A Phase 3, Multicenter, Randomized, Double-Blinded, Placebo-Controlled Study to Evaluate the Safety and Efficacy of L606 (Treprostinil Liposome Inhalation Suspension) in Participants With Pulmonary Hypertension Associated With Interstitial Lung Disease (WHO Group 3)

The purpose of this study is to find out if L606 is safe and if it helps people with high blood pressure in the lungs (pulmonary hypertension) caused by interstitial lung disease (PH-ILD, WHO Group 3). One of the main ways the study will check this is by seeing if people can walk further in six minutes (called the "six-minute walk test"). Another important (secondary) goal is to see how long it takes for PH-ILD to get worse while people are taking L606. One way the study will measure this is by looking at the distance people walk at different times in the study on different doses of L606. Throughout the study, doctors will also closely monitor for any side effects people have to make sure L606 is safe. People who decide to join this study will be randomly (like tossing a coin) placed into one of two groups: one group will get L606 and the other group will get a placebo, which will look like L606 but will not contain any active medicine. At the end of the initial blinded portion of the study, study participants may have the opportunity to enroll in an open-label extension portion of the study where all participants will receive L606.

开始日期2026-04-23

申办/合作机构

Liquidia Technologies, Inc.

NCT04691154

/ Active, not recruiting临床3期

A Phase 3, 2-Part, Open-Label Study to Evaluate the Safety and Tolerability of Liposomal Treprostinil Inhalation Suspension (L606) in Subjects With Pulmonary Arterial Hypertension or Pulmonary Hypertension Associated With Interstitial Lung Disease

This Phase 3, 2-part, open-label, multicenter study aims to demonstrate the safety and tolerability of L606 in patients with PAH or PH-ILD. The study will determine the short-term and long-term safety and tolerability of L606 in this patient population.

开始日期2021-08-01

申办/合作机构

Liquidia Technologies, Inc.

[+1]

NCT04041648

/ Completed临床1期

A Phase I, Randomized, Double-blind, Placebo-controlled Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of Single Ascending Dose of L606 for Inhalation in Healthy Subjects

The primary objective of this study is to evaluate the Pharmacokinetics, Safety and Tolerability of L606 (Liposomal Treprostinil) Inhalation Solution in Single Ascending Dose study design in healthy volunteers.

开始日期2018-11-09

申办/合作机构

国邑药品科技股份有限公司

[+1]

100 项与曲前列尼尔脂质体相关的临床结果

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100 项与曲前列尼尔脂质体相关的转化医学

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100 项与曲前列尼尔脂质体相关的专利（医药）

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项与曲前列尼尔脂质体相关的文献（医药）

2010-01-01·Ontario health technology assessment series

Clinical utility of vitamin d testing: an evidence-based analysis.

Article

CLINICAL NEED:

CONDITION AND TARGET POPULATION Vitamin D deficiency may lead to rickets in infants and osteomalacia in adults. Factors believed to be associated with vitamin D deficiency include: darker skin pigmentation,winter season,living at higher latitudes,skin coverage,kidney disease,malabsorption syndromes such as Crohn's disease, cystic fibrosis, andgenetic factors.Patients with chronic kidney disease (CKD) are at a higher risk of vitamin D deficiency due to either renal losses or decreased synthesis of 1,25-dihydroxyvitamin D. Health Canada currently recommends that, until the daily recommended intakes (DRI) for vitamin D are updated, Canada's Food Guide (Eating Well with Canada's Food Guide) should be followed with respect to vitamin D intake. Issued in 2007, the Guide recommends that Canadians consume two cups (500 ml) of fortified milk or fortified soy beverages daily in order to obtain a daily intake of 200 IU. In addition, men and women over the age of 50 should take 400 IU of vitamin D supplements daily. Additional recommendations were made for breastfed infants. A Canadian survey evaluated the median vitamin D intake derived from diet alone (excluding supplements) among 35,000 Canadians, 10,900 of which were from Ontario. Among Ontarian males ages 9 and up, the median daily dietary vitamin D intake ranged between 196 IU and 272 IU per day. Among females, it varied from 152 IU to 196 IU per day. In boys and girls ages 1 to 3, the median daily dietary vitamin D intake was 248 IU, while among those 4 to 8 years it was 224 IU. VITAMIN D TESTING: Two laboratory tests for vitamin D are available, 25-hydroxy vitamin D, referred to as 25(OH)D, and 1,25-dihydroxyvitamin D. Vitamin D status is assessed by measuring the serum 25(OH)D levels, which can be assayed using radioimmunoassays, competitive protein-binding assays (CPBA), high pressure liquid chromatography (HPLC), and liquid chromatography-tandem mass spectrometry (LC-MS/MS). These may yield different results with inter-assay variation reaching up to 25% (at lower serum levels) and intra-assay variation reaching 10%. The optimal serum concentration of vitamin D has not been established and it may change across different stages of life. Similarly, there is currently no consensus on target serum vitamin D levels. There does, however, appear to be a consensus on the definition of vitamin D deficiency at 25(OH)D < 25 nmol/l, which is based on the risk of diseases such as rickets and osteomalacia. Higher target serum levels have also been proposed based on subclinical endpoints such as parathyroid hormone (PTH). Therefore, in this report, two conservative target serum levels have been adopted, 25 nmol/L (based on the risk of rickets and osteomalacia), and 40 to 50 nmol/L (based on vitamin D's interaction with PTH). ONTARIO CONTEXT: VOLUME #ENTITYSTARTX00026; COST: The volume of vitamin D tests done in Ontario has been increasing over the past 5 years with a steep increase of 169,000 tests in 2007 to more than 393,400 tests in 2008. The number of tests continues to rise with the projected number of tests for 2009 exceeding 731,000. According to the Ontario Schedule of Benefits, the billing cost of each test is $51.7 for 25(OH)D (L606, 100 LMS units, $0.517/unit) and $77.6 for 1,25-dihydroxyvitamin D (L605, 150 LMS units, $0.517/unit). Province wide, the total annual cost of vitamin D testing has increased from approximately $1.7M in 2004 to over $21.0M in 2008. The projected annual cost for 2009 is approximately $38.8M. EVIDENCE-BASED ANALYSIS: The objective of this report is to evaluate the clinical utility of vitamin D testing in the average risk population and in those with kidney disease. As a separate analysis, the report also sought to evaluate the prevalence of vitamin D deficiency in Canada. The specific research questions addressed were thus: What is the clinical utility of vitamin D testing in the average risk population and in subjects with kidney disease?What is the prevalence of vitamin D deficiency in the average risk population in Canada?What is the prevalence of vitamin D deficiency in patients with kidney disease in Canada?Clinical utility was defined as the ability to improve bone health outcomes with the focus on the average risk population (excluding those with osteoporosis) and patients with kidney disease.

LITERATURE SEARCH:

A literature search was performed on July 17th, 2009 using OVID MEDLINE, MEDLINE In-Process and Other Non-Indexed Citations, EMBASE, the Cumulative Index to Nursing & Allied Health Literature (CINAHL), the Cochrane Library, and the International Agency for Health Technology Assessment (INAHTA) for studies published from January 1, 1998 until July 17th, 2009. Abstracts were reviewed by a single reviewer and, for those studies meeting the eligibility criteria, full-text articles were obtained. Reference lists were also examined for any additional relevant studies not identified through the search. Articles with unknown eligibility were reviewed with a second clinical epidemiologist, then a group of epidemiologists until consensus was established. The quality of evidence was assessed as high, moderate, low or very low according to GRADE methodology. Observational studies that evaluated the prevalence of vitamin D deficiency in Canada in the population of interest were included based on the inclusion and exclusion criteria listed below. The baseline values were used in this report in the case of interventional studies that evaluated the effect of vitamin D intake on serum levels. Studies published in grey literature were included if no studies published in the peer-reviewed literature were identified for specific outcomes or subgroups. Considering that vitamin D status may be affected by factors such as latitude, sun exposure, food fortification, among others, the search focused on prevalence studies published in Canada. In cases where no Canadian prevalence studies were identified, the decision was made to include studies from the United States, given the similar policies in vitamin D food fortification and recommended daily intake.

INCLUSION CRITERIA:

Studies published in EnglishPublications that reported the prevalence of vitamin D deficiency in CanadaStudies that included subjects from the general population or with kidney diseaseStudies in children or adultsStudies published between January 1998 and July 17(th) 2009 EXCLUSION CRITERIA: Studies that included subjects defined according to a specific disease other than kidney diseaseLetters, comments, and editorialsStudies that measured the serum vitamin D levels but did not report the percentage of subjects with serum levels below a given threshold

OUTCOMES OF INTEREST:

Prevalence of serum vitamin D less than 25 nmol/LPrevalence of serum vitamin D less than 40 to 50 nmol/LSerum 25-hydroxyvitamin D was the metabolite used to assess vitamin D status. Results from adult and children studies were reported separately. Subgroup analyses according to factors that affect serum vitamin D levels (e.g., seasonal effects, skin pigmentation, and vitamin D intake) were reported if enough information was provided in the studies

QUALITY OF EVIDENCE:

The quality of the prevalence studies was based on the method of subject recruitment and sampling, possibility of selection bias, and generalizability to the source population. The overall quality of the trials was examined according to the GRADE Working Group criteria. (ABSTRACT TRUNCATED)

项与曲前列尼尔脂质体相关的新闻（医药）

2026-01-30

·哽啾糖水铺

JPM速递：慢阻肺（COPD）新药研发烽烟四起，谁将主导下一个十年？

2026年1月12日至15日，第44届J.P.摩根医疗健康大会（JPM 2026）在美国旧金山举行；作为全球生物医药产业的风向标，本届大会中包含了诸多COPD相关的研发趋势引言：为何慢阻肺（COPD）成为全球新药研发的必争之地？慢性阻塞性肺疾病（COPD），作为一种常见的呼吸系统疾病，正给全球医疗系统带来沉重的负担。仅在美国，就有超过1600万成年人受其影响，其中约41%的患者因病情加重需要住院治疗。然而，与高发病率形成鲜明对比的是，现有治疗手段正面临严峻瓶颈。数据显示，约85%的COPD患者在一线治疗中未能获得理想改善，且在急性发作后四周内，治疗失败率高达50%。巨大的未满足需求与清晰的临床痛点，正吸引全球顶尖药企以前所未有的力度布局COPD赛道。本文将深度解析其最新的研发管线与战略动向，揭示主导下一个十年的核心趋势与关键玩家。1. 破局者登场：二十年沉寂后，吸入疗法迎来首个新机制吸入式维持治疗是COPD管理的基石，但令人惊讶的是，这一核心领域已有二十余年未曾迎来任何真正意义上的新机制药物。长期的沉寂，终于被默沙东的重磅收购所打破，标志着一个创新时代的重启。默沙东 (Merck & Co.) - OHTUVAYRE (ensifentrine) 默沙东通过收购Verona Pharma，将一款首创的PDE3/PDE4双重抑制剂——OHTUVAYRE (ensifentrine)——收入囊中。此举不仅填补了市场的长期空白，更被默沙东视为一个“数十亿美元的商业机会”。作为20多年来首个新型吸入式维持治疗药物，其商业化表现被寄予厚望，有望成为驱动公司2025年增长的强劲引擎。除了对传统维持疗法的革新，生物制剂的入局正在从另一个维度——精准靶向炎症，来改变COPD的治疗格局。图来源于JPM默沙东ppt 2. 生物制剂的精准打击：靶向炎症通路，开辟治疗新维度 COPD远非单纯的气道阻塞，其背后复杂的炎症机制才是疾病迁延与恶化的关键。近年来，靶向特定炎症通路的生物制剂已成为研发热点，它们代表了COPD治疗从“对症”到“对因”的重大转变，开启了个体化治疗的新篇章。赛诺菲 (Sanofi) & 再生元 (Regeneron) - 度普利尤单抗 (Dupixent) 与 Itepekimab 作为该领域的先行者，赛诺菲与再生元凭借其明星产品度普利尤单抗 (Dupixent)取得了里程碑式的突破。该药已在美国和欧盟获批，成为首个获批用于此类慢性炎症疾病的生物制剂，为特定炎症表型的COPD患者带来了新希望。在此基础上，其后续管线Itepekimab (抗IL-33抗体)也已进入3期临床阶段。尽管在其两项关键研究中仅有一项成功，暴露出其临床路径的不确定性，但这依然体现了赛诺菲/再生元在IL-33这一前沿靶点上的探索决心与战略纵深。 Connect Biopharma - Rademikibart Connect Biopharma则将目光瞄准了急性COPD加重这一关键场景，其在研的抗IL-4Rα单抗Rademikibart显示出独特的竞争优势。临床节奏：其针对急性COPD加重的2期研究已于2025年第二季度启动，预计2026年中期将公布顶线数据。技术优势：临床前研究结果尤为亮眼，显示Rademikibart在改善对β-受体激动剂的反应性方面显著优于度普利尤单抗，并具备更快的起效潜力。安进 (Amgen) - TEZSPIRE 生物医药巨头安进同样未缺席这场竞赛。其重磅药物TEZSPIRE已被列入针对COPD的3期研发项目，凭借其靶向上游炎症因子TSLP的独特机制，有望在更广泛的COPD患者群体中发挥作用，体现了安进在呼吸领域的战略雄心。整体来看，生物制剂赛道呈现出“先驱占位、新秀追赶、巨头入局”的竞争格局。赛诺菲/再生元的Dupixent凭借先发优势已抢占市场，但Connect Biopharma的Rademikibart在临床前数据上展现了差异化优势，而安进TEZSPIRE则可能凭借更上游的靶点覆盖更广泛患者群体。未来的竞争焦点将不仅在于疗效，更在于对特定炎症表型患者的精准识别与市场教育。尽管生物制剂展现了强大的潜力，但其注射给药的方式也催生了对更便捷疗法的追求，比如口服药物和更高效的吸入技术。 3. 便利与精专：口服药与新剂型带来的想象空间在追求疗效最大化的同时，提升患者用药的便利性，以及针对特殊患者群体开发“精专化”治疗方案，正成为当前COPD药物研发的另外两大趋势。 Kymera Therapeutics - 口服STAT6降解剂 KT-621 Kymera Therapeutics另辟蹊径，利用其前沿的蛋白质降解平台，开发了一款口服STAT6降解剂 KT-621。STAT6是IL-4/IL-13介导的2型炎症通路中的关键转录因子。KT-621的核心价值在于，它“旨在提供类似生物制剂的疗效，同时具备每日一次口服的便利性”。这款口服药物所瞄准的STAT6相关呼吸系统适应症（包括COPD和哮喘）的潜在患者群体高达3400万人，其市场潜力不言而喻。 Liquidia Technologies - 聚焦PH-COPD的特异性疗法 Liquidia Technologies则选择了更为精专的赛道，利用其独特的PRINT®技术，重点改善伴有肺高压的COPD患者（PH-COPD）的药物递送。这是一个长期被忽视但需求迫切的细分市场。核心产品：其产品管线包括 YUTREPIA 和 L606。临床计划：公司计划于2027年启动针对PH-COPD的3期临床研究。市场潜力：据估计，PH-COPD患病人数超过30万，市场规模预计超过50亿美元，凸显了其在细分赛道中的巨大价值。通过聚焦PH-COPD这一服务不足的“利基市场”，Liquidia采取了典型的差异化竞争策略，旨在避开主流COPD疗法的激烈竞争，建立起稳固的商业护城河。从新机制吸入剂、靶向生物药到口服小分子，COPD的研发管线呈现出前所未有的活力。 4. 总结与展望：COPD治疗的未来图景回顾全球COPD的研发管线，我们可以清晰地看到三大核心趋势正在重塑未来的治疗格局：机制创新：研发重点已从传统的支气管扩张剂，转向了如PDE3/PDE4双重抑制剂、STAT6降解剂等全新作用机制，旨在从更根本的层面干预疾病进程。精准靶向：生物制剂的全面入局，通过靶向IL-4Rα、IL-33、TSLP等关键炎症通路，正引领COPD治疗进入精准化、个体化的新时代。剂型优化与人群细分：口服疗法带来的便利性，与针对PH-COPD等特殊亚群的特异性疗法，共同提升了治疗方案的深度与广度，更好地满足了多样化的临床需求。面对COPD领域巨大的未满足需求，全球药企的积极布局与持续创新，预示着一个治疗突破的黄金时代即将到来。随着这些创新疗法的陆续获批，未来的COPD患者将拥有更多、更有效、更便捷的治疗选择，从而有望从根本上改变这一慢性疾病的管理面貌。后台私信获取JPM2026各公司ppt

2024-10-03

·Endpoints

FDA lifts partial hold on Avidity’s myotonic dystrophy drug; Orum's IPO in South Korea

Plus, news about Caliway Pharmaceuticals, argenx, Halozyme Therapeutics, Verrica, Liquidia and Pharmosa: FDA lifts partial clinical hold on Avidity Biosciences’ drug: The treatment , called delpacibart etedesiran, is in the Phase 3 HARBOR trial in patients with myotonic dystrophy type 1, a progressive and often fatal neuromuscular disease with no approved therapies. The FDA first handed down a partial clinical hold in 2022, following a serious adverse event in an earlier-stage study. Avidity’s stock $RNA rose about 3% on Thursday. — Katherine Lewin Orum Therapeutics outlines IPO in South Korea: The biotech plans to list on the KOSDAQ market to help fund its protein degrader at a time when antibody-drug conjugates are still hot. Orum is seeking between $67 million to $80 million in the IPO. Earlier this year, it partnered with Vertex , and last year it sold an asset to Bristol Myers Squibb. — Kyle LaHucik Caliway Biopharmaceuticals also has IPO plans, in Taiwan: The biotech uplisted to the Taipei Exchange and raised about $206 million, or NT $6.4 billion, which it described as the “largest IPO in Taiwan’s biotech industry history.” The company expects to have Phase 2b results by the end of the year for a subcutaneous fat reduction injectable. It also has preclinical injections for hyperpigmentation and osteoarthritis. — Kyle LaHucik Argenx picks four new targets per deal with Halozyme Therapeutics: The company is using Halozyme’s ENHANZE drug delivery technology to pick a total of six targets. It will now pay Halozyme $30 million upfront for the exclusive rights to the four new targets. Argenx could also pay up to $85 million per target for milestone payments, regulatory approvals, and sales. Halozyme’s stock $HALO gained about 2% in premarket trading on Thursday. — Katherine Lewin Verrica slashes commercial team headcount: The West Chester, PA-based biotech said it cut its workforce by 47 employees in a bid to “streamline operations, reduce costs and preserve capital.” The layoffs are part of the company’s broader push to trim its commercial organization, which includes focusing on 35 sales territories, down from an original 80. It had 100 full-time employees as of Dec. 31. — Ayisha Sharma Liquidia expands drug licensing deal with Pharmosa: Under the terms of the updated pact , Liquidia will get the rights to Pharmosa’s pulmonary arterial hypertension candidate, L606, in markets outside of North America, including Japan and parts of Europe. Liquidia is set to pay Pharmosa $3.5 million upfront and up to $157.75 million in milestones in the new territories. — Ayisha Sharma

引进/卖出临床3期IPO

2024-07-04

·药融圈

独特吸入技术傍身，仿制与创新并进，这家biotech不一般

▲8月15-16日 NDC2024生物医药创新者峰会扫码报名注：本文不构成任何投资意见和建议，以官方/公司公告为准；本文仅作医疗健康相关药物介绍，非治疗方案推荐（若涉及），不代表平台立场。任何文章转载需得到授权。肺动脉高压（PAH）是一种罕见且危及生命的慢性进行性疾病，其会导致肺动脉压升高和局部高血压，从而导致心力衰竭并最终死亡。Mordor Intelligence预测，2024年到2029年肺动脉高压市场的年均复合增长率为5.0%，由于医疗保健行业高度发达，以及传染病和慢性病患病率不断上升，北美在肺动脉高压市场中处于领先地位。肺动脉高压的治疗可以通过使用前列环素和前列环素类似物、钙通道阻滞剂、磷酸二酯酶5、内皮素受体拮抗剂等药物来治疗，前列环素及其类似物是最早用于PAH的靶向治疗药物。曲前列尼尔是由UnitedTherapeutics研发的一款人工合成前列环素药品，可以促进血管舒张，同时可抑制血小板的聚集，用于肺动脉高压的症状治疗。2005年，曲前列尼尔注射液获得FDA批准上市，商品名为Remodulin（瑞莫杜林），但其零售价极高，曾一度成为美国药房零售价最贵的20个药物之一。2019年3月，Sandoz宣布FDA批准其曲前列尼尔注射液上市，可完全替代Remodulin，同时还宣布其将与RareGen公司合作进行曲前列尼尔注射液的商业推广和销售。图源：Mordor Intelligence 从药融云数据库获悉，主要为罕见病药品提供商业化服务的RareGen于2020年被Liquidia Technologies收购，Liquidia Technologies是2004年由Joe DeSimone和Ed Samulski从北卡罗来纳大学教堂山分校分离出来的，并于当年在特拉华州注册成立，随着上述合并交易的完成，以上的两家公司均成为Liquidia Corporation的全资运营子公司。目前的Liquidia Corporation（以下简称Liquidia）是一家生物制药公司，主要专注于肺动脉高压产品的开发以及PRINT技术的其他应用，通过两家全资子公司Liquidia Technologies和Liquidia PAH作为单一实体运营。其中，Liquidia Technologies主要负责肺动脉高压相关产品的开发，Liquidia PAH负责治疗肺部疾病药品的商业化。药融云数据库：公司信息 Liquidia专有的PRINT颗粒工程技术使其能够设计和制造高度均匀的药物颗粒，并对颗粒的尺寸、三维几何形状和化学成分进行精确控制，通过控制颗粒的这些物理和化学参数，PRINT能够将所需的药理学益处设计到开发产品中，包括延长药物释放的持续时间、增加药物负载、设计更方便的给药途径、创造新的组合产品的能力、增强储存和稳定性以及减少副作用的潜力。专攻PAH和PH-ILD Liquidia旗下Liquidia PAH主要负责仿制药曲前列尼尔注射液的商业化，Liquidia Technologies负责治疗肺动脉高压（PAH）和间质性肺病相关肺动脉高压（PH-ILD）相关疾病的产品研发，肺动脉高压市场的主要领导者有拜耳、吉利德、强生、辉瑞和United Therapeutics等。当前公司管线的具体进展情况如下：一仿制药曲前列尼尔注射液（仿制药）通过收购RareGen获得，由Liquidia PAH与Sandoz合作进行曲前列尼尔注射液的商业化。2019年，Sandoz宣布FDA批准曲前列尼尔注射液上市，用于治疗肺动脉高压，可完全替代United Therapeutics公司2002年获批上市的原研药Remodulin。截图来自：国家药品监督管理局 2010年7月，李氏大药厂与United Therapeutics公司达成独家协议，获得Remodulin在中国的独家分销代理权和定价许可权。2020年，李氏大药厂子公司兆科药业按新4类申报的曲前列尼尔注射液获得国家药监局批准上市，视同通过一致性评价。2023年3月，江苏众强药业有限公司的曲前列尼尔注射液仿制药获批上市，同时也是继兆科药业之后获批的国产第二家曲前列尼尔注射液企业。二在研管线 1、YUTREPIA（曲前列尼尔吸入粉剂） YUTREPIA是一种研究性吸入干粉制剂，使用Liquidia的PRINT技术设计，通过方便、省力、手掌大小的装置给药。2021年11月，FDA发布了YUTREPIA用于PAH患者的暂定批准。2023年7月，Liquidia提交了对其YUTREPIA新药申请的修正案，寻求在标签中添加PH-ILD适应症。 Liquidia在505(b)(2)监管途径下开发了YUTREPIA，使用雾化形式的曲前列尼尔（Tyvaso，美国唯一一个被批准治疗PH-ILD的疗法）作为参考上市药物。2020年1月Liquidia提交了YUTREPIA的新药上市申请，FDA分别于2021年8月和2021年10月对公司的两家美国生产工厂进行了现场批准前检查，2021年11月，FDA发布了YUTREPIA的初步批准，表明该NDA已满足最终批准的所有要求，但尚不能上市。2023年7月，Liquidia提交了NDA修正案，将PH-ILD添加到YUTREPIA的标签中。FDA的最终批准和YUTREPIA的推出直接受到United Therapeutics于2020年6月4日提起的Hatch-Waxman诉讼以及United Therapeutics授予PH-ILD的监管排他性（于2024年3月31日到期）的影响。2024年3月下旬，法官撤销了在最初的Hatch-Waxman诉讼中发布的禁令，该禁令阻止FDA批准YUTREPIA的新药上市申请，截止2024年4月1日，没有进一步的法律阻碍FDA批准YUTREPIA用于PAH和PH-ILD。 Liquidia提交的新药上市申请基于已经完成的INSPIRE试验，即曲前列尼尔干粉吸入的安全性和药理学研究，这是一项开放标签、多中心临床试验3期研究，用于在未吸入曲前列尼尔或正在从Tyvaso（雾化曲前列尼尔）过渡的PAH患者，该试验的主要目的是评估YUTEREPIA的长期安全性，主要终点是评估第2个月的安全性和耐受性。目前正在进行ASCENT试验，这是一项开放标签前瞻性多中心研究，旨在评估干粉吸入曲前列尼尔治疗肺动脉高压的安全性和耐受性，目的是告知YUTEREPIA在PH-ILD患者中的剂量和耐受性特征。 2、L606（脂质体曲前列尼尔）吸入混悬液 L606混悬液是一种吸入式、持续释放的研究性曲前列尼尔缓释制剂，使用Pharmosa Biopharm专有的脂质体配方来封装曲前列尼尔，曲前列尼尔可以以受控速率缓慢释放到肺部，在很长一段时间内增强药物暴露，并减轻对上呼吸道的局部刺激。药融云数据库：药品信息 2023年6月28日，Liquidia与Pharmosa Biopharm宣布已就L606在北美的开发和商业化达成一项独家许可协议，根据协议条款，Liquidia将负责L606在北美的开发、监管和商业活动，Pharmosa Biopharm将生产L606的临床和商业用品，并支持Liquidia建立一个全球供应链。作为这些独家权利的对价，Liquidia将向Pharmosa Biopharm支付1000万美元预付款，与PAH和PH-ILD适应症相关的高达2.15亿美元的潜在开发和销售里程碑付款，以及L606净销售额的特许权使用费。Pharmosa Biopharm还将获得1000万美元的里程碑付款，用于批准每个额外的适应症和额外的产品。如果Pharmosa Biopharm寻求合作伙伴，Liquidia保留在欧洲和其他地区就L606的开发和商业化进行谈判的优先权，但须满足许可协议中规定的某些条件。 Liquidia打算根据505(b)(2)监管途径在美国寻求L606的首次监管批准。计划中的NDA预计将包括：（1）已完成的临床试验1期研究，证明其耐受性和与Tyvaso相当的药代动力学；（2）在美国的PAH和PH-ILD患者正在进行的开放标签临床试验3期研究数据；（3）来自一项双盲、随机、安慰剂对照研究的临床数据。L606目前正在美国进行一项用于治疗PAH和PH-ILD的开放标签研究，并计划进行一项治疗PH-ILD的全球关键性安慰剂对照疗效研究，计划在2024年上半年启动PH-ILD试验。财务状况 Liquidia Corporation 2023年度收入1750万美元，上年同期收入1590万美元，总的收入主要与Sandoz公司的推广协议销售曲前列尼注射液有关，160万美元的增加主要是由于有利的总退款、回扣和管理式医疗调整，但与上一年相比，销售数量下降的影响抵消了这些调整。2023年度收入成本与上年同期差别不大。研发费用4320万美元，与上年同期相比增长了122%，主要是由于向Pharmosa Biopharm支付了1000万美元的预付款许可费，以获得L606在北美的独家许可。一般及行政费用4470万美元，上年同期3240万美元，增加了38%，主要是由于人事和咨询费用（包括股票薪酬）增加了980万美元，以及为YUTREPIA的潜在商业化做准备而增加的140万美元。2023年度净亏损7850万美元，或每股基本和摊薄亏损1.21美元，上年同期净亏损为4100万美元，或每股基本和摊薄亏损0.67美元。截止2024年3月31日，2024年第一季度的收入为300万美元，而上年同期为450万美元，与上年同期相比，本年度销售数量减少的影响；收入成本为150万美元，上年同期的收入成本为70万美元，与上一年相比的增长主要是由于公司在2023年第四季度扩大了销售队伍；研发费用为1010万美元，上年同期的研发费用为530万美元，增加了91%，主要是由于与员工人数增加相关的人事费用（包括股票薪酬）增加了200万美元，以及与L606计划相关的临床费用增加了170万美元。此外，与YUTREPIA计划相关的费用增加了130万美元，原因是与2023年下半年启动的ASCENT研究相关的临床和供应费用增加；一般及行政费用为2020万美元，上年同期只有780万美元，增加了1240万美元（160%），主要是由于与正在进行的YUTEREPIA诉讼相关的法律费用增加了310万美元，人事费用（包括股票薪酬）增加了590万美元，以及为YUTREPIA的潜在商业化做准备的商业和咨询费用增加了220万美元；净亏损为4090万美元或每股基本和摊薄亏损0.54美元，上年同期净亏损为1170万美元，或每股基本和摊薄亏损0.18美元。 2024年1月，Liquidia宣布获得1亿美元的新融资，主要与公司在当月第一周完成的两笔交易有关。其中，和Patient Square Capital（医疗保健投资公司）的一家附属公司签订了普通股私募购买协议，总收益为7500万美元；同一周，与HealthCare Royalty Partners（HCRx）对收入利息融资协议（RIFA）进行了第四次修订，HCRx额外资助2500万美元，该公司已先投资了6750万美元的非稀释性资本，而最初计划在RIBA下分四批投资1亿美元。第四次修订将2500万美元从第三部分转移到第二部分，因此HCRx在第二部分下总共提供了3500万美元，剩余的第三笔1000万美元和第四笔2250万美元将在未来经HCRx和Liquidia双方同意后提供资金。2024年3月31日，公司的现金和现金等价物总额为1.58亿美元。截止2024年5月30日，Liquidia公司市值还不到10亿美元。截止发稿前为9.14亿美元。参考： NMPA/CDE；药融云数据，www.pharnexcloud.com FDA/EMA/PMDA；相关公司公开披露（除标注外，正文图片均来自企业官网）； https://liquidia.com/； https://mp.weixin.qq.com/s/owUXdtUVUE5Cq6aMki-a6A； https://www.mordorintelligence.com/zh-CN/industry-reports/pulmonary-arterial-hypertension-market；等等。 [ 重磅苏州新药峰会推荐 ] 8月15日论坛1：新药创始人峰会 09:00-09:20复星医药如何应对复杂的生物医药产业环境 09:20-09:40跨越周期，承压下的中国Biotech公司还有哪些机会把握？ 09:40-10:00绕不开的商业化，Biotech公司该怎么看待商业化 10:00-10:20齐鲁制药如何应对复杂的生物医药产业环境 10:20-10:40华东医药如何应对复杂的生物医药产业环境 1040-11:002024中国医药企业综合实力排行榜系列榜单发布 11:00-1120勃林格殷格翰的研发战略及项目需求 11:20-11:40洞察未来：中国生物医药产业发展未来还有哪些机会 11:40-12:20圆桌：中国Biotech公司未来突破点有哪些？ 12:20-13:30午餐 13:30-13:50创新药是选FIC还是BIC？解决临床未满足需要是关键！ 13:50-14:10计算加速新药研发：多肽GPCR药物发现新机遇 14:10-14:30细胞治疗产品动物药效评价 14:30-14:50从AI赛道杀出来，给予CRO和Biotech的发展启发 14:50-15:10和誉医药FGFR抑制剂开发进展 15:10-15:30 富龙康泰BIC多激酶抑制剂开发进展 15:30-15:50YAP/TEAD抑制剂的开发 15:50-16:10浅谈“前药”的前世今生-长乘医药的前药研发 16:10-16:30 慢乙肝功能性治愈创新药物的研究与开发初步确认嘉宾排名不分先后，按照姓名首字母A-Z 蔡学钧，前 BMS，全球研发副总裁曹保红，美迪西，药理部副总裁陈椎，和誉医药，联合创始人兼CSO 邓永奇，凯复医药，CEO 党群，真实生物，总裁杜镇建，北京富龙康泰，副总经理刘谦，阿斯利康中国，副总裁金方，健康元，首席科学家毛化，沙利文，大中华区合伙人兼董事总经理王兴利，复星医药，执行总裁&创新药事业部联席CEO 王中健，药融云，总经理&CTO 许茗彦，长乘医药，CEO 朱继东，奕拓医药，CEO 张弛，新通药物，项目负责人钟文革，齐鲁锐格，CTO 恒瑞医药（嘉宾行程确定中）勃林格殷格翰外部创新合作中心齐鲁制药（嘉宾行程确定中）扫码报名参会【关于药融圈】药融圈PRHub旨在帮助生物医药科技型企业进行品牌推广及商务拓展服务，针对客户的真实需求制定系统化解决方案，通过“翻译-降维-场景化”将客户的品牌信息以直白易懂的方式被公众知悉，同时在流量渠道覆盖100万+垂直用户基础上实现合作目的，帮助合作伙伴完成从品牌开始到商务为终的闭环营销服务。我们已经完成了数十场线下1000人规模的生物医药研发类会议，涵盖小分子新药，大分子新药，改良型新药，BD跨境交易等多个领域，服务了百余家上市/独角兽/生物技术/制药企业。

并购上市批准

100 项与曲前列尼尔脂质体相关的药物交易

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研发状态

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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	临床3期	美国	Liquidia Technologies, Inc.	2026-04-23
间质性肺病导致的肺动脉高压	临床3期	德国	Liquidia Technologies, Inc.	2026-04-23
间质性肺病导致的肺动脉高压	临床3期	拉脱维亚	Liquidia Technologies, Inc.	2026-04-23
间质性肺病导致的肺动脉高压	临床3期	英国	Liquidia Technologies, Inc.	2026-04-23
肺部疾病和缺氧引起的肺动脉高压	临床3期	美国	Liquidia Technologies, Inc.	2021-08-01
肺动脉高压	临床1期	美国	国邑药品科技股份有限公司	2018-11-09

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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


NCT04691154 (ATS2024) 人工标引	临床3期	肺动脉高压 \| 间质性肺病导致的肺动脉高压	15	L606	艱觸鏇齋網獵繭廠蓋鹽(顧範網淵鏇餘餘築齋範) = 窪鹽鹽鑰觸鹽齋願網網壓獵顧淵蓋製範觸壓淵 (鬱餘膚廠窪糧夢獵遞遞 ) 更多	积极	2024-05-22
ATS2021	N/A	-	-	Liposomal Treprostinil Inhalation Suspension (L606)	鹽鹹衊壓夢夢艱襯製範(範選網醖築蓋築製顧壓) = No unexpected treatment-emergent adverse event (TEAE) was found. All AEs resolved within 24 hours of dosing 鹽鹹夢憲願構製顧襯鏇 (衊獵築蓋淵遞願窪膚積 ) 更多	-	2021-05-03