Ipilimumab

Burixafor (GPC-100) Phase 2 Trial in Multiple Myeloma Nears Key Readout Preparing for Expansion into Sickle Cell Disease and Acute Myeloid Leukemia New Leadership Appointments Bring Deep Drug Development Expertise REDWOOD CITY, Calif., Oct. 06, 2025 (GLOBE NEWSWIRE) -- Exicure, Inc. (Nasdaq: XCUR), a clinical-stage biotechnology company developing therapeutics for hematologic diseases, today shared recent progress for its lead program, burixafor (GPC-100), outlined upcoming strategic priorities, and introduced new members of its leadership team who will help drive the company’s next phase of growth. “The progress we’ve achieved since integrating GPCR Therapeutics earlier this year reflects both the strength and potential of the burixafor program and the dedication of our team,” said Andy Yoo, Chief Executive Officer of Exicure. “As we look ahead to our upcoming Phase 2 data readout and plan for expansion into new indications, I am energized by the opportunity to advance a program that could transform treatment approaches across multiple diseases.” Burixafor Phase 2 Trial in Multiple Myeloma Nears Key Clinical Readout Exicure’s ongoing Phase 2 study (NCT05561751) is a randomized, open-label, multicenter trial evaluating burixafor, a small molecule CXCR4 antagonist, in autologous stem cell transplant (ASCT) for multiple myeloma. By blocking CXCR4, burixafor is designed to mobilize hematopoietic stem cells out of the bone marrow and into the bloodstream, where they can be collected for use in transplant procedures. Interim results to date have been highly encouraging, with 100% of patients (10/10) achieving the primary endpoint of successful CD34+ stem cell mobilization, including patients previously treated with daratumumab. Burixafor has a faster kinetics of mobilization with a well-tolerated safety profile, enabling same-day administration of the mobilizing agent and leukapheresis. This differentiates burixafor from FDA-approved agents such as plerixafor and motixafortide, which require overnight pre-treatment. In August, the company announced that the final patient had completed their last study visit. The clinical database has since been locked, and Exicure remains on track to report topline data in Q4 2025. Preparations are also underway for a potential Phase 3 trial. A full data publication from the ongoing Phase 2 trial (NCT05561751) is anticipated in 2026. In addition, a publication from a previous Phase 2 study (NCT02104427) evaluating burixafor in combination with G-CSF in patients with multiple myeloma, non-Hodgkin lymphoma, and Hodgkin disease, is currently under peer review. Expanding Development Opportunities Building on progress in multiple myeloma, Exicure is preparing to expand burixafor into additional indications: Sickle Cell Disease: Exicure is in discussions with key clinicians at leading institutions to initiate an investigator-sponsored trial evaluating burixafor for improving stem cell mobilization in patients undergoing gene editing and autologous transplant.Acute Myeloid Leukemia (AML): The company is also planning a Phase 1 chemosensitization study in AML. Preclinical data suggest that burixafor may mobilize tumor cells from protective bone marrow niches, potentially enhancing the efficacy of chemotherapy. New Leadership Strengthen Scientific and Clinical Capabilities Following its acquisition of GPCR Therapeutics USA earlier this year, Exicure has been advancing the Phase 2 trial with dedicated internal drug development experts. These scientific leaders bring broad experience spanning from basic research to regulatory approval and have decades of proven track records in developing innovative medicines. Josephine (Pina) Cardarelli, Ph.D. joined as President and Chief Scientific Officer. Dr. Cardarelli is a seasoned drug development executive whose career spans oncology, immunology, pharmacology, and translational medicine. She most recently served as Vice President of Cell Biology & Pharmacology at Bristol-Myers Squibb, where she played a key role in the approvals of Yervoy® and Opdivo® and Fucosyl GM-1 antibody program that is currently in Phase 3. Earlier in her career at Medarex, she advanced multiple programs into clinical development, including CXCR4 (Ulocuplumab), CXCL10, CD30, and CD19 antibodies and multiple ADCs. She also identified the lead antibody for the IFNα receptor program that ultimately became AstraZeneca’s FDA-approved Saphnelo™. With more than 100 global patents and over 50 peer-reviewed publications, Dr. Cardarelli brings extensive expertise in biologics, antibody-drug conjugates, IND strategy, and regulatory affairs. She received her Ph.D. in physiology from Albany Medical College. Niña Caculitan, Ph.D. has been appointed Head of Clinical, overseeing activities spanning manufacturing, regulatory affairs, clinical development, clinical operations and data analysis. She brings over 15 years of experience from both academia and industry, with a strong background in antibody and small molecule chemistry for therapeutics in oncology and immune-mediated disorders. Previously, she was a key contributor to understanding cellular mechanisms of antibody drug conjugate processing at Genentech. Dr. Caculitan earned her Ph.D. in chemistry from the University of California, Berkeley. Devki Sukhtankar, Ph.D. has joined as Head of Preclinical Research and Translational Medicine, guiding cross-functional translational research efforts with a focus on integrating clinical data and advancing the pipeline into new indications. She brings over 15 years of experience in oncology, neurology and inflammatory diseases, where she has led preclinical programs with a focus on pharmacology and contributed to 20 peer-reviewed publications. Dr. Sukhtankar earned her Ph.D. in cancer biology from the University of Arizona and has extensive experience collaborating with partner organizations to expand therapeutic opportunities. About Burixafor (GPC-100)Burixafor (GPC-100) is a highly selective small molecule antagonist of CXCR4, a chemokine receptor that plays a central role in retaining hematopoietic stem cells in the bone marrow niche. By blocking CXCR4, burixafor may enhance the mobilization of these cells into the peripheral blood for collection and use in autologous stem cell transplant (ASCT) procedures. Originally developed by GPCR Therapeutics, Inc., burixafor became part of Exicure’s pipeline following the company’s acquisition in January 2025. In addition to multiple myeloma, burixafor is also being considered in other diseases where improved stem cell mobilization could help enable more efficient and effective treatment approaches, such as sickle cell disease, rare diseases requiring autologous transplant, and cell and gene therapy settings. A chemosensitization trial in AML is also being planned, leveraging burixafor’s mechanism of mobilizing malignant cells from protective bone marrow niches into the peripheral blood, where they may be more effectively targeted by chemotherapy. About Exicure Exicure, Inc. (Nasdaq: XCUR) is a clinical-stage biotechnology company developing therapies to address key challenges in hematologic diseases. The company’s lead program, burixafor (GPC-100), is being evaluated for its ability to improve stem cell mobilization in multiple myeloma, sickle cell disease, and in support of cell and gene therapy. It is also being studied as a potential chemosensitizing agent in acute myeloid leukemia (AML). For more information, visit www.exicuretx.com. Contact:Exicure, Inc.847.673.1700 (Tel)847.556.6411 (Fax)

点击蓝字 关注我们 本 期 看 点 2025年欧洲肿瘤内科学会（ESMO 2025）将于10月17-21日在德国柏林举行。作为全球肿瘤学领域的顶尖盛会，多项即将改变临床实践的重磅研究将在本届大会发布。目前，大会“突破性摘要”（LBA）的标题已经公布。 结直肠癌（CRC）长期是固态肿瘤治疗中最复杂的系统之-其分子异质性、免疫景观差异及治疗路径多层交错。然而，2025 年的 ESMO 大会，让我们看到 CRC 领域迎来一场真正意义上的“多维突破”： ctDNA 指导的辅助与再挑战策略、免疫治疗新布局、以及 VEGF/多靶点联合方案的临床深化，正在系统性地重塑 CRC 的治疗逻辑。本文详细梳理2025ESMO大会的重磅七项研究。 本期内容 01 七大重磅研究全景速览 02 ctDNA在结直肠癌领域的应用 03 免疫从MSI-H到MMR-p的前移探索 04 MSS人群的系统突破 【01 七大重磅研究全景速览】 LBA9：DYNAMIC-III 研究 标题： ctDNA-Guided Adjuvant Chemotherapy De-Escalation in Stage III Colon Cancer: Primary Analysis of the ctDNA-Negative Cohort from the Randomized AGITG DYNAMIC-III Trial (Intergroup Study of AGITG and CCTG) ctDNA指导下III期结肠癌辅助化疗降阶治疗：AGITG DYNAMIC-III随机试验（AGITG与CCTG多中心研究）中ctDNA阴性队列的初步分析 讲者： Jeanne Tie（彼得·麦卡勒姆癌症中心） LBA33：CITRIC 研究 标题： Circulating Tumor (ct) DNA-Guided Anti-EGFR Rechallenge Strategy in Metastatic Colorectal Cancer (mCRC): Final results of the phase II randomized CITRIC trial ctDNA指导下的抗EGFR药物再挑战策略用于转移性结直肠癌：II期随机CITRIC试验的最终结果 讲者： Clara Montagut（Hospital del Mar） LBA32：PARERE 研究 标题： Panitumumab retreatment followed by regorafenib versus the reverse sequence in chemorefractory metastatic colorectal cancer patients with RAS and BRAF wild-type circulating tumor DNA (ctDNA): Final results of the randomized PARERE trial by GONO 帕尼单抗再治疗后接受瑞戈非尼对比反向治疗顺序用于经ctDNA检测为RAS和BRAF野生型化疗难治性转移性结直肠癌患者：GONO随机PARERE研究的最终结果 讲者： Marco Maria M. Germani（比萨大学） LBA29：CheckMate 8HW 研究 标题： Nivolumab plus ipilimumab vs nivolumab monotherapy for microsatellite instability-high/mismatch repair-deficient (MSI-H/dMMR) metastatic colorectal cancer (mCRC): new results from CheckMate 8HW 纳武利尤单抗联合伊匹木单抗对比纳武利尤单抗单药治疗MSI-H/dMMR转移性结直肠癌：CheckMate 8HW研究最新结果 讲者： Sara Lonardi（Veneto Institute of Oncology） LBA30：STELLAR-303 研究 标题： Zanzalintinib plus atezolizumab (zanza + atezo) vs regorafenib (rego) in patients (pts) with previously treated metastatic colorectal cancer (mCRC): Primary overall survival (OS) analysis from the randomized, open-label, phase 3 STELLAR-303 study Zanzalintinib +阿替利珠单抗（zanza + atezo） vs 瑞戈非尼（rego）用于既往接受过治疗的转移性结直肠癌患者：随机、开放标签、III期STELLAR-303研究的主要总生存期分析 讲 者： Anwaar Saeed（UPMC 希尔曼癌症中心） LBA31 标题： Neoadjuvant immunotherapy induces immune activation and responses in MMR-proficient colon cancers 新辅助免疫疗法在MMR功能正常的结肠癌中诱导免疫激活和应答 讲者： Yara L. Verschoor（荷兰癌症研究中心） LBA34：DeFianCe研究 标题： DeFianCe Trial: A randomized phase 2 trial of sirexatamab (DKN-01) plus bevacizumab and chemotherapy versus bevacizumab and chemotherapy as second-line therapy in advanced microsatellite stable (MSS) colorectal cancer (CRC) DeFianCe研究：一项比较sirexatamab（DKN-01）+贝伐珠单抗+化疗 vs 贝伐珠单抗+化疗作为晚期MSS型结直肠癌的二线治疗的随机II期试验 讲者： Zev A. A. Wainberg（加州大学洛杉矶分校） 【02 ctDNA在结直肠癌领域的应用】 本届 ESMO 汇总的结直肠癌领域的重磅研究中，其中三项研究聚焦 ctDNA。 LBA9（DYNAMIC-III）在Ⅲ期结肠癌术后人群中，首次以随机对照的方式验证 ctDNA 阴性患者可实施辅助化疗降阶的可行性：在维持无病生存（DFS）不受影响的前提下，降低治疗强度与毒性负担，为“以分子残留状态（MRD）而非单一病理分期定强度”的新范式奠基。 面向转移性结直肠癌（mCRC），LBA33（CITRIC）通过ctDNA 动态确认 RAS/BRAF 野生型后实施抗 EGFR 再挑战，为“分子转阴→再用药”的闭环提供临床证据。 LBA32（PARERE，GONO）进一步比较帕尼单抗再治疗→瑞戈非尼与反向顺序：结果支持“先分子精准、后多靶点抗血管”的顺序优化思路。三项研究共同指向：ctDNA 不只是“测”，更要“用”；从辅助到再治疗，动态分层 + 精准时机将成为下一代标准路径的核心。 03 免疫从MSI-H到MMR-p的前移探索 免疫治疗板块两项 LBA 形成“巩固一线标准 + 前移新证据”的互补格局。LBA29（CheckMate 8HW）在MSI-H/dMMR mCRC中，纳武利尤单抗 + 伊匹木单抗相较单药纳武利尤单抗，持续在PFS、缓解持续时间（DOR）乃至 OS上展示优势，进一步稳固双免作为一线标准的地位，并以可管理的安全性支持长期用药。更具前瞻性的是 LBA31：在传统意义上的“免疫冷肿瘤”——MMR 功能正常（MMR-p）结肠癌中，新辅助免疫亦可诱导显著的免疫活化与病理应答，提示通过术前窗口期“预热”肿瘤微环境，或有望拓宽免疫获益人群。由此，CRC 免疫策略正从“静态分层（MSI 与否）”走向“时空重塑（围手术期免疫激活）”，边界被重新定义。 04 MSS人群的系统突破 面对占比最大的 MSS/mCRC，2025 年的两项Ⅲ/Ⅱ期研究带来可操作的联合思路。LBA30（STELLAR-303）显示，多靶点 TKI（Zanzalintinib）+ PD-L1 抑制剂（阿替利珠单抗）在既往治疗后的 mCRC 中对比瑞戈非尼实现总生存期（OS）层面的提升，为“抗血管 + 免疫”在 MSS 人群的应用提供了分层证据与安全性参考。LBA34（DeFianCe）则以DKK1 抑制剂 sirexatamab（DKN-01）+ 贝伐珠单抗 + 化疗对比“贝伐珠单抗 + 化疗”作为 MSS 型 CRC 的二线方案，在客观缓解率（ORR）与无进展生存（PFS）上取得优势，提示“WNT 通路调节 + 抗血管”有望成为免疫冷型肿瘤的可复用模板。配合前述 ctDNA 与免疫窗口策略，MSS 人群的组合—顺序—时机三维优化正加速成形：先以分子与微环境“做减法”，再以多通路“做加法”，把更多患者推向可获益的治疗轨道。 ★