XCUR FXN

近期（9月27日），一家利用球形核酸（SNA）药物治疗神经障碍和脱发的Biotech公司Exicure宣布一些重大事项，在公司控制权方面，在满足一些成交条件的情况下，现有投资者CBI USA将通过购买公司普通股的形式获得实际控制权。此外，行政和研发方面，该公司将裁员约66%，同时实施其他成本削减措施。不仅如此，这家公司还暂停了所有临床前研究，暂停了所有合作事项，停止了所有R&D研发事项。可即使如此，公司的现金流也只能维持到2023年的第二季度。从股市来看，该公司股价也已经从曾经最高位的170多美元下跌至如今的1.41美元，甚至要通过反向分割股权维持股价不跌至1美元大关才能避免摘牌，是什么让Exicure变成现如今深陷寒潭的境地呢？临床前数据误报导致管线被砍从该公司的研发管线来看，主要是集中在一些神经障碍疾病和脱发上，但是问题也出现在这里，如果临床前数据并不正确，向后推进的临床进度那也很可能是白用功。根据该公司前神经科学小组负责人Grant Corbett，Ph.D指控，治疗弗里德里希共济失调的XCUR-FXN临床前项目中具有一些数据造假不当行为。随后，该公司进行了内部调查，他们对此进行回击认为是Corbett单方面误报，其中误报内容有：①从2020年秋季开始，Corbett博士误报了与XCUR-FXN相关的某些研发实验的原始数据；②Corbett博士误报了至少三个不同实验的结果，这些实验至少进行到2021年2月；③错误报告的数据仅涉及XCUR-FXN的疗效，而非安全性；④从2021年1月7日到2021年8月12日，错误报告的数据被包括在各种公开陈述和SEC文件中；Corbett博士独自误报了数据，没有得到公司其他任何人的帮助或知情，包括公司管理层和其他研发员工，并且直到2021年11月辞职前没有向公司任何人通知他的行为；⑤公司管理层在发表包含Corbett博士误报数据的公开声明时，合理地依赖了Corbett博士的分析；⑥Corbett博士对XCUR-FXN数据的误报没有影响到其他公司的项目。但无论到底是哪一方在误报数据，起码临床前数据不可信是实锤了的。这一数据误报直接导致公司此前裁员50%，XCUR-FXN和cavrotolimod (AST-008)两款管线被砍，CEO David Giljohann博士辞职，而新接任的CEO Brian C. Bock上任未过多久也辞去总裁兼首席执行官的职务。对于这一困境，Exicure选择孤注一掷，调整其研发资源，以第一优先级支持针对SCN9A治疗神经性疼痛的临床前计划的开发，并同步展开与艾伯维和Ipsen关于脱发和罕见病的临床开发。临床前又迎来失利然而，SCN9A治疗神经性疼痛的临床前研究计划也遭遇了重挫，根据该公司的陈述，在非人灵长类模型中，SCN9A作为非阿片药物的替代品治疗神经性疼痛未能达到之前在体外临床研究中的效果。考虑到这一点，Exicure停止了SCN9A项目的进一步临床前研究，并打算将其剥离。那从现在来看，该公司不仅仅终止了所有的合作，也打算将SCN9A剥离，现在的这家公司的现状实际上相当于除了未披露的什么管线都没有进行。小编总结在资本寒冬下，小企业的生存变得更为艰难，Exicure正面临生死存亡的关键。与一些公司抵达临床阶段后才出现不利情况不同，Exicure在临床前就遭遇了重大危机，如果不重新确定正确的战略方向，那么Exicure很可能成为一家Biotech消亡的案例之一。参考来源：https://exicuretx.com/扫描下方二维码助力佰傲谷洞见生物医药，把握行业脉搏（20份神秘好礼先到先得）点亮在看，传递信息♥

Exicure is going through its second round of layoffs in less than a year. One could be forgiven if they expected Exicure to have stopped the bleeding after a tumultuous first quarter of 2022. It was then that two top executives, including the CEO, resigned and three board members departed. As it turns out, the preclinical crack that preceded the initial exodus has only continued to wreak havoc on the company’s financial future. After shaving half of its staff in January, the company is at it again, cutting 66% of its remaining workforce, according to a release Tuesday. All R&D work, including partnered programs, has been shelved, and the company is looking to offload its preclinical programs, irrespective of how weak they appear.  To keep its head above water, Exicure is selling $5.4 million in shares to its existing investor, CBI USA, giving the latter more than 50% of voting power in the company. In the release, Exicure CEO Matthias Schroff said the decision to let people go was in “no way a reflection on them.” Exicure says its executive team is “expected” to stay on following the downsizing and restructuring.  Likely contributing to the decision to press pause on all R&D work were new data disclosed by the company showing that its top preclinical asset, SCN9A, flunked in vivo studies in nonhuman primates. Exicure says the med “did not meet desired target engagement levels as observed in previous in vitro preclinical studies” and, as a result, would require additional preclinical work that it can’t take on.  It's more of the same for Exicure, which for almost a full year now has tried to stave off the collapse of the company and its pipeline. But data trump all in biotech, as the company has brutally learned.  On the Nasdaq, Exicure was living off of the public market sugar high that guided much of the biotech space in 2020 and early 2021. In fact, the company was able to extend the ride longer than many, until November 2021 when Exicure disclosed that a former senior researcher confessed to “alleged improprieties” regarding its XCUR-FXN preclinical program for patients with Friedreich’s ataxia. The share price plummeted on the news, falling nearly $9 the day after the disclosure, from some $32 per share to around $23.  After more details came to light, Exicure ended development of the program and announced it would cut half of its staff by January 2022. The company explained that it would redirect its focus toward the SCN9A assets and other partnered-programs.  Exicure had two collaborations in the works that will now be shelved: one with Ipsen to develop treatments for Huntington’s disease and Angelman syndrome, and another with AbbVie to find treatments for hair loss. Both were based on Exicure’s nucleic acid tech, dubbed “spherical nucleic acid,” which it has touted as being able to more efficiently enter a swath of cell types than competitor nucleic acid-based treatments.  As of the end of June, Exicure had just over $23 million left in cash and short-term investments, estimating that was enough to last at least six more months. The company was bleeding money, however, having spent more than half of the reserves it had available entering 2022.