BOSTON--(BUSINESS WIRE)--Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced the first accepted medical presentations of the Phase 3 data on the investigational once daily vanzacaftor/tezacaftor/deutivacaftor (“vanza triple”) — the potential next-in-class triple combination medicine — will take place at the North American Cystic Fibrosis Conference (NACFC).

波士顿--（商业新闻短讯）--Vertex Pharmaceuticals Incorporated（Nasdaq:VRTX）今天宣布，将在北美囊性纤维化会议（NACFC）上首次接受关于每日一次的研究性vanzacaftor/tezacaftor/deutivacaftor（“vanza triple”）的第三阶段数据的医学介绍，这是潜在的下一类三联组合药物。

Vertex also announced presentations describing long-term outcomes in people with cystic fibrosis (CF) ages 2 to 11 years taking TRIKAFTA®, demonstrating consistent and sustained improvements across multiple measures of disease..

Vertex公司还宣布了对2至11岁服用TRIKAFTA®的囊性纤维化（CF）患者的长期治疗结果，结果表明患者的多种疾病指标都得到了一致和持续的改善。

“TRIKAFTA has transformed the treatment of CF in the 5 years since its approval, changing the outlook for patients with CF,” said Carmen Bozic, M.D., Executive Vice President, Global Medicines Development and Medical Affairs, and Chief Medical Officer at Vertex. “Toward our goal to bring all eligible patients to normal levels of CFTR function, we have made global regulatory submissions for our next-in-class vanza triple combination medicine for patients with CF aged 6 years and older.

。“为了使所有符合条件的患者达到正常的CFTR功能水平，我们已经为6岁及以上CF患者的下一批vanza三联药物提交了全球监管报告。

We are excited to bring this promising new medicine, which has the potential to deliver even greater improvements in sweat chloride, to patients with CF.”.

我们很高兴为CF患者带来这种有前途的新药，它有可能在汗液氯化物方面取得更大的改善。”。

Data on the Investigational Once Daily Next-in-Class Triple Combination Therapy, vanzacaftor/tezacaftor/deutivacaftor

每日一次的下一类三联疗法研究数据，vanzacaftor/tezacaftor/deutivacaftor

Vertex will present data on the vanza triple in an oral presentation and two posters on Friday, September 27. This is the first time that the clinical data from the Phase 3 clinical trials of the vanza triple in patients 6 years and older with CF have been accepted for presentation at a medical meeting.

Vertex将于9月27日（星期五）在口头演示和两张海报中展示vanza triple的数据。这是vanza triple在6岁及以上CF患者中进行的3期临床试验的临床数据首次被接受在医学会议上发表。

These data formed the basis of global regulatory submissions..

这些数据构成了全球监管提交的基础。。

“The additional, significant reductions in sweat chloride we see in the vanza triple clinical trials are noteworthy. I believe this improvement in CFTR function may lead to important benefits for people with CF,” said Claire L. Keating, M.D., Co-Director of the Gunnar Esiason Adult Cystic Fibrosis and Lung Program at Columbia University.

哥伦比亚大学Gunnar Esiason成人囊性纤维化和肺项目联合主任Claire L.Keating医学博士说：“我们在vanza三重临床试验中看到的汗液氯化物的额外显着减少值得注意。我认为CFTR功能的改善可能会为CF患者带来重要益处。”。

“If approved, as a clinician, I’m looking forward to being able to offer patients an option, with once daily dosing, that could advance the treatment for people living with CF.”.

“如果获得批准，作为一名临床医生，我期待着能够为患者提供一种选择，每天服用一次，这可能会促进CF患者的治疗。”。

Data from the Phase 3 clinical studies of the vanza triple will be presented in the following sessions:

vanza triple的3期临床研究数据将在以下会议中介绍：

“Vanzacaftor/Tezacaftor/Deutivacaftor (VNZ Triple) in Adolescents and Adults with CF: Results from Two Randomized, Active-Controlled Phase 3 Trials,” will be an oral presentation during a session entitled: “Cutting Edge Research: What’s New” on Friday, September 27, from 10:15 a.m. to 12:15 p.m. EDT and be a poster presentation on Friday, September 27, from 1:15-2:15 p.m.

“Vanzacaftor/Tezacaftor/Deutivacaftor（VNZ Triple）在青少年和成人CF患者中的应用：两项随机，主动对照的3期临床试验的结果”，将于9月27日（美国东部夏令时）星期五上午10:15至下午12:15在题为“前沿研究：最新进展”的会议上进行口头演示，并于9月27日（美国东部夏令时）星期五下午1:15-2:15进行海报演示。

“Safety and Efficacy of Vanzacaftor/Tezacaftor/Deutivacaftor (VNZ Triple) in Children 6 Through 11 Years of Age with Cystic Fibrosis,” will be a poster presentation on Friday, September 27, from 1:15-2:15 p.m. EDT.

“Vanzacaftor/Tezacaftor/Deutivacaftor (VNZ Triple) 对 6 至 11 岁囊性纤维化儿童的安全性和有效性 "将于美国东部时间 9 月 27 日星期五下午 1:15-2:15 进行海报展示。

The vanza triple was granted Fast Track and Orphan Drug Designations from the U.S. Food and Drug Administration (FDA) for the treatment of CF and has been submitted for regulatory approval in the U.S., Canada, U.K., EU, Switzerland, Australia and New Zealand. Vertex has been assigned a Prescription Drug User Fee Act (PDUFA) date by the FDA of January 2, 2025, for this submission.

vanza triple已获得美国食品和药物管理局（FDA）的快速通道和孤儿药指定，用于治疗CF，并已提交美国，加拿大，英国，欧盟，瑞士，澳大利亚和新西兰的监管部门批准。Vertex已被FDA指定为2025年1月2日的处方药用户费用法案（PDUFA）日期。

The vanza triple has not been approved by any global health authority..

vanza triple尚未获得任何全球卫生当局的批准。。

Long-Term Benefits of TRIKAFTA®

TRIKAFTA®的长期益处

Vertex will present new data on TRIKAFTA® from long-term (96 week and 192 week) studies in patients ages 2-11 years old that reinforce the sustained benefit seen in studies in older people with CF. Specifically, that early treatment with TRIKAFTA® is associated with sustained improvements in lung function.

Vertex将从2-11岁患者的长期（96周和192周）研究中提供有关TRIKAFTA®的新数据，这些数据加强了老年CF患者研究中所见的持续益处。具体而言，TRIKAFTA®的早期治疗与肺功能的持续改善有关。

These new data demonstrate that in these young children, TRIKAFTA® could also lead to improved exocrine pancreatic function over time. The data presented at NACFC highlight the safety and tolerability of TRIKAFTA®, which were generally consistent with the established safety profile..

这些新数据表明，在这些幼儿中，TRIKAFTA®也可以随着时间的推移改善外分泌胰腺功能。NACFC上提供的数据突出了TRIKAFTA®的安全性和耐受性，这与既定的安全性概况基本一致。。

“I have seen first-hand the positive long-term impact that improvement of CFTR function by TRIKAFTA can have on patients’ clinical outcomes. These improvements are particularly striking for me as a physician caring for young children, where improvements in things like lung function, pancreatic function and quality of life are so meaningful,” said Professor Marcus A.

马库斯a教授说：“我亲眼目睹了TRIKAFTA改善CFTR功能对患者临床结局的长期积极影响。作为一名照顾幼儿的医生，这些改善对我来说尤其引人注目，因为肺功能，胰腺功能和生活质量等方面的改善非常有意义。”。

Mall, M.D., Professor and Chair of the Department of Pediatric Respiratory Medicine, Immunology and Critical Care Medicine and Cystic Fibrosis Center at Charité - Universitätsmedizin Berlin..

Mall，医学博士，柏林Charité-Universitätsmedizin儿科呼吸医学，免疫学和重症监护医学以及囊性纤维化中心的教授兼主席。。

Vertex will have four poster presentations that include clinical trial and real-world evidence data, three specifically on TRIKAFTA® and one showing sustained benefits from KALYDECO®, which is approved for treatment in the youngest ages.

Vertex将有四张海报展示，其中包括临床试验和现实世界的证据数据，其中三张专门针对TRIKAFTA®，另一张展示了KALYDECO®的持续益处，KALYDECO®已被批准在最年轻的年龄进行治疗。

“Long-term safety and efficacy of elexacaftor/tezacaftor/ivacaftor in children 2 years and older with cystic fibrosis and at least one F508del allele: 96-week results from an open-label extension study,” will be a poster presentation on Friday, September 27, at 12:15-1:15 p.m. EDT.

“elexacaftor/tezacaftor/ivacaftor在2岁及以上患有囊性纤维化和至少一个F508del等位基因的儿童中的长期安全性和有效性：开放标签扩展研究的96周结果”，将于9月27日星期五（美国东部夏令时下午12:15-1:15）发布海报。

“Long-term safety and efficacy of elexacaftor/tezacaftor/ivacaftor in children 6 years and older with cystic fibrosis and at least one F508del allele: Results from a 192-week extension study,” will be a poster presentation on Friday, September 27, at 1:15-2:15 p.m. EDT.

“elexacaftor/tezacaftor/ivacaftor对6岁及以上患有囊性纤维化和至少一个F508del等位基因的儿童的长期安全性和有效性：192周延长研究的结果”，将于9月27日星期五，美国东部夏令时下午1:15-2:15发布海报。

“Effectiveness of elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) in people with cystic fibrosis and non-F508del CFTR variants: Interim results from a registry-based study,” will be a poster presentation on Friday, September 27, from 12:15-1:15 p.m. EDT.

“elexacaftor/tezacaftor/ivacaftor（ELX/TEZ/IVA）在囊性纤维化和非F508del-CFTR变异患者中的有效性：基于注册表的研究的中期结果”，将于9月27日星期五（美国东部夏令时下午12:15-1:15）发布海报。

“Long-term benefits of early ivacaftor (IVA) initiation in people with cystic fibrosis (CF),” will be a poster presentation on Friday, September 27, from 1:15-2:15 p.m. EDT.

“囊性纤维化（CF）患者早期服用依伐卡托（IVA）的长期益处”，将于9月27日（星期五）美国东部夏令时下午1:15-2:15发布海报。

About Cystic Fibrosis

关于囊性纤维化

Cystic fibrosis (CF) is a rare, life-shortening genetic disease affecting more than 92,000 people globally. CF is a progressive, multi-organ disease that affects the lungs, liver, pancreas, GI tract, sinuses, sweat glands and reproductive tract. CF is caused by a defective and/or missing CFTR protein resulting from certain mutations in the CFTR gene.

囊性纤维化（CF）是一种罕见的缩短寿命的遗传疾病，影响全球92000多人。CF是一种进行性多器官疾病，会影响肺，肝，胰腺，胃肠道，鼻窦，汗腺和生殖道。CF是由CFTR基因的某些突变导致的CFTR蛋白缺陷和/或缺失引起的。

Children must inherit two defective CFTR genes — one from each parent — to have CF, and these mutations can be identified by a genetic test. While there are many different types of CFTR mutations that can cause the disease, the vast majority of people with CF have at least one F508del mutation. CFTR mutations lead to CF by causing CFTR protein to be defective or by leading to a shortage or absence of CFTR protein at the cell surface.

儿童必须继承两个有缺陷的CFTR基因（每个父母一个）才能患有CF，这些突变可以通过基因测试来鉴定。尽管有许多不同类型的CFTR突变可导致该疾病，但绝大多数CF患者至少有一个F508del突变。CFTR突变通过导致CFTR蛋白缺陷或导致细胞表面CFTR蛋白短缺或缺失而导致CF。

The defective function and/or absence of CFTR protein results in poor flow of salt and water into and out of the cells in a number of organs. In the lungs, this leads to the buildup of abnormally thick, sticky mucus, chronic lung infections and progressive lung damage that eventually leads to death for many patients.

。在肺部，这会导致异常粘稠的粘液积聚，慢性肺部感染和进行性肺损伤，最终导致许多患者死亡。

The median age of death is in the 30s, but with treatment, projected survival is improving..

中位死亡年龄在30多岁，但经过治疗，预计生存率正在提高。。

About Vertex

关于Vertex

Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases. The company has approved medicines that treat the underlying causes of multiple chronic, life-shortening genetic diseases — cystic fibrosis, sickle cell disease and transfusion-dependent beta thalassemia — and continues to advance clinical and research programs in these diseases.

Vertex是一家全球生物技术公司，投资于科学创新，为患有严重疾病的人创造变革性药物。。

Vertex also has a robust clinical pipeline of investigational therapies across a range of modalities in other serious diseases where it has deep insight into causal human biology, including acute and neuropathic pain, APOL1-mediated kidney disease, IgA nephropathy, autosomal dominant polycystic kidney disease, type 1 diabetes, myotonic dystrophy type 1 and alpha-1 antitrypsin deficiency..

Vertex还在其他严重疾病的一系列模式中拥有强大的临床研究治疗渠道，对因果人类生物学有深入的了解，包括急性和神经性疼痛，APOL1介导的肾脏疾病，IgA肾病，常染色体显性多囊肾病，1型糖尿病，1型强直性肌营养不良和α-1抗胰蛋白酶缺乏症。。

Vertex was founded in 1989 and has its global headquarters in Boston, with international headquarters in London. Additionally, the company has research and development sites and commercial offices in North America, Europe, Australia, Latin America and the Middle East. Vertex is consistently recognized as one of the industry's top places to work, including 14 consecutive years on Science magazine's Top Employers list and one of Fortune’s 100 Best Companies to Work For.

Vertex成立于1989年，全球总部位于波士顿，国际总部位于伦敦。此外，该公司在北美、欧洲、澳大利亚、拉丁美洲和中东设有研发基地和商业办事处。Vertex一直被公认为行业最佳工作地点之一，包括连续14年入选《科学》杂志的最佳雇主名单，以及《财富》杂志的100家最佳工作公司之一。