A Dose Escalation Study Using Eflornithine (DFMO) and AMXT 1501 Followed by a Randomized Controlled Trial of DFMO With or Without AMXT 1501 for Neuroblastoma, CNS Tumors, and Sarcomas

The purpose of this study is to evaluate the investigational oral drug AMXT 1501 in combination with oral eflornithine (DFMO). An investigational drug is one that has not been approved by the U.S. Food & Drug Administration (FDA), or any other regulatory authorities around the world for use alone or in combination with any drug, for the condition or illness it is being used to treat.
The goals of this part of the study are:
* Establish a recommended dose of AMXT 1501 in combination with DFMO
* Test the safety and tolerability of AMXT 1501 in combination with DFMO
* To determine the activity of study treatments chosen based on:
* How each subject responds to the study treatment
* How long a subject lives without their disease returning/progressing

开始日期2026-04-28

申办/合作机构

Hershey Medical Center

[+2]

NCT07287917

/ Recruiting临床1/2期

A Phase 1b/2 Trial Investigating the Safety and Efficacy of Oral AMXT 1501 and Oral DFMO in Combination With Standard of Care in Patients With Advanced Solid Tumors Who Progressed After Prior Therapies

This study will evaluate the safety, tolerability, and preliminary effectiveness of AMXT 1501 and DFMO when combined with standard treatments for advanced solid tumors. The trial includes two groups:
* Cohort 1: Patients with ER+ / HER2- breast cancer receiving fulvestrant and capivasertib
* Cohort 2: Patients with unresectable or metastatic cutaneous melanoma receiving pembrolizumab
The Phase 1b portion will find the recommended Phase 2 dose (RP2D). The Phase 2 portion will further evaluate clinical activity at the RP2D using response criteria for solid tumors (RECIST 1.1).
The study will also evaluate pharmacokinetics, pharmacodynamics, disease control, and overall safety.

开始日期2026-01-26

申办/合作机构

Aminex Therapeutics, Inc.

NCT05717153

/ Recruiting早期临床1期IIT

Intratumoral Extracellular Metabolic Impact of DFMO and AMXT 1501

This early phase I trial studies brain tumor (glioma) metabolism in response to eflornithine (DFMO) and polyamine transport inhibitor AMXT-1501 dicaprate (AMXT 1501) in patients with diffused or high grade glioma. Brain tumors use and produce certain molecules to survive and grow. DFMO is an irreversible inhibitor of ornithine decarboxylase, the enzyme catalyzing polyamine synthesis. AMXT 1501 is a polyamine transport inhibitor which prevents uptake of polyamines from the extracellular environment. This trial is being done to analyze how DFMO and AMXT 1501 affect brain tumor metabolism based on the molecules in the tumor's fluid.

开始日期2023-10-01

申办/合作机构

Mayo Clinic

[+1]

100 项与 Dicaprate 相关的临床结果

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100 项与 Dicaprate 相关的转化医学

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100 项与 Dicaprate 相关的专利（医药）

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项与 Dicaprate 相关的文献（医药）

2025-09-01·ESMO Open

Phase I dose-escalation trial of AMXT 1501 dicaprate plus difluoromethylornithine: a dual-agent approach targeting immunosuppressive polyamine metabolism

Article

作者: Piha-Paul, S A ; Vandross, A L ; Tolcher, A W ; Burns, M R ; Spira, A I

BACKGROUND:

Dysregulation of polyamine synthesis has been observed in various cancer cell types. A novel approach to depriving cancer cells of polyamines involves the use of difluoromethylornithine (DFMO) to block polyamine biosynthesis in combination with AMXT 1501, a potent inhibitor of polyamine transport. Preclinical mouse tumor models showed that the combination of AMXT 1501 plus DFMO had strong antitumor activity, together with evidence of a stimulated immune response against tumors.

MATERIALS AND METHODS:

This was a multicenter phase I, open-label, two-part dose-escalation study, with expansion, to evaluate the safety and preliminary efficacy of oral AMXT 1501 in combination with oral DFMO in patients with advanced solid tumors. In part 1, patients were treated with ascending doses of AMXT 1501 alone for 2 weeks and then in combination with DFMO for an additional 2 weeks. In part 2, AMXT 1501 was dosed with escalating DFMO doses to determine the recommended phase II dose (RP2D), with an expansion cohort to confirm the RP2D. Patients with unresectable, locally advanced, or metastatic solid tumors for which no standard therapy was recognized were eligible.

RESULTS:

A total of 56 patients were enrolled and treated (20, 22, and 14 in part 1, part 2, and expansion, respectively). Patients were heavily pretreated; the median number of prior cancer treatments was 10.0. The most common treatment-emergent adverse events (TEAEs) were diarrhea (39.3%), nausea (37.5%), and vomiting (33.9%). There were no grade 4 or 5 TEAEs and no deaths due to TEAEs. Moderate antitumor activity was observed with 2 patients with confirmed responses and 16 patients with stable disease for an overall response rate of 6% and a clinical benefit rate of 49%.

CONCLUSIONS:

Overall, AMXT 1501 in combination with DFMO was safe and tolerated with evidence of preliminary clinical activity. The RP2D was determined to be AMXT 1501 600 mg twice daily plus DFMO 500 mg.

CLINICAL TRIAL IDENTIFICATION:

NCT03536728.

2025-06-15·INTERNATIONAL JOURNAL OF CANCER

Polyamine depletion limits progression of acute leukaemia

Article

作者: Verhelst, Steven H. L. ; Lock, Richard B. ; Norris, Murray D. ; Mayoh, Chelsea ; Spurling, Dayna ; Ronca, Emma ; Xiao, Lin ; Tanaka, Katsunori ; Somers, Klaartje ; Guo, Xinyi ; Azfar, Mujahid ; Cheung, Laurence C. ; Gao, Weiman ; Burns, Mark R. ; Haber, Michelle ; Karsa, Mawar ; Bongers, Angelika ; Kotecha, Rishi S. ; Vangheluwe, Peter ; Karsa, Ayu

Abstract:

Cancer cells are addicted to polyamines, polycations essential for cellular function. While dual targeting of cellular polyamine biosynthesis and polyamine uptake is under clinical investigation in solid cancers, preclinical and clinical studies into its potential in haematological malignancies are lacking. Here we investigated the preclinical efficacy of polyamine depletion in acute leukaemia. The polyamine biosynthesis inhibitor difluoromethylornithine (DFMO) inhibited growth of a molecularly diverse panel of acute leukaemia cell lines, while non‐malignant cells were unaffected. Responsiveness to DFMO was linked to decreased levels of its molecular target, the rate‐limiting polyamine biosynthesis enzyme ODC1, and of the polyamine transporters ATP13A2 and ATP13A3. DFMO increased polyamine uptake and upregulated expression of polyamine transporters in acute leukaemia cells, a compensatory effect abolished by treatment with the polyamine transport inhibitor AMXT 1501. This drug, currently in a phase 1 clinical trial in solid tumours in combination with DFMO, potentiated the inhibitory effects of DFMO, and their combination synergistically inhibited the growth of acute leukaemia cell lines by inducing apoptosis. DFMO and AMXT 1501 limited disease progression in highly aggressive xenograft models of infant KMT2A‐rearranged leukaemia, even when treatment was initiated at high disease burden. Increased expression of c‐MYC was associated with enhanced sensitivity to the combination of DFMO and AMXT 1501, suggesting this oncoprotein as a potential predictive marker of response to the drug combination. In conclusion, targeting polyamine biosynthesis and polyamine uptake limits disease progression in models of acute leukaemia, supporting further preclinical and clinical investigation into this approach for acute leukaemia.

2025-03-01·Molecular Oncology

The polyamine transporter ATP13A3 mediates difluoromethylornithine‐induced polyamine uptake in neuroblastoma

Article

作者: Chris Van den Haute ; Weiman Gao ; Ayu Karsa ; Steven H. L. Verhelst ; Mawar Karsa ; Mujahid Azfar ; Peter Vangheluwe ; Emma Ronca ; Murray D. Norris ; Klaartje Somers ; Michelle Haber ; Ruby Pandher ; Chelsea Mayoh ; Dayna Spurling ; Xinyi Guo ; Angelika Bongers ; Lin Xiao ; Youri Fayt ; Arthur Schoofs ; Mark R. Burns

High‐risk neuroblastomas, often associated with MYCN protooncogene amplification, are addicted to polyamines, small polycations vital for cellular functioning. We have previously shown that neuroblastoma cells increase polyamine uptake when exposed to the polyamine biosynthesis inhibitor difluoromethylornithine (DFMO), and this mechanism is thought to limit the efficacy of the drug in clinical trials. This finding resulted in the clinical development of polyamine transport inhibitors, including AMXT 1501, which is presently under clinical investigation in combination with DFMO. However, the mechanisms and transporters involved in DFMO‐induced polyamine uptake are unknown. Here, we report that knockdown of ATPase 13A3 (ATP13A3), a member of the P5B‐ATPase polyamine transporter family, limited basal and DFMO‐induced polyamine uptake, attenuated MYCN‐amplified and non‐MYCN‐amplified neuroblastoma cell growth, and potentiated the inhibitory effects of DFMO. Conversely, overexpression of ATP13A3 in neuroblastoma cells increased polyamine uptake, which was inhibited by AMXT 1501, highlighting ATP13A3 as a key target of the drug. An association between high ATP13A3 expression and poor survival in neuroblastoma further supports a role of this transporter in neuroblastoma progression. Thus, this study identified ATP13A3 as a critical regulator of basal and DFMO‐induced polyamine uptake and a novel therapeutic target for neuroblastoma.

项与 Dicaprate 相关的新闻（医药）

2026-03-14

·PRNewswire

Aminex Therapeutics Receives Second FDA Orphan Drug Designation for AMXT 1501 for Malignant Glioma Including DIPG - a Highly Aggressive Childhood Brain Cancer

- Designation Underscores the Significant Unmet Medical Need as AMXT 1501 Advances in Two Active National Clinical Trials — Including a Pediatric Study Now Enrolling Children with DIPG March 12, 2026 -- Aminex Therapeutics, Inc., a clinical-stage biotechnology company focused on developing a novel metabolic-targeted therapy to treat adult and pediatric cancers, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to AMXT 1501 in combination with difluoromethylornithine (DFMO) for the treatment of malignant glioma, including diffuse intrinsic pontine glioma (DIPG). This is the company's second Orphan Drug Designation, following the ODD granted in October 2025 for neuroblastoma. "This second Orphan Drug Designation is a powerful validation of AMXT 1501's potential to make a meaningful difference for patients facing some of the most devastating cancers," said Mark Burns, PhD, Chief Scientific Officer and President of Aminex Therapeutics. "DIPG is heartbreaking — children diagnosed with this brain tumor have virtually no effective treatment options and very little time. We are committed to changing that." DIPG is an aggressive brainstem tumor that primarily strikes children aged 5 to 10, with a median survival of less than 12 months from diagnosis. There is currently no cure. For Families of Children with DIPG and Other High-Risk Cancers: A Trial Is Now Enrolling The Beat Childhood Cancer Research Consortium at Penn State College of Medicine, in partnership with Aminex, is actively enrolling patients in a national Phase 1/2 clinical trial of AMXT 1501 plus DFMO in pediatric patients with DIPG, neuroblastoma, sarcomas and other high-risk childhood cancers. The trial is planned to open at 50 clinics nationwide and enroll patients. AMXT 1501 is also being evaluated in adults with solid tumors including breast cancer and metastatic melanoma in an active Phase 1b/2 multicenter trial (NCT07287917.) The FDA's Orphan Drug Designation provides Aminex with development incentives including tax credits, fee waivers, and seven years of market exclusivity upon potential approval. Aminex Therapeutics, Inc. is a clinical-stage biotechnology company developing AMXT 1501, a novel small molecule polyamine transport inhibitor, in combination with DFMO, a polyamine synthesis inhibitor. The content above comes from the network. if any infringement, please contact us to modify.

孤儿药临床研究

2026-02-08

·药明康德

三特异性NK细胞衔接器、创新JAK2变构抑制剂获FDA批准进入临床…… | 一周盘点

本期看点： 1. GT Biopharma公司宣布，其新型B7-H3靶向三特异性自然杀伤（NK）细胞衔接器GTB-5550的IND申请已获得美国FDA批准，将在7种不同的转移性癌症队列中开展研究。 2. Prelude Therapeutics公司宣布，其新型JAK2V617F突变选择性抑制剂PRT12396的IND申请已获美国FDA批准，1期临床试验将在高危真红细胞增多症（PV）以及中高危骨髓纤维化（MF）患者中开展。 GTB-5550：IND申请获得FDA许可 GT Biopharma公司宣布，其新型候选药物GTB-5550的IND申请已获得美国FDA批准。GTB-5550是一种靶向B7-H3的三特异性NK细胞衔接器，用于治疗表达B7-H3抗原的实体肿瘤。该分子由三种组分组成，通过柔性接头连接：1）靶向NK细胞表面CD16激活受体的纳米体臂；2）野生型IL-15（WT IL-15）连接臂，用于促进NK细胞的增殖、活化和存活；3）靶向肿瘤细胞上B7-H3抗原的纳米体臂，从而将NK细胞精准引导至肿瘤部位。计划开展的1期临床试验共分为两个部分：1a期为剂量递增阶段，将评估最多6个剂量水平以确定最大耐受剂量（MTD）；1b期为剂量扩展阶段，将在7种不同的转移性癌症队列中（包括去势抵抗性前列腺癌、卵巢癌、乳腺癌、头颈癌、非小细胞肺癌、胰腺癌和膀胱癌）验证1a期研究确定的MTD，并进一步评估药物的耐受性。 PRT12396：IND申请获得FDA许可 Prelude Therapeutics公司宣布，其新型JAK2V617F突变选择性抑制剂PRT12396的IND申请已获美国FDA批准，将启动一项1期临床研究。该研究为开放标签、多中心试验，旨在评估PRT12396在高危真红细胞增多症以及中高危骨髓纤维化患者中的安全性、初步疗效和药代动力学特征。公司预计将于2026年第二季度完成首例患者给药。 PRT12396是Prelude公司设计并验证的新型变构抑制剂，能够结合到V617F突变所在的JAK2 JH2“深口袋”。该突变是骨髓增殖性肿瘤（MPNs）的主要驱动突变，在约95%的PV、60%的原发性血小板增多症（ET）和55%的MF患者中存在。Prelude公司开发的这类变构抑制剂在MPN临床前模型中展现了突变特异性抑制作用，有望降低突变等位基因负荷、延缓甚至逆转疾病进展，从而改善MPN患者的治疗结果。 AMXT 1501：启动1b/2期联合治疗试验 Aminex Therapeutics公司宣布启动一项AMXT 1501联合二氟甲基鸟氨酸（DFMO）的1b/2期临床试验。这项多中心、开放标签研究将评估口服AMXT 1501与口服DFMO联合标准治疗在两类人群中的安全性、耐受性和初步疗效，包括既往治疗失败、绝经前/后、雌激素受体（ER）阳性、人表皮生长因子受体2（HER2）阴性乳腺癌和转移性黑色素瘤患者。 AMXT 1501是一种新型多胺转运抑制剂，旨在阻断肿瘤细胞摄取外源性多胺——这些分子对肿瘤生长和存活至关重要。DFMO是一种已知的多胺生物合成抑制剂。两者联合旨在全面抑制多胺代谢，从而抑制肿瘤生长。参考资料： [1] Onchilles Pharma Announces IND Clearance for N17350, Advancing the First Next-Generation Cytotoxic Therapeutic Leveraging the ELANE Pathway into the Clinic. Retrieved February 6, 2026, from https://www.onchillespharma.com/post/onchilles-pharma-announces-ind-clearance-for-n17350 [2] GT Biopharma Announces FDA Clearance of Investigational New Drug (IND) Application for GTB-5550 TriKE®, a B7-H3-Targeted Natural Killer (NK) Cell Engager for Solid Tumors Expressing B7-H3. Retrieved February 6, 2026, from https://www.globenewswire.com/news-release/2026/02/03/3231077/0/en/GT-Biopharma-Announces-FDA-Clearance-of-Investigational-New-Drug-IND-Application-for-GTB-5550-TriKE-a-B7-H3-Targeted-Natural-Killer-NK-Cell-Engager-for-Solid-Tumors-Expressing-B7-H.html [3] Prelude Therapeutics Receives FDA Clearance of Investigational New Drug Application (IND) for PRT12396, a Mutant-selective JAK2V617F Inhibitor. Retrieved February 6, 2026, from https://www.globenewswire.com/news-release/2026/02/03/3231175/0/en/Prelude-Therapeutics-Receives-FDA-Clearance-of-Investigational-New-Drug-Application-IND-for-PRT12396-a-Mutant-selective-JAK2V617F-Inhibitor.html [4] Affinia Therapeutics Announces FDA Acceptance of IND Application to Advance AFTX-201 to a Phase 1/2 Trial for the Treatment of BAG3-Associated Dilated Cardiomyopathy (DCM). Retrieved February 6, 2026, from https://affiniatx.com/affinia-therapeutics-announces-fda-acceptance-of-ind-application-to-advance-aftx-201-to-a-phase-1-2-trial-for-the-treatment-of-bag3-associated-dilated-cardiomyopathy-dcm/ [5] PharmaResearch Receives U.S. FDA Clearance to Initiate Phase 1 Clinical Trial of Nano Anticancer Drug, PRD-101. Retrieved February 6, 2026, from https://www.prnewswire.com/news-releases/pharmaresearch-receives-us-fda-clearance-to-initiate-phase-1-clinical-trial-of-nano-anticancer-drug-prd-101-302681109.html [6] Spur Therapeutics Presents New Phase 1/2 Data on Its Gene Therapy Candidate in Gaucher Disease at 22nd Annual WORLDSymposium™. Retrieved February 6, 2026, from https://www.globenewswire.com/news-release/2026/02/04/3231939/0/en/Spur-Therapeutics-Presents-New-Phase-1-2-Data-on-Its-Gene-Therapy-Candidate-in-Gaucher-Disease-at-22nd-Annual-WORLDSymposium.html [7] Aminex Therapeutics Announces Multiple Sites Activated for Phase 1b/2 Clinical Trial of Novel Investigational Cancer Treatment AMXT 1501 and DFMO in Patients with Breast Cancer or Melanoma. Retrieved February 6, 2026, from https://www.prnewswire.com/news-releases/aminex-therapeutics-announces-multiple-sites-activated-for-phase-1b2-clinical-trial-of-novel-investigational-cancer-treatment-amxt-1501-and-dfmo-in-patients-with-breast-cancer-or-melanoma-302678969.html [8] Neomorph Announces First Patient Dosed in Phase 1/2 Trial Evaluating NEO-811 For the Treatment of Locally Advanced or Metastatic Non-Resectable Clear Cell Renal Cell Carcinoma. Retrieved February 6, 2026, from https://www.globenewswire.com/news-release/2026/02/03/3231475/0/en/Neomorph-Announces-First-Patient-Dosed-in-Phase-1-2-Trial-Evaluating-NEO-811-For-the-Treatment-of-Locally-Advanced-or-Metastatic-Non-Resectable-Clear-Cell-Renal-Cell-Carcinoma.html [9] Ultragenyx Announces Positive Longer-Term Data Demonstrating Treatment with UX111 Gene Therapy Results in Sustained, Significant Reductions in CSF-HS and Continued Meaningful Improvements in Clinical Function Across Multiple Developmental Domains in Children with Sanfilippo Syndrome (MPS IIIA). Retrieved February 6, 2026, from https://ir.ultragenyx.com/news-releases/news-release-details/ultragenyx-announces-positive-longer-term-data-demonstrating [10] uniQure Announces Updated Preliminary AMT-191 Phase I/IIa Data Showing Sustained Increases in α-Gal A Enzyme Activity in Patients with Fabry Disease. Retrieved February 6, 2026, from https://www.globenewswire.com/news-release/2026/02/06/3233740/0/en/uniQure-Announces-Updated-Preliminary-AMT-191-Phase-I-IIa-Data-Showing-Sustained-Increases-in-%CE%B1-Gal-A-Enzyme-Activity-in-Patients-with-Fabry-Disease.html 免责声明：本文仅作信息交流之目的，文中观点不代表药明康德立场，亦不代表药明康德支持或反对文中观点。本文也不是治疗方案推荐。如需获得治疗方案指导，请前往正规医院就诊。版权说明：欢迎个人转发至朋友圈，谢绝媒体或机构未经授权以任何形式转载至其他平台。转载授权请在「药明康德」微信公众号回复“转载”，获取转载须知。分享，点赞，在看，聚焦全球生物医药健康创新

2025-11-04

·盛诺一家

盘点｜10月美国加速审评18种抗癌新药/新疗法，多个癌种迎来治疗新突破！

本文由盛诺一家原创编译，转载需经授权 2025年10月是肿瘤治疗领域非常重要的一个月，美国食品药品监督管理局（FDA）接连发布了一系列关键审批决定。这期间，多个针对不同癌症类型的药物获得了“完全批准”或“加速批准”，为实体瘤和血液系统恶性肿瘤患者带来了更多治疗选择。 ⏳点击跳转，查看详情：《盘点｜10月美国重磅批准5款抗癌新疗法，为多种癌症患者带来新曙光！》此外，FDA继续通过授予“快速通道”、“孤儿药”、“优先审评”和“突破性疗法”资格，加速有前景新药的研发。这些动态显示出未来癌症治疗领域将迎来一批创新疗法。本文将盘点这些对肿瘤治疗格局有重大影响的FDA新动向。来源：FIERCE Biotech网站 FDA的“快速通道认证”，旨在加速药物研发和审批，提供更频繁的FDA沟通机会，并使药物有资格获得加速批准或优先审评。 FDA授予“孤儿药”资格的药物和生物制剂，旨在为罕见疾病的治疗、诊断或预防提供安全有效的解决方案。获得该资格的药物将享受一系列激励政策。 FDA授予的“优先审评”资格，意味着该药物若获批，将显著优于现有疗法，比如更安全、更有效，或能预防严重疾病，或能提高患者依从性（让患者更好地按照医生的建议去做治疗和随访）。 FDA的“突破性疗法”认定，用于加速开发和审评在严重或危及生命疾病中相较现有疗法有显著改善潜力的药物。 1、FDA受理T-DXd联合THP用于HER2阳性乳腺癌的补充申请（sBLA） 10月1日，FDA受理了T-DXd（Enhertu，德曲妥珠单抗）联合紫杉醇（Taxol）、曲妥珠单抗（Herceptin）和帕妥珠单抗（Perjeta）（简称THP方案），用于HER2阳性II或III期乳腺癌成人患者新辅助治疗的补充生物制品许可申请（sBLA）。该申请的目标审评截止日期（PDUFA日期）定为2026年5月18日，将在此之前做出审评决定。 T-DXd是由日本第一三共（Daiichi Sankyo）研发的一款抗体偶联药物（ADC）。截至目前，该药已在多个国家/地区获批上市，包括中国。 2、FDA授予ETX-636在PIK3CA突变、HR阳性乳腺癌中的快速通道资格 10月1日，FDA授予ETX-636授予快速通道资格，用于治疗携带PIK3CA突变的激素受体阳性（HR+）、HER2阴性晚期乳腺癌患者。来源：ChatGPT生成AI示意图 ETX-636是由美国Ensem Therapeutics公司研发的一种靶向PIK3CA突变的变异特异性别构调节抑制剂。目前该药仍处于临床开发阶段，尚未在全球任何国家/地区获批上市。 ⏳点击跳转，查看详情：《PIK3CA突变乳腺癌患者新希望：美国新药ETX-636获快速通道资格！》 3、AMXT 1501与DFMO联合获FDA孤儿药资格，用于神经母细胞瘤 10月2日，FDA授予AMXT 1501与DFMO（商品名Iwilfin，学名difluoromethylornithine）联合用于神经母细胞瘤的孤儿药资格。来源：摄图网 AMXT 1501是一种新型的“多胺转运抑制剂”，其作用机制是阻断肿瘤细胞对多胺的吸收，而多胺是肿瘤生长和存活所必需的物质。目前该药仍处于临床开发阶段，尚未在全球任何国家/地区获批上市。 ⏳点击跳转，查看详情：《AMXT 1501联合DFMO获孤儿药资格，为神经母细胞瘤患者带来新希望》 4、Orca-T获FDA优先审评资格，用于血液系统恶性肿瘤 10月6日，FDA受理了Orca-T的生物制品许可申请（BLA），并授予其优先审评资格，用于治疗血液系统恶性肿瘤。 Orca-T是一种异体T细胞免疫疗法，由美国Orca Bio公司开发，用于治疗血液系统恶性肿瘤，包括急性髓系白血病（AML）、急性淋巴细胞白血病（ALL）及骨髓增生异常综合征（MDS）。目前该药仍处于临床开发阶段，尚未在全球任何国家/地区获批上市。 FDA设定的PDUFA目标审评日期为2026年4月6日，将在此之前做出审评决定。 5、WTX-124获FDA快速通道资格，用于难治性黑色素瘤 10月8日，FDA授予WTX-124快速通道资格，用于治疗经过标准免疫治疗后仍进展的局部晚期或转移性皮肤黑色素瘤患者。 WTX-124是由美国Werewolf Therapeutics公司研发的一款条件激活型白介素-2（IL-2）免疫疗法。截至目前，该药尚未在任何国家/地区获批上市。 6、FDA授予ADCE-D01在软组织肉瘤中的快速通道资格 10月9日，FDA授予ADCE-D01快速通道资格，用于治疗软组织肉瘤。 ADCE-D01是由丹麦Adcendo ApS公司开发的一种“首创”抗体偶联药物（ADC），靶向uPARAP（尿激酶型纤溶酶原激活物受体相关蛋白）。截至目前，该药尚未在任何国家/地区获批上市。 7、Ficerafusp Alfa获FDA突破性疗法资格，用于HPV阴性头颈癌 10月13日，FDA授予Ficerafusp Alfa（代号BCA101）与帕博利珠单抗（Keytruda）联合，用于HPV阴性、复发或转移性头颈鳞状细胞癌（HNSCC）一线治疗的突破性疗法资格。 Ficerafusp Alfa是由美国Bicara Therapeutics公司研发的一款双功能抗体药物，同时靶向EGFR（表皮生长因子受体）和 TGF-β（转化生长因子β）。截至目前，该药尚未在任何国家/地区获批上市。 8、FDA授予Sonrotoclax用于复发/难治型套细胞淋巴瘤的突破性疗法资格 10月14日，FDA授予Sonrotoclax（代号BGB-11417）突破性疗法资格，用于经BTK抑制剂及抗CD20药物治疗后复发或难治的成人套细胞淋巴瘤（MCL）患者。 Sonrotoclax是由瑞士BeOne Medicines公司研发的新一代BCL-2抑制剂。截至目前，该药尚未在任何国家/地区获批上市。 9、FDA授予NG-350A快速通道资格，用于pMMR型直肠癌 10月14日，FDA授予NG-350A快速通道资格，用于治疗错配修复功能正常（pMMR）的局部晚期直肠癌患者。来源：ChatGPT生成AI示意图 NG-350A是由美国Akamis Bio公司研发的一款溶瘤免疫疗法。截至目前，该药尚未在任何国家/地区获批上市。 ⏳点击跳转，查看详情：《直肠癌新疗法NG-350A获快速通道资格，让更多患者有机会免于手术！》 10、FDA授予MNV-201孤儿药资格，用于低危骨髓增生异常综合征（MDS） 10月15日，FDA授予MNV-201孤儿药资格，用于治疗骨髓增生异常综合征（MDS）。 MNV-201是由以色列Minovia Therapeutics公司研发的一款线粒体细胞疗法。截至目前，该药尚未在任何国家/地区获批上市。 11、癌症疫苗EO2463获FDA快速通道资格，用于滤泡性淋巴瘤 10月16日，FDA授予创新免疫疗法EO2463快速通道资格，用于治疗滤泡性淋巴瘤。来源：ChatGPT生成AI示意图 EO2463是由法国Enterome公司研发的一种OncoMimics™癌症疫苗，属于免疫疗法。截至目前，该疗法尚未在任何国家／地区获批上市。 ⏳点击跳转，查看详情：《46%患者肿瘤显著缩小或完全消失！癌症疫苗EO2463治疗淋巴瘤疗效突出，获加速审批》 12、FDA受理XS003用于慢性髓性白血病的新药申请 10月21日，FDA受理XS003的新药申请（NDA），用于治疗慢性髓性白血病（CML）。 XS003是由瑞典Xspray Pharma AB公司研发的一种以尼洛替尼（Tasigna）为参考的酪氨酸激酶抑制剂（TKI）新制剂。 FDA设定的PDUFA目标日期为2026年6月18日，将在此之前做出审评决定。 13、FDA授予Enfortumab Vedotin联合帕博利珠单抗，在MIBC中的优先审评资格 10月22日，FDA授予Enfortumab Vedotin-ejfv（Padcev）联合帕博利珠单抗（Keytruda），用于接受根治性膀胱切除术及标准盆腔淋巴结清扫术患者的补充申请（sBLA）优先审评资格。适应症为不适合或拒绝含铂化疗的肌层浸润性膀胱癌（MIBC）患者的围手术期治疗。来源：博鳌乐城医疗先行区中心药房公众号 Padcev是一种新型抗体偶联药物（ADC），由美国Seagen公司和日本安斯泰来（Astellas）制药公司共同研发。截至目前，该药已在多个国家/地区获批上市，包括中国。 14、FDA授予MT-125快速通道资格，用于胶质母细胞瘤 10月22日，FDA授予MT-125快速通道资格，用于治疗胶质母细胞瘤（GBM）。 MT-125由美国Myosin Therapeutics研发，是一种首创双靶点小分子药物。截至目前，该药尚未在任何国家/地区获批上市。 ⏳点击跳转，查看详情：《胶质母细胞瘤新药MT-125获美国药监局授予“快速通道”资格，年底将开展人体试验》 15、Pamlectabart Tismanitin获FDA快速通道资格，用于多发性骨髓瘤 10月23日，FDA授予Pamlectabart Tismanitin（代号HDP-101）快速通道资格，用于治疗复发或难治性多发性骨髓瘤患者。 Pamlectabart Tismanitin是由德国 Heidelberg Pharma AG公司研发的一款新型抗体偶联药物（ADC）。截至目前，该药尚未在任何国家/地区获批上市。 16、FDA授予Zenocutuzumab突破性疗法资格，用于NRG1融合型胆管癌 10月24日，FDA授予Zenocutuzumab（商品名Bizengri）突破性疗法资格，用于治疗带有NRG1基因融合的晚期或不可手术胆管癌患者。来源：ChatGPT生成AI示意图 Zenocutuzumab是由荷兰Merus N.V.公司研发的一款双特异性抗体药物，靶向HER2和HER3受体，用于治疗携带NRG1基因融合的实体瘤。截至目前，该药已在美国获批上市，用于接受过系统治疗后、携带NRG1基因融合的晚期不可切除或转移性非小细胞肺癌（NSCLC）或胰腺腺癌成人患者。该药在中国大陆尚未获批上市。 ⏳点击跳转，查看详情：《新一代抗癌药Zenocutuzumab获突破性疗法认定，为罕见胆管癌患者带来新希望！》 17、Daraxonrasib获FDA孤儿药资格，用于胰腺癌 10月27日，FDA授予Daraxonrasib（代号RMC-6236）孤儿药资格，用于治疗胰腺癌。来源：ChatGPT生成AI示意图 Daraxonrasib（研发代号RMC-6236）是由美国Revolution Medicines公司开发的一种口服、直接作用的RAS（ON）多靶点抑制剂，具有潜力治疗多种由RAS致癌突变驱动的肿瘤。截至目前，该药尚未在任何国家/地区获批上市。 18、FDA授予ZEN-3694孤儿药资格，用于NUT癌 10月27日，FDA授予ZEN-3694孤儿药资格，用于治疗罕见的NUT癌。 ZEN3694是由加拿大Zenith Epigenetics Ltd.公司研发的一种口服BET溴结构域抑制剂（BETi）。截至目前，该药尚未在任何国家/地区获批上市。编者按从今年10月FDA密集加速审评的抗癌新药/新疗法可以看出，全球肿瘤治疗正进入一个“精准与突破并行”的新时代——无论是针对罕见癌症的孤儿药、加速审批的免疫疗法，还是新一代抗体偶联药物（ADC）与细胞疗法等，都在不断刷新患者的生存曲线。盛诺一家始终关注全球最前沿的抗癌药物与治疗进展，并已与美国、英国、日本等多个国家的知名医院建立官方签约合作关系，致力于帮助中国患者第一时间了解并获得这些创新药物和疗法。如果您或家人正面临复杂、难治的癌症诊疗抉择，想了解更多癌症等重大、复杂疾病的海外前沿研究成果、药物、疗法信息，请扫描下方二维码联系我们，盛诺一家的全球就医规划师可为您提供专业的就医路径规划和全球优质医疗资源快速对接服务，包括预约国外权威专家一对一远程咨询、快速办理医疗签证前往海外权威医院就医等。请在备注中输入口令：11042 资料来源： https://www.targetedonc.com/view/october-2025-a-look-at-fda-oncology-approvals-and-designations 关于我们盛诺一家只做一件事情，就是为国内患者在全球范围内寻找就医途径。只要人类还有办法，不论是创新的药物还是疗法，不论是在美国哈佛，还是英国、日本著名医院，我们都会快速找到，将信息提供给患者，帮助患者快速和全球专家视频咨询，或直接飞到发达国家进行治疗。很多患者因此重获新生。点赞、转发、收藏，一键三连！

突破性疗法快速通道优先审批孤儿药

100 项与 Dicaprate 相关的药物交易

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研发状态

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适应症	最高研发状态	国家/地区	公司	日期
脑干胶质瘤	临床2期	美国	Aminex Therapeutics, Inc.	2026-04-28
室管膜母细胞瘤	临床2期	美国	Aminex Therapeutics, Inc.	2026-04-28
尤文肉瘤	临床2期	美国	Aminex Therapeutics, Inc.	2026-04-28
复发性神经母细胞瘤	临床2期	美国	Aminex Therapeutics, Inc.	2026-04-28
骨肉瘤	临床2期	美国	Aminex Therapeutics, Inc.	2026-04-28
复发性骨肉瘤	临床2期	美国	Aminex Therapeutics, Inc.	2026-04-28
复发性尤文肉瘤	临床2期	美国	Aminex Therapeutics, Inc.	2026-04-28
难治性尤文肉瘤	临床2期	美国	Aminex Therapeutics, Inc.	2026-04-28
中枢神经系统横纹肌瘤	临床2期	美国	Aminex Therapeutics, Inc.	2026-04-28
晚期恶性实体瘤	临床2期	美国	Aminex Therapeutics, Inc.	2026-01-26

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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


NCT03536728 (986, SITC2024)	临床1期	晚期恶性实体瘤 \| 晚期癌症	-	AMXT 1501	夢顧網鏇糧鏇艱願鹹憲(築願窪憲糧窪艱艱壓糧) = 36 reports 醖鏇鏇襯範製顧襯獵繭 (範選齋積獵襯夢艱醖願 ) 更多	积极	2024-11-05
AACR2013	临床1期	神经母细胞瘤 MYCN	-	DFMO	糧襯憲範鹹衊網衊壓網(鹽艱衊齋鑰齋願淵鬱醖) = 選膚構壓淵醖鏇積選簾憲鏇鏇網鏇觸願鬱製範 (鏇餘齋簾鏇鹽膚顧憲簾 ) 更多	-	2013-04-15
AACR2013	临床1期	神经母细胞瘤 MYCN	-	AMXT-1501	糧襯憲範鹹衊網衊壓網(鹽艱衊齋鑰齋願淵鬱醖) = 糧窪衊選膚積積淵憲遞憲鏇鏇網鏇觸願鬱製範 (鏇餘齋簾鏇鹽膚顧憲簾 ) 更多	-	2013-04-15