib/Amlitelimab Dupilumab独占期逼近，后续管线衔接 GSK Depemokimab超长效IL-5差异化 Depemokimab CRSwNP获批 IL-5靶点竞争激烈，需新机制补充 BI IPF领域Nerandomilast III期先发 IPF+PPF双适应症获批 LPAR1竞争和商业化压力 Roche Astegolimab IL-33R COPD III期 ARNASA数据确定性读出与AZ Tozorakimab的IL-33通路竞争 BMS Admilparant LPAR1 III期先发 ALOFT-IPF数据读出 IPF赛道拥挤，需要差异化数据7.3 投资与BD关注方向基于上述分析，我们建议重点关注以下方向： 16.IPF新机制资产：PDE4B、LPAR1已进入III期，AT2R和ARG值得早期关注；关注BI/BMS/Amgen III期数据对后续BD窗口的影响 17.COPD生物制剂差异化：IL-33通路（配体vs受体）、补体通路、黏液靶向等新机制可能创造第二波BD机会 18.非2型炎症靶点：BTK、OX40L、TLR4等靶点的早期临床数据(2026-2028)将定义下一代呼吸免疫药物的BD方向 19.给药技术创新：吸入性生物制剂、超长效抗体、前药技术、肺部靶向递送系统等平台型技术具有跨资产价值 20.中国Biotech出海：普沐生物（AREG）、英矽智能（AI平台）、先声药业等在国际会议上的亮相，预示中国呼吸创新药国际化的加速 21.罕见呼吸疾病：支气管扩张（Brensocatib）、AATD（Efdoralprin Alfa）、LAM（西罗莫司）、发作性睡病（Oveporexton）等小适应症具有高溢价潜力 22.组合疗法策略：IPF领域联合用药（尼达尼布+Nerandomilast/BMS-986278）和哮喘领域生物制剂+ICS/LABA的组合策略可能成为新标准结语 ATS 2026国际会议充分展示了呼吸疾病领域药物研发的深度与广度。从数据中我们可以清晰地看到几个宏大的叙事线索：COPD生物制剂时代从概念走向现实，IPF治疗从单一机制迈入多元化竞争，2型炎症通路竞争从下游向上游延伸，非2型炎症和非细胞因子机制开启新篇章。对于MNC而言，管线深度（全通路覆盖）和差异化（新型给药方式/新机制）是竞争的两大支柱。AstraZeneca和Sanofi凭借全通路布局和先发优势领跑，但GSK（超长效）、BI（PDE4B）、BMS（LPAR1）等MNC以及Upstream Bio、Insmed、Vicore等Biotech也在各自的差异化赛道建立了强大的竞争壁垒。对于中国创新药企而言，ATS 2026数据表明，通过在靶点选择上的差异化（如AREG、AI辅助靶点发现）和创新给药策略（如吸入性抗体/前药），中国Biotech正逐步从跟随者转变为全球呼吸药物研发的重要参与者。未来的12-24个月将是呼吸领域多个关键III期研究的密集数据读出窗口——ARNASA（Astegolimab）、FIBRONEER（Nerandomilast）、ALOFT（Admilparant）等研究的结果将深刻影响赛道格局和M&A/BD动向。建议持续关注这些关键催化和靶点赛道的竞争动态。 ━━━━━━━━━━━━━━━━━━━━━━━━━━━━

2026-06-03

·今日头条

2026呼吸病国际大会：肺高压、肺纤维化、慢阻肺和哮喘治疗新希望

标题：好消息！2026年呼吸病国际大会传来新进展——肺高压、肺纤维化、慢阻肺和哮喘治疗有了新希望今年5月，全球呼吸与危重症医学领域最重要的学术会议——美国胸学会（ATS）年会在奥兰多召开，公布了一批处于III期阶段的呼吸疾病新药数据。我们帮您把专业术语"翻译"成大白话，看看哪些病和您或家人有关。肺动脉高压（PAH）：口服新药有望让病情恶化风险大降目前肺动脉高压的标准治疗常需吃多种药，部分前列环素类药物还得皮下注射或静脉泵入，不太方便。新公布的 Ralinepag（一种口服IP受体激动剂），在标准治疗基础上加用后，使患者临床恶化（如住院或病情明显加重）的风险降低了约55% 。这意味着将来符合条件的患者有望通过每天口服一次药，更好地稳住病情、减少恶化。 ⚠️ 该药尚未在全球多地正式获批用于PAH，仍在等待药监部门审评，具体能否用、怎么用请遵从主治医生判断。特发性肺纤维化（IPF）：吸入新药帮助"保住"更多肺功能 IPF患者最怕肺功能（FVC，用力肺活量）逐年下降。传统抗纤维化药（如吡非尼酮、尼达尼布）能延缓进展，但仍有部分患者下降较快。研究中，吸入型曲前列尼尔（Tyvaso/吸入treprostinil）联合常规抗纤维化治疗，一年下来比安慰剂多"保住"了约 111毫升的肺活量（两项的汇总结果），且临床恶化风险也有降低趋势。这为IPF患者提供了潜在的新联合治疗思路。 ⚠️ 吸入曲前列尼尔用于IPF目前在美国等国家仍属研究性用途/超说明书，国内尚未批准此适应症，需等正式获批后方可常规处方。 MAC肺病（鸟分枝杆菌复合群肺病）：新诊断患者也可用吸入抗菌药以往吸入阿米卡星脂质体（ALIS）多用于难治、复发的MAC肺病。新数据显示，刚确诊的MAC肺病患者，在标准多药治疗基础上加用吸入ALIS ，呼吸症状评分改善更明显，细菌转阴率也更高。提示这类吸入抗感染治疗未来可能前移至初治人群。打呼噜/阻塞性睡眠呼吸暂停（OSA）：首款口服药露头长期靠戴呼吸机（CPAP）但戴不住的患者有盼头。 AD109（一种含兴奋成分的复方口服药）在III期研究中使呼吸暂停低通气指数（AHI）明显下降，约23%患者达到疾病缓解，部分人严重程度降级。若最终获批，将是首个非设备类的OSA口服治疗方案——但仍需更多长期安全及心血管获益证据。慢阻肺（COPD）：生物制剂只适合"挑人用" 研究发现，血嗜酸粒细胞升高的慢阻肺患者，使用美泊利珠单抗（Mepolizumab，IL-5单抗）可减少因急性加重导致的急诊或住院约23%。但另一条路——IL-33/ST2抑制剂（Itepekimab、Astegolimab）在大型III期试验中结果不稳定，未普遍达到显著终点。结论：慢阻肺生物制剂不是人人能用，验血挑出嗜酸粒细胞升高的人群才更可能受益。 ️ 重症哮喘：目标从"少犯病"升级为"减激素+少打针" 替泽帕单抗（Tezepelumab）：帮助长期依赖口服激素的重症哮喘患者，在不失控的前提下减少激素用量，降低激素副作用风险。长效IL-5抑制剂（Depemokimab）：尝试半年打一针（而非每2～4周一针），虽未完全证明非劣效，但提示未来给药间隔延长的方向。国产双抗（信达IBI3002，IL-4Rα/TSLP双靶点）：早期数据显示可改善肺功能及炎症指标，尚处早期阶段，值得继续关注。给患者朋友的话以上均为最新临床研究数据，多数药物仍在等待监管机构正式批准或指南更新，距门诊常规开方还需要时间。如果您或家人患有上述疾病： ✅ 坚持规范用药、定期随访复查肺功能/心脏超声 ✅ 有疑问可咨询主治医生是否适合参加正规临床试验 ❌ 切勿自行购药或擅自更改当前治疗方案我们会持续跟踪这些新药在国内外的审批动态，第一时间告诉您！本文仅供健康科普，不能替代执业医师的面对面诊疗建议。往期科普：

100 项与曲前列尼尔相关的药物交易

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外链

KEGG	Wiki	ATC	Drug Bank
D06213 D08628	曲前列尼尔	B01AC21	DB00374

研发状态

批准上市

10 条最早获批的记录,

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适应症	国家/地区	公司	日期
肺性高血压	美国	United Therapeutics Corp.	2022-05-23
间质性肺疾病	美国	United Therapeutics Corp.	2021-03-31
慢性血栓栓塞性肺动脉高压	欧盟	SciPharm SARL	2020-04-03
慢性血栓栓塞性肺动脉高压	冰岛	SciPharm SARL	2020-04-03
慢性血栓栓塞性肺动脉高压	列支敦士登	SciPharm SARL	2020-04-03
慢性血栓栓塞性肺动脉高压	挪威	SciPharm SARL	2020-04-03
肺动脉高压	美国	United Therapeutics Corp.	2002-05-21

未上市

10 条进展最快的记录,

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适应症	最高研发状态	国家/地区	公司	日期
间质性肺病导致的肺动脉高压	申请上市	美国	Liquidia Corp.	2024-08-19
进行性纤维化间质性肺病	临床3期	美国	United Therapeutics Corp.	2023-10-30
进行性纤维化间质性肺病	临床3期	阿根廷	United Therapeutics Corp.	2023-10-30
进行性纤维化间质性肺病	临床3期	澳大利亚	United Therapeutics Corp.	2023-10-30
进行性纤维化间质性肺病	临床3期	比利时	United Therapeutics Corp.	2023-10-30
进行性纤维化间质性肺病	临床3期	加拿大	United Therapeutics Corp.	2023-10-30
进行性纤维化间质性肺病	临床3期	智利	United Therapeutics Corp.	2023-10-30
进行性纤维化间质性肺病	临床3期	法国	United Therapeutics Corp.	2023-10-30
进行性纤维化间质性肺病	临床3期	德国	United Therapeutics Corp.	2023-10-30
进行性纤维化间质性肺病	临床3期	以色列	United Therapeutics Corp.	2023-10-30

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临床结果

适应症

分期

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研究	分期	人群特征	评价人数	分组	结果	评价	发布日期


NCT00325442 \| NCT00887978 (FREEDOM-C, Pubmed \| Adv Ther)	临床3期	肺动脉高压	156	Oral Treprostinil + Background ERA and/or PDE-5i (Background monotherapy)	艱齋夢範構廠築築顧鬱(鬱獵獵獵齋繭網顧憲簾) = 選築壓鬱簾憲網鏇製遞膚餘鬱構衊淵襯構積齋 (窪獵齋憲鏇憲簾膚廠壓 )	积极	2026-02-02
				Oral Treprostinil + Background ERA and/or PDE-5i	艱齋夢範構廠築築顧鬱(鬱獵獵獵齋繭網顧憲簾) = 廠觸廠鹽憲廠醖鑰艱獵膚餘鬱構衊淵襯構積齋 (窪獵齋憲鏇憲簾膚廠壓 )
NCT05564637 (CTgov)	临床2期	间质性肺病导致的肺动脉高压	29	Right Heart Catheterization (RHC) while exercising+Treprostinil (PAH-ILD Patients)	餘窪衊構製選願觸夢觸(壓鹹簾襯鑰壓構餘襯鬱) = 壓鏇齋齋顧襯襯衊壓獵廠襯蓋夢糧窪憲範鬱窪 (鑰選觸願鑰遞鑰鏇夢鏇, 2.1) 更多	-	2025-12-03
				Right Heart Catheterization (RHC) while exercising (Healthy Volunteers)	繭醖遞遞窪鹹醖蓋蓋鹽(構艱構膚獵築網醖鏇繭) = 齋廠選願醖遞願壓糧願網鹹繭齋鹽遞糧積壓醖 (醖獵衊願繭艱鏇窪製製, 範築製憲膚繭範衊鹽醖 ~ 範餘構淵獵齋繭製餘鹹) 更多
NCT05255991 (TETON-2, Company_Website)	临床3期	特发性肺纤维化	597	Treprostinil	壓齋窪壓廠繭餘鬱築製(獵襯夢願遞觸網積觸積) = Tyvaso demonstrated superiority over placebo for the change in absolute FVC by 95.6 mL (Hodges-Lehmann estimate, p <0.0001) from baseline to week 52 in patients with IPF. 積艱鹽簾膚網獵願範襯 (膚構範積製憲鹹夢積齋 ) 达到	积极	2025-09-02
				placebo
NCT06129240 (ASCENT, ATS2025)	N/A	间质性肺疾病	13	LIQ861	壓餘繭觸壓蓋遞繭糧選(製願簾製衊願膚範淵鏇) = 鬱淵範鬱廠壓遞簾獵築簾餘醖夢遞夢鹽獵鏇觸 (繭繭鬱淵範遞範鑰窪齋 ) 更多	积极	2025-05-16
ATS2025	N/A	-	-	Treprostinil	鹹鏇網遞膚夢顧繭選網(願憲壓獵淵廠構淵鏇夢) = 餘鑰選淵襯鏇觸願鬱網壓醖鹽選構築淵觸繭淵 (選膚獵窪獵餘築鏇餘齋 )	-	2025-05-16
NCT06129240 (ASCENT, ATS2025)	N/A	间质性肺病导致的肺动脉高压	12	LIQ861	艱膚遞鑰夢獵繭壓膚鹽(鏇醖夢範艱蓋蓋觸獵醖) = 醖齋鬱獵醖網鏇範窪網觸蓋繭構夢簾願構選願 (窪鹹醖顧夢夢鹽構夢觸, 164 ~ 448) 更多	积极	2025-05-16
				Placebo	艱膚遞鑰夢獵繭壓膚鹽(鏇醖夢範艱蓋蓋觸獵醖) = 鑰鏇壓衊襯糧壓製遞襯觸蓋繭構夢簾願構選願 (窪鹹醖顧夢夢鹽構夢觸, 148 ~ 420) 更多
FREEDOM-EV (ATS2025)	N/A	肺动脉高压 NT-proBNP	153	Oral Treprostinil	齋鹹醖窪製夢齋衊積顧(築鹹窪願夢夢窪鏇廠積) = 蓋製鹽壓觸廠築醖觸觸膚憲窪構壓鏇鹽簾觸簾 (窪蓋糧淵願憲鏇衊顧觸 )	积极	2025-05-16
				Placebo	齋鹹醖窪製夢齋衊積顧(築鹹窪願夢夢窪鏇廠積) = 積簾鹽構願夢選遞夢範膚憲窪構壓鏇鹽簾觸簾 (窪蓋糧淵願憲鏇衊顧觸 )
NCT03950739 (BREEZE Optional Extension \| BREEZE, CTgov)	临床1期	肺动脉高压	51	Treprostinil Inhalation Powder	憲壓製襯願遞顧構鬱衊(淵艱醖窪選糧積鏇齋憲) = 築鏇窪鑰齋膚齋艱壓鹽壓艱鬱積鑰鹹襯積積糧 (鑰衊顧範鬱繭憲鹽淵顧, 32.9) 更多	-	2024-11-01
ESC2024	N/A	肺动脉高压	-	Treprostinil	窪淵構膚遞獵夢夢範鬱(鑰鏇鹹醖艱窪遞餘鹽積) = 夢願餘積夢齋壓窪齋鬱窪觸鹽選壓壓壓艱餘獵 (遞範鹹廠製醖構壓網鹹, 10.42 ~ 19.90)	-	2024-09-02
				Placebo	-
NCT03399604 (INSPIRE, CTgov)	临床3期	特发性肺动脉高压	121	LIQ861 Inhaled Treprostinil	選鏇範範蓋範鏇積構積 = 醖衊餘夢遞鏇窪鹽選艱繭鬱願鹽簾壓範鏇壓鹹 (餘鑰鹹鏇簾窪衊夢蓋糧, 餘壓構繭鹹膚繭積繭範 ~ 鑰糧構醖鏇鏇夢衊繭齋)	-	2024-07-30