Chinook Therapeutics, Inc.

正文共： 1348字 8图预计阅读时间： 4分钟诺华计划以8亿美元预付款收购位于圣地亚哥的生物科技公司Regulus Therapeutics，该公司专注于microRNA药物的研发。此次收购的核心资产是farabursen，这是一种针对与肾病相关的miR-17的候选药物，刚完成针对多囊肾病患者的1b期临床试验。收购Regulus的协议中，股东将获得每股7美元的现金，这一价格是周二收盘价的两倍多。此外，诺华还承诺，若达到某些未公开的审批标准，将额外支付每股7美元。Regulus成立于2007年，由Alnylam和Ionis联合创立，其技术灵感来源于《自然》杂志上一篇关于合成寡核苷酸抑制microRNA的论文。公司成立初期发展势头良好，曾与赛诺菲和阿斯利康等大型药企合作。然而，近年来研发遭遇连续失败，股价自2021年以来一直未能突破4美元。farabursen于2022年进入临床试验，针对的是研究人员认为与肾病相关的miR-17。目前，多囊肾病患者治疗选择有限，主要依赖托伐普坦等药物来延缓肾功能恶化。诺华在肾病领域的布局并非首次。2023年，诺华以30亿美元收购Chinook Therapeutics，获得了治疗IgA肾病的新药Venrafia。此外，诺华还计划扩展其已获批的IgA肾病药物Fabhalta的适应症，以覆盖更多肾病类型。诺华首席医疗官Shreeram Aradhye在领英上表示，此次收购加强了公司在肾病产品线上的实力，延续了在IgA肾病和补体3肾小球病治疗领域获批后的创新势头。诺华生物医学研究总裁Fiona Marshall此前向BioPharma Dive透露，公司一直在寻找有潜力的早期项目，并将多囊肾病视为肾病产品线的重点攻克目标。她强调，虽然内部研发项目很重要，但如果市场上有更优秀的项目，诺华也会果断收购。参考资料：Novartis to acquire Regulus Therapeutics and farabursen, an investigational microRNA inhibitor to treat ADPKD, the most common genetic cause of renal failureNovartis to acquire Regulus in deal for kidney disease drug其它公开资料图片来源：微信订阅号AI38笔！596亿美元！跨国药企2025年重磅交易达成，中国公司贡献很大2025-05-02【直击现场】「星起点」宣讲会直播：50万资金+专家辅导，寻找下一个代谢病创新药突破者！2025-04-30百济神州索托克拉冲刺上市，引领BCL2抑制剂新纪元！2025-04-30版权声明/免责声明本文为原创文章。本文仅作信息交流之目的，不提供任何商用、医用、投资用建议。文中图片、视频、字体、音乐等素材或为药时代购买的授权正版作品，或来自微信公共图片库，或取自公司官网/网络，部分素材根据CC0协议使用，版权归拥有者，药时代尽力注明来源。如有任何问题，请与我们联系。衷心感谢!药时代官方网站:www.drugtimes.cn联系方式:电话:13651980212微信:27674131邮箱:contact@drugtimes.cn点击这里，查看更多精彩!

Novartis has identified farabursen as a good fit for its burgeoning kidney disease portfolio.\n Novartis has committed another $1.7 billion to its ambition to become a kidney disease leader. The Swiss drugmaker is making the outlay, around half of which is upfront, to buy Regulus Therapeutics for a rare renal disease drug that is set to enter phase 3 in the third quarter.Regulus published top-line data from a phase 1b trial of its lead candidate, farabursen, in March. The trial enrolled patients with autosomal dominant polycystic kidney disease (ADPKD). People who received the oligonucleotide every other week for three months had statistically significant increases in urinary PC1 and PC2, proteins that inversely correlate with disease severity.Height-adjusted total kidney volume (htTKV), a predictor of renal insufficiency, increased 0.05% over four months in patients on farabursen. HtTKV grew 2.58% in the placebo cohort. Buoyed by the data, Regulus forged ahead with plans to start a phase 3 trial that could support a filing for accelerated approval.Novartis has identified farabursen as a good fit for its burgeoning kidney disease portfolio. The company has agreed to pay $7 a share upfront to acquire Regulus. The upfront payment, which amounts to $800 million, is more than double the closing price of Regulus’ stock Tuesday. Novartis has agreed to pay another $7 a share if Regulus achieves a milestone tied to the regulatory approval of farabursen. The total value, should all these targets be hit, would reach $1.7 billion.  Regulus has designed its phase 3 trial to support a filing for accelerated approval based on htTKV after 12 months. The trial will serve as its own confirmatory study, with the biotech tracking changes in a measure of kidney function, eGFR, out to 24 months to support full approval.If approved, farabursen will enter a market already served by Otsuka’s Jynarque. Otsuka said (PDF) U.S. sales “increased significantly” over the first three months of the year because ADPKD prescriptions rose. Regulus told investors in January that Jynarque is only used in about 7% of the addressable population, a fact the biotech’s CEO Joseph Hagan said is “in large part because of its safety and tolerability profile.”Regulus claimed farabursen is a multibillion-dollar opportunity. Novartis wants to seize the opportunity to advance the kidney care business that it has built out through deals such as the $3.2 billion Chinook Therapeutics acquisition and development of internal programs. The commercial portfolio now includes Fabhalta and Vanrafia. 

Novartis will pay $800 million upfront to acquire Regulus Therapeutics, a San Diego biotechnology company that launched nearly two decades ago with plans to make drugs capable of targeting small strips of nucleic acid known as microRNA.Announced Wednesday, the acquisition will hand the Swiss pharmaceutical firm a drug prospect called farabursen, which recently completed a Phase 1b study in people with autosomal dominant polycystic kidney disease, or ADPKD.Per deal terms, Regulus shareholders will receive $7 in cash per share, a premium of more than 100% to the stocks closing price Tuesday. Additionally, Novartis has committed to pay an additional $7 per share via a so-called contingent value right thats linked to the achievement of an unspecified regulatory milestone.Regulus was launched in 2007 as a joint venture between Alnylam Pharmaceuticals and Ionis Pharmaceuticals, building on a paper published in Nature on how a kind of synthetic oligonucleotide could silence microRNA, which plays a role in genetic regulation.After early successes, such as a 2010 partnership with Sanofi and a 2012 deal with AstraZeneca, the biotech hit setbacks from which it struggled to recover. It also had difficulty finding the right application for its drugmaking technology, trying its hand at a hepatitis C treatment that didnt pan out. Since 2021, Regulus shares have traded below $4 per share.The drug at the heart of its deal with Novartis, called farabursen, entered the clinic in 2022. Farabursen targets miR-17, which researchers have identified as potentially relevant to kidney disease.People with ADPKD have few treatment options, relying on the drug tolvaptan to slow the rate of kidney function decline, as well as pain relievers and blood pressure-lowering drugs.Acquiring Regulus is one of several investments Novartis has made in kidney disease, most notably a $3 billion deal in 2023 for Chinook Therapeutics that netted it Venrafia, which won accelerated approval to treat IgA nephropathy earlier this month. Its also developing Fabhalta, already cleared in IgAN, for several other kidney-related conditions.This is a meaningful addition to our renal portfolio as we continue driving innovation in kidney care, following our recent approvals of treatments for IgAN and C3G, Shreeram Aradhye, Novartis chief medical officer, wrote in a LinkedIn post.The pharma hinted earlier this year that it would continue to be active in dealmaking. Its president of biomedical research, Fiona Marshall, told BioPharma Dive that it was looking for early-stage assets. Novartis previously identified ADPKD as a target in its renal portfolio.So often, its having our own program that makes us really like the project, and then if we see somebody else is doing it better than us externally, well still bring that in, she said in January. '