J&J buys an ear ly-stage gene ther a py for the eye — the on ly known pro gram for an un der-the-radar biotech

强生公司为眼睛购买了一种耳后期基因,这是众所周知的一种不受雷达监视的生物技术

2020-12-03 01:33:19 ENDPOINTS NEWS

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Just weeks af­ter MeiraGTx tout­ed their lat­est up­date on da­ta emerg­ing from their gene ther­a­py al­liance with J&J on in­her­it­ed reti­nal dis­eases, the phar­ma gi­ant is back with an ac­qui­si­tion tai­lored to beef up their R&D ef­forts in the field. This deal is fo­cused on HMR59, an ear­ly-stage gene ther­a­py at a small biotech called Hemera Bio­sciences. They’re keep­ing the terms un­der wraps for now, but J&J is grab­bing a once-and-done gene ther­a­py that is de­signed to pro­tect lev­els of CD59, a key pro­tein that guards the reti­na. Very lit­tle is on dis­play about this Waltham, MA-based biotech. The on­ly drug they re­fer to on their web­site is HMR59. There’s no word what, if any­thing, is left to work on. Ac­cord­ing to LinkedIn, Adam Rogers, who has an MD from Emory, launched the com­pa­ny 10 years ago. By pro­tect­ing CD59 in the eye, they be­lieve the treat­ment can play a big role pro­tect­ing the vi­sion of pa­tients suf­fer­ing from ge­o­graph­ic at­ro­phy, a se­vere form of age-re­lat­ed mac­u­lar de­gen­er­a­tion. J&J based the deal on the Phase I study that’s been com­plet­ed, with an­oth­er ear­ly-stage study now in the clin­ic fo­cused on safe­ty. There’s been con­sid­er­able work launched for gene ther­a­pies in this field since Spark — now owned by Roche — won a pi­o­neer­ing OK for their treat­ment for Leber con­gen­i­tal amau­ro­sis, a rare ge­net­ic reti­nal dis­ease 3 years ago. Just last sum­mer Bio­gen added to that with an in-li­cens­ing pact on a gene ther­a­py for in­her­it­ed reti­nal dis­eases. “Ge­o­graph­ic at­ro­phy is a dev­as­tat­ing form of AMD that im­pacts the abil­i­ty to ac­com­plish every­day tasks, such as read­ing, dri­ving, cook­ing, or even see­ing faces,” said James List, Janssen’s glob­al ther­a­peu­tic area head, car­dio­vas­cu­lar & me­tab­o­lism. “Our aim with this nov­el, sin­gle-ad­min­is­tra­tion gene ther­a­py is to use our de­vel­op­ment ex­per­tise and deep her­itage in vi­sion care to help im­prove pa­tient out­comes by in­ter­ven­ing ear­ly, halt­ing the pro­gres­sion to blind­ness, and pre­serv­ing more years of sight.” In 2006, a clinical trial called ILLUMINATE halted abruptly. Trial subjects treated with an investigational drug called torcetrapib started experiencing unexpected cardiovascular problems, in some cases resulting in death. After 15 years and nearly 1 billion dollars, the development of torcetrapib froze. Nine years later, a paper published in the journal Circulation explained how the ILLUMINATE disaster could have been predicted and prevented if just 9 proteins had been measured in clinical trial participants following initial treatments. In the oncology world, there’s no better hunting ground for cancer R&D execs than Genentech. The biotech franchise at Roche has worked on some of the leading drugs in the field, proven themselves with blockbuster returns, and carries weight for whatever it says and does. The exodus of R&D talent out of the South San Francisco hub is a testament to their success. Now one of their top research execs has been raided by a top China biotech player to satisfy not just their need for an oncology R&D chief as they build up their muscle in discovery and drug development, but also add a spotter for new cancer drug deals. Pfizer and BioNTech have scored a historic OK for their Covid-19 vaccine in the UK, where the first doses are expected to be delivered within days. Days after the British government authorized its Medicines & Healthcare Products Regulatory Agency to make a call independent of the European Union — from which it’s in the process of exiting — the regulators gave the green light for emergency use of BNT162b2. Two years after tiptoeing into the protein degradation space with a discovery pact centered on neurodegeneration, AbbVie is wading deeper to explore the more common applications in cancer and immunological diseases. The pharma giant is handing Frontier Medicines $55 million upfront to get things started while promising to reimburse its preclinical R&D costs. In addition to drug candidates that target E3 ligases — a key member of the body’s natural degradation system — Frontier will also be scouting small molecule binders to targets. After recording close to a billion dollars in investment earnings over 9 months, driven mostly by Moderna’s soaring stock, Merck is cashing out. The company sold its stake in $MRNA — which it’s held since 2015 — in the first half of the quarter, causing a temporary 2.5% dip in Moderna’s share price before the market opened on Wednesday. By late morning, the stock was back up 3.4%. The news comes just two weeks before an FDA advisory committee meeting to discuss Moderna’s application for emergency use of its Covid-19 vaccine. Eighteen months after Exelixis signed on to partner with Iconic Therapeutics on an antibody-drug conjugate, more money is flowing. Exelixis is picking up its exclusive option on the tissue factor-targeting ADC now known as XB002 (formerly ICON-2), Iconic’s lead program. Exelixis is adding $20 million to the $7.5 million it paid upfront. Part of that goes to ZymeWorks, which assisted with their ZymeLink ADC tech. Six years after Merck and Bristol Myers Squibb captured the attention of the oncology world with the first approval of their PD-1 drugs Keytruda and Opdivo, sales revenue has started to level off after a host of rivals joined the hunt for new OKs for metastatic conditions, where the FDA has proven quick to act. But a key analyst covering biopharma believes that there’s a vast, still largely untapped frontier for new approvals to come in the adjuvant setting that could once again ignite the growth of these leading cancer franchises. And once again, he’s pointing to the 2 leaders in the field as the most likely players to come out ahead — way, way ahead. Roughly five weeks after buying out Asklepios — also known as AskBio — for up to $4 billion, Bayer is hammering home its focus on cell and gene therapies. The Big Pharma announced Wednesday that it is establishing a cell and gene therapy platform within its pharmaceuticals division in order to consolidate all of its related projects under one umbrella. Researchers will continue working and developing experimental drugs on their own, Bayer said, while the execs will lay out and define the overall strategy and look to swing some more deals. Roche and Blueprint Medicines are continuing to put pressure on rivals Eli Lilly and Loxo, as their Gavreto drug picked up a new approval Wednesday. The companies announced that the FDA has given the thumbs-up for Gavreto to treat RET-mutation and RET-fusion positive thyroid cancers, less than three months after the drug received its first approval in non-small cell lung cancer with RET fusions. Gavreto is now set to fully compete with Lilly’s Retevmo after the latter’s brief period as the only developer with an OK’ed medicine for this specific gene alteration.
就在MeiraGTx发布了他们与强生在基因治疗方面的最新数据后的几周,这家公司又回来了,并要求加强他们在该领域的研发力量。 这笔交易涉及HMR59,这是一家名为Hemera Bio-Sciences的小型生物技术公司的一种耳期基因疗法。他们暂时保留了这些条款,但强生正在抢夺一种一次性的基因疗法,它被设计用于保护CD59的水平,CD59是保护视网膜的关键基因。 关于这个位于马萨诸塞州沃尔瑟姆的生物技术项目,很少有人提出质疑。他们在网站上使用的药物是HMR59。没有任何文字,如果有任何东西,留下了什么工作。拥有埃默里医学博士的亚当•罗杰斯10年前与LinkedIn建立了联系,并推出了这款产品。 通过在眼睛中保护CD59,他们认为这种治疗可以在保护患者的视力方面起到很大的作用,这是一种不同形式的年龄相关的Mac Degraphic Atrophy(一种年龄相关的Mac Degraphic)。 强生的协议基于已经完成的第一阶段研究,以及目前在临床上以安全性为基础的第二阶段研究。 自从Spark(现为罗氏公司所有)3年前因治疗Leber Congeni-tal Amaurosis(一种罕见的遗传病)而获得PioNeering OK后,该领域的基因治疗就开始了一些可持续的工作。就在去年夏天,mer bio-gen公司又加入了一项针对她的视网膜疾病的基因治疗的协议。 Janssen的全球治疗和医疗领域主管James List说:“地理图形技术是AMD的一种发展形式,它使人们能够完成每天的工作,比如阅读,烘焙,烹饪,甚至是看脸。”“我们的目的是利用我们在视力护理方面的经验和深度,帮助通过提高听力,阻止失明,并提前更长时间的视力,来改善视力。” 2006年,一项名为ILLUMINATE的临床试验突然中止。试验对象使用一种名为torcetrapib的研究药物治疗后,开始出现意想不到的心血管问题,在某些情况下导致死亡。经过15年和近10亿美元,torcetrapib的开发冻结了。 九年后,发表在《循环》杂志上的一篇论文解释了如果在最初治疗后的临床试验参与者中只测量了9种蛋白质,那么照明灾难是如何被预测和预防的。 在肿瘤学领域,没有比基因泰克更适合癌症研发高管的猎场了。罗氏的生物技术专营权已经开发出了该领域的一些领先药物,并以惊人的回报证明了自己,而且无论它说什么,做什么都有分量。 研发人才从旧金山南部的研发中心大量外流,是他们成功的证明。 如今,他们的一位顶级研究高管被一位中国顶尖生物技术公司突袭,这不仅是为了满足他们在研发和药物开发方面增强实力时对肿瘤研发主管的需求,还为新的癌症药物交易增加了一名观察家。 辉瑞和BioNTech公司的Covid-19疫苗在英国获得了历史性的认可,首批疫苗预计将在几天内交付。 在英国政府授权其药品和医疗保健产品监管机构独立于欧盟发出呼吁几天后,监管机构为紧急使用BNT162B2开了绿灯,该机构目前正处于退出欧盟的过程中。 在凭借一项以神经变性为中心的发现协议蹑手蹑脚地进入蛋白质降解领域两年后,AbbVie正在更深入地探索癌症和免疫学疾病中更为常见的应用。 这家制药巨头向Frontier Medicinals支付了5500万美元的预付款,以启动项目,同时承诺偿还其临床前研发费用。除了靶向E3连接酶(人体自然降解系统的关键成员)的候选药物外,Frontier还将寻找靶向小分子结合剂。 默克公司在过去9个月里取得了近10亿美元的投资收益,主要是受Moderna股价飙升的推动,现在正在进行套现。 该公司在上半季度出售了自2015年以来持有的$mRNA股份,导致Moderna股价在周三开盘前短暂下跌2.5%。截至上午晚些时候,该公司股价回升3.4%。这一消息是在FDA顾问委员会开会讨论Moderna紧急使用其Covid-19疫苗申请的两周前发布的。 在Exelixis签约与Iconic Therapeutics合作研制抗体-药物结合物18个月后,更多的资金正在流入。 Exelixis正在选择组织因子靶向ADC的独家选项,现在被称为XB002(原ICON-2),ICONIC的主导项目。Exelixis公司在预付750万美元的基础上又增加了2000万美元。其中一部分归ZymeWorks公司,该公司协助他们的ZymeLink ADC技术。 默克公司和百时美施贵宝公司首次批准其PD-1药物Keytruda和Opdivo,引起了肿瘤学界的关注。六年后,随着许多竞争对手加入到针对转移性疾病的新型OKs的搜寻中来,销售收入开始趋于平稳,FDA证明这方面的行动很快。 但一位负责生物制药的主要分析师认为,在辅助药物方面,仍有一个巨大的,基本上尚未开发的新领域,可能会再次点燃这些领先的癌症专营权的增长。再一次,他指出这两个领域的领导者是最有可能取得领先的球员--非常非常领先。 在斥资40亿美元收购Asklepios(又名AskBio)大约五周后,拜耳公司将重点放在了细胞和基因疗法上。 这家大型制药公司周三宣布,它正在其制药部门内建立一个细胞和基因治疗平台,以便将其所有相关项目整合在一个伞下。拜耳公司表示,研究人员将继续自行研发试验药物,而高管们将制定和定义总体战略,并寻求达成更多交易。 罗氏和Blueprint医药公司继续对竞争对手礼来和洛索施加压力,他们的Gavreto药物周三获得了新的批准。 两家公司宣布,FDA对Gavreto治疗RET突变和RET融合阳性甲状腺癌给予了大拇指的赞赏,这距离Gavreto首次被批准用于RET融合的非小细胞肺癌还不到三个月。在礼来公司的Retevmo短暂地作为唯一一个开发这种特定基因改变药物的公司之后,Gavreto现在将与后者展开全面竞争。

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