Vertex Selects Drug Combo, a Potential Therapy for 90% of CF Patients

福泰三联疗法VX-445 III期试验达主要终点,预计治疗高达90%的囊性纤维化患者

2019-05-31 13:41:00 Xconomy

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Vertex Pharmaceuticals is already a leader in cystic fibrosis therapies. The company is placing its bet on an experimental drug that could strengthen its dominant position. Vertex said it will file for regulatory approval of VX-445, an experimental drug that the company estimates could treat up to 90 percent of cystic fibrosis (CF) patients. It’s one of two experimental three-drug combination therapies that the Boston company had advanced to late-stage testing. After reporting earlier this year that both experimental drug combos met Phase 3 goals, Vertex said it would decide in the second quarter which of the two it would submit to regulators. Vertex said Thursday that it determined VX-445 could benefit the greatest number of CF patients. Shares of Vertex (NASDAQ: VRTX) rose about 1.3 percent in mid-morning trading. Cystic fibrosis is an inherited disorder caused by mutations to the gene that produces a protein that helps cells pump water in and out. Without that protein, the lungs, pancreas, and other organs develop a thick buildup of mucus that impairs breathing and serves as a breeding ground for bacterial infections. Vertex’s earlier CF drugs treat patients with specific genetic mutations. But the company has tried to broaden its reach through drug combinations. VX-445 combines the experimental Vertex drug elexacaftor with two of the company’s approved CF therapies, tezacaftor and ivacaftor. The other Vertex three-drug cocktail combines the experimental VX-659 with tezacaftor and ivacaftor. After four weeks of treatment, Vertex said VX-659 and VX-445 improved lung function by an average of 14 and 13.8 percentage points respectively, compared to a placebo. Showing lung function improvement was the main goal of the Phase 3 studies. With both drugs performing comparably, Vertex said in March that it would further evaluate the two treatments after 24 weeks. Vertex released 24-week data Thursday showing that patients given VX-445 showed improved lung function by an average 14.3 percentage points. The company did not provide 24-week data for VX-659. Vertex said its selection of VX-445 for regulatory submission was based on a number of factors including safety, tolerability, and how it interacts with other drugs.
福泰制药公司已经是囊性纤维化治疗的领导者。该公司正押注于一种可以巩固其主导地位的实验药物。 福泰(Vertex)说,它将申请 VX-445的监管批准,该公司估计这种实验药物可以治疗高达90%的囊性纤维化( CF )患者。这是波士顿公司提出的两种实验性的三种药物联合疗法中的一种。 福泰(Vertex)今年早些时候曾报道称,这两个实验药物组合都达到了第3阶段的目标,并表示将在第二季度决定向监管机构提交这两个药物组合中的哪一个。福泰(Vertex)周四表示,它确定 VX-445能使最多的 CF 患者受益。 福泰(Vertex)( NASDAQ : VRTX )股价在早盘中上涨约1.3%。 细胞纤维化是一种遗传性疾病,由基因突变引起,产生一种蛋白质,帮助细胞泵入和流出水。如果没有这种蛋白质,肺、胰腺和其他器官就会形成一层粘稠的粘液,损害呼吸,成为细菌感染的繁殖基地。 福泰(Vertex)早期的 CF 药物治疗特定基因突变患者。但该公司一直试图通过药物组合扩大业务范围。VX-445将实验福泰(Vertex)药物 elexacaffer 与公司批准的两种 CF 疗法( tezacaffer 和 ivacaffer )相结合。另一个福泰(Vertex)三药鸡尾酒结合实验 VX-659与 tezacaffer 和 ivacaver 。 福泰(Vertex)说,经过4周的治疗, VX-659和 VX-445分别改善肺功能14和13.8个百分点。显示肺功能改善是第三阶段研究的主要目标。福泰(Vertex)在3月份表示,由于这两种药物的表现都相当不错,它将在24周后进一步评估这两种治疗方法。 福泰(Vertex)周四公布的24周数据显示, VX-445患者肺功能改善平均14.3个百分点。该公司没有提供 VX-659的24周数据。福泰(Vertex)说,选择 VX-445作为提交给监管机构的药物是基于一些因素,包括安全性、耐受性以及它与其他药物的相互作用。

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