A Phase 3, Randomized, Double-blind, Placebo-controlled Study of Ralinepag to Evaluate Safety and Effects on Exercise Capacity Assessed by CPET in Subjects With WHO Group 1 Pulmonary Hypertension Who Recently Initiated Therapy

Study ROR-PH-302, ADVANCE CAPACITY, was designed to evaluate the effects of ralinepag therapy on exercise capacity as assessed by change in peak oxygen consumption (VO2) derived from cardiopulmonary exercise testing (CPET) after 28 weeks of treatment.

开始日期2021-01-20

申办/合作机构

United Therapeutics Corp.

NCT04613999

/ Completed临床1期

A Phase 1 Open-label, Non-randomized, Single Ascending Dose Escalation Study To Evaluate The Pharmacokinetics, Safety, And Tolerability Of A Ralinepag Extended Release (XR) Tablet Formulation In Healthy Chinese Subjects

开始日期2020-10-09

申办/合作机构

云顶新耀（上海）医药科技有限公司

NCT03683186

/ Enrolling by invitation临床3期

A Phase 3 Open-label Extension (OLE) Study to Evaluate the Long-term Safety and Efficacy of Ralinepag in Subjects With World Health Organization (WHO) Group 1 Pulmonary Arterial Hypertension (PAH)

Study ROR-PH-303, ADVANCE EXTENSION, is an open-label extension (OLE) study for participants with WHO Group 1 PAH who have participated in another Phase 2 or Phase 3 study of ralinepag.

开始日期2019-09-23

申办/合作机构

United Therapeutics Corp.

100 项与 Ralinepag 相关的临床结果

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100 项与 Ralinepag 相关的转化医学

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100 项与 Ralinepag 相关的专利（医药）

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项与 Ralinepag 相关的文献（医药）

2026-09-01·American Journal of Cardiovascular Drugs

Food Effects on the Pharmacokinetics of Single Dose and Steady-State Ralinepag, a Novel Oral Prostacyclin Agonist, in Healthy Volunteers

Article

作者: Lachant, Daniel ; Giannakoulas, George ; Guido, Maria ; Safdar, Zeenat ; Botta, Cristian E ; Patzlaff, Natalie ; Mellemkjær, Søren ; Broderick, Meredith ; Seaman, Scott

BACKGROUND:

Pulmonary arterial hypertension (PAH) is a rare, progressive disorder characterized by pathological pulmonary vascular remodeling leading to right heart failure. Ralinepag extended-release (XR), is a once-daily oral prostacyclin (IP) receptor to treat PAH. This study evaluated the safety, tolerability, and pharmacokinetics (PK) of multiple ascending doses of ralinepag XR tablets in fed and fasted healthy adult volunteers.

STUDY DESIGN AND METHODS:

A single-center, open-label, nonrandomized, multiple-dose titration study of two cohorts (fed (n = 13) or fasted (n = 15)) with once-daily dosing of ralinepag XR for 5 days, with up to four sequential dose escalations. Dosing began at 60 µg and escalated every 5 days as tolerated (120, 180, 240, and 300 µg). Doses were administered after a 10-h overnight fast or a moderate-fat breakfast. PK parameters assessed were maximum observed concentration (C_max), area under the concentration-time curve from time 0 to 24 h postdose (AUC_(0-24)), and time of maximum observed concentration (T_max) on day 1 (single 60 μg dose); and C_max, AUC_(0-24), and T_max at steady state.

RESULTS:

Ralinepag XR exhibited minimal differences in PK parameters between fed and fasted states in the first 24 h. Multiple-dose administration showed a dose-dependent increase in exposure and a median 4-10 h T_max at steady state, independent of fed status. Ralinepag accumulation was approximately 2.1-fold after 5 days of dosing at 60 μg in both fed and fasted states, consistent with a mean half-life of 20-36 h. There were no serious adverse events attributable to ralinepag. The most commonly reported adverse events were headache, jaw pain, and nausea with no differences between groups.

CONCLUSIONS:

Food status did not significantly affect the PK or safety of ralinepag XR.

2026-08-01·LANCET

Ralinepag for the treatment of pulmonary arterial hypertension (ADVANCE OUTCOMES): a randomised, double-blind, placebo-controlled phase 3 study

Article

作者: Gaine, Sean ; Chang, Sung-A ; Ataya, Ali ; Kim, Nick H ; Oudiz, Ronald ; Solum, Derek ; Torres, Fernando ; Barberà, Joan A ; Sood, Namita ; Escudero, Miluska ; Vachiery, Jean-Luc ; Pulido, Tomás ; Jerjes-Sanchez, Carlos ; Giannakoulas, George ; Deng, Chunqin ; Church, Colin ; Channick, Richard ; Lachant, Daniel ; Khanna, Dinesh ; Lacasse, Victoria ; Benza, Raymond ; McLaughlin, Vallerie V ; Feenstra, John ; Cella, Dana ; Preston, Ioana R ; Meyer, Gisela Bohns ; Peterson, Leigh ; Humbert, Marc

BACKGROUND:

Pulmonary arterial hypertension (PAH) is a rare, progressive disease characterised by elevated pulmonary vascular resistance that can lead to right ventricular failure and premature death. Ralinepag is an oral, once-daily, selective prostacyclin IP receptor agonist developed to treat PAH. We aimed to evaluate the efficacy and safety of ralinepag in patients with PAH.

METHODS:

ADVANCE OUTCOMES was a randomised, double-blind, placebo-controlled, event-driven, phase 3 trial of ralinepag in patients with PAH. Eligible patients were aged 18 years or older and PAH was diagnosed on the basis of the 2022 European Society of Cardiology and European Respiratory Society guidelines (mean pulmonary artery pressure >20 mm Hg, pulmonary artery wedge pressure ≤15 mm Hg, and pulmonary vascular resistance of >2 Wood units). Patients were randomly assigned (1:1) to ralinepag or placebo, initiated at a dose of 50 μg once daily and titrated weekly until the highest tolerated individualised dose was reached. Randomisation was stratified by baseline 6-minute walk distance (6MWD), PAH aetiology, and oral background therapy. A block size of four was used within each combination of stratification factors. The sponsor, patients, and all personnel directly involved with the conduct of the study were masked to study drug identity and randomisation assignments. The primary outcome was time to first clinical worsening event, a composite of death from any cause, admission to hospital due to worsening PAH or right heart failure, initiation of parenteral or inhaled prostacyclin-pathway therapy, disease progression, or unsatisfactory long-term clinical response. Efficacy analyses were done in the full analysis set and safety analyses in the safety set; both sets comprised 687 patients after exclusion of 41 randomly assigned and treated patients from sites in China (exclusions due to regulatory challenges and data integrity concerns). This study is registered with ClinicalTrials.gov (NCT03626688) and euclinicaltrials.eu (2023-509304-16-00) and is complete.

FINDINGS:

Patients were enrolled between Jan 24, 2019, and June 20, 2025. Of 1037 patients screened for eligibility, 728 were randomly assigned and received at least one dose of ralinepag or placebo; 687 were included in the full analysis and safety sets, of whom 350 received ralinepag and 337 received placebo. Median follow-up from randomisation to clinical worsening event, censoring, or study closure was 85·0 weeks (IQR 27·6-160·9) in the ralinepag group and 78·4 weeks (34·6-137) in the placebo group. At baseline, patients had a mean 6MWD of 438·9 m (SD 104·8) and 548 (80%) of 687 patients were receiving dual background PAH therapy. Overall, 64 (18%) of 350 patients in the ralinepag group and 121 (36%) of 337 patients in the placebo group had a first clinical worsening event (hazard ratio 0·45 [95% CI 0·33-0·62]; p<0·0001). The largest numerical between-group differences in components of the composite outcome were observed for disease progression, initiation of parenteral or inhaled prostacyclin-pathway therapy, and unsatisfactory long-term clinical response. Adverse event was the primary reason for treatment discontinuation in 65 (19%) of 350 patients in the ralinepag group and ten (3%) of 337 patients in the placebo group. Serious adverse events occurred in 98 (28%) patients in the ralinepag group and 104 (31%) patients in the placebo group. Adverse events leading to death occurred in 15 (4%) and 14 (4%) patients, respectively.

INTERPRETATION:

In patients with PAH receiving contemporary background therapy, ralinepag significantly reduced the risk of first clinical worsening compared with placebo, but was associated with more adverse-event-related treatment discontinuations. These results support the use of ralinepag as an oral, once-daily prostacyclin-pathway treatment option for PAH.

FUNDING:

United Therapeutics Corporation.

2025-08-01·JHLT Open

Safety and pharmacokinetics of ralinepag, a novel oral prostacyclin receptor agonist

Article

作者: Patzlaff, Natalie ; Sood, Namita ; Ataya, Ali ; Broderick, Meredith ; Coons, James C ; Patricia George, M ; Seaman, Scott ; Chakinala, Murali M

Background:

Ralinepag is an oral, potent, highly selective prostacyclin receptor agonist and is in development for pulmonary arterial hypertension. Ralinepag was formulated as an immediate-release (IR) capsule, later modified to an extended-release (XR) tablet to optimize administration for once-daily dosing.

Methods:

This phase I study evaluated the pharmacokinetic (PK) profile and relative bioavailability of ralinepag XR. There were two cohorts of healthy participants (n = 12 each). One received a single dose of ralinepag IR (30 µg) followed by single ascending doses of XR (60, 120, and 180 µg), and the other received single ascending doses of selexipag (200, 400, and 600 µg). Participants titrated to the highest tolerated dose. There was a 7-day washout period between each dose.

Results:

The ralinepag XR formulation showed a reduced maximum observed concentration (C_max), delayed time of maximum observed concentration (T_max), and similar area under the curve compared with the IR formulation. Ralinepag XR demonstrated a gradual increase in plasma concentration over 8 to 12 hours, followed by a slow decline, with a half-life of 19-23 hours. In contrast, the active metabolite of selexipag (MRE-269) exhibited a sharp peak with a half-life of 9-10 hours. Ralinepag XR was well-tolerated by healthy volunteers, with 9 of 12 participants reaching the highest dose. Adverse events were qualitatively similar to oral prostacyclin class therapies.

Conclusion:

Ralinepag XR has a half-life suitable for once-daily dosing. The reduced C_max and delayed T_max contributes to lower peak-to-trough fluctuations and may provide favorable effects for sustained efficacy and improved tolerability.

项与 Ralinepag 相关的新闻（医药）

2026-10-13

·BioSpace

United Therapeutics Presents New Data at the CHEST 2026 Annual Meeting

A pre-specified subgroup analysis from the phase 3 ADVANCE OUTCOMES study showed that ralinepag reduced the risk of clinical worsening in patients with pulmonary arterial hypertension ( PAH ), regardless of how many noninvasive low-risk criteria they met at baseline A post-hoc analysis of the phase 3 TETON-2 study showed that nebulized treprostinil significantly reduced the risk of multiple disease progression events compared to placebo in patients with idiopathic pulmonary fibrosis ( IPF ) Interim analyses using validated mortality risk models in the prospective PHINDER study showed that patients with interstitial lung disease ( ILD ), particularly those with IPF, have numerically higher mortality risk scores, supporting the need for earlier pulmonary hypertension ( PH ) screening in these patients An interim analysis of the baseline characteristics of patients enrolled in DeciPHer-ILD , a multicenter registry of PH-ILD patients, found that these patients exhibit poor hemodynamics, impaired functional class, and reduced exercise capacity indicative of advanced disease SILVER SPRING, Md. & RESEARCH TRIANGLE PARK, N.C.--(BUSINESS WIRE)--United Therapeutics Corporation (Nasdaq: UTHR ), a public benefit corporation, today announced that two oral and five poster presentations will be presented across its commercial and development portfolio at the CHEST 2026 Annual Meeting hosted by the American College of Chest Physicians taking place October 18-21, 2026, in Phoenix, Arizona. United Therapeutics will also sponsor and host Time to Look Closer: Emerging Data and Screening Insights in PH-ILD , featuring preliminary data from PHINDER , a study designed to help develop a screening tool for PH-ILD. “CHEST offers the opportunity to share important insights about the composition and diverse needs of patients with PAH, PH-ILD, and IPF as we strive to improve care for these progressive, life-threatening diseases,” said Franck Rahaghi, M.D. , Chief Medical Officer, United Therapeutics. “United Therapeutics is dedicated to improving outcomes in these disease areas by developing new treatment options and advancing research that can help lead to earlier screening and disease detection and improved understanding of treatment paradigms. A number of our presentations at this year’s CHEST meeting, as well as our ‘Time to Look Closer’ symposium, are directed toward these essential goals.” Oral presentations Efficacy of Inhaled Treprostinil on Multiple Disease Progression Events in Patients with Idiopathic Pulmonary Fibrosis in the TETON-IPF Program Oral Presentation, Tuesday, October 20, 1:59 - 2:03 PM MST. Presented by Steven Nathan, M.D., MBBCh, FCCP. Mortality Risk Scoring in Interstitial Lung Disease with and Without Pulmonary Hypertension Using the ILD-Gap Model: Interim Analysis from the PHINDER Study Oral Presentation, Tuesday, October 20, 3:05 – 3:13 PM MST. Presented by Mary Beth Scholand, M.D. Poster presentations Ralinepag Treatment Effects Across Noninvasive Low-Risk Pro Results from ADVANCE OUTCOMES Poster Presentation, Poster #3222 Tuesday, October 20, 1:45 PM MST. Presented by Jean Elwing, M.D., FCCP. Neighborhood-Level Social Risk and Patient-Reported Outcomes in Patients with Pulmonary Arterial Hypertension Poster Presentation, Poster #3498, Tuesday, October 20, 1:45 PM MST. Presented by Karim El-Kersh, M.D. Risk Stratification in Interstitial Lung Disease Associated with Pulmonary Hypertension Using the PVD-B65 Model: Interim Analysis from the PHINDER Study Poster Presentation, Poster #3555, Tuesday, October 20, 1:45 PM MST. Presented by Parth Rali, M.D., MBBS, FCCP. Baseline Characteristics of ILD-PH Patients Enrolled in the DeciPHer-ILD Registry: An Interim Analysis Poster Presentation, Poster #4259, Wednesday, October 21, 10:20 AM MST. Presented by Kristin Highland, M.D. Real-World Outcomes of Patients with Idiopathic Pulmonary Fibrosis Initiating Antifibrotic Therapy by Pulmonary Fibrosis Foundation Center Status Poster Presentation, Poster #4290, Wednesday, October 21, 10:20 AM MST. Presented by Manisha Lin, PharmD. Sponsored and hosted events Time to Look Closer: Emerging Data and Screening Insights in PH-ILD Tuesday, October 20 10:30 – 11:15 AM MST The FDA has accepted United Therapeutics’ New Drug Application for ralinepag to treat PAH and its supplemental New Drug Application for nebulized treprostinil to treat IPF. Ralinepag has not been approved for use in any indication by the FDA and remains investigational for PAH. Nebulized treprostinil remains investigational for IPF. About United Therapeutics Founded by CEO Martine Rothblatt to discover a cure for her daughter's life-threatening rare disease, pulmonary arterial hypertension, United Therapeutics transforms the treatment of rare diseases and pioneers alternatives to expand the supply of transplantable organs. From our innovative therapies to our groundbreaking manufactured organs, we are bold and unconventional. We move quickly from scientific theory to practical technologies that can save lives. As a public benefit corporation, even our legal structure reflects our commitments. We serve patients, act with integrity, create long-term shareholder value, and operate with sustainable practices that protect the future we are working to build. Visit us at and follow us on LinkedIn , Facebook , and Instagram . Forward-Looking Statements Statements included in this press release that are not historical in nature are “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements include, among others: our efforts to improve patient care for patients with PAH, PH-ILD, and IPF; our efforts to develop new treatment options and advance research that can help lead to earlier screening and disease detection and improved understanding of treatment paradigms; and our goals of expanding the supply of transplantable organs, developing practical technologies that can save lives, creating long-term shareholder value, and operating with sustainable practices. These forward-looking statements are subject to certain risks and uncertainties, such as those described in our periodic reports filed with the Securities and Exchange Commission, that could cause actual results to differ materially from anticipated results. Consequently, such forward-looking statements are qualified by the cautionary statements, cautionary language, and risk factors set forth in our periodic reports and documents filed with the Securities and Exchange Commission, including our most recent Annual Report on Form 10-K, Quarterly Reports on Form 10-Q, and Current Reports on Form 8-K. We claim the protection of the safe harbor contained in the Private Securities Litigation Reform Act of 1995 for forward-looking statements. We are providing this information as of October 12, 2026, and assume no obligation to update or revise the information contained in this press release whether as a result of new information, future events or any other reason. Contacts For Further Information Contact: Investor Inquiries Media Inquiries communications@unither.com

临床结果临床3期

2026-10-03

·BLOODSCREEN

柳叶刀呼吸病学 | ERS 2026会议纪要

ATS2026会议披露的6大研究Tozorakimab治疗COPD急性加重尽管接受标准吸入维持治疗，仍有超过一半的慢性阻塞性肺疾病（COPD）患者会发生急性加重。目前，获批用于控制不佳COPD的生物制剂主要限于血嗜酸性粒细胞升高的患者，因此对于更广泛COPD人群仍存在未满足的治疗需求。在ERS 2026大会上，来自美国匹兹堡大学的Frank C Sciurba介绍了两项设计完全相同的Ⅲ期临床试验OBERON和TITANIA的研究结果。两项研究比较了IL-33抑制性单克隆抗体tozorakimab与安慰剂在标准治疗基础上的疗效，纳入既往或当前吸烟、且过去一年内有COPD急性加重史的患者。患者随机接受300 mg皮下注射tozorakimab或匹配安慰剂，每4周一次，持续52周。OBERON和TITANIA研究中分别有446例和438例患者接受tozorakimab治疗，431例和435例接受安慰剂治疗，各组至少75%的患者为既往吸烟者。在既往吸烟者中，与安慰剂相比，tozorakimab使中重度急性加重的年化发生率在OBERON研究中降低29%，分别为1.34次和1.90次，率比（RR）为0.71（95% CI 0.57–0.88，p=0.002）；在TITANIA研究中降低34%，分别为1.37次和2.07次，RR为0.66（95% CI 0.55–0.80，p<0.001）。在总体人群中，无论气流受限严重程度还是吸烟状态如何，结果均保持一致。此外，在血嗜酸性粒细胞计数低于150 cells/μL、150–300 cells/μL以及≥300 cells/μL的患者中，tozorakimab分别使急性加重年化发生率降低23%、34%和43%。Sciurba表示，这些结果最值得关注之处在于，tozorakimab的疗效覆盖了广泛的患者群体，并不受肺功能严重程度、嗜酸性粒细胞水平和吸烟状态影响。在广泛患者群体中急性加重约减少30%，而血嗜酸性粒细胞计数超过300 cells/μL的患者中急性加重减少达到43%，因此tozorakimab有潜力改变COPD的治疗模式。ERS 2026和NEJM同步发布｜靶向IL-33预防慢阻肺急性加重Ralinepag治疗肺动脉高压此前已经发表的Ⅲ期ADVANCE OUTCOMES研究显示，口服、每日一次的选择性前列环素受体激动剂ralinepag，在标准治疗或背景治疗基础上加用后，与安慰剂相比能够显著延长PAH患者首次临床恶化的时间，中位随访时间在ralinepag组和安慰剂组分别为85.0周和78.4周。在ERS 2026大会上，巴黎萨克雷大学和Inserm的Marc Humbert进一步介绍了ADVANCE OUTCOMES研究中有关临床改善的结果。该研究共纳入687例成人患者，中位年龄为53岁（范围18–84岁），其中350例随机接受ralinepag治疗，337例接受安慰剂，患者确诊PAH的平均时间为4.5年。研究将临床改善定义为未发生临床恶化，同时满足以下三项标准中的至少两项，即6分钟步行距离较基线增加至少10%或至少30 m、WHO功能分级改善至或维持在Ⅱ级或Ⅲ级，以及NT-proBNP较基线下降至少30%。在第28周，ralinepag组44.5%的患者达到临床改善，而安慰剂组为34.6%，共同优势比为1.47（95% CI 1.07–2.02，p=0.015）。 Humbert表示，这些结果进一步支持ralinepag作为接受现代背景治疗PAH患者的一种治疗选择，但未来仍需研究其在现有PAH治疗中的最佳定位、如何优化剂量递增期间的耐受性，以及长期治疗是否能够改善更为重要的临床结局。Lancet | 创新药前列环素IP受体激动剂Ralinepag治疗动脉性肺动脉高压老年慢性肺病患者的流感疫苗高剂量流感疫苗在老年人中能够产生比标准剂量疫苗更强的免疫反应，此前FLUNITY-HD两项随机试验的汇总分析已经显示，高剂量疫苗能够降低老年人因肺炎或流感住院的风险。在ERS 2026大会上，来自哥本哈根大学Herlev和Gentofte医院的Lisa Steen Duus进一步介绍了FLUNITY-HD研究中年龄≥65岁且患有慢性肺病老年患者的相关结果。该亚组共包含466,320名参与者，其中31,976例（6.9%）患有慢性肺病，包括哮喘、支气管扩张症或COPD，所有参与者中一半接受一次高剂量流感疫苗，另一半接受一次标准剂量疫苗。在没有慢性肺病的患者中，高剂量疫苗组发生922例肺炎或流感住院事件，标准剂量组为1031例，粗疫苗有效性（rVE）为10.7%（95% CI 2.8–18.4）；而在患有慢性肺病的患者中，两组分别发生390例和406例住院事件，rVE为4.4%（95% CI −10.2–17.0）。 Duus表示，在FLUNITY-HD研究中，高剂量流感疫苗降低住院风险的获益在有无慢性肺病的人群中表现出一致性，这些结果支持在包括慢性肺病患者在内的老年人群中使用高剂量流感疫苗，同时未来仍需进一步研究这一获益是否适用于不同类型的慢性肺病以及不同疾病严重程度的人群。Astegolimab与COPD住院此前发表的ALIENTO和ARSANA两项Ⅲ期研究汇总分析显示，抗ST2人源IgG2单克隆抗体、IL-33抑制剂astegolimab，与安慰剂相比能够显著降低年龄≥40岁COPD患者中中重度急性加重的年化发生率。在ERS 2026大会上，密歇根大学的MeiLan K Han进一步介绍了这项汇总分析中与急性加重相关的住院结局。汇总研究中，892例患者接受476 mg astegolimab、每2周一次，896例患者接受476 mg、每4周一次，888例患者接受安慰剂、每2周一次，治疗持续52周。与安慰剂相比，每2周一次的astegolimab使导致24小时内住院或死亡的重度COPD急性加重年化发生率降低32%，调整后RR为0.68（95% CI 0.52–0.87，nominal p=0.0028）；同时，导致急诊就诊、24小时内住院或死亡的急性加重发生率也降低32%，分别为0.19次和0.28次/年，RR为0.68（95% CI 0.53–0.86，nominal p=0.0015）。 Han表示，这些数据进一步强调了IL-33通路在COPD中的作用，以及astegolimab降低严重临床事件的潜力，而这些严重事件与更高的死亡率以及较大的医疗资源和医疗费用消耗相关。柳叶刀 | IL-33受体单抗治疗频繁急性加重慢阻塞肺病：非2型炎症同样获益？Lunsekimig治疗中重度哮喘 Lunsekimig能够同时阻断胸腺基质淋巴细胞生成素（TSLP）和IL-13，从而靶向多个炎症通路。密歇根大学肺与危重症医学科的Njira L Lugogo介绍了AIRCULESⅡ期b期研究结果，该研究纳入685例接受中高剂量吸入性糖皮质激素治疗的中重度哮喘成人患者，所有患者在前一年至少发生过一次急性加重。研究采用剂量探索设计，患者分别接受lunsekimig 60 mg每4周一次、300 mg每4周一次、300 mg每8周一次或安慰剂每4周一次。在300 mg每4周一次的治疗组中，哮喘急性加重年化发生率较安慰剂显著降低55.3%（95% CI 27.8–72.3，校正p=0.0016）。这一效果在既往一年发生≥3次急性加重的患者中进一步增强，RRR达到86.9%（95% CI 54.2–96.3）；在具有2型炎症生物标志物的患者中也观察到更明显的获益，其中基线FeNO≥25 ppb患者的RRR为65.1%（95% CI 30.5–82.5），血嗜酸性粒细胞≥300 cells/μL患者的RRR为70.6%（95% CI 40.5–85.5）。 Lugogo表示，该研究显示lunsekimig能够显著改善哮喘急性加重，而且这种改善在既往急性加重次数较多以及存在2型炎症生物标志物升高的患者中更加明显。她认为，同时靶向多个炎症通路可能为哮喘患者带来更广泛的获益，包括肺部疾病以及部分患者同时存在的肺外2型炎症相关合并症，而针对具有冗余性的炎症通路可能成为未来中重度哮喘治疗的一个方向。

2026-09-22

·亦弘商学院

课堂丨TPP&CDP：以终为始，筑牢创新药临床开发与商业化决策根基

2026年9月17-20日，“目标产品特征（TPP）和临床开发计划（CDP）的制定”课程在北京顺利开课。来自国内国际药企的医学、临床、注册、研发等领域的33位同学在师资团队的指导下，共同学习如何准备、制定和撰写TPP和CDP，并掌握如何运用这两个科学工具有效规划和决策临床开发路径。高层经理研修项目目标产品特征（TPP）和临床开发计划（CDP）的制定 2026/9/17-20 苏州课程内容：回顾新药临床开发全过程课程主席：徐宁博士前再鼎医药执行副总裁亦弘商学院研究员临床研究管理课程建设委员会委员课程主席徐宁博士开篇，带领同学们回顾了新药研发“高风险、高投入、长周期、低成功率”的复杂性，介绍了课程目标、师资与日程，并剖析中国创新药同质化、原始创新不足等痛点。帮助同学们建立起「以终为始」的方法学认知，理解 TPP/CDP 在明方向、助决策、降风险、提成功率与延长产品生命周期中的核心价值。课程内容：目标产品特征（TPP）课程主席：朱煦老师北京正融智讯联合创始人/首席医学官临床研究管理课程建设委员会委员 TPP的概念以及TPP 在一体化药物开发策略中的重要意义 TPP制定的各阶段活动及涵盖的要素如何制定TPP？课程主席朱煦老师讲解目标产品特征（TPP）的定义、意义与制定过程：TPP 是「以终为始」的研发思路与跨部门沟通契约，呼应 FDA 说明书章节，由 DTPP→TPP→TPC 逐层细化，依托证据平台（PoE）与 Go/No-Go 标准驱动决策；并以类风湿关节炎药物 T66 为案例展示应用，帮助同学们理解TPP 是整合研发效率、统一多方利益相关人价值预期的核心管理工具。课程内容：如何在商务拓展中运用TPP和CDP？课程教授：包骏博士 Apuri BioVenture创始及管理合伙人 TPP及CDP在商务拓展中的应用商务拓展和TPP、CDP的关系？ TPP及CDP如何在商务拓展中应用？如何运用TPP和CDP帮助BD的决策？具有丰富商务拓展经验的包骏博士从项目的综合评估角度，给同学们阐述了 TPP/CDP 在商务拓展（BD）中的关键作用：作为项目综合评估（技术、商业、财务、注册、IP）、估值（rNPV/实物期权等）、尽职调查与合同谈判的核心文件与参数来源；结合案例解析成功（PTS/PRS/PTRS）测算与交易成败因素。帮助同学们掌握用 TPP/CDP 支撑“引进/授权”全链条理性决策的方法。课程内容：临床开发计划（CDP）临床开发计划（CDP）的概念及与TPP的关系 CDP分阶段涵盖的要素及重点考量如何设计和规划CDP？制定CDP前的主要思路？法规监管的考量：分析法规事务在TPP和CDP制定中的角色 TPP是新药开发的明确目标，是制定CDP的基础，而CDP是达到TPP所规划目标的线路图。CDP作为描述一个候选药物如何从首次人体试验到注册上市的蓝图，需要多学科、有激情的专业团队通过不懈思索与努力，花费大量时间和精力来制定、实施和评估。徐博士系统讲解临床开发计划（CDP）的概念、分阶段（临床前/早期/晚期）、与 TPP 的「战略—战术」关系及六大核心考量（患者需求、科学依据、TPP、研究设计、法规、决策风险）；涵盖 FDA/NMPA 加速通道、与监管会议类型、CDP 团队组建与项目负责人角色，帮助同学们掌握「以终为始」制定 CDP、用数据支撑注册上市与产品生命周期管理的框架。课程内容：临床阶段市场准入的考量课程教授：宣建伟教授中山大学药学院医药经济研究所所长亦弘商学院研究员课程教授：谭宝滢老师中山大学医药经济研究所助理研究员晚期临床视角下的医保准入与证据构建 DRG/DIP 改革下医院科室的运营逻辑临床科室典型痛点与精细化运营策略创新药全链条准入的企业实践路径宣建伟老师围绕晚期临床阶段市场准入展开，讲解市场准入概念、国内医保谈判制度，强调创新药要在晚期临床布局卫生经济学与真实世界证据，支撑医保准入；同时介绍药品临床综合评价方法，区分药监审评与医保支付评价差异。谭宝滢老师聚焦DRG/DIP支付改革，解读CMI、病例倍率、医院盈亏逻辑及DRG/DIP3.0下各级医院定位，结合血液科、肝胆外科、乳腺科实例，剖析科室运营痛点，提出病例结构优化、药术联动、战略性亏损等精细化运营策略。帮助同学们理解创新药从证据生成到多端准入的完整逻辑与实操要点。课程内容：早期临床开发阶段CDP的考量——“Go/No go”的决策课程教授：黑永疆博士上海慧思博生物创始人临床开发早期如何确定适应症、目标患者人群？如何科学有效地快速确定患者的耐受性、安全性、用药剂量选择？临床标记物的选择、adaptive study design、如何尽早验证临床有效的概念（POC）？如何进行获益和风险评估点及“go/no go”决策标准？黑永疆博士给同学们讲解了早期临床开发计划（eCDP）的蓝图，覆盖 IND 准备、首次人体（FIH）/Ⅰ期试验设计、起始剂量（MRSD/MABEL）、剂量递增（3+3/BOIN）、临床药理与生物标志物、概念验证（POC）及 Go/No-Go 决策框架，并以肺动脉高压药 Ralinepag 为例，帮助同学们掌握从 IND 到 POC 的早期开发路径、剂量优化工具与跨学科团队协作方法。课程内容：晚期临床开发阶段CDP制定的核心考量晚期临床视角下的医保准入与证据构建 DRG/DIP 改革下医院科室的运营逻辑临床科室典型痛点与精细化运营策略创新药全链条准入的企业实践路径朱老师聚焦晚期临床开发计划（CDP）的制定，系统讲解从概念验证（PoC）到上市的开发路径、预设的 Go/No-Go 决策标准，以及 CDP 与 TPP/TPC 的呼应关系；并以易瑞沙（IRESSA）、抗 MRSA 新药 Tedizolid 为案例，剖析监管沟通、风险评估（5R/6R 原则）与 MRCT 策略。帮助同学们理解晚期 CDP 需跨部门协作、动态应对竞争格局与政策演变，掌握在关键节点降低风险、支撑产品成功上市的方法。案例分享课程教授：陈洁博士开拓药业资深副总裁案例分享环节，陈博士以中国创新药 BD 窗口期为背景，通过三个真实案例——外用雄激素受体拮抗剂 CB-03-01（痤疮/脱发）、PD-1/VEGF 双抗 AK112、CDK2/4/6 三重抑制剂 RGT-419B——给同学们展示了如何以 TPP 锁定差异化定位、以 CDP 分步验证并控制风险。帮助同学们掌握运用 TPP/CDP 整合科学、商业与注册要求，系统校验立项假设、优化开发路径与交易决策的实战方法。小组报告随着课程的进行，同学们根据小组报告的要求，通过课后信息收集及组内分工合作，经过了几轮充分讨论，在共同努力下，完成了某产品的TPP和CDP的制定，将三天所学内容在实践模拟中学以致用，融会贯通。授课老师们对各组方案的科学性、合理性和完整性进行了提问、点评和总结，经过充分的互动讨论，进一步帮助同学们更加深刻地理解TPP和CDP制定过程中需要注意的关键点。敬请持续关注“临床研究管理”精彩课堂！问卷调研新版GCP之数据治理需求调研信息严格保密，感谢支持！ // 咨询电话：0512-6556 6063 刘老师（电话同微信）：135 2222 4003 联系邮箱：apply@yeehongedu.cn 相关链接：迭代革新 | 临床研究管理专业能力培养项目第七期重磅升级焕新赋能｜药物研发管理专业能力培养项目第九期重磅升级

100 项与 Ralinepag 相关的药物交易

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外链

KEGG	Wiki	ATC	Drug Bank
D10725	-	-	DB12462

研发状态

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
肺动脉高压	申请上市	美国	United Therapeutics Corp.	2026-08-24
结缔组织病	临床3期	美国	United Therapeutics Corp.	2019-01-24
结缔组织病	临床3期	中国	United Therapeutics Corp.	2019-01-24
结缔组织病	临床3期	阿根廷	United Therapeutics Corp.	2019-01-24
结缔组织病	临床3期	澳大利亚	United Therapeutics Corp.	2019-01-24
结缔组织病	临床3期	奥地利	United Therapeutics Corp.	2019-01-24
结缔组织病	临床3期	比利时	United Therapeutics Corp.	2019-01-24
结缔组织病	临床3期	巴西	United Therapeutics Corp.	2019-01-24
结缔组织病	临床3期	保加利亚	United Therapeutics Corp.	2019-01-24
结缔组织病	临床3期	加拿大	United Therapeutics Corp.	2019-01-24

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临床结果

适应症

分期

评价

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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


NCT03626688 (ADVANCE OUTCOMES, Pubmed \| Lancet)	临床3期	肺动脉高压	687	Ralinepag	鹹蓋選鹹餘憲積積淵鹽(壓鏇糧網鏇獵壓願觸鏇) = 襯襯遞網觸積願網憲蓋顧獵鹽蓋鏇憲鏇窪鹹鹹 (網醖獵獵鏇鹹醖艱鹹憲 ) 更多	积极	2026-08-01
				Placebo	鹹蓋選鹹餘憲積積淵鹽(壓鏇糧網鏇獵壓願觸鏇) = 淵積醖糧遞範選餘齋鹽顧獵鹽蓋鏇憲鏇窪鹹鹹 (網醖獵獵鏇鹹醖艱鹹憲 ) 更多
NCT04084678 (CAPACITY, CTgov)	临床3期	肺动脉高压	10	Ralinepag (Ralinepag)	網蓋製鏇鏇壓淵鹽壓憲(選鹽顧築觸艱壓製鑰蓋) = 遞鏇憲築廠選壓衊範憲築範製築鬱獵網壓鹹遞 (襯壓網鏇廠壓選觸願積, 鬱願衊範艱觸窪糧蓋醖 ~ 構遞餘襯夢衊鏇淵願範) 更多	-	2026-07-06
				Placebo (Placebo)	網蓋製鏇鏇壓淵鹽壓憲(選鹽顧築觸艱壓製鑰蓋) = 鹹鏇憲顧願選鏇繭網窪築範製築鬱獵網壓鹹遞 (襯壓網鏇廠壓選觸願積, 醖鑰膚鏇餘鏇鹹獵積壓 ~ 鹹衊網憲獵憲壓膚製鹽) 更多
NCT03626688 (ADVANCE OUTCOMES, Corporate Publications) 人工标引	临床3期	肺动脉高压	687	Ralinepag	鏇鹹夢窪憲齋餘網艱築(鹹廠艱顧鹹憲鑰艱積鑰): HR = 0.45 (95.0% CI, 0.33 ~ 0.62), P-Value = <0.0001 达到更多	积极	2026-03-02
				Placebo
NCT03683186 (ATS2024) 人工标引	临床3期	肺动脉高压	96	Ralinepag (Weeks 28)	願艱鹹淵積醖憲願顧網(構鹽鹹艱觸觸選壓鏇顧) = Adverse events in the OLE have been consistent with the known safety profile of prostacyclin therapies, with headache (69.8%), diarrhea (39.6%), and nausea (31.3%) reported most commonly. 鹽積簾蓋遞齋廠遞鏇鹽 (網獵簾願膚壓繭淵窪鹹 )	积极	2024-05-22
				Ralinepag (Weeks 52)
NCT02279745 (APD811-007, ATS2022)	临床2期	肺动脉高压	61	Ralinepag	蓋鑰鏇窪繭願糧醖製憲(餘鹽膚衊製襯獵淵夢簾) = 憲餘艱簾獵齋鬱觸繭糧鏇廠廠遞壓衊鹹餘襯觸 (願壓蓋構鹽壓夢鏇範鬱 )	积极	2022-05-15
NCT02279745 (APD811-007, CTgov)	临床2期	肺动脉高压	45	Ralinepag	鏇膚繭構壓憲顧夢繭鏇(繭觸壓憲觸壓構遞鹹衊) = 餘簾艱膚鬱鑰醖網淵淵餘衊夢鹹範構簾築獵窪 (範鬱鬱膚觸積餘鑰鹽製, 願網遞夢願製繭顧衊繭 ~ 蓋廠繭製築憲築願淵獵) 更多	-	2021-12-22
NCT02279160 (CTgov)	临床2期	家族性肺动脉高压 \| 肺动脉高压 \| 特发性肺动脉高压	61	APD811 (APD811)	廠顧膚憲衊遞鬱鬱繭廠(鑰淵憲製醖獵齋鹽範壓) = 壓艱廠築鏇艱艱廠襯範願鏇觸廠顧窪願蓋窪鬱 (範廠築蓋鑰顧顧淵艱廠, 1.85) 更多	-	2020-07-14
				Placebo (Placebo)	廠顧膚憲衊遞鬱鬱繭廠(鑰淵憲製醖獵齋鹽範壓) = 顧範憲鹹簾齋選艱製襯願鏇觸廠顧窪願蓋窪鬱 (範廠築蓋鑰顧顧淵艱廠, 1.62) 更多
NCT02279160 (Pubmed \| Eur Respir J)	临床2期	肺动脉高压	61	Ralinepag	鑰鹽膚繭醖廠觸築齋窪(遞網糧壓艱鑰壓選艱窪) = 鏇顧遞簾築鑰壓憲膚鏇築廠積淵糧網鹽鹽觸遞 (廠範艱艱蓋膚襯構鏇鹽 ) 更多	积极	2019-10-01
				Placebo	鑰鹽膚繭醖廠觸築齋窪(鏇齋繭繭膚鑰遞襯觸獵) = 鹹製網齋獵鬱獵襯膚糧積齋觸築範衊壓顧獵簾 (鬱製鹹衊鬱構顧鏇襯膚 ) 更多