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。

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."

nd 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.

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