Delving into the Latest Updates on ACT-001(Accendatech) with Synapse

Overview

Basic Info

Drug Type

Small molecule drug

Synonyms

dimethylaminomicheliolide Fumarate, ACT 001(Accendatech), ACT001-B

Target

LY96 x PAI-1 x TLR4

Action

inhibitors, antagonists

Mechanism

LY96 inhibitors(lymphocyte antigen 96 inhibitors), PAI-1 inhibitors(Plasminogen activator inhibitor 1 inhibitors), TLR4 antagonists(Toll like receptor 4 antagonists)

Therapeutic Areas

NeoplasmsNervous System DiseasesRespiratory Diseases+ [6]

Active Indication

Small Cell Lung CancerHigh grade gliomaDiffuse Intrinsic Pontine Glioma+ [11]

Inactive Indication

EpendymomaRecurrent Glioblastoma

Originator Organization

Accendatech Co. Ltd.

Active Organization

Accendatech Co. Ltd.Tianjin Xinyi Shangde Biomedical Technology Co., Ltd.Accendatech AU Pty Ltd.

+ [1]

Inactive Organization-

License Organization-

Drug Highest PhasePhase 3

First Approval Date-

RegulationFast Track (United States), Orphan Drug (United States), Rare Pediatric Disease (United States), Orphan Drug (European Union), Breakthrough Therapy (China)

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Structure/Sequence

Molecular FormulaC21H31NO7

InChIKeyPDWGVXJIDIRQME-DCNFBYMVSA-N

CAS Registry1582289-91-5

主要目的：评估ACT001联合全脑放疗对小细胞肺癌脑转移患者颅内外肿瘤的疗效。次要目的：以其他疗效终点评估ACT001联合全脑放疗对小细胞肺癌脑转移患者颅内肿瘤的疗效；评估ACT001降低全脑放疗毒副反应的作用；评估ACT001对小细胞肺癌脑转移患者生活质量的影响；评估ACT001对小细胞肺癌脑转移患者认知功能的影响。探索性目的：评估ACT001联合全脑放疗治疗小细胞肺癌脑转移患者颅外肿瘤的疗效；基于群体药代动力学（PopPK）分析方法，表征ACT001及其代谢产物MA·MCL（如适用）的药代动力学（PK）特征；评估治疗前后血液生物标志物的变化及其与疗效之间的关系；探索ACT001及其代谢产物MA·MCL（如适用）的暴露与效应之间的关系；评估治疗前后脑脊液循环肿瘤DNA（ctDNA）基因特征的变化及其与疗效之间的关系、以及脑脊液中的药物浓度及相关的PK特征。

[Translation]

Primary Objective: To evaluate the efficacy of ACT001 combined with whole-brain radiotherapy for the treatment of extracranial and extracranial tumors in patients with brain metastases from small cell lung cancer. Secondary Objectives: To evaluate the efficacy of ACT001 combined with whole-brain radiotherapy for the treatment of intracranial tumors in patients with brain metastases from small cell lung cancer using other efficacy endpoints; to evaluate the effect of ACT001 on reducing the toxicities of whole-brain radiotherapy; to evaluate the effect of ACT001 on the quality of life in patients with brain metastases from small cell lung cancer; and to evaluate the effect of ACT001 on cognitive function in patients with brain metastases from small cell lung cancer. Exploratory Objectives: To evaluate the efficacy of ACT001 combined with whole-brain radiotherapy for the treatment of extracranial tumors in patients with brain metastases from small cell lung cancer; to characterize the pharmacokinetic (PK) profile of ACT001 and its metabolite, MA·MCL (if applicable), using a population pharmacokinetic (PopPK) analysis approach; to evaluate changes in blood biomarkers before and after treatment and their relationship with efficacy; to explore the exposure-effect relationship of ACT001 and its metabolite, MA·MCL (if applicable); to evaluate changes in the genomic profile of circulating tumor DNA (ctDNA) in cerebrospinal fluid before and after treatment and its relationship with efficacy, as well as drug concentrations in cerebrospinal fluid and related PK profiles.

Start Date03 Sep 2025

Sponsor / Collaborator

Tianjin Xinyi Shangde Biomedical Technology Co., Ltd.

NCT06838676

/ RecruitingPhase 2IIT

A Phase II Trial of ACT001 in Children and Adolescents With Diffuse Intrinsic Pontine Gliomas and H3K27-altered High Grade Gliomas

This is a Phase II open-label study to investigate the safety and efficacy of ACT001 in patients with DIPG and H3K27-altered HGG.

Start Date10 Jul 2025

Sponsor / Collaborator

Nationwide Children's Hospital

[+1]

CTR20232173

/ RecruitingPhase 2

一项单臂、多中心评价ACT001联合放疗治疗初诊弥漫内生型桥脑胶质瘤患者安全性和有效性的II期临床研究

[Translation] A single-arm, multicenter Phase II clinical study evaluating the safety and efficacy of ACT001 combined with radiotherapy in patients with newly diagnosed diffuse intrinsic pontine glioma

主要目的：评估ACT001联合放疗治疗初诊DIPG受试者的肿瘤缓解情况和安全性（疗效探索期）、验证ACT001联合放疗对初诊DIPG受试者的肿瘤缓解情况和总生存时间的影响（疗效验证期）；次要目的：初诊DIPG受试者对ACT001联合放疗的疗效（疗效探索期）、验证ACT001联合放疗对初诊DIPG受试者的其他疗效验证ACT001联合放疗对初诊DIPG受试者的安全性（疗效验证期）；探索性目的：评估治疗对生物标志物的影响及其与疗效之间的关系、基于PopPK分析方法，表征ACT001及代谢产物的PK特征、探索ACT001的暴露与效应之间的关系

[Translation]

Primary objectives: To evaluate the tumor remission and safety of ACT001 combined with radiotherapy in newly diagnosed DIPG subjects (efficacy exploration phase), and to verify the effect of ACT001 combined with radiotherapy on tumor remission and overall survival time in newly diagnosed DIPG subjects (efficacy verification phase); Secondary objectives: To evaluate the efficacy of ACT001 combined with radiotherapy in newly diagnosed DIPG subjects (efficacy exploration phase), to verify other efficacy of ACT001 combined with radiotherapy in newly diagnosed DIPG subjects, and to verify the safety of ACT001 combined with radiotherapy in newly diagnosed DIPG subjects (efficacy verification phase); Exploratory objectives: To evaluate the impact of treatment on biomarkers and its relationship with efficacy, to characterize the PK characteristics of ACT001 and metabolites based on PopPK analysis methods, and to explore the relationship between exposure and effect of ACT001

Start Date30 Aug 2023

Sponsor / Collaborator

Tianjin Xinyi Shangde Biomedical Technology Co., Ltd.

100 Clinical Results associated with ACT-001(Accendatech)

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100 Translational Medicine associated with ACT-001(Accendatech)

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100 Patents (Medical) associated with ACT-001(Accendatech)

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54

Literatures (Medical) associated with ACT-001(Accendatech)

01 Sep 2025·JOURNAL OF CLINICAL LABORATORY ANALYSIS

ACT001 Inhibits Tumor Progression and Modulates Immune Responses in Non‐Small Cell Lung Cancer

Article

Author: Shi, Zhijing ; Yu, Yang ; Wang, Zhitao ; Song, Yan ; Guo, Hui ; Li, Yiman ; Hou, Huijie ; Chen, Zhe

ABSTRACT:

Background:

Advancements in non‐small cell lung cancer (NSCLC) therapies have improved outcomes, but challenges like low immune response, drug resistance, and side effects persist. ACT001, a novel small‐molecule inhibitor, shows promise in addressing these issues.

Methods:

We evaluated ACT001's anti‐tumor and immunomodulatory effects in NSCLC. In vitro, its impact on proliferation, migration, invasion, and cell cycle arrest was assessed. In vivo, its effect on tumor growth in C57BL/6 mice was studied. Pull‐down assays and mass spectrometry identified ACT001's interaction with STAT1/STAT3 and its regulation of PD‐L1 expression.

Results:

ACT001 inhibited NSCLC cell proliferation, migration, and invasion, induced cell cycle arrest, and suppressed tumor growth in vivo. It enhanced granzyme B release in CD3+ T cells, promoting NSCLC cell apoptosis. Mechanistically, ACT001 bound to STAT1/STAT3, suppressing their phosphorylation and reducing PD‐L1 expression.

Conclusion:

ACT001 exhibits antitumoral and immunomodulatory potential by targeting STAT1/STAT3 and regulating PD‐L1, offering a promising therapeutic approach for NSCLC.

01 Sep 2025·Synthetic and Systems Biotechnology

Engineering the cytochrome P450 to enhance parthenolide production in Saccharomyces cerevisiae

Article

Author: Liu, Haoyu ; Yang, Tongqing ; Jiang, Yixun ; Xiao, Wenhai ; Wang, Ying ; Yao, Mingdong ; Dong, Tianyu

Parthenolide is confirmed to be an important component of the anticancer drug-ACT001. However, parthenolide biosynthesis in Saccharomyces cerevisiae (S. cerevisiae) was greatly hindered by the low conversion rate of its precursor, costunolide. In this study, the Position Specific Scoring Matrix (PSSM) was used to analyze the sequence evolutionary information of parthenolide synthase from Tanacetum parthenium (TpPTS), and a series of mutants were designed and validated. Notably, when the mutant of TpPTS-Y22G was introduced in S. cerevisiae, the parthenolide titer increased by 110 % compared to that of the TpPTS wild-type. Considering TpPTS as an endoplasmic reticulum-localized cytochrome P450 and the importance of heme supply, endoplasmic-associated molecular chaperone HRD1 (hydroxymethyl glutaryl-coenzyme A reductase degradation protein 1) and heme biosynthesis gene HEM2 (aminolevulinate dehydratase) were overexpressed in S. cerevisiae to improve TpPTS expression and catalytic activity. As a result, a titer of 27.08 mg/L parthenolide was achieved in a shake flask, which was further increased by 209 %. Additionally, the conversion rate of costunolide to parthenolide increased from 20.4 % to 51.8 % compared to the initial strain yYTQ001. Eventually, a parthenolide titer of 99.71 mg/L was achieved in a 5-L bioreactor. Our research provides effective strategies and valuable references for engineering rate-limiting cytochrome P450 enzymes to improve sesquiterpenes production in S. cerevisiae.

01 Jun 2025·Clinical and Translational Medicine

Targeting the MDK/c‐Myc complex to overcome temozolomide resistance in glioma

Article

Author: Wang, Bangmao ; Liu, Ning ; Wang, Genbei ; Wang, Qianqian ; Ding, Xiaojing ; Xi, Xiaonan ; Lu, Yaxin ; Zhong, Weilong

Abstract:

Background:

Temozolomide (TMZ), which is an alkylating agent, is the standard chemotherapeutic drug used for glioma treatment. However, the development of resistance to TMZ limits its efficacy. Thus, identifying novel therapeutic targets is necessary.

Methods:

In this study, the levels of midkine (MDK) and c‐Myc expression in glioma patient samples downloaded from TCGA were analyzed. Their interactions were also demonstrated through microthermometry and immunocoprecipitation. Furthermore, proteomics technology and Western blot showed that MDK interacted with c‐Myc and influenced its ubiquitination, thereby activating a prosurvival signalling pathway and epithelial–mesenchymal transition mechanism, which contributed to TMZ resistance. To target the MDK/c‐Myc complex, we screened for a small‐molecule inhibitor (ACT001) that specifically disrupts the interaction between MDK and c‐Myc. Treatment with ACT001 greatly sensitized TMZ‐resistant glioma cells to TMZ, promoting cell death and inhibiting cell proliferation. Moreover, combination therapy with ACT001 and TMZ showed synergistic effects that inhibit tumour growth in glioma xenograft models and glioma in situ models.

Results:

ACT001 facilitated the degradation of c‐Myc by focusing on the MDK/c‐Myc complex and controlled the Wnt/β‐catenin signalling pathway via MDK, ultimately halting the advancement of glioma. When combined with TMZ, ACT001 showed good therapeutic potential for the treatment of glioma.

Conclusion:

Focusing on the MDK/c‐Myc complex could be an effective approach to combat resistance to TMZ in glioma. Therapy with ACT001 may be a novel approach to improve the efficacy of TMZ‐based chemotherapy in patients with glioma. Further preclinical and clinical studies are warranted to validate the therapeutic potential of targeting the MDK/c‐Myc complex in glioma treatment.

100 Deals associated with ACT-001(Accendatech)

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R&D Status

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Small Cell Lung Cancer	Phase 3	China	Tianjin Xinyi Shangde Biomedical Technology Co., Ltd.	03 Sep 2025
High grade glioma	Phase 2	United States	Accendatech Co. Ltd.	10 Jul 2025
High grade glioma	Phase 2	Australia	Accendatech Co. Ltd.	10 Jul 2025
High grade glioma	Phase 2	Canada	Accendatech Co. Ltd.	10 Jul 2025
High grade glioma	Phase 2	Germany	Accendatech Co. Ltd.	10 Jul 2025
Diffuse Intrinsic Pontine Glioma	Phase 2	China	Tianjin Xinyi Shangde Biomedical Technology Co., Ltd.	30 Aug 2023
Non-Small Cell Lung Cancer	Phase 2	China	Tianjin Xinyi Shangde Biomedical Technology Co., Ltd.	05 Apr 2023
Recurrent Glioblastoma	Phase 2	United States	Accendatech Co. Ltd.	22 Sep 2021
Breast Cancer	Phase 2	China	Tianjin Xinyi Shangde Biomedical Technology Co., Ltd.	16 Feb 2021
Hepatocellular Carcinoma	Phase 2	China	Tianjin Xinyi Shangde Biomedical Technology Co., Ltd.	16 Feb 2021

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Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


CTR20230452 (ACT001-CN-051, Company_Website) Manual	Phase 2	Extensive stage Small Cell Lung Cancer \| Lung Cancer with Brain Metastasis Second line \| Third line	60	Radiotherapy+ACT001	rhzzngbidr(zfpjztzdel) = krrhsxxtcu eynlovfmjm (tfjrkiaaje )	Positive	28 Aug 2025
				Radiotherapy	rhzzngbidr(zfpjztzdel) = ylsdljbncg eynlovfmjm (tfjrkiaaje )
ChiCTR2000037739 (ASCO2022) Manual	Phase 1/2	Solid tumor \| Brain metastases	33	ACT001 200mg (Cohort A)	izxjdcnofv(yrzbinvygc) = oaiqvqlkyf cdnigffrrk (tgoyvsvhra ) View more	Positive	02 Jun 2022
				ACT001 400mg,bid (Cohort B)	izxjdcnofv(yrzbinvygc) = ctdaxhhhxi cdnigffrrk (tgoyvsvhra ) View more