Efzofitimod

每日呼吸病学全球动态本专栏汇总过去24小时全球呼吸领域前沿资讯，聚焦临床研究、药物获批、指南更新与学术会议重磅成果，供临床及学术参考，内容仅作信息交流，不构成诊疗建议。1. 依沃西（PD-1/VEGF双抗）HARMONi-2研究期中分析OS显著阳性，头对头击败帕博利珠单抗核心内容：2026年9月3日，康方生物（9926.HK）宣布，其全球首创PD-1/VEGF双特异性抗体依沃西（商品名：依达方®，AK112）对比帕博利珠单抗（K药），一线治疗PD-L1表达阳性（TPS≥1%）局部晚期或转移性非小细胞肺癌（NSCLC）的随机、双盲、多中心注册性III期HARMONi-2研究，期中分析达到总生存期（OS）关键次要终点，OS结果具有统计学显著性和临床意义。该研究此前已达到无进展生存期（PFS）主要终点。重要性理由：这是全球首个PD-1/VEGF双抗在头对头III期试验中证实OS优于标准PD-1单抗的研究，可能重塑晚期NSCLC一线免疫治疗格局。依沃西作为中国原创双抗药物，其全球多中心III期数据阳性标志着国产肿瘤免疫药物从”跟随”走向”引领”，对临床实践和全球肺癌治疗市场均有深远影响。信息来源：康方生物官方公告 / 东方财富网（2026-09-03发布）链接：https://caifuhao.eastmoney.com/news/202609030503304589220902. 阿斯利康完成舒沃替尼全球许可协议，EGFR exon20ins靶向药加速全球化核心内容：2026年9月1日，阿斯利康宣布完成与迪哲医药的全球许可协议，获得口服EGFR抑制剂ZEGFROVY®（舒沃替尼，sunvozertinib）的全球开发和商业化权益。该药物用于一线治疗EGFR外显子20插入突变（exon20ins）NSCLC的补充新药申请（sNDA）已获美国FDA受理，基于全球多中心WU-KONG28 III期试验的阳性结果，该数据曾在2026年ASCO年会上作为Late-Breaking口头报告公布。重要性理由：EGFR exon20ins约占NSCLC的2-3%，传统EGFR-TKI疗效差，长期缺乏有效靶向治疗。舒沃替尼是首个由中国企业自主研发并实现全球权益授权的EGFR exon20ins高选择性抑制剂，阿斯利康的全球商业化能力将显著加速该药物在全球范围内的可及性。一线治疗适应症若获FDA批准，将成为该亚型患者的新标准治疗。信息来源：1stOncology / 阿斯利康官方公告（2026-09-01发布）链接：https://www.1stoncology.com/blog/astrazeneca-completes-global-license-agreement-for-oral-egfr-inhibitor-zegfrovy-sunvozertinib-for-lung-cancer1234670507/3. FDA公开Depemokimab（Exdensur）REMS评估文件，新型长效IL-5抑制剂治疗重度哮喘核心内容：美国FDA公开了Depemokimab（商品名Exdensur）的风险评估和缓解策略（REMS）评估文件。该药物是一种新型长效IL-5单克隆抗体，用于治疗重度嗜酸性粒细胞性哮喘。其上市申请基于两项关键52周全球多中心III期试验（SWIFT-1 [NCT04719832]和SWIFT-2 [NCT04718103]），采用随机、双盲、平行组、安慰剂对照设计，受试者按2:1随机接受depemokimab 100mg或匹配安慰剂。重要性理由：目前已获批的哮喘IL-5抑制剂（美泊利珠单抗、瑞利珠单抗、贝那利珠单抗）均需每4-8周给药一次，Depemokimab作为长效IL-5抑制剂，有望实现每6个月一次的超长效给药，显著提升患者依从性。若获批，将为重度嗜酸性粒细胞性哮喘提供更便捷的生物制剂选择，可能改变哮喘生物制剂的给药模式。信息来源：FDA Access Data（REMS评估文件，2026年公开）链接：https://www.accessdata.fda.gov/drugsatfda_docs/nda/2026/761458Orig1s000RiskR.pdf4. COPD生物制剂系统综述与网络Meta分析发表，首次全面比较多种靶向药物疗效核心内容：2026年8月28日，Cureus发表一项系统综述和网络Meta分析，全面评估了靶向2型炎症通路和上皮警报素（alarmin）通路的生物制剂在慢性阻塞性肺疾病（COPD）中的疗效和安全性。研究纳入多项随机对照试验，比较了dupilumab（抗IL-4/IL-13）、mepolizumab（抗IL-5）等不同机制生物制剂在减少COPD急性加重、改善肺功能和生活质量方面的相对疗效。重要性理由：随着dupilumab于2024年成为首个获批的COPD生物制剂，COPD生物制剂治疗进入快速发展期，多种不同机制的药物正在研发中。该网络Meta分析是目前最全面的COPD生物制剂间接比较研究，为临床医生在嗜酸粒细胞性COPD患者中选择生物制剂提供了循证依据，也为后续头对头研究设计提供了参考。信息来源：Cureus（2026-08-28发表，DOI: 10.7759/cureus.115334）链接：https://www.cureus.com/articles/522885-efficacy-and-safety-of-biologic-therapies-targeting-type-2-and-epithelial-alarmin-pathways-in-chronic-obstructive-pulmonary-disease-a-systematic-review-and-network-meta-analysis-of-randomized-controlled-trials5. 睡眠呼吸暂停首个口服药物进入FDA审批程序，有望改变OSA治疗格局核心内容：2026年9月2日报道，一种用于治疗阻塞性睡眠呼吸暂停（OSA）的口服药物已正式进入FDA审批程序。根据与FDA的沟通安排，如果FDA正式受理该申请，预计将于2027年第一季度做出最终审批决定。该药物为睡前口服制剂，旨在通过药物方式改善OSA患者的气道塌陷问题。全球约有10亿人受OSA困扰，目前标准治疗为持续气道正压通气（CPAP），但患者依从性普遍较差。重要性理由：OSA是最常见的睡眠呼吸障碍，与心血管疾病、代谢综合征、认知功能下降等多种并发症密切相关，但治疗手段长期局限于CPAP、口腔矫治器和手术，患者依从性是主要瓶颈。如果口服药物获批，将成为OSA领域首个药物治疗选择，可能彻底改变OSA的治疗模式，显著提升患者治疗可及性和依从性，对公共卫生具有重大意义。信息来源：西雅图韩国日报 / 医药新闻（2026-09-02报道）链接：https://www.seattlekdaily.com/news/articleView.html?idxno=219936. aTyr Pharma更新Efzofitimod治疗肺结节病III期研究FDA监管回应时间核心内容：2026年9月2日，aTyr Pharma宣布，已向FDA提交了efzofitimod治疗伴限制性肺病的肺结节病（pulmonary sarcoidosis）患者的计划中III期研究方案，预计将于2026年9月中旬收到FDA的监管回应。Efzofitimod是一种首创的重组免疫调节蛋白，通过结合细胞表面的神经纤维蛋白（neuropilin-2，NRP2）发挥作用，在II期试验中已显示出改善肺结节病患者肺功能和症状的潜力。重要性理由：肺结节病是一种原因不明的多系统肉芽肿性疾病，肺部受累最常见，严重者可进展为肺纤维化和呼吸衰竭。目前治疗主要依赖全身糖皮质激素和免疫抑制剂，长期使用副作用大，且缺乏针对结节病发病机制的靶向治疗。Efzofitimod作为首创免疫调节蛋白，如果III期成功，将为肺结节病提供首个靶向治疗选择，填补该领域长期未满足的临床需求。

Company anticipates response from FDA by mid-September 2026 on protocol submitted for planned Phase 3 study of efzofitimod in patients with pulmonary sarcoidosis with restrictive lung disease. SAN DIEGO, Sept. 01, 2026 (GLOBE NEWSWIRE) -- aTyr Pharma, Inc. (Nasdaq: ATYR) (“aTyr” or the “Company”), a clinical stage biotechnology company engaged in the discovery and development of first-in-class medicines from its proprietary tRNA synthetase platform, today provided an update on the expected timing of a response from the U.S. Food and Drug Administration (FDA) related to the protocol the Company submitted in June 2026 for a planned Phase 3 study of efzofitimod in patients with chronic, symptomatic pulmonary sarcoidosis with restrictive lung disease. Based on feedback from the FDA, the Company anticipates receiving a response by mid-September 2026. The planned Phase 3 trial is expected to be a global, randomized, double-blind, placebo-controlled study to evaluate the efficacy and safety of efzofitimod in patients with moderate to severe pulmonary sarcoidosis. The 54-week study will consist of two parallel cohorts randomized equally to either 5.0 mg/kg efzofitimod or placebo dosed intravenously once every 3 weeks for a total of 17 doses. The study is intended to enroll up to approximately 372 patients with symptomatic pulmonary sarcoidosis with restrictive lung disease who are receiving a stable dose of ≤ 5.0 mg daily oral corticosteroid and/or a background immunosuppressant. All background treatment will remain stable throughout the duration of the study. The primary endpoint of the study will be change from baseline in forced vital capacity at week 48 and the key secondary endpoint will be change from baseline in the King’s Sarcoidosis Questionnaire-Lung score at week 48. Abo ut Efzofitimod ezstandalone.cmd.push(function () { ezstandalone.showAds(131); }); Efzofitimod is a novel biologic immunomodulator in clinical development for the treatment of interstitial lung disease (ILD), a group of immune-mediated disorders that can cause inflammation and fibrosis, or scarring, of the lungs. Efzofitimod is a tRNA synthetase derived therapy that selectively modulates activated myeloid cells through neuropilin-2 to resolve inflammation without immune suppression and potentially prevent the progression of fibrosis. Efzofitimod is currently being investigated in the Phase 2 EFZO-CONNECT™ study in patients with systemic sclerosis (SSc, or scleroderma)-related ILD, and aTyr recently submitted a protocol to the FDA for a global Phase 3 study of efzofitimod in patients with pulmonary sarcoidosis, a major form of ILD. These forms of ILD have limited therapeutic options and there is a need for safer and more effective, disease-modifying treatments that improve outcomes. About aTyr aTyr is a clinical stage biotechnology company leveraging evolutionary intelligence to translate tRNA synthetase biology into new therapies for fibrosis and inflammation. tRNA synthetases are ancient, essential proteins that have evolved novel domains that regulate diverse pathways extracellularly in humans. aTyr’s discovery platform is focused on unlocking hidden therapeutic intervention points by uncovering signaling pathways driven by its proprietary library of domains derived from all 20 tRNA synthetases. aTyr’s lead therapeutic candidate is efzofitimod, a novel biologic immunomodulator in clinical development for the treatment of interstitial lung disease, a group of immune-mediated disorders that can cause inflammation and progressive fibrosis, or scarring, of the lungs. For more information, please visit . Forward-Looking Statements ezstandalone.cmd.push(function () { ezstandalone.showAds(132); }); This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements are usually identified by the use of words such as "anticipate," “believes,” “can,” “could,” “designed,” “expects,” “intends,” “may,” “plans,” “potential,” “upcoming,” “will,” and variations of such words or similar expressions. We intend these forward-looking statements to be covered by such safe harbor provisions for forward-looking statements and are making this statement for purposes of complying with those safe harbor provisions. These forward-looking statements include, among others, statements regarding our continued development of efzofitimod in pulmonary sarcoidosis; timelines and plans with respect to certain regulatory and development milestones, activities and goals, including the potential receipt of a response from the FDA by mid-September 2026 on a protocol submitted to the FDA in June 2026 for a planned Phase 3 study of efzofitimod in pulmonary sarcoidosis; the proposed design of our planned Phase 3 study of efzofitimod in pulmonary sarcoidosis, including the dosing regimen, enrollment expectations, targeted endpoints, and strategy to focus on a more limited patient population; and our interpretation of the results of the Phase 3 EFZO-FIT™ study and the meaning of those interpretations for our planned Phase 3 study. These forward-looking statements also reflect our current views about our plans, intentions, expectations, strategies and prospects, which are based on the information currently available to us and on assumptions we have made. Although we believe that our plans, intentions, expectations, strategies and prospects, as reflected in or suggested by these forward-looking statements, are reasonable, we can give no assurance that the plans, intentions, expectations, strategies or prospects will be attained or achieved. All forward-looking statements are based on estimates and assumptions by our management that, although we believe to be reasonable, are inherently uncertain. Furthermore, actual results may differ materially from those described in these forward-looking statements and will be affected by a variety of risks and factors that are beyond our control including, without limitation, uncertainty related to interactions with the FDA in general, uncertainty regarding geopolitical and macroeconomic events, risks associated with the discovery, development and regulation of efzofitimod, the risks associated with targeting a more limited patient population in our planned Phase 3 study of efzofitimod in pulmonary sarcoidosis, the risk that we or future partners may cease or delay preclinical or clinical development activities for efzofitimod for a variety of reasons (including difficulties or delays in patient enrollment in planned clinical trials), the possibility that future collaborations could be terminated early, and the risk that we may not be able to raise the additional funding required for our business and product development plans, as well as those risks set forth in our most recent Annual Report on Form 10-K, Quarterly Reports on Form 10-Q and in our other SEC filings. Except as required by law, we assume no obligation to update publicly any forward-looking statements, whether as a result of new information, future events or otherwise. Contact: Ashlee Dunston Sr. Director, Investor Relations and Public Affairs adunston@atyrpharma.com