SAN DIEGO, Jan. 2, 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 in the third cohort of patients in the Phase 1b MAD study of RGLS8429 for the treatment of ADPKD..

圣地亚哥，2024年1月2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研究的第三组患者登记。。

'The completion of our third and final randomized placebo-controlled cohort in the Phase 1b MAD study is an exciting step for Regulus, and we are happy to reach this stage of development for RGLS8429,' said Jay Hagan, CEO of Regulus. 'Our team is motivated and gearing up for 2024 when we expect topline data from our second cohort in Q1 2024, topline data from our third cohort in mid-2024, and the initiation of the fourth cohort, an open label fixed dose, of RGLS8429.'.

Regulus首席执行官杰伊·哈根（JayHagan）说：“我们在1b期MAD研究中完成了第三个也是最后一个随机安慰剂对照队列，这对Regulus来说是一个令人兴奋的步骤，我们很高兴RGLS8429达到了这一发展阶段。”当我们预计2024年第一季度第二个队列的顶线数据，2024年年中第三个队列的顶线数据以及RGLS8429的第四个队列（开放标签固定剂量）的启动时，我们的团队有动力并为2024年做好准备。

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 weight-based 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）。该研究将在三种不同的基于体重的剂量水平上评估RGLS8429治疗，包括测量多囊藻毒素的变化，身高调整后的总肾体积（htTKV），囊肿结构和整体肾功能。

The third cohort is being dosed at 3 mg/kg of RGLS8429 or placebo every other week for three months. The protocol was amended to include a fourth cohort of subjects who will receive an open label fixed dose of RGLS8429 to compare biomarker and safety data to the weight-based dosing..

第三组每隔一周服用3 mg/kg的RGLS8429或安慰剂，持续三个月。该方案被修改为包括第四组受试者，他们将接受开放标签固定剂量的RGLS8429，以比较生物标志物和安全性数据与基于体重的剂量。。

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 ADPKDAutosomal 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.

关于ADPKD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，在肝脏和其他器官中的程度较小。

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 RGLS8429RGLS8429 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RGLS8429RGLS8429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。RGLS8429的给药在临床前模型中显示出可靠的数据，与Regulus的第一代化合物RGLS4326相比，肾功能，大小和其他疾病严重程度指标的明显改善以及临床前研究中优越的药理学特征。

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于2022年9月宣布完成第一阶段SAD研究。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，血浆暴露在四个测试剂量中大致呈线性，与第一代化合物的PK数据相似。

In the Phase 1b MAD study Regulus announced both top line data from the first cohort of patients, and completion of enrollment for the second cohort of patients in September 2023. After review of all available safety data, Regulus has advanced to the third cohort where dosing has begun, and patients will receive 3 mg/kg of RGLS8429 or placebo every other week for three months.

在1b期MAD研究中，Regulus宣布了第一组患者的一线数据，并于2023年9月完成了第二组患者的登记。在审查了所有可用的安全性数据后，Regulus已进入第三个队列，开始给药，患者将每隔一周接受3 mg/kg的RGLS8429或安慰剂，持续三个月。

Regulus announced completion of enrollment in the third cohort in January 2024..

Regulus宣布于2024年1月完成第三组的注册。。

About RegulusRegulus 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RegulusRegulus 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 StatementsStatements 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.

前瞻性声明本演示文稿中包含的关于非历史事实的声明是1995年《私人证券诉讼改革法案》所指的“前瞻性声明”，包括与公司RGLS8429计划相关的声明，启动临床研究的预期时间，可能为ADPKD患者实现治疗效果和临床翻译，报告基线数据的预期时间，其他临床前和临床活动的时间和未来发生以及我们现金跑道的预期长度。

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 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 commercial.

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，其中包括但不限于，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，初步或初步结果可能无法表明未来的结果，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，开发和商业过程相关的风险。

SOURCE Regulus Therapeutics Inc.

SOURCE Regulus Therapeutics Inc。