A Phase 2b, Placebo-Controlled, Randomized Study of BPZE1 Intranasal Pertussis Vaccine in Healthy Adults to Assess Protection Against Colonization Following Challenge With Virulent Wild-Type Bordetella Pertussis

This is a randomised, double-blinded, placebo-controlled trial of BPZE1 that includes virulent B. pertussis challenge followed by a safety follow-up.

开始日期2022-06-20

申办/合作机构

ILiAD Biotechnologies LLC

NCT05136599

/ Completed临床1期IIT

Open-label, Phase 1, Dose-escalation Clinical Trial to Establish a Controlled Human Infection Model by Determining the Optimal and Safe Bordetella Pertussis Dose That Induces Mild Symptomatic Infection and Colonization in Healthy Adults

This study aims to establish a Controlled Human Infection Model of Bordetella pertussis by determining a reproducible and safe infectious bacterial dose (challenge inoculum) that achieves colonization and mild symptomatic infection in healthy adults.

开始日期2022-01-28

申办/合作机构

Dalhousie University

[+3]

NCT05116241

/ Completed临床2期

Phase 2b Placebo-Controlled Randomized Study of BPZE1 Intranasal Pertussis Vaccine in Healthy School-Age Children to Assess Immunological Response and Safety of a Single Dose BPZE1 With/Without Coadministration of Tdap (Boostrix™)

This study evaluates the safety and immunogenicity of the BPZE1 live, attenuated pertussis vaccine, intended to prevent nasopharyngeal colonization and pertussis disease, and compares BPZE1 vaccine vs Boostrix vaccine vs both BPZE1 and Boostrix vaccines. This is a multi-center, randomized, placebo- and active-comparator-controlled study in healthy, school-age children with a 6-month safety follow-up after the first vaccination.

开始日期2021-11-11

申办/合作机构

ILiAD Biotechnologies LLC

100 项与 Pertussis vaccine(INSERM) 相关的临床结果

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100 项与 Pertussis vaccine(INSERM) 相关的转化医学

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100 项与 Pertussis vaccine(INSERM) 相关的专利（医药）

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项与 Pertussis vaccine(INSERM) 相关的文献（医药）

2026-10-01·LANCET INFECTIOUS DISEASES

Immunogenicity and safety of BPZE1, an intranasal live attenuated pertussis vaccine, evaluated with and without tetanus-reduced diphtheria-acellular pertussis vaccine in healthy children aged 6–17 years: a randomised, active-controlled and placebo-controlled, phase 2b trial

Article

作者: Richmond, Peter ; Goldstein, Peter ; Bernatoniene, Jolanta ; Ivankovich-Escoto, Gabriela ; Rubin, Keith ; Locht, Camille ; Bandi, Srini ; Avila, Lydiana ; Weissfeld, Lisa ; Marshall, Helen S ; Faust, Saul N ; Nolan, Terry ; Lang, Wei ; Rayner, Svetlana ; Noviello, Stephanie ; Céspedes, Javier ; Samal, Vivek ; Shenoy, Anil

BACKGROUND:

Pertussis remains an important public health issue, in part because current vaccines fail to induce potent mucosal immunity against the causative agent, Bordetella pertussis. In contrast, the live attenuated vaccine BPZE1 induces robust nasal secretory IgA (S-IgA) and protects against B pertussis infection in adults primed with whole-cell vaccines. We evaluated safety and immunogenicity of BPZE1 administered either alone or with tetanus-reduced diphtheria-acellular pertussis vaccine (Tdap) in children primed with acellular pertussis vaccines.

METHODS:

In this randomised, observer-blinded, placebo-comparator and active-comparator-controlled, phase 2b trial, conducted at 16 paediatric hospital-based research centres and academic clinical sites (eight in the UK, five in Australia, and three in Costa Rica), children aged 6-17 years were randomly assigned by an interactive response technology system using permuted blocks of three (1:1:1 ratio) to receive approximately 10⁹ colony-forming units of BPZE1 intranasally with intramuscular placebo, intramuscular Tdap with intranasal placebo, or BPZE1 plus Tdap. Eligible participants had not received pertussis vaccination or been diagnosed with pertussis in the past three years. The primary immunogenicity outcome was geometric mean fold rises (GMFRs) in nasal S-IgA to whole-cell extract from baseline to day 29 in the per-protocol immunogenicity analysis set, with the outcome met if the lower limit of the 95% CI for the GMFR was greater than 1 for both BPZE1 and BPZE1 plus Tdap groups. The key secondary immunogenicity outcome was baseline-adjusted day 29 geometric mean ratios (GMRs) of anti-acellular pertussis vaccine antigen serum IgG levels between the BPZE1 plus Tdap and Tdap groups, and differences between these groups in percentages of participants with seroprotective antibody concentrations (≥0·1 IU/mL) against diphtheria and tetanus toxoids. Reactogenicity was evaluated through the 7 days after vaccination (primary safety outcome), unsolicited adverse events for 28 days post-vaccination, and serious adverse events throughout the study. The trial was registered at ClinicalTrials.gov (NCT05116241) and is complete.

FINDINGS:

Between Nov 11, 2021, and Oct 24, 2023, 381 children aged 6-17 years were screened, of whom 368 were enrolled in the study and randomly assigned to receive study vaccine. Two participants were not vaccinated, and 366 received either intranasal BPZE1 and intramuscular placebo (n=123), intranasal BPZE1 and intramuscular Tdap (n=121), or intranasal placebo and intramuscular Tdap (n=122). 354 participants completed the study. Overall, the mean age was 10·8 years (SD 2·79), 196 (54%) were male and 170 (46%) were female, and most participants were categorised as other race (176 [48%]) or White (161 [44%]). The primary endpoint of S-IgA induction was met for B pertussis whole-cell extracts (WCE) in the BPZE1 (n=111) and BPZE1 plus Tdap (n=113) groups, with GMFRs at day 29 post-vaccination of 3·8 (95% CI 3·1-4·7; p<0·0001) and 3·5 (2·9-4·3; p<0·0001), respectively. BPZE1 plus Tdap induction of serum IgG against all acellular pertussis antigens was comparable with those induced by Tdap at day 29, with GMRs of 1·5 (95% CI 1·1-2·1), 1·1 ( 0·9-1·4), and 1·1 ( 0·9-1·4), for anti-pertactin, anti-filamentous haemagglutinin, and anti-pertussis toxin, respectively. All participants (100%) in both Tdap groups had protective anti-diphtheria and anti-tetanus toxoid antibody levels of 0·1 IU/mL or greater at day 29. Solicited adverse events occurred in 59 (48%) of 123, 81 (67%) of 121, and 88 (72%) of 122 participants in the BPZE1, BPZE1 plus Tdap, and Tdap groups, respectively, and events were predominantly mild to moderate. Frequencies of unsolicited adverse events were similar across groups. No vaccine-related serious adverse events, deaths, or adverse events leading to study discontinuation were reported.

INTERPRETATION:

BPZE1 was well tolerated in children aged 6-17 years, and induced robust nasal S-IgA responses to B pertussis WCE without interference from simultaneously administered Tdap. BPZE1 vaccination did not interfere with Tdap-induced serum IgG to B pertussis antigens and tetanus and diphtheria toxoids. These findings support further development of BPZE1 as a vaccine candidate against pertussis in acellular pertussis vaccine-primed children 6 years and older.

FUNDING:

ILiAD Biotechnologies, Weston, FL, USA.

TRANSLATION:

For the Spanish translation of the abstract see Supplementary Materials section.

2026-01-02·Expert Opinion on Drug Delivery

The potential public health benefit of live-attenuated pertussis vaccines

Review

作者: Rubin, Keith ; Locht, Camille

INTRODUCTION:

Despite high vaccination coverage, pertussis remains endemic, and epidemic cycles continue to occur every 3-5 years, illustrating the shortcomings of current vaccination strategies. Unlike current vaccines, especially acellular pertussis vaccines, which prevent disease for short duration but do not prevent nasal colonization by the etiological agent Bordetella pertussis, natural infection can induce durable immunity against disease and infection. Accordingly, live-attenuated nasal pertussis vaccine candidates have been developed to mimic the immunogenicity of natural infection without causing disease.

AREAS COVERED:

We summarize the current status of live-attenuated nasal pertussis vaccines based on a literature search with keywords 'pertussis vaccine,' 'live,' and 'attenuated.' These candidates were designed based on different strategies, including dependence on critical metabolites for bacterial growth, genetic attenuation of virulence, and modification of immunomodulatory properties of Bordetella.

EXPERT OPINION:

Only vaccines based on genetic attenuation of virulence have reached clinical development thus far. The most advanced candidate is BPZE1, which has successfully completed six clinical studies, demonstrating safety, systemic and mucosal immunogenicity, and the ability to prevent or substantially reduce B. pertussis infection. If BPZE1 is eventually adopted for global use, it will likely be a major breakthrough in the ultimate control of pertussis, which has plagued humanity for centuries.

2025-12-01·Lancet Microbe

Efficacy, immunogenicity, and safety of the live attenuated nasal pertussis vaccine, BPZE1, in the UK: a randomised, placebo-controlled, phase 2b trial using a controlled human infection model with virulent Bordetella pertussis

Article

作者: Raud, Lucy ; Samal, Vivek ; Gbesemete, Diane ; Goldstein, Peter ; Laver, Jay R ; Hill, Alison R ; Faust, Saul N ; Locht, Camille ; Coutinho, Tyween ; Weissfeld, Lisa ; Dale, Adam P ; Ferreira, Daniela M ; Ramasamy, Maheshi N ; Noviello, Stephanie ; Rubin, Keith ; Guy, Jonathan ; Reed, Thomas A N ; Read, Robert C ; Babbage, Gavin ; Lang, Wei ; Ibrahim, Muktar ; Solovay, Ken

BACKGROUND:

Pertussis is a severe respiratory disease caused by Bordetella pertussis. Although vaccines prevent disease for a limited duration, they do not prevent infection and transmission. We aimed to assess the safety and efficacy of BPZE1 at preventing or substantially reducing colonisation by virulent B pertussis using a robust controlled human infection model.

METHODS:

This randomised, placebo-controlled, phase 2b trial was conducted at University Hospital Southampton and University of Oxford in the UK. Eligible participants were healthy adults aged 18-50 years, who complied with the protocol, refrained from smoking and nasal sprays, and were fully vaccinated against SARS-CoV-2. Exclusion criteria were pertussis vaccination or illness (<5 years), baseline anti-pertussis toxin serum IgG (>20 International Units [IU]/mL) or anti-pertactin serum IgG (>30 IU/mL) concentrations, and a positive SARS-CoV-2 test. Participants were randomly assigned (1:1), using permuted blocks with a block size of four, to receive an intranasal dose of 10⁹ colony-forming units (CFU) of BPZE1 or placebo (lyophilised buffer) and were challenged 60-120 days later with 10⁵ CFU virulent B pertussis. Masked staff administered the study vaccine. Nasal mucosal secretion and blood samples were collected. The primary outcome was negative B pertussis cultures of nasal washes at days 9, 11, and 14 after virulent challenge in the modified intention-to-treat (mITT; defined as all participants randomly assigned to treatment who were vaccinated, challenged, and had at least one culture result at day 9, 11, or 14 post-challenge) and per protocol adequate inoculum populations (defined as all participants in the mITT population who received a challenge inoculum equal to or higher than the target (≥0·5 × 10⁵ CFU; sensitivity analysis). This trial is registered with ClinicalTrials.gov, NCT05461131.

FINDINGS:

Between June 23, 2022, and Oct 26, 2023, 141 participants were assessed for eligibility, of whom 88 were ineligible and 53 were randomly assigned (26 to the BPZE1 group and 27 to the placebo group). 26 (49%) participants were male and 27 (51%) were female, with a mean age of 30·42 years (SD 8·49). Participants self-identified as White (42 [79%]), Black (six [11%]), or Asian (five [9%]). Five (9%) participants did not receive virulent challenge and two (4%) were lost to follow-up before virulent challenge. 46 (87%) participants received virulent challenge at 60-120 days (24 in the BPZE1 group vs 22 in the placebo group). One in the BPZE1 group withdrew consent and one in the placebo group was not evaluable due to COVID-19. 44 (83%) completed the challenge trial unit stay (23 [88%] vs 21 [78%]). 24 (92%) participants in the BPZE1 group and 21 (78%) in the placebo group were included in the mITT population. In the mITT population, the number of participants with no detectable colonisation on days 9, 11, and 14 post-challenge was higher in the BPZE1 group (14 [58%; 95% CI 39-76] of 24 vs seven [33%; 17-55] of 21 in the placebo group; p=0·091). Four (17%) in the BPZE1 group and five (24%) in the placebo group received a lower challenge dose than the target. In the per protocol adequate inoculum population, 12 (60%; 39-78) of 20 in the BPZE1 group and four (25%; 10-50) of 16 in the placebo group had no detectable colonisation by B pertussis B1917 on days 9, 11, and 14 post-challenge (p=0·033). Most participants reported at least one solicited adverse event during the 7 days after vaccination (22 [85%] of 26 in the BPZE1 group vs 22 [81%] of 27 in the placebo group), which were mostly mild (grade 1) in severity. Unsolicited adverse events were reported in similar frequency in the BPZE1 and placebo groups during the 28 days after vaccination (seven [27%] of 26 vs nine [33%] of 27). No serious adverse events or discontinuations due to adverse events were reported during the trial.

INTERPRETATION:

Intranasal BPZE1 vaccination prevented or substantially reduced infection after challenge with virulent B pertussis and is an attractive vaccine candidate. Given the favourable safety profile, large phase 3 trials are warranted to confirm these initial findings.

FUNDING:

ILiAD Biotechnologies.

项与 Pertussis vaccine(INSERM) 相关的新闻（医药）

2026-10-15

·创新药物递送

全球经口吸入和鼻腔给药领域 · 头条速览（2026年10月上）

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Importantly, YUTREPIA® (treprostinil) inhalation powder will remain available to patients with PAH without disruption. Liquidia will continue to advance YUTREPIA and L606 clinical programs as previously outlined. Liquidia宣布与United Therapeutics达成全球和解，确保YUTREPIA在PAH领域持续商业化 Liquidia宣布与United Therapeutics就全部未决诉讼达成和解，确保YUTREPIA®（曲前列尼尔吸入粉雾剂）在肺动脉高压（PAH）领域持续商业化供应。公司同时将按计划推进YUTREPIA及L606项目开发。 2 LTR Pharma Brings Roxus® Intranasal Vardenafil to U.S. Prescribers: Peak Plasma Levels in a Median of 10 Minutes 媒体：PR Newswire 2026/10/8 LTR Pharma announced the U.S. availability of Roxus®, an intranasal vardenafil formulation compounded through a Section 503A pharmacy, offering men with erectile dysfunction a faster-acting alternative to oral PDE5 inhibitors.Clinical studies showed Roxus reached peak plasma concentrations in a median of 10 minutes, approximately six times faster than oral vardenafil LTR Pharma将Roxus®伐地那非鼻喷引入美国市场：中位达峰时间仅10分钟 LTR Pharma宣布其用于治疗勃起功能障碍（ED）的鼻喷盐酸伐地那非产品 Roxus® 已在美国上市。该产品由503A复方药房提供处方服务，为患者提供较口服PDE5抑制剂起效更快的治疗选择。临床研究显示，Roxus中位达峰时间仅约10分钟，较口服伐地那非快6倍。 3 Aptar Expands Pharma Operations in Mumbai, Reinforcing India as a Strategic Growth Market 媒体：Aptar Pharma 2026/10/8 Aptar announced the expansion of its operations in Mumbai, India to add local production of several pharma inhalation technologies, including Aptar’s pressurized metered dose inhaler (pMDI) valve, as well as breath-actuated systems and multi dose nasal spray systems. Aptar扩大孟买医药业务运营，进一步强化印度作为战略增长市场的地位 Aptar宣布扩建其位于印度孟买的运营基地，并新增多项医药吸入给药技术的本地化生产能力，包括Aptar的定量吸入气雾剂阀门、呼吸触发式给药系统以及多剂量鼻喷雾系统。 4 Lupin Launches Formoflo-G® MDI, World’s First Fixed-dose Triple Combination Drug for COPD Management 媒体：Lupin 2026/10/6 Lupin Limited (Lupin) has launched Formoflo-G® MDI, the world's first fixed-dose triple combination MDI for COPD in India. The product is available as a twice-daily, single-strength fixed dose, bringing hope to more than 55 million people living with COPD in India, a condition that remains a leading cause of death and disability. Lupin推出全球首个用于COPD管理的固定剂量三联吸入气雾剂 Lupin在印度推出Formoflo-G®吸入气雾剂，这是全球首个用于COPD治疗的固定剂量三联复方MDI。该产品采用每日两次给药方案，为印度超过5500万名COPD患者提供新的治疗选择。 5 Belhaven Biopharma Initiates a Pivotal Biocomparability Study to Evaluate the Pharmacokinetics, Pharmacodynamics, and Safety of Nasdepi(R) vs. Epinephrine Intramuscular Injections 媒体：Newswire 2026/10/6 Belhaven Biopharma, announced that the U.S. FDA has accepted their IND application for Nasdepi® (BBP01), a novel, investigational nasal powder epinephrine formulation, designed as a needle-free alternative for the emergency treatment of anaphylaxis. Belhaven Biopharma启动关键性生物可比性研究 Belhaven Biopharma宣布，美国FDA已受理其 Nasdepi®（BBP01）的IND申请，公司启动可比性研究用以评估Nasdepi®与肾上腺素肌肉注射剂的药代动力学、药效学及安全性。Nasdepi®是一种新型在研肾上腺素鼻用粉剂，旨在为过敏性休克紧急治疗提供一种无针给药替代方案。 6 Bespak announces collaboration with Shin Nippon Biomedical Laboratories to expand global access to FDA-approved nasal dry powder device 媒体：Bespak 2026/09/21 Bespak has partnered with Shin Nippon Biomedical Laboratories (SNBL) to broaden global access to its FDA-approved nasal dry powder delivery device. The collaboration will combine Bespak’s device technology with SNBL’s drug development expertise to support the development and commercialization of intranasal therapies. Bespak宣布与SNBL达成合作，扩大FDA批准鼻用干粉给药装置的全球可及性 Bespak宣布与新日本科学（SNBL）达成合作，进一步扩大其FDA批准的鼻用干粉给药装置在全球市场的可及性。双方将结合Bespak的装置技术与SNBL的药物开发能力，加速鼻腔给药产品的开发和商业化。 7 ILiAD Biotechnologies Publishes Pediatric Phase 2b SUPER Results for Intranasal Pertussis Vaccine BPZE1 in The Lancet Infectious Diseases 媒体：ScienceDirect 2026/10/1 ILiAD Biotechnologies published Phase 2b SUPER trial results for its intranasal pertussis vaccine candidate BPZE1 in The Lancet Infectious Diseases. The study demonstrated a favorable safety profile and immune response in children, supporting further development of BPZE1 as a next-generation vaccine against pertussis. LiAD公布鼻喷百日咳疫苗BPZE1儿童IIb期研究结果 ILiAD Biotechnologies在《柳叶刀·感染病学》发表了其鼻喷百日咳疫苗BPZE1的IIb期SUPER研究结果。研究显示BPZE1单次鼻腔给药后,在儿童中具有良好的安全性和免疫原性，为其作为新一代百日咳疫苗的进一步开发提供了支持。 8 Decoy Therapeutics Reports Preclinical Data Supporting Once-Weekly Intranasal Dosing for DCOY-CoV Program 媒体：Decoy Therapeutics 2026/9/29 Decoy Therapeutics, a biotechnology company pioneering Designable Multi-Antivirals (D-MAVs™), announced preclinical pharmacokinetic (PK) data supporting once-weekly intranasal administration for its lead program, DCOY-CoV. Results demonstrate that a single intranasal dose maintains therapeutic drug concentrations in both upper respiratory mucosal tissues and deep lung tissue for up to seven days. Decoy Therapeutics公布临床前数据，支持其DCOY-CoV项目每周一次鼻腔给药方案 Decoy Therapeutics是一家专注于可设计多重抗病毒药物（D-MAVs™）开发的生物技术公司。公司近日公布了其领先项目 DCOY-CoV 的临床前药代动力学（PK）数据，支持采用每周一次鼻腔给药的治疗方案。 9 Nasus Pharma Announces Enrollment and Dosing of First Patient in Phase 1 Study of NS003 Intranasal Formulation of Ondansetron 媒体：Nasus Pharma 2026/9/29 Nasus Pharma announced that the first patient has been enrolled and dosed in the Company’s Phase 1 pharmacokinetic (PK) trial of NS003, its proprietary intranasal powder formulation of ondansetron being developed for chemotherapy-induced nausea and vomiting (CINV) and post-operative nausea and vomiting (PONV). Nasus Pharma宣布NS003鼻腔给药制剂I期临床研究完成首例受试者入组并给药 Nasus Pharma宣布其NS003项目已在I期药代动力学研究中完成首例受试者入组并给药。NS003是一款在研昂丹司琼鼻用粉剂，拟用于化疗相关恶心呕吐（CINV）和术后恶心呕吐（PONV）的治疗与预防。头条速览（2026年9月下） ► 点击阅读头条速览（2026年9月上） ► 点击阅读本公众号致力于分享药物递送领域的前沿信息，促进同行交流与学习。所引用的文章内容均来源于网络，版权归原作者所有。如涉及侵权，请及时联系我们核实处理。本公众号所发布的信息、文章和观点，旨在为读者提供一般性的参考和交流，不构成任何专业建议。本公众号对转载、分享的内容、陈述、观点判断保持中立，不对所包含内容的准确性、可靠性或完善性提供任何明示或暗示的保证，仅供读者参考。对于因使用或信赖本公众号信息而引致的任何损失，本公众号概不负责。如需转载，请注明来源于本公众号。

2026-10-04

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全球生物医药与创新药动态日报

本期导读：ADC临床数据密集发布——Genmab Rina-S在铂耐药卵巢癌ORR达45.9%，成为本期最重磅数据；Biogen抗干扰素受体抗体52周皮肤型红斑狼疮数据、Agenus免疫组合3年生存数据同日亮相；Zipalertinib一线EGFR外显子20插入突变NSCLC启动NDA提交；中国小核酸企业中美瑞康展示首个saRNA激活utrophin治疗DMD的人体证据；疫苗、CGT、酶替代疗法与监管动态多点开花。需如实说明：10月1—3日海外无大型并购交易，海外大额一级市场融资事件亦较少；本期融资动态以国内小核酸企业融资（内嵌于第4条）为代表。01ADCGenmab Rina-S（FRα靶向ADC）2期RAINFOL-01：铂耐药卵巢癌ORR 45.9% 在109例铂耐药卵巢癌（PROC）患者中，Rina-S 120mg/m²每3周给药确认ORR达45.9%（95%CI 36.3–55.7），含5例完全缓解，中位缓解持续时间12.1个月、51%的应答者一年后仍保持应答，中位PFS 9.5个月。无论FRα表达水平（包括低表达与无表达）均有抗肿瘤活性，且不受既往mirvetuximab治疗影响；53%的患者已接受过3—4线治疗。涉及公司/机构：Genmab（Rina-S为FRα靶向、exatecan载荷的TOPO1抑制剂ADC）信息来源：Genmab官网02抗体药自身免疫Biogen litifilimab（抗IFNAR1单抗）皮肤型红斑狼疮52周2期数据：快速持久清除 AMETHYST研究（2/3期）Part A新52周数据显示，litifilimab在皮肤型红斑狼疮（CLE）患者中实现快速且持久的皮肤疾病清除，52周仍维持改善。该药2026年1月已获FDA突破性疗法认定，为潜在first-in-class疗法；AMETHYST Part 3关键数据预计2027年上半年读出。涉及公司/机构：Biogen 信息来源：Biogen官网03创新药FDA申报Zipalertinib+化疗一线EGFR外显子20插入突变NSCLC启动NDA提交（FDA RTOR） Cullinan Therapeutics与Taiho启动zipalertinib联合化疗一线治疗EGFR外显子20插入突变非小细胞肺癌的NDA提交，纳入FDA实时肿瘤学审评（RTOR）项目。该提交基于全球III期REZILIENT3研究（zipalertinib+化疗 vs 化疗），为这一难治基因亚型的重要注册进展。涉及公司/机构：Cullinan Therapeutics、Taiho Oncology 信息来源：Cullinan Therapeutics官网04小核酸新技术融资中美瑞康（Ractigen）WMS展示首个saRNA激活utrophin治疗DMD的人体证据 RAG-18（小激活RNA，RNAa机制）在杜氏肌营养不良（DMD）患者肌肉活检中显示utrophin蛋白上调，为全球首个在人体中证明该机制可激活治疗性靶点的数据；该机制不依赖具体突变类型，有望惠及几乎所有DMD患者（研究为北京协和医院牵头的研究者发起试验）。公司7月刚完成超3100万美元新一轮融资（国中资本领投），累计融资约8350万美元；RAG-18已获FDA孤儿药与罕见儿科疾病认定。涉及公司/机构：中美瑞康（Ractigen Therapeutics）、国中资本、北京协和医院信息来源：AInvest05疫苗ILiAD鼻内减毒活百日咳疫苗BPZE1儿科2b期研究发表于《柳叶刀-感染病学》 BPZE1（鼻内减毒活百日咳疫苗）SUPER儿科2b期临床试验结果发表于The Lancet Infectious Diseases：随机双盲、安慰剂与活性对照研究显示，BPZE1有望阻断百日咳杆菌定植与传播，扩展其公共卫生与商业化潜力。涉及公司/机构：ILiAD Biotechnologies 信息来源：Morningstar（Business Wire）06抗体药肿瘤免疫Agenus BOT+BAL复发性卵巢癌：IGCS 2026报告48%估计3年生存率 botensilimab（CTLA-4）+balstilimab（PD-1）免疫组合在复发性卵巢癌中报告48%的估计3年生存率（C-800-01研究），并按铂敏感性与既往治疗分层展示长期生存结果，为化疗后复发卵巢癌免疫治疗的重要长期随访数据。涉及公司/机构：Agenus 信息来源：Agenus官网07创新药酶替代疗法BioMarin POMBILITI+OPFOLDA晚发型庞贝病5年3期数据（WMS） PROPEL开放标签延伸研究5年分析（82例持续接受POMBILITI+OPFOLDA治疗的患者）显示持久运动功能获益与肺功能相对稳定，支持cipaglucosidase alfa+miglustat联合方案在晚发型庞贝病中的长期疗效与安全性。涉及公司/机构：BioMarin 信息来源：BioMarin官网08细胞与基因治疗CAR-TCRISPR Therapeutics将携zugo-cel（CRISPR编辑同种异体CAR-T）自免1期数据亮相ACR 2026 CRISPR/Cas9基因编辑的同种异体CD19 CAR-T疗法zugo-cel（原CTX112）在自身免疫病中的1期临床数据将在ACR Convergence 2026以poster形式展示。zugo-cel无需HLA配型、现货型设计，正在系统性红斑狼疮、系统性硬化症、炎症性肌病等适应症中推进。涉及公司/机构：CRISPR Therapeutics 信息来源：CRISPR Therapeutics官网09BD/授权酶替代疗法Italfarmaco与JCR达成JR-141（Hunter综合征）美欧拉美独家授权 JCR授予Italfarmaco其JR-141（pabinafusp alfa，基于J-Brain Cargo®技术的脑穿透酶替代疗法）在美国、欧洲、拉丁美洲获批后的独家开发与商业化权益。JR-141正开展全球III期研究（NCT04573023），用于治疗黏多糖贮积症II型（Hunter综合征）的神经病变与体细胞症状。涉及公司/机构：Italfarmaco、JCR Pharmaceuticals 信息来源：Morningstar（Business Wire）｜ JCR Pharmaceuticals官网10监管政策CDE：9月28日—10月3日12款1类创新药首次获批临床根据CDE官网与公开资料，本周（9月28日—10月3日）12款1类创新药首次在中国获得临床试验默示许可，涵盖小分子、抗体、寡核苷酸疗法、细胞疗法等；其中包括Atalanta Therapeutics申报的ATL-101鞘内注射液等。国产创新药临床申报持续活跃。涉及公司/机构：CDE及各申报企业信息来源：医药观澜/新浪财经今日要点总结 ADC重磅数据：Genmab Rina-S铂耐药卵巢癌ORR 45.9%、mDOR 12.1个月，且不依赖FRα表达水平，为卵巢癌ADC治疗再添强证据。自免与肿瘤免疫双线进展：Biogen litifilimab皮肤型红斑狼疮52周持久清除；Agenus免疫组合卵巢癌48%估计3年生存率。中国创新药出海与新技术：Zipalertinib一线NDA进入FDA RTOR通道；中美瑞康首个saRNA激活utrophin治疗DMD人体证据（并披露超3100万美元新一轮融资）。酶替代与疫苗稳步推进：BioMarin庞贝病5年数据、ILiAD百日咳疫苗儿科2b期发表于Lancet ID、Italfarmaco/JCR达成Hunter综合征授权。 CGT与监管：CRISPR同种异体CAR-T自免数据将亮相ACR；CDE本周12款1类新药获批临床。注：本文信息均来自公开渠道整理，每条均标注实际发布日期，仅供行业参考，不构成任何投资建议。完整原文链接可点击底部「阅读原文」查看。

突破性疗法临床2期临床3期并购疫苗

2026-10-03

·生物制品圈

鼻喷百日咳疫苗首次儿童试验阳性：BPZE1到底走通了哪一步？

一项在6—17岁儿童中开展的2b期国际多中心试验显示：减毒活鼻喷百日咳疫苗BPZE1单次鼻腔给药后，呼吸道黏膜显著产生分泌型IgA；与常规Tdap加强针联用互不干扰；全程无疫苗相关严重不良事件。这是BPZE1首次在儿童人群中完成的对照试验，由英国南安普顿大学牵头，联合澳大利亚、哥斯达黎加共16个临床研究中心完成，试验代号SUPER（Stand Up to Pertussis）。01 疫苗覆盖率已经很高，百日咳为什么还在暴发？百日咳由百日咳博德特菌（Bordetella pertussis）引起，传染性强，婴幼儿感染后可出现呼吸暂停、肺炎甚至死亡。在多数国家，儿童全程接种率已达90%以上，但近十多年来，美、英、澳等国仍反复出现暴发，青少年和成人病例占比持续上升。原因不在"没打"，而在于—— 现有注射用百日咳疫苗主要保护人体远离重症，却不能阻止细菌在鼻咽部定植和传播。目前使用的无细胞疫苗（Tdap等）诱导的是血液里的IgG抗体，能中和细菌毒素，却不在呼吸道黏膜表面建立第一道防线。接种者仍可能无症状带菌，成为社区里的隐性传播源。换句话说，现有疫苗让人"不生病"，但没有让人"不带菌"。这正是新一代百日咳疫苗想补上的缺口。02 BPZE1是什么？为什么做成鼻喷？ BPZE1是一种减毒活百日咳疫苗，由野生百日咳博德特菌敲除三个主要毒素基因（皮肤坏死毒素、气管细胞毒素、百日咳毒素）后获得，以单次鼻腔喷雾给药，模拟细菌自然感染呼吸道的过程，但毒力已被削弱到不会致病。研发思路很直接：既然细菌从鼻腔钻进来，就把疫苗也送到鼻腔去。黏膜局部免疫的核心武器是分泌型IgA（S-IgA）——一种覆盖在呼吸道、消化道黏膜表面的抗体，能在细菌黏附上呼吸道上皮之前就把它拦下。2025年公布的成人CHAMPION-1人体挑战研究已经提示：BPZE1可以减少细菌在鼻咽部定植。但此前儿童数据完全空白。已经打过无细胞疫苗的学龄儿童，鼻腔里还能不能被BPZE1"重新教会"产生S-IgA？和常规Tdap一起打，会不会互相干扰？——这次2b期试验，就是来回答这两个问题的。03 试验怎么做的？三组人，单次鼻喷这是一项多中心、随机、安慰剂对照、同时设主动对照的2b期试验。2021年11月至2024年5月，共筛查368名6—17岁健康儿童，其中366名接受接种，按1:1:1随机分组： · BPZE1组（n=123）：鼻喷BPZE1 + 肌注安慰剂 · BPZE1+Tdap组（n=121）：鼻喷BPZE1 + 肌注Tdap加强针 · Tdap组（n=122）：鼻喷安慰剂 + 肌注Tdap 所有儿童此前均已按程序完成无细胞百日咳疫苗基础免疫。主要免疫原性终点：接种后第29天，鼻分泌物中抗百日咳全菌提取物（WCE）的S-IgA几何平均倍数升高（GMFR）。04 关键数据：鼻子里的抗体涨了多少？结果非常直接—— · BPZE1单喷组：抗WCE鼻S-IgA 升高 3.8倍（95%CI 3.1—4.7） · BPZE1+Tdap联合组：升高 3.5倍（95%CI 2.9—4.3） · Tdap单针组：仅升高 1.1倍（95%CI 0.9—1.4），几乎无变化两个BPZE1组的95%置信区间下限都大于1，正式达到预设的主要终点。也就是说，无论是否同时打Tdap，单次鼻喷BPZE1都能在鼻腔黏膜诱导出显著的S-IgA反应。针对丝状血凝素（FHA）、黏着素（pertactin）、菌毛（Fim2/3）和百日咳毒素（PT）的鼻S-IgA也都出现有统计学意义的升高。亚组的血清杀菌活性（SBA）检测中，BPZE1单喷组约60%的受试者SBA滴度较基线升高至少4倍，而Tdap单针组约为20%。另一个被专门检验的问题是"互相打架"：BPZE1是否会削弱Tdap加强针针对破伤风、白喉、百日咳的血清IgG应答？结果显示—— BPZE1与Tdap联用后，Tdap诱导的血清IgG水平与单独接种Tdap相比达到了非劣效标准。BPZE1没有"抢戏"。 ▲ 主要免疫原性结果对照表。05 安全性：孩子耐受得怎么样？整体耐受性良好。整个试验没有报告与疫苗相关的严重不良事件，也没有出现因不良反应而终止试验的病例。接种后7天内的反应以轻、中度为主，通常1—3天内自行缓解。BPZE1鼻喷组最常见的是与鼻喷装置直接相关的症状：鼻充血或流涕（约48%—52%）、打喷嚏（约25%—32%）、头痛（约22%—35%）。Tdap注射相关反应主要是注射部位疼痛（约37%—41%）和压痛（约50%—58%）。联合接种组是两组反应的叠加，没有出现新的、意外的安全信号。研究期间共报告3例严重不良事件（1例哮喘加重、1例女性胃肠炎、1例16岁女性呼吸困难），独立数据安全监察委员会判断均与疫苗或鼻喷装置无关。 ▲ 接种后7天内主动监测的鼻/呼吸道、全身和注射部位不良反应发生率。06 这一步意味着什么？边界也要看清把这次儿童2b期结果，和2025年公布的CHAMPION-1成人人体挑战研究放在一起看，BPZE1的研发路径正在逐步清晰： · 在成人中，已被证明可减少细菌在鼻咽部定植 · 在6—17岁已接种过无细胞疫苗的儿童中，可在呼吸道黏膜重新唤起显著S-IgA · 可与常规Tdap加强针联用，互不干扰 · 单次鼻喷给药，儿童耐受性良好这意味着BPZE1有潜力成为一种"阻断传播型"疫苗——不仅保护接种者本人，还可能通过减少鼻咽带菌，降低家庭和社区内的传播链，间接保护最脆弱的小婴儿。但作为长期跟踪疫苗研发的观察者，有四个边界必须讲清楚： ① 这是2b期免疫原性试验，不是3期效力试验。终点是"抗体涨了多少"，不是"真实世界里少生了多少病人"。鼻S-IgA与保护力之间的相关性，仍缺大规模真实数据锁定。 ② 黏膜抗体持久性有限。鼻S-IgA在第29天达峰后逐渐下降，到第169天左右接近基线。是否需要加强、多久加强一次，目前没有答案。 ③ 试验对象是6岁以上、已完成无细胞疫苗基础免疫的健康儿童。能否用于6月龄以下、最易重症的婴儿，以及免疫低下人群，这次试验没有回答。 ④ 黏膜定植减少能否真正转化为社区传播下降，仍需等待后期试验。南安普顿团队也明确表示将继续推进后期临床试验。写在最后百日咳疫苗的下一个十年，竞争焦点已经从"防不防重症"转向"能不能同时阻断传播"。BPZE1这次在儿童身上走通的，是把鼻喷黏膜免疫从动物实验、成人挑战试验，推进到了学龄儿童这一真实传播人群——这一步不大，但方向是对的。对普通读者来说，现阶段最实际的信息仍然是：按国家免疫规划完成儿童百日咳基础免疫和青少年、成人Tdap加强针，仍然是预防百日咳最可靠的手段。 BPZE1距离上市还有3期试验和审批的距离，不必现在就把它当作"即将到来的新选择"。但它的出现，让我们看到疫苗学正在认真对待一个老问题——为什么不能在细菌进门的地方，就把它拦住。 — 完 — 你怎么看"鼻喷疫苗"这种思路？欢迎在评论区聊聊。如果觉得有用，欢迎转发给身边有孩子的朋友。参考来源： 1. Faust SN, Cevas J, Avila L, et al. Immunogenicity and safety of BPZE1, an intranasal live attenuated pertussis vaccine, evaluated with and without tetanus-reduced diphtheria-acellular pertussis vaccine in healthy children aged 6–17 years: a randomised, active-controlled and placebo-controlled, phase 2b trial. The Lancet Infectious Diseases, 2026-10-01 在线发表. DOI: 10.1016/S1473-3099(26)00470-6. 临床试验注册号：NCT05116241。 2. University Hospital Southampton / NIHR Clinical Research Facility 官方新闻稿：First children's study of nasal whooping cough vaccine reports positive results, 2026-10-02. 链接：https://research.uhs.nhs.uk/news/first-childrens-study-of-nasal-whooping-cough-vaccine-reports-positive-results 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不本站。

100 项与 Pertussis vaccine(INSERM) 相关的药物交易

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KEGG	Wiki	ATC	Drug Bank
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研发状态

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适应症	最高研发状态	国家/地区	公司	日期
百日咳	临床2期	美国	National Institute of Allergy & Infectious Diseases	2018-10-23

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适应症

分期

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研究	分期	人群特征	评价人数	分组	结果	评价	发布日期


NCT05116241 (Pubmed \| Lancet Infect Dis)	临床2期	-	366	BPZE1	醖衊壓繭膚齋網襯鏇壓(網製廠選構齋鹽觸鏇獵) = 範襯積窪構簾鹹願壓範壓築鏇遞衊簾餘夢網鑰 (願範廠壓鹹夢餘製憲鹹 )	积极	2026-10-01
				BPZE1 + Tdap	醖衊壓繭膚齋網襯鏇壓(網製廠選構齋鹽觸鏇獵) = 願窪鑰獵鏇遞觸鹽蓋繭壓築鏇遞衊簾餘夢網鑰 (願範廠壓鹹夢餘製憲鹹 )
NCT05136599 (CTgov)	临床1期	百日咳	79	Bordetella pertussis D420 (Dose #3)	艱窪壓鏇構鏇艱網鏇餘 = 壓淵餘獵遞糧網鑰範淵夢壓醖願顧蓋簾顧鑰蓋 (觸廠網夢鑰積製積壓鹹, 夢襯餘艱鬱廠齋膚築獵 ~ 鹽夢顧顧廠願憲憲襯築) 更多	-	2026-06-22
				Bordetella pertussis D420 (Dose #5)	艱窪壓鏇構鏇艱網鏇餘 = 襯顧夢餘衊艱艱廠鬱襯夢壓醖願顧蓋簾顧鑰蓋 (觸廠網夢鑰積製積壓鹹, 範鏇繭願構艱簾鏇顧蓋 ~ 艱築積鏇鑰鑰積廠醖選) 更多
NCT05461131 (CHAMPION-1, Pubmed \| Lancet Microbe) 人工标引	临床2期	百日咳	53	BPZE1 (intranasal 10⁹ CFU)	廠夢壓艱觸壓選夢壓窪(衊廠窪簾鏇壓齋壓壓簾) = 醖範鑰繭夢選齋鑰顧鹽壓網觸築製憲壓壓衊網 (選願願餘鏇選淵構糧齋, 39 ~ 76) 更多	积极	2025-12-01
				Placebo	廠夢壓艱觸壓選夢壓窪(衊廠窪簾鏇壓齋壓壓簾) = 夢襯壓鏇壓蓋窪網鹹築壓網觸築製憲壓壓衊網 (選願願餘鏇選淵構糧齋, 17 ~ 55) 更多
NCT05116241 (SUPER, CTgov)	临床2期	百日咳	368	Placebo+Boostrix	蓋廠艱繭鬱醖淵淵繭醖(範糧鹹製衊蓋網範築築) = 憲構壓獵製範淵蓋網壓衊鏇願鬱積夢選願廠繭 (選醖糧襯網襯繭積壓範, 淵襯築積鹹窪築構積選 ~ 鹽艱網獵膚齋獵壓鬱顧) 更多	-	2025-11-06
NCT05461131 (CHAMPION-1, CTgov)	临床2期	百日咳	53	Bordetella Pertussis Challenge Strain+Azithromycin+BPZE1 (BPZE1)	網遞鹽憲積醖鬱夢鹹衊 = 壓遞艱獵觸齋構築鑰顧鹹願顧鏇簾廠鏇襯遞廠 (衊選獵醖鬱鬱壓簾選憲, 襯遞選壓繭壓夢廠襯獵 ~ 獵鹹簾網鹹顧觸鹽鏇鹽) 更多	-	2025-03-12
				Bordetella Pertussis Challenge Strain+Azithromycin (Placebo)	網遞鹽憲積醖鬱夢鹹衊 = 淵選醖繭願艱鏇夢蓋構鹹願顧鏇簾廠鏇襯遞廠 (衊選獵醖鬱鬱壓簾選憲, 積鹹構簾餘糧繭繭夢鏇 ~ 構齋衊範築網壓觸鹹壓) 更多
NCT05116241 (SUPER, Biospace) 人工标引	临床2期	百日咳	366	BPZE1	膚製蓋廠構鹹齋醖鬱築(構繭艱艱廠齋構網選廠) = Minimal reactogenicity was observed across all groups in this pediatric study, consistent with the favorable safety profile previously established in 5 adult clinical trials. 衊窪餘壓遞憲餘觸遞鬱 (憲簾鏇獵壓鹹鏇壓鏇廠 )	积极	2024-05-28
				BPZE1+Tdap
NCT03942406 (CTgov)	临床2期	百日咳	300	BPZE1 pertussis vaccine and VaxINator(TM) Atomization Device (BPZE1 Intranasal Prime, BPZE1 Boost)	鏇鹽夢獵繭醖壓醖蓋鹽 = 鹹鏇餘築觸繭範憲願襯遞夢淵範鑰鏇顧觸鹹糧 (遞選憲夢築鏇觸鹽襯壓, 鹽鹹鏇遞積簾蓋鏇願遞 ~ 壓簾膚糧繭憲廠願淵遞) 更多	-	2023-06-27
				BPZE1 pertussis vaccine and VaxINator(TM) Atomization Device (BPZE1 Intranasal Prime, Placebo Boost)	鏇鹽夢獵繭醖壓醖蓋鹽 = 壓鏇窪選淵獵鬱淵艱齋遞夢淵範鑰鏇顧觸鹹糧 (遞選憲夢築鏇觸鹽襯壓, 衊願鬱獵製蓋繭餘窪構 ~ 鑰鹽窪製餘艱繭壓築製) 更多
NCT03541499 (CTgov)	临床2期	百日咳	50	BPZE1 (BPZE1 10^7 CFU by VaxINator)	繭觸製遞選餘繭淵廠壓 = 膚鑰憲築窪構淵構餘艱淵構醖醖繭鹽網簾艱憲 (窪製遞醖觸鏇糧憲鹽願, 醖窪鬱憲淵醖鬱蓋築構 ~ 構選鏇蓋窪餘簾廠繭繭) 更多	-	2022-02-03
				BPZE1 (BPZE1 10^9 CFU by VaxINator)	繭觸製遞選餘繭淵廠壓 = 襯鬱鹹網蓋餘醖膚壓鹽淵構醖醖繭鹽網簾艱憲 (窪製遞醖觸鏇糧憲鹽願, 夢糧淵積壓醖餘鹽獵淵 ~ 網築積積淵積壓網窪糧) 更多
NCT02453048 (Pubmed \| Lancet Infect Dis) 人工标引	临床1期	-	48	BPZE1	醖積衊遞網選窪衊觸網(構鬱齋簾夢膚製簾餘網) = 鹹繭窪遞淵醖壓範製膚膚糧鹽鹹顧觸襯網襯網 (鬱鑰簾遞蓋鏇襯壓衊獵 ) 更多	积极	2020-11-01
				Placebo	憲願願觸網鏇窪鹹壓選(壓顧簾蓋顧鹹鑰製積遞) = 醖餘蓋鏇衊壓廠鏇積構艱夢衊艱餘憲醖襯獵廠 (範觸膚積網積繭鏇衊壓 )