MARBURG, Germany, July 04, 2024 (GLOBE NEWSWIRE) -- Global biotechnology leader CSL Behring (ASX: CSL) today announced that two hemophilia B patients were treated with the gene therapy HEMGENIX® (etranacogene dezaparvovec) at Hemophilia Treatment Centers in France. This milestone achievement makes HEMGENIX® the first gene therapy administered as a treatment in a real-world setting for hemophilia B in Europe..

2024年7月4日，德国马尔堡（环球通讯社）--全球生物技术领导者CSL Behring（ASX:CSL）今天宣布，两名血友病B患者在法国血友病治疗中心接受了基因治疗HEMGENIX®（etranacogene dezaparvovec）治疗。这一里程碑式的成就使HEMGENIX®成为欧洲第一个在现实环境中治疗血友病B的基因疗法。。

HEMGENIX® is the first one-time gene therapy approved in Europe for the treatment of adults with severe and moderately severe hemophilia B, an inherited bleeding disorder caused by the lack of Factor IX (a protein needed to produce blood clots to stop bleeding). It is used in adults without a history of Factor IX inhibitors.1.

HEMGENIX®是欧洲首次批准用于治疗成人重度和中度重度血友病B的一次性基因疗法，血友病B是一种遗传性出血性疾病，由缺乏因子IX（产生血块止血所需的蛋白质）引起。它用于没有IX因子抑制剂病史的成年人。

Following European Commission approval, HEMGENIX® was the first ever therapy to be granted Direct Access in France2, thus enabling the first patients to be treated in Europe outside of the clinical program.

在欧盟委员会批准后，HEMGENIX®是法国有史以来第一个被允许直接进入的治疗方法，从而使第一批患者能够在临床计划之外的欧洲接受治疗。

Though effective, current therapies can be time intensive and require regular treatment that can have a substantial impact on a patient’s daily life.3 HEMGENIX® offers a one-time treatment, allowing people living with hemophilia B to produce their own Factor IX, which can lower the risk of bleeding.4.

虽然有效，但目前的治疗可能需要大量时间，需要定期治疗，这可能会对患者的日常生活产生重大影响。3 HEMGENIX®提供一次性治疗，使血友病B患者能够产生自己的IX因子，从而降低出血风险。

“Only a few decades ago, gene therapy for hemophilia was a distant concept, which has now become reality. Accordingly, the first two patients treated with HEMGENIX® since receiving European approval is a major accomplishment and a testament to the joint commitment of the hemophilia B community, as well as the access and reimbursement authorities, in bringing innovative therapies to patients,” said Dr Lutz Bonacker SVP and General Manager, CSL Behring Commercial Operations Europe.

“仅仅几十年前，血友病的基因治疗还是一个遥远的概念，现在已经成为现实。因此，自获得欧洲批准以来，首批接受HEMGENIX®治疗的两名患者是一项重大成就，证明了血友病B社区以及获取和报销机构在为患者带来创新疗法方面的共同承诺，”CSL Behring Commercial Operations Europe总经理Lutz Bonacker博士说。

“This milestone has been made possible by the innovative Direct Access scheme adopted in France, allowing patients to benefit from early access to pioneering treatments. We are encouraged to see increasing access to gene therapies in European countries and are fully committed to ensuring that access to potentially life-changing treatment continues.”.

“法国采用的创新性直接获取计划使这一里程碑成为可能，使患者能够从早期获得开创性治疗中受益。我们感到鼓舞的是，欧洲国家越来越多地获得基因治疗，并充分致力于确保继续获得可能改变生命的治疗。”。

HEMGENIX® was granted conditional marketing authorisation by the European Commission (EC) for the European Union and European Economic Area in February 2023, following approval from the U.S. Food and Drug Administration (FDA) in November 2022. It has also been approved by Health Canada, the United Kingdom's Medicines and Healthcare products Regulatory Agency (MHRA), Switzerland’s Swissmedic and Australia’s Therapeutic Goods Administration (TGA)..

2022年11月，经美国食品和药物管理局（FDA）批准，HEMGENIX®于2023年2月获得欧盟委员会（EC）针对欧盟和欧洲经济区的有条件上市许可。它还得到了加拿大卫生部、英国药品和保健品管理局（MHRA）、瑞士瑞士瑞士医学会（Swissmedic）和澳大利亚治疗用品管理局（TGA）的批准。。

The multi-year clinical development of HEMGENIX® was led by uniQure and sponsorship of the clinical trials transitioned to CSL after it licensed global rights to commercialise the treatment.

HEMGENIX®的多年临床开发由uniQure领导，在获得全球治疗商业化权利后，临床试验的赞助转向了CSL。

About Hemophilia B

关于血友病B

Hemophilia B is a life-threatening rare disease. People with the condition are particularly vulnerable to bleeds in their joints, muscles, and internal organs, leading to pain, swelling, and joint damage. Current treatments for moderate to severe hemophilia B include life-long prophylactic infusions of factor IX to temporarily replace or supplement low levels of the blood-clotting factor..

血友病B是一种危及生命的罕见疾病。患有这种疾病的人特别容易出现关节、肌肉和内脏出血，导致疼痛、肿胀和关节损伤。目前治疗中度至重度血友病B的方法包括终身预防性输注IX因子，以暂时替代或补充低水平的凝血因子。。

About HEMGENIX®

关于HEMGENIX®

HEMGENIX® is a gene therapy that reduces the rate of abnormal bleeding in eligible people with hemophilia B by enabling the body to continuously produce factor IX, the deficient protein in hemophilia B. It uses AAV5, a non-infectious viral vector, called an adeno-associated virus (AAV). The AAV5 vector carries the Padua gene variant of Factor IX (FIX-Padua) to the target cells in the liver, generating factor IX proteins that are 5x-8x more active than normal.

HEMGENIX®是一种基因疗法，可通过使身体持续产生因子IX（血友病B中的缺陷蛋白）来降低符合条件的B型血友病患者的异常出血率。它使用AAV5（一种非感染性病毒载体，称为腺相关病毒（AAV））。AAV5载体将因子IX的Padua基因变体（FIX-Padua）携带到肝脏中的靶细胞，产生比正常活性高5倍至8倍的因子IX蛋白。

These genetic instructions remain in the target cells, but generally do not become a part of a person’s own DNA. Once delivered, the new genetic instructions allow the cellular machinery to produce stable levels of factor IX..

这些基因指令保留在靶细胞中，但通常不会成为一个人自身DNA的一部分。一旦交付，新的基因指令允许细胞机制产生稳定水平的因子IX。。

About the Pivotal HOPE-B Trial

The pivotal Phase III HOPE-B trial is an ongoing, multinational, open-label, single-arm study to evaluate the safety and efficacy of HEMGENIX®. Fifty-four adult hemophilia B patients classified as having moderately severe to severe hemophilia B and requiring prophylactic factor IX replacement therapy were enrolled in a prospective, six-month or longer observational period during which time they continued to use their current standard of care therapy to establish a baseline Annual Bleeding Rate (ABR).

关键的III期HOPE-B试验是一项正在进行的多国开放标签单臂研究，旨在评估HEMGENIX®的安全性和有效性。54名成人血友病B患者被归类为中度重度至重度血友病B，需要预防性IX因子替代治疗，他们参加了一个为期6个月或更长时间的前瞻性观察期，在此期间，他们继续使用目前的护理标准治疗来建立基线年出血率（ABR）。

After the six-month lead-in period, patients received a single intravenous administration of HEMGENIX® at the 2x10^13 gc/kg dose. Patients were not excluded from the trial based on pre-existing neutralizing antibodies (NAbs) to AAV5..

在六个月的导入期后，患者以2x10 ^ 13 gc/kg剂量单次静脉注射HEMGENIX®。基于预先存在的AAV5中和抗体（NAb），患者未被排除在试验之外。。

A total of 54 patients received a single dose of HEMGENIX® in the pivotal trial, with 52 patients completing at least three years of follow-up. The primary endpoint in the pivotal HOPE-B study was ABR 52 weeks after achievement of stable factor IX expression (months 7 to 18) compared with the six-month lead-in period.

在关键试验中，共有54名患者接受了单剂量的HEMGENIX®，其中52名患者完成了至少三年的随访。关键的HOPE-B研究的主要终点是与六个月的导入期相比，在达到稳定的因子IX表达（7至18个月）后52周的ABR。

For this endpoint, ABR was measured from month seven to month 18 after infusion, ensuring the observation period represented a steady-state factor IX transgene expression. Secondary endpoints included assessment of factor IX activity..

对于该终点，在输注后第7个月至第18个月测量ABR，确保观察期代表稳态因子IX转基因表达。次要终点包括评估因子IX活性。。

No serious treatment-related adverse reactions were reported. One death resulting from urosepsis and cardiogenic shock in a 77-year-old patient at 65 weeks following dosing was considered unrelated to treatment by investigators and the company sponsor. A serious adverse event of hepatocellular carcinoma was determined to be unrelated to treatment with HEMGENIX® by independent molecular tumour characterization and vector integration analysis.

没有报告严重的治疗相关不良反应。研究人员和公司赞助商认为，一名77岁患者在给药后65周因尿脓毒症和心源性休克导致的一例死亡与治疗无关。通过独立的分子肿瘤表征和载体整合分析，确定肝细胞癌的严重不良事件与HEMGENIX®治疗无关。

No inhibitors to factor IX were reported..

。。

Long-term three-year data presented at the 17th Annual Congress of the European Association for Haemophilia and Allied Disorders (EAHAD) 2024 continue to reinforce the potential long-lasting efficacy and safety of HEMGENIX® and the ongoing benefit of this treatment for people living with hemophilia B..

2024年欧洲血友病和相关疾病协会（EAHAD）第17届年会上提供的长期三年数据继续加强了HEMGENIX®的潜在长期疗效和安全性，以及这种治疗对血友病B患者的持续益处。。

About CSL

关于CSL

CSL (ASX:CSL; USOTC:CSLLY) is a global biotechnology company with a dynamic portfolio of lifesaving medicines, including those that treat hemophilia and immune deficiencies, vaccines to prevent influenza, and therapies in iron deficiency and nephrology. Since our start in 1916, we have been driven by our promise to save lives using the latest technologies.

CSL（ASX:CSL；USOTC:CSLLY）是一家全球生物技术公司，拥有一系列充满活力的救生药物，包括治疗血友病和免疫缺陷的药物，预防流感的疫苗以及缺铁和肾脏病的疗法。自1916年成立以来，我们一直致力于使用最新技术拯救生命。

Today, CSL – including our three businesses: CSL Behring, CSL Seqirus and CSL Vifor – provides lifesaving products to patients in more than 100 countries and employs 32,000 people. Our unique combination of commercial strength, R&D focus and operational excellence enables us to identify, develop and deliver innovations so our patients can live life to the fullest.

如今，CSL（包括我们的三大业务：CSL Behring、CSL Sequirus和CSL Vifor）为100多个国家的患者提供救生产品，拥有32000名员工。。

For inspiring stories about the promise of biotechnology, visit CSL.com/Vita. For more information about CSL, visit CSL.com..

有关生物技术前景的鼓舞人心的故事，请访问CSL.com/Vita。有关CSL的更多信息，请访问CSL.com。。

Media Contacts

媒体联系人

Stephanie Fuchs

Stephanie Fuchs

Mobile: +49 151 584 388 60

手机：+49 151 584 388 60

Email: Stephanie.Fuchs@cslbehring.com

电子邮件Stephanie.Fuchs@cslbehring.com

References

参考文献

1 European Medicines Agency. First Gene therapy to treat haemophilia B. Available at: https://www.ema.europa.eu/en/news/first-gene-therapy-treat-haemophilia-b. [Accessed May 2024].

1欧洲药品管理局。第一种治疗血友病B的基因疗法。可在以下网址获得：https://www.ema.europa.eu/en/news/first-gene-therapy-treat-haemophilia-b.[2024年5月访问]。

2 Republique Française. Légifrance: Article 62 of Law No. 2021-1754. Available at: https://www.legifrance.gouv.fr/jorf/id/JORFTEXT000048551003 [Accessed May 2024].

2法国共和国。Legifrance：第2021-1754号法律第62条。网址：https://www.legifrance.gouv.fr/jorf/id/JORFTEXT000048551003[2024年5月访问]。

3 Leebeek, F & Miesbach, W. (2021) Gene therapy for haemophilia: a review on clinical benefit, limitations, and remaining issues. Blood. Vol 138, Issue 11. pp923-931.

3 Leebeek，F＆Miesbach，W。（2021）血友病的基因治疗：临床益处，局限性和剩余问题的综述。血。第138卷，第11期。pp923-931。

4 Coppens M et al. Etranacogene dezaparvovec gene therapy for haemophilia B (HOPE-B): 24-month post-hoc efficacy and safety data from a single-arm, multicentre, phase 3 trial. The Lancet Haematology 2024; 11(4):E265-E275.

4 Coppens M等人。Etranacogene dezaparvovec基因治疗血友病B（HOPE-B）：来自单臂，多中心，3期试验的24个月事后疗效和安全性数据。柳叶刀血液学2024；11（4）：E265-E275。