Vertex Pharma Challenger Sionna为囊性纤维化管道获得1.91亿美元的IPO现金-动脉网

Vertex Pharma Challenger Sionna Secures $191M in IPO Cash for Cystic Fibrosis Pipeline

MedCity News 等信源发布 2025-02-10 03:32

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Vertex Pharmaceuticals dominates the market for cystic fibrosis drugs, but Sionna Therapeutics contends the pharma company’s products still have efficacy and safety limitations. Sionna has mapped out a clinical trial strategy that could validate its different approach to the chronic respiratory disease.

Vertex Pharmaceuticals在囊性纤维化药物市场上占据主导地位，但Sionna Therapeutics认为该制药公司的产品仍然存在有效性和安全性限制。辛纳制定了一项临床试验策略，可以验证其治疗慢性呼吸道疾病的不同方法。

Now the biotech can execute those plans with $191 million from an upsized IPO..

现在，这家生物技术公司可以通过大规模IPO获得1.91亿美元来执行这些计划。。

Sionna had planned to offer more than 8.8 million shares in the range of $16 and $18 each, which would have raised nearly $150 million at the pricing midpoint. The company was able to

辛纳曾计划发行880多万股股票，每股价格在16美元至18美元之间，这将在定价中点筹集近1.5亿美元。该公司能够

boost the deal size

扩大交易规模

to more than 10.5 million shares offered at the top of the targeted price range. Sionna’s shares debuted on the Nasdaq Friday under the stock symbol “SION.” Strong investor interest in the new stock enabled Sionna’s shares to close their first day of trading at $25 apiece, up nearly 39% from the IPO price..

在目标价格范围的顶部发行1050多万股。Sionna的股票于周五在纳斯达克上市，股票代码为“SION”。投资者对新股的浓厚兴趣使Sionna的股票在第一天的交易中以每股25美元的价格收盘，比IPO价格上涨了近39%。。

In cystic fibrosis (CF), genetic mutations lead to faulty versions of CFTR, a protein key to moving ions in and out of cells. Improperly functioning CFTR leads to mucus buildup in the lungs of CF patients. Two types of drugs improve CFTR function. Correctors bind to the protein and help it fold into the proper shape.

在囊性纤维化（CF）中，基因突变导致CFTR的错误版本，CFTR是将离子进出细胞的关键蛋白质。CFTR功能异常会导致CF患者肺部粘液积聚。两种药物可改善CFTR功能。校正剂与蛋白质结合并帮助其折叠成正确的形状。

Potentiators open channels of the protein. Vertex’s drugs combine both mechanisms to improve CFTR function at the surface of a cell..

增强剂打开蛋白质的通道。Vertex的药物结合了这两种机制来改善细胞表面的CFTR功能。。

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Health IT

健康IT

Transforming Patient Care: The Role of AI-Powered Assistants

改变病人护理：人工智能辅助人员的作用

The progress in artificial intelligence (AI) is reshaping patient care, across various dimensions, from facilitating faster discharge to curating treatment plans and suggesting lifestyle changes.

人工智能（AI）的进步正在从各个方面重塑患者护理，从促进更快的出院到策划治疗计划并建议改变生活方式。

By IT Medical

通过IT Medical

Waltham, Massachusetts-based Sionna is developing CF drugs that target NBD1, a domain on the CFTR protein. NBD1 has long been thought to be important for normalizing CFTR function because it’s the location of the F508del mutation — the most common mutation that causes CF. Sionna aims to succeed where others have fallen short developing drugs that stabilize this target..

位于马萨诸塞州沃尔瑟姆的辛纳正在开发针对CFTR蛋白结构域NBD1的CF药物。长期以来，人们一直认为NBD1对于CFTR功能的正常化很重要，因为它是F508del突变的位置，F508del突变是导致CF的最常见突变。Sionna的目标是在其他人无法开发稳定该靶标的药物的情况下取得成功。。

“Approximately 90% of people with CF carry at least one copy of the F508del genetic mutation,” Sionna said in the

“大约90%的CF患者携带至少一份F508del基因突变，”辛纳在

IPO filing

IPO备案

. “We believe stabilizing NBD1 is central to unlocking dramatic improvements in clinical outcomes and quality of life for CF patients. We have employed biophysical, cell-based and virtual screening campaigns and extensive use of structural biology to guide the optimization of novel small molecule NBD1 stabilizers.”.

“我们认为稳定NBD1是实现CF患者临床结果和生活质量显着改善的关键。我们采用了生物物理，基于细胞和虚拟的筛查活动，并广泛使用结构生物学来指导新型小分子NBD1稳定剂的优化。”。

Sionna expects NBD1 stabilization could be used as part of a dual combination therapy or as an add-on to standard CF therapies. The company has three NBD1 stabilizers in clinical development. SION-109, which targets NBD1’s interface with the CFTR intracellular loop 4 (ICL4) region, has completed Phase 1 testing in healthy volunteers.

辛纳预计NBD1稳定化可以作为双重联合疗法的一部分，也可以作为标准CF疗法的附加品。该公司在临床开发中有三种NBD1稳定剂。针对NBD1与CFTR细胞内环4（ICL4）区域的界面的SION-109已在健康志愿者中完成了第一阶段测试。

Sionna said results showed this molecule was well tolerated and achieved exposure in the body that could support its use as part of a combination with two other Sionna stabilizers, SION-719 and SION-451. These two NBD1 stabilizers .

辛纳说，结果表明，这种分子具有良好的耐受性，并在体内暴露，可以支持其与其他两种辛纳稳定剂SION-719和SION-451组合使用。这两个NBD1稳定剂。

began clinical testing

开始临床测试

last summer. Interim data as of mid-January indicate that both have been well tolerated by patients so far, the company said in the filing.

去年夏天。该公司在文件中表示，截至1月中旬的中期数据表明，到目前为止，患者对这两种药物的耐受性都很好。

Vertex’s twice-daily drug Trikafta, a combination of two correctors and one potentiator, has become a standard of care therapy for CF patients who have at least one F508del mutation in the CFTR gene. This drug, approved in 2019, has also become the company’s top-selling product. In the nine months ended Sept.

Vertex的每日两次药物Trikafta是两种校正剂和一种增强剂的组合，已成为CFTR基因中至少有一个F508del突变的CF患者的标准治疗方法。这种药物于2019年获得批准，也成为该公司最畅销的产品。在截至9月9日的9个月内。

30, 2024, Trikafta (marketed as Kaftrio in Europe) accounted for $7.5 billion in sales, up 14% compared to the same period in 2023. This past December, the FDA .

2024年3月30日，Trikafta（在欧洲以Kaftrio的名义销售）销售额达到75亿美元，与2023年同期相比增长了14%。去年12月，FDA。

approved Vertex’s Alyftrek

批准Vertex的Alyftrek

, which offers the same drug combination in a once-daily pill. Neither Trikafta nor Alfytrek address NBD1.

，它在每日一次的药丸中提供相同的药物组合。Trikafta和Alfytrek都没有地址NBD1。