In side Bio gen's scram ble to sell Aduhelm: Pro ject 'Javelin' and pres sure to ID as many pa tients as pos si ble

在Bio Gen的scramble出售aduhelm:Pro ject‘标枪’和pres肯定ID和pos si ble一样多的pa times

2021-07-22 15:00:30 ENDPOINTS NEWS

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In anticipation of Aduhelm’s approval for Alzheimer’s in June, Biogen employees were directed to identify and guarantee treatment centers would administer the drug through a program called “Javelin,” a senior Biogen employee told Endpoints News. The program identified 800 centers for use, he said, and Biogen now pays for the use of bioassays to identify beta amyloid in potential patients having undergone a lumbar puncture procedure, the employee said — and one center preparing to administer the drug confirmed its participation in the bioassay program. Always remember just how risky it is to gamble big on small studies. A little more than 4 years ago, Novartis reportedly put up a package worth up to $1 billion for the dry eye drug ECF843 after a small biotech called Lubris put it through its paces in a tiny study of 40 moderate to severe patients, tracking some statistically significant markers of efficacy. By last fall, the program had risen up to become one CEO Vas Narasimhan’s top “wild card” programs in line for a potential breakthrough year in 2021. These drugs were all considered high-risk, high-reward efforts. And in this case, risk won. Earlier this summer, Magenta Therapeutics was forced to say goodbye to its head of R&D at a pivotal time as the biotech read out data for its lead stem cell conditioning hopeful. Just weeks later, the company’s second drug is facing a clinical hold from the FDA as the agency demands more info on how the candidate works in humans. Magenta will be required to submit an additional bioassay to help determine the best path forward on dose escalation for the biotech’s Phase I/II study of MGTA-117, an antibody-drug conjugate used to deplete hematopoietic stem cells prior to a transplant of HSC-based gene therapy, the biotech said Wednesday. The manufacturing expansion into Africa continued Wednesday with Pfizer and BioNTech’s announcement that the two companies signed on with the Biovac Institute to manufacture its Covid-19 vaccine to distribute throughout Africa. But while the continent is in dire need of doses, vaccines from the expansion won’t be available until the start of next year. Biovac will only handle distribution and fill-finish duties. Drug substance for these batches will come from European facilities. When fully operational, the facility will pump out 100 million doses a year, set to be evenly distributed among the 55 member states of the African Union. With the recent and unexpected discovery of probable human carcinogen impurities prompting recalls of popular heartburn drugs like Zantac and nizatidine, as well as metformin diabetes medicines, ARBs and more, the FDA has made clear the need for an industry-wide risk assessment for the so-called nitrosamines in even more drugs that may be at risk. But industry groups are pushing back on the need for such widespread testing, arguing that the FDA should be more consistent and collaborative with other regulators in its approach, and that the timelines for such testing are too constricted and may lead to serious drug shortages. With the wind of the pandemic at its back, the biopharma industry saw a surging tide of fundraising in 2020 that has only exponentially grown this year. But with VC dollars flowing like never before, are there some cracks showing in biopharma’s gold-tinted façade? In the first half of 2021, total healthcare venture spending in the US has already outstripped 2020’s record-breaking totals across biotech, healthtech and beyond, putting the industry on a funding pace that could be hard to match in coming years, according to a new report from Silicon Valley Bank released Wednesday. As the saga over how to pay for Aduhelm plays out, biopharma companies are now looking closely at how Biogen was able to keep such a special relationship with FDA officials ahead of the drug’s accelerated approval, and whether or not they might be able to leverage those takeaways into their own relationships with the agency. What makes Biogen unique, however, is how deep its relationship with the FDA ran, and the way in which FDA’s neuroscience head Billy Dunn worked alongside the company for years prior to Aduhelm’s quick OK. For instance, back in 2017, Dunn co-chaired a data sharing initiative at the nonprofit C-Path Institute alongside Biogen SVP Samantha Budd Haeberlein, as first reported by the New York Times. In 2018, Dunn and Budd Haeberlein also jointly presented on strengthening communication across the Alzheimer’s drug development community. As clinical trial transparency and the sharing of positive and negative trial results slowly become the norm rather than the exception, a new study published in the BMJ revealed struggles from smaller companies to keep up with their larger rivals when it comes to transparency. AbbVie, Amgen, Bayer, Merck KGaA/EMD Serono, Novartis, Roche and Takeda all tied for the top spot as the most transparent biopharma companies, the study found. Like so many people around the world, my life has been profoundly shaped by cancer. Those personal experiences, along with a deep love of clinical laboratory science and a passion to apply the power of genomics in medicine, motivated me to launch a company that would improve cancer care through better diagnostics. Thirteen years later, I am proud that we are delivering more accurate information at multiple points along the patient journey, with a focus on eight of the 10 cancers that are most commonly diagnosed in the United States. Albireo $ALBO CEO Ron Cooper is getting his long-awaited shot at marketing the first drug approved for pruritus in all subtypes of progressive familial intrahepatic cholestasis. The biotech put out word Tuesday afternoon that the FDA came through with the approval right on deadline, something that had been expected since the agency handed the company a priority review for the drug. That now gives them a green light to see how much of a head start they can get before Mirum finds out about the ultimate fate of its own PFIC program.
一名高级Biogen员工告诉Endpoints News,预计Aduhelm将在6月份批准阿尔茨海默氏症,Biogen员工被指示识别并保证治疗中心将通过一个名为“标枪”的项目来管理这种药物。 他说,该项目确定了800个使用中心,Biogen现在支付使用生物测试来识别接受腰椎穿刺手术的潜在患者的β淀粉样蛋白的费用,一个准备服用该药物的中心证实了其参与生物测试项目。 永远记住,把大赌注押在小研究上是多么危险。 据报道,4年多前,诺华公司为干眼症药物ECF843提供了价值高达10亿美元的包装,此前一家名为润滑的小型生物技术公司对40名中度至重度患者进行了一项小型研究,跟踪了一些具有统计学意义的疗效标志。 到去年秋天,该项目已经成为首席执行官瓦斯·纳拉西姆汉(Vas Narasimhan)的顶级“外卡”项目之一,有望在2021年取得突破性进展。这些药物都被认为是高风险、高回报的努力。在这种情况下,风险赢了。 今年夏天早些时候,品红治疗公司在一个关键时刻被迫告别其研发负责人,因为这家生物技术公司宣读了其领先的干细胞调节希望的数据。就在几周后,该公司的第二种药物正面临FDA的临床搁置,因为该机构要求更多关于候选药物在人类中如何工作的信息。 生物技术公司周三表示,洋红将被要求提交一份额外的生物测试,以帮助确定生物技术公司对MGTA-117的I/II期研究的最佳剂量升级途径。MGTA-117是一种抗体药物结合物,用于在移植基于HSC的基因治疗之前耗尽造血干细胞。 周三,辉瑞和生物技术公司宣布,这两家公司与Biovac研究所签署协议,生产其新冠肺炎疫苗,并在整个非洲分发,从而继续向非洲扩张生产。但是,尽管非洲大陆急需剂量,但扩张后的疫苗要到明年初才能上市。 Biovac将只处理分配和填充完成的职责。这些批次的原料药将来自欧洲设施。全面投入运营后,该设施将每年泵出1亿剂,将平均分配给非洲联盟55个成员国。 随着最近意外发现可能的人类致癌物杂质,引发了赞他克和尼扎替丁等热门烧心药物以及二甲双胍糖尿病药物、ARBs等的召回,美国食品和药物管理局明确表示,需要对更多可能有风险的药物中的所谓亚硝胺进行全行业风险评估。 但行业团体正在反对进行如此广泛的测试的必要性,认为美国食品和药物管理局应该在其方法上与其他监管机构更加一致和合作,这种测试的时间表过于狭窄,可能导致严重的药物短缺。 随着疫情的蔓延,生物制药行业在2020年迎来了激增的融资浪潮,今年只呈指数级增长。但是,随着风投资金前所未有地流动,Biopharma金色的外观是否出现了一些裂缝? 硅谷银行(Silicon Valley Bank)周三发布的一份新报告显示,2021年上半年,美国的医疗保健风险投资总额已经超过了2020年生物技术、医疗技术及其他领域的创纪录总额,使该行业的融资速度在未来几年可能很难跟上。 随着关于如何支付Aduhelm的传奇的展开,生物制药公司现在正在密切关注Biogen如何在药物加速批准之前与FDA官员保持如此特殊的关系,以及他们是否能够将这些外卖纳入自己与该机构的关系。 然而,Biogen的独特之处在于它与美国食品和药物管理局的关系有多深,以及在Aduhelm的《快速确定》之前,美国食品和药物管理局神经科学负责人比利·邓恩(Billy Dunn)与该公司合作多年的方式。例如,据《纽约时报》首次报道,早在2017年,邓恩就与生物技术高级副总裁萨曼莎·巴德·海伯莱一起在非营利组织C-Path研究所共同主持了一项数据共享倡议。2018年,Dunn和Budd Haeberlein还联合介绍了加强阿尔茨海默氏症药物开发社区的沟通。 随着临床试验透明度和阳性和阴性试验结果的共享慢慢成为常态而不是例外,发表在《英国医学杂志》上的一项新研究显示,在透明度方面,较小的公司难以跟上较大的竞争对手。 研究发现,艾伯维、安进、拜耳、默克集团/EMD雪兰诺、诺华、罗氏和武田都并列最透明生物制药公司的榜首。 像世界上许多人一样,癌症深刻地影响了我的生活。这些个人经历,加上对临床实验室科学的热爱和在医学中应用基因组学力量的热情,激励我创办了一家公司,通过更好的诊断来改善癌症护理。13年后,我感到自豪的是,我们在病人旅程的多个点提供了更准确的信息,重点是美国最常见的10种癌症中的8种。 Albireo$Albo首席执行官罗恩·库珀(Ron Cooper)正在获得他期待已久的机会,即将推出第一种被批准用于所有进行性家族性肝内胆汁淤积症亚型瘙痒的药物。 这家生物技术公司周二下午发布消息称,美国食品和药物管理局在截止日期前获得了批准,这是自该机构将该药物的优先审查交给该公司以来一直在意料之中的。这给了他们一个绿灯,看看在Mirum发现自己的PFIC项目的最终命运之前,他们可以领先多少。

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