SAN DIEGO, Sept. 13, 2023 /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 in the second cohort of patients in the ongoing Phase 1b MAD study of RGSL8429 for the treatment of ADPKD..

圣地亚哥，2023年9月13日/PRNewswire/-Regulus Therapeutics Inc.（纳斯达克股票代码：RGLS），一家专注于发现和开发针对microRNA的创新药物（“公司”或“Regulus”）的生物制药公司，今天宣布已完成RGSL8429正在进行的用于治疗ADPKD的1b期MAD研究中第二批患者的入组。。

'The completion of enrollment in our second cohort is another exciting milestone in our efforts to bring RGLS8249 to individuals living with ADPKD,' said Jay Hagan, CEO of Regulus. 'I am looking forward to watching this cohort progress through the study as well as sharing topline data from our first cohort in the coming weeks.'.

Regulus首席执行官Jay Hagan说：“在我们努力将RGLS8249带到ADPKD患者身上的另一个令人兴奋的里程碑。”我期待通过研究观察这一队列的进展，并在未来几周内分享我们第一批队列的顶级数据。

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 will evaluate RGLS8429 treatment across three different dose levels, including measuring changes in polycystins, 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）。该研究将评估三种不同剂量水平的RGLS8429治疗，包括测量多囊蛋白的变化，高度调整的总肾体积（htTKV），囊肿结构和整体肾功能。

The first cohort was dosed at 1 mg/kg of RGLS8429 or placebo every other week for three months, with top-line data expected to be reported in the coming weeks. Patients in the second cohort are receiving 2 mg/kg of RGLS8429 or placebo every other week for three months. The Company expects to begin dosing the third cohort following a review of all available cohort 2 safety data, which is planned for October 2023..

第一组每隔一周给予1mg/kg的RGLS8429或安慰剂三个月，预计在未来几周内报告顶线数据。第二组患者每隔一周接受2 mg/kg RGLS8429或安慰剂治疗3个月。该公司预计在审查所有可用的第2组安全性数据后开始给药第三组，计划于2023年10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 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ADPKD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小。

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种主要的病理特征，到60岁时，约有50%的ADPKD患者最终导致终末期肾脏疾病。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 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 robust data in preclinical models, where clear improvements in kidney function, size, and other measures of disease severity have been demonstrated along with a superior pharmacologic profile in preclinical studies compared to Regulus' first-generation compound, RGLS4326.

关于RGLS8429 RGLS8429是用于治疗ADPKD的新型下一代寡核苷酸，其设计用于抑制miR-17并优先靶向肾脏。RGLS8429的给药在临床前模型中显示出稳健的数据，其中与Regulus的第一代化合物RGLS4326相比，已经证明肾功能，大小和其他疾病严重程度测量的明显改善以及临床前研究中的优异药理学特征。

Regulus announced completion of the Phase 1 SAD study in September 2022. 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 and is similar to the PK data from the first-generation compound.

Regulus宣布于2022年9月完成第一阶段SAD研究。1期SAD研究表明RGLS8429具有良好的安全性和PK特性。RGLS8429耐受性良好，未报告严重不良事件，血浆暴露在所测试的四个剂量中近似线性，与第一代化合物的PK数据相似。

In April 2023, Regulus announced completion of enrollment for the first cohort of patients in the Phase 1b MAD study and after review of all available safety data, advanced to the second cohort where patients will receive 2 mg/kg of RGLS8429 or placebo every other week for three months. The Company also recently completed the 27-week chronic toxicity study of RGLS8429 in mice.

2023年4月，Regulus宣布完成1b期MAD研究中第一批患者的入组，并在审查所有可用安全性数据后，进入第二队列，患者每隔一周接受2 mg/kg RGLS8429或安慰剂治疗为期三个月。该公司最近还完成了RGLS8429在小鼠中进行的为期27周的慢性毒性研究。

No RGLS8429-related toxicity, including CNS effects, was observed at any dose level up to the top dose of 300 mg/kg administered every other week..

在任何剂量水平下均未观察到RGLS8429相关毒性，包括CNS效应，直至每隔一周施用300mg/kg的最高剂量。。

About 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.

关于Regulus Regulus Therapeutics Inc.（纳斯达克股票代码：RGLS）是一家生物制药公司，致力于发现和开发针对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个管道，并在microRNA领域拥有丰富的知识产权。

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

Regulus在加利福尼亚州圣地亚哥维持其公司总部。。

Forward-Looking Statements Statements contained in this presentation 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, the expected timing for initiating clinical studies, potentially achieving therapeutic efficacy and clinical translation for ADPKD patients, the expected timing for reporting topline data, the timing and future occurrence of other preclinical and clinical activities and the expected length of our cash runway.

本演示文稿中包含的有关非历史事实事项的前瞻性陈述是1995年“私人证券诉讼改革法案”含义内的“前瞻性陈述”，包括与公司RGLS8429计划相关的声明，启动临床研究的预期时机，可能为ADPKD患者实现治疗效果和临床翻译，报告一线数据的预期时机，其他临床前和临床活动的时机和未来发生以及我们cash跑道的预期长度。

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.

由于此类陈述存在风险和不确定性，因此实际结果可能与此类前瞻性陈述所表达或暗示的结果大不相同。“相信”，“预期”，“计划”，“期望”，“意图”，“将会”，“目标”，“潜力”等词语旨在确定前瞻性陈述。

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 approach we are taking to discover and develop drugs is novel and may never lead to marketable products, preliminary or initial results may not be indicative of future results, preclinical and clinical studies may not be successful, risks related to regulatory review and approval, risks related to our reliance on third-party collaborators and other third parties, risks related to intellectual property, risks associated with the process of discovering, developing and commercia.

这些前瞻性陈述基于Regulus当前的预期，涉及可能永远不会实现或可能证明不正确的假设。由于各种风险和不确定性，实际结果和事件发生时间可能与此类前瞻性声明中预期的结果有很大差异，其中包括但不限于我们发现和开发药物的方法新颖且可能永远不会导致到可销售的产品，初步或初步结果可能无法指示未来的结果，临床前和临床研究可能不成功，与监管审查和批准相关的风险，与我们依赖第三方合作者和其他第三方相关的风险，与知识产权相关的风险，与发现，开发和商业化过程相关的风险。

SOURCE Regulus Therapeutics Inc.

来源Regulus Therapeutics Inc。