Novartis has reached an agreement to buy Regulus Therapeutics and its experimental therapy for autosomal dominant polycystic kidney disease (ADPKD) for $800 million upfront in a deal that could have a total value of up to $1.7 billion.

诺华已达成协议，将以8亿美元的预付款收购Regulus Therapeutics及其用于常染色体显性多囊肾病（ADPKD）的实验性疗法，该交易的总价值可能高达17亿美元。

Shares in the San Diego-based microRNA-directed medicine specialist rocketed more than 130% in pre-market trading on the announcement of the $7-per-share transaction, approaching a value of $8 at the time of writing.

总部位于圣地亚哥的微RNA导向药物 specialist 的股价在宣布每股7美元的交易后，在盘前交易中飙升了130%以上，截至撰写本文时已接近8美元。

Another $7 per share could be payable on approval of Regulus' lead drug, farabursen (formerly RGLS8429), which met the mark in a

每股7美元的额外付款可能在Regulus的主导药物farabursen（前称RGLS8429）获批时支付，该药物在一项...中达到了标准。

phase 1b trial

1b期试验

reported in March and has been due to start a phase 3 trial in the third quarter of this year.

该药物于今年3月上报，并计划在今年第三季度进入第三阶段试验。

ADPKD is a leading cause of end-stage renal disease and affects around 160,000 people in the US, with a global population between four and seven million. It currently has just one FDA-approved treatment – Otsuka's oral drug Jynarque (tolvaptan) – which was cleared in 2018 and had estimated US sales of $1.5 billion last year..

常染色体显性多囊肾病（ADPKD）是导致终末期肾病的主要原因之一，在美国影响约16万人，全球患者人数在四百万到七百万之间。目前，该疾病仅有一种获得FDA批准的治疗药物——大冢制药的口服药Jynarque（托伐普坦），该药物于2018年获批，去年在美国的销售额估计达到15亿美元。

Regulus' phase 1b data showed that miR-17-directed oligonucleotide farabursen achieved reductions in an ADPKD biomarker (polycystin) and slowed down the increase in kidney size that occurs in patients with the disease, as well as the formation of abnormal cysts.

Regulus的1b期数据显示，miR-17导向的寡核苷酸farabursen减少了ADPKD生物标志物（多囊蛋白）的水平，减缓了患者肾脏增大的速度，并抑制了异常囊肿的形成。

A fixed dose of 300mg given as a subcutaneous injection every other week looks set to be the dose taken forward into the phase 3 study, which is expected to include a 12-month kidney growth endpoint to support accelerated approval, and 24-month endpoint on estimated glomerular filtration rate (eGFR) – a widely-used measure of kidney function – for full approval..

每两周一次固定剂量300mg的皮下注射似乎将成为推进到第三阶段研究的剂量，该研究预计包括一个12个月的肾脏生长终点，以支持加速审批，以及一个24个月的估算肾小球滤过率（eGFR，一种广泛使用的肾功能指标）终点，以获得完全批准。

'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, Novartis' chief medical officer..

“对于目前治疗选择有限的ADPKD患者来说，farabursen代表了一种潜在的首创药物，其特性可能在疗效、耐受性和安全性方面优于现有标准疗法，”诺华首席医学官Shreeram Aradhye表示。

'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,' he added.

“Regulus 团队在 farabursen 方面做了有意义的基础工作，我们期待进一步研究其潜力，目标是为有需要的患者带来更好的治疗选择，”他补充道。

Regulus said earlier this year that it sees a 'multi-billion dollar opportunity' for farabursen based on the pressing need for new ADPKD treatments and a high level of willingness among physicians to prescribe the drug for high-risk patients.

今年早些时候，Regulus 表示，基于对新型 ADPKD 治疗方法的迫切需求以及医生为高危患者开药的高度意愿，公司认为 farabursen 存在“数十亿美元的机会”。

Buying Regulus ties neatly into Novartis' push into kidney disease therapies, with a portfolio that includes a trio of drugs to treat IgA nephropathy (IgAN) – two of which are

购买Regulus与诺华进军肾脏疾病治疗领域的战略紧密相连，其产品组合包括三种治疗IgA肾病（IgAN）的药物，其中两种是

already FDA-approved

已获FDA批准

– and

– 和

Fabhalta

法布阿尔塔

(iptacopan), which recently became the first licensed treatment in the US for C3 glomerulopathy (C3G).

(iptacopan)，该药物最近成为美国首个获批用于治疗C3肾小球病（C3G）的药物。

The group's IgAN pipeline stems in large part from its $3.5 billion takeover of

该集团的IgAN管线在很大程度上源于其35亿美元的收购

Chinook Therapeutics

奇努克治疗公司

in 2023, while last year Novartis set up a new biotech with the help of venture capital firm Versant, called

2023年，而去年诺华在风险投资公司Versant的帮助下成立了一家新的生物技术公司，名为

Borealis Biosciences

博瑞利斯生物科学公司

, that aims to deliver two new kidney disease candidates.

，目标是推出两种新的肾病候选药物。

The transaction is expected to close in the second half of 2025. Under the terms of the deal, Regulus will merge with a Novartis unit and become an indirect wholly owned subsidiary of the Swiss group.

该交易预计将在2025年下半年完成。根据协议条款，Regulus将与诺华的一个部门合并，并成为这家瑞士集团间接全资拥有的子公司。