BOSTON--(BUSINESS WIRE)--Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that the U.S. Food and Drug Administration (FDA) has cleared the Investigational New Drug Application (IND) for VX-407, an investigational first-in-class small molecule corrector that targets the underlying cause of autosomal dominant polycystic kidney disease (ADPKD) in patients with a subset of PKD1 genetic variants.

波士顿--（商业新闻短讯）--Vertex Pharmaceuticals Incorporated（Nasdaq:VRTX）今天宣布，美国食品和药物管理局（FDA）已批准VX-407的研究性新药申请（IND），VX-407是一种研究性一流的小分子校正剂，针对具有PKD1基因变异子集的患者常染色体显性多囊肾病（ADPKD）的根本原因。

ADPKD is the most common inherited kidney disease, with an estimated 250,000 people in the U.S. and Europe living with ADPKD; however, there are no treatments currently available that address the underlying causal biology of the disease..

ADPKD是最常见的遗传性肾脏疾病，美国和欧洲估计有250000人患有ADPKD；然而，目前还没有可用的治疗方法来解决该疾病的潜在因果生物学。。

ADPKD is a life-shortening genetic kidney disease characterized by the growth of numerous kidney-enlarging cysts that impair kidney function and can ultimately lead to kidney failure, requiring dialysis or kidney transplantation, and premature death.

ADPKD是一种缩短寿命的遗传性肾脏疾病，其特征是大量肾脏增大的囊肿会损害肾功能，并最终导致肾衰竭，需要透析或肾脏移植以及过早死亡。

The majority of ADPKD cases are caused by variants in the PKD1 gene, which encodes the polycystin 1 (PC1) protein. These inherited variants lead to a loss of PC1 function that results in cyst growth. VX-407 is a first-in-class small molecule corrector that is designed to target the underlying cause of ADPKD in a subset of patients with PKD1 variants, estimated at ~25,000 (or ~10%) of the overall ~250,000 ADPKD patient population, by restoring function to the variant PC1 protein.

大多数ADPKD病例是由编码多囊蛋白1（PC1）蛋白的PKD1基因变异引起的。这些遗传变异导致PC1功能丧失，导致囊肿生长。VX-407是一流的小分子校正剂，旨在针对PKD1变异患者亚组中ADPKD的根本原因，通过恢复变异PC1蛋白的功能，估计约占总ADPKD患者总数的25000（或10%）。

Vertex plans to initiate a Phase 1 clinical trial of VX-407 in healthy volunteers this month..

Vertex计划本月在健康志愿者中启动VX-407的1期临床试验。。

“The advancement of VX-407, a first-in-class molecule for the treatment of ADPKD, into the clinic represents another important opportunity to transform the treatment of a serious disease,” said Carmen Bozic, M.D., Executive Vice President, Global Medicines Development and Medical Affairs, and Chief Medical Officer at Vertex.

Vertex全球药物开发和医疗事务执行副总裁兼首席医疗官Carmen Bozic医学博士说：“VX-407（一种用于治疗ADPKD的一流分子）进入临床代表了改变严重疾病治疗方法的另一个重要机会。”。

“Just as our approach in cystic fibrosis allowed us to reach more patients over time, our goal here is to serially innovate to reach the 250,000 people suffering from ADPKD.”.

“正如我们在囊性纤维化方面的方法使我们能够随着时间的推移接触更多的患者一样，我们的目标是不断创新，以接触25万患有ADPKD的患者。”。

About Autosomal Dominant Polycystic Kidney Disease (ADPKD)

关于常染色体显性遗传性多囊肾病（ADPKD）

ADPKD is the most common inherited kidney disease and one of the most common severe Mendelian genetic diseases, affecting approximately 250,000 diagnosed people in the U.S. and Europe. As an autosomal dominant disease, one affected parent can pass on the disease to their children.

ADPKD是最常见的遗传性肾脏疾病，也是最常见的严重孟德尔遗传疾病之一，在美国和欧洲约有250000名确诊患者受到影响。作为常染色体显性遗传疾病，一名受影响的父母可以将疾病传给他们的孩子。

In most cases, ADPKD is caused by variants in the PKD1 and PKD2 genes, which express proteins known as polycystins. The majority of ADPKD patients (~80%) have a variant in the PKD1 gene, resulting in a loss of function of polycystin 1 (PC1). This leads to the proliferation of kidney epithelial cells, increased fluid secretion and the formation and expansion of numerous fluid-filled cysts.

在大多数情况下，ADPKD是由PKD1和PKD2基因的变异引起的，这些基因表达称为多囊蛋白的蛋白质。大多数ADPKD患者（约80%）的PKD1基因存在变异，导致多囊蛋白1（PC1）功能丧失。这导致肾上皮细胞增殖，液体分泌增加以及许多充满液体的囊肿的形成和扩张。

The progressive cyst formation causes an increase in kidney size and decline in kidney function. Around half of patients with ADPKD experience kidney failure by the age of 60. Kidney cysts can also lead to severe abdominal pain, cyst infection, blood in the urine and kidney stones, all of which significantly impair quality of life..

进行性囊肿形成导致肾脏大小增加和肾功能下降。大约一半的ADPKD患者在60岁时会出现肾衰竭。肾囊肿还会导致严重的腹痛、囊肿感染、尿液中的血液和肾结石，所有这些都会严重影响生活质量。。

About VX-407

关于VX-407

VX-407 is a first-in-class small molecule corrector that is designed to treat ADPKD in patients with a subset of PKD1 variants, estimated at ~25,000 (or ~10%) of the overall ~250,000 ADPKD patient population, by correcting defective PC1 folding to restore function. In doing so, the aim is to stop growth of kidney cysts and reduce kidney volume, thereby preventing progression to kidney failure..

VX-407是一流的小分子校正剂，旨在通过校正缺陷的PC1折叠来恢复功能，从而治疗具有PKD1变体子集的患者的ADPKD，估计约占整个约250000 ADPKD患者人群的25000（或约10%）。这样做的目的是阻止肾囊肿的生长并减少肾脏体积，从而防止进展为肾衰竭。。

About Vertex

关于顶点

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。该公司已批准用于治疗多种慢性、缩短寿命的遗传疾病（囊性纤维化、镰状细胞病和输血依赖性β地中海贫血）的根本原因的药物，并继续推进这些疾病的临床和研究计划。

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, 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，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。

For company updates and to learn more about Vertex's history of innovation, visit www.vrtx.com or follow us on LinkedIn, Facebook, Instagram, YouTube and Twitter/X..

有关公司更新和Vertex创新历史的更多信息，请访问www.vrtx.com或在LinkedIn、Facebook、Instagram、YouTube和Twitter/X上关注我们。。

Special Note Regarding Forward-Looking Statements

关于前瞻性声明的特别说明

This press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, including, without limitation, statements made by Carmen Bozic, M.D., in this press release, statements regarding plans to initiate a Phase 1 clinical trial of VX-407 in healthy volunteers this month, and estimates regarding the potential eligible patient population.

本新闻稿包含1995年《私人证券诉讼改革法案》中定义的前瞻性声明，包括但不限于医学博士卡门·博齐奇在本新闻稿中的声明，关于本月在健康志愿者中启动VX-407第一阶段临床试验计划的声明，以及关于潜在合格患者人群的估计。

While Vertex believes the forward-looking statements contained in this press release are accurate, these forward-looking statements represent the company's beliefs only as of the date of this press release and there are a number of risks and uncertainties that could cause actual events or results to differ materially from those expressed or implied by such forward-looking statements.

虽然Vertex认为本新闻稿中包含的前瞻性陈述是准确的，但这些前瞻性陈述仅代表截至本新闻稿发布之日的公司信念，并且存在许多风险和不确定性，可能导致实际事件或结果与此类前瞻性陈述所表达或暗示的事件或结果存在重大差异。

Those risks and uncertainties include, among other things, that data from the company’s research and development programs may not support registration or further development of its compounds due to safety, efficacy, and other risks listed under the heading “Risk Factors” in Vertex's annual report and in subsequent filings filed with the Securities and Exchange Commission and available through the company's website at www.vrtx.com and www.sec.gov.

这些风险和不确定性包括，除其他外，公司研发计划的数据可能不支持其化合物的注册或进一步开发，因为Vertex的年度报告和随后提交给证券交易委员会的文件中“风险因素”标题下列出了安全性、有效性和其他风险，可通过公司网站www.vrtx.com和www.sec.gov获得。

You should not place undue reliance on these statements. Vertex disclaims any obligation to update the information contained in this press release as new information becomes available..

你不应该过分依赖这些陈述。Vertex不承担在新信息可用时更新本新闻稿中包含的信息的任何义务。。

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