打开APP

La Jolla治疗慢性肾病药物二期临床结果良好

  1. Galectin-3
  2. La Jolla
  3. 慢性肾病

来源:生物谷 2014-03-13 11:04

La Jolla生物技术公司上周刚刚完成了其治疗慢性肾病的药物GCS-100得二期临床试验,其理想结果增强了投资者信心,助涨公司股价。

2014年3月12日讯 /生物谷BIOON/ --La Jolla生物技术公司上周刚刚完成了其治疗慢性肾病的药物GCS-100得二期临床试验。在这项有121名慢性肾病患者参加的研究中,研究人员测试了两种剂量GCS-100的治疗效果。其中低剂量GCS-100能明显改善患者病情延缓其纤维化进程;而高剂量GCS-100疗效并不明显。GCS-100是通过阻断一种galectin-3的蛋白质表达来实现这一作用的。研究人员分析认为,高剂量GCS-100不明显的原因在于生物体反馈调节导致更多的galectin-3产生。不过在此次二期临床研究中,这两种剂量均未发现有明显副作用。而投资者也并未因为这点瑕疵而失去信心,此消息一经公布,公司股价应声上扬。

该项目负责人Pablo Pergola表示GCS-100将可以帮助越来越多的慢性肾病患者,目前这类疾病尚未能有特效疗法。而评论人士认为仅保守估计,这一市场到2024年就将达到200亿美元之多。(生物谷Bioon.com)

详细英文报道:

La Jolla Pharmaceutical's ($LJPC) chronic kidney disease drug hit its primary endpoint of improving organ function in a Phase II trial, the company said, and while the results sent the biotech's shares skyward by 75%, a quirk in dosing gave some analysts pause.

The study tested GCS-100 at two doses in 121 CKD patients, finding that the lower dose improved renal function at a statistically significant rate and met its secondary endpoint of cutting levels of galectin-3, a protein tied to tissue fibrosis. The higher dose, however, charted no statistically significant difference in either goal.

La Jolla blames the high-dose failure on what it calls a biologic feedback loop, with CEO George Tidmarsh telling investors that overinhibition of galectin-3 might spur the body to redouble production of the protein, negating GCS-100's effect. However, that explanation would appear to be inconsistent with the drug's Phase I results, in which La Jolla reported that a "statistically significant post-dose reduction of serum galectin-3 was also observed at the higher doses" of GCS-100.

But the dosing issue didn't scare off investors, who sent La Jolla's shares to an all-time high $19.17 in premarket trading on Wednesday, buying into the idea that GCS-100 could be the first drug approved for CKD, a big market with bigger unmet need.

"Chronic kidney disease is an enormous and growing medical problem worldwide," trial investigator and University of Texas professor Pablo Pergola said in a statement. "A disease-modifying agent that could slow and potentially reverse the tissue fibrosis that is a hallmark of this disease would be a welcome advance in the field and could have a major impact on patients' lives."

And Chardan Capital Markets analyst Ling Wang is all in on the drug's potential, telling Reuters that, if approved, it could gross more than $2 billion a year by 2024--"and that's a conservative estimate," she said.

On the safety side, both doses of GCS-100 were well-tolerated with no serious adverse events in the lower amount and two in the higher that were later deemed unrelated to the drug, La Jolla said.

 

版权声明 本网站所有注明“来源:生物谷”或“来源:bioon”的文字、图片和音视频资料,版权均属于生物谷网站所有。非经授权,任何媒体、网站或个人不得转载,否则将追究法律责任。取得书面授权转载时,须注明“来源:生物谷”。其它来源的文章系转载文章,本网所有转载文章系出于传递更多信息之目的,转载内容不代表本站立场。不希望被转载的媒体或个人可与我们联系,我们将立即进行删除处理。

87%用户都在用生物谷APP 随时阅读、评论、分享交流 请扫描二维码下载->