PARIS, FRANCE, 18 November, 2024 Ipsen (Euronext: IPN; ADR: IPSEY) today announced data at the American Association for the Study of Liver Diseases (AASLD) assessing the long-term efficacy and safety of patients treated with Bylvay® from two Phase III open-label extension studies: late-breaking abstract (#5045) on PEDFIC 2 in Progressive Familial Intrahepatic Cholestasis (PFIC) and oral presentation ASSERT-EXT (#50) in Alagille syndrome (ALGS).

2024年11月18日，法国巴黎Ipsen（泛欧交易所：IPN；ADR：IPSEY）今天在美国肝病研究协会（AASLD）上公布了数据，评估了两项III期开放标签扩展研究中Bylvay®治疗患者的长期疗效和安全性：进行性家族性肝内胆汁淤积症（PFIC）中PEDFIC 2的晚期突破性摘要（#5045）和Alagille综合征（ALGS）中的口服ASSERT-EXT（#50）。

Sustained efficacy data and improvements in height, weight and sleep measures were observed for patients treated with Bylvay for at least 72 weeks in both rare cholestatic diseases..

在这两种罕见的胆汁淤积性疾病中，Bylvay治疗至少72周的患者观察到持续的疗效数据以及身高，体重和睡眠指标的改善。。

“We know from our work with patient communities that receiving a diagnosis of PFIC and ALGS can be overwhelming in a patient or caregivers’ life. Disease symptoms like severe itch can have an impact on the whole family,” said Sandra Silvestri, EVP Chief Medical Officer, Ipsen. “Data suggesting Bylvay-treated patients experienced sustained efficacy, and which support the safety and tolerability profile seen in previous clinical trials, are important.

Ipsen执行副总裁桑德拉·西尔维斯特里（SandraSilvestri）说：“我们从与患者社区的合作中了解到，接受PFIC和ALGS诊断可能会对患者或护理人员的生活产生巨大影响。严重瘙痒等疾病症状可能会影响整个家庭。”。“数据表明，接受Bylvay治疗的患者经历了持续的疗效，并且支持了先前临床试验中所见的安全性和耐受性特征，这一点很重要。

Ipsen is committed to being the leader across rare cholestatic liver diseases and we are just getting started.”.

Ipsen致力于成为罕见胆汁淤积性肝病的领导者，我们才刚刚开始。”。

PEDFIC 2 Study in PFIC

PFIC中的PEDFIC 2研究

“These open-label extension data from PEDFIC 2 suggest that the initial reduction in pruritus and in serum bile acid levels achieved following initiation of odevixibat are being sustained into the longer term,” said Dr. Richard J. Thompson, Professor of Molecular Hepatology, King’s College London and principal investigator of the PEDFIC 2 trial.

伦敦国王学院分子肝病学教授兼PEDFIC 2试验首席研究员Richard J.Thompson博士说：“这些来自PEDFIC 2的开放标签扩展数据表明，奥德维西巴特开始后瘙痒和血清胆汁酸水平的初步降低正在长期持续。”。

“We are also observing reductions in both pruritus and serum bile acid across a number of PFIC subtypes. This is important information for our understanding of the therapeutic management of our patients living with PFIC.” .

“我们还观察到许多PFIC亚型的瘙痒和血清胆汁酸均降低。这对于我们了解PFIC患者的治疗管理至关重要。”。

PEDFIC 2 was an open-label extension study (n=116; patients from PEDFIC 1 Bylvay and placebo cohorts at week 24, and new Bylvay-naïve patients of any age and PFIC subtype), evaluating the efficacy and safety of Bylvay through 72 weeks (n=83).1 The data showed a clinically meaningful 1-point reduction in pruritus score at week 72 in 42 percent of patients <18 years old with PFIC 1 and 2 who transitioned to Bylvay at 24 weeks (n=5/12) and 61 percent of patients with any type of PFIC and of any age excluding episodic (n=19/31).

PEDFIC 2是一项开放标签的扩展研究（n=116；第24周来自PEDFIC 1 Bylvay和安慰剂组的患者，以及任何年龄和PFIC亚型的新Bylvay初治患者），评估了Bylvay在72周内的疗效和安全性（n=83）。数据显示，在第72周，42%的18岁以下PFIC 1和2患者在24周时转变为Bylvay（n=5/12），61%的任何类型PFIC患者和任何年龄（不包括发作性）的患者瘙痒评分在临床上有意义降低1分（n=19/31）。

Rapid initial pruritus scores achieved by week 4 were sustained for patients who remained on treatment. At 72 weeks, the mean change in serum bile acid (sBA) levels from patients who transition to Bylvay at week 24 (n=15) was –104.00 µmol/L and Bylvay-treated patients (n=43) was -57.97 µmol/L . .

对于仍在接受治疗的患者，在第4周达到的快速初始瘙痒评分得以维持。在72周时，在第24周（n=15）过渡到Bylvay的患者血清胆汁酸（sBA）水平的平均变化为–104.00µmol/L，接受Bylvay治疗的患者（n=43）的平均变化为-57.97µmol/L。

Beyond the clinically meaningful and sustained improvements seen in pruritus and sBA levels, height, weight and sleep increases were reported at 72 weeks in Bylvay-treated patients. Most adverse events in Bylvay-treated patients over the duration of the study were reported as mild or moderate. The most common were gastrointestinal (17.2 percent; n=20/116), including diarrhea (12 percent; n=14/116).

除了瘙痒症和sBA水平的临床意义和持续改善外，据报道，接受Bylvay治疗的患者在72周时身高，体重和睡眠增加。在研究期间，接受Bylvay治疗的患者的大多数不良事件报告为轻度或中度。最常见的是胃肠道（17.2%；n=20/116），包括腹泻（12%；n=14/116）。

In two cases, diarrhea led to one treatment interruption and one discontinuation..

在两例中，腹泻导致一次治疗中断和一次停药。。

Assert-EXT Study in ALGS“The sustained improvements we’ve seen in Bylvay-treated individuals living with Alagille syndrome are encouraging,” said Dr. Nadia Ovchinsky, Chief, Division of Gastroenterology and Hepatology, Hassenfeld Children’s Hospital at NYU Langone, New York. and principal investigator of the ASSERT trial.

ALGS的Assert EXT研究“我们在接受Bylvay治疗的Alagille综合征患者中看到的持续改善令人鼓舞”，纽约州纽约大学兰贡分校哈森菲尔德儿童医院胃肠病和肝病科科长兼Assert试验首席研究员Nadia Ovchinsky博士说。

“These results not only show the potential to manage symptoms like pruritus, which can be extremely difficult for children and their parents to manage, but we’re also seeing a consistent safety profile over the longer term with sustained tolerability.”.

“这些结果不仅显示了控制瘙痒症等症状的潜力，这对儿童及其父母来说可能是极其困难的，而且我们还看到了长期持续耐受性的一致安全性。”。

In ASSERT-EXT, the open-label extension study (n=50) evaluating the long-term efficacy and safety of Bylvay in ALGS patients (ages 1-15.9 years) through 72 weeks (n=44), sustained improvements were observed in pruritus and sBA levels through 72 weeks.2 At week 72, 93 percent (n=28/30) of patients who received Bylvay throughout the 24 weeks ASSERT trial and 77 percent (n=10/13) of those who transitioned from placebo to Bylvay at week 24 experienced a clinically meaningful ≥1 point reduction in pruritus score.

在ASSERT-EXT中，开放标签扩展研究（n=50）评估了Bylvay在ALGS患者（年龄1-15.9岁）至72周（n=44）中的长期疗效和安全性，在72周内观察到瘙痒和sBA水平持续改善。在72周时，在24周ASSERT试验中接受Bylvay的患者中有93%（n=28/30），在24周时从安慰剂过渡到Bylvay的患者中有77%（n=10/13）的瘙痒评分降低≥1分。

Reductions in sBA levels were also observed in patients treated with Bylvay for 72 weeks showing a mean reduction of 124 µmol/L in those who continuously received Bylvay and a mean reduction of 139 µmol/L in patients who transitioned from placebo to Bylvay. Mean changes from baseline were observed in height (8.2 cm) and weight (2.8 kg) on continuous Bylvay use and for patients who transitioned from placebo to Bylvay, height (10.7 cm) and weight (3.3 kg) mean changes were also reported.

在接受Bylvay治疗72周的患者中也观察到sBA水平降低，持续接受Bylvay的患者平均降低124µmol/L，从安慰剂过渡到Bylvay的患者平均降低139µmol/L。在连续使用Bylvay时，观察到身高（8.2厘米）和体重（2.8公斤）与基线的平均变化，对于从安慰剂过渡到Bylvay的患者，也报告了身高（10.7厘米）和体重（3.3公斤）的平均变化。

Improvements in sleep were observed from weeks 24 to 72 across all four sleep parameters (n=43), including proportion of days seeing blood due to scratching, proportion of days needing help falling asleep, proportion of days needing soothing and daytime tiredness. Data supports the safety profile in the ASSERT clinical trial for Bylvay.

在所有四个睡眠参数（n=43）中，从第24周至第72周观察到睡眠改善，包括由于抓挠而看到血液的天数比例，需要帮助入睡的天数比例，需要缓解的天数比例和白天疲劳。数据支持Bylvay ASSERT临床试验的安全性。

Treatment emergent adverse event (TEAE) occurred in 18 percent (n=6/33) of patients who continuously received Bylvay and 41 percent (n=7/17) of patients who transitioned from placebo to Bylvay. Most adverse events were mild or moderate with diarrhea as the most common TEAE. One TEAE led to discontinuation..

持续接受Bylvay治疗的患者中有18%（n=6/33）发生治疗紧急不良事件（TEAE），从安慰剂过渡到Bylvay的患者中有41%（n=7/17）发生TEAE。。一次TEAE导致停药。。

About PFIC and ALGSPFIC is a group of rare genetic disorders in which bile acids build up in the liver, causing damage, which may result in liver failure. ALGS is also a rare genetic disorder, affecting multiple organs including the liver, heart, skeleton, eyes and kidneys. Without early diagnosis and effective management, people living with PFIC and ALGS may need a liver transplant.

关于PFIC和ALGSPFIC是一组罕见的遗传性疾病，胆汁酸在肝脏中积聚，引起损伤，可能导致肝衰竭。ALGS也是一种罕见的遗传性疾病，影响肝脏，心脏，骨骼，眼睛和肾脏等多个器官。。

Debilitating itch, caused as a result of the serum bile acid build up, is one of the most common symptoms of both PFIC and ALGS, significantly impacting sleep and daily activities and resulting in skin mutilation, loss of sleep, irritability, and poor attention..

血清胆汁酸积聚引起的衰弱性瘙痒是PFIC和ALGS最常见的症状之一，严重影响睡眠和日常活动，导致皮肤受损、睡眠不足、烦躁不安和注意力不集中。。

Bylvay (odevixibat) posters presented at AASLD

拜尔维（odevixibat）海报在AASLD展出

Abstract

摘要

Poster or Oral #

海报或口头#

Full title

完整标题

Authors

作者

ASSERT-EXT final results

ASSERT-EXT最终结果

Oral, Abstract Parallel, ePoster [50]Monday 18 November 11:45–12:00Human Cholestatic, PBC and other Biliary Disorders in Children and Adults

11月18日星期一11:45-12:00儿童和成人的胆汁淤积症、PBC和其他胆道疾病

ASSERT-EXT: Final data from an open-label, Phase 3 study of odevixibat in patients with Alagille syndrome

ASSERT-EXT:Alagille综合征患者奥德维西巴特开放标签3期研究的最终数据

Nadia Ovchinsky et al.

Nadia Ovchinsky等人。

Hepatic parameters with ODX in PFIC

PFIC中ODX的肝脏参数

Poster, Abstract [4277]Monday 18 November13:00–14:00Poster Session IV

海报，摘要[4277]11月18日星期一13:00–14:00海报第四课

Tassos Grammatikopoulos et al.

Tassos Grammatikopoulos等人。

Phase I DDI results

第一阶段DDI结果

Poster, Abstract [4280]Monday 18 November13:00–14:00Poster Session IV

海报，摘要[4280]11月18日星期一13:00–14:00海报第四课

A Phase 1, open-label, fixed-sequence, crossover study to evaluate the interaction of multiple-dose odevixibat with the pharmacokinetics of single-dose combined oral contraceptive steroids in healthy female participants

第一阶段，开放标签，固定序列，交叉研究，以评估健康女性参与者中多剂量奥迪昔布与单剂量联合口服避孕药类固醇药代动力学的相互作用

Florent Mazuir et al.

Florent-Mazuir等人。

PEDFIC1/2 OLE final results (LB)

PEDFIC1/2 OLE最终结果（LB）

Poster, Abstract [5045]Monday 18 November13:00–14:00Poster Session IV

海报，摘要[5045]11月18日星期一13:00–14:00海报第四课

Sustained, long-term efficacy and safety of odevixibat in patients with progressive familial intrahepatic cholestasis: Results from the PEDFIC2 Phase 3, open-label extension study

奥德维西巴特对进行性家族性肝内胆汁淤积症患者的持续，长期疗效和安全性：PEDFIC2 3期开放标签扩展研究的结果

Richard Thompson et al.

理查德·汤普森等人。

About Bylvay (odevixibat)Odevixibat is a once-daily non-systemic ileal bile acid transport (IBAT) inhibitor approved under the brand name Bylvay® in the U.S. as the first drug treatment option for patients 3 months of age and older living with cholestatic pruritus due to progressive familial intrahepatic cholestasis (PFIC).

关于Bylvay（odevixibat）odevixibat是一种每日一次的非全身性回肠胆汁酸转运（IBAT）抑制剂，在美国以Bylvay®品牌获得批准，是3个月及以上因进行性家族性肝内胆汁淤积（PFIC）而患有胆汁淤积性瘙痒症的患者的第一种药物治疗选择。

BYLVAY may not be effective in a subgroup of PFIC type 2 patients with specific ABCB11 variants resulting in non-functional or complete absence of the bile salt export pump protein..

BYLVAY可能对具有特定ABCB11变异的PFIC 2型患者亚组无效，导致胆汁盐输出泵蛋白无功能或完全缺失。。

Odevixibat was also approved in June 2021 in the E.U. under the brand name Bylvay®, as the first drug treatment option for all types of PFIC in patients aged 6 months or older. Bylvay has received orphan exclusivity for the treatment of PFIC in the U.S. and E.U.

2021年6月，奥德维西巴特也在欧盟以Bylvay®品牌获得批准，成为6个月或以上患者所有类型PFIC的第一种药物治疗选择。Bylvay在美国和欧盟获得了PFIC治疗的孤儿专有权。

In June 2023 Bylvay was approved in the U.S. for the treatment of cholestatic pruritus in patients from 12 months of age with Alagille syndrome (ALGS) and received orphan exclusivity for ALGS. In September 2024, odevixibat was approved in the E.U under the brand name Kayfanda® for the treatment of cholestatic pruritus in ALGS in patients aged 6 months or older..

2023年6月，Bylvay在美国被批准用于治疗12个月大的Alagille综合征（ALGS）患者的胆汁淤积性瘙痒症，并获得ALGS的孤儿专享权。2024年9月，奥德维西巴特在欧盟以Kayfanda®品牌获得批准，用于治疗6个月或以上患者的ALGS胆汁淤积性瘙痒症。。

IMPORTANT SAFETY INFORMATION – U.S.Warnings and Precautions:

重要安全信息–美国警告和注意事项：

Liver Test AbnormalitiesPatients who enrolled in PFIC and ALGS clinical trials had abnormal liver tests at baseline. In clinical trials, treatment-emergent elevations of liver tests or worsening of liver tests relative to baseline values were observed. Most abnormalities included elevations in aspartate aminotransferase (AST), alanine transaminase (ALT) in PFIC and ALGS, and total and direct bilirubin in PFIC clinical trials.

肝脏检查异常参加PFIC和ALGS临床试验的患者在基线时肝脏检查异常。在临床试验中，观察到治疗引起的肝脏检查升高或肝脏检查相对于基线值恶化。大多数异常包括PFIC和ALGS中天冬氨酸转氨酶（AST），丙氨酸转氨酶（ALT）升高，以及PFIC临床试验中的总胆红素和直接胆红素升高。

No patients permanently discontinued treatment due to liver test abnormalities..

没有患者因肝脏检查异常而永久停止治疗。。

Obtain baseline liver tests and monitor during treatment. Dose reduction or treatment interruption may be required if abnormalities occur. For persistent or recurrent liver test abnormalities, consider treatment discontinuation.

。如果发生异常，可能需要减少剂量或中断治疗。对于持续性或复发性肝功能异常，请考虑停止治疗。

Permanently discontinue Bylvay if a patient progresses to portal hypertension or experiences a hepatic decompensation event.

如果患者进展为门静脉高压症或发生肝代偿失调事件，则永久停用Bylvay。

DiarrheaDiarrhea occurred in both PFIC and ALGS clinical trials in BYLVAY-treated patients at a rate greater than placebo treated patients. If diarrhea occurs with use of BYLVAY, monitor for dehydration and treat promptly. Treatment interruption or discontinuation may be required for persistent diarrhea with no alternate etiology..

在接受BYLVAY治疗的患者的PFIC和ALGS临床试验中，腹泻发生率均高于安慰剂治疗的患者。如果使用BYLVAY导致腹泻，请监测脱水情况并及时治疗。对于没有其他病因的持续性腹泻，可能需要中断或停止治疗。。

Fat-Soluble Vitamin (FSV) DeficiencyFat-soluble vitamins (FSV) include vitamin A, D, E, and K. PFIC and ALGS patients can have FSV deficiency at baseline, as part of their disease. BYLVAY may affect absorption of fat-soluble vitamins.

脂溶性维生素（FSV）缺乏脂溶性维生素（FSV）包括维生素A，D，E和K。PFIC和ALGS患者在基线时可能患有FSV缺乏症，这是其疾病的一部分。BYLVAY可能会影响脂溶性维生素的吸收。

Obtain baseline levels and monitor during treatment, along with any clinical manifestations. Supplement if deficiency is observed. If FSV deficiency persists or worsens despite FSV supplementation, discontinue treatment.

获得基线水平并在治疗期间监测，以及任何临床表现。如果观察到缺陷，请补充。如果尽管补充了FSV，但FSV缺乏症仍然存在或恶化，请停止治疗。

ADVERSE REACTIONSALGS: The most common adverse reactions (>5%) are diarrhea, abdominal pain, hematoma, and decreased weight.PFIC: The most common adverse reactions (>2%) are diarrhea, liver test abnormalities, vomiting, abdominal pain, and fat-soluble vitamin deficiency.

。PFIC：最常见的不良反应（>2%）是腹泻，肝脏检查异常，呕吐，腹痛和脂溶性维生素缺乏症。

DRUG INTERACTIONSFor patients taking bile acid binding resins, take BYLVAY at least 4 hours before or 4 hours after administering, as bile acid binding resins may bind to and reduce BYLVAY efficacy.

药物相互作用对于服用胆汁酸结合树脂的患者，至少在给药前4小时或给药后4小时服用BYLVAY，因为胆汁酸结合树脂可能结合并降低BYLVAY疗效。

USE IN SPECIFIC POPULATIONSThere are no human data on BYLVAY use in pregnant persons to establish a drug-associated risk of major birth defects, miscarriage, or adverse developmental outcomes. Based on findings from animal reproduction studies, BYLVAY may cause cardiac malformations when a fetus is exposed during pregnancy..

在特定人群中使用没有关于孕妇使用BYLVAY来确定与药物相关的重大出生缺陷，流产或不良发育结局风险的人类数据。根据动物繁殖研究的结果，当胎儿在怀孕期间暴露时，BYLVAY可能会导致心脏畸形。。

There is a pregnancy safety study that monitors pregnancy outcomes in women exposed to BYLVAY during pregnancy. Pregnant women exposed to BYLVAY, or their healthcare providers, should report BYLVAY exposure by calling 1-855-463-5127.

有一项妊娠安全研究监测怀孕期间接触BYLVAY的女性的妊娠结局。接触BYLVAY的孕妇或其医疗保健提供者应致电1-855-463-5127报告BYLVAY暴露情况。

To report SUSPECTED ADVERSE REACTIONS, contact Ipsen Biopharmaceuticals, Inc. at +1-855-463-5127, or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.

要报告疑似不良反应，请联系Ipsen Biopharmaceuticals，Inc.，电话：+1-855-463-5127，或联系FDA，电话1-800-FDA-1088或www.FDA.gov/medwatch。

Indications and Usage U.S.Bylvay is an ileal bile acid transporter (IBAT) inhibitor indicated for the treatment of cholestatic pruritus in:Patients 12 months of age and older with Alagille syndrome (ALGS)Patients 3 months of age and older with progressive familial intrahepatic cholestasis (PFIC)

适应症和用法美国Bylvay是一种回肠胆汁酸转运蛋白（IBAT）抑制剂，用于治疗胆汁淤积性瘙痒症：12个月及以上的Alagille综合征（ALGS）患者3个月及以上的进行性家族性肝内胆汁淤积症（PFIC）

Please see full U.S. Prescribing Information.

请参阅完整的美国处方信息。

Indications of use E.U.Bylvay is indicated for the treatment of progressive familial intrahepatic cholestasis (PFIC) in patients aged 6 months or older. Please see full E.U. Prescribing Information.

E.U.Bylvay的使用指征适用于治疗6个月或以上患者的进行性家族性肝内胆汁淤积症（PFIC）。请参阅完整的欧盟处方信息。

Kayfanda is indicated for the treatment of cholestatic pruritus in Alagille syndrome (ALGS) in patients aged 6 months or older. Please see full E.U. Prescribing Information.

Kayfanda适用于治疗6个月或以上患者的Alagille综合征（ALGS）胆汁淤积性瘙痒症。请参阅完整的欧盟处方信息。

ENDS

结束

About PEDFIC 1 and 2PEDFIC 1 was a 24-week double-blind, randomized, placebo-controlled trial that evaluated the efficacy and tolerability of two doses of odevixibat in reducing pruritus and serum bile acid levels in children with PFIC 1 or 2. PEDFIC 2 is a 72-week open label extension trial, which consisted of children from PEDFIC 1 who received either Bylvay (cohort 1a) or placebo (cohort 1b) and a new cohort (2) of Bylvay-naïve patients of any age and PFIC subtype.

关于PEDFIC 1和2PEDFIC 1是一项为期24周的双盲，随机，安慰剂对照试验，该试验评估了两种剂量的奥得昔布在降低PFIC 1或2儿童瘙痒和血清胆汁酸水平方面的疗效和耐受性。。

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PEDFIC is the largest, global, Phase III trial ever conducted in PFIC. PEDFIC 1 (NCT03566238) was a 24-week double-blind, randomized (1:1:1), placebo-controlled trial that evaluated the efficacy and tolerability of two doses of odevixibat in reducing pruritus and serum bile acid levels in children with PFIC 1 or 2.

PEDFIC是PFIC有史以来规模最大的全球III期试验。。

Participants were randomly allocated to receive placebo (n=20), odevixibat 40 μg/kg (n=23), or odevixibat 120 μg/kg (n=19) once a day. The results were published in The Lancet.3.

参与者被随机分配每天一次接受安慰剂（n=20），奥迪昔布40μg/kg（n=23）或奥迪昔布120μg/kg（n=19）。研究结果发表在《柳叶刀》上。

PEDFIC 2 (NCT03659916), an open-label extension of PEDFIC 1, is a 72-week trial that aimed to evaluate the efficacy and tolerability of odevixibat 120 µg/kg once a day in patients with PFIC. Patients were divided into two cohorts: Cohort 1 (n=56) which consisted of children with PFIC 1 or 2 from PEDFIC 1 who received odevixibat (Cohort 1a: n= 37) or placebo (Cohort 1b: n=19), respectively, and Cohort 2 (n=60) which consisted of newly enrolled, odevixibat-naïve patients of any age and PFIC subtype.

PEDFIC 2（NCT03659916）是PEDFIC 1的开放标签扩展，是一项为期72周的试验，旨在评估PFIC患者每天一次120µg/kg奥地昔布的疗效和耐受性。患者分为两个队列：队列1（n=56），其中包括来自PEDFIC 1的PFIC 1或2的儿童，他们分别接受奥迪昔布（队列1a：n=37）或安慰剂（队列1b：n=19），以及队列2（n=60），其中包括新登记的任何年龄和PFIC亚型的奥迪昔布初治患者。

Interim results were published in The Journal of Hepatology.4.

中期结果发表在《肝病学杂志》上。

About ASSERT and ASSERT-EXTASSERT (NCT04674761) was a 24-week double-blind, randomized, placebo-controlled trial with an open-label long term extension. ASSERT evaluated the safety and efficacy of 120 µg /kg once-daily odevixibat vs placebo for the treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS).

关于ASSERT和ASSERT-EXTSSERT（NCT04674761）是一项为期24周的双盲，随机，安慰剂对照试验，具有开放标签的长期延长。ASSERT评估了每日一次120µg/kg奥迪昔布与安慰剂治疗Alagille综合征（ALGS）患者胆汁淤积性瘙痒症的安全性和有效性。

The trial enrolled 52 patients of any age with a genetically confirmed diagnosis of ALGS. The results were published in The Lancet.5.

该试验招募了52名经遗传学确诊为ALGS的任何年龄的患者。研究结果发表在《柳叶刀》上。

In ASSERT-EXT (NCT05035030), ASSERT’s ongoing open-label extension, all trial participants received 120 μg/kg of odevixibat once daily for 72-weeks after the double-blind treatment period completed. In both ASSERT and ASSERT-EXT, the investigators looked for changes in pruritus, serum bile acid concentrations, sleep, and treatment-emergent adverse events..

在ASSERT-EXT（NCT05035030）中，ASSERT正在进行的开放标签扩展，所有试验参与者在双盲治疗期结束后72周内每天一次接受120μg/kg的奥德维西巴特。在ASSERT和ASSERT-EXT中，研究人员寻找瘙痒，血清胆汁酸浓度，睡眠和治疗紧急不良事件的变化。。

About IpsenWe are a global biopharmaceutical company with a focus on bringing transformative medicines to patients in three therapeutic areas: Oncology, Rare Disease and Neuroscience.

关于IpsenWe是一家全球生物制药公司，专注于在三个治疗领域为患者带来变革性药物：肿瘤学，罕见病和神经科学。

Our pipeline is fueled by external innovation and supported by nearly 100 years of development experience and global hubs in the U.S., France and the U.K. Our teams in more than 40 countries and our partnerships around the world enable us to bring medicines to patients in more than 80 countries.

我们的渠道由外部创新推动，并由近100年的发展经验和美国、法国和英国的全球中心提供支持。我们在40多个国家的团队和我们在世界各地的合作伙伴使我们能够为80多个国家的患者提供药物。

Ipsen is listed in Paris (Euronext: IPN) and in the U.S. through a Sponsored Level I American Depositary Receipt program (ADR: IPSEY). For more information, visit ipsen.com

Ipsen通过赞助的一级美国存托凭证计划（ADR：IPSEY）在巴黎（泛欧交易所：IPN）和美国上市。有关更多信息，请访问ipsen.com