SAN DIEGO, Oct. 8, 2024 /PRNewswire/ -- Regulus Therapeutics Inc. (Nasdaq: RGLS), a biopharmaceutical company focused on the discovery and development of innovative medicines targeting microRNAs (the 'Company' or 'Regulus'), today announced it has completed enrollment of 26 patients in the fourth cohort in the Phase 1b MAD study of RGLS8429 for the treatment of ADPKD..

圣地亚哥，2024年10月8日/PRNewswire/--Regulus Therapeutics Inc.（纳斯达克：RGLS），一家专注于发现和开发针对microRNA的创新药物的生物制药公司（“公司”或“Regulus”），今天宣布已完成RGLS8429治疗ADPKD的1b期MAD研究第四组26名患者的登记。。

'The completion of enrollment of our final cohort marks an important milestone as we near the end of our Phase 1b study. We have been pleased to see positive results from each of the prior three cohorts, showing continued evidence of a mechanistic dose response based on increase in urinary polycystins (PC1 and PC2), across cohorts.

“随着我们接近1b期研究的结束，我们最终队列的注册完成标志着一个重要的里程碑。我们很高兴看到前三个队列中的每一个都取得了积极的结果，显示出基于队列中尿多囊蛋白（PC1和PC2）增加的机制剂量反应的持续证据。

Additionally, exploratory imaging analysis has shown mean reductions in height adjusted total kidney volume (htTKV), a key measurement which we will be evaluating in the fourth cohort and in the potentially pivotal Phase 3 trial,' said Jay Hagan, CEO of Regulus. 'We remain on track for an End of Phase 1 meeting with the FDA by the end of 2024, and we plan to share topline data from a substantial number of patients in cohort four in early 2025.'.

此外，探索性影像学分析显示，身高调整后的总肾脏体积（htTKV）平均减少，这是我们将在第四组和潜在关键的3期试验中评估的关键指标，”Regulus首席执行官杰伊·哈根（JayHagan）说我们仍有望在2024年底前与FDA完成第一阶段会议，并计划在2025年初分享第四组大量患者的主要数据。”。

The Phase 1b MAD study is a double-blind, placebo-controlled, weight-based dosing and an open-label fixed dose 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 (cohort 1-3) and one fixed dose level (cohort 4), including measuring changes in urinary polycystins PC1 and PC2, height adjusted total kidney volume (htTKV), cyst architecture, and overall kidney function.

1b期MAD研究是一项双盲，安慰剂对照，基于体重的剂量和开放标签的固定剂量试验，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（队列1-3）和一种固定剂量水平（队列4）的RGLS8429治疗，包括测量尿多囊蛋白PC1和PC2的变化，身高调整后的总肾体积（htTKV），囊肿结构和整体肾功能。

PC1 and PC2 are the protein products of the PKD1 and PKD2 genes and have been shown to inversely correlate with disease severity. Patients in the fourth cohort are receiving a fixed dose of 300 mg of RGLS8429 administered every other week for three months..

PC1和PC2是PKD1和PKD2基因的蛋白质产物，已显示与疾病严重程度呈负相关。第四组患者每隔一周服用300 mg固定剂量的RGLS8429，持续三个月。。

More information about the MAD clinical trial is available at clinicaltrials.gov (NCT05521191).

有关MAD临床试验的更多信息，请访问clinicaltrials.gov（NCT05521191）。

About ADPKD

关于ADPKD

Autosomal dominant polycystic kidney disease (ADPKD), caused by mutations in the PKD1 or PKD2 genes, is among the most common human monogenic disorders and a leading cause of end-stage renal disease. The disease is characterized by the development of multiple fluid filled cysts primarily in the kidneys, and to a lesser extent in the liver and other organs.

由PKD1或PKD2基因突变引起的常染色体显性多囊肾病（ADPKD）是最常见的人类单基因疾病之一，也是终末期肾病的主要原因。该疾病的特征是主要在肾脏中形成多个充满液体的囊肿，而在肝脏和其他器官中则较少。

Excessive kidney cyst cell proliferation, a central pathological feature, ultimately leads to end-stage renal disease in approximately 50% of ADPKD patients by age 60. Approximately 160,000 individuals are diagnosed with the disease in the United States alone, with an estimated global prevalence of 4 to 7 million..

肾囊肿细胞过度增殖是一个重要的病理特征，最终导致60岁左右的ADPKD患者中约50%的终末期肾病。仅在美国就有大约160000人被诊断出患有这种疾病，估计全球患病率为400万至700万。。

About RGLS8429

关于RGLS8429

RGLS8429 is a novel, next generation oligonucleotide for the treatment of ADPKD designed to inhibit miR-17 and to preferentially target the kidney. Administration of RGLS8429 has shown clear improvements in kidney function, size, and other measures of disease severity in preclinical models. Regulus announced completion of the Phase 1 SAD study in September 2022.

RGLS8429是一种新型的下一代寡核苷酸，用于治疗ADPKD，旨在抑制miR-17并优先靶向肾脏。在临床前模型中，RGLS8429的给药显示出肾功能，大小和其他疾病严重程度指标的明显改善。Regulus于2022年9月宣布完成第一阶段SAD研究。

The Phase 1 SAD study demonstrated that RGLS8429 has a favorable safety and PK profile. RGLS8429 was well-tolerated with no serious adverse events reported and plasma exposure was approximately linear across the four doses tested. In the Phase 1b MAD study Regulus announced topline data from the first cohort of patients in September 2023, from the second cohort of patients in March 2024 and from the third cohort of patients in June 2024.

第一阶段SAD研究表明，RGLS8429具有良好的安全性和PK特征。RGLS8429耐受性良好，未报告严重不良事件，血浆暴露在四个剂量的测试中大致呈线性。。

Regulus completed enrollment in the fourth cohort of patients in October 2024. Patients in the fourth cohort are receiving a 300 mg fixed dose of RGLS8429 administered every other week for three months..

Regulus于2024年10月完成了第四组患者的登记。第四组患者每隔一周服用300 mg固定剂量的RGLS8429，持续三个月。。

About Regulus

关于Regulus

Regulus Therapeutics Inc. (Nasdaq: RGLS) is a biopharmaceutical company focused on the discovery and development of innovative medicines targeting microRNAs. Regulus has leveraged its oligonucleotide drug discovery and development expertise to develop a pipeline complemented by a rich intellectual property estate in the microRNA field.

Regulus Therapeutics Inc.（纳斯达克：RGLS）是一家生物制药公司，专注于发现和开发针对microRNA的创新药物。Regulus利用其寡核苷酸药物发现和开发专业知识开发了一条管道，并在microRNA领域拥有丰富的知识产权。

Regulus maintains its corporate headquarters in San Diego, CA..

Regulus的公司总部位于加利福尼亚州圣地亚哥。。

Forward-Looking Statements

前瞻性声明

Statements contained in this press release regarding matters that are not historical facts are 'forward-looking statements' within the meaning of the Private Securities Litigation Reform Act of 1995, including statements associated with the Company's RGLS8429 program and preclinical pipeline, the potential that RGLS8429 may be eligible for an Accelerated Approval pathway, potentially achieving therapeutic efficacy and clinical translation for patients, the expected timing for reporting interim or topline data, and the timing and future occurrence of other preclinical and clinical activities.

本新闻稿中关于非历史事实的声明是1995年《私人证券诉讼改革法》所指的“前瞻性声明”，包括与公司RGLS8429计划和临床前管道相关的声明，RGLS8429可能有资格获得加速批准途径的可能性，可能为患者实现治疗效果和临床翻译，报告中期或topline数据的预期时间，以及其他临床前和临床活动的时间和未来发生。

Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Words such as 'believes,' 'anticipates,' 'plans,' 'expects,' 'intends,' 'will,' 'goal,' 'potential' and similar expressions are intended to identify forward-looking statements.

由于此类报表存在风险和不确定性，因此实际结果可能与此类前瞻性报表明示或暗示的结果存在重大差异。诸如“相信”、“预期”、“计划”、“期望”、“打算”、“意志”、“目标”、“潜力”等词语以及类似的表达方式旨在识别前瞻性陈述。

These forward-looking statements are based upon Regulus' current expectations and involve assumptions that may never materialize or may prove to be incorrect. Actual results and the timing of events could differ materially from those anticipated in such forward-looking statements as a result of various risks and uncertainties, which include, without limitation, the risk that the approach we are taking to discover and develop drugs is novel and may never lead to marketable products, that preliminary or topline results are based on a preliminary analysis of key efficacy and safety data, and such data may change following a more comprehensive review of the data related to the clinical trial and may not be indicative of future results, the FDA has not designated RGLS8429 for an Accelerated Approval pathway and such designation may not lead to a fa.

这些前瞻性陈述基于Regulus目前的预期，并涉及可能永远不会实现或可能被证明不正确的假设。由于各种风险和不确定性，实际结果和事件发生的时间可能与此类前瞻性声明中预期的结果存在重大差异，这些风险和不确定性包括但不限于，我们发现和开发药物的方法是新颖的，可能永远不会产生适销对路的产品，初步或一线结果是基于对关键疗效和安全性数据的初步分析，这些数据可能会在对与临床试验相关的数据进行更全面的审查后发生变化，并且可能不会指示未来的结果，FDA尚未将RGLS8429指定为加速批准途径，这种指定可能不会导致fa。

SOURCE Regulus Therapeutics Inc.

SOURCE Regulus Therapeutics Inc。

WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM?

想在PRNEWSWIRE上发布贵公司的新闻。COM？

440k+

44万+

Newsrooms &

新闻编辑室&

Influencers

影响者

9k+

9千+

Digital Media

数字媒体

Outlets

出口

270k+

27万+

Journalists

记者

Opted In

选择加入

GET STARTED

快速入门