Chagas Cardiomyopathy

Phase 2/3 trial (EBO-301) in treatment-refractory Mycobacterium avium Complex (TR-MAC) continues with Phase 2 topline results expected in summer 2024 and Phase 3 enrollment pause in place during continued analysis of incoming data; plan to discuss with FDA Cash, cash equivalents, and investments of $134.5 million at December 31, 2023 MENLO PARK, Calif.--(BUSINESS WIRE)-- AN2 Therapeutics, Inc., (Nasdaq: ANTX), a clinical-stage biopharmaceutical company focused on developing treatments for rare, chronic, and serious infectious diseases with high unmet needs, today reported financial results for the fourth quarter and year ended December 31, 2023. “AN2’s cash position remains strong as we advance our innovative boron-based pipeline. Following the recent meeting of the independent Data Safety Monitoring Board, we continue to believe in the potential of epetraborole to become an important component of the backbone therapy for NTM lung disease,” said Eric Easom, Co-Founder, President and Chief Executive Officer. “Patients enrolled in the epetraborole Phase 2/3 trial in treatment refractory lung disease caused by MAC continue to be dosed while new enrollment in the Phase 3 part of the trial remains paused. Patients in the Phase 2 trial have reached the six-month mark, and we plan to report topline results this summer. Analysis of the baseline patient demographics from the trial show a highly complex and refractory patient population, which we plan to discuss with the FDA in the coming months.” Easom continued, “We are pleased with the momentum across our boron chemistry pipeline. We signed a licensing agreement last fall with the University of Georgia Research Foundation to develop a novel therapy for chronic Chagas disease, which is in late preclinical development and we are also progressing an IV formulation of epetraborole for melioidosis, in addition to advancing other research programs. We believe epetraborole, along with these other promising boron therapy candidates, has the opportunity to help address significant unmet patient need.” Fourth Quarter & Recent Business Updates: Epetraborole Pivotal Phase 2/3 Clinical Study in TR-MAC Lung Disease This double-blind, placebo-controlled trial is comparing epetraborole plus a background regimen versus placebo plus background regimen in patients with TR-MAC lung disease. In February 2024, the Company announced that it had voluntarily paused Phase 3 enrollment in the seamless Phase 2/3 clinical trial, pending further data review. The voluntary pause was instituted following an analysis of blinded aggregate data from the ongoing Phase 2 study, which showed potentially lower than expected efficacy. The aggregate baseline characteristics reveal patients with complex comorbidities, prolonged NTM lung disease, and high levels of cavitary disease, as well as patients who are refractory to Arikayce, the only FDA-approved drug for refractory NTM caused by MAC, as part of their background regimen. The decision to pause Phase 3 enrollment was not due to safety concerns. While the Phase 3 part of the trial is paused for new enrollment (n=97), the Company is continuing to dose existing patients enrolled in the Phase 2/3 trial under the existing protocol. The Company plans to provide further details on the epetraborole development strategy after discussion with FDA and continued review of the accumulated blinded data. The Company expects to announce topline data from the Phase 2 part of the trial in summer 2024. Presented New Epetraborole Data at Infectious Disease Society of America (IDSA) IDWeek 2023 Conference. In October 2023, three poster presentations and two oral presentations were presented at the IDWeek 2023 conference that detailed the potential of epetraborole in treating NTM (nontuberculous mycobacteria) lung disease caused by MAC and Mycobacterium abscessus. The Company also presented data on its melioidosis program. Executed License from University of Georgia Research Foundation to Develop Boron-Containing Product Candidates for Chagas Disease. In October 2023, AN2 announced that it had executed an exclusive license from the University of Georgia Research Foundation to advance the development of a boron-containing small molecule, AN2-502998 (formerly AN15368), for Chagas disease. This boron-based molecule was originally discovered by researchers at Anacor Pharmaceuticals, Inc. (a wholly owned subsidiary of Pfizer), which included some of the current AN2 team, and the University of Georgia, with grant funding from the Wellcome Trust. AN2-502998 has advanced into late preclinical development for chronic Chagas disease. Received Grant from Bill & Melinda Gates Foundation. In September 2023, AN2 announced that it received a grant from the Bill & Melinda Gates Foundation to discover novel, boron containing small molecules for the treatment of tuberculosis and malaria. Selected Fourth Quarter and Full Year 2023 Financial Results Research and Development (R&D) Expenses: R&D expenses for the full year 2023 were $54.9 million, compared to $29.5 million in the prior year. R&D expenses for the fourth quarter of 2023 were $14.9 million compared to $8.8 million for the same period during 2022 due to increased clinical trial expenses, personnel-related expenses, consulting and outside services, and other expenses, partially offset by lower chemistry manufacturing and controls expenses. General and Administrative (G&A) Expenses: G&A expenses for the full year 2023 were $14.8 million, compared to $12.8 million in the prior year. G&A expenses for the fourth quarter of 2023 were $3.9 million, compared to $3.7 million for the same period during 2022 due to increased personnel-related expenses and professional service fees, partially offset by a decrease in insurance expenses. Other Income, Net: Other income, net for the full year 2023 was $4.9 million, compared to $1.3 million in the prior year. Other income, net for the fourth quarter of 2023 was $1.9 million, compared to $0.6 million for the same period in 2022 due to increased interest and investment income based on higher interest rates and higher cash, cash equivalents and investment balances. Net loss: Net loss for the full year 2023 was $64.7 million, compared to $41.0 million in the prior year. Net loss for the fourth quarter of 2023 was $16.9 million, compared to $11.8 million for the same period during 2022. Cash Position: The Company had cash, cash equivalents, and investments of $134.5 million at December 31, 2023. About AN2 Therapeutics, Inc. AN2 Therapeutics, Inc. is a clinical-stage biopharmaceutical company developing treatments for rare, chronic, and serious infectious diseases with high unmet needs. Our initial candidate is epetraborole, which we are studying as a once-daily, oral treatment with a novel mechanism of action for patients with nontuberculous mycobacteria (NTM) lung disease, a rare, chronic, and progressive infectious disease caused by bacteria known as mycobacteria, that leads to irreversible lung damage and can be fatal. For more information, please visit our website at . Forward-Looking Statements This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements expressed or implied in this press release include, but are not limited to, statements regarding: continued analysis of incoming data; continuation of the Phase 2 and 3 parts of the EBO-301 trial; continuation of the enrollment pause in Phase 3 of the EBO-301 trial; FDA meetings and discussions; reporting of topline data from Phase 2 of the EBO-301 trial and other updates; potential of epetraborole, AN-502998, and the Company’s boron chemistry pipeline; timing and initiation of preclinical and clinical studies; the advancement of candidates for Chagas disease and melioidosis; and other statements that are not historical fact. These statements are based on AN2’s current estimates, expectations, plans, objectives, and intentions, are not guarantees of future performance and inherently involve significant risks and uncertainties. Actual results and the timing of events could differ materially from those anticipated in such forward-looking statements as a result of these risks and uncertainties, which include, but are not limited to, risks and uncertainties related to: the recommendations of the Data Safety Monitoring Board following future reviews of unblinded data; the number of patients who remain enrolled in the Phase 2/3 trial; the ability of AN2 to resume enrollment in the Phase 2/3 trial, in the event the determination to resume enrollment is made; meetings with FDA; the ability of AN2 to effectively and timely make amendments to the Phase 2/3 pivotal trial design based on its analysis of the Phase 2 portion of the study and/or pursuant to additional FDA feedback; possible changes to AN2’s plans or priorities as it assesses study data; potential for protocol modifications, redesign, or study termination; timely enrollment of patients in AN2’s existing and future clinical trials; AN2’s ability to procure sufficient supply of its product candidate for its existing and future clinical trials; the potential for results from clinical trials to differ from preclinical, early clinical, preliminary, or expected results; significant adverse events, toxicities, or other undesirable side effects associated with AN2’s product candidate; the significant uncertainty associated with AN2’s product candidate ever receiving any regulatory approvals; AN2’s ability to obtain, maintain, or protect intellectual property rights related to its current and future product candidates; implementation of AN2’s strategic plans for its business and current and future product candidates; the sufficiency of AN2’s capital resources and need for additional capital to achieve its goals; global macroeconomic conditions and global conflicts; and other risks, including those described under the heading “Risk Factors” in AN2’s Annual Reports on Form 10-K and Quarterly Reports on Form 10-Q, and AN2’s other reports filed with the U.S. Securities and Exchange Commission (SEC). These filings, when made, are available on the investor relations section of AN2’s website at and on the SEC’s website at . Forward-looking statements contained in this press release are made as of this date, and AN2 undertakes no duty to update such information except as required under applicable law. AN2 THERAPEUTICS, INC. CONDENSED STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS (in thousands, except share and per share data) (unaudited) Three Months Ended December 31, Year Ended December 31, 2023 2022 2023 2022 Operating expenses: Research and development $ 14,919 $ 8,752 $ 54,871 $ 28,511 Research and development—related party — — — 1,000 General and administrative 3,896 3,724 14,764 12,751 Total operating expenses 18,815 12,476 69,635 42,262 Loss from operations (18,815 ) (12,476 ) (69,635 ) (42,262 ) Other income, net 1,917 634 4,903 1,306 Net loss (16,898 ) (11,842 ) (64,732 ) (40,956 ) Accretion to redemption value and cumulative dividends on preferred stock — — — (1,820 ) Net loss attributable to common stockholders $ (16,898 ) $ (11,842 ) $ (64,732 ) $ (42,776 ) Net loss per share attributable to common stockholders, basic and diluted $ (0.57 ) $ (0.61 ) $ (2.74 ) $ (2.79 ) Weighted-average number of shares used in computing net loss per share, basic and diluted 29,735,397 19,352,854 23,600,107 15,340,134 Other comprehensive loss: Unrealized gain (loss) on investments 397 167 649 (347 ) Comprehensive loss $ (16,501 ) $ (11,675 ) $ (64,083 ) $ (41,303 ) AN2 THERAPEUTICS, INC. CONDENSED BALANCE SHEETS (in thousands) December 31, 2023 2022 Assets Cash and cash equivalents $ 15,647 $ 27,219 Short-term investments 91,648 68,840 Prepaid expenses and other current assets 3,212 2,562 Long-term investments 27,194 3,219 Other assets, long-term 1,043 720 Total assets $ 138,744 $ 102,560 Liabilities and stockholders’ equity Accounts payable $ 2,676 $ 2,122 Other current liabilities 11,367 5,064 Other liabilities, long-term — 2 Total liabilities 14,043 7,188 Stockholders’ equity 124,701 95,372 Total liabilities and stockholders’ equity $ 138,744 $ 102,560

药物研发进展 1.兴齐眼药硫酸阿托品滴眼液申报上市 4月25日，中国国家药监局药品审评中心（CDE）官网最新公示，兴齐眼药递交了硫酸阿托品滴眼液的新药上市申请并获得受理。根据兴齐眼药公告，该产品是以硫酸阿托品为活性成分的眼用制剂（SQ-729），此次申报适应症为：用于延缓儿童近视进展。近视是由于眼球直径过长，导致图像无法聚焦在视网膜上的一种疾病。由于儿童在阅读、学习和使用数字设备上的时间增多，在户外的时间变少，近视的发病率也在急剧增加，并逐渐成为一个日益增长的公共卫生挑战。在没有治疗的情况下，高度或快速进展的近视可导致严重的眼部健康问题，如青光眼、白内障和视网膜脱离，并可引起不可逆的视力丧失。目前近视的治疗选择是佩戴框架或隐性眼镜，这并不能减缓其进展。在中国国家卫健委颁布的《儿童青少年近视防控适宜技术指南》中，低浓度阿托品是唯一推荐的抗胆碱类药物。这种产品可阻断中枢神经系统以及周围神经系统中神经递质的作用，通过禁用聚焦机制来控制近视加深。不过，阿托品需要稀释后才能使用。兴齐眼药于2016年6月与新加坡国立眼科中心（SNEC）签署合作协议，在获得SNEC中国独家授权的硫酸阿托品滴眼液处方及5年临床试验数据的基础上，该公司开展了该产品的3期临床试验。研究结果显示，试验组对比安慰剂组在主要疗效指标上有统计学意义的显著性差异，试验组优于安慰剂组；试验中这款硫酸阿托品滴眼液安全性良好，患者使用依从性好。 2.甘李药业甘精胰岛素在玻利维亚获批 4月24日，甘李药业宣布已于近日取得了玻利维亚国家药品和卫生技术局（AGEMED）下发的甘精胰岛素注射液（预充式）的注册批件，也意味着该公司获得向玻利维亚销售甘精胰岛素注射液的商业化许可。公开资料显示，甘李药业研发的甘精胰岛素（商品名为长秀霖）是一种长效胰岛素类似物，用于糖尿病患者基础血糖控制，每日只需注射1次。糖尿病是一种影响身体将食物转化为能量的慢性疾病。患有糖尿病的人，身体不能产生足够的胰岛素，或者不能正常使用胰岛素。当体内没有足够的胰岛素或细胞对胰岛素停止反应时，过多的葡萄糖就会留在血液中。久而久之，这会导致严重的健康问题，如心脏病、视力下降和肾脏疾病。甘李药业研发的甘精胰岛素是一种长效胰岛素类似物，也称基础胰岛素。该产品经过修饰后可在较长时间里提供稳定水平的血浆胰岛素，每天需注射1次，注射起效后作用时间可持续24h，能较好地模拟生理性基础胰岛素的分泌特性。由于该产品在体内吸收缓慢，作用时间较长，患者的血浆胰岛素水平不会出现明显峰值，可平稳降低患者血糖，有较好的疗效、安全性和患者依从性。甘精胰岛素注射液已经成为糖尿病治疗的重要的基础胰岛素类似物。 3.阿斯利康反义寡核苷酸疗法达到所有3期临床终点 4月25日，阿斯利康（AstraZeneca）和Ionis Pharmaceuticals公司联合宣布，在研反义寡核苷酸（ASO）疗法eplontersen在治疗转甲状腺素蛋白（TTR）介导的淀粉样变性多发性神经病（ATTRv-PN）患者的3期临床研究中，与外部安慰剂组相比，在66周时达到了所有共同主要终点和次要终点。这款疗法目前正在接受美国FDA的审评。ATTRv-PN是一种由于TTR蛋白不正常形成，并累积在全身各种器官组织的严重、渐进性并危及生命的疾病。TTR蛋白会累积于周围神经、心脏与肠道，进而造成难以治疗的周围感觉运动神经病变、自主神经病变以及/或心肌病变等症状。此疾病会造成许多的并发症，并逐渐地影响患者的生活品质与日常活动。此病进展迅速，通常会造成患者早夭，患者在确诊后的中位存活期仅有4.7年。Eplontersen为一款一月一次，可由患者自己皮下注射的在研反义寡核苷酸药物，其采用的反义寡核苷酸配体偶联（LICA）技术，可以将ASO药物与能和细胞表面特定受体结合的配体分子偶联在一起。LICA技术能有效地将ASO选择性递送到表达这些受体的细胞中。Eplontersen可用以抑制TTR蛋白的生产，从而治疗遗传性与非遗传性的淀粉样变性多发性神经病。此药已获得美国FDA授予孤儿药资格。 4.Bluebird提交镰状细胞贫血基因疗法BLA申请 4月24日，bluebird bio宣布已向FDA提交了治疗镰状细胞贫血（SCD）患者的lovo-cel基因疗法的生物制品许可申请（BLA）。此次BLA包括优先审查请求，如果获得批准，FDA对申请的审查将从提交之日起缩短至六个月，而标准审查时间为10个月。Lovo-cel是一种在研、用于治疗SCD的一次性基因疗法，其设计目的是将改良形式的β-珠蛋白基因（βA-T87Q珠蛋白基因）的功能拷贝添加到患者自身的造血干细胞（HSCs）中。一旦患者具有βA-T87Q珠蛋白基因，他们的红细胞可以产生抗镰状血红蛋白（HbAT87Q），从而降低HbS的比例，目标是减少镰状红细胞、溶血和其他并发症。BLA的递交是基于迄今最大型的SCD基因疗法临床项目的结果。基于HGB-206C组队列中36名患者的疗效结果，中位随访时间为32个月，HGB-210研究中有两名患者的随访时间为18个月。BLA提交的文件还包括在整个lovo-cel项目中接受治疗的50名患者的安全数据，其中包括6名接受了6年或更长时间随访的患者。bluebird bio在2022ASH大会上公布的数据显示：在29名可评估的患者中，截至最后一次随访，在2年的母研究中，28名患者在输注lovo-cel后严重血管闭塞发作（VOEs）完全解决，而在入组前2年，每年3.5例(1.0-13.5例)。FDA此前授予了lovo-cel孤儿药资格、快速通道资格、再生医学高级疗法（RMAT）资格和用于治疗SCD的罕见儿科疾病资格。 5.伟德杰生物IL-6R抗体2a期试验获积极结果 4月24日，伟德杰生物发布新闻稿称，其重组人源化白介素-6受体单克隆抗体注射液VDJ-001治疗特发性多中心型Castleman病（iMCD）的2a期前瞻性研究结果，已由北京协和医院血液科张路副教授于近期在学术会议上进行了口头报告。初步结果分析显示，VDJ-001在iMCD患者中有效率高、起效快，有望成为该病重要的治疗选择。基于此，伟德杰生物计划今年四季度启动该产品治疗iMCD的关键性2b期临床研究。伟德杰生物成立于2011年，致力于研发治疗自身免疫病、罕见病、肿瘤的创新型抗体/蛋白药。VDJ-001正是由该公司基于人B细胞的抗体进化与筛选平台研发的一款重组人源化单克隆抗体注射液，具有生物学活性高、安全性好、剂量低、表达量高、生产成本低等特点。VDJ-001能与可溶性和细胞膜上IL-6受体（sIL-6R和 mIL-6R）特异性结合，从而阻断IL-6经典、反式及反式呈递三种通路。临床前研究显示，VDJ-001在亲和力、体外生物学活性等主要药效学特性方面具有一定优势；在动物模型中，该产品表现出较好的抑制淋巴组织增生的效力；在毒理实验中，它具有较高的最大耐受剂量（MTD）及未见明显毒性反应剂量(NOAEL)，安全性良好。 6.γδ T细胞疗法最新数据公布 4月24日，IN8bio在2023年欧洲血液与骨髓移植学会（EBMT）年会上公布了同种异体 γ-δ T 细胞疗法INB-100的1期积极数据。INB-100是一种同种异体γδT 细胞疗法，其I期是对接受造血干细胞移植（HSCT）的急性白血病患者注输扩增/活化的γδT 细胞，主要终点为安全性和耐受性，次要终点GvHD的比率、复发率和总生存率。γδT细胞是T细胞的一种，约占T细胞总数的5%。其TCR由γ链和δ链组成，又根据δ链的不同表达可分为三种亚群，γδ T细胞是一种极具有天然免疫学特性，又具有适应性免疫应答的淋巴细胞。γδ T细胞能够通过分泌穿孔素和颗粒酶、表达CD16产生ADCC作用，分泌细胞因子IFN-γ与TNF-δ、表达Fas-L与TRAIL产生细胞凋亡，和充当抗原递呈细胞进行间接肿瘤杀伤。基于γδ T细胞独特的细胞杀伤机制，γδ T细胞在近年来得到大力开发，诸如CAR γδ-T细胞。传统CAR-T所用的T细胞大多是αβ T细胞，因为其在T细胞群体中分布最广泛，数量也最多。而T细胞的另一种亚群γδ T细胞被发现具有开发成CAR-T细胞治疗的潜力，与目前CAR-T所采用的αβ T细胞不同，γδ T细胞能识别其目标抗原而不受MHC（人类白细胞表面抗原）与限制，并介导抗肿瘤反应，而不会引起GvHD。目前不少的候选CAR γδ T细胞已经进入临床开发阶段。 7.杜氏肌营养不良新药在中国申报临床 4月25日，中国国家药监局药品审评中心（CDE）官网最新公示，Santhera Pharmaceuticals和曙方医药（Sperogenix）联合申报了vamorolone口服混悬液的临床试验申请。公开资料显示，这是曙方医药1.24亿美元引进的一款罕见病新药，已在美国递交用于治疗杜氏肌营养不良（DMD）的新药上市申请（NDA）。此前，vamorolone已获得FDA授予治疗DMD的孤儿药资格、快速通道资格与罕见儿科疾病认定（PRDD）。DMD是一种罕见的X-染色体连锁遗传疾病，是由于在X染色体上编码抗肌萎缩蛋白的基因出现突变而导致，几乎只影响男性。该病的特征为在出生时或出生后短时间内出现炎症反应，进而导致肌肉纤维化，以及临床上肌肉的萎缩与退化。皮质类固醇是DMD儿童与成人患者的标准治疗药物，这些激素虽然可以延缓疾病进展，但却存在一些副作用。Vamorolone是一种创新类固醇药物，虽然可与之结合的受体与皮质类固醇相同，但它能够选择性地激活类固醇的某些信号通路，因此被认为属于解离型抗炎药。即在引发抗炎症效果的同时，具有一定潜力避免传统类固醇所带来的安全性顾虑与副作用，进而可能取代现有疗法。Vamorolone最初由ReveraGen BioPharma开发。2018年，Santhera公司获得了该药的全球研发许可（除日本和韩国以外）。2022年1月，曙方医药与Santhera公司宣布就vamorolone达成独家授权协议，从而获得该产品在大中华区用于DMD及其他罕见病适应症的独家开发和商业化权益。 8.科伦药业第 4 款 ADC 启动临床 4 月 25 日，科伦药业的 Nectin-4 ADC SKB410 启动了 I 期临床试验，评价该药用于晚期实体瘤受试者的安全性、耐受性、药代动力学和初步疗效。这是 SKB410 的首个临床试验，标志着这一项目正式进入了临床阶段，这也是科伦启动临床的第 4 个 ADC 项目。SKB410 是在去年 12 月首次在国内递交临床试验申请，并于今年 2 月获批临床，今日刚刚启动临床。Nectin-4 在多种类型癌症中表达被重新激活并有高表达量，包括膀胱癌、三阴性乳腺癌、肺癌、结直肠癌、胰腺癌及卵巢癌。Nectin-4 的正调控也为一些癌症类型中总体生存率较差的独立生物标记物。由于在癌症中的选择性表达，Nectin-4 已成为有潜力用于治疗各种癌症的靶点。与 HER2、TROP2 等热门靶点相比，Nectin-4 ADC 当前赛道并不拥挤，据 Insight 数据库显示，当前全球共有 5 款临床阶段在研产品，另有 1 款获批上市，Seagen/安斯泰来 Enfortumab Vedotin（Padcev）。 9.全球首款！智翔金泰TSLP双表位双抗获批临床 4月24日，智翔金泰GR2002GR注射液获批临床，用于治疗哮喘。GR2002是智翔金泰自主研发的抗TSLP双特异性抗体，也是全球首个获批临床的TSLP双表位双抗。GR2002是智翔金泰利用其双抗药物发现技术平台开发的一款TSLP双抗。据智翔金泰招股说明书介绍，智翔金泰为解决双抗药物开发过程中的轻链错配开发了两种双抗平台，第一种为比较经典的scFv+Fab双抗结构，第二种为基于共同轻链（common light chain）的Fab+Fab双抗结构。GR2002即源于第二种。Fab+Fab双抗的优势是其结构与单抗高度类似，免疫原性低，而且可以借鉴单抗的制备工艺。此类双抗更适合于临床上需要多次给药的双抗药物的开发。但是共同轻链双抗的开发具有比较高的技术门槛。智翔金泰基于专利的双载体噬菌体呈现抗体库技术，以构建的大容量人抗体轻链库资源为基础，可以快速（3个月）筛选到选定的两个抗体的共同轻链，用于构建共同轻链双抗。 行业资讯 1. 10.75亿美元！礼来出售鼻喷升血糖疗法 4月24日，礼来宣布已将高血糖素鼻用粉雾剂（Baqsimi）的全球权益出售给Amphastar。根据协议，Amphastar将在交易完成时支付5亿美元现金给礼来，交易完成一周年时再支付1.25亿美金。此外，礼来也有资格获得总金额高达4.5亿美元的销售里程金。该协议预计2023年第二季度或第三季度完成。高血糖素鼻用粉雾剂装在一次性、即用型喷雾器中，给药后通过患者鼻粘膜被动吸收，无需患者进行吸气配合。2019年7月，Baqsimi获FDA批准用于4岁及以上年龄糖尿病患者严重低血糖的治疗，成为首款上市的胰高血糖素鼻喷剂，无需注射。它体积小、便于携带、单次固定给药剂量为3毫克，随时可用（无需混悬）。礼来财报显示，2022年Baqsimi全球销售额总计1.393亿美元。严重低血糖症对1型和2型糖尿病患者而言是一种严重的、需要紧急救治的疾病。其特征是出现精神和/或生理功能的改变，且只有在他人的协助下才能恢复。严重低血糖症如果不能得到及时的治疗可能导致十分严重的后果，如意识丧失，癫痫发作，昏迷、甚至死亡。 2.荣灿生物与国药中生上海生物制品研究所达成合作 4月23日，荣灿生物医药技术(上海)有限公司（荣灿生物）与国药集团中国生物上海生物制品研究所有限责任公司（上海生物制品研究所）签署合作协议，双方将充分利用各自资源与优势，合作打造自主可控的mRNA疫苗（药物）研发技术平台，并基于该平台研发多款mRNA疫苗（药物）。上海生物制品研究所总经理李秀玲表示：“mRNA技术作为通用的平台型技术，应用场景广泛，潜力巨大，协同推进mRNA关键核心技术攻关、自主技术开发和产品转化应用，对增强生物医药产业技术创新能力和保障人民群众身体健康具有重要意义。荣灿生物是国内为数不多的拥有mRNA递送载体核心技术的创新型企业，我们期待通过本次与荣灿生物的合作，共建具有独立自主知识产权的mRNA疫苗技术平台，全面提升自主可控的mRNA疫苗的可及性，为中国患者提供更优质的治疗方案，和更全面的医疗健康保障。”荣灿生物首席科学家章雪晴表示：“荣灿生物的LNP递送系统具有高效、安全、创新等优势，且在肺部靶向、心脑血管炎症靶向及特定细胞靶向的核酸载体研究领域走在国际前列。本次与上海生物制品研究所达成战略合作，将依托于国药集团中国生物国内领先的药物研发和产业化能力，共同开发出全新、高效、具有自主知识产权的mRNA疫苗（药物），满足未被满足的临床需求。” 3.启辰生与华润生物就mRNA预防性传染病疫苗达成战略合作协议 近日，北京启辰生生物科技有限公司与华润生物医药有限公司宣布，由启辰生生物旗下宜兴辰功新药开发有限公司与华润生物医药就mRNA RSV疫苗合作开发签订战略合作协议。双方以“互惠互补、创新发展”为原则，基于启辰生生物已获得临床批件的质粒、mRNA平台，纳米技术与递送系统平台、CMC平台成熟优势，借助华润生物医药的行业领先地位与临床经验，双方将在预防性传染病mRNA疫苗展开深度合作。呼吸道合胞病毒（Respiratory syncytial virus，RSV）是一种副黏病毒科肺炎病毒属的负单链RNA病毒，1956年从黑猩猩呼吸道中首次分离，由于和呼吸道感染有关，并且能把感染的细胞融合起来，因此得名。RSV在婴幼儿和老年人群体中疾病负担较重，儿童和老年人等高危人群感染后治疗方法以对症支持治疗/缓解症状为主，因此预防是RSV管理的关键，自然感染RSV无法诱导针对保守免疫原的长期免疫记忆反应，RSV免疫预防需要依靠被动预防（抗体药物）或主动预防（预防性疫苗）。RSV病毒首次发现至今已超过60年，但全球尚无有效预防和治疗药物上市。