两周见效，近90%患者获完全缓解的单抗疗法；使致病菌数量下降约500倍的噬菌体疗法…… | 一周盘点

▎药明康德

本期看点

1. 单抗疗法briquilimab在针对慢性自发性荨麻疹患者开展的1b/2a期研究中表现亮眼，两个单剂量队列中有89%的患者达成完全缓解（CR）。

2. 噬菌体疗法BX004用于治疗囊性纤维化（CF）患者因铜绿假单胞菌引起的慢性肺部感染，在一项早期临床试验中与安慰剂相比显著降低了细菌数量约500倍，无耐药发生，同时有效保护了健康微生物组。

Briquilimab：公布1b/2a期临床试验数据

Jasper Therapeutics公司公布了其候选单抗briquilimab用于治疗慢性自发性荨麻疹的1b/2a期研究BEACON的初步数据。Briquilimab是一种非糖基化单克隆抗体，可通过靶向细胞表面受体c-Kit（也称为CD117）以避免与干细胞因子结合，从而阻断肥大细胞中的关键生存信号，造成细胞凋亡与耗竭，从而消除肥大细胞驱动疾病（如慢性荨麻疹）中炎症反应的潜在来源。

此次公布的结果显示，briquilimab在240 mg和360 mg单剂量队列中表现出深度且快速的疾病控制效果，两组共9名受试者中有8人（89%）实现了CR，7人（78%）在第2周即达到临床缓解。此外，在BEACON研究中完成治疗并转入开放标签扩展研究、接受每8周180 mg剂量治疗的受试者中，也显示出强大的临床疗效：在第12周时，11名受试者中有8人（73%）达到了CR。安全性方面，研究期间未报告任何≥3级的治疗相关不良事件。

BX004：公布1b/2a期临床试验数据

BiomX公司宣布，其噬菌体疗法BX004的首个人体试验结果已在学术期刊

Nature Communications

论文中的数据表明，在针对抗生素耐药性铜绿假单胞菌感染的1b/2a期临床试验中，BX004的安全性和耐受性良好，在所有患者中和所有测试剂量水平下均未发生治疗相关安全事件。与接受安慰剂治疗的患者相比，接受BX004治疗患者的细菌数量下降了2.7 log₁₀（约500倍），且未出现耐药现象，同时有效保护了患者的健康微生物组。该公司正在推进BX004的2b期临床试验，预计将于2026年第一季度获得顶线结果。

Paxalisib：公布1b期联合治疗试验中首例患者的数据

Kazia Therapeutics公司公布了一项1b期临床试验中首例患者的初步结果，该研究旨在评估其可穿越血脑屏障的在研口服PI3K/Akt/mTOR通路抑制剂paxalisib联用帕博利珠单抗和标准化疗的疗效与安全性。该公司于2016年底从基因泰克（Genentech）获得了paxalisib的许可。该疗法此前已获得多项资格，包括美国FDA授予的针对胶质母细胞瘤的孤儿药资格和快速通道资格（FTD），针对携带PI3K通路突变、需联合放疗治疗的实体瘤脑转移适应症的FTD，以及针对弥漫性内生性桥脑胶质瘤（DIPG）和非典型畸胎样/横纹肌样肿瘤的孤儿药资格与罕见儿科疾病认定。

此次公布的结果显示，一名61岁、肿瘤已局部转移至左肺上叶的三阴性乳腺癌女性患者在接受治疗仅21天后，其循环肿瘤细胞（CTCs）减少了超过50%，且CTC细胞簇也显著减少。这些早期临床数据与此前公布的临床前数据一致。

ELU42：IND申请获得FDA许可

Eluciderm公司宣布，美国FDA已批准其针对新药ELU42的IND申请，可开展一项针对糖尿病足溃疡（DFUs）患者的1/2a期开放标签研究，以评估ELU42的安全性和疗效。ELU42是一款新型外用小分子Wnt通路调节剂，具有抑菌特性，旨在治疗慢性开放性伤口。

参考资料（可上下滑动查看）

[1] Eleva administers first dose of its Factor H biological treatment in C3-Glomerulopathy to healthy volunteers. Retrieved July 11, 2025, from https://elevabiologics.com/eleva-administers-first-dose-of-its-factor-h-biological-treatment-in-c3-glomerulopathy-to-healthy-volunteers/

[2] First patient dosed in the Phase I clinical trial of PulseSight Therapeutics’ PST-611 treatment for dry AMD/Geographic Atrophy. Retrieved July 11, 2025, from https://pulsesight.com/2025/07/07/first-patient-dosed-in-the-phase-i-clinical-trial-of-pulsesight-therapeutics-pst-611-treatment-for-dry-amd-geographic-atrophy/

[3] Eluciderm, Inc. Announces U.S. FDA Clearance of IND Application for ELU42. Retrieved July 11, 2025, from https://www.businesswire.com/news/home/20250708620364/en/Eluciderm-Inc.-Announces-U.S.-FDA-Clearance-of-IND-Application-for-ELU42

[4] Solid Biosciences Announces FDA IND and Health Canada CTA Approval for First-in-Class Cardiac Gene Therapy to Treat Catecholaminergic Polymorphic Ventricular Tachycardia (CPVT). Retrieved July 11, 2025, from https://www.globenewswire.com/news-release/2025/07/08/3112195/0/en/Solid-Biosciences-Announces-FDA-IND-and-Health-Canada-CTA-Approval-for-First-in-Class-Cardiac-Gene-Therapy-to-Treat-Catecholaminergic-Polymorphic-Ventricular-Tachycardia-CPVT.html

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[7] Purespring Therapeutics announce FDA IND clearance for Phase I/II clinical trial for primary IgA nephropathy (IgAN). Retrieved July 11, 2025, from https://www.globenewswire.com/news-release/2025/07/09/3112303/0/en/Purespring-Therapeutics-announce-FDA-IND-clearance-for-Phase-I-II-clinical-trial-for-primary-IgA-nephropathy-IgAN.html

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[9] BlueRock Therapeutics announces first patient receives investigational therapy in Phase 1/2a clinical trial of OpCT-001 for the treatment of primary photoreceptor diseases. Retrieved July 11, 2025, from https://www.bayer.com/en/us/news-stories/treatment-of-primary-photoreceptor-diseases-0

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[14] Cocrystal Pharma to Present Data from Phase 1 Study of First-in-Class Norovirus Protease Inhibitor CDI-988 at the 9th International Calicivirus Conference. Retrieved July 11, 2025, from https://www.globenewswire.com/news-release/2025/07/10/3113201/0/en/Cocrystal-Pharma-to-Present-Data-from-Phase-1-Study-of-First-in-Class-Norovirus-Protease-Inhibitor-CDI-988-at-the-9th-International-Calicivirus-Conference.html

[15] Krystal Biotech Announces First Patient Dosed in Phase 1/2 Trial of KB801 for the Treatment of Neurotrophic Keratitis. Retrieved July 11, 2025, from https://www.globenewswire.com/news-release/2025/07/09/3112425/0/en/Krystal-Biotech-Announces-First-Patient-Dosed-in-Phase-1-2-Trial-of-KB801-for-the-Treatment-of-Neurotrophic-Keratitis.html

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