A Phase 3, Randomized, Double-blind, Placebo-controlled, Multicenter Study of Mavorixafor in Participants With Congenital and Acquired Primary Autoimmune and Idiopathic Chronic Neutropenic Disorders Who Are Experiencing Recurrent and/or Serious Infections

The purpose of this study is to demonstrate the efficacy and evaluate the safety, and tolerability of mavorixafor in participants with congenital or acquired primary autoimmune and idiopathic chronic neutropenic disorders who are experiencing recurrent and/or serious infections as assessed by demonstrating its clinical benefit and increasing levels of circulating neutrophils.

开始日期2024-06-06

申办/合作机构

X4 Pharmaceuticals, Inc.

NCT04154488 / Active, not recruiting临床1/2期

A Phase 1b/2, Open-Label, Multicenter Study of Mavorixafor in Patients With Congenital Neutropenia and Chronic Neutropenia Disorders

This is a 2-part study of mavorixafor in participants diagnosed with chronic neutropenia. The main goal of Part 1 (Phase 1b) is to help researchers learn more about how the investigational medicine, mavorixafor, impacts people living with chronic neutropenia (including congenital, idiopathic, and cyclic). In Part 2 (Phase 2), the safety and tolerability of chronic dosing of mavorixafor will be evaluated in a larger participant population and the impact of 6-month chronic dosing of mavorixafor on participant neutropenia.

开始日期2020-10-16

申办/合作机构

X4 Pharmaceuticals, Inc.

NCT04274738 / Completed临床1期

A Phase 1b Trial of Mavorixafor, an Oral CXCR4 Antagonist, in Combination With Ibrutinib in Patients With Waldenstrom's Macroglobulinemia (WM) Whose Tumors Express Mutations in MYD88 and CXCR4

The primary objective of the study is to establish a pharmacologically active dose of mavorixafor in combination with ibrutinib based on pooled safety, clinical response, pharmacokinetic (PK) and pharmacodynamic (PD) data to select the recommended dose for a randomized registrations trial.

开始日期2020-04-30

申办/合作机构

X4 Pharmaceuticals, Inc.

100 项与 X4 Pharmaceuticals, Inc. 相关的临床结果

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0 项与 X4 Pharmaceuticals, Inc. 相关的专利（医药）

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项与 X4 Pharmaceuticals, Inc. 相关的文献（医药）

2024-07-23·Blood Advances

A novel transmembrane CXCR4 variant that expands the WHIM genotype-phenotype paradigm

Article

作者: Ellison, Maryssa ; Walter, Jolan E. ; Gordon, Sumai ; Badarau, Adriana ; Nguyen, Chi ; Pawar, Sumit ; Neff, Jadee L. ; Geier, Christoph B. ; Polyakova, Ekaterina ; Aleshkevich, Svetlana ; Sharapova, Svetlana O. ; Chen, Kelly ; Bledsoe, Jacob R. ; Grimbacher, Bodo ; Proietti, Michele ; Zmajkovicova, Katarina ; Sondheimer, Neal ; Sleasman, John W. ; Ujhazi, Boglarka ; Warnatz, Klaus ; Monticelli, Halenya ; Yilmaz, Melis ; Tarrant, Teresa K. ; Sakovich, Inga ; Wiest, Ivana ; Maier-Munsa, Sabine ; Taveras, Arthur G.

Warts, Hypogammglobulinemia, Infections, Myelokathexis (WHIM) syndrome is a rare immunodeficiency disease that results from impaired leukocyte trafficking (myelokathexis) predominately caused by gain-of-function variants in C-X-C chemokine receptor type 4 (CXCR4). Clinical manifestations of WHIM syndrome can differ in familial forms or in people harboring identical CXCR4 variants. All known pathogenic CXCR4 variants associated with WHIM syndrome (CXCR4WHIM) to date are localized in the intracellular C-terminus of CXCR4. We identified 4 unrelated patients with variable WHIM-like clinical presentations harboring a novel heterozygous CXCR4 variant (c.250G>C; p.D84H) localized at a highly conserved position in the transmembrane domain of the receptor outside the C-terminus. Functional characterization of the CXCR4D84Hvariant (CXCR4D84H) using patient-derived peripheral blood mononuclear cells and in vitro cellular assaysshow decreased CXCR4 internalization and increased chemotaxis in response to CXCL12, similar to known CXCR4WHIM, but also revealed unique features of CXCR4D84H signaling to cAMP, Ca2+ mobilization and AKT/ERK pathways. These findings are consistent with molecular dynamics simulations that show disruption of the Na+ binding pocket by D84H, resulting in collapse of the hydrophobic gate above and destabilization of the inactive state of CXCR4. Mavorixafor, a CXCR4 antagonist being evaluated in clinical trials for chronic neutropenia and WHIM syndrome, normalized CXCL12-mediated chemotaxis of CXCR4D84H patient lymphocytes ex vivo and improved WBC and subset counts in 1 patient with CXCR4D84H enrolled in the chronic neutropenia phase 1b clinical trial (NCT04154488). The present study expands the current understanding of CXCR4 function and genotype-phenotype correlations in WHIM syndrome and in people with WHIM-like phenotypes.

2024-07-04·Blood

A phase 3 randomized trial of mavorixafor, a CXCR4 antagonist, for WHIM syndrome

Article

作者: Peake, Jane ; Olbrich, Peter ; Ezra, Navid ; Kiani-Alikhan, Sorena ; Vossen, Matthias G. ; Stewart, Murray ; Bolyard, Audrey A. ; Hasle, Henrik ; Bertrand, Yves ; Levin, Carina ; Rodina, Yulia ; Donadieu, Jean ; Belschner, Andrea ; Kang, Hyoung Jin ; Jiang, Honghua ; Neth, Olaf ; Langguth, Daman ; Dale, David ; Wysocki, Christian A. ; Shcherbina, Anna ; Hu, Yanping ; Kulagin, Alexander ; MacLeod, Rick ; Dubuc, Susan ; Taveras, Arthur G. ; Alsina, Laia ; Badolato, Raffaele ; Azar, Antoine ; Rutten, Caroline E. ; Deyà-Martínez, Àngela ; Bridger, Gary J. ; Chen, Kelly ; Yan, Tina ; Tarrant, Teresa K. ; Dickerson, Kathryn E. ; Kuijpers, Taco W. ; Li, Sunny

AbstractWe investigated efficacy and safety of mavorixafor, an oral CXCR4 antagonist, in participants with warts, hypogammaglobulinemia, infections, and myelokathexis (WHIM) syndrome, a rare immunodeficiency caused by CXCR4 gain-of-function variants. This randomized (1:1), double-blind, placebo-controlled, phase 3 trial enrolled participants aged ≥12 years with WHIM syndrome and absolute neutrophil count (ANC) ≤0.4 × 103/μL. Participants received once-daily mavorixafor or placebo for 52 weeks. The primary end point was time (hours) above ANC threshold ≥0.5 × 103/μL (TATANC; over 24 hours). Secondary end points included TAT absolute lymphocyte count ≥1.0 × 103/μL (TATALC; over 24 hours); absolute changes in white blood cell (WBC), ANC, and absolute lymphocyte count (ALC) from baseline; annualized infection rate; infection duration; and total infection score (combined infection number/severity). In 31 participants (mavorixafor, n = 14; placebo, n = 17), mavorixafor least squares (LS) mean TATANC was 15.0 hours and 2.8 hours for placebo (P < .001). Mavorixafor LS mean TATALC was 15.8 hours and 4.6 hours for placebo (P < .001). Annualized infection rates were 60% lower with mavorixafor vs placebo (LS mean 1.7 vs 4.2; nominal P = .007), and total infection scores were 40% lower (7.4 [95% confidence interval [CI], 1.6-13.2] vs 12.3 [95% CI, 7.2-17.3]). Treatment with mavorixafor reduced infection frequency, severity, duration, and antibiotic use. No discontinuations occurred due to treatment-emergent adverse events (TEAEs); no related serious TEAEs were observed. Overall, mavorixafor treatment demonstrated significant increases in LS mean TATANC and TATALC, reduced infection frequency, severity/duration, and was well tolerated. The trial was registered at www.clinicaltrials.gov as #NCT03995108.

2023-11-02·Blood

Mavorixafor for Patients with Chronic Neutropenic Disorders Treated with G-CSF: Preliminary Response Data and G-CSF Dose Reduction in an Ongoing Phase 2, Open-Label, Multicenter Study Support Reduction in G-CSF Dosing

作者: Taveras, Arthur G. ; Wilson, David B. ; Walkovich, Kelly ; Dickerson, Kathryn E. ; Warren, Julia T. ; MacLeod, Rick ; Stewart, Murray ; Peters, Katie ; Adell, Katrina P. ; Walter, Jolan E. ; Sharathkumar, Anjali A. ; Dubuc, Susan ; Chen, Kelly

Introduction: Chronic neutropenia (CN) encompasses multiple disorders characterized by an absolute neutrophil count (ANC) <1500 cells/µL for >3 months, leading to increased susceptibility to recurrent and/or severe infections. CN can be sub-classified as congenital neutropenia defined by genetic variants ( ELANE variants are the most common), cyclic neutropenia (CyN) supported by documentation of ANC cycling with reciprocal monocytosis most often associated with certain ELANE variants, or chronic idiopathic neutropenia (CIN) defined as acquired neutropenia without an identifiable cause. The current treatment approach for CN involves long-term use of injectable granulocyte colony-stimulating factor (G-CSF), which can have associated adverse events (AEs) [Donadieu J. Expert Rev Hematol. 2021; Fioredda F. Hemasphere. 2023; Newburger PE. Semin Hematol. 2013]. Therefore, efforts are under way to discover other therapies for CN. In a phase 3 trial (NCT03995108), mavorixafor, an investigational oral CXCR4 antagonist, showed durable increases in ANC and reduction in total infection rate and severity in people with WHIM (Warts, Hypogammaglobulinemia, Infections, and Myelokathexis) syndrome [Badolato R. Clin Immunol. 2023]. Mavorixafor is being studied in a phase 2 trial for chronic neutropenic disorders (NCT04154488) [Warren JT. Blood. 2022] to evaluate the durability, safety, and tolerability of chronic dosing of mavorixafor with or without concurrent G-CSF. Here, we describe preliminary results for 3 participants in the ongoing phase 2 trial.Methods: Individuals (aged ≥12 years) with diagnosis of congenital neutropenia, CyN, or CIN ≥6 months prior and ANC <1000/µL (or no lower limit but <10,000/µL if on G-CSF) at screening visits were enrolled in the phase 2 trial. Eligible adult participants received once-daily 400 mg of mavorixafor while adolescents received weight-based dosing (>50 kg, 400mg; ≤50 kg, 200 mg) for 6 months with or without G-CSF. Participants already on G-CSF continued their individualized G-CSF dosing for ≥8 weeks, followed by potential dose and/or frequency reduction based on monthly ANC assessments. Hematologic parameters, including ANC, were assessed over 8 hours on day 1 and at months 1 and 3, with continued assessment at month 6. At months 2 and 4 (assessment to continue at month 5), hematologic parameters were assessed within 2 to 4 hours of dosing to reduce trial burden.Results: Data are reported as of July 17, 2023. Preliminary data from the first 3 participants on G-CSF receiving chronic once-daily oral mavorixafor for at least 2 months showed increases in mean ANC compared with baseline (BL) at all time points assessed in all participants. ANCs reached normal ranges for all participants. Increases in ANC supported physicians' decisions to reduce G-CSF dosing.Two participants (P1 and P2) with CIN achieved increases in ANC that enabled initiation of G-CSF tapering at month 2. For P1, G-CSF dose was tapered to 50% at month 2, and further G-CSF tapering occurred at month 3. ANC remained within normal ranges during this period. At month 4, G-CSF dosing was fully withdrawn, and the participant continued daily mavorixafor monotherapy. For P2, ANC increased ~3x baseline by month 2. G-CSF dose was tapered to 50% after month 2. ANC remained within normal ranges during this period. At month 3, G-CSF dosing was fully withdrawn, and the participant continued daily mavorixafor monotherapy with an ANC ~1000 cells/µL.A third participant (P3) with CyN receiving mavorixafor daily with concurrent G-CSF showed increases in ANC over BL for 4 months.All 3 participants remain on study, and have not experienced any serious AEsConclusions: Our report shows that chronic treatment with oral, once-daily mavorixafor in combination with G-CSF resulted in durable increases in ANC compared with BL for ≥3 months in 3 study participants. Reduction of injectable G-CSF dosing was implemented in 2 study participants with CIN following robust ANC increase observed after the first 2 months of combination treatment. These preliminary data also support the safety of concomitant treatment with G-CSF and mavorixafor. Additional data from the ongoing phase 2 study will further elucidate the potential of daily, oral mavorixafor as a treatment for CN to reduce the burden and associated AEs of injectable G-CSF. A phase 3 trial of people with CN is being planned.

152

项与 X4 Pharmaceuticals, Inc. 相关的新闻（医药）

2024-10-24

·GlobeNewswire-Health Care

X4 Pharmaceuticals to Report Third-Quarter 2024 Financial and Phase 2 Chronic Neutropenia Study Results During Conference Call and Webcast on November 13, 2024

BOSTON, Oct. 24, 2024 (GLOBE NEWSWIRE) -- X4 Pharmaceuticals, Inc. (Nasdaq: XFOR), a company driven to improve the lives of people with rare diseases of the immune system, today announced that it will report financial results for the third quarter ended September 30, 2024 and present results from its concluded Phase 2 clinical trial evaluating mavorixafor in people with chronic neutropenia on Wednesday, November 13, 2024. The company will host a conference call and webcast on the same day at 8:00 a.m. ET. The conference call can be accessed by dialing 1-800-267-6316 from the United States or 1-203-518-9783 internationally, followed by the conference ID: X4PHARMA. The live webcast and accompanying slide presentation will be accessible through the investor relations section of X4 Pharmaceuticals’ website at www.x4pharma.com. Following the conclusion of the call and webcast, a replay will be available on the company’s website. About X4 PharmaceuticalsX4 is delivering progress for patients by developing and commercializing innovative therapies for those with rare diseases of the immune system and significant unmet needs. Leveraging our expertise in CXCR4 and immune system biology, we have successfully developed mavorixafor, which has received U.S. approval as XOLREMDI® (mavorixafor) capsules in its first indication. We are also evaluating the use of mavorixafor in additional potential indications. X4 corporate headquarters are in Boston, Massachusetts and our research center of excellence is in Vienna, Austria. For more information, please visit our website at www.x4pharma.com. Company Contact:José Juves Head of Corporate & Patient Affairsjose.juves@x4pharma.com Investor Contact:Daniel Ferry Managing Director, LifeSci Advisorsdaniel@lifesciadvisors.com (617) 430-7576

上市批准财报临床2期

2024-08-30

·Endpoints

Ipsen sells priority review voucher for highest price since 2016

Ipsen Pharmaceuticals sold its priority review voucher to an unidentified “large global pharmaceutical company” for $158 million, it said this week, the highest publicly announced price for one of the vouchers in about eight years. Ipsen obtained the rare pediatric disease voucher in August 2023 thanks to the approval of Sohonos (palovarotene), which is intended to help adults and children with the rare disease known as fibrodysplasia ossificans progressiva. Priority review vouchers, or PRVs, can be acquired through the approval of rare pediatric disease or rare tropical disease drugs, and then can be sold or used by companies to speed the FDA’s reviews of any new drug. The $158 million sale is about $50 million more than any of the other PRV sales since last August, which includes sales of between $100 million and $108 million for Krystal Biotech, Valneva, X4 Pharmaceuticals and Day One Biopharmaceuticals. The last time a PRV sold for more than $158 million was in 2016, when PaxVax Bermuda sold its PRV, acquired via the approval of its cholera vaccine Vaxchora, for $290 million to an undisclosed buyer. PRVs have typically sold for about $100 million since the voucher programs began more than a decade ago, with higher prices of more than $150 million only coming between 2014 and 2016. “The sale of the PRV generates further firepower for our ongoing medicine launches and external-innovation strategy, which has seen multiple additions to our portfolio at every stage of development in 2024,” Philippe Lopes-Fernandes, chief business officer at Ipsen, said in a statement.

优先审批上市批准并购

2024-08-20

·生物制品圈

世界亟需金葡菌疫苗，但它仍在失败的路上

2011年春天，数据监测委员会要求默克公司及其合作者停止他们正在开展的金葡菌疫苗III期临床试验——这项针对全球细菌感染主要死因而开发的候选疫苗，在26个国家的8000名患者中进行了试验，但试验效果不佳（抗体显著增高，但VE=13.8%；-52.5, 51.6%，无显著意义），无法继续进行。接下来一年的深入研究发现，在接受心胸手术的患者中测试候选疫苗，接种了候选疫苗的试验者比注射安慰剂的患者更有可能死亡。默克公司负责该疫苗研发的首席医学官福勒博士指出，许多死亡的疫苗接种者都是死于多器官系统衰竭，专家推测这种风险可能部分源于炎症反应的扩大，但仍不确定这种试验性疫苗是如何使受试者变得更加脆弱的。反复失败的金葡菌候选疫苗： 10余款候选疫苗和3款单抗前赴后继的失败当疫苗界还沉浸在默克试验带来的伤害中时，2012年辉瑞公司推出了自己的候选疫苗试验，其中包括4种不同的金葡菌抗原。基于在小鼠中获得的良好疗效数据，辉瑞首先在健康成年人中进行了试验，随后又在3000多名接受择期手术的患者中进行了2b试验。但到2018年，这款投入5亿美金的重组工艺候选疫苗希望也已破灭。血清数据显示，虽然接种疫苗的患者产生了金葡菌抗体，但疫苗组的感染率与安慰剂组相同（针对12个主要疗效终点的VE均无显著意义）。与默克公司之前的研究一样，这项2b期试验也被终止了。后来人们进行了“曲线救国”策略，输注单克隆抗体以期提供短期保护的被动免疫。但研究发现，两种正在开发中的单克隆抗体——Arsanis公司的ASN100和Aridis公司的suvratoxumab和tosatoxumab相继在中期试验中失败。事实上在过去的25年时间里，大约有14种在研金葡菌疫苗在2期或3期临床试验中失败。一个又一个疫苗开发商投入了数百万美元，开发出了一个又一个拥有高超科学依据的候选疫苗。但无论疫苗研发策略多么有前途，无论候选疫苗在临床前研究中效果多么好，最终都没有一种疫苗能成功保护人类免受金葡菌的感染。金葡菌疫苗失败原因探索：若人体预先暴露金葡菌，疫苗就会失去作用加州大学圣迭戈分校儿科传染病科主任George Liu说：默克公司试验失败后，一连串无法解释的疫苗失败成了“房间里的大象”（意思是一些非常显而易见的，可是却一直被忽略的问题）。科学家和制造商们逐渐意识到，在投入研发金葡菌疫苗之前，需要先弄清楚什么原因导致了疫苗失效。 George Liu的实验室研究表明，金葡菌疫苗并不像其他有有效疫苗那样与免疫系统相互作用。金葡菌有办法操纵免疫系统来保护自己，使宿主防御系统和传统疫苗策略失去保护作用。这就是多年来试验失败的原因，这也为可能有效的疫苗带来了希望。金葡菌是一种与人类共存的病原体，在大多数情况下不会对人类造成伤害。多达1/3的成人和50%的儿童在鼻腔中持续携带这种病菌，但都没有任何症状。然而一旦金葡菌进入血液、肺部或软组织，就会致命。医疗器械、外科手术、未愈合的伤口和注射毒品都是人们感染金葡菌的常见途径，可导致败血症、肺炎、心内膜炎和骨感染。每年造成全球100多万人死亡，美国因此的住院费用高达数十亿美元。这种微生物对抗生素的耐药性不断增加，也限制了治疗方案的选择，增加了疫苗的紧迫性。几乎所有失败的金葡菌疫苗人体试验背后都有一个成功的小鼠临床前研究，但小鼠模型与人类有本质区别——人生活在金葡菌的环境中，而实验小鼠则不是。 George Liu在小鼠接种的疫苗与默克公司试验中使用的疫苗类似，让它们预先感染金葡菌，以观察接触金葡菌是否会影响它们随后对疫苗的反应。在从未接触过金葡菌的对照组小鼠中，疫苗 “效果很好”。但在预先暴露于金葡菌的小鼠中，疫苗没有保护作用。 2022年，研究人员在Cell Host & Microbe杂志上报告了这一结果，它反映了研究人员在临床试验中看到的情况：当金葡菌预先启动了免疫系统时，疫苗就不起作用了。金葡菌疫苗无效的机制：预先暴露的金葡菌留下了错误的免疫记忆免疫系统会产生针对抗原的特异性抗体，在第一次遇到抗原后，免疫系统会产生免疫记忆。如果再次遇到病原体/抗原，这些抗体就会被迅速召回，这种免疫记忆对长期保护至关重要。对小鼠体内抗体的深入研究表明：金葡菌扰乱了它们的免疫记忆。但金葡菌已经学会了破解它：当实验室小鼠暴露于金葡菌时，病原体会留下错误的免疫反应印记。免疫系统学会了制造抗金葡菌抗体，这些抗体会迅速赶到现场，但却无法产生保护作用。通常情况下，抗体会与细菌细胞壁上的抗原结合，然后将细菌拖到巨噬细胞或中性粒细胞等免疫细胞中，在一个称为吞噬的过程中被消化和消灭。但在George Liu的研究中，接触金葡菌的小鼠产生了带有额外糖基的抗体。这些抗体仍然附着在细菌表面，但当它们到达巨噬细胞时，免疫细胞并没有消灭病原体。当研究人员移除糖基后，吞噬作用就恢复了。这种免疫操作使金葡菌得以与人类共生，也破坏了疫苗策略。当小鼠或已经接触过这种细菌的人接种疫苗后，他们的免疫系统会优先恢复原来的免疫反应，而不是根据疫苗建立新的保护性反应。去年年底George Liu发表在Cell Reports Medicine上的一项后续研究中扩大了研究范围，研究了10种金葡菌抗原，其中许多抗原在临床试验中都失败了。这些抗原包括8种能促使机体产生自身抗体的活性疫苗抗原，以及2种候选单克隆抗体：suvratoxumab和tefibazumab。与金葡菌细胞壁相关的活性疫苗抗原对之前接触过金葡菌的小鼠没有保护作用，这些疫苗抗原只是唤醒并强化了无保护性的免疫印记。单克隆抗体对暴露于金葡菌的小鼠也无效，但原因不同——它们无法与金葡菌预编程的非保护性内源抗体竞争。这些内源抗体与细菌细胞结合在一起，所以当单克隆抗体到来时，它们无处可躲。正常情况下，内源性抗体会扑上去杀死病原体，但相反内源性葡萄球菌抗体会进入病原体，并保护细菌不被治疗药物杀死。新的变通办法：优势抗原/T细胞免疫/多价/调整人群策略尽管Liu在2023年的研究中检测的大多数抗原都被金葡菌的免疫逃避技术所击败，但这项工作表明仍有乐观的余地，以排除法的形式留给了金葡菌疫苗新思路：优势抗原策略（针对α毒素、抗原表面决定簇B）、T细胞免疫策略、多价疫苗策略、调整接种人群策略（考虑暴露于金葡菌前的生命早期人群接种）。优势抗原诱导策略：其中一种活性疫苗抗原能诱导出针对金葡菌关键毒力因子α毒素的中和抗体——即使是之前接触过这种毒素的小鼠也能产生中和抗体。这种细菌毒素使细菌在宿主免疫反应中占据优势，中和它们可以给免疫系统提供更多抗感染的机会。其次，优势抗原铁调控表面决定簇B（或称IsdB）会诱发非保护性抗体，针对引起较小免疫反应但可能因免疫印记缺陷而得不到保护的亚优势抗原区的研究更为有效。 T细胞免疫策略：研究发现了T细胞对抑制金葡菌的重要性，2014年对默克公司临床试验的一部分患者进行的跟踪分析发现，死亡率与疫苗接种者血清中某些T细胞细胞因子水平低密切相关。虽然这项研究规模较小，但死亡患者似乎对接种疫苗的T细胞反应不恰当或不存在，尽管他们体内有大量抗体，全国儿童医院重症监护医师Chris Montgomery在一封电子邮件中写道。实验室的数据表明，金葡菌α毒素会损害T细胞发育，阻碍疫苗应答。T细胞功能障碍患者也更容易反复感染金葡菌，这可能表明这些免疫细胞发挥着关键的防御作用。杜克大学福勒教授说，最终未来疫苗的最佳方法可能是多价疫苗，这些疫苗将针对金葡菌生命周期不同阶段和感染阶段的毒素、T细胞和其他多种抗原的某种组合。还有一个问题是谁应该接种疫苗。疫苗研发人员一直把重点放在已被金葡菌定植并有高感染风险的患者身上。但一些专家表示，为婴儿接种疫苗是更好的策略。欧林生物的金葡菌疫苗仍在“不确定”中 2015年，欧林生物与陆军军医大学联合开发的重组金葡菌疫苗获批临床试验。2023年II期临床研究结果发表于Vaccine杂志，安全性和免疫原性良好，疫苗组抗体水平显著提高，重复了默克和辉瑞等公司的结果。 2023年末，其适用于骨科手术人群的多中心RCT 3期临床试验中期数据，向DSMB（数据监查委员会）提交审查前期临床试验结果和III期临床试验方案，以判断试验无效的可能性及是否需要终止试验，从而减少I类新药研发的风险。但DSMB给出的建议是：继续进行研究。这款“诺奖级”的新疫苗，有希望“花落”我国进度最快的欧林吗？ JAMA. Published online August 2, 2024. doi:10.1001/jama.2024.1015 欧林生物：重组金葡菌疫苗Ⅲ期临床试验已入组3,600余例，临床入组医院增加至60家以上 (baidu.com) 欧林生物重组金葡菌疫苗Ⅱ期临床试验研究论文在《疫苗》杂志发表 (cls.cn) 识别微信二维码，添加生物制品圈小编，符合条件者即可加入生物制品微信群！请注明：姓名+研究方向！版权声明本公众号所有转载文章系出于传递更多信息之目的，且明确注明来源和作者，不希望被转载的媒体或个人可与我们联系(cbplib@163.com)，我们将立即进行删除处理。所有文章仅代表作者观点，不代表本站立场。

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