News brief ing: FDA re quests new tri al for Reata's Friedre ich's atax ia pro gram; J&J's Trem fya picks up ex pand ed la bel ...

新闻简报:FDA寻求新的三种药物,为Reata的Friedre ich的atax ia Program;强生的Trem fya接了ex pand ed la bel.。。

2020-11-26 02:01:20 ENDPOINTS NEWS


Three months af­ter Rea­ta Phar­ma­ceu­ti­cals sug­gest­ed its Friedre­ich’s atax­ia pro­gram omavelox­olone could be de­layed, the com­pa­ny re­vealed that is in­deed go­ing to be the case. Rea­ta $RE­TA shares took a nose­dive Wednes­day af­ter the biotech re­vealed that the FDA said sup­ple­men­tal da­ta for its piv­otal tri­al did not strength­en the case for ap­proval. As a re­sult, the drug is like­ly to need an­oth­er study be­fore the FDA takes up the case. In ear­ly Wednes­day morn­ing trad­ing, Rea­ta stock was down rough­ly 8% off the news. It’s a small­er dive than when the com­pa­ny first said the pro­gram could be set back, though, as shares lost about a third of their val­ue at the time. As such, this news was not en­tire­ly un­ex­pect­ed but still proved dis­ap­point­ing to an­a­lysts. “While the path for­ward for omavelox­olone in the US re­mains to be de­ter­mined, we be­lieve it is large­ly de­risked by the pos­i­tive re­sults seen” in the study, SVB Leer­link’s Joseph Schwartz wrote to in­vestors. Baird’s Bri­an Sko­r­ney took a more bull­ish view, de­scrib­ing the up­date as mere­ly an “in­cre­men­tal neg­a­tive.” Sko­r­ney said he has been op­er­at­ing with a de­lay be­ing the “base-case as­sump­tion” and that the news doesn’t af­fect Rea­ta’s oth­er pro­gram, bar­dox­olone for rare kid­ney dis­eases, which is ex­pect­ed to be filed in the first quar­ter of 2021. The FDA did not rule out re­con­sid­er­ing omavelox­olone’s ap­pli­ca­tion once the new study has been com­plet­ed, Rea­ta not­ed. Four months af­ter nab­bing an ex­pand­ed la­bel in the US for ac­tive pso­ri­at­ic arthri­tis, J&J’s Trem­fya has got­ten the OK for the in­di­ca­tion in Eu­rope as well. Trem­fya, al­so known as guselkum­ab, had pre­vi­ous­ly been ap­proved on the con­ti­nent for mod­er­ate to se­vere plaque pso­ri­a­sis. The drug is a mon­o­clon­al an­ti­body tar­get­ing p19 in the IL-23 and in­hibits its in­ter­ac­tion with the IL-23 re­cep­tor. Wednes­day’s ap­proval comes on the ba­sis of two Phase III stud­ies look­ing at adults who had ac­tive PsA and those who were bi­o­log­ic-naïve. Both stud­ies looked at pa­tients who had pre­vi­ous­ly failed stan­dard ther­a­pies. Back when it first got US ap­proval in 2017, an­a­lysts pegged peak an­nu­al sales for the drug at $1.6 bil­lion, which would make it a top 20 drug. Through the first three quar­ters of 2020, Trem­fya had net­ted J&J and Janssen $965 mil­lion world­wide. Since January 2020, the clinical trials ecosystem has quickly evolved to manage roadblocks impeding clinical trial integrity, and patient care and safety amid a global pandemic. Closed borders, reduced air traffic and delayed or canceled flights disrupted global distribution, revealing how flexible logistics and supply chains can secure the timely delivery of clinical drug products and therapies to sites and patients. In mid-August, as Kodak’s $765 million government-backed push into drug manufacturing slowly fell apart in national headlines, Ginkgo Bioworks CEO Jason Kelly got a message from his company’s government liaison: HHS wanted to know if they, too, might want a loan. The government’s decision to lend Kodak three quarters of a billion dollars raised eyebrows because Kodak had never made drugs before. But Ginkgo, while not a manufacturing company, had spent the last decade refining new ways to produce materials inside cells and building automated facilities across Boston. Two biotechs said they got turned away by the FDA on Wednesday, in part due to pandemic-related travel restrictions. North Carolina-based Liquidia Technologies was handed a CRL for its lead pulmonary arterial hypertension drug, citing the need for more CMC data and on-site pre-approval inspections, which the FDA hasn’t been able to conduct due to travel restrictions. The agency also deferred its decision on Revance Therapeutics’ BLA for its frown line treatment, because it needs to inspect the company’s northern California manufacturing facility. The action, Revance emphasized, was not a CRL. → San Diego protein degradation startup Vividion Therapeutics has made a change at the top with Jeffrey Hatfield taking the helm as CEO, replacing Diego Miralles six months after Roche forked over $135 million to collaborate with Vividion on their small molecule degraders. Hatfield is chairman of the board at miRagen Therapeutics and previously held the CEO job at Zafgen and Vitae Pharmaceuticals. He also had a series of leadership roles at Bristol Myers Squibb from 1996-2004, including SVP, immunology and virology divisions. When Shanghai’s STAR board began opening its doors to biotech, it was considered not just a rival to Nasdaq but also the stock exchange in Hong Kong. Those perceptions may take an amicable turn as China expands a mutual access program with the city. The changes mean investors in mainland China will be able to own Hong Kong biotech chapter stocks, while those in Hong Kong — a much more internationally connected group — would have access to those listed on STAR. In effect, it turns the Shanghai market into a globally accessible exchange overnight while also broadening a key source of revenue for HKEX. It’s been a banner year for the once humdrum business of manufacturing drugs, particularly vaccines. Billions have been spent ramping up facilities for Covid-19 jabs, while individual CDMOs have expanded their facilities, apparently anticipating demand or responding to a government-led push to onshore drug manufacturing. Now Baxter Biopharma Solutions, the CDMO wing of the many-armed healthcare giant Baxter, is getting in on the game. On Tuesday, they announced plans to spend $50 million to expand their flagship, 600,000 square-foot facility in Bloomington, IN. Novartis is always one of the industry’s biggest R&D spenders. As they often do toward the end of each year, company execs are highlighting the drugs they expect will most likely be winners in 2021. And they’re also dreaming about some potential big-time lottery tickets. As part of its annual investor presentation Tuesday, where the company allows investors and analysts to virtually schmooze with the bigwigs, Novartis CEO Vas Narasimhan will outline what he thinks are the pharma’s “Wild Cards.” The slate of five experimental drugs are those that Novartis hopes can be high-risk, high-reward entrants into the market over the next half-decade or so, and cover a wide range of indications. On the third straight Monday that the world wakes up to positive vaccine news, AstraZeneca and Oxford are declaring a new Phase III milestone in the fight against the pandemic. Not everyone is convinced they will play a big part, though. With an average efficacy of 70%, the headline number struck analysts as less impressive than the 95% and 94.5% protection that Pfizer/BioNTech and Moderna have boasted in the past two weeks, respectively. But the British partners say they have several other bright spots going for their candidate. One of the two dosing regimens tested in Phase III showed a better profile, bringing efficacy up to 90%; the adenovirus vector-based vaccine requires minimal refrigeration, which may mean easier distribution; and AstraZeneca has pledged to sell it at a fraction of the price that the other two vaccine developers are charging. The European Union is looking at ways to bypass patent protections and make it easier to make generic drugs in cases of emergency such as the Covid-19 pandemic, a new document says. Normally, under WTO regulations, the practice known as “compulsory licensing” is allowed in exceptional circumstances and could be applied as a waiver to bypass patent holders. Wednesday’s document was published as part of the EU’s plan to shore up the intellectual property rights of its member states.
三个月后,ReaTa Pharmaceuticals建议其Friedreich的Ataxia Program OmaveloxoLone可以被删除,事实就是如此。 周三,美国食品药品监督管理局发布生物技术报告称,食品药品监督管理局对其“三价”产品的支持并不支持该产品的上市。此后,该公司股价大幅下挫,原因是食品药品监督管理局发布的生物技术报告称,食品药品监督管理局对其“三价”产品的支持并不支持该产品的上市。作为一个结果,在FDA开始处理这个病例之前,这种药物需要进一步的研究。 在周三早盘的交易中,ReaTa的股价在这一消息传出后大幅下跌了8%。不过,与委员会最初表示该计划可能会倒退时相比,这次下跌幅度较小,当时该公司股价下跌了约三分之一。 因此,这条新闻并不是完全不正确的,但事实证明,它仍然是不正确的。 SVB Leer-Link的约瑟夫•施瓦茨在给投资者的信中写道:“虽然奥马维洛龙在美国市场的发展道路有待确定,但我们相信,从研究中看到的结果来看,它的风险大大降低了。” Baird的Brian Skorney则持更为牛气的观点,将最新的新闻描述为仅仅是一个“不折不扣的坏账”。Skorney表示,他一直在用一个“基本案例”来描述这个问题,而这个新闻并不影响Rea的另一个计划,也就是“罕见的孩子们的坏账”,该计划预计将在2021年第一季度提交。 FDA并不排除在新研究完成后重新使用奥美洛龙的可能性。 四个月前,强生的Tremifya在美国接受了一次治疗关节疼痛的试用期,在欧盟也获得了10次试用期的认可。 Tremifya,也被称为Guselkumab,已经被证明是一个现代化的斑块PSOria。该药物是IL-23中p19的单克隆抗体,并抑制其与IL-23受体的相互作用。 周三的实验是基于两个III期研究的基础上进行的,研究对象分别是患有急性PsA的成年人和那些虚幻的成年人。这两个研究对象都是那些以前在标准治疗中不及格的学生。 当它在2017年首次获得美国批准时,科学家们预计该药的峰值销售额为16亿美元,这将使它跻身前20大药物之列。在2020年的前三个季度,Tremifya为强生和杨森带来了9.65亿美元的全球净收入。 自2020年1月以来,临床试验生态系统迅速发展,以管理在全球大流行中阻碍临床试验完整性,患者护理和安全的路障。边境关闭,空中交通减少以及航班延误或取消扰乱了全球分销,这揭示了灵活的物流和供应链如何能够确保临床药物产品和疗法及时送达现场和患者。 8月中旬,随着柯达政府支持的7.65亿美元药品生产计划在全国新闻头条上慢慢告吹,Ginkgo Bioworks的首席执行官杰森•凯利从公司的政府联络人那里得到了一个信息:HHS想知道他们是否也需要贷款。 政府借给柯达3.5亿美元的决定令人侧目,因为柯达以前从未制造过药品。但Ginkgo虽然不是一家制造公司,但在过去的十年里,它一直在完善在细胞内生产材料的新方法,并在波士顿各地建造自动化设施。 两名生物技术公司表示,他们周三被FDA拒之门外,部分原因是与大流行相关的旅行限制。 总部位于北卡罗来纳州的Liquidia Technologies获得了其主要肺动脉高压药物的CRL,理由是需要更多的CMC数据和现场审批前检查。由于旅行限制,FDA一直未能进行这些检查。该机构还推迟了对Revance Therapeutics的BLA用于其皱眉线治疗的决定,因为它需要检查该公司在北加利福尼亚的制造设施。Revance强调,这次行动不是一次CRL。 圣地亚哥蛋白质降解初创公司Vividion Therapeutics进行了高层调整,Jeffrey Hatfield接替Diego Miralles担任首席执行官,六个月前,罗氏花了1.35亿美元与Vividion合作研发小分子降解剂。哈特菲尔德是miRagen Therapeutics公司的董事长,之前曾在Zafgen和Vitae Pharmaceuticals公司担任首席执行官。1996年至2004年期间,他还在百时美施贵宝公司担任过一系列领导职务,包括免疫学和病毒学部门高级副总裁。 当上海的明星板开始向生物科技领域敞开大门时,它不仅被认为是纳斯达克的竞争对手,也被认为是香港证交所的竞争对手。随着中国扩大与这座城市的互通有无项目,这些看法可能会出现友好的转变。 这些变化意味着,中国内地的投资者将能够持有香港生物科技分部股票,而香港的投资者--一个国际化程度高得多的集团--将能够获得在星空上市的股票。实际上,它使上海市场在一夜之间变成了一个面向全球的交易所,同时也拓宽了港交所的一个关键收入来源。 对于曾经单调乏味的药品生产,尤其是疫苗生产来说,今年是辉煌的一年。数十亿美元的资金已经用于增加Covid-19针剂的生产设施,而个别的CDMO也扩大了它们的生产设施,这显然是预期到了需求,或是对政府主导的国内药品生产的推动做出了回应。 现在,百特生物制药解决方案公司,多臂医疗保健巨头百特的CDMO之翼,正在加入这一游戏。周二,他们宣布计划斥资5000万美元扩建其位于布鲁明顿的旗舰工厂,该工厂占地60万平方英尺。 诺华一直是该行业最大的研发支出者之一。就像他们每年年底经常做的那样,公司高管们都在强调他们预计2021年最有可能成为赢家的药物。 他们还梦想着一些潜在的大乐透彩票。 诺华公司首席执行长纳拉西姆汉将在周二举行的年度投资者陈述会上概述他认为这家制药公司的“不确定因素”。诺华公司希望这五种试验性药物能够在未来五年左右的时间里成为高风险,高回报的进入者,并涵盖广泛的适应症。 阿斯利康和牛津大学宣布在抗击流感的第三阶段取得新的里程碑,这是世界连续第三个星期一从积极的疫苗消息中醒来。不过,并不是每个人都相信他们会扮演重要角色。 平均有效率为70%,这一头条数字让分析师们觉得不如辉瑞/生物技术公司和Moderna在过去两周分别吹嘘的95%和94.5%的保护效果那么令人印象深刻。但是英国的合作伙伴说,他们还有其他几个亮点适合他们的候选人。在第三阶段测试的两种剂量方案中的一种显示出更好的轮廓,使功效达到90%;基于腺病毒载体的疫苗需要最低限度的冷藏,这可能意味着更容易分发;阿斯利康公司承诺以其他两家疫苗开发商的零头价格出售该疫苗。 一份新的文件称,欧盟正在研究绕过专利保护的方法,使仿制药在诸如Covid-19大流行等紧急情况下更容易生产。 通常情况下,根据世贸组织的规定,被称为“强制许可”的做法在特殊情况下是允许的,并可作为一种豁免适用,以绕过专利持有者。周三公布的文件是欧盟支持其成员国知识产权计划的一部分。