Cidofovir

GAITHERSBURG, Md., Sept. 26, 2024 (GLOBE NEWSWIRE) -- Emergent BioSolutions Inc. (NYSE: EBS) (“Emergent”) today announced that two contract options have been secured to procure additional treatment courses of TEMBEXA® (brincidofovir) to sustain the U.S. government’s national preparedness posture against human smallpox. TEMBEXA® is indicated for the treatment of human smallpox disease in adult and pediatric patients, including neonates. The order is valued at $67.4 million and executes procurement options CLIN0004A and CLIN0005A under Emergent’s existing 10-year contract. “This announcement highlights the strength of the longstanding partnership between Emergent and the U.S. government to meet the country’s preparedness needs,” said Paul Williams, senior vice president, products business, Emergent. We look forward to continuing to deliver TEMBEXA® to support smallpox preparedness.” The existing contract includes optional procurement CLINs that can be exercised throughout the 10-year contract period of performance with a maximum potential value of $568 million. The exercise of CLIN0004A and CLIN0005A commits funding through 2027 and will help ensure continued supply of TEMBEXA® to address the threat of smallpox. This project has been funded in whole or in part with federal funds from the Department of Health and Human Services; Administration for Strategic Preparedness and Response; Biomedical Advanced Research and Development Authority (BARDA) under contract number 75A50122C00047. About TEMBEXA®TEMBEXA® is an oral antiviral approved by the FDA in June 2021 for the treatment of human smallpox disease caused by variola virus in adult and pediatric patients, including neonates. TEMBEXA® is formulated as 100 mg tablets and 10 mg/mL oral suspension dosed once weekly for two weeks. The oral suspension formulation is particularly important for patients who have difficulty swallowing due to age or medical status. Important Information about TEMBEXA® (brincidofovir)TEMBEXA® is an orthopoxvirus nucleotide analog DNA polymerase inhibitor and is indicated for the treatment of human smallpox disease in adult and pediatric patients, including neonates. Limitations of Use: TEMBEXA® is not indicated for the treatment of diseases other than human smallpox disease. The effectiveness of TEMBEXA® for the treatment of smallpox disease has not been determined in humans because adequate and well-controlled field trials have not been feasible and inducing smallpox disease in humans to study the drug’s efficacy is not ethical. TEMBEXA® efficacy may be reduced in immunocompromised patients based on studies in immune deficient animals. TEMBEXA® has a BOXED WARNING for increased risk for mortality when used for longer duration. An increased incidence of mortality was seen in TEMBEXA®-treated subjects compared to placebo-treated subjects in a 24-week clinical trial when TEMBEXA® was evaluated in another disease. The Warnings and Precautions for TEMBEXA® include: Elevations in Hepatic Transaminases and Bilirubin: May cause increases in serum transaminases (ALT or AST) and serum bilirubin. Monitor liver laboratory parameters before and during treatment.Diarrhea and Other Gastrointestinal Adverse Events: Diarrhea and additional gastrointestinal adverse events including nausea, vomiting, and abdominal pain may occur. Monitor patients, provide supportive care, and if necessary, do not give the second and final dose of TEMBEXA®.Coadministration with Related Products: TEMBEXA® should not be coadministered with intravenous cidofovir.Embryo-fetal Toxicity: May cause fetal harm. Advise individuals of childbearing potential of the potential risk to the fetus and to use effective contraception.Carcinogenicity: TEMBEXA® should be considered a potential human carcinogen. Do not crush or divide TEMBEXA® tablets.Male Infertility: Based on testicular toxicity in animal studies, TEMBEXA® may irreversibly impair fertility in individuals of reproductive potential. Common adverse reactions (occurring in at least 2% of TEMBEXA®-treated subjects) were diarrhea, nausea, vomiting, and abdominal pain. Please read full Prescribing Information for TEMBEXA® for additional safety information here. About SmallpoxSmallpox is a highly contagious disease caused by the variola virus. Historically, smallpox was one of the deadliest diseases in history with a case fatality rate of approximately 30 percent. Despite successful eradication of smallpox in the 1970s, there is considerable concern that variola virus could reappear, either through accidental release or as a weapon of bioterrorism. According to the U.S. Centers for Disease Control and Prevention, variola virus is ranked in the highest risk category for bioterrorism agents (Category A) due to its ease of transmission, high mortality rate, and potential to cause public panic and social disruption. Based on a recent report – The Department of Health and Human Services Fiscal Year 2023 Public Health and Social Services Emergency Fund Justification of Estimates for Appropriations Committee – smallpox remains a threat of high concern to both the domestic and international community. About Emergent BioSolutionsAt Emergent, our mission is to protect and enhance life. For 25 years, we’ve been at work defending people from things we hope will never happen—so we are prepared just in case they ever do. We provide solutions for complex and urgent public health threats through a portfolio of vaccines and therapeutics that we develop and manufacture for governments and consumers. We also offer a range of integrated contract development and manufacturing services for pharmaceutical and biotechnology customers. To learn more about how we help protect public health, visit our website and follow us on LinkedIn, X, Instagram, Apple Podcasts and Spotify. Safe Harbor StatementThis press release includes forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements, other than statements of historical fact, including statements regarding the exercise by BARDA of any optional future procurements under the contract (75A50122C00047) to supply TEMBEXA® to the U.S. government, are forward-looking statements. We generally identify forward-looking statements by using words like “anticipate,” “believe,” “continue,” “could,” “estimate,” “expect,” “forecast,” “goal,” “intend,” “may,” “plan,” “should,” “will,” “would,” and similar expressions or variations thereof, or the negative thereof, but these terms are not the exclusive means of identifying such statements. Forward-looking statements are based on Emergent’s current intentions, beliefs, and expectations regarding future events. Emergent cannot guarantee that any forward-looking statement will be accurate. Readers should realize that if underlying assumptions prove inaccurate or unknown risks or uncertainties materialize, actual results could differ materially from Emergent’s expectations. Readers are, therefore, cautioned not to place undue reliance on any forward-looking statement. Any forward-looking statement speaks only as of the date of this press release, and, except as required by law, Emergent does not undertake to update any forward-looking statement to reflect new information, events or circumstances. There are a number of important factors that could cause Emergent’s actual results to differ materially from those indicated by any forward-looking statements. Readers should consider this cautionary statement, as well as the risk factors identified in Emergent’s periodic reports filed with the U.S. Securities and Exchange Commission when evaluating Emergent’s forward-looking statements. Investor Contact:Richard S. LindahlExecutive Vice President, CFOlindahlr@ebsi.com Media Contact:Assal HellmerVice President, Communicationsmediarelations@ebsi.com

©氨基观察-创新药组原创出品 作者 | 武月 盈利问题，如同达摩克利斯之剑般悬挂在每一家biotech的头顶。 放眼国内市场，所有的公司都在为此努力，开源节流、降本增效，甚至有的公司开展全员BD、砍管线、转型CDMO。 残酷的是，大多数biotech，还在寻找并努力靠近自己的盈亏平衡点。 那么，究竟卖多少药，biotech才能不亏钱？我们不妨把视线放到海外，观察那些成功的过来者，走过的路。 关于biotech的盈亏之问，看起来没有标准答案。 有的企业，在其营收接近25亿美元才实现扭亏为赢；大部分，重磅品种上市后5年以上，销售额达到10亿美元以上才得以扭亏；而也有的企业，在营收仅2.6亿美金时已经顺利“上岸”。 / 01 / 5年、10亿美元 放眼全球，biotech都是一个“新陈代谢”极高的群体。 据广发证券统计数据显示，1990年以来，超过600家biotech企业在纳斯达克IPO。但截止2020年6月30日，仅有12家biotech有相对稳定的收入和正向现金流。 将时间线拉长，从上世纪80年代biotech概念兴起至今，40年时间里，靠自己买迈入pharma阵营的，更是只有寥寥无几。 为什么biotech很难长久地活下去？这是由商业模式决定的。相比成长性一般但胜在确定性强的大药企，biotech则是发散性创新模式，成长过程充满了不确定性。 换句话说，由于研发的高风险和销售的不确定性，大多数biotech压根没法跑通商业模式，盈利更是无从谈起。 那么，那些全球顶尖biotech，是如何上岸的？又为此付出了多大的代价？ 无论再生元、吉利德还是福泰制药、渤健，大都是在重磅品种上市后5年以上，销售额达到10亿美元以上才得以扭亏。 以再生元为例，2008年推出首款药物Rilonacept后，由于适应症限制，销售额一直不理想，亏损也在不断扩大。 直到2011年，再生元推出眼科神药Eylea，销售额迅速起飞，上市第一年销售额达8.4亿美元，第二年便成为重磅炸弹药物，带动公司整体营收达13.78亿美元，并顺利扭亏。 吉利德的商业化故事起步于抗病毒药物Cidofovir，1996年上市后，销售额一直维持在1-2亿美元左右，直到2001年HIV重磅药物Tenofovir上市，销售额在3年内迅速突破10亿美元大关。在重磅炸弹的带领下，吉利德于2007年成功扭亏，此后盈利水平一路飙升。 对比之下，福泰制药、渤健的盈利则花了更长时间。后者治疗多发性硬化症的重组干扰素1996年就上市，近十年后销售额18亿才得以扭亏； 福泰制药命途更是多舛，从1999年的免疫抑制剂再到HIV，再到2011年的丙肝抗病毒疗法，福泰制药经过了20年的试错，其中替拉瑞韦首年销售额超过5亿美元，远超过市场预期。福泰制药也因为这款药物，首次实现了盈利。 本以为公司命运能够就此逆转，但两年后，吉利德推出了一款丙肝神药 Sovaldi，能够彻底治愈丙肝，公司的抗病毒条线遭到了无情的毁灭性打击。最后在囊性纤维化这片应许之地，2012年推出Kalydeco、2015年推出Orkambi，才稳住局面。 直到2017年，福泰制药才彻底走向盈利大道，当年其营收接近25亿美元。 / 02 / 更早的盈利 从这些顶级biotech的盈利之路，不难看出，推出重磅炸弹后，平均要在10亿美元以上的收入，才能跨过盈亏平衡点。 当然，翻看海外其他叫得上名号的biotech历年财报，你会发现，这里还有其他情况。 比如，专注罕见病领域，并被阿斯利康以390亿美元收购的Alexion制药，营收达到仅仅2.6亿美金时已经扭亏为盈。 这与Alexion制药的研发策略密切相关。公司的核心产品是全球首个上市的C5补体抑制剂，eculizumab。或许是因为该靶点开发难度巨大，该领域的竞争显得并不那么激烈。 2007年3月，FDA批准eculizumab，用于治疗阵发性睡眠性血红蛋白尿症（PNH）。随后又获批典型溶血性尿毒综合征(aHUS)、乙酰胆碱抗体阳性重症肌无力等适应症，并获得了多项孤儿药资格认证。 Eculizumab全球销售额一路飙升，2012年以11.24亿美元的销售额步入重磅炸弹梯队，2017年达31.44亿美元。 从作用机制上来看，C5补体抑制剂理论上在类风湿性关节炎、银屑病、心脏病炎症并发症等风湿免疫性疾病领域均有较大潜力。公司早期也在这些领域做了大量研究。 然而，事与愿违，在经历一系列的临床试验之后，eculizumab对类风湿性关节炎和膜性肾病的治疗效果均未达预期。相反，其在PNH领域展现出不错的效果。Alexion也借此转变阵地至罕见病领域。 令人意想不到的是，这个金矿非常耐挖。正如Alexion创始人所说：与那些患者群达几万、几十万的非罕用药面临的价格压力相比，Alexion开发的药品多年来几乎不受影响。 尽管eculizumab每年的用药费用高达44万美元，但由于其疗效确切及不可替代性，保险公司和国家性医保机构都愿意为其买单。守着这样一座“金山”，Alexion的研发投入很明显低了一个档次。 被收购前，Alexion 2017年的研发费用率最高，不过也仅有25%。当年，其营收超35亿美元，只有6个产品，其中3个已上市，临床I期、II期、III期的产品各一个。 看惯大药企动辄数十甚至上百的产品管线，Alexion的产品线显得单薄得多。而随着营收的增长，Alexion的研发费用率更是下降至16%左右。远低于同期，再生元等动辄30%的研发费用率。 当然，研发费用率低，也与Alexion更青睐并购有关。2017年上市的3款产品中，2款来自于并购。 这与吉利德有些类似。吉利德2007年首次盈利，净利率便超过30%，随后多年一直保持着这样的盈利水平，似乎并不存在盈利爬坡的困扰。 2001年，吉利德首个抗艾滋病药物替诺福韦酯获得FDA批准。而就在一年之后，吉利德耗资4.64亿美元收购Triangle制药，获得emtricitabine。 以后来者眼光看，正是对于emtricitabine的收购，让吉利德在HIV领域迅速成为龙头企业。丙肝神药 Sovaldi也是通过收购Pharmasset公司获得的。2012年，吉利德耗资110亿美元收购Pharmasset，在2014年上市后的前3个月，Sovaldi就创造了23亿美元的惊人数据，创下当时美国新药上市的销售纪录。 一个共识是，即使销售额再高，药企也必须保持着研发烧钱的属性。典型如再生元，Eylea挣来的钱几乎全被扔回了研发，金额比同期biotech高出一个数量级，因此，其在商业化的前十年盈利水平都并不高。 而Alexion制药、吉利德等依托并购路径成长的企业，要么能够实现更早的盈利，要么能够更早实现高质量的盈利。 / 03 / “反面”教材 当然，在头部biotech阵营，也并非没有“反面”教材。 所谓“反面”是指，即使公司产品的销售额已经不低，但由于研发投入体量巨大，使得公司盈利还遥遥无期。 典型如被辉瑞428亿美元收购的ADC元老Seagen。 早在1997年，ADC技术尚未成熟时，Seagen就成立并瞄准ADC领域。经过20多年的探索，如今Seagen建立了一条优质的ADC产品管线。 目前，Seagen共有四款产品上市，分别为Adcetris、Padcev、Tivdak、Polivy。2022年，这四款产品为Seagen带来了17亿美元的收入，占总营收的87%。 同一年，Seagen的研发费用达到13.4亿美元，研发费用率接近70%。加之销售费用，公司全年亏损超过6亿美元。 事实上，Seagen的商业化能力并不算差，但公司持续不断砸钱在平台和后续管线搭建，导致亏损不断放大。2018年，其研发费用率达到86%，尽管随着收入的增长，费用率有所下降，但依然接近70%，亏损额也由2.6亿美元扩大至6.1亿美元。 这也不难理解。面对DS-8201带来的巨大压力，Seagen必须要用更多投入换回领跑机会。就在去年9月，害怕被颠覆的Seagen，还与PROTAC领域的领军企业Nurix达成了总价高达34.6亿美元的合作，希望研发出具有新作用机制的降解抗体偶物（DAC）。 投入与冒险还在继续。 再比如RNAi赛道领头羊Alnylam，自2018年推出全球第一款获批上市的RNAi药物，验证了RNAi技术路线；如今，手握5款上市的RNAi药物，销售额持续攀升；股价也一路高歌猛进，市值超过340亿美元，成功完成阶级跃迁。 去年，靠着销售额及授权收入，Alnylam的营收更是首次超过百亿人民币，今年上半年继续保持超80%的高增长，营收超过80亿元，然而，公司仍在持续亏损。 为了维持领先地位，Alnylam不得不持续投入研发，验证自家平台的价值，这也使得公司的研发投入居高不下，2023年至2021年，研发费用率分别为55%、85%和94%。 同时，由于多款药物同时销售，公司的销售和管理费用率也一直维持在较高水平，过去三年分别为44%、74%和74%。这些因素，共同导致了公司迟迟无法盈利。 当然，好消息是，随着Alnylam营收的快速增长，各项费用正在迅速被摊薄。也就是说，Alnylam仍走在通往盈利的道路上，只不过，用时长了些。 / 04 / 总结 研发与商业化，对于创新药企同样重要。而过高的费用水平，会构成企业从销售盈利到整体盈利之间的鸿沟。如何在保持研发竞争实力的同时，提高销售额、摊薄费用率，是一门学问。 对于国内正在奋力前行的biotech来说，学习这些成功案例不仅能够更好地理解行业规律，还能够为其在未来的道路上提供宝贵的经验借鉴。 毕竟，在这个充满挑战的领域中，每一个成功的例子都是通往成功的宝贵财富。而那些成功者的盈利轨迹，或许也能让市场，更客观、理性地看待biotech的盈利问题。 药渡媒体商务合作 媒体公关 | 新闻&会议发稿 张经理：18600036371（微信同号） 点击下方“药渡“，关注更多精彩内容 免责声明 “药渡”公众号所转载该篇文章来源于其他公众号平台，主要目的在于分享行业相关知识，传递当前最新资讯。图片、文章版权均属于原作者所有，如有侵权，请及时告知，我们会在24小时内删除相关信息。 微信公众号的推送规则又双叒叕改啦，如果您不点个“在看”或者没设为"星标"，我们可能就消散在茫茫文海之中~点这里，千万不要错过药渡的最新消息哦！👇👇