Novartis announced that it has entered into an agreement to acquire Regulus Therapeutics, a San Diego-based, publicly traded clinical-stage biopharmaceutical company focused on developing microRNA therapeutics. Regulus’ lead asset, farabursen, is a potential first-in-class, next-generation oligonucleotide targeting miR-17 for the treatment of autosomal dominant polycystic kidney disease (ADPKD)..

诺华宣布已达成协议，收购位于圣地亚哥的公开上市临床阶段生物制药公司Regulus Therapeutics，该公司专注于开发微RNA疗法。Regulus的核心资产farabursen是一种潜在的首创、新一代寡核苷酸药物，靶向miR-17用于治疗常染色体显性多囊肾病（ADPKD）。

The agreed deal is fully in line with the therapeutic area focus of Novartis and leverages our strength and expertise in renal disease. “With limited treatment options currently available for patients suffering from ADPKD, farabursen represents a potential first-in-class medicine with a profile that may provide enhanced efficacy, tolerability and safety versus standard of care,” said Shreeram Aradhye, President, Development and Chief Medical Officer, Novartis.

双方达成的协议完全符合诺华在治疗领域的重点，并利用了我们在肾脏疾病方面的优势和专业知识。 “对于目前有限的常染色体显性多囊肾病（ADPKD）患者治疗选择，Farabursen代表了一种潜在的首创药物，其特性可能会比现有标准治疗提供更高的疗效、耐受性和安全性，”诺华公司开发总裁兼首席医学官Shreeram Aradhye表示。

“ADPKD is the most common genetic cause of renal failure worldwide. The team at Regulus has done meaningful foundational work with farabursen, and we look forward to investigating its potential further as we aim to bring a better treatment option to patients in need.” Farabursen is an investigational microRNA inhibitor designed to target miR-17 with preferential kidney exposure, aiming to reduce the growth of cysts and kidney size, as well as delay progression of disease severity in ADPKD.

“ADPKD是全球肾衰竭最常见的遗传原因。Regulus团队在farabursen方面开展了卓有成效的基础工作，我们期待进一步研究其潜力，以期为有需要的患者提供更好的治疗选择。” Farabursen是一种在研的微RNA抑制剂，旨在优先靶向肾脏中的miR-17，目标是减少囊肿生长和肾脏体积，并延缓ADPKD疾病严重程度的进展。

In March 2025, Regulus announced the successful completion of its Phase 1b multiple-ascending dose clinical trial for farabursen..

2025年3月，Regulus宣布其farabursen的1b期多剂量递增临床试验已成功完成。

The Phase 1b trial data showed promising clinical efficacy and safety, including consistent impact on urinary polycystin (PC), a biomarker of mechanistic response, and height-adjusted total kidney volume (htTKV), a measure of progressive disease.

1b期试验数据显示出良好的临床疗效和安全性，包括对尿多囊蛋白（PC）这一机制反应的生物标志物以及身高调整后的总肾体积（htTKV）这一疾病进展指标的一致影响。

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