CC-90006

据新浪网报道，2025年6月24日，曾在84版《鹿鼎记》中饰演韦小宝的知名武打演员李小飞，在确诊肝癌仅4个月后不幸离世，享年76岁。据悉，他于今年2月因昏迷被紧急送医，确诊时已处于肝癌三期，此时已失去手术机会。 在为这位老艺术家的离世深感痛心与惋惜之余，我们不禁追问：为何许多肝癌一经发现便是晚期？当前有哪些手段可及时发现肝癌？又有哪些抗癌新药与新技术能为患者争取一线生机？ ▲图源“sina”，版权归原作者所有，如无意中侵犯了知识产权，请联系我们删除 为何大多肝癌发现即晚期？ 肝癌之所以常常在发现时就已是晚期，主要与肝脏的生理特性、早期症状的隐匿性、高危人群早期筛查的不足、疾病本身的进展速度等有关。 1、 肝脏的“沉默”特性 ：肝脏是人体再生与代偿功能最强的器官之一。即便约 70%-80% 的肝细胞受损或癌变，剩余健康肝细胞仍能维持基本的代谢、解毒等功能，人体通常不会出现明显不适。此外，肝脏内部缺乏痛觉神经，只有当肿瘤生长到一定程度，压迫到肝脏包膜（包膜上有神经）或侵犯周围组织时，才会引起疼痛等明显症状，而此时病情往往已进展到中晚期。 2、 肝癌早期症状隐匿且缺乏特异性 ：肝癌早期几乎没有典型症状，少数患者可能出现乏力、食欲减退、轻微腹胀等表现，但易被误认为是普通的消化道不适（如胃病、肝炎等），甚至被当作“劳累”而忽视。等到出现黄疸（皮肤、眼白发黄）、腹水、肝区剧痛、体重骤降等明显症状时，肿瘤通常已较大或发生转移。 3、 高危人群筛查意识不足 ：肝癌的发生多与慢性肝病（如乙肝、丙肝病毒感染）、肝硬化、长期饮酒、非酒精性脂肪肝等因素相关，这些人群属于肝癌高危人群。但很多高危人群缺乏定期筛查的意识，未能及时通过超声、甲胎蛋白（AFP）检测等手段早期发现病变，导致病情延误。 4、 肝癌进展速度较快 ：肝癌细胞的增殖速度相对较快，尤其是中低分化的肝癌，从早期发展到晚期可能仅需数月~1年左右。若未及时干预，病情会迅速恶化，增加了“发现即晚期” 的概率。 如何尽早发现肝癌？ 早期发现肝癌是提高治疗效果、改善预后的关键。由于肝癌早期症状隐匿，主动筛查是早期发现的核心手段，尤其是针对高危人群。通过规范筛查，多数早期肝癌（如直径≤3 厘米的小肝癌）可被及时发现，此时通过手术切除、射频消融等治疗，5 年生存率可达 70% 以上，远高于晚期肝癌（不足 10%）。以下建议有助于早期发现肝癌： 明确高危人群并重点筛查 PART 1 肝癌的发生与多种因素密切相关，以下人群属于高危人群，需重点关注筛查： 1、慢性乙型肝炎病毒（HBV）或丙型肝炎病毒（HCV）感染者（包括携带状态）； 2、有肝硬化病史者（无论病因，如乙肝肝硬化、酒精性肝硬化等）； 3、非酒精性脂肪肝患者（尤其是合并代谢综合征，如肥胖、糖尿病等）； 4、长期大量饮酒者（日均酒精摄入≥40g，持续 10 年以上）； 5、有肝癌家族史者（一级亲属患肝癌）； 6、曾因其他癌症接受过腹部放射治疗者。 警惕肝癌的早期信号 PART 2 虽然肝癌早期症状不明显，但若出现以下轻微不适，需及时就医排查（尤其是高危人群更应引起重视）： 1、不明原因的乏力、食欲减退，且持续 2 周以上； 2、右上腹（肝区）隐痛、胀痛或不适感； 3、短期内体重莫名下降（1 个月内下降 5% 以上）； 4、皮肤、眼睛巩膜轻微发黄（黄疸早期表现）。 肝癌早期筛查的核心手段 PART 3 目前临床公认的肝癌早期筛查方法包括影像学检查和肿瘤标志物检测，两者结合可提高早期检出率。 1、 腹部超声检查 ：具有无创、便捷、价格较低的优势，能清晰显示肝脏内的占位性病变（如结节、肿块），可发现直径＞1厘米的早期病灶。但对极微小病灶（＜1 厘米）敏感性较低。 2、 血清甲胎蛋白（AFP）检测 ：AFP 是肝癌相对特异的肿瘤标志物，约 70% 的肝癌患者会出现 AFP 升高。但部分早期肝癌患者 AFP 可能正常（即 “AFP 阴性肝癌”），因此不能仅靠 AFP 判断，需结合超声。 3、 CT或核磁 ：若超声或 AFP 发现异常，可进一步做肝脏增强 CT 或增强磁共振（MRI），精准判断病灶性质（区分良性结节与恶性肿瘤）。 4、 异常凝血酶原检测 ：对于高危人群，还可检测异常凝血酶原（PIVKA-II），作为 AFP 的补充指标，提高早期诊断率。 肝癌术后复查的重要手段 PART 4 肝癌术后仍需警惕复发，以下检查有助于及时发现复发或转移征兆： 1、 循环肿瘤细胞（CTC）检测 ：作为先进的液体活检技术，CTC 检测通过分析血液中从原发肿瘤或转移灶脱落的恶性细胞，可精准评估肿瘤的存在状态、进展程度及治疗效果。 2、 ctDNA（循环肿瘤 DNA）检测 ：该技术能捕捉肿瘤早期甚至癌前阶段基因水平的异常变化，先于影像学发现癌变迹象，实现“早筛查、早发现、早诊断、早治疗”，适用于临床提示可能患癌但无法确诊的人群。 3、 微小残留病灶（MRD）检测 ：可发现治疗后体内残留的微量癌细胞或肿瘤相关异常分子，为评估复发风险、制定后续治疗方案提供依据，有助于提升患者治愈率与生存质量。 2025最新获批上市的肝癌新药 菲诺利单抗注射液+贝伐珠单抗注射液：新的联合治疗选择 PART 1 2025年2月28日，神州细胞研发的菲诺利单抗注射液（SCT-I10A）与贝伐珠单抗注射液（SCT510）联合疗法，获中国国家药品监督管理局（NMPA）批准，用于既往未接受过系统治疗的不可切除或转移性肝细胞癌治疗。 其中，菲诺利单抗注射液（Finotonlimab，SCT-I10A）是神州细胞自主研发的一款重组人源化抗PD-1IgG4型单克隆抗体注射液。而贝伐珠单抗注射液（Bevacizumab，SCT510）为其自主研发的一款重组人源化抗VEGF单克隆抗体注射液，作为贝伐珠单抗的生物类似药，此前已获批用于多种癌症治疗。 纳武利尤单抗注射液+伊匹木单抗注射液：开启肝癌双免治疗新时代 PART 2 2025年3月31日，百时美施贵宝公司研发的两款肿瘤免疫治疗药物——纳武利尤单抗注射液（欧狄沃）+伊匹木单抗注射液（逸沃）的新适应证，获得中国国家药品监督管理局（NMPA）批准，用于不可切除或晚期肝细胞癌(HCC) 成人患者的一线治疗。阅读原文了解详情：刚刚！“双免王炸”纳武利尤单抗+伊匹木单抗新适应证在华获批！2年中位OS照进现实 特瑞普利单抗+贝伐珠单抗：显著延长生存期 PART 3 2025年3月21日，君实生物创新研发的抗PD-1单抗药物——特瑞普利单抗注射液与贝伐珠单抗联合用于不可切除或转移性肝细胞癌的首线治疗，获国家药品监督管理局批准。这是特瑞普利单抗在中国获批的第11项适应症。 此次新适应症的获批主要基于HEPATORCH研究（NCT04723004）的研究数据。与索拉非尼相比， 特瑞普利单抗联合贝伐珠单抗可显著延长转移性肝细胞癌患者的无进展生存期（PFS）和总生存期（OS） ， 两组中位PFS分别为5.8vs.4.0个月，疾病进展或死亡风险降低31% （HR=0.69，95%CI：0.525-0.913；P=0.0086）， 中位OS分别为20.0vs14.5个月，死亡风险降低24% （HR=0.76，95%CI：0.579-0.987；P=0.0394）。同时， 联合治疗组客观缓解率（ORR）达25.3%，显著高于索拉非尼组的6.1% 。 肝癌患者必知的九大抗癌新技术，每一项都藏着生存希望 GPC3 CAR-T：肝癌疾病控制率达90.9% PART 1 GPC3在72%的晚期肝细胞癌（HCC）组织中呈高表达，且与患者术后转移显著相关，而在正常肝脏中无表达，因此被视为精准打击肝癌细胞的“黄金”靶标——既能精准识别癌细胞，又可避免损伤健康肝组织。 2024年ASCO大会上，一款靶向GPC3的自体CAR-T疗法——C-CAR031的首次人体临床研究（IIT）公布了惊艳数据：全部入组的24例晚期肝细胞癌患者，接受C-CAR031输注后， 疾病控制率（DCR）高达90.9%，客观缓解率（ORR）达50.0% （详见下图）。在22例可评估疗效的患者中， 90.9%出现肿瘤缩小，肝内及肝外病灶中位缩小率达44.0% （范围为3.4%-94.4%）， 中位无进展生存期（PFS）为4.27个月 （95%CI，2.86-8.90）。 ▲图源“ASCO”，版权归原作者所有，如无意中侵犯了知识产权，请联系我们删除 其中，C023号患者（DL4剂量组）接受治疗后肿瘤持续退缩：治疗1.5个月时， 肺部转移病灶即出现深度应答，影像学检查显示肿瘤显著缩小 。 ▲图源“ASCO”，版权归原作者所有，如无意中侵犯了知识产权，请联系我们删除 SCG101 TCR-T：抗癌与清除乙肝病毒两手抓 PART 2 在国际细胞与基因治疗大会（ISCT）上，一项应用SCG101TCR-T治疗HBV相关肝细胞癌的突破性案例公布。该患者接受单剂SCG101治疗后即展现惊艳疗效：不仅达成部分缓解（PR），肿瘤体积缩小74.5%，体内乙肝感染更被100%清除。 影像学显示，治疗第28天， 肿瘤靶病灶较基线缩小66%，率先达成部分缓解（PR） ；治疗第4个月， 肿瘤进一步缩小至74.5%，另有一处病灶完全消失 （详见下图）。值得注意的是，治疗全程未联合其他抗肿瘤干预，截至数据统计时， 患者无进展生存期（PFS）已超6.9个月，且持续维持缓解状态 。 ▲图源“SCG”，版权归原作者所有，如无意中侵犯了知识产权，请联系我们删除 此外，肝脏免疫组化分析显示，回输SCG101TCR-T后， 乙肝表面抗原阳性肝细胞被100%清除 ；血清学指标亦印证这一结果： HBsAg水平从输注前的557.96IU/mL，第7天骤降至1.3IU/mL，第28天近乎清零（0.08IU/mL） （详见下图）。 ▲图源“SCG”，版权归原作者所有，如无意中侵犯了知识产权，请联系我们删除 CIK细胞产品-爱可仑赛注射液：上市在即 PART 3 2025年3月31日，国产细胞治疗药物爱可仑赛注射液（EAL®）的新药上市申请，被中国国家药监局药品审评中心（CDE）正式受理（受理号：CXSS2500039），适用于原发性肝细胞癌根治术后高复发风险人群（患者需满足以下标准中的至少两项：无微血管侵犯；术后血小板和淋巴细胞比值（PLR）＜110；术后中性粒细胞和淋巴细胞比值（NLR）＜3），预防术后复发。 它从患者自身外周血提取单核细胞，经实验室扩增和功能增强，形成以CD8⁺细胞毒性T细胞为主的混合T淋巴细胞群。与传统化疗不同，EAL®能唤醒人体自身“抗肿瘤部队”，识别并清除肿瘤残留与微小病灶，从源头阻断复发。且因是自体细胞制备，几乎无排异反应。在430例II期临床试验患者中，已展现出延缓复发和降低死亡风险的积极信号。 此次受理，标志着中国首款针对实体瘤的细胞因子诱导杀伤细胞（CIK）疗法，正式进入上市审批阶段。当前，国内尚无CIK细胞治疗产品上市，也没有细胞疗法获批用于治疗实体瘤。若爱可仑赛注射液能顺利获批，将填补国内实体瘤细胞药物空白，实现中国免疫细胞药品治疗实体瘤领域的零突破，为肝癌术后高复发风险患者带来新的治疗希望！ ▲截图源自“NMPA” BST02 TIL：全球首个进入临床阶段的肝癌TIL产品 PART 4 百吉生物研发的BST02是全球首个进入临床阶段的肝癌专用TIL（肿瘤浸润淋巴细胞）产品，属于过继免疫细胞治疗技术。其基于MSE-TIL平台开发，通过扩增患者自身肿瘤浸润淋巴细胞制成，有望治疗包括肝细胞癌、胆管癌在内的所有类型肝癌。 2023年10月26日，该产品获美国FDA批准开展Ⅰ/Ⅱ期临床试验，适应症覆盖各类肝癌。其核心优势在于无需联用高剂量白介素-2（IL-2），既能大幅降低治疗相关副作用（如细胞因子风暴风险），又能显著提升患者生活质量。 在Ⅰ期临床试验中，BST02取得重大进展：一位晚期肝癌患者此前接受“介入+靶向+免疫”联合治疗及二线系统治疗均失败，肿瘤持续进展且副作用令其痛苦不堪，入组后接受低剂量BST02回输治疗。结果显示： 回输6周后病灶缩小57.1%；18周时靶病灶完全消退（CR），癌栓显著缩小，无疾病进展生存时间（PFS）达162天（约24周）。 ▼利用MSE-TIL技术平台开发的CAR-TIL对多种肿瘤的抗肿瘤效果 ▲图源“Biosyngen”，版权归原作者所有，如无意中侵犯了知识产权，请联系我们删除 个性化新抗原疫苗：将肝癌术后中位无复发生存期延长至8.3个月 PART 5 一项关于个性化新抗原疫苗治疗肝细胞癌的临床研究引发广泛关注。该研究纳入10例伴有门静脉分支侵犯的可切除肝细胞癌患者（均为术后高复发风险），包括5例肝细胞癌（HCC）、5例HCC合并肝内胆管癌（cHCC-CC），均接受个性化肿瘤新抗原疫苗的初免-加强方案接种。 结果显示：入组患者首次接种后的 中位无复发生存期（RFS）为7.4个月 （详见下图）； 根治术后中位RFS达8.3个月，其中2例患者持续无复发 （平均随访21.4个月）。 ▲图源“BMC”，版权归原作者所有，如无意中侵犯了知识产权，请联系我们删除 其中，46岁男性患者N22的案例尤为典型。该患者确诊为乙肝相关肝细胞癌，MRI显示肝5段有5.0×4.5×4.0cm肿瘤，手术切除1个月后接受预防性TACE治疗。术后病理诊断为侵袭性cHCC-CC，伴血管癌栓及循环肿瘤细胞，提示高复发风险。 患者于术后3个月启动新抗原疫苗接种（详见下图）。术后1年MRI发现1.4cm复发病灶，二次肝切除病理显示：复发肿瘤组织中CD8⁺T细胞浸润及颗粒酶B分泌量较原发灶显著增加，充分证实新抗原疫苗可诱导免疫记忆效应。 ▲图源“BMC”，版权归原作者所有，如无意中侵犯了知识产权，请联系我们删除 国研个性化mRNA肿瘤疫苗：肝癌复发率显著降低 PART 6 2024 ASCO大会上，公布了我国自研的一款个性化mRNA肿瘤疫苗的首次人体临床研究（NCT03674073）数据。 结果显示：该疫苗与消融疗法的联合使用具有可控的安全性，显示出免疫激活的证据和延长生存期的潜力。 1年复发率分别为18.2%（疫苗接种组） vs 33.3%（对照组）；2 年复发率分别为36.4%（疫苗接种组） vs. 51.4%（对照组） ， 显著降低了肝细胞癌患者的复发率 。 ▲截图源自“ASCO” 同种异体NK细胞：60%肝癌患者治疗三年无复发！ PART 7 一项针对乙肝病毒相关孤立性HCC患者的MG4101研究（NCT02008929）证实，肝切除术后应用体外扩增的同种异体自然杀伤（NK）细胞辅助治疗具有潜在临床价值。共纳入5例接受肝切除术的乙肝相关肝细胞癌患者，中位年龄44.8岁（范围36-54岁），肿瘤中位直径2.2cm（范围2.1-8.2cm），入组接受5个周期的体外扩增同种异体NK细胞治疗，中位随访期为43个月（范围：7-49个月）。 结果显示：NK细胞输注后 3年无复发生存率（RFS）为60%，总生存率（OS）为80% （详见下图）。 肝切除术后1年存活的4例患者，至3年时均存活 。其中1例患者（病例1）在根治性肝切除术后7个月出现肝内复发，经射频消融（RFA）治疗后， 连续42个月未再复发 。 ▼接受NK治疗患者的RFS和OS ▲图源“AHPS”，版权归原作者所有，如无意中侵犯了知识产权，请联系我们删除 Vγ9Vδ2T细胞注肝癌患者，中位总生存提升近3倍 PART 8 港大医学院团队与暨南大学合作，在全球率先完成同种异体γδ-T细胞治疗晚期肝癌、肺癌等实体瘤的临床研究，相关数据同步发表于《细胞与分子免疫学》杂志。 该研究共纳入132名晚期癌症患者（涵盖肺癌、肝癌、胰腺癌、乳腺癌等实体瘤），均接受来自健康供体的NF扩增同种异体Vγ9Vδ2T细胞（属γδ-T细胞的一种）治疗。 结果显示：肝癌亚组中7例（7/8）患者生存期≥10个月；截至2020年6月最新随访，3例肝癌患者接受过继性γδ-T细胞治疗后， 生存期已达30-35个月 （详见下图）。对比来看， 肝癌组未治疗患者的中位生存期（OS）仅8.1个月，而接受γδ-T细胞治疗者的中位生存期达23.1个月 （P=0.0002）， 中位OS提升近3倍 。 ▲图源“Cell Mol Immunol”，版权归原作者所有，如无意中侵犯了知识产权，请联系我们删除 粪便微生物群移植（FMT）联合PD-1抑制剂，助转移性肝癌患者肿瘤显著缩小近50% PART 9 近期，《临床与转化报告》发表的一项临床研究（NCT04264975）揭示，粪便微生物群移植（FMT）联合PD-1抑制剂在抗PD-1难治性晚期实体瘤治疗中展现惊人疗效：该联合疗法可有效重塑患者肠道微生物组，显著提升临床应答。 其中一例转移性肝癌（纳武利尤单抗原发性耐药）患者R7的疗效尤为显著，首次FMT联合免疫治疗仅减缓肿瘤生长；二次FMT移植8周后， 肿瘤体积显著缩小30.5%，最终实现部分缓解（PR），较首次治疗前基线缩小47.7% （详见下图），且免疫应答显著增强。 ▲图源“Cell Host & Microbe”，版权归原作者所有，如无意中侵犯了知识产权，请联系我们删除 小编寄语 相信通过上文的科普，肝癌患者对肝癌高发人群、筛查手段、2025年最新获批的肝癌新药及国内外最新抗癌技术进展等已有详细了解。 全球肿瘤医生网小编也希望，更多患者能实现早期发现——一旦出现异常症状，切勿拖延，及时就医干预可显著改善预后，避免错失最佳手术时机，防止类似“确诊仅四个月便不幸离世”的悲剧重演。对于已确诊的患者，应积极通过定期复查、病因控制、综合管理及必要的辅助治疗等方式，最大限度降低复发风险，助力更多肝癌患者延长生存期，跨越一个又一个10年！ 参考资料 [1]Zhang Q,et al.Phase I study of C-CAR031, a GPC3-specific TGFβRIIDN armored autologous CAR-T, in patients with advanced hepatocellular carcinoma (HCC)[J]. 2024. https://meetings.asco.org/abstracts presentations/234377 [2]Cai Z,et al.Personalized neoantigen vaccine prevents postoperative recurrence in hepatocellular carcinoma patients with vascular invasion[J]. Molecular cancer, 2021, 20: 1-13. https://molecular cancer.biomedcentral.com/articles/10.1186/s12943-021-01467-8 [3]Liang P,et al.A first-in-human study to evaluate a personalized neoantigen-based mRNA-loaded dendritic cell vaccine, in combination with ablation, in patients with hepatocellular carcinoma[J]. 2024. https://ascopubs.org/doi/abs/10.1200/JCO.2024.42.16_suppl.e14513 [4]Kim JM,et al.Adjuvant therapy using ex vivo-expanded allogenic natural killer cells in hepatectomy patients with hepatitis B virus related solitary hepatocellular carcinoma: MG4101 study. Ann Hepatobiliary Pancreat Surg. 2021 May 31;25(2):206-214. https://pmc.ncbi.nlm.nih.gov/articles/PMC8180393/ [5]Xu Y,et al.Allogeneic Vγ9Vδ2 T-cell immunotherapy exhibits promising clinical safety and prolongs the survival of patients with late-stage lung or liver cancer. Cell Mol Immunol. 2021 Feb;18(2):427-439. https://pmc.ncbi.nlm.nih.gov/articles/PMC8027668/ [6]Kim Y,et al. Fecal microbiota transplantation improves anti-PD-1 inhibitor efficacy in unresectable or metastatic solid cancers refractory to anti-PD-1 inhibitor[J]. Cell Host & Microbe, 2024, 32(8): 1380-1393. e9. https://www.cell.com/cell-host-microbe/abstract/S1931-3128(24)00228-2 [7]https://finance.sina.com.cn/jjxw/2025-06-30/doc-infcvpnn9965153.shtml 本文为全球肿瘤医生网原创，未经授权严禁转载

SAN DIEGO, CA, USA I February 12, 2025 I AnaptysBio, Inc. (Nasdaq: ANAB), a clinical-stage biotechnology company focused on delivering innovative immunology therapeutics, today announced statistically significant Week 12 data from the global 424-patient Phase 2b RENOIR trial of investigational rosnilimab, a depleter and agonist of PD-1+ T cells, for moderate-to-severe rheumatoid arthritis (RA). Rosnilimab was safe and well tolerated with similar adverse event rates vs. placebo. The Phase 2b RENOIR trial is evaluating the efficacy, safety, tolerability, pharmacokinetics and pharmacodynamics of rosnilimab in patients with moderate-to-severe RA on background conventional disease-modifying antirheumatic drugs (cDMARDs) (e.g., methotrexate). The trial enrolled 424 patients with a mean baseline disease activity score — 28 joints (DAS-28) C-Reactive Protein (CRP) score of 5.64 and mean baseline clinical disease activity index (CDAI) score of 37.7 across the U.S., Canada and Europe, who were either biologic or targeted synthetic DMARD (b/tsDMARD) naïve (n=250; 59%) or experienced (n=174; 41%). Patients classified as b/tsDMARD-experienced reported prior utilization of at least one biologic or targeted synthetic therapy, such as TNFα inhibitors, B cell inhibitors, selective costimulatory modulators or JAK inhibitors. Patients were randomized to receive either 100mg of subcutaneous rosnilimab every four weeks (Q4W), 400mg Q4W, 600mg every two weeks (Q2W), or placebo. The primary endpoint was assessed at Week 12 and secondary endpoints were assessed at both Week 12 and Week 14. Following completion of the Week 14 visit, rosnilimab-treated patients who achieved CDAI low disease activity (LDA) of ≤ 10, continued their assigned treatment through Week 28 in a blinded, all-active treatment period. Rosnilimab Achieved Primary and Secondary Efficacy Endpoints The trial achieved its primary endpoint of the mean change from baseline in DAS-28 CRP at Week 12 for all three doses of rosnilimab vs. placebo. Rosnilimab achieved statistical significance in at least one dose and numerical superiority at all doses, including once monthly administration, on key secondary endpoints of ACR20, ACR50 and CDAI LDA at Week 12, even though higher than typical placebo rates were observed. Also, rosnilimab demonstrated the highest ever reported responses for these key secondary endpoints at Week 14. 69% of rosnilimab-treated patients achieved CDAI LDA at Week 14 and appear to show sustained CDAI LDA and ACR50 responses, as well as potentially deepening ACR70 responses out to Week 28. Translational blood biomarker data, across all doses, showed similar immunological impact with robust on-target pharmacological activity in rosnilimab-treated patients that was not observed on placebo. Rosnilimab demonstrated rapid and sustained reduction of ~90% PD-1 high T cells and ~50% of PD-1+ T cells, and an increase in total Tregs. Together, this resulted in a minimal impact on total T cell counts and favorable T cell composition reflective of healthy immune homeostasis. Additionally, a ~50% reduction in the mean CRP from baseline, an objective measure of inflammation, was observed in rosnilimab-treated patients through the entire trial period that was not observed on placebo. “We are excited about these trial results and the impact they could have on patients living with RA. Rosnilimab is safe and well tolerated with the highest ever reported CDAI low disease activity at ~3 months that, to date, is sustained and potentially deepening over 6 months. These findings, further supported by objective translational data, surpass our target product profile in the ~$20 billion U.S. RA market,” said Daniel Faga, president and chief executive officer of Anaptys. “In Q2 2025, we will report full six-month data and additional translational data that we expect will further substantiate rosnilimab’s impact on restoring healthy immune homeostasis in RA and additional diseases, such as ulcerative colitis (UC). We also look forward to reporting top-line Week 12 Phase 2 data for rosnilimab in UC, now in Q4 2025.” Results for trial endpoints at Week 12 and Week 14 were as follows: At Week 14, 69% (71% of b/tsDMARD-naïve and 66% of b/tsDMARD-experienced) or 220 of the 318 rosnilimab-treated patients across all doses achieved CDAI LDA and were eligible to remain on continued active therapy through Week 28. As of the Dec. 10, 2024 data cutoff, these patients appear to show sustained CDAI LDA and ACR50 responses and potentially deepening ACR70 responses out to Week 28. This portion of the ongoing trial remains blinded, and complete Week 28 data are anticipated in Q2 2025. “In RA, there is an urgent need for innovative therapies such as rosnilimab, which have the potential of reducing the debilitating effects of this disease for a longer period of time for a broader range of patients,” said Jonathan Graf, M.D., professor of Medicine, Division of Rheumatology at the University of California, San Francisco and RENOIR investigator. “RA patients have an abnormal population of PD-1 high expressing T cells circulating in their bloodstream and joints. I am strongly encouraged by these Phase 2 data, which support the hypothesis that by depleting these PD-1 high expressing T cells and agonizing the remaining PD-1+ T cells, rosnilimab offers a fundamentally different approach to treating RA by resetting the immune system, potentially offering more durable relief of symptoms and disease modification.” Rosnilimab Safe and Well Tolerated with AEs Similar Across Treatment Groups and Placebo Consistent with prior studies, these rosnilimab Phase 2b data demonstrate a favorable safety and tolerability profile. The data through Week 12 show: The table below shows safety data for all patients through Week 12: 1. SAEs (severe unless otherwise noted): pneumonia – mild (100 mg Q4W); meniscus tear – moderate (400 mg Q4W); anaphylaxis from wasp sting, ureter stone, and cholecystitis/pericardial effusion (600mg Q2W); cellulitis/diarrhea (placebo) 2. Severe AE (excluding SAEs): flu (100mg Q4W); RA flare (600mg Q2W); macular degeneration/retinal hemorrhage (placebo) As of the Dec. 10, 2024 data cutoff, the safety profile for patients who achieved CDAI LDA through Week 14, and continued active therapy up to Week 28, remains consistent with the reported profile of all rosnilimab-treated patients through Week 12. “Despite multiple advances in the treatment of patients with RA, a large number remain difficult to treat. Unfortunately, no new drug classes have been approved for RA in the last decade. As we continue to advance our understanding of RA and look to reduce long-term damage to the body’s joints and organs caused by this disease, it is imperative that we develop treatment options with different modes of action, that are not only effective but also safe for long-term use,” said Paul Emery, M.D., Versus Arthritis professor of rheumatology at the University of Leeds and Leeds Biomedical Research Centre, UK. “Rosnilimab’s efficacy data paired with a favorable safety and tolerability profile present a promising new option for people living with RA.” “Today’s data offer new hope for patients living with RA and I am particularly encouraged by the combined efficacy and safety profile in both b/tsDMARD-naïve and -experienced patients. This advance in our understanding of RA would not have been possible without the patients and clinicians who participated in this important trial, and we are sincerely grateful,” added Paul Lizzul, M.D., Ph.D., chief medical officer of Anaptys. “Importantly, these positive clinical and translational data validate our scientific approach to target the PD-1 co-inhibitory receptor on activated immune cells in RA, as well as other heterogeneous, systemic autoimmune and inflammatory diseases, including UC, that would benefit from this novel approach targeting a central node of inflammation.” Further details are available on: Trial Details | ClinicalTrials.gov About the Primary and Secondary Endpoints The primary endpoint of mean change in DAS28-CRP at Week 12 is calculated based on differential weighting of individual measures, including the patient’s general health, CRP and a count of 28 swollen and tender joints, with a score ranging from 0 to 9.4. Secondary endpoints include the CDAI score, a composite assessment used to measure the severity of RA based on the sum of four assessment tools; the number of swollen and tender joints, the patient’s global disease activity index, and the physician’s global disease activity index. The score ranges from 0 to 76, with a score ≤ 10 is the threshold for LDA. Additionally, secondary endpoint ACR20/50/70 responses are used to measure change in RA disease activity. For example, an ACR50 response requires a patient to have a 50% reduction in the number of swollen and tender joints, and a reduction of 50% in three of the following five parameters: physician global assessment of disease, patient global assessment of disease, patient assessment of pain, CRP or erythrocyte sedimentation rate, and degree of disability in Health Assessment Questionnaire (HAQ) score. ACR20 and ACR70 responses require 20% and 70% reductions, respectively, across the measures listed above. About Rosnilimab Rosnilimab is a novel therapeutic antibody that directly targets PD-1, a co-inhibitory receptor preferentially expressed on the surface of activated T cells, which broadly impacts the pathogenic drivers of inflammatory diseases such as RA and UC. Rosnilimab is a targeted therapy designed to deplete PD-1 high T cells and agonize the remaining PD-1+ T cells to restore the immune system back to a state of homeostasis. This is anticipated to result in specific immunological outcomes in both inflamed tissue and the periphery, such as reduction in T cell proliferation, migration and cytokine secretion, and reduction of plasma cell generation and autoantibody levels. Rosnilimab is currently under clinical investigation, and its safety and efficacy have not been evaluated by any regulatory authority. Efficacy and safety data from this trial, including translational data, will be presented at future medical meetings. Anaptys Investor Call Anaptys management will host an investor call and live webcast, with an accompanying slide presentation, to review results of the data from the Phase 2b RA trial, today, Feb. 12, 2025, at 8:30am ET / 5:30am PT. A live webcast of the call will be available on the Anaptys website at: https://ir.anaptysbio.com/events . A replay of the webcast will be available for at least 30 days following the event. About Anaptys Anaptys is a clinical-stage biotechnology company focused on delivering innovative immunology therapeutics for autoimmune and inflammatory diseases. Its lead program, rosnilimab, a depleter and agonist targeting PD-1+ T cells, is in a Phase 2b trial for the treatment of rheumatoid arthritis and in a Phase 2 trial for the treatment of ulcerative colitis. Other antibodies in its portfolio include ANB033, an anti-CD122 antagonist, in a Phase 1 trial and ANB101, a BDCA2 modulator, entering a Phase 1 trial. Anaptys has also discovered multiple therapeutic antibodies licensed to GSK in a financial collaboration for immuno-oncology, including an anti-PD-1 antagonist ( Jemperli (dostarlimab-gxly)) and an anti-TIM-3 antagonist (cobolimab, GSK4069889). To learn more, visit www.AnaptysBio.com or follow us on LinkedIn . SOURCE: AnaptysBio