The End points 100 gives a C-suite thumbs down on 'crip pling' price pro pos als and Aduhelm. Plus: Is any one go ing to JP ...

端点100给了一个C组拇指向下‘瘫痪’价格亲pos als和Aduhelm。另外:有人去JP吗...

2021-10-11 22:30:07 ENDPOINTS NEWS


After tracking the opinion of the Endpoints 100 — biotech execs, mostly CEOs, who give us semi-annual feedback on key issues and trends — for the past four years, one thing stands out: Opinions tend to run along lines of consensus; by and large they track as a pack. So when right at 60% of the 81 execs participating in the H2 survey turn thumbs down on whether the FDA should have OK’d Aduhelm — that ultra controversial and very expensive Alzheimer’s drug waved through at the agency despite the clear lack of positive efficacy data — it’s not hard to conclude that a clear majority aren’t having it. With another 22% undecided still, you have some lingering doubts that need to be resolved. But only 18% are in the solid ‘yes’ camp — leaving Biogen with little in the way of professional industry support in its lonely corner. The biotech sector has come down firmly from the highs of February as negative sentiment takes hold. The sector had a major boost of optimism from the success of the COVID-19 vaccines, making investors keenly aware of the potential of biopharma R&D engines. But from early this year, clinical trial, regulatory and access setbacks have reminded investors of the sector’s inherent risks. RBC Capital Markets recently surveyed investors to take the temperature of the market, a mix of specialists/generalists and long-only/ long-short investment strategies. Heading into the second half of the year, investors mostly see the sector as undervalued (49%), a large change from the first half of the year when only 20% rated it as undervalued. Around 41% of investors now believe that biotech will underperform the S&P500 in the second half of 2021. Despite that view, 54% plan to maintain their position in the market and 41% still plan to increase their holdings. Once riding high with its non-opioid drug for knee pain, Flexion was at the center of the M&A rumor mill for a 10-figure buyout. Years later, that shine has worn off — and now Flexion will hand over the keys in a significantly smaller acquisition. Pacira BioSciences will acquire Flexion for $8.50 per share in a deal valued at $450 million, a steep discount for a biotech that once commanded attention from the likes of drug giant Sanofi, the companies said Monday. The FDA has slapped a clinical hold on Allogene’s clinical trials after a patient in one of their early-stage studies for the off-the-shelf cell therapy ALLO-501A developed a “chromosomal abnormality” in their CAR-T cells. The biotech $ALLO reported after the market close on Thursday that executives plan to follow up in the coming weeks with regulators as the FDA considers the biotech’s plan to take this drug into a pivotal trial. Supernus is marking its expansion into the Parkinson’s disease arena, striking a deal to buy out Adamas Pharmaceuticals in a deal worth more than $400 million. A year after swooping in on its first Parkinson’s acquisition, the two companies announced that they had reached an agreement for Supernus to pay up to $9.10 per share in cash as part of the acquisition — close to $450 million. Two longtime trustees of Dana-Farber Cancer Institute have resigned in the wake of an investigation by the Boston Globe’s Spotlight team into its somewhat unorthodox practice of allowing trustees to invest in its biotech spinouts and even — at one time — negotiate on behalf of those startups. Marc Cohen, a serial biotech entrepreneur and investor who co-founded the protein degradation pioneer C4 Therapeutics with Jay Bradner, and retired Harvard Business School professor Malcolm Salter said they will now focus on advising the institute’s venture philanthropy program, launched last year. They joined the board in 2004 and 1998, respectively, before resigning last week. Sumitovant Biopharma’s subsidiary Enzyvant late Friday won approval for its first treatment, which is also the first promising treatment for babies without a thymus, an organ that plays a crucial role in helping to fight infections. The treatment, known as Rethymic (allogeneic processed thymus tissue-agdc), is a one-time regenerative therapy for what’s formally known as congenital athymia. Enzyvant, which licensed the drug from Duke University in 2017, said that usually about 20 babies per year in the US are born with the condition, and many often die from infections or autoimmune manifestations by age two or three. Two years after Bayer swallowed the remaining shares of his regenerative cell therapy company, Emile Nuwaysir has found himself a new “big hairy goal”: in vivo cell therapy. Ensoma Therapeutics, an ambitious and well-backed startup out of Fred Hutch and the University of Washington, has tapped Nuwaysir as the new CEO. It’ll be the serial executive’s second stint as a CEO after BlueRock, where he shepherded a first-of-its-kind cell therapy for Parkinson’s disease into clinical trials. Biotech deal cash continued to gush through Q3, but a careful look at the details in aggregate suggests that the rosy tint to the biotech boom has started to lose some of its color. Once again, deal master Chris Dokomajilar at DealForma has crunched the numbers on all the gauges we use to assess the performance of the biotech sector. And there’s been a distinct cooling of overall activity, particularly around IPOs and venture cash infusions. Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here. R&D strategies are central to any biopharma company — and especially the larger ones. John Carroll has invited a stellar slate of guests to talk about exactly that at the European Biopharma Summit coming up next week. You can view the line up and reserve a spot here.
在过去的四年里,跟踪了终点100人的观点--生物技术高管,大多数是CEO,他们每半年向我们提供一次关键问题和趋势的反馈--后,有一件事很突出:观点往往沿着共识的路线运行;总的来说,他们是一群人跟踪的。 因此,当参与H2调查的81名高管中有60%的人对FDA是否应该批准Aduhelm持反对意见时--尽管明显缺乏积极的疗效数据,但这种极具争议且非常昂贵的阿尔茨海默氏症药物在该机构获得了通过--不难得出结论,显然大多数人没有使用它。还有22%的人还没有决定,你有一些挥之不去的疑问需要解决。但只有18%的人坚定地支持Biogen--这使得Biogen在其孤独的角落几乎没有专业行业的支持。 由于负面情绪占据主导地位,生物技术板块已从2月份的高点稳步回落。新冠肺炎疫苗的成功大大增强了该行业的乐观情绪,使投资者敏锐地意识到生物制药研发引擎的潜力。但从今年年初开始,临床试验、监管和准入方面的挫折提醒投资者该行业固有的风险。 加拿大皇家银行资本市场(RBC Capital Markets)最近对投资者进行了调查,以了解市场的温度,这是专家/通才和只做多/多空投资策略的混合。进入下半年,投资者大多认为该行业被低估(49%),与上半年只有20%的人认为该行业被低估相比有了很大变化。大约41%的投资者现在认为,生物技术将在2021年下半年表现低于标准普尔500。尽管有这种观点,54%的人计划维持市场头寸,41%的人仍计划增持。 Flexion曾凭借其治疗膝盖疼痛的非阿片类药物一路高歌猛进,成为10位数收购的并购谣言的中心。多年后,这种光芒已经消失--现在Flexion将在一项规模明显较小的收购中交出钥匙。 两家公司周一表示,帕奇拉生物科学公司将以每股8.50美元的价格收购Flexion,交易价值4.5亿美元,这对一家曾引起制药巨头赛诺菲等公司关注的生物技术公司来说是一个巨大的折扣。 美国食品和药物管理局暂停了Allogene的临床试验,因为在他们的一项现成细胞疗法ALLO-501A的早期研究中,一名患者在他们的CAR-T细胞中出现了“染色体异常”。 生物技术公司$allo在周四收盘后报告称,随着美国食品和药物管理局考虑生物技术公司将这种药物进行关键试验的计划,高管们计划在未来几周与监管机构跟进。 Supernus标志着其向帕金森病领域的扩张,达成了一项价值超过4亿美元的收购Adamas Pharmaceuticals的协议。 在首次收购帕金森一年后,两家公司宣布,他们已经达成协议,Supernus将支付高达每股9.10美元的现金作为收购的一部分--接近4.5亿美元。 达纳-法伯癌症研究所(Dana-Farber Cancer Institute)的两名长期受托人辞职,此前《波士顿环球报》(Boston Globe)的聚光灯团队(Spotlight)调查了该研究所(Boston Globe)允许受托人投资其生物技术分拆公司,甚至一度代表这些初创公司谈判的有点非正统的做法。 马克·科恩(Marc Cohen)是一名连续的生物技术企业家和投资者,他与杰伊·布拉德纳(Jay Bradner)共同创建了蛋白质降解先驱C4治疗学(C4 Therapeutics),退休的哈佛商学院教授马尔科姆·索尔特(Malcolm Salter)表示,他们现在将专注于为该研究所去年启动的风险慈善项目提供建议。他们分别于2004年和1998年加入董事会,上周辞职。 Sumitovant Biopharma的子公司酵素周五晚些时候赢得了其第一种治疗方法的批准,这也是第一种对没有胸腺的婴儿有希望的治疗方法,胸腺是一种在帮助抗击感染方面发挥关键作用的器官。 这种治疗被称为Rethymic(异体处理胸腺组织-AGDC),是一种一次性的再生治疗,用于治疗形式上被称为先天性胸腺炎的疾病。2017年从杜克大学获得该药物许可的酵素公司表示,通常美国每年约有20名婴儿出生时患有这种疾病,许多人经常在两三岁时死于感染或自身免疫表现。 拜耳吞并其再生细胞治疗公司剩余股份两年后,埃米尔·努韦希尔(Emile Nuwaysir)为自己找到了一个新的“大目标”:体内细胞治疗。 Fred Hutch和华盛顿大学的雄心勃勃、资金雄厚的初创公司Ensoma Therapeutics已经任命Nuwaysir为新首席执行官。这将是这位连续高管继BlueRock之后的第二次首席执行官任期,在BlueRock中,他带领一种首次用于帕金森病的细胞疗法进入临床试验。 生物技术交易现金在第三季度继续涌出,但仔细观察总体细节表明,生物技术繁荣的玫瑰色已经开始失去一些颜色。 DealForma的交易大师克里斯·多科马吉拉尔(Chris Dokomajilar)再次分析了我们用来评估生物技术行业表现的所有指标的数字。总体活动明显降温,尤其是围绕IPO和风险投资的现金注入。 欢迎回到终结点周刊,您对本周顶级生物制药头条的评论。想每周六早上把这个放在你的收件箱里吗?当前端点读者可以访问他们的读者配置文件以每周添加端点。新端点?在这里报名。 研发战略是任何生物制药公司的核心--尤其是较大的公司。约翰·卡罗尔邀请了一大群嘉宾在下周即将举行的欧洲生物制药峰会上谈论这个问题。你可以在这里查看排队并预订一个位置。