▎藥明康德

本期看點

1. 單抗療法briquilimab在針對慢性自發(fā)性蕁麻疹患者開展的1b/2a期研究中表現(xiàn)亮眼，兩個單劑量隊列中有89%的患者達(dá)成完全緩解（CR）。2. 噬菌體療法BX004用于治療囊性纖維化（CF）患者因銅綠假單胞菌引起的慢性肺部感染，在一項早期臨床試驗中與安慰劑相比顯著降低了細(xì)菌數(shù)量約500倍，無耐藥發(fā)生，同時有效保護(hù)了健康微生物組。

Briquilimab：公布1b/2a期臨床試驗數(shù)據(jù)

Jasper Therapeutics公司公布了其候選單抗briquilimab用于治療慢性自發(fā)性蕁麻疹的1b/2a期研究BEACON的初步數(shù)據(jù)。Briquilimab是一種非糖基化單克隆抗體，可通過靶向細(xì)胞表面受體c-Kit（也稱為CD117）以避免與干細(xì)胞因子結(jié)合，從而阻斷肥大細(xì)胞中的關(guān)鍵生存信號，造成細(xì)胞凋亡與耗竭，從而消除肥大細(xì)胞驅(qū)動疾病（如慢性蕁麻疹）中炎癥反應(yīng)的潛在來源。

此次公布的結(jié)果顯示，briquilimab在240 mg和360 mg單劑量隊列中表現(xiàn)出深度且快速的疾病控制效果，兩組共9名受試者中有8人（89%）實現(xiàn)了CR，7人（78%）在第2周即達(dá)到臨床緩解。此外，在BEACON研究中完成治療并轉(zhuǎn)入開放標(biāo)簽擴(kuò)展研究、接受每8周180 mg劑量治療的受試者中，也顯示出強(qiáng)大的臨床療效：在第12周時，11名受試者中有8人（73%）達(dá)到了CR。安全性方面，研究期間未報告任何≥3級的治療相關(guān)不良事件。

BX004：公布1b/2a期臨床試驗數(shù)據(jù)

BiomX公司宣布，其噬菌體療法BX004的首個人體試驗結(jié)果已在學(xué)術(shù)期刊Nature Communications上發(fā)表。BX004是一種固定的多噬菌體組合療法，用于治療囊性纖維化患者因銅綠假單胞菌引起的慢性肺部感染。銅綠假單胞菌是導(dǎo)致CF患者發(fā)病和死亡的主要原因之一。

論文中的數(shù)據(jù)表明，在針對抗生素耐藥性銅綠假單胞菌感染的1b/2a期臨床試驗中，BX004的安全性和耐受性良好，在所有患者中和所有測試劑量水平下均未發(fā)生治療相關(guān)安全事件。與接受安慰劑治療的患者相比，接受BX004治療患者的細(xì)菌數(shù)量下降了2.7 log??（約500倍），且未出現(xiàn)耐藥現(xiàn)象，同時有效保護(hù)了患者的健康微生物組。該公司正在推進(jìn)BX004的2b期臨床試驗，預(yù)計將于2026年第一季度獲得頂線結(jié)果。

Paxalisib：公布1b期聯(lián)合治療試驗中首例患者的數(shù)據(jù)

Kazia Therapeutics公司公布了一項1b期臨床試驗中首例患者的初步結(jié)果，該研究旨在評估其可穿越血腦屏障的在研口服PI3K/Akt/mTOR通路抑制劑paxalisib聯(lián)用帕博利珠單抗和標(biāo)準(zhǔn)化療的療效與安全性。該公司于2016年底從基因泰克（Genentech）獲得了paxalisib的許可。該療法此前已獲得多項資格，包括美國FDA授予的針對膠質(zhì)母細(xì)胞瘤的孤兒藥資格和快速通道資格（FTD），針對攜帶PI3K通路突變、需聯(lián)合放療治療的實體瘤腦轉(zhuǎn)移適應(yīng)癥的FTD，以及針對彌漫性內(nèi)生性橋腦膠質(zhì)瘤（DIPG）和非典型畸胎樣/橫紋肌樣腫瘤的孤兒藥資格與罕見兒科疾病認(rèn)定。

此次公布的結(jié)果顯示，一名61歲、腫瘤已局部轉(zhuǎn)移至左肺上葉的三陰性乳腺癌女性患者在接受治療僅21天后，其循環(huán)腫瘤細(xì)胞（CTCs）減少了超過50%，且CTC細(xì)胞簇也顯著減少。這些早期臨床數(shù)據(jù)與此前公布的臨床前數(shù)據(jù)一致。

ELU42：IND申請獲得FDA許可

Eluciderm公司宣布，美國FDA已批準(zhǔn)其針對新藥ELU42的IND申請，可開展一項針對糖尿病足潰瘍（DFUs）患者的1/2a期開放標(biāo)簽研究，以評估ELU42的安全性和療效。ELU42是一款新型外用小分子Wnt通路調(diào)節(jié)劑，具有抑菌特性，旨在治療慢性開放性傷口。

參考資料（可上下滑動查看）

[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

[5] Sensorium Therapeutics Receives FDA IND Clearance for SNTX-2643, a First-in-Class, Rapid-Acting Anxiolytic. Retrieved July 11, 2025, from https://www.prnewswire.com/news-releases/sensorium-therapeutics-receives-fda-ind-clearance-for-sntx2643-a-firstinclass-rapidacting-anxiolytic-302499309.html

[6] BiomX Announces Publication in Nature Communications of Phage Cocktail BX004 Phase 1b/2a Part 1 Data Demonstrating Strong Activity in Cystic Fibrosis. Retrieved July 11, 2025, from https://www.globenewswire.com/news-release/2025/07/08/3111723/0/en/BiomX-Announces-Publication-in-Nature-Communications-of-Phage-Cocktail-BX004-Phase-1b-2a-Part-1-Data-Demonstrating-Strong-Activity-in-Cystic-Fibrosis.html

[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

[8] Avenzo Therapeutics Announces First Patient Dosed in Phase 1/2 Clinical Study of AVZO-1418, a Potential Best-in-Class EGFR/HER3 Bispecific Antibody-Drug Conjugate. Retrieved July 11, 2025, from https://avenzotx.com/press-releases/avenzo-therapeutics-announces-first-patient-dosed-in-phase-1-2-clinical-study-of-avzo-1418-a-potential-best-in-class-egfr-her3-bispecific-antibody-drug-conjugate/

[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

[10] Jasper Therapeutics Reports Clinical Data Update from Briquilimab Studies in Chronic Spontaneous Urticaria. Retrieved July 11, 2025, from https://ir.jaspertherapeutics.com/news-releases/news-release-details/jasper-therapeutics-reports-clinical-data-update-briquilimab#:~:text=REDWOOD%20CITY%2C%20Calif.%2C%20July%2007%2C%202025%20%28GLOBE%20NEWSWIRE%29,CSU%20and%20providing%20an%20update%20on%20the%20program.

[11] Kazia Therapeutics Reports Early Efficacy Data from First Triple-Negative Breast Cancer Patient Receiving Paxalisib Combination Regimen achieving >50% Reduction in Circulating Tumor Cells in Phase 1b Trial. Retrieved July 11, 2025, from https://www.prnewswire.com/news-releases/kazia-therapeutics-reports-early-efficacy-data-from-first-triple-negative-breast-cancer-patient-receiving-paxalisib-combination-regimen-achieving-50-reduction-in-circulating-tumor-cells-in-phase-1b-trial-302500778.html

[12] The Institute for Research in Immunology and Cancer (IRIC) of the Université de Montréal and IRICoR Announce initiation of Phase 1 Trial of Licensed Small Molecule Therapy for Solid Tumors. Retrieved July 11, 2025, from https://www.prnewswire.com/news-releases/the-institute-for-research-in-immunology-and-cancer-iric-of-the-universite-de-montreal-and-iricor-announce-initiation-of-phase-1-trial-of-licensed-small-molecule-therapy-for-solid-tumors-302499345.html

[13] Neumora Therapeutics Announces Initiation of Phase 1 Clinical Study of M4 Positive Allosteric Modulator NMRA-861. Retrieved July 11, 2025, from https://ir.neumoratx.com/news-releases/news-release-details/neumora-therapeutics-announces-initiation-phase-1-clinical-study

[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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網(wǎng)址: 兩周見效，近90%患者獲完全緩解的單抗療法；使致病菌數(shù)量下降約500倍的噬菌體療法…… http://m.u1s5d6.cn/newsview1552752.html