An Open-label, Non-randomized Study to Evaluate the Pharmacokinetics (PK), Safety and Tolerability of a Single Dose of Mavorixafor in Participants With Hepatic Impairment (HI) Compared to Matched Healthy Volunteers With Normal Hepatic Function

The purpose of this study is to measure the effect of HI on the PK, safety, and tolerability of a single dose of mavorixafor compared to matched healthy volunteers (HVs) with normal hepatic function.

开始日期2025-02-28

申办/合作机构

X4 Pharmaceuticals, Inc.

NCT06914869

/ Completed临床1期

A Phase I, Randomized, Open-label, Drug-drug Interaction Study to Assess the Effect of Multiple Doses of a Moderate and a Strong CYP3A Inducer on the Pharmacokinetics and Safety of Mavorixafor in Healthy Male and Female Participants

The main purpose of this study is to evaluate drug-drug interaction (DDI) of orally administered mavorixafor with cytochrome P3A (CYP3A) inducers carbamazepine (a strong CYP3A inducer) or efavirenz (a moderate CYP3A inducer) in healthy male and female participants.

开始日期2025-02-18

申办/合作机构

X4 Pharmaceuticals, Inc.

NCT06056297

/ Recruiting临床3期

A Phase 3, Randomized, Double-blind, Placebo-controlled, Multicenter Study of Mavorixafor in Participants With Congenital and Acquired Primary Autoimmune and Idiopathic Chronic Neutropenic Disorders Who Are Experiencing Recurrent and/or Serious Infections

The purpose of this study is to demonstrate the efficacy and evaluate the safety and tolerability of mavorixafor in participants with congenital or acquired primary autoimmune and idiopathic chronic neutropenic disorders who are experiencing recurrent and/or serious infections as assessed by demonstrating its clinical benefit and increasing levels of circulating neutrophils.

开始日期2024-06-06

申办/合作机构

X4 Pharmaceuticals, Inc.

100 项与 Mavorixafor 相关的临床结果

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100 项与 Mavorixafor 相关的转化医学

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100 项与 Mavorixafor 相关的专利（医药）

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项与 Mavorixafor 相关的文献（医药）

2025-08-02·ChemMedChem

Synthesis and ⁶⁴Cu‐Radiolabeling Strategies of Small Organic Radioconjugates Based on the AMD070 Scaffold

Article

作者: Chérel, Michel ; Tripier, Raphaël ; Faivre‐Chauvet, Alain ; Troadec, Thibault ; Le Saëc, Patricia ; Le Roy, Marie M.

CXCR4 is a transmembrane receptor overexpressed in a large variety of cancer cells. In addition to classical antibody‐ and peptide‐targeting for the development of positron emission tomography (PET) radiopharmaceuticals, this receptor possesses a range of small organic inhibitors that can be exploited. These are based mainly on nitrogen‐rich scaffolds, such as AMD070, and cyclic polyamines, such as cyclam, in the AMD3100 skeleton. The latter has been explored as a direct 64Cu chelator, but examples of CXCR4 PET imaging with small organic targeting units are still scarce in the literature. Herein, the synthesis of two novel CXCR4‐directed radiopharmaceuticals is described, combining the AMD070 scaffold as a targeting unit and bifunctional te1pa macrocycle as a strong 64Cu chelator. The synthesis of the conjugates and optimization of 64Cu‐radiolabeling are presented, opening the way for future in vitro and in vivo studies.

2025-05-04·IMMUNOPHARMACOLOGY AND IMMUNOTOXICOLOGY

Mavorixafor: a CXCR4 antagonist for WHIM syndrome

Review

作者: Chen, Jixiang ; Xu, Bo ; Zhou, Jiecan ; Chen, Canyu ; Li, Weiyi ; He, Zunbo

BACKGROUND:

WHIM syndrome is a rare primary immune deficiency and chronic neutropenia caused by overactivation of the C-X-C motif chemokine receptor 4/C-X-C motif chemokine ligand 12 (CXCR4/CXCL12) signaling pathway. On April 26th, 2024, Xolremdi (mavorixafor) capsules received its approval from US FDA, is the first targeted treatment specifically for patients aged ≥12 years with WHIM syndrome. Mavorixafor, as a selective CXCR4 antagonist, is able to increase the number of mature neutrophils and lymphocytes in the blood.

OBJECTIVE:

This review is to describe the pharmacological properties of mavorixafor and evaluate its clinical efficacy and safety profile.

METHODS:

A literature search was conducted using keywords mavorixafor, XOLREMDI, AMD070, AMD11070, X4P-001, WHIM Syndrome, and CXCR4/CXCL12 on Web of Science, Google Scholar, and PubMed. Drug information was obtained from the FDA website.

RESULTS:

In the pivotal 52-week phase III trial, time above absolute neutrophil count threshold (TAT_ANC) values in the mavorixafor group were higher than those in the placebo group at 4 different time points (15.04 h vs 2.75 h; p < 0.0001), and mavorixafor group had lower infection frequency, severity and duration. The most common adverse events are thrombocytopenia, pityriasis, rash, rhinitis, epistaxis, vomiting, and dizziness.

CONCLUSION:

Mavorixafor 400mg daily effectively increases WBC count, reduces disease symptoms and infection burden in WHIM syndrome patients ≥12 years. Future clinical programs will continue to evaluate the safety and efficacy of mavorixafor in patients with chronic neutropenic disease.

2025-04-01·Annals of Medicine and Surgery

Mavorixafor, CXCR4 antagonist, a novel treatment for WHIM syndrome, first FDA approval 2024

作者: Hussaini, Helai ; Ahmad, Sarah ; Wardak, Abdul Baseer ; Sadeed, FNU ; Ilmaguook, Badr ; Waheed, Aiman ; Gul, Muhammad Hamza

Warts, Hypogammaglobulinemia, recurrent bacterial Infections, and Myelokathexis syndrome is a rare autosomal dominant disorder characterized by gain-of-function mutations in chemokine receptor type-4 (CXCR4 receptor). The acronym WHIM stands for Warts, Hypogammaglobulinemia, recurrent bacterial Infections, and Myelokathexis. Traditionally, WHIM syndrome has been managed with intravenous immunoglobulin (IVIG) and filgrastim (granulocyte colony-stimulating factor). Intravenous immunoglobulin is used to provide passive immunity to counteract hypogammaglobulinemia, while filgrastim stimulates the production and release of neutrophils from the bone marrow to address neutropenia. However, these treatments have limited efficacy, require frequent administration, and involve significant costs and patient burden. The recent Food and Drug Administration (FDA) approval of Mavorixafor, a small-molecule CXCR4 antagonist, marks a significant advancement in the management of WHIM syndrome. Mavorixafor enhances white blood cell mobilization from the bone marrow, directly targeting the underlying cause of the disease. In a pivotal phase-3 trial conducted by Badalato et al, involving 30 participants randomized to receive Mavorixafor 400 mg daily or placebo for 52 weeks, Mavorixafor demonstrated a statistically significant increase in absolute neutrophil count (P < 0.01) and a notable reduction in annual infection frequency compared to placebo. Additionally, a Phase-2 study reported a 75% reduction in cutaneous warts and an annual infection rate of 2.27 occurrences with adequate dosing. Mavorixafor maintained a favorable safety profile and required fewer administration complexities than IVIG and filgrastim, with 78% of patients achieving an overall response compared to 10% in the placebo group. These findings underscore the potential of Mavorixafor to significantly improve clinical outcomes for patients with WHIM syndrome, offering a more effective and targeted treatment option. The approval of Mavorixafor not only enhances current therapeutic strategies but also paves the way for future research into CXCR4 antagonists, potentially revolutionizing the management of WHIM syndrome and similar immunodeficiencies.

171

项与 Mavorixafor 相关的新闻（医药）

2025-08-13

·investors.x4pharma.com

X4 Pharmaceuticals Announces Closing of Upsized $85 Million Private Placement

BOSTON, Aug. 13, 2025 (GLOBE NEWSWIRE) -- X4 Pharmaceuticals (Nasdaq: XFOR), a company driven to improve the lives of people with rare diseases of the immune system, today closed an upsized private placement of 11,040,776 shares of common stock and pre-funded warrants to purchase 48,852,772 shares of common stock for total offering proceeds of $85 million. The originally announced offering of $60 million was increased to allow for the inclusion of an investor who had a pre-existing investment right. The offering was led by Coastlands Capital with support from existing investors Empery Asset Management, LP, Bain Capital Life Sciences, New Enterprise Associates (NEA) and other leading life science investors, including, BVF Partners LP, Deep Track Capital, Kalehua Capital, Nantahala Capital, Stonepine Capital Management, and Trails Edge Capital Partners. X4 expects to use the net proceeds from the financing for continued development towards a potential additional approval of mavorixafor in chronic neutropenia in addition to the commercialization of WHIM. The securities sold in this financing are being made in a transaction not involving a public offering and have not been registered under the Securities Act of 1933, as amended, and may not be offered or sold in the United States except pursuant to an effective registration statement or an applicable exemption from the registration requirements. X4 has agreed to file a registration statement with the Securities and Exchange Commission registering the resale of the shares of common stock and the shares of its common stock underlying the pre‐funded warrants sold in this financing. This press release shall not constitute an offer to sell or a solicitation of an offer to buy these securities, nor shall there be any sale of these securities in any state or other jurisdiction in which such offer, solicitation or sale would be unlawful prior to the registration or qualification under the securities laws of any such state or other jurisdiction. About X4 Pharmaceuticals X4 is delivering progress for patients by developing and commercializing innovative therapies for those with rare diseases of the immune system and significant unmet needs. Leveraging expertise in CXCR4 and immune system biology, X4 has successfully developed mavorixafor, an orally available CXCR4 antagonist that is currently being marketed in the U.S. as XOLREMDI® in its first indication. The company is also evaluating additional uses of mavorixafor and is conducting a global, pivotal Phase 3 clinical trial (4WARD) in people with certain chronic neutropenic disorders. X4 is headquartered in Boston, Massachusetts. For more information, please visit www.x4pharma.com. X4 Forward-Looking Statements This press release contains forward-looking statements within the meaning of applicable securities laws, including the Private Securities Litigation Reform Act of 1995, as amended. These statements may be identified by the words “may,” “will,” “could,” “would,” “should,” “expect,” “plan,” “anticipate,” “intend,” “believe,” “estimate,” “predict,” “project,” “potential,” “continue,” “target,” or other similar terms or expressions that concern X4’s expectations, strategy, plans, or intentions. Forward-looking statements include, without limitation, implied or express statements regarding the aggregate amount of proceeds to be received from the PIPE financing, the closing of the PIPE financing; and the use of proceeds from the PIPE financing. Any forward-looking statements in this press release are based on management’s current expectations and beliefs. These forward-looking statements are neither promises nor guarantees of future performance, and are subject to a variety of risks and uncertainties, many of which are beyond X4’s control, which could cause actual results to differ materially from those contemplated in these forward-looking statements, including the risks that: X4’s future financial performance and position may not improve, resulting in difficulties in implementing X4’s business strategy, and plans and objectives for future operations; the expected sufficiency of X4’s existing cash resources and runway may not be accurate resulting in the need for additional financing sooner than anticipated or unexpected liquidity constraints; the internal and external costs required for X4’s ongoing and planned activities, and the resulting impact on expense and use of cash, may be higher than expected, which may cause the company to use cash more quickly than expected or to change or curtail some of X4’s plans or both; the expected availability, content, and timing of clinical data from X4’s ongoing clinical trials of mavorixafor may be delayed or unavailable, including its ongoing Phase 3 clinical trial; trials and studies may be delayed and may not have satisfactory outcomes, earlier trials and studies may not be predictive of later trials and studies; the design and rate of enrollment for clinical trials, including the current design of the ongoing Phase 3 clinical trial evaluating mavorixafor in certain chronic neutropenic disorders may not enable successful completion of the trial(s); the commercial opportunity for mavorixafor in chronic neutropenic disorders may be smaller than anticipated; X4 may be unable to obtain and maintain regulatory approvals; the company may experience uncertainties inherent in the initiation and completion of preclinical studies and clinical trials and clinical development; adverse safety effects might arise from the testing or use of X4 product and product candidates; the need to align with collaborators may hamper or delay development and commercialization efforts or increase costs; business may be adversely affected and costs may increase if any of the company’s key collaborators fails to perform its obligations or terminates the collaboration; X4’s ability to advance and commercialize and increase sales in mavorixafor to treat chronic neutropenia or to optimize the U.S. promotion of XOLREMDI (mavorixafor), approved for the treatment of WHIM; and other risks and uncertainties, including those described in the section entitled “Risk Factors” in X4’s most recent Annual Report on X4’s Form 10-K, as well as in other filings X4 makes with the Securities and Exchange Commission, including its quarterly report on Form 10-Q, from time to time. X4 undertakes no obligation to update the information contained in this press release to reflect new events or circumstances, except as required by law. X4 Investor Contact: Candice Masse astr partners candice.masse@astrpartners.com (978) 879-7273 Source: X4 Pharmaceuticals

上市批准临床3期

2025-06-27

·靶点圈

趋化因子受体CXCR4的长文解读：揭秘靶向药物的研发现状以及在疾病治疗中的作用机制

1CXCR4的分子结构与信号通路CXCR4的分子结构CXCR4作为G蛋白偶联受体(GPCR)，由352个氨基酸组成，具有典型的七跨膜α螺旋拓扑结构，属于CXC趋化因子受体家族成员，又称CD184。图1 CXCR4的结构示意图CXCR4的表达调控CXCR4的表达受表观遗传(如启动子低甲基化、组蛋白乙酰化)、非编码RNA(如miR-340-5p、miR-588)及转录因子(如NF-κB、HIF-1α)的调控。CXCR4的结合配体CXCR4主要结合天然配体CXCL12(又称SDF-1)，有α、β、γ等多种亚型，二者结合后调控细胞迁移、免疫应答和神经可塑性。其中，α亚型在骨髓干细胞归巢和神经系统发育中起关键作用；β亚型与血管生成相关；γ亚型与癌细胞的转移密切相关。图2 CXCR4/CXCL12生物轴中的信号通路CXCR4的信号传导CXCR4与CXCL12结合后，通过G蛋白亚基(如Gαi、Gα12/13、Gβ/Gγ)激活下游通路：● PI3K/Akt通路：调控细胞存活、增殖和代谢。● PLC通路：参与细胞内钙动员和蛋白激酶C(PKC)激活。● JAK/STAT通路：调节基因转录，影响细胞增殖和免疫应答。● MAPK/ERK通路：控制细胞生长、分化和凋亡。● β-抑制蛋白(β-arrestin)通路：参与受体脱敏和内吞作用，调控信号持续时间。2CXCR4的生物学功能与病理作用CXCR4的生理功能● 免疫系统调控①免疫细胞迁移：CXCR4表达于T/B细胞、树突状细胞等表面，介导免疫细胞向感染或炎症部位的趋化运动。②HIV共受体：作为人类免疫缺陷病毒(HIV)感染免疫细胞的共受体，与CD4协同促进病毒进入细胞。● 造血与组织修复①造血干细胞(HSC)归巢：在骨髓微环境中，CXCR4/CXCL12轴维持HSC的滞留和自我更新，调控血细胞生成。②组织再生：CXCR4参与心血管和消化系统的稳态维持、伤口愈合及血管生成(如内皮细胞迁移和新生血管形成)。● 神经系统作用①神经发育：CXCR4在神经元突触前/后膜均有分布，调控神经元和神经胶质细胞的迁移，参与血脑屏障形成。②神经保护：在神经炎症(如多发性硬化)中，CXCR4信号可减轻神经损伤，促进轴突修复和神经元干细胞分化。图3 CXCR4的生理功能CXCR4的病理功能● 肿瘤进展与转移①癌症干细胞(CSCs)标记：CXCR4+细胞具有干细胞特性(自我更新、肿瘤起始能力)，与肿瘤的侵袭性密切相关。②增殖：通过CXCR4PI3K/AKT、JAK/STAT、MAPK等通路促进肿瘤细胞增殖。③血管生成：诱导VEGF表达、招募内皮祖细胞，激活血管生成相关基因，并通过激活Rac1和MMP-2/MMP-14蛋白酶，增强肿瘤细胞侵袭和血管内渗能力。④转移：促进上皮-间质转化(EMT)、细胞黏附(如整合素表达)和基质金属蛋白酶(MMPs)分泌，介导肿瘤细胞向高表达CXCL12的器官(如肺、骨髓、淋巴结)迁移，形成转移灶。● 治疗抵抗与免疫逃逸①耐药性：激活PI3K/Akt和JAK/STAT通路，上调抗凋亡蛋白(如Bcl-2)，降低化疗和放疗敏感性。②免疫抑制微环境：肿瘤相关成纤维细胞(CAFs)分泌CXCL12，与癌细胞表面CXCR4结合，通过招募调节性T细胞(Tregs)、肿瘤相关巨噬细胞(TAMs)和髓源性抑制细胞(MDSCs)，抑制抗肿瘤免疫应答。● 预后价值CXCR4在超20种癌症(如非小细胞肺癌、乳腺癌、胰腺癌、白血病等)中高表达，与肿瘤的恶性程度、转移和预后不良显著相关，包括更高的转移率、化疗抵抗和更短的生存期。图4 CXCR4的病理功能3CXCR4靶向药物类型及临床进展CXCR4靶向治疗的核心策略● 阻断CXCR4/CXCL12相互作用：通过拮抗剂阻止配体结合，抑制下游PI3K/Akt、MAPK等促癌通路，减少肿瘤细胞增殖、迁移和耐药性。● 诱导受体内吞与降解：部分抗体(如Ulocuplumab)通过促进CXCR4内吞，降低细胞表面受体水平，阻断信号传导。● 癌症干细胞(CSCs)靶向：选择性清除CSCs，减少肿瘤复发和转移。例如，肽类拮抗剂Motixafortide通过结合CXCR4，抑制CSCs的归巢和存活。图5 不同CXCR4抑制剂的作用机制小分子拮抗剂● Plerixafor(AMD3100)：首个FDA批准的CXCR4拮抗剂，双环胺衍生物，竞争性结合CXCR4，阻断CXCL12信号，用于干细胞动员。联合化疗或免疫疗法(如抗PD-1抗体)用于血液肿瘤和实体瘤。● Mavorixafor(X4P-001)：提高口服生物利用度高，可增强CD8+细胞浸润，调节肿瘤微环境免疫细胞浸润，减少心脏毒性。FDA批准用于治疗WHIM综合征，联合免疫检查点抑制剂用于肾细胞癌、黑色素瘤等。● 其他药物：Burixafor(亲和力高于Plerixafor，用于干细胞动员)、GMI-1359(双重抑制CXCR4和E-selectin，用于乳腺癌)。表1 靶向CXCR4/CXCL12轴的小分子肽类拮抗剂● T22及其衍生物：如T140、Motixafortide(BL-8040)，源自马蹄蟹蛋白，通过β-折叠结构结合CXCR4。其中，Motixafortide联合G-CSF已获批用于MM的干细胞动员，并联合免疫疗法用于胰腺癌。● POL系列：如Balixafortide(POL6326)，环肽类，用于乳腺癌、前列腺癌的临床研究，可增强化疗敏感性。单克隆抗体● Ulocuplumab(BMS-936564)：人源化IgG4抗体，通过活性氧(ROS)诱导凋亡，不依赖补体或ADCC效应。用于MM和AML，联合疗法显示响应率提升。● PF-06747143：IgG1抗体，兼具ADCC/CDC效应及细胞动员作用，用于AML、NHL。其他● 纳米抗体：如ALX-0651，双位点结合CXCR4，用于干细胞动员。● 天然产物：①海洋黄酮Penicillixanthone-A：双重抑制CXCR4/CCR5，抗HIV和肿瘤。②柴胡皂苷A：通过下调CXCR4表达，抑制乳腺癌转移。③清热活血方(QRHX)：通过抑制CXCL12/CXCR4/JAK2/STAT3通路，调控肿瘤相关巨噬细胞，用于肺癌模型。④Turmeric(姜黄)：通过下调CXCR4和STAT3，抑制多种肿瘤的增殖和转移。⑤Sinomenine(青藤碱)：通过抑制CXCR4/STAT3通路诱导骨肉瘤细胞周期阻滞和凋亡。● 适配体：NOX-A12(Spiegelmer适配体)，靶向CXCL12/CXCR4轴，用于血液肿瘤。● 抗体-药物偶联物(ADCs)：抗CXCR4抗体偶联Auristatin，选择性杀灭CXCR4+肿瘤细胞，减少对正常干细胞的毒性。● 放射性核素：如⁷⁷Lu/⁹⁰Y标记Pentixather，用于多发性骨髓瘤的内放射治疗，联合化疗显著降低肿瘤负荷。表2 CXCR4的特异性配体目录包括受体激动剂和拮抗剂联合治疗● 化疗增敏：CXCR4抑制剂与化疗药物联用，逆转肿瘤细胞对紫杉醇、阿糖胞苷等的耐药性(如Plerixafor联合化疗用于AML)。● 免疫治疗协同：通过重塑免疫微环境(如减少Tregs和MDSCs浸润)，增强PD-1抑制剂等免疫疗法的响应率(如Mavorixafor联合Nivolumab用于肾细胞癌)。靶向药物递送系统利用CXCR4介导的内吞作用，设计靶向载体，实现化疗药物或siRNA的肿瘤选择性递送。● 脂质纳米颗粒：表面修饰CXCR4配体(如SDF1α、AMD3100)，递送化疗药物(如阿霉素)、siRNA或光热剂(IR780)，用于乳腺癌、肝癌等，兼具靶向性与信号阻断双重作用。● 聚合物纳米颗粒：如PLGA纳米粒结合LFC131肽，递送索拉非尼或p53mRNA，逆转耐药并调节免疫微环境；氧化还原响应型胶束结合T22肽，共递送Venetoclax和索拉非尼，用于AML治疗。● 蛋白纳米颗粒：如T22肽修饰的多价蛋白纳米颗粒，递送化疗药物(如5-FdU、Ara-C)、毒素(如PE24、蓖麻毒素)或促凋亡蛋白，诱导肿瘤选择性焦亡，用于结直肠癌和AML的选择性杀伤。● 无机纳米粒：如金纳米粒结合抗CXCR4抗体，增强放疗敏感性。表3 CXCR4靶向药物递送系统CXCR4的诊断与预后价值CXCR4过表达与肿瘤分期、转移负荷及不良预后相关，可作为分子标志物指导患者分层和治疗监测。● ⁶⁸Ga-Pentixafor(诊断性放射性药物)：高亲和力结合CXCR4，通过PET成像可视化受体分布，肾排泄快，背景积累低。①血液系统肿瘤：对多发性骨髓瘤(MM)的诊断灵敏度远高于¹⁸F-FDGPET(93.3%vs.53.3%)，定量指标与肿瘤分期和生物标志物高度相关。在弥漫大B细胞淋巴瘤、边缘区淋巴瘤中，可提高分期准确性并指导治疗策略调整。②实体瘤：在小细胞肺癌(SCLC)、肾上腺皮质癌中摄取显著，卵巢癌、胆管癌中肿瘤/背景比(TBR)较高，但在多数实体瘤中表达异质性限制其广泛应用。③其他疾病：可用于心血管疾病(如心肌梗死)和炎症性疾病的CXCR4表达评估。● ¹⁷⁷Lu/⁹⁰Y-Pentixather(治疗性放射性药物)：通过放射性核素(如¹⁷⁷Lu、⁹⁰Y)标记CXCR4拮抗剂，精准递送辐射至肿瘤细胞，诱导凋亡并抑制微环境支持。①血液系统肿瘤：在复发难治性急性髓系白血病(AML)中，联合异基因造血干细胞移植(HSCT)可实现完全缓解；在MM中，单药治疗部分患者出现部分缓解，但总体生存率未显著提升。②实体瘤：初步研究显示对SCLC、肾上腺皮质癌有效，但因实体瘤异质性和骨髓抑制毒性，应用受限。● AMD3100衍生物：如⁶⁴Cu-AMD3465，用于CXCR4表达可视化，但肝、肾摄取较高需优化。● 荧光成像：如Cy5标记Pentixafor，用于Barrett食管的荧光内镜检测。图6 在癌症诊断和治疗中靶向趋化因子受体CXCR44CXCL12/CXCR4轴在不同疾病中的应用肿瘤● 黑色素瘤：CXCL12/CXCR4通过激活PI3K-AKT、MAPK、NF-κB等下游信号通路，促进肿瘤细胞增殖、迁移、血管生成及免疫逃逸，同时通过β1整合素增强肿瘤细胞与血管内皮细胞黏附，引导肿瘤细胞向CXCL12富集的淋巴结、肝、骨等器官迁移，形成转移灶。图7 CXCL12/CXCR4在黑色素瘤中的作用机制● 白血病：CXCL12/CXCR4维持骨髓微环境黏附，在急性T细胞淋巴细胞白血病(T-ALL)中，CXCL12诱导H3K9甲基化，改变染色质构象，增强白血病细胞的核变形能力和迁移性；慢性淋巴细胞白血病(CLL)中，CXCL12/STAT3通路促进IL-10表达，抑制T细胞功能，介导免疫逃逸。此外，CXCL12/CXCR4轴通过激活STAT3，保护肿瘤细胞免受化疗诱导的凋亡。● 乳腺癌：CXCL12/CXCR4可间接诱导CyclinD1和VEGF表达，促进肿瘤增殖和血管生成。临床样本中，CXCR4与磷酸化STAT3(p-STAT3)的共表达与肿瘤大小、淋巴结转移及组织学分级呈正相关。● 肺癌：CXCL12/CXCR4通过JAK2/STAT3通路抑制顺铂诱导的肺癌细胞凋亡，临床样本中CXCL12与p-STAT3表达呈正相关，且与恶性进展相关。● 膀胱癌：癌症相关成纤维细胞(CAFs)分泌的CXCL12通过JAK2/STAT3通路诱导p62蛋白积累，抑制PD-L1的自噬降解，促进免疫逃逸。表4 癌症中针对CXCR4的治疗干预(临床试验)神经退行性疾病● 阿尔茨海默病(AD)：CXCL12/CXCR4激活NF-κB通路，促进小胶质细胞释放TNF-α，加速Aβ聚集和tau过度磷酸化，导致突触丢失，损害神经祖细胞(NPC)增殖和记忆形成。● 帕金森病(PD)：α-synuclein通过TLR4激活CXCL12/CXCR4信号，招募微胶质细胞并释放IL-1β，损伤黑质多巴胺能神经元。低浓度CXCL12通过CXCR4促进多巴胺释放(神经保护)，高浓度则通过Rac1通路诱导神经元凋亡。执行功能障碍与前额叶CXCL12/CXCR4介导的小胶质细胞活化相关。● 多发性硬化(MS)：CXCL12在MS斑块处异常分布，吸引CXCR4+免疫细胞穿越血脑屏障(BBB)，加剧脱髓鞘和轴突损伤。信息处理速度下降与CXCL12/CXCR4轴激活相关，CSF中CXCL12水平可作为疾病严重程度的生物标志物。● 肌萎缩侧索硬化(ALS)：CXCL12/CXCR4在脊髓前角运动神经元中上调，参与TDP-43蛋白聚集和应激颗粒形成，加速神经元死亡。约1/3患者伴前额叶认知障碍，与运动皮层微胶质细胞活化和CXCL12/CXCR4信号增强相关。图8 神经炎症和认知障碍图9 干细胞在神经炎症和再生中的作用和功能其他疾病● 银屑病：皮损中CXCL12和CXCR4表达升高，CXCL12+成纤维细胞招募CXCR4高表达中性粒细胞和T细胞，促进IL-17依赖性炎症和表皮增生。CXCR4阻断可改善咪喹莫特诱导的银屑病样病变，但角质形成细胞中CXCR4缺失反而加剧炎症，提示细胞类型特异性机制。● 脑损伤(弓形虫感染)：CXCL12与STAT3的mRNA表达升高，协同调控先天免疫和T细胞介导的保护性免疫，控制脑内弓形虫感染。● 心肌缺血：心脏干细胞通过旁分泌CXCL12激活STAT3，促进血管再生和心肌修复；miR-135a-5p/CXCL12/JAK-STAT轴则通过调控炎症和凋亡，减轻心肌梗死损伤。● 动脉损伤：JAK/STAT3通路诱导血管内皮细胞表达CXCL12，促进血管重塑过程中的增殖反应。● 皮肤疾病：CXCL12通过CXCR4激活真皮乳头层和外根鞘细胞的STAT3/STAT5，抑制毛发生长；在伤口愈合中，IL-10通过STAT3促进成纤维细胞合成透明质9178和VEGF，加速组织再生。● 胃炎(幽门螺杆菌感染)：cagA蛋白激活STAT3，后者与转录因子BHLHE40结合，上调CXCL12表达，促进胃黏膜CD4+T细胞浸润，加剧炎症反应。● 肾纤维化：CXCL12α/CXCR4通过JAK2/STAT3/GSK3β/β-catenin通路，抑制GSK3β活性，促进β-catenin核转位，诱导肾小管细胞纤维化；糖尿病肾病中，该轴还通过STAT3介导DPP4诱导的线粒体功能障碍。● 肝纤维化：CAFs分泌的CXCL12通过ACKR3/STAT3通路增强基质金属蛋白酶(MMP2)和VEGF表达，促进肿瘤内皮细胞迁移和血管生成。图10 CXCL12/CXCR4与STAT3通路表5 靶向CXCL12/STAT3通路的治疗药物纲要疼痛● 神经病理性疼痛：在多种神经病理性疼痛模型中，如坐骨神经损伤、糖尿病神经病变、HIV相关神经痛等，CXCL12/CXCR4轴的表达均上调。它通过激活下游信号通路、促进炎症因子(TNF-α、IL-1β)释放等机制，增强神经元兴奋性(如Nav1.8钠通道表达增加)，导致痛觉过敏和异常疼痛行为。在坐骨神经损伤模型中，损伤后CXCL12和CXCR4在背根神经节(DRG)和脊髓背角的表达上调，参与了神经病理性疼痛的发展和维持。● 炎症性疼痛：在CFA诱导的炎症性疼痛中，CXCR4基因启动子去甲基化，导致其表达升高，激活NF-κB和ERK通路，引发机械性痛敏。在慢性胰腺炎疼痛中，TLR4与CXCL12/CXCR4相互作用形成正反馈，激活星形胶质细胞和小胶质细胞，释放炎症因子，加重疼痛。● 癌症相关疼痛：在骨癌痛中，肿瘤细胞植入会诱导脊髓CXCL12表达，激活RhoA/ROCK2和CaMKII/CREB通路，增强神经元敏感性。在胰腺癌中，早期肿瘤通过CXCL12吸引施万细胞形成神经微环境，可能抑制疼痛，但晚期促进神经病理性疼痛。● 术后疼痛：以足底切口模型为例，术后脊髓背角CXCL12/CXCR4激活NF-κB和Akt，介导机械痛敏。抑制CXCR4可减少细胞外信号调节激酶(ERK)磷酸化，从而缓解疼痛。● 阿片类药物诱导的痛觉过敏(OIH)：CXCL12通过NMDAR(NR2B亚基)增强神经元兴奋性，导致痛觉过敏。使用CXCR4拮抗剂(如AMD3100)可降低NMDAR磷酸化，减轻痛觉过敏症状。● 复杂性区域疼痛综合征(CRPS-I)：在CRPS-I的慢性缺血疼痛(CPIP)模型中，脊髓背角CXCL12/CXCR4轴激活ERK通路，参与疼痛信号的传递。阻断CXCR4可减少c-Fos阳性染色细胞，降低胶质细胞激活，缓解机械性异常性疼痛。图11 慢性疼痛中的CXCL12/CXCR4轴的作用机制5挑战与未来方向现有挑战● 毒性与脱靶效应：小分子及抗体药物对正常组织(如肝、肾、骨髓等)的影响需通过优化靶向特异性解决(如纳米载体靶向)。● 实体瘤渗透限制：CXCR4⁺肿瘤细胞常位于基质丰富区域，纳米药物递送效率受限，需结合超声成像或微泡技术增强渗透。● 耐药性机制：长期治疗可能诱导CXCR4表达上调或信号通路代偿，需联合多靶点抑制剂(如同时阻断PI3K和CXCR4)。未来方向● 精准医学：通过⁶⁸Ga-PentixaforPET成像量化CXCR4表达，筛选高表达患者开展“篮子试验”(跨癌种治疗)。● 联合治疗策略：①免疫-靶向联合：CXCR4拮抗剂与化疗、免疫检查点抑制剂(如PD-1抗体)、TLR激动剂或、CAR-T细胞疗法或放疗联用，增强抗肿瘤免疫和敏感性，克服耐药。②双特异性分子：开发同时靶向CXCR4和其他CSC标记(如CD44、LGR5)的双抗或纳米颗粒，克服异质性。● 新型递送技术：①仿生纳米载体：模拟巨噬细胞膜的CXCR4靶向纳米囊泡，递送化疗药物并重塑免疫微环境。②智能响应型系统：pH或酶响应性纳米颗粒，在肿瘤微环境中释放药物，减少正常组织暴露。抗体发现服务 & 产品1羊驼免疫&骆驼免疫—自建现代化养殖农场2万亿级天然抗体库产品—轻松DIY科研抗体3配套产品—助您轻松搭建基因工程抗体平台关于仁域生物成都仁域生物成立于2019年1月，是一家专注基因工程抗体技术和天然抗体库开发的公司，拥有优化的噬菌体展示抗体库技术和现代化的骆驼/羊驼养殖免疫基地。可为客户提供14天、100%成功率的先导抗体分子发现服务，彻底解决传统抗体定制的周期长、失败率高、成本高三大难题。目前已经成功完成300+靶点抗体筛选项目！protocol 获取 / 产品咨询邮箱｜find@renyubio.com电话｜19136178673地址｜成都市经开区科技产业孵化园关注我们，小编将持续更新相关内容～参考文献Rueda A, Serna N, Mangues R, Villaverde A, 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The role of CXCL12/CXCR4/CXCR7 axis in cognitive impairment associated with neurodegenerative diseases. Brain Behav Immun Health. 2024 Dec 24;43:100932.Dzhuzha DO. RADIOPHARMACEUTICALS BASED ON ANTAGONISTS OF CHEMOCINE RECEPTOR CXCR4 IN DIAGNOSTICS AND TREATMENT OF ONCOLOGICAL DISEASES. Probl Radiac Med Radiobiol. 2024 Dec;(29):19-33. English, Ukrainian. Korbecki J, Bosiacki M, Kupnicka P, Barczak K, Chlubek D, Baranowska-Bosiacka I. CXCR4 as a therapeutic target in acute myeloid leukemia. Leukemia. 2024 Nov;38(11):2303-2317. Martino EA, Bruzzese A, Labanca C, Mendicino F, Lucia E, Olivito V, Stanzione G, Zimbo A, Pozzi S, Neri A, Morabito F, Vigna E, Gentile M. Investigational CXCR4 inhibitors in early phase development for the treatment of hematological malignancies. Expert Opin Investig Drugs. 2024 Sep;33(9):915-924.Yang J, Tian E, Chen L, Liu Z, Ren Y, Mao W, Zhang Y, Zhang J. Development and therapeutic perspectives of CXCR4 antagonists for disease therapy. 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CSCO会议

2025-06-16

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X4 Pharmaceuticals Presents Positive Phase 2 Chronic Neutropenia Trial Data in Poster Presentations at the 30th Annual Congress of the European Hematology Association (EHA)

Poster #1654 highlights clinically meaningful and durable increases in circulating neutrophils observed with mavorixafor treatment +/- G-CSF over 6-month study Poster #1669 highlights findings that investigators were willing and able to reduce injectable G-CSF while maintaining ANC levels in the normal range Data continue to strengthen confidence in potential success of ongoing global, pivotal Phase 3 trial evaluating mavorixafor in primary chronic neutropenia BOSTON, June 16, 2025 (GLOBE NEWSWIRE) -- X4 Pharmaceuticals (Nasdaq: XFOR), a company driven to improve the lives of people with rare diseases of the immune system, today announced the presentation of positive data from its completed Phase 2 trial of mavorixafor in the treatment of certain chronic neutropenic (CN) conditions at the 30th Annual Congress of the European Hematology Association (EHA) in Milan, Italy. “We are very proud to have sponsored this trial – the first of its kind – evaluating an oral agent for the chronic treatment of certain primary chronic neutropenic disorders,” said Christophe Arbet-Engels, M.D., Ph.D., Chief Medical Officer of X4 Pharmaceuticals. “In addition to helping us design our ongoing Phase 3 pivotal clinical trial of mavorixafor in CN, the trial also provided a first glimpse into the potential real-world use of mavorixafor in a market currently served by only one approved therapy, with both clinical investigators and participants willing and then able to significantly reduce or discontinue the dose of injectable granulocyte colony-stimulating factor, or G-CSF. The results from this Phase 2 study continue to strengthen our confidence in a successful conclusion to our Phase 3 trial, which we expect will fully enroll in the third or fourth quarter of this year.” Julia A. Warren, M.D., Ph.D., study investigator and hematologist within both the Department of Pediatrics, Division of Hematology, Perelman School of Medicine, University of Pennsylvania, and the Division of Pediatric Hematology, Children’s Hospital of Philadelphia, commented on the results: “I was pleased to participate in this clinical trial and to present the results at this year’s EHA meeting. I am grateful to the participants with neutropenia who are currently enrolling in the Phase 3 clinical trial, as this rigorous study will help us understand which neutropenia patients will ultimately benefit from this drug should it gain regulatory approval.” The Phase 2 study of oral, once-daily mavorixafor was a six-month, open-label clinical trial that enrolled a total of 23 participants diagnosed with idiopathic, congenital, or cyclic chronic neutropenia, and had two subsets: mavorixafor monotherapy (n = 10 at baseline) and mavorixafor in combination with injectable G-CSF (n=13 at baseline). The two poster presentations at EHA today focused on clinical results based on the following goals of the study: Assessing safety and the ability of oral, once-daily mavorixafor to sustainably raise absolute neutrophil count (ANC) as a monotherapy and in combination with injectable G-CSF; and Assessing whether treatment of participants with mavorixafor could enable the reduction of G-CSF dosage while maintaining clinically targeted ANC levels and physicians’ willingness to do so. Key Findings from the Poster Presentations: Results from participants receiving oral, once-daily mavorixafor monotherapy showed that mavorixafor durably increased mean ANC from baseline over the 6-month trial. Further analysis showed that those with severe CN achieved nearly 3-fold increases in mean ANC levels out to six months, reaching levels typically targeted by physicians for patients with severe chronic neutropenia. The majority of participants (89%) and investigators were willing and elected to substantially reduce or discontinue injectable G-CSF use with oral mavorixafor treatment. All participants with congenital neutropenia concurrently treated with G-CSF, including one with the ELANE variant, were able to decrease G-CSF while maintaining mean ANC at normal levels. This study provides the first evidence that a significant subset of patients with primary CN may be able to successfully transition off injectable G-CSF to mavorixafor, potentially providing the first oral option to treat chronic neutropenia. In addition, a sub-study comparing the mean percentage of functional neutrophils in samples from healthy donors (n=5) to participants from the Phase 2 CN study (n=9) showed that the mean percentage of functional circulating neutrophils in CN participants in this sub-study was comparable to that of healthy donors after six months of mavorixafor dosing. Mavorixafor was generally well tolerated as monotherapy and in combination with injectable G-CSF during the trial, with no drug-related serious adverse events reported; the overall safety profile observed in the study was consistent with previous clinical studies of mavorixafor. Further analysis showed that those with severe CN achieved nearly 3-fold increases in mean ANC levels out to six months, reaching levels typically targeted by physicians for patients with severe chronic neutropenia. This study provides the first evidence that a significant subset of patients with primary CN may be able to successfully transition off injectable G-CSF to mavorixafor, potentially providing the first oral option to treat chronic neutropenia. X4 is currently conducting a global, pivotal Phase 3 trial in chronic neutropenia to assess the ability of mavorixafor to safely and durably increase ANC levels while also reducing participants’ annualized infections rates over a 52-week period. About Chronic Neutropenia and Mavorixafor Chronic neutropenia is a rare blood condition lasting more than three months and characterized by persistently or intermittently low levels of circulating neutrophils, increased risk of infections, and reduced quality of life. Neutrophils are retained in the bone marrow by the CXCR4/CXCL12 axis, creating a reserve of cells. Downregulation of the CXCR4 receptor by mavorixafor, an orally active CXCR4 antagonist, has been shown to mobilize functional neutrophils from the bone marrow into the peripheral blood across multiple disease states. The level of circulating neutrophils is typically measured by drawing blood to determine the absolute neutrophil count (ANC). About the Phase 1b/Phase 2 Chronic Neutropenia Trial The Phase 1b/Phase 2 clinical trial (NCT04154488) was a proof-of-concept, open-label, multicenter study designed to assess oral mavorixafor, with or without injectable G-CSF, in participants with chronic neutropenic disorders, including idiopathic, cyclic, and congenital neutropenia. In the Phase 1b portion of the study (n=25), participants received one dose of oral mavorixafor and were assessed for magnitude of absolute neutrophil count (ANC) response and tolerability. The Phase 2 portion of the trial (n=23) assessed the safety, tolerability, and the impact on participants’ neutropenia of oral, once-daily mavorixafor with and without concurrent injectable G-CSF therapy over a six-month period in the same patient population. About the 4WARD Global, Pivotal Phase 3 Clinical Trial The 4WARD trial is a global, pivotal Phase 3 clinical trial (NCT06056297) evaluating the efficacy, safety, and tolerability of oral, once-daily mavorixafor (with or without G-CSF) in people with congenital, acquired primary autoimmune, or idiopathic chronic neutropenia who are experiencing recurrent and/or serious infections. The 52-week trial is a randomized, double-blind, placebo-controlled, multicenter study aiming to enroll 150 participants with confirmed trough ANC levels less than 1,000 cells per microliter at baseline screening and histories of two or more serious and/or recurrent infections in the prior year. The primary endpoint of the trial is based on two outcome measures: annualized infection rate and positive ANC response. About X4 Pharmaceuticals X4 is delivering progress for patients by developing and commercializing innovative therapies for those with rare diseases of the immune system and significant unmet needs. Leveraging expertise in CXCR4 and immune system biology, X4 has successfully developed mavorixafor, an orally available CXCR4 antagonist that is currently being marketed in the U.S. as XOLREMDI® in its first indication. The company is also evaluating additional uses of mavorixafor and is conducting a global, pivotal Phase 3 clinical trial (4WARD) in people with certain chronic neutropenic disorders. X4 is headquartered in Boston, Massachusetts. For more information, please visit www.x4pharma.com. X4 Forward Looking Statements This press release contains forward-looking statements within the meaning of applicable securities laws, including the Private Securities Litigation Reform Act of 1995, as amended. These statements may be identified by the words “may,” “will,” “could,” “would,” “should,” “expect,” “plan,” “anticipate,” “intend,” “believe,” “estimate,” “predict,” “project,” “potential,” “continue,” “target,” or other similar terms or expressions that concern X4’s expectations, strategy, plans, or intentions. Forward-looking statements include, without limitation, implied or express statements regarding the initiation, timing, enrollment progress, and results of our current and future preclinical studies and clinical trials, including our ongoing pivotal Phase 3 clinical trial; the timing and period during which the results of the trials will become available and reported, as well as our research and development programs; and expectations regarding the commercial potential of mavorixafor and ongoing engagement and feedback from regulatory authorities. Any forward-looking statements in this press release are based on management’s current expectations and beliefs. These forward-looking statements are neither promises nor guarantees of future performance, and are subject to a variety of risks and uncertainties, many of which are beyond X4’s control, which could cause actual results to differ materially from those contemplated in these forward-looking statements, including the risks that: the expected availability, content, and timing of clinical data from X4’s ongoing clinical trials of mavorixafor may be delayed or unavailable, including the ongoing Phase 3 clinical trial; trials, studies and research programs may be delayed and may not have satisfactory outcomes, additionally earlier trials and studies may not be predictive of later trials and studies, including assessing the ability of mavorixafor monotherapy to durably increase absolute neutrophil count in patients with chronic neutropenic; the design and rate of enrollment for current clinical trials may not enable successful completion of the trial(s); X4 may be unable to obtain and maintain regulatory approvals; adverse safety effects may arise from the testing or use of X4’s product and product candidates; the need to align with collaborators may hamper or delay development and commercialization efforts or increase costs; and business may be adversely affected and clinical trials may be delayed if any of the company’s key collaborators fails to perform its obligations or terminates the collaboration; and other risks and uncertainties, including those described in the section entitled “Risk Factors” in X4’s most recent Annual Report on X4’s Form 10-K , as well as in other filings X4 makes with the Securities and Exchange Commission, including its quarterly report on Form 10-Q, from time to time. X4 undertakes no obligation to update the information contained in this press release to reflect new events or circumstances, except as required by law. Source: X4 Pharmaceuticals

临床结果临床3期临床2期

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KEGG	Wiki	ATC	Drug Bank
D11510	-	-	DB05501

研发状态

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适应症	国家/地区	公司	日期
WHIM综合征	美国	X4 Pharmaceuticals, Inc.	2024-04-26

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适应症	最高研发状态	国家/地区	公司	日期
常染色体隐性遗传性严重先天性中性粒细胞减少症3型	临床3期	美国	X4 Pharmaceuticals, Inc.	2024-06-06
常染色体隐性遗传性严重先天性中性粒细胞减少症3型	临床3期	阿根廷	X4 Pharmaceuticals, Inc.	2024-06-06
常染色体隐性遗传性严重先天性中性粒细胞减少症3型	临床3期	澳大利亚	X4 Pharmaceuticals, Inc.	2024-06-06
常染色体隐性遗传性严重先天性中性粒细胞减少症3型	临床3期	加拿大	X4 Pharmaceuticals, Inc.	2024-06-06
常染色体隐性遗传性严重先天性中性粒细胞减少症3型	临床3期	哥伦比亚	X4 Pharmaceuticals, Inc.	2024-06-06
常染色体隐性遗传性严重先天性中性粒细胞减少症3型	临床3期	捷克	X4 Pharmaceuticals, Inc.	2024-06-06
常染色体隐性遗传性严重先天性中性粒细胞减少症3型	临床3期	法国	X4 Pharmaceuticals, Inc.	2024-06-06
常染色体隐性遗传性严重先天性中性粒细胞减少症3型	临床3期	德国	X4 Pharmaceuticals, Inc.	2024-06-06
常染色体隐性遗传性严重先天性中性粒细胞减少症3型	临床3期	希腊	X4 Pharmaceuticals, Inc.	2024-06-06
常染色体隐性遗传性严重先天性中性粒细胞减少症3型	临床3期	匈牙利	X4 Pharmaceuticals, Inc.	2024-06-06

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研究	分期	人群特征	评价人数	分组	结果	评价	发布日期


PS1654 (EHA2025)	临床2期	中性粒细胞减少	-	Mavorixafor alone	夢膚選鹽鬱範壓觸製壓(製製窪鹽遞醖選繭願糧) = The most frequently reported treatment-related, treatment-emergent adverse events were gastrointestinal, all of mild or moderate severity 積鏇簾顧鑰網願製鏇壓 (齋積醖衊遞齋範淵淵淵 ) 更多	积极	2025-05-14
				Mavorixafor plus G-CSF
NCT04154488 (GlobeNewswire) 人工标引	临床2期	中性粒细胞减少	23	Mavorixafor monotherapyMavorixafor monotherapy	膚膚製餘繭鏇淵選遞繭(鬱蓋觸鏇獵遞獵築鏇憲) = 憲夢憲選齋齋願鏇壓積餘窪艱餘艱醖糧網積衊 (繭鹽遞鑰積繭壓鏇觸鹽 )	积极	2024-11-13
				Mavorixafor in combination with injectable G-CSFMavorixafor in combination with injectable G-CSF	膚膚製餘繭鏇淵選遞繭(鬱蓋觸鏇獵遞獵築鏇憲) = 觸艱範餘醖醖構積襯夢餘窪艱餘艱醖糧網積衊 (繭鹽遞鑰積繭壓鏇觸鹽 )
NCT03005327 (CTgov)	临床2期	WHIM综合征	8	X4P-001 (X4P-001 50 mg)	膚鬱餘壓觸鑰壓範鏇構(鹹窪鹹鬱觸夢鏇壓壓鑰) = 壓簾範鏇願餘選鏇顧繭艱網糧遞築糧襯憲鏇鹽 (醖簾觸簾衊鑰築鏇鹹繭, 156.094) 更多	-	2024-10-30
				X4P-001 (X4P-001 100 mg)	膚鬱餘壓觸鑰壓範鏇構(鹹窪鹹鬱觸夢鏇壓壓鑰) = 廠壓壓夢夢網網窪衊構艱網糧遞築糧襯憲鏇鹽 (醖簾觸簾衊鑰築鏇鹹繭, 4238.280) 更多
NCT02667886 (X4P-001-RCCA, CTgov)	临床1/2期	晚期肾细胞癌	74	X4P-001+Axitinib (X4P-001 200 mg BID With Axitinib)	範獵獵夢鑰餘鹽餘壓範 = 鏇淵範醖觸願觸顧艱齋廠廠憲餘遞壓蓋願艱簾 (觸鑰壓簾艱鹹艱築膚齋, 選壓鏇鹽壓夢積願膚廠 ~ 範膚顧鬱鬱製廠淵齋築) 更多	-	2024-08-14
				X4P-001+Axitinib (X4P-001 400 mg QD With Axitinib)	範獵獵夢鑰餘鹽餘壓範 = 簾憲餘鑰觸鏇遞醖鑰鑰廠廠憲餘遞壓蓋願艱簾 (觸鑰壓簾艱鹹艱築膚齋, 獵願網選選鹽鏇繭衊膚 ~ 窪衊鹹簾鹹襯醖築鹹糧) 更多
NCT03995108 (phase 3, Pubmed \| Blood)	临床3期	WHIM综合征 CXCR4 gain-of-function variants	31	Mavorixafor	糧襯鹽蓋淵蓋鬱鏇餘齋(蓋蓋觸糧願夢齋窪築願) = 淵獵衊齋艱製醖襯製廠衊選築憲衊齋襯艱膚鏇 (顧築範艱觸壓網鬱淵製 ) 更多	积极	2024-07-04
				Placebo	糧襯鹽蓋淵蓋鬱鏇餘齋(蓋蓋觸糧願夢齋窪築願) = 蓋製鏇夢簾鏇願構淵醖衊選築憲衊齋襯艱膚鏇 (顧築範艱觸壓網鬱淵製 ) 更多
NCT04154488 (Biospace) 人工标引	临床2期	中性粒细胞减少	23	Mavorixafor monotherapy	醖鹹艱艱餘繭壓獵憲窪(顧繭簾顧鹹壓觸艱窪壓) = 鏇鏇淵憲鹽艱積網積夢鏇鑰餘壓簾築積製醖鏇 (蓋糧繭醖餘壓鬱醖鑰遞 ) 更多	积极	2024-06-27
				Mavorixafor with stable-dose G-CSF	鹽齋糧選構廠簾壓鏇醖(艱構遞觸憲鏇選選構糧) = 獵構選網網憲構範願壓廠範艱網蓋憲範顧簾繭 (夢淵鹹願憲壓鏇製鬱餘 )
NCT03995108 (FDA_CDER) 人工标引	临床3期	WHIM综合征	31	XOLREMDI	構鏇顧鬱鬱壓蓋選餘窪(範艱鏇齋製鹽獵積衊積) = 顧糧糧觸選簾製憲獵遞醖鬱憲艱齋壓糧願簾齋 (遞壓壓廠醖廠構醖淵憲, 1.89) 更多	积极	2024-04-26
NCT03995108 (Phase 3 Trial of an Oral CXCR4 Antagonist, AAAAI2024)	临床3期	WHIM综合征	-	Mavorixafor	鹹糧製鏇膚襯遞夢鏇膚(範窪淵獵齋窪壓網膚艱) = 積網網壓膚膚蓋憲餘壓廠廠壓積觸觸壓製積遞 (衊膚網製壓鬱繭糧製築 ) 更多	积极	2024-02-23
				Placebo	鹹糧製鏇膚襯遞夢鏇膚(範窪淵獵齋窪壓網膚艱) = 鹽齋膚觸願築繭願廠範廠廠壓積觸觸壓製積遞 (衊膚網製壓鬱繭糧製築 ) 更多
NCT03995108 (EHA2023)	临床3期	WHIM综合征	31	Mavorixafor	淵憲餘築選範窪鹽顧鬱(願願膚憲蓋鑰獵願憲憲) = increased from baseline into normal range and sustained at each timepoint assessed over 52 weeks with mavorixafor versus placebo 淵獵蓋網簾蓋餘觸網艱 (壓製選憲鹹網築淵網膚 ) 更多	积极	2023-06-08
				Placebo
NCT02923531 (CTgov)	临床1/2期	局部晚期透明细胞肾细胞癌	9	Nivolumab+X4P-001	鑰壓願鏇選積積壓願鏇 = 獵鹹選築觸鏇積願壓襯蓋衊餘選選構築構襯製 (鑰齋鏇範製簾襯積鏇餘, 艱築鏇醖鹽願鹽遞鬱築 ~ 膚鑰淵鹽築壓網願觸繭) 更多	-	2022-12-29