BOSTON--(BUSINESS WIRE)--Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced important advancements across its suzetrigine pain program, which has the potential to be the first new class of medicine for acute and neuropathic pain in more than two decades. Suzetrigine is an oral selective NaV1.8 pain signal inhibitor (formerly known as VX-548)..

波士顿--（商业新闻短讯）--Vertex Pharmaceuticals Incorporated（Nasdaq:VRTX）今天宣布了其suzetrigine疼痛计划的重要进展，该计划有可能成为20多年来第一类治疗急性和神经性疼痛的新型药物。Suzetrigine是一种口服选择性NaV1.8疼痛信号抑制剂（以前称为VX-548）。。

Following the positive Phase 3 results in acute pain announced in January 2024, the Food and Drug Administration (FDA) has granted a rolling New Drug Application (NDA) submission for suzetrigine in moderate-to-severe acute pain. Vertex has started the rolling submission process and is on track to complete the submission in the second quarter of 2024.

在2024年1月宣布急性疼痛的3期阳性结果后，美国食品和药物管理局（FDA）批准了suzetrigine在中度至重度急性疼痛中的滚动新药申请（NDA）。Vertex已开始滚动提交过程，有望在2024年第二季度完成提交。

Suzetrigine was previously granted FDA Fast Track and Breakthrough Therapy designations in moderate-to-severe acute pain..

Suzetrigine之前被FDA授予中度至重度急性疼痛的快速通道和突破性治疗指定。。

In neuropathic pain, Vertex released positive results from its Phase 2 study in December 2023 and recently completed a successful end-of-phase 2 meeting with the FDA. Vertex is now preparing to initiate a Phase 3 pivotal program of suzetrigine in patients with DPN in 2H 2024. In addition, the FDA recently granted suzetrigine Breakthrough Therapy designation for the treatment of pain associated with DPN..

在神经性疼痛方面，Vertex于2023年12月发布了第二阶段研究的阳性结果，最近与FDA成功完成了第二阶段会议的结束。Vertex目前正准备于2024年下半年在DPN患者中启动suzetrigine的3期关键计划。此外，FDA最近批准了suzetrigine突破疗法，用于治疗与DPN相关的疼痛。。

The Phase 3 program will include two identical 12-week randomized, double-blind, placebo-controlled studies evaluating the efficacy and safety of suzetrigine (70 mg once daily) in patients with DPN. The primary endpoint for both studies will be the change from baseline in weekly average of daily pain intensity on the numeric pain rating scale (NPRS) assessed at Week 12 compared to placebo.

第三阶段计划将包括两项相同的12周随机，双盲，安慰剂对照研究，评估suzetrigine（70 mg，每日一次）对DPN患者的疗效和安全性。这两项研究的主要终点是与安慰剂相比，在第12周评估的数字疼痛评定量表（NPRS）上，每日疼痛强度的周平均值与基线的变化。

Both studies will also include a key secondary endpoint of change from baseline in the weekly average of daily pain intensity on the NPRS at Week 12 compared to pregabalin. Approximately 1,100 patients are expected to enroll in each Phase 3 study. After completing participation in the randomized controlled studies, patients may roll over into an open-label study to evaluate the long-term safety and effectiveness of suzetrigine in DPN..

与普瑞巴林相比，这两项研究还将包括第12周NPRS每日疼痛强度周平均值与基线变化的关键次要终点。预计大约1100名患者将参加每个3期研究。在完成随机对照研究的参与后，患者可以转入开放标签研究，以评估suzetrigine在DPN中的长期安全性和有效性。。

Additionally, Vertex continues to enroll its Phase 2 study of suzetrigine in patients with lumbosacral radiculopathy, or LSR, which is pain caused by impairment or injury to nerve roots in the area of the lumbar spine. The company is on track to complete enrollment in the Phase 2 LSR study by the end of the year..

此外，Vertex继续在腰骶神经根病（LSR）患者中纳入其suzetrigine的2期研究，LSR是由腰椎区域神经根受损或损伤引起的疼痛。该公司有望在年底前完成第二阶段LSR研究的注册。。

“Today marks a significant milestone on our journey to redefine the treatment of pain,” said Carmen Bozic, M.D., Executive Vice President, Global Medicines Development and Medical Affairs, and Chief Medical Officer at Vertex. “Given the favorable benefit/risk profile demonstrated by suzetrigine across the entire clinical program and the positive interactions with regulators, we are excited by the opportunity to rapidly advance suzetrigine, a new non-opioid potential treatment, for the millions of patients suffering from acute and peripheral neuropathic pain.”.

Vertex全球药物开发和医疗事务执行副总裁兼首席医疗官CarmenBozic医学博士说：“今天标志着我们重新定义疼痛治疗之旅的一个重要里程碑。”。“鉴于suzetrigine在整个临床计划中表现出良好的益处/风险概况以及与监管机构的积极互动，我们很高兴有机会迅速推进suzetrigine，一种新的非阿片类药物潜在治疗方法，用于数百万患有急性和周围神经性疼痛的患者。”。

Next Steps for the Pain Portfolio

疼痛组合的下一步

In line with its pain portfolio serial innovation strategy, Vertex continues to advance preclinical and clinical development of additional NaV1.8 and NaV1.7 pain signal inhibitors, for use alone or in combination, in acute and neuropathic pain. The company intends to advance its next generation NaV1.8 pain signal inhibitor VX-993 oral formulation into Phase 2 acute pain and peripheral neuropathic pain studies later this year.

根据其疼痛组合系列创新策略，Vertex继续推进额外的NaV1.8和NaV1.7疼痛信号抑制剂的临床前和临床开发，用于单独或联合用于急性和神经性疼痛。该公司打算在今年晚些时候将其下一代NaV1.8疼痛信号抑制剂VX-993口服制剂推进2期急性疼痛和周围神经性疼痛研究。

Vertex also anticipates initiating a Phase 1 study of an intravenous formulation of VX-993 later this year..

Vertex还预计在今年晚些时候启动VX-993静脉制剂的第一阶段研究。。

About Acute Pain

关于急性疼痛

Acute pain is a disabling condition and is defined as pain lasting less than 3 months. It is estimated that over 80 million people are prescribed a medicine for acute pain every year in the U.S. Due to limited treatment options, there is an unmet need in acute pain management to improve the patient experience and reduce the economic and societal burden..

急性疼痛是一种致残性疾病，定义为持续不到3个月的疼痛。据估计，美国每年有超过8000万人服用治疗急性疼痛的药物。由于治疗选择有限，急性疼痛管理的需求尚未得到满足，以改善患者体验并减轻经济和社会负担。。

About Peripheral Neuropathic Pain

关于周围神经性疼痛

Peripheral neuropathic pain, or PNP, is a significant area of unmet need for patients suffering from pain. PNP is a collection of chronic conditions including pain associated with diabetic peripheral neuropathy (DPN), lumbosacral radiculopathy (LSR), small fiber neuropathy and trigeminal neuralgia. DPN and LSR are two of the largest segments within the estimated 10 million patients who are prescribed a medicine for PNP every year in the U.S..

周围神经性疼痛或PNP是疼痛患者未满足需求的重要领域。PNP是一组慢性病，包括与糖尿病周围神经病变（DPN），腰骶神经根病（LSR），小纤维神经病和三叉神经痛相关的疼痛。在美国每年约有1000万名患者服用PNP药物，DPN和LSR是其中最大的两个部分。。

About Suzetrigine (VX-548)

关于Suzetrigine（VX-548）

Suzetrigine (formerly VX-548) is an investigational oral, selective NaV1.8 pain signal inhibitor that is highly selective for NaV1.8 relative to other NaV channels. NaV1.8 is a voltage-gated sodium channel that plays a critical role in pain signaling in the peripheral nervous system. NaV1.8 is a genetically validated target for the treatment of pain, and suzetrigine has demonstrated a favorable benefit/risk profile in three Phase 3 studies and two Phase 2 studies in moderate-to-severe acute pain.

Suzetrigine（以前称为VX-548）是一种研究性口服选择性NaV1.8疼痛信号抑制剂，相对于其他NaV通道对NaV1.8具有高度选择性。NaV1.8是一种电压门控钠通道，在周围神经系统的疼痛信号传导中起关键作用。NaV1.8是一种经过基因验证的疼痛治疗靶点，suzetrigine在中度至重度急性疼痛的三期研究和两期研究中表现出良好的益处/风险特征。

Suzetrigine also demonstrated positive results and a well-tolerated profile in a Phase 2 study in patients with pain associated with diabetic peripheral neuropathy, a type of peripheral neuropathic pain. Vertex’s approach is to selectively inhibit NaV1.8 using small molecules with the objective of creating a new class of pain signal inhibitors that have the potential to provide effective relief of pain without the limitations of currently available therapies, including the addictive potential of opioids..

Suzetrigine在与糖尿病周围神经病变（一种周围神经性疼痛）相关的疼痛患者的2期研究中也显示出积极的结果和良好的耐受性。Vertex的方法是使用小分子选择性抑制NaV1.8，目的是创建一类新的疼痛信号抑制剂，该抑制剂有可能在不受当前可用疗法（包括阿片类药物成瘾潜力）限制的情况下有效缓解疼痛。。

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 Vertex’s expectations for the rolling submission to the FDA for suzetrigine in acute pain, including expectations to complete the submission in the second quarter of 2024, expectations to initiate the Phase 3 program of suzetrigine in patients with DPN in the second half of 2024, plans for the design of and enrollment expectations for the Phase 3 DPN program, plans to evaluate the long-term efficacy and safety of suzetrigine in patients with DPN, including enrollment expectations for this open-label study, and enrollment expectations for the Phase 2 study in LSR, including plans to complete enrollment in this study by the end of the year, our expectations for the anticipated benefits of suzetrigine, our plans to continue advancing additional NaV1.8 and NaV1.7 pain signal inhibitors in acute and neuropathic pain, our plans to advance VX-993 oral formulation into Phase 2 acute pain and PNP studies later this year, and our plans to initiate a Phase 1 study of an intravenous formulation of VX-993 later this year.

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（CarmenBozic）在本新闻稿中发表的声明，其中包括Vertex对向FDA滚动提交急性疼痛中的suzetrigine的期望，包括在2024年第二季度完成提交的期望，2024年下半年在DPN患者中启动suzetrigine第三阶段计划的期望，第三阶段DPN计划的设计和注册期望，评估suzetrigine在DPN患者中的长期疗效和安全性的计划，包括这项开放标签研究的注册期望以及LSR第二阶段研究的入学期望，包括计划在年底前完成这项研究的入学，我们对suzetrigine的预期收益的期望，我们计划在急性和神经性疼痛中继续推进额外的NaV1.8和NaV1.7疼痛信号抑制剂，我们计划在今年晚些时候将VX-993口服制剂推进2期急性疼痛和PNP研究，以及我们计划在今年晚些时候启动VX-993静脉注射制剂的1期研究。

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 factors that could cause actual events or results to differ materially from those indicated 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公司的信念，并且有许多因素可能导致实际事件或结果与此类前瞻性陈述所表明的大不相同。

Those risks and uncertainties include, among other things, that our clinical studies for suzetrigine may be delayed, and that data from the company's research and development pro.

这些风险和不确定性包括，我们对suzetrigine的临床研究可能会延迟，以及该公司研发专业人员的数据。