勃林格殷格翰今日宣布，公司II期临床试验BALANCE-CF^{TM [1]}招募到首位患者，该试验旨在评估应对囊性纤维化的全新潜在治疗手段。^[1],[2]试验将探索在患有囊性纤维化的成人和青少年患者中，在现有标准治疗的基础上加用不同剂量的吸入性上皮钠通道(ENaC)抑制剂与安慰剂相比对肺功能的影响。^[1],[3],[4]

囊性纤维化患者中存在的基因突变，可导致各种器官的粘液变得粘稠。^[5]在肺部，粘稠的粘液可以堵塞气道，滞留细菌，继而引发感染、炎症、呼吸衰竭和其他并发症。^[6]吸入的ENaC抑制剂(BI 1265162)被设计用于阻止钠的吸收，从而保持气道表面湿润，有助于稀释粘液，使囊性纤维化患者更易保持气道通畅。^[7],[8]

ENaC抑制剂旨在用于治疗所有类型的囊性纤维化突变的患者。^[1],[9] 该药可通过能倍乐® ^[1] 吸入器吸入。能倍乐®是勃林格殷格翰研发的一款吸入装置，以软雾的形式将药物缓慢送出，即便对有呼吸困难的患者而言也十分便于吸入。^[10],[11]囊性纤维化患者通常面临很高的治疗负担，有时需花费数小时进行多种治疗。^[12] BI 1265162 ENaC抑制剂通过能倍乐® ^[1]喷雾器递送药物，让患者可快速吸入。其给药方式不受空间限制，旨在作为标准治疗的添加治疗。

“我们很高兴为这项2期试验招募到首位患者，我们也希望公司的ENaC抑制剂能被证实为囊性纤维化患者带来获益。”炎症治疗领域医学负责人Kay Tetzlaff博士说道，“勃林格殷格翰致力于研究新的治疗手段，用以满足严重的、未被满足的医疗需求。我们希望这个药物，连同其给药方式一起，可以为饱受这一致残性疾患困扰的患者带来重要的改变。”

囊性纤维化是一种进行性遗传性疾病，可导致持续的肺部感染，并随着时间的推移而限制患者的呼吸能力。^[6] 尽管近年来治疗选择得到了改善，患者仍面临着不断恶化的健康状况，中位预期寿命仅为四十多岁。^[12]

参考文献

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