Efzofitimod

Meeting with the FDA to review the results from the Phase 3 EFZO-FIT™ study and determine the path forward for efzofitimod in pulmonary sarcoidosis is scheduled for mid-April 2026. SAN DIEGO, Feb. 03, 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 announced that the U.S. Food and Drug Administration (FDA) has accepted the Company’s meeting request to discuss its lead therapeutic candidate, efzofitimod, for the treatment of pulmonary sarcoidosis. The Type C meeting is scheduled for mid-April 2026. “We look forward to meeting with the FDA in mid-April to review the results of our Phase 3 EFZO-FIT™ study and determine the path forward for efzofitimod in pulmonary sarcoidosis, a major form of interstitial lung disease,” said Sanjay S. Shukla, M.D., M.S., President and Chief Executive Officer of aTyr Pharma. “We expect to provide an update regarding the outcome of the meeting following the receipt of the official meeting minutes.” EFZO-FIT™ was a Phase 3 study of efzofitimod in 268 patients with symptomatic pulmonary sarcoidosis. While the study did not meet its primary endpoint of change from baseline in mean daily oral corticosteroid dose at week 48, clinical benefit for 5.0 mg/kg efzofitimod was observed across multiple study efficacy parameters at week 48 compared to placebo, including improvement in change from baseline for the King’s Sarcoidosis Questionnaire (KSQ)-Lung score (p=0.0479), Fatigue Assessment Scale score (p=0.0226), KSQ-General Health score (p=0.0197), and complete steroid withdrawal with KSQ-Lung score improvement (p=0.0196). Additionally, treatment with efzofitimod maintained lung function as a measure of forced vital capacity and was well-tolerated with a safety pro with prior trials conducted to date. Abo ut Efzofitimod 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. In addition to the global Phase 3 EFZO-FIT™ study of efzofitimod in patients with pulmonary sarcoidosis, a major form of ILD, efzofitimod is also being investigated in the Phase 2 EFZO-CONNECT™ study in patients with systemic sclerosis (SSc, or scleroderma)-related 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 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 the potential therapeutic benefits and applications of efzofitimod; and timelines and plans with respect to certain development activities and goals, including the occurrence and timing of our meeting with the FDA to review the results of the Phase 3 EFZO-FIT™ study and determine the path forward for efzofitimod in pulmonary sarcoidosis, as well as our expectations with respect to the outcome of that meeting, the timing of our update for that meeting and next steps for the development of efzofitimod in pulmonary sarcoidosis. 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 risk that we or our 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 existing 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

各位呼吸科同行，咱们临床中碰到结节病，是不是常有种 “明明知道它难缠，却总在治疗上差点意思” 的感觉？这病堪称 “多器官突袭手”——90% 会累及肺部，30%-50% 还会 “搞事情” 到皮肤、眼睛、心脏这些地方，从轻症自愈到重症器官衰竭，病情谱宽得让人头疼。 常规操作里，激素是一线 “排头兵”，但长期用副作用扛不住；甲氨蝶呤、硫唑嘌呤这些二线药，又不是人人有效、人人耐受。之前大家公认的 “第三梯队援军” 是抗 TNF 药物里的英夫利西单抗，但除了它，其他生物制剂、靶向合成药到底行不行？最近发表在《Thorax》上的一篇系统综述 + meta 分析，专门给这些 “候选援军” 做了次 “全面体检”，今天咱们就用接地气的方式聊聊结果！先搞清楚：这篇研究是怎么 “体检” 的？ 研究团队从 MEDLINE、EMBASE 这些数据库里翻了个底朝天，从建库一直查到 2024 年 6 月，还补查了 ClinicalTrials.gov，最后筛出 16 篇符合要求的研究（含 15 个试验）—— 其中 8 个是随机对照试验（RCT），8 个是单臂试验，覆盖了抗 TNF、非抗 TNF 生物制剂，还有 JAK 抑制剂这类靶向合成药。 他们主要看两个核心：一是肺功能里的 “预测用力肺活量（% predicted FVC）” 变化（这是少数能统一对比的指标），二是用 “投票计数法”（按效果方向分类，不纠结统计显著性）汇总不同器官的疗效。简单说，就是既算 “硬数据”，也看 “整体趋势”，尽量客观评价每款药的表现。各 “援军” 表现大揭秘：有惊喜也有遗憾 咱们按 “表现等级” 给这些药分个队，方便大家快速 get 重点：1. 「久经沙场老将」：英夫利西单抗（抗 TNF） 作为早已获批用于难治性结节病的 “熟面孔”，这次研究再次确认了它的 “主力地位”： 肺功能：meta 分析显示，它能让 % predicted FVC 平均提升 5.7%（抗 TNF 类药物整体贡献最大），虽然不算 “飞跃式”，但刚好落在 “临床能感觉到改善” 的 2%-6% 范围内（也就是 MCID）； extrapulmonary ：对皮肤、眼睛、心脏这些 extrapulmonary 病变也有用，比如有试验里它能改善 “extrapulmonary 器官严重程度评分（ePOST）”； 结论：目前证据最足，是难治性结节病（尤其是肺或重要器官受累）在激素 + 二线药无效后的 “首选援军”。2. 「潜力副将」：阿达木单抗（抗 TNF） 作为英夫利西单抗的 “同门师弟”，它的表现有点 “偏科” 但值得期待： 优势领域：皮肤结节病表现突出 —— 有 RCT 显示，它能明显缩小皮肤病灶面积、减轻体积，只是患者生活质量评分（DLQI、SHQ）没到显著差异； 肺部表现：单臂试验里有 1/3 患者肺功能提升≥5%，但 RCT 里和安慰剂比没显出优势，可能和患者选择、剂量有关； 实用价值：如果患者对英夫利西单抗不耐受、有禁忌，或者需要长期自注射（方便居家使用），它可以作为 “备选副将”。3. 「新锐选手」：托法替布（JAK 抑制剂）、efzofitimod（抗 neuropilin-2） 这俩是这次研究里的 “新人”，证据虽少但方向很对，属于 “值得重点观察” 梯队： 托法替布（靶向合成药）：专攻皮肤和激素减量 —— 单臂试验里 10 个皮肤结节病患者全改善，6 个实现 “完全缓解”；5 个肺受累患者里，3 个成功停了激素还没让肺功能恶化，适合 “激素依赖 + 皮肤受累” 的难治患者； efzofitimod（新型生物制剂）：瞄准肺部 ——RCT 里它能改善肺功能、帮助减激素，还呈 “剂量依赖性”（剂量越高效果可能越好），只是目前结果没到统计显著，需要更大试验验证； 提醒：目前还在 “考察期”，临床用的话得经过多学科讨论，优先在研究或极难治患者中尝试。4. 「暂时出局」：这些药目前不推荐 有些 “候选援军” 这次表现不太理想，暂时没法加入治疗队列： 依那西普（抗 TNF）：最 “惨” 的一个 —— 治疗眼部结节病时反而让病情恶化，肺部试验也因为 “治疗失败太多” 提前叫停； 利妥昔单抗（抗 CD20）、sarilumab（抗 IL-6）：利妥昔单抗对肺功能改善不稳定，还有患者因病情进展死亡；sarilumab 甚至让肺功能和 CT 表现变差，直接 “负分出局”； 乌司奴单抗（抗 IL-12/23）、戈利木单抗（抗 TNF）：肺功能改善不显著，没显出比安慰剂更优的效果。这些 “体检结果” 靠谱吗？得说清 3 个局限 虽然研究很全面，但咱们临床用的时候，得知道这些结果的 “边界”： 「样本量太小」：不少试验像 “小范围摸底”—— 单臂试验中位样本量才 12 人，RCT 也有很多人数不足 50，结果可能不够 “普适”； 「设计不统一」：不同研究的患者类型（比如是否有纤维化）、疗效指标、随访时间不一样，比如有的只看肺，有的只看皮肤，对比起来有点 “各说各话”； 「偏倚风险高」：8 个 RCT 里 5 个 “高偏倚风险”，单臂试验几乎都有至少一项偏倚，可能让结果 “有点水分”。给咱们临床的 3 个核心启示 「首选不变」：英夫利西单抗仍是难治性结节病（尤其是肺 / 重要器官受累）的 “第一选择”，别轻易换； 「替代有谱」：阿达木单抗可作为英夫利西单抗的替代，尤其适合需要长期自用药、皮肤受累的患者； 「新锐慎用」：托法替尼、efzofitimod 虽有潜力，但目前别常规用 —— 要么入组临床试验，要么多学科（呼吸、风湿、皮肤科等）一起评估后，用在 “实在没辙” 的患者身上。未来还得解决这些 “老大难” 研究最后也指出，结节病治疗要想再进阶，还得闯 3 关： 「精准分组」：结节病太 “杂”，得先把不同亚型（比如纤维化型、非纤维化型）分清楚，再针对性做试验，避免 “一锅烩”； 「统一指标」：现在疗效指标五花八门，得定一套 “通用标准”（比如同时包含肺功能、症状、器官受累），让不同研究能对比； 「 timing 问题」：什么时候从激素 / 二线药升级到生物制剂？哪些患者 early 用效果更好？这些还没答案，需要更多研究。 最后想跟各位同行唠两句：结节病虽然难治，但这次研究至少让我们看到了更多 “潜在援军” 的方向。你们在临床中有没有碰到过 “用常规药没效果，试了新药用出惊喜” 的病例？或者对这些药物有什么疑问？欢迎在评论区分享经验、一起讨论 —— 毕竟，好的治疗方案，从来都是 “临床实战 + 证据总结” 磨出来的！