. Positive results from the global ElevAATe phase 2 study (clinical study identifier:

。ElevAATe 全球二期研究（临床研究标识符：

NCT05856331

NCT05856331

) showed that efdoralprin alfa (SAR447537, formerly known as INBRX-101), met all primary and key secondary endpoints when dosed every three weeks (Q3W) or four weeks (Q4W) in adults with alpha-1 antitrypsin deficiency (AATD) emphysema, a rare disease. Efdoralprin alfa is an investigational recombinant human alpha-1 antitrypsin (AAT)-Fc fusion protein.

）显示，Efdoralprin alfa（SAR447537，曾用名 INBRX-101）在每三周（Q3W）或每四周（Q4W）给药一次的情况下，达到了所有主要和关键次要终点，用于治疗患有α1-抗胰蛋白酶缺乏症（AATD）肺气肿的成人患者，这是一种罕见病。Efdoralprin alfa 是一种研究中的重组人α1-抗胰蛋白酶（AAT）-Fc融合蛋白。

It demonstrated a statistically significant greater mean increase in functional AAT levels within normal range as measured by trough concentrations at steady state compared to those receiving weekly plasma-derived augmentation therapy at week 32 [p<0.0001]. The study also met key secondary endpoints, demonstrating superior mean increase in fAAT average concentration as well as higher percentage of days above the lower limit of the normal range for both Q3W and Q4W dosing..

它在稳态下通过谷浓度测量的功能性AAT水平的平均增加，在正常范围内显示出统计学上显著高于接受每周一次血浆衍生增强疗法的患者（第32周，p<0.0001）。该研究还达到了关键的次要终点，证明了fAAT平均浓度的优越平均增幅，以及在Q3W和Q4W剂量下高于正常范围下限的天数百分比更高。

The recombinant efdoralprin alfa was well tolerated with a similar adverse event profile to plasma-derived therapy. Additional safety follow-up will be assessed in the ElevAATe OLE phase 2 study (clinical study identifier:

重组eftoralprin alfa耐受性良好，不良事件特征与血浆源性疗法相似。 ElevAATe OLE 2期研究（临床研究标识符：）将进一步评估安全性随访。

NCT05897424

NCT05897424

“These data demonstrate that efdoralprin alfa achieved consistently higher normal functional AAT levels, with less frequent dosing, compared to a current standard of care,”

“这些数据表明，与目前的标准治疗相比，efdoralprin alfa 能够以更少的给药频率持续达到更高的正常功能性 AAT 水平。”

Christopher Corsico

克里斯托弗·科西科

, Global Head of Development at Sanofi.

赛诺菲全球开发主管。

“This is particularly meaningful as maintaining protective protein levels is the cornerstone of pulmonary management of AATD and currently available treatments require weekly therapeutic infusions. The ElevAATe results represent the potential for efdoralprin alfa to be a restorative recombinant therapeutic option for the AATD community, reinforcing our commitment to develop treatments for both rare and respiratory conditions with great unmet medical need.” .

“这一点尤为重要，因为维持保护性蛋白质水平是AATD肺部管理的基石，而目前可用的治疗方法需要每周进行治疗性输注。ElevAATe研究结果代表了efdoralprin alfa有望成为AATD群体的一种修复性重组治疗选择，进一步巩固了我们为罕见病和呼吸系统疾病开发治疗方案的承诺，这些疾病存在巨大的未满足医疗需求。”

“AATD is a debilitating condition that can be challenging to treat,”

“AATD 是一种令人衰弱的疾病，治疗起来可能具有挑战性，”

Igor Barjaktarevic

伊戈尔·巴尔贾克塔雷维奇

, MD, PhD, Associate Professor, David Geffen School of Medicine at UCLA and primary investigator on the ElevAATe phase 2 study.

医学博士，哲学博士，加州大学洛杉矶分校大卫·格芬医学院副教授，ElevAATe 2期研究的首席研究员。

“Achieving and maintaining normal AAT levels with less frequent dosing and with complete independence from blood donation programs would be a welcome change to the current treatment experience for people living with AATD. With the current standard of care, patients reach but do not maintain normal protein levels between the infusions, leaving a remaining unmet need.

“通过减少给药频率并完全摆脱献血计划来实现和维持正常的AAT水平，将是对目前AATD患者治疗体验的可喜改变。按照目前的护理标准，患者在输注之间能够达到但无法维持正常的蛋白质水平，这仍然是一个未满足的需求。

I’m encouraged by the ElevAATe trial results and what efdoralprin alfa could mean for the AATD community.” .

ElevAATe 试验结果让我备受鼓舞，efdoralprin alfa 可能对 AATD 患者群体产生重要意义。

Efdoralprin alfa was previously granted fast track and orphan drug designation by the US Food and Drug Administration (FDA) for the treatment of AATD emphysema. Efdoralprin alfa is currently under clinical investigation, and its safety and efficacy have not been evaluated by any regulatory authority.

Efdoralprin alfa 之前被美国食品药品监督管理局 (FDA) 授予快速通道和孤儿药资格，用于治疗 AATD 肺气肿。Efdoralprin alfa 目前正在临床研究中，其安全性和有效性尚未得到任何监管机构的评估。

Sanofi plans to present the data at a forthcoming medical meeting and engage with global regulatory authorities on the appropriate next steps..

赛诺菲计划在即将召开的医学会议上展示这些数据，并与全球监管机构就适当的下一步行动进行沟通。

About AATD

关于AATD

AATD is a rare, inherited disorder characterized by low levels or absence of AAT, a protein produced by the liver that protects the lungs from inflammation and damage.

AATD是一种罕见的遗传性疾病，其特征是AAT水平低或缺失，AAT是由肝脏产生的一种保护肺部免受炎症和损伤的蛋白质。

The disease causes progressive deterioration of the tissue of the lungs and liver.

该疾病会导致肺和肝组织的进行性恶化。

Without adequate AAT levels, affected individuals often experience lung damage and develop COPD, including emphysema, and in severe forms of the disease, patients can sometimes require lung transplantation. Plasma-derived therapies were introduced in 1987 to treat the condition but since then, no new therapies have been introduced.

没有足够的AAT水平，受影响的个体通常会出现肺损伤并发展为慢性阻塞性肺病（COPD），包括肺气肿，在严重的情况下，患者有时需要进行肺移植。1987年引入了血浆衍生疗法来治疗这种疾病，但自那时以来，尚未有新的疗法问世。

About 235,000 people worldwide live with AATD, with nearly 100,000 people in the US, but about 90% of individuals with AATD are likely undiagnosed..

全世界约有 23.5 万人患有 AATD，其中美国约有 10 万人，但大约 90% 的 AATD 患者可能尚未确诊。

About efdoralprin alfa

关于efdoralprin alfa

Efdoralprin alfa (SAR447537, formerly known as INBRX-101) is a recombinant human AAT-Fc fusion protein being investigated as a restorative therapy in adults with AATD emphysema, with Q3W or Q4W dosing. The investigational treatment is being studied to restore functional AAT levels to the normal range and inhibit neutrophil elastase, an enzyme that can cause lung tissue damage in patients with AATD.

Efdoralprin alfa（SAR447537，曾用名 INBRX-101）是一种重组人 AAT-Fc 融合蛋白，正在研究用于治疗患有 AATD 肺气肿的成人，采用每 3 周或每 4 周一次的给药方案。该研究性治疗旨在将功能性 AAT 水平恢复至正常范围，并抑制中性粒细胞弹性蛋白酶，这种酶可能导致 AATD 患者的肺组织损伤。

Efdoralprin alfa was granted fast track designation and orphan drug designation by the FDA for the treatment of AATD emphysema. .

Efdoralprin alfa 获得 FDA 授予的快速通道资格和孤儿药资格，用于治疗 AATD 肺气肿。

About ElevAATe

关于ElevAATe

The ElevAATe phase 2 study was a double-blind, randomized study evaluating efdoralprin alfa versus a standard of care plasma-derived augmentation therapy in patients with AATD emphysema. ​Ninety-seven patients were randomized 2:2:1 to receive efdoralprin alfa every three weeks or every four weeks, or plasma-derived augmentation therapy once weekly.

ElevAATe 第二阶段研究是一项双盲、随机研究，评估了efdoralprin alfa与标准的血浆源性增强疗法在AATD肺气肿患者中的疗效。97名患者被随机分为2:2:1的比例，分别每三周或每四周接受一次efdoralprin alfa治疗，或每周一次接受血浆源性增强疗法。

The primary endpoint was the mean change in average fAAT concentrations as measured from baseline to average serum trough fAAT concentrations at steady state in patients treated with efdoralprin alfa Q3W or Q4W compared with weekly plasma-derived augmentation therapy, following a treatment period of up to 32 weeks.

主要终点是从基线到稳态时平均血清谷浓度的平均 fAAT 浓度变化，在接受每3周或每4周一次 efdoralprin alfa 治疗的患者中，与每周一次血浆衍生增强疗法进行比较，治疗期最长为32周。

Key secondary endpoints included mean change in serum fAAT concentration from baseline to fAAT average concentrations at steady state and percentage of days that steady-state functional AAT levels were above the lower limit of the normal range..

关键的次要终点包括从基线到稳态的血清 fAAT 浓度平均变化，以及稳态功能性 AAT 水平高于正常范围下限的天数百分比。

About Sanofi

关于赛诺菲

Sanofi is an R&D driven, AI-powered biopharma company committed to improving people’s lives and delivering compelling growth. We apply our deep understanding of the immune system to invent medicines and vaccines that treat and protect millions of people around the world, with an innovative pipeline that could benefit millions more. Our team is guided by one purpose: we chase the miracles of science to improve people’s lives; this inspires us to drive progress and deliver positive impact for our people and the communities we serve, by addressing the most urgent healthcare, environmental, and societal challenges of our time..

赛诺菲是一家以研发为驱动、以人工智能为支撑的生物制药公司，致力于改善人们的生活并实现令人瞩目的增长。我们凭借对免疫系统的深刻理解，研发药物和疫苗，为全球数百万人提供治疗和保护，同时通过创新的研发管线，有望惠及更多人群。我们的团队秉承一个使命：追寻科学的奇迹以改善人们的生活；这激励我们不断推动进步，为员工及所服务的社区带来积极影响，直面当今最紧迫的医疗、环境和社会挑战。