A Phase III, Open-label, Single Arm Study to Investigate the Efficacy and Safety of Elafibranor 80 mg in Adult Japanese Participants With Primary Biliary Cholangitis (PBC)

The purpose of this study is to find out about the safety and how well the study intervention (elafibranor) works in participants with PBC. The participants in this study will have confirmed PBC with inadequate response or intolerance to UDCA, which is a medication used in the management and treatment of cholestatic liver disease.
PBC is a slowly progressive disease characterised by damage of the bile ducts in the liver, leading to a build-up of bile acids which causes further damage. The liver damage in PBC may lead to scarring (cirrhosis).
PBC may also be associated with multiple symptoms. Many patients with PBC may require a liver transplant or may die if the disease progresses and a liver transplant is not done. In this study all participants will receive a daily dose of elafibranor (the study intervention).
The main aim of this study is to determine if elafibranor reduces alkaline phosphatase (ALP) and total bilirubin levels. High ALP and bilirubin levels in the blood can indicate liver disease.
There will be 4 periods in this study: A screening period (up to 10 weeks) to assess whether the participant can take part. A treatment period (52 weeks) where all eligible participants will receive elafibranor. A variable treatment extension period (2-5 years) from End Of Treatment (EOT) period up to the commercial availability of elafibranor in Japan. A follow-up period (4 weeks) where participants' health will be monitored.
Participants will undergo blood sampling, urine collections, physical examinations, clinical evaluations, electrocardiograms (ECG: recording of the electrical activity of heart), ultrasound examinations (a non-invasive test that passes a probe over skin to look at the bladder, urinary tract, and liver), and Fibroscan® examinations (to measure stiffness of the liver). They will also be asked to fill in questionnaires. Each participant will be in this study for up to approximately 6 years

开始日期2025-01-16

申办/合作机构

Ipsen SA

NCT06383403

/ Active, not recruiting临床3期

A Phase IIIb Randomised, Parallel-Group, Double-Blind, Placebo-Controlled, Two-Arm Study to Evaluate the Effect of Elafibranor 80 mg on Normalisation of Alkaline Phosphatase in Adult Participants With Primary Biliary Cholangitis (PBC) and Inadequate Response or Intolerance to Ursodeoxycholic Acid.

The participants in this study will have confirmed PBC with inadequate response or intolerance to Ursodeoxycholic acid (UDCA), which is a medication used in the management and treatment of cholestatic liver disease.
Primary biliary cholangitis is a slowly progressive disease characterised by damage of the bile ducts in the liver, leading to a build-up of bile acids which causes further damage. The liver damage in PBC may lead to scarring (cirrhosis). PBC may also be associated with multiple symptoms.
Many patients with PBC may require a liver transplant or may die if the disease progresses and a liver transplant is not done. This study will compare a daily dose of elafibranor (the study drug) to a daily dose of placebo (a dummy treatment).
The main aim of this study is to determine if elafibranor is better than placebo in reducing ALP levels to a normal value. High ALP levels in the blood can indicate liver disease.
There will be three periods in this study: A screening period (up to 8 weeks) to assess whether the participant can take part; a treatment period (up to 52 weeks) where eligible participants will be grouped as per their blood ALP levels and randomly assigned to either receive elafibranor or placebo, and a follow-up period (4 weeks) where participants' health will be monitored.
Participants will be twice as likely to receive elafibranor than placebo (2:1 ratio).
Participants will undergo blood sampling, urine collections, physical examinations, clinical evaluations, electrocardiograms (ECG: recording of the electrical activity of heart), ultrasound examinations (a noninvasive test that passes a probe over skin to look at the bladder, urinary tract, and liver), and Fibroscan® examinations (a noninvasive test that passes a probe on skin to measure stiffness of the liver).
They will also be asked to fill in questionnaires. Each participant will be in this study for up to 64 weeks (15 months).

开始日期2024-07-09

申办/合作机构

Ipsen SA

NCT06016842

/ Recruiting临床3期

A Phase III Randomised, Parallel-Group, Double-Blind, Placebo-Controlled, Two-Arm Study to Evaluate the Efficacy and Safety of Elafibranor 80 mg on Long-Term Clinical Outcomes in Adult Participants With Primary Biliary Cholangitis (PBC)

The participants of this study will have confirmed Primary Biliary Cholangitis (PBC) and cirrhosis (scarring of the liver).
PBC is a slowly progressive disease, characterised by damage to the bile ducts in the liver, leading to a build-up of bile acids which causes further damage.
The liver damage in PBC may lead to cirrhosis. PBC may also be associated with multiple symptoms. Many patients with PBC may require liver transplant or may die if the disease progresses and a liver transplant is not done.
This study will compare a daily dose of elafibranor (the study drug) to a daily dose of placebo (a dummy treatment) and will last up to 3.5 years for each participant.
The main aim of this study is to determine if elafibranor is better than placebo in preventing clinical outcome events showing disease worsening (including progression of disease leading to liver transplant or death).
This study will also study the safety of long-term treatment with elafibranor, as well as the impact on symptoms such as itching and tiredness.

开始日期2023-08-31

申办/合作机构

Ipsen SA

100 项与 Elafibranor 相关的临床结果

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100 项与 Elafibranor 相关的转化医学

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100 项与 Elafibranor 相关的专利（医药）

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141

项与 Elafibranor 相关的文献（医药）

2026-01-01·JOURNAL OF HEPATOLOGY

Safety and efficacy of elafibranor in primary sclerosing cholangitis: The ELMWOOD phase II randomized-controlled trial

Article

作者: Demir, Münevver ; Bonder, Alan ; Jaitly, Seema ; Gilroy, Richard ; Patel, Bhaktasharan C ; Shiffman, Mitchell L ; Bowlus, Christopher L ; Cazzagon, Nora ; Trivedi, Palak J ; Jacobson, Ira M ; Ko, Hin Hin ; Kugelmas, Marcelo ; Cheent, Kuldeep ; Olveira, Antonio ; Turnes, Juan ; Thorburn, Douglas ; Mukhopadhya, Ashis ; Kowdley, Kris V ; Gomes da Silva, Hugo ; Bilir, Bahri M ; Cortez-Pinto, Helena ; Dill, Michael T ; Levy, Cynthia ; Kallis, Yiannis ; Pietrangelo, Antonello ; Miller, Benjamin ; Abouda, George F ; Eksteen, Bertus ; Pradhan, Faruq ; Milligan, Claire ; Luketic, Velimir ; Montano-Loza, Aldo J ; Mangia, Alessandra ; Salcedo, Magdalena ; Sprinzl, Kathrin ; Zein, Claudia O ; Swann, Rachael ; Thuluvath, Paul J ; Chandok, Natasha ; Fenkel, Jonathan M ; Tavenard, Aude ; Campos-Varela, Isabel

BACKGROUND & AIMS:

Primary sclerosing cholangitis (PSC) is a rare, chronic liver disease. Elafibranor, a dual peroxisome proliferator-activated receptor-α/δ agonist, was investigated in the phase II ELMWOOD trial (NCT05627362).

METHODS:

This 12-week, double-blind trial enrolled adults with PSC and alkaline phosphatase (ALP) ≥1.5× the upper limit of normal. The primary endpoint was elafibranor safety vs. placebo. Additional endpoints included relative mean change from baseline in ALP and enhanced liver fibrosis (ELF) score.

RESULTS:

A total of 68 participants (male: 54.4%; mean age: 46.3 years; inflammatory bowel disease: 55.9%) were randomized to elafibranor 80 mg (n = 22), elafibranor 120 mg (n = 23), or placebo (n = 23). At baseline, 70.6% were on ursodeoxycholic acid, 48.5% had ELF scores >9.8, and the mean ALP level was 369.5 U/L. At Week 12, rates of treatment-emergent adverse events (TEAEs) and TEAEs leading to discontinuation in participants on elafibranor 80 mg, 120 mg, and placebo were 68.2%, 78.3%, and 69.6%, and 4.5%, 4.3%, and 8.7%, respectively. Serious TEAEs occurred only in participants on placebo (4.3%). Participants on elafibranor 80 mg and 120 mg had reductions in ALP vs. placebo (least squares mean treatment difference [95% CI]: -35.3% [-49.2, -21.4] and -54.7% [-68.3, -41.0], respectively). ALP normalization occurred only in participants on elafibranor 80 mg (9.1%) and 120 mg (17.4%). The LS mean treatment differences (95% CI) in change from baseline in ELF scores in participants on elafibranor 80 mg and 120 mg vs. placebo were -0.19 (-0.52, +0.15) and -0.28 (-0.62, +0.06), respectively.

CONCLUSIONS:

Elafibranor was well tolerated in people with PSC and associated with greater biochemical improvements over 12 weeks compared with placebo. A greater magnitude of response was observed with elafibranor 120 mg compared with 80 mg.

IMPACT AND IMPLICATIONS:

For people with primary sclerosing cholangitis (PSC), there is a need for a well-tolerated and effective treatment that will enhance quality of life, prevent disease progression, and improve long-term outcomes. Here, we present results from the double-blind period of the phase II ELMWOOD trial in PSC, wherein elafibranor, a peroxisome proliferator-activated receptor-α/δ agonist, demonstrated a favorable safety profile, provided greater biochemical improvements over 12 weeks compared with placebo, and appeared to stabilize markers of fibrosis and improve pruritus. These findings support larger and longer term investigations of elafibranor to explore its therapeutic potential as a treatment for people with PSC.

2025-11-01·JOURNAL OF MOLECULAR GRAPHICS & MODELLING

Unveiling the therapeutic potential of artopetelin flavonoids through computational approaches as peroxisome proliferator-activated receptor-delta (PPARδ) agonists

Article

作者: Poudel, Manila ; Parajuli, Nirmal ; Tamang, Ashika ; Bharati, Samjhana ; Maharjan, Binita ; Shrestha, Aakar ; Shrestha, Ram Lal Swagat ; Subin, Jhashanath Adhikari ; Shiva, M C ; Marasini, Bishnu P ; Shrestha, Timila

Diabetes mellitus is a growing global health concern, with peroxisome proliferator-activated receptor-delta (PPARδ) emerging as a promising therapeutic target due to its role in glucose regulation. Flavonoids, a class of plant-derived bioactive compounds, are known for their anti-diabetic properties. The present study aims to explore the potential of artopetelin flavonoids as PPARδ activators using a range of computational approaches. Molecular docking (MD) calculations identified six artopetelin ligands with binding affinities surpassing those of the native ligand (-10.4 kcal/mol) and the reference drugs such as elafibranor (-10.0 kcal/mol) and seladelpar (-9.8 kcal/mol). The highest binding affinity was obtained for artopetelin A, with a value of -11.8 kcal/mol. All candidates, except artopetelin B, were bound at the receptor's catalytic site, suggesting potential for competitive activation. Molecular dynamics simulations (MDS) of the top five complexes revealed structural stability, with consistent root mean square deviation (RMSD) profiles and stable hydrogen bonding patterns. Gibbs free energy changes (ΔG_BFE < 0) confirmed the sustained thermodynamic spontaneity of complex formation reactions where the values ranged from -39.88 to -26.66 kcal/mol. Pharmacokinetic predictions via pkCSM indicated favorable drug-likeness and ADMET profiles. These preliminary in silico findings highlight the strong potential of top five artopetelin flavonoids as PPARδ activators for type 2 diabetes management, underscoring their promise as plant-derived therapeutic agents and warranting further experimental validation.

2025-10-09·Cureus Journal of Medical Science

Beyond Ursodeoxycholic Acid: A Comprehensive Review of Second-Line Agents in Primary Biliary Cholangitis

Review

作者: Morris, Liam ; Lakshmanan, Vijay

Primary biliary cholangitis (PBC) is a chronic autoimmune liver disease characterised by progressive bile duct injury and cholestasis. It predominantly affects middle-aged women and typically presents with symptoms such as fatigue, pruritus, and deranged liver enzymes. While ursodeoxycholic acid (UDCA) remains the first-line therapy, a significant proportion of patients fail to achieve adequate biochemical response, leaving them vulnerable to disease progression. Until recently, treatment options for these individuals in the United Kingdom (UK) were limited. However, the therapeutic landscape is evolving with the recent approval of elafibranor and seladelpar, offering new hope for patients and clinicians alike. This review highlights key characteristics of these emerging second-line agents, including their mechanisms of action, administration, safety profiles, and regulatory status in the UK. Special attention is given to the clinical implications of their approval and accessibility within NHS pathways. In addition to disease-modifying therapies, adjunctive strategies for symptom control, particularly for pruritus and fatigue, are also discussed, along with a brief overview of future therapeutic directions. By summarising the expanding treatment arsenal, this review aims to support evidence-informed decision-making and promote timely specialist referral in patients with suboptimal response to UDCA.

290

项与 Elafibranor 相关的新闻（医药）

2025-12-30

·广州天润药业

40% 患者需求未满足！这款加速获批的罕见病新药有何硬实力？

省时总结版依非兰诺（Elafibranor）是一款具有创新作用机制的双重PPARα/δ激动剂，凭借在原发性胆汁性胆管炎（PBC）领域的优异临床数据，于2024年在全球多个主要市场获批上市。其良好的药代动力学特性和可接受的安全性，使其成为对标准疗法UDCA应答不佳或不耐受的PBC患者的一个重要治疗选择。随着其商业化的推进和确认性研究的深入，Elafibranor有望在肝病治疗领域占据重要地位，并可能在未来探索更广泛的适应症。往下读了解更多资讯市场规模依非兰诺片（商品名：Iqirvo），最早于2024年6月获美国FDA加速批准。 2024年9月获欧盟EMA附条件上市许可，在这两个地区均需在后续提供进一步的临床数据来确认其长期临床获益。此外，Iqirvo已在英国、澳大利亚、加拿大等地区获批上市，还有多个国家和地区在进行临床试验。根据Ipsen财务报告显示，2024年Iqirvo的全球销售总额达到了2190万欧元。 2019年上海拓臻生物取得了依非兰诺大中华区独家产品授权，目前尚未查询到该品种的国内临床进展。市场前景尽管PBC是罕见病，但目前治疗需求远未满足（约40%患者对一线治疗不应答），新药获批有望驱动市场增长。该市场已形成刚需、高价值的“小而美”格局。竞争格局熊去氧胆酸（UDCA）是治疗PBC的一线治疗药物，对于许多对一线标准疗法UDCA应答不佳或不耐受的PBC患者而言，临床上存在着巨大的未满足需求。当前PBC的二线治疗药物主要包括奥贝胆酸及贝特类药物等。值得一提的是，近一年内奥贝胆酸原研药由于安全性问题已在欧美市场撤市。优势与机遇地位认证在美国具有first-in-class地位，获FDA突破性治疗认定和孤儿药认定。机制明确，多效合一 PPAR双激动剂的作用机制在代谢、炎症、纤维化等多环节有效，理论优势明显。罕见病切入，加速上市在美欧加速获批，临床有效性和短期安全性得到监管机构认可，建立了市场基础，为后续开发铺平了道路。拓展空间大 PBC存在未满足的医疗需求，且其作用机制支持向NASH、酒精性肝病等更大患者人群拓展。挑战与风险 PBC领域的深化研究当前在美国和欧盟的批准均为有条件批准，目前数据来自约1年的临床试验，长期用药的心血管、代谢等方面风险需进一步评估，这将是其能否获得完全批准并巩固市场地位的关键。市场竞争大肝病领域新药研发竞争激烈，在刚上市即需面对其他靶点药物的挑战，市场渗透压力大。商业不确定性作为新上市药物，其市场渗透率、定价和医保覆盖情况仍在形成中。专利分析根据药智网查询到的专利数据显示，原研在国内并未进行化合物或制剂相关的专利申请。技术分析 1.参考配方及原辅包推荐参考FDA原研说明书，推荐处方如下： 2.工艺分析查询到原料在pH值范围1.2至6.8的水性介质中几乎不溶；在pH值为7.5时，其溶解度非常低。结合原研的处方组成，可推测其采用湿法制粒工艺。【免责声明】文章只做科普分享，涉及的药物仅用于科学原理阐释，不构成任何具体产品推广或用药建议。申请样品想试用我们的样品吗？识别左侧二维码申请吧！

2025-12-25

·秀刀师XDS

柳叶刀综述：代谢功能障碍相关脂肪性肝炎的治疗进展

1. 流行病学与疾病进程代谢功能障碍相关脂肪性肝病（MASLD），旧称非酒精性脂肪性肝病（NAFLD），全球患病率估计已达38.2%，其自然病程为从相对良性的肝脏脂肪变性发展到具有进行性特征的代谢功能障碍相关脂肪性肝炎（MASH），后者的病理特征为肝细胞损伤、炎症和纤维化。大约20%的脂肪变性患者会发展为MASH，其中又有约20%可能进展为肝硬化。肝硬化患者每年发生肝细胞癌的风险约为1.5–2%。肝纤维化分期是预测肝脏相关结局的独立指标。纤维化进展，特别是从F2期开始（即“高危MASH”），与失代偿和死亡风险显著增加相关。 MASLD患者的主要死因依次为心血管疾病、肝外癌症和肝硬化相关并发症，凸显了其作为系统性代谢疾病的本质。 2. 现有管理与治疗方法 MASH的基础管理依赖于生活方式干预，包括减肥、地中海饮食模式和体育锻炼。然而，只有10-20%的个体能够长期维持必要的体重减轻水平，成功率有限。在Resmetirom和Semaglutide获批之前，仅有维生素E和吡格列酮被专业指南建议用于部分MASH患者，属于超适应症使用。减重手术可显著改善MASH的组织学特征和纤维化，但其应用受限于手术可及性以及手术相关的并发症。随着高效减重药物（如GLP-1受体激动剂）的出现，患者接受手术的意愿可能降低。3. 治疗领域的重大突破在经历了包括Selonsertib、Emricasan和Elafibranor在内的多种药物试验失败后，MASH治疗在2024-2025年迎来了两个里程碑式的药物批准。 Resmetirom 机制：一种每日口服一次的肝脏靶向性甲状腺激素受体-β（THR-β）激动剂。它能选择性激活肝脏中的THR-β，从而增强肝脏脂肪酸氧化，降低脂毒性，并改善血脂水平。关键试验：MAESTRO-NASH（3期）。疗效数据：治疗52周后，与安慰剂组（9.7%）相比，80mg和100mg剂量组分别有25.9%和29.9%的患者实现MASH消退且纤维化无恶化。在纤维化改善方面，两个剂量组分别为24.2%和25.9%，而安慰剂组为14.2%。批准状态：于2024年3月获美国FDA批准，2025年8月获EMA批准，用于治疗伴有F2至F3期纤维化的MASH。 Semaglutide 机制：一种每周皮下注射一次的GLP-1受体激动剂。由于肝细胞不表达GLP-1受体，其对肝脏的改善作用被认为是间接的，主要通过抑制食欲、减轻体重和改善全身代谢实现。关键试验：ESSENCE（3期）。疗效数据：治疗72周后，62.9%的Semaglutide组患者实现MASH消退且纤维化无恶化，而安慰剂组为34.1%。37.0%的治疗组患者实现纤维化改善且MASH无恶化，安慰剂组为22.5%。批准状态：于2025年8月获得FDA的加速批准，用于治疗伴有中度至重度纤维化的MASH。这两款药物批准是基于肝脏的组织学终点，这两项试验仍在进行中，已评估药物治疗对肝硬化进展、肝癌预防等长期临床结局的影响。4. 研发中的新兴疗法 MASH的复杂病理生理学催生了多样化的药物研发管线，主要可分为作用于代谢的药物和肝脏特异性药物。作用于代谢的药物 PPAR激动剂：通过激活过氧化物酶体增殖物激活受体（PPARs）来调节脂质代谢和炎症。 GLP-1受体激动剂及其组合：利用肠促胰素的多效性来改善血糖、减轻体重和改善肝脏组织学。 FGF21类似物：成纤维细胞生长因子21是一种代谢应激激素，可增强脂肪酸氧化并提高胰岛素敏感性。肝脏特异性药物与基因疗法 THR-β激动剂：VK2809机制与Resmetirom类似，在2b期VOYAGE研究中也取得了积极结果，预计将进入3期试验。基因疗法：靶向与MASLD风险相关的基因变异。其他机制：包括磷酸二酯酶抑制剂（ZSP1601）、A3腺苷受体激动剂（Namodenoson）和DGAT2抑制剂（ION224）等，均处于2期开发阶段。 5. 联合疗法与代偿期肝硬化联合疗法由于MASH病理机制复杂，联合用药被认为是提高疗效的潜在策略。然而，临床试验结果好坏参半。例如，TANDEM（Tropifexor + Cenicriviroc）和ATLAS研究均未达到主要终点。但近期一项Lanifibranor联合Empagliflozin（SGLT-2抑制剂）的2期研究在改善非侵入性肝脏标志物方面取得了积极成果。将作用于全身代谢的药物与肝脏靶向药物相结合，是未来联合治疗的合理方向。 MASH代偿期肝硬化为已发展至肝硬化的患者开发疗法是当务之急，但挑战巨大。多项针对肝硬化患者的试验（包括Semaglutide、Selonsertib等）均以失败告终。近期研究带来了一些新启示：非侵入性标志物的价值：研究发现纤维化消退与临床结局改善相关，且增强型肝纤维化（ELF）评分的变化与组织学改善有良好的一致性，支持将NITs作为肝硬化试验的替代终点。更长的治疗周期：Efruxifermin在代偿期肝硬化患者中的SYMMETRY研究在延长至96周后，观察到组织学纤维化分期和NITs均有显著改善，提示肝硬化治疗可能需要更长的观察时间。 6. 未来挑战与展望随着新药进入临床使用，两大实践性问题凸显。如何在临床实践中识别合适的患者？新药获批的标签并未强制要求肝活检，这表明监管机构认可了使用非侵入性检测（NITs）来筛选患者。然而，挑战在于：阈值问题：MAESTRO-NASH试验入组患者的基线FIB-4和ELF评分均值落在人群水平的“不确定”范围内，提示广泛应用的转诊阈值可能排除部分符合治疗条件的患者。种族差异：NITs在不同种族人群中的表现存在差异，统一阈值可能导致对某些人群的过度或不足治疗。美国MASLD专家组建议，在使用Resmetirom时，不应单独使用FIB-4评分，而应结合多种NITs（如VCTE、MRE、ELF等）来综合判断患者是否符合F2/F3期纤维化的标准。如何评估药物的真实世界疗效？由于缺乏长期疗效数据，如何监测治疗反应尚不明确。 Resmetirom的监测：基于12个月数据，专家组提出了一些潜在的监测指标。例如，治疗12个月后，若肝脏硬度（VCTE）增加>30%，或肝酶增加>20%，或肝脏脂肪含量（MRI-PDFF）相对基线降低<30%，可能预示着治疗无反应，应考虑停药。 Semaglutide的监测：其疗效与体重减轻密切相关。但体重减轻通常在52周内达到平台期，此后药物对肝脏的长期影响以及如何通过NITs进行评估，仍然未知。 MASH药物治疗的最新进展令人振奋，但是任何单一药物都不太可能成为解决MASH问题的“万能药”。生活方式干预、食品技术、社会变革和政府政策在疾病防控中仍扮演着不可或缺的角色。当前，长期疗效和成本效益数据的缺乏限制了对新药卫生经济学影响的评估。而且Resmetirom和Semaglutide的早期积极信号是否能在长期临床结局研究中得到验证仍未可知。未来的研究应关注将创新疗法与生活方式干预和稳健的医疗护理路径相结合，以应对真实世界中的挑战。阅读原文： Li W, Alazawi W, Loomba R. Current and emerging therapeutic landscape for metabolic dysfunction-associated steatohepatitis. Lancet Gastroenterol Hepatol. 2025 Nov 27:S2468-1253(25)00260-2. doi: 10.1016/S2468-1253(25)00260-2 . Epub ahead of print. PMID: 41319672. 由于公众号平台的最新推荐机制，如您想第一时间收到更新的文章，请将公众号加入星标，如下图所示：

2025-12-22

·EndPoints

Boehringer taps Rectify to create pills for chronic kidney disease

Boehringer Ingelheim will work with a Boston startup to develop oral medicines for chronic kidney diseases in a partnership worth up to $448 million in biobucks. The move boosts the profile of the nimble Rectify Pharma, which has not needed to raise money since its $100 million launch in September 2021, CEO Rajesh Devraj said in an interview. The biotech, founded by three-decade Vertex veteran Jonathan Moore, is backed by Atlas Venture, Omega Funds, Forbion and Longwood Fund. Upfront terms for the Monday morning collaboration were kept under wraps. The tie-up represents Rectify’s first pharma partnership, and talks between the duo have been going on for about two years, Devraj said. The partnership revolves around the ABCC6 protein. That protein is critical to regulating calcification. Lower amounts of the protein are found in patients with CKD and rare genetic conditions such as pseudoxanthoma elasticum (PXE) and generalized arterial calcification of infancy (GACI), Rectify said. Rectify could help broaden Boehringer’s established presence in the chronic kidney disease field, which includes Jardiance, its approved medicine in collaboration with Eli Lilly. Boehringer also has an experimental aldosterone synthase inhibitor in combination with Jardiance in Phase 3 testing. The drugmaker has made cardio-renal-metabolic a core focus, with obesity as a key part of that R&D apparatus. Rectify adds to a flurry of biotech partnerships inked by Boehringer since the beginning of August. The family-owned German drug giant has made alliances with CDR-Life , Kyowa Kirin , AimedBio , Quantro Therapeutics and Palatin Technologies . Meanwhile, it has dropped some programs, including a schizophrenia drug from Nxera Pharma. Rectify is developing a pill that targets the ABC transporters ABCB4 and BSEP. The ABCB4 protein “addresses bile homeostasis,” and BSEP is the “most important transporter that excretes bile acids away from the liver,” Devraj said. The near-term goal is to send the asset into Phase 1 in healthy volunteers “very early next year” and then into Phase 1b for primary sclerosing cholangitis, or PSC, Devraj said. The chronic liver disease leads to inflamed bile ducts. Since there are no FDA-approved medicines for the condition, “the chances are high” for an accelerated approval route for Rectify’s unnamed drug prospect, Devraj said. But he said it’s yet to be determined what the exact path the medicine will take. There’s precedent in an adjacent condition called PBC, or primary biliary cholangitis. In 2024, Ipsen’s Iqirvo and Gilead’s Livdelzi both gained FDA accelerated approval for PBC, and other drugmakers are looking to get onto the market, as well. Intercept, meanwhile, is pulling its PBC drug from the market. PBC could be one of the indications Rectify explores for its drug candidate after getting into PSC. The medicine could be a “pipeline-in-a-pill” product, the CEO said. MASH could be another area Rectify explores, according to its website. Rectify is also expanding the “remit” of its platform to “other membrane proteins” beyond ABC transporters, Devraj said. “We found this very unique hot spot in these membrane proteins that we can plug and play, rinse and repeat,” the CEO said. “You build a platform. The question is, can you deploy it at scale? Can you then transfer it from one set of targets to the other? And, three, can you do it repeatedly and consistently?” With the Boehringer pact and remaining Series A funds, Rectify can answer some of those questions. The company has enough runway to get to clinical proof-of-mechanism with the PSC drug, Devraj said.

加速审批临床3期上市批准

100 项与 Elafibranor 相关的药物交易

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外链

KEGG	Wiki	ATC	Drug Bank
D11208	Elafibranor	-	DB05187

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
原发性胆汁性胆管炎	美国	Ipsen Biopharmaceuticals, Inc.	2024-06-10

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
纤维化	临床3期	美国	Genfit SA	2016-03-01
纤维化	临床3期	阿根廷	Genfit SA	2016-03-01
纤维化	临床3期	澳大利亚	Genfit SA	2016-03-01
纤维化	临床3期	比利时	Genfit SA	2016-03-01
纤维化	临床3期	加拿大	Genfit SA	2016-03-01
纤维化	临床3期	智利	Genfit SA	2016-03-01
纤维化	临床3期	哥伦比亚	Genfit SA	2016-03-01
纤维化	临床3期	捷克	Genfit SA	2016-03-01
纤维化	临床3期	丹麦	Genfit SA	2016-03-01
纤维化	临床3期	芬兰	Genfit SA	2016-03-01

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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


NCT05627362 (ELMWOOD, Pubmed \| J Hepatol)	临床2期	原发性硬化性胆管炎 alkaline phosphatase (ALP)	68	Elafibranor 80 mg	積鹹積襯顧壓壓觸廠醖(淵鑰鹹選衊鏇夢範觸鹹) = 繭網顧鬱遞鏇糧鹽鏇遞簾網衊製顧選艱繭蓋憲 (鏇網憲齋願顧餘糧憲壓 ) 更多	积极	2025-05-01
				Elafibranor 120 mg	積鹹積襯顧壓壓觸廠醖(淵鑰鹹選衊鏇夢範觸鹹) = 艱壓簾觸艱鏇鬱築夢淵簾網衊製顧選艱繭蓋憲 (鏇網憲齋願顧餘糧憲壓 ) 更多
NCT05627362 (ELMWOOD, NEWS) 人工标引	临床2期	原发性硬化性胆管炎	68	elafibranor	構網夢窪獵鹹簾窪獵構(鑰鹽夢衊鏇鹽壓鑰願餘) = Data from ELMWOOD demonstrated a positive safety and tolerability profile. 糧憲鏇窪獵醖壓壓醖願 (憲範築繭鏇願簾願鹹艱 ) 更多	积极	2025-04-25
				Placebo
NCT04526665 (ELATIVE, CTgov)	临床3期	原发性胆汁性胆管炎	161	Placebo	網繭糧鏇鑰願廠糧艱蓋 = 膚範蓋選餘艱襯襯獵夢遞憲積獵範鑰窪簾艱製 (夢遞餘齋鬱顧憲艱鏇壓, 範鹹窪築遞壓淵醖獵簾 ~ 齋選製餘鹽鹹願鬱鏇構) 更多	-	2024-09-05
NCT04526665 (FDA_CDER) 人工标引	临床3期	原发性胆汁性胆管炎	161	IQIRVO 80mg Once Daily	淵廠襯醖餘積鹽膚窪觸(製鹽獵廠蓋構願鏇獵鏇) = 網壓廠糧簾鑰繭壓鑰淵齋鹹壓鹽製顧廠蓋鹽艱 (窪夢鑰願獵顧艱顧範獵 ) 更多	积极	2024-06-10
				Placebo	淵廠襯醖餘積鹽膚窪觸(製鹽獵廠蓋構願鏇獵鏇) = 齋築壓遞淵製範網鬱衊齋鹹壓鹽製顧廠蓋鹽艱 (窪夢鑰願獵顧艱顧範獵 ) 更多
NCT04526665 (ELATIVE, EASL2024) 人工标引	临床3期	原发性胆汁性胆管炎	161	Elafibranor	鹽鹽簾網遞艱餘遞選遞(襯淵齋壓廠獵淵窪遞壓) = 獵獵鬱選齋築觸憲鑰膚顧窪蓋遞醖壓鑰淵艱鏇 (範製膚範鏇齋製夢夢範 ) 更多	积极	2024-06-01
				Placebo	糧窪窪襯獵鹽築繭鏇鬱(範範蓋獵鏇繭糧構構製) = 構構窪襯繭壓襯鏇壓範鑰繭積製衊製齋構範餘 (壓鹽範遞觸醖簾網積憲 )
NCT04526665 (ELATIVE, Pubmed) 人工标引	临床3期	原发性胆汁性胆管炎	161	Elafibranor 80 mg	積夢鹽範築廠觸鏇獵齋(網憲製艱窪壓蓋襯獵觸) = 夢窪遞壓鹽遞積網範鹽顧窪廠範積糧遞鹹蓋鹹 (網窪獵餘選蓋壓襯窪餘 ) 更多	积极	2024-02-29
				Placebo	積夢鹽範築廠觸鏇獵齋(網憲製艱窪壓蓋襯獵觸) = 積膚膚醖醖積糧醖網襯顧窪廠範積糧遞鹹蓋鹹 (網窪獵餘選蓋壓襯窪餘 ) 更多
NEWS 人工标引	临床3期	原发性胆汁性胆管炎	161	elafibranor	構簾獵簾繭築壓選鏇膚(積築鏇選淵鏇蓋構夢糧): 3.0 更多	积极	2023-11-14
				placebo
NCT04526665 (ELATIVE, Biospace) 人工标引	临床3期	原发性胆汁性胆管炎	161	elafibranor 80mg	衊糧衊獵衊壓繭憲鏇淵(襯鑰觸築獵鏇願夢築製) = 餘衊製顧獵選鏇觸顧鑰願鑰獵夢憲膚選願餘遞 (遞願餘醖願構選淵醖鹹 )	积极	2023-09-20
				placebo	衊糧衊獵衊壓繭憲鏇淵(襯鑰觸築獵鏇願夢築製) = 構積膚顧糧鏇壓淵憲膚願鑰獵夢憲膚選願餘遞 (遞願餘醖願構選淵醖鹹 )
NCT03883607 (Open Label, Randomized, Multicenter Study of Elafibranor in Children With Nonalcoholic Steatohepatitis, Pubmed \| J Pediatr Gastroenterol Nutr)	临床2期	代谢功能障碍相关脂肪性肝炎 alanine aminotransferase (ALT)	-	Elafibranor 80 mg	淵構餘夢獵鏇選鑰壓鬱(夢鹽醖壓遞繭鬱遞齋餘) = 夢蓋範鹽襯齋範繭選壓憲艱艱選襯衊選餘艱蓋 (簾範獵窪選顧鑰繭襯窪, 13)	积极	2023-08-01
				Elafibranor 120 mg	淵構餘夢獵鏇選鑰壓鬱(夢鹽醖壓遞繭鬱遞齋餘) = 鑰鹹餘顧觸網餘鬱鹹憲憲艱艱選襯衊選餘艱蓋 (簾範獵窪選顧鑰繭襯窪, 20)
NCT01694849 (CTgov)	临床2期	代谢功能障碍相关脂肪性肝炎	275	GFT505 80mg (GFT505 80mg)	夢積構鏇蓋築積衊醖鬱(壓淵壓鹹鏇簾衊醖願鬱) = 獵鏇淵鹹築製鹽築艱餘構製網網衊淵顧廠糧蓋 (襯遞襯窪廠襯鹽淵範築, 1.33) 更多	-	2022-11-03
				GFT505 120mg (GFT505 120mg)	夢積構鏇蓋築積衊醖鬱(壓淵壓鹹鏇簾衊醖願鬱) = 憲壓鬱廠鏇繭顧製簾鑰構製網網衊淵顧廠糧蓋 (襯遞襯窪廠襯鹽淵範築, 1.68) 更多