新功能、新界面、新体验,扫描即可下载生物谷APP!
首页 » BIOON报道 » Cytokinetics修正ALS药物tirasemtiv研发数据错误

Cytokinetics修正ALS药物tirasemtiv研发数据错误

来源:生物谷 2013-07-24 22:35

 

2013年7月24日讯 /生物谷BIOON/ --制药公司Cytokinetics公司被迫修订其研发的ALS药物tirasemtiv的研发数据错误。为了修正这一错误,公司重新制定了新的中期研究计划,并将原定的志愿者规模由500人扩大至680人。据了解,这项错误发生在参与患者数据采集系统中,有58名志愿者本应该服用实验药物却被错误的提供了安慰剂。这一错误也使得该药物的开发成本增加了近500万美元。

肌萎缩性脊髓侧索硬化症(ASL)又被称为路格里克氏病,是一种中枢神经系统遭受破坏的病症,可导致瘫痪甚至死亡,目前并未有太多治疗方案。(生物谷Bioon.com)

详细英文报道:

Forced to remedy an error in a study for a potential ALS drug, Cytokinetics ($CYTK) has revealed plans to amend the protocol of the midstage trial with expanded enrollment. The change boosts the number of patients in the study from 500 to 680 people, and at least one analyst thinks the larger data set could bolster the case for an expedited route to market for the drug.

South San Francisco-based Cytokinetics tackled fixes for the so-called Benefit-ALS study after discovering a programming error in its vendor's electronic data capture system for the trial, which caused 58 patients who were supposed to receive the company's experimental drug tirasemtiv to get placebo. The company is increasing enrollment and changing the statistical methods section of the study, expecting $5 million in added costs and a three-month delay in the trial, which is expected to report results in early 2014.

However, there could be a silver lining to the changes, especially if the data from the Phase IIb study are positive. Leerink Swann analyst Joseph Schwartz commented on the potential for Cytokinetics to use the larger data set in a case for accelerated approval of tirasemtiv, as the FDA sometimes allows companies to commercialize their therapies for patients with serious unmet medical needs after successful midstage studies.

Amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig's disease, certainly fits the bill of a disease without ample treatment options. The incurable disease attacks the central nervous system, leading to eventual paralysis and in most cases death after patients lose their ability to control muscles needed to sustain life. Biogen Idec ($BIIB), Regeneron Pharmaceuticals ($REGN) and other companies have left a trail of trial failures in the ALS field with various treatment approaches that never panned out.

Cytokinetics could reverse the trend with positive data on tirasemtiv, a fast skeletal muscle troponin activator. The experimental drug follows the company's lead candidate, omecamtiv mecarbil, a potential med for heart failure under development with partner Amgen ($AMGN). Key data from the lead program are expected in September.

 

温馨提示:87%用户都在生物谷APP上阅读,扫描立刻下载! 天天精彩!


相关标签

最新会议 培训班 期刊库