Regulus Therapeutics Inc. (RGLS) announced Monday positive topline results from the third cohort of patients in its Phase 1b Multiple-Ascending Dose (MAD) clinical trial of RGLS8429 for the treatment of Autosomal Dominant Polycystic Kidney Disease or ADPKD.In pre-market activity on the Nasdaq, Regulus shares were gaining around 14 percent to trade at $2.86.The Phase 1b MAD study is a double-blind, placebo-controlled trial evaluating the safety, tolerability, pharmacokinetics and pharmacodynamics (PK/PD) of RGLS8429 in adult patients with ADPKD.The study is evaluating RGLS8429 treatment across three different weight-based dose levels and one fixed dose level, including measuring changes in urinary polycystins 1 and 2 (PC1 and PC2), htTKV, cyst architecture, and overall kidney function.The company noted that enrollment in the fourth cohort, which will include up to 30 patients, has been initiated and patients will receive an open label fixed dose of 300 mg of RGLS8429 every other week for three months.

Regulus Therapeutics Inc.（RGLS）周一宣布，在RGLS8429治疗常染色体显性多囊肾病或ADPKD的1b期多重递增剂量（MAD）临床试验中，第三组患者的topline结果呈阳性。在纳斯达克的上市前活动中，Regulus股价上涨约14%，收于2.86美元。1b期MAD研究是一项双盲安慰剂对照试验，评估RGLS8429在成人ADPKD患者中的安全性，耐受性，药代动力学和药效学（PK/PD）。该研究正在评估三种不同的基于体重的剂量水平和一种固定剂量水平的RGLS8429治疗，包括测量尿多囊蛋白1和2（PC1和PC2），htTKV，囊肿结构和整体肾功能的变化。该公司注意到，第四组（最多包括30名患者）的登记已经开始，患者将每隔一周接受300毫克RGLS8429的开放标签固定剂量，为期三个月。

In addition to PC1 and PC2 and safety, imaging biomarkers will also be evaluated..

除了PC1和PC2以及安全性外，还将评估成像生物标志物。。

In the third cohort, RGLS8429 was well tolerated with no safety concerns, and continued evidence of a mechanistic dose response was observed following completion of 3 mg/kg dose level based on urinary biomarker analyses PC1 and PC2.Jay Hagan, CEO of Regulus Therapeutics, said, 'Building on the positive results from our first two cohorts, these cohort 3 data, particularly the reduction in htTKV seen in the majority of patients, further strengthen our conviction in RGLS8429's ability to potentially address the underlying, genetic cause of ADPKD.

在第三个队列中，RGLS8429耐受性良好，没有安全性问题，并且根据尿液生物标志物分析PC1和PC2，在完成3 mg/kg剂量水平后，观察到持续的机械剂量反应证据。Regulus Therapeutics首席执行官Jay Hagan说，“基于我们前两个队列的积极结果，这些队列3数据，特别是大多数患者中htTKV的降低，进一步加强了我们对RGLS8429潜在解决ADPKD潜在遗传原因的能力的信念。

We anticipate requesting an End-of-Phase 1 meeting with the FDA in the fourth quarter of this year and look forward to providing a data update from the open-label fourth and final cohort of the Phase 1b MAD study by year-end.'For More Such Health News, visit rttnews.com For comments and feedback contact: editorial@rttnews.comBusiness News.

我们预计将于今年第四季度结束与FDA的第一阶段会议，并期待在年底前提供1b期MAD研究开放标签第四也是最后一组的数据更新。”有关更多此类健康新闻，请访问rttnews.com获取评论和反馈联系方式：editorial@rttnews.comBusiness新闻。

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