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.Xinyi Shangde YaoYuan Pharmaceutical Co., 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]

ChiCTR2500108747

/ Not yet recruitingPhase 3IIT

A Randomized, Blinded, Placebo-controlled Phase III clinical Trial to Evaluate the Efficacy of ACT001 to Enhance the Result of Radiotherapy and the Reduction of Toxic and Side Effect of whole-Brain Radiotherapy for Patients with Brain Metastasis of Small Cell Lung Cancer

Start Date01 Jul 2025

Sponsor / Collaborator

Self Finance, Inc.

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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53

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

01 Oct 2025·CNS Neuroscience & Therapeutics

Article

Author: Li, Xiaobo ; Lu, Guotao ; Gong, Weijuan ; Wu, Qi ; Wei, Junjie ; Gu, Min ; Liu, Fangzhou ; Qian, Li

ABSTRACT:

Aims:

Myeloid‐derived suppressor cells (MDSCs) are elevated in Parkinson's disease (PD), but their functional role remains unclear. This study investigated whether GSDMD deficiency enhances the immunosuppressive activity of granulocytic MDSCs (G‐MDSCs) to mitigate PD progression.

Methods:

Flow cytometry and Western blot analyzed G/M‐MDSCs in 37 PD patients and 21 controls. An MPTP‐induced PD mouse model was used to assess GSDMD‐deficient G‐MDSCs through behavioral tests, immunohistochemistry, and adoptive transfer experiments. The NLRP3 inhibitor ACT001 was evaluated for therapeutic potential.

Results:

PD patients and MPTP‐treated mice showed increased peripheral G‐MDSCs with reduced NLRP3/GSDMD activation. GSDMD knockout mice exhibited attenuated PD symptoms, reversed by MDSCs depletion. Adoptive transfer of GSDMD‐deficient G‐MDSCs suppressed microglial activation and improved motor function. ACT001 enhanced G‐MDSCs immunosuppression and alleviated PD pathology.

Conclusion:

GSDMD deficiency promotes immunosuppressive G‐MDSCs that inhibit neuroinflammation and PD progression. Targeting GSDMD to modulate MDSCs' function represents a novel therapeutic strategy for PD.

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 ; Hou, Huijie ; Li, Yiman ; 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.

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	ajgahmknle(qusbrbuoen) = qehicbicwh xilgjrkzsq (zqwbmodhqw )	Positive	28 Aug 2025
				Radiotherapy	ajgahmknle(qusbrbuoen) = jebsnqeouk xilgjrkzsq (zqwbmodhqw )
ChiCTR2000037739 (ASCO2022) Manual	Phase 1/2	Solid tumor \| Brain metastases	33	ACT001 200mg (Cohort A)	zhrlwiplsi(dtjbagwjlb) = pfptnisbad wcnndftawp (wvmzkiznbs ) View more	Positive	02 Jun 2022
				ACT001 400mg,bid (Cohort B)	zhrlwiplsi(dtjbagwjlb) = kdgifkiqhb wcnndftawp (wvmzkiznbs ) View more