An experimental Novartis drug helped bring an autoimmune condition causing low platelet counts

一种实验性的诺华药物有助于改善导致血小板计数低的自身免疫状况

under control in a Phase 3 trial

在三期试验中得到控制

, further lifting the prospects of a therapy the company acquired in a multibillion-dollar deal last year.

，进一步提升了该公司去年在一项数十亿美元的交易中获得的疗法的前景。

The drug, ianalumab, acts by destroying misfiring immune cells and blocking signaling that creates new ones. Novartis has been testing it in a disorder called immune thrombocytopenia, in which the body erroneously wipes out blood-clotting platelets. The company intends for the drug to work hand-in-hand with another therapy, Promacta, that it sells for the condition..

这种药物名为伊那鲁单抗，通过摧毁错误激活的免疫细胞并阻断生成新细胞的信号起作用。诺华公司一直在一种称为免疫性血小板减少症的疾病中进行测试，该疾病中身体会错误地清除帮助血液凝固的血小板。该公司打算让这种药物与其销售的另一种用于治疗该疾病的药物普罗玛克塔协同作用。

In a study called Vayhit2, the Swiss drugmaker tested whether a short course of the combination would help people whose platelet levels have fallen after initial treatment with steroids, and reduce the number of medications they require going forward.

在一项名为 Vayhit2 的研究中，这家瑞士制药商测试了这种组合的短期疗程是否能够帮助那些在最初使用类固醇治疗后血小板水平下降的患者，并减少他们后续所需的药物数量。

Strategies to shorten long-term care with a durable, short-course therapy have had “limited success,” said Hanny Al-Samkari, a hematologist at Massachusetts General Hospital. “There is a need for other treatment options.”

麻萨诸塞州总医院的血液科医生汉尼·阿尔-萨姆卡里说，用持久、短期治疗来缩短长期护理的策略“成效有限”，“需要其他治疗选择”。

Data Al-Samkari presented at the American Society of Hematology meeting Tuesday suggest ianalumab can help. The trial enrolled 152 people and randomized them to one of two different doses of ianalumab taken with Promacta, or Promacta and a placebo. Enrollees received four monthly infusions of ianalumab and daily doses of Promacta for 16 weeks, with an eight-week tapering of Promacta afterwards..

阿尔·萨姆卡里在周二的美国血液学会会议上提交的数据显示，依那鲁单抗可能有帮助。该试验招募了152人，并将他们随机分配到两组，分别接受两种不同剂量的依那鲁单抗与普罗玛塔联合治疗，或普罗玛塔与安慰剂联合治疗。参与者在16周内接受了四次每月一次的依那鲁单抗输注以及每日剂量的普罗玛塔，随后进行了为期八周的普罗玛塔逐渐减量。

The regimen’s main objective was to extend “disease control” by delaying the time it took for patients to require a rescue therapy or for their platelet counts to drop below a key threshold.

该方案的主要目标是通过延迟患者需要抢救治疗的时间或血小板计数降至关键阈值以下的时间来延长“疾病控制”。

Both doses of ianalumab achieved that objective. The higher dose reduced the risk of treatment failure by 45% compared to the placebo group, while the lower dose reduced that risk by 42%. People in the higher-dose arm went a median of 13 months before treatment failure, compared to 4.7 months for the placebo.

两种剂量的伊那鲁单抗都达到了这个目标。较高剂量组与安慰剂组相比，治疗失败的风险降低了45%，而较低剂量组则降低了42%。高剂量组的患者在治疗失败前的中位时间为13个月，而安慰剂组为4.7个月。

Higher-dose recipients also had a significantly higher rate of stable responses at six months, with 62% hitting that mark versus 39% of placebo patients..

高剂量组患者在六个月时的稳定反应率也显著更高，达到该标准的比例为62%，而安慰剂组患者为39%。

Adverse event rates were similar in all groups, and only one severe adverse event, heart palpitations, was considered related to ianalumab.

所有组的不良事件发生率相似，只有一例严重不良事件——心悸被认为与伊那鲁单抗有关。

Helping people with immune thrombocytopenia wean off of medications will improve their quality of life, said Vinod Pullarkat, professor of hematology at City of Hope cancer treatment center. “This could be paradigm-changing,” he said in an interview.

帮助免疫性血小板减少症患者逐渐停药将改善他们的生活质量，希望之城癌症治疗中心的血液学教授维诺德·普拉卡特说道。“这可能会改变范式，”他在一次采访中说道。

Novartis gained control of ianalumab through a $2.9 billion acquisition of

诺华通过一笔29亿美元的收购获得了伊那鲁单抗的控制权

German biotech MorphoSys

德国生物技术公司MorphoSys

. The main drug in that deal, pelabresib, has faced development setbacks, but ianalumab has succeeded in testing in

. 该交易中的主要药物pelabresib遭遇了研发挫折，但ianalumab在测试中取得了成功

Sjögren’s syndrome

干燥综合征

and, now, immune thrombocytopenia.

现在，免疫性血小板减少症。