Delving into the Latest Updates on Chidamide with Synapse

RegulationOrphan Drug (United States), Priority Review (China), Breakthrough Therapy (China), Orphan Drug (Japan), Conditional marketing approval (China), Special Review Project (China)

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Structure/Sequence

Molecular FormulaC22H19FN4O2

InChIKeySZMJVTADHFNAIS-BJMVGYQFSA-N

CAS Registry1616493-44-7

Hemophagocytic lymphohistiocytosis (HLH) is a severe, life-threatening hyperinflammatory syndrome characterized by dysregulated activation of cytotoxic T-lymphocytes and macrophages, with lymphoma-associated HLH (LA-HLH) carrying a particularly poor prognosis due to delayed diagnosis and lack of optimal treatment regimens. While the systemic features of LA-HLH-such as persistent fever, pancytopenia, and hepatosplenomegaly-are well-documented, its oral manifestations have not been previously described in the literature. We herein report a case of LA-HLH in a 28-year-old female patient who presented with intermittent fever along with recurrent oral ulcers. The patient had been diagnosed with ENKTL (non-nasal type, stage IVB, PINK-E 4, high risk) two years prior, with skin and muscle involvement, and had achieved remission after chemotherapy and autologous hematopoietic stem cell transplantation. A comprehensive diagnostic workup, including bone marrow biopsy and laboratory assessments, confirmed the diagnosis of LA-HLH secondary to relapsed extranodal NK/T-cell lymphoma (non-nasal type, stage IVB). The oral lesions were managed with Kangfuxin mouthwashes, while the underlying lymphoma and HLH were treated with dexamethasone and etoposide, combined with chidamide and tislelizumab. To the best of our knowledge, this is the first report documenting oral mucosal involvement by ENKTL occurring in a patient with LA-HLH. These oral lesions, as a manifestation of localized lymphoma progression, may serve as early clinical clues to lymphoma relapse with secondary HLH, rather than being a specific early sign of LA-HLH itself. Increased awareness of these potential oral findings among clinicians and dentists could facilitate earlier diagnosis of lymphoma relapse in LA-HLH patients and improve the multidisciplinary management of associated complications.

01 Nov 2026·British Journal of Pharmacology

Histone deacetylase inhibitors as venetoclax‐sensitising partners in acute myeloid leukaemia: Mechanisms, pharmacology and translational perspectives

Review

Author: Lee, Jaebok ; Diederich, Marc

Abstract:

Venetoclax combined with hypomethylating agents has improved treatment for older or unfit patients with acute myeloid leukaemia (AML), but resistance and relapse remain common. This review analyses the rationale for combining venetoclax with inhibitors of histone deacetylase (HDAC). Class I histone deacetylase 1/2/3‐dependent suppression of myeloid cell leukaemia 1 and de‐repression of pro‐apoptotic BCL‐2 homology domain 3 (BH3)‐only genes provide the strongest mechanistic basis, whereas HDAC6/heat shock protein 90, autophagy, immune‐modulatory and super‐enhancer mechanisms remain context‐dependent and incompletely validated in primary AML under venetoclax exposure. Effects of HDAC inhibition must also be interpreted in the context of tumour protein 53 status. Early HDAC inhibitor‐containing composite regimens, particularly chidamide‐based CACAG‐VEN (chidamide, venetoclax, azacitidine, low‐dose cytarabine, aclarubicin and granulocyte colony‐stimulating factor), report high response rates, including overall response rate of 76.5–98.0%, complete remission/complete remission with incomplete count recovery or composite complete remission of 73.5–93.3%, and measurable residual disease negativity of 44–61%. This six‐component regimen does not isolate the contribution of HDAC inhibition. Future development should prioritise biomarker‐selected trials that hold non‐HDAC components constant, incorporate pharmacodynamic HDAC engagement and BH3 profiling, and account for cytochrome P450 3A‐mediated drug interactions. HDAC inhibitor–venetoclax combinations are mechanistically rational, but standard‐of‐care positioning requires randomised or pharmacodynamic validation.

13 Oct 2026·JAMA-JOURNAL OF THE AMERICAN MEDICAL ASSOCIATION

Tucidinostat Plus R-CHOP for Large B-Cell Lymphoma—Reply

Article

Author: Xu, Peng-Peng ; Chen, Bo ; Zhao, Wei-Li

74

News (Medical) associated with Chidamide

18 Oct 2026

·www.biospace.com

Chipscreen Biosciences Announces NMPA IND Approval for NW001, a PD-L1-Targeted Epigenetic Immune-Modulating Antibody Conjugate

SHENZHEN, China--(BUSINESS WIRE)-- #ChipscreenBiosciences --Shenzhen Chipscreen Biosciences Co., Ltd. (“Chipscreen Biosciences”) (SSE: 688321) today announced that NW001, a first-in-class epigenetic immune-modulating antibody conjugate (EIMAC) co-developed with Chengdu Chipscreen Newway Biosciences Co., Ltd., has obtained IND approval from China’s National Medical Products Administration (NMPA). The Phase 1 trial will assess the safety, tolerability, pharmacokinetics and preliminary antitumor activity of NW001 in patients with advanced tumors. Although immune checkpoint inhibitors have reshaped the treatment landscape for many advanced cancers, immunologically "cold" tumors and primary or acquired resistance remain major clinical challenges. Improving the efficacy of immunotherapy and overcoming resistance are therefore key priorities in developing next-generation immuno-oncology (IO) drugs. NW001 is an epigenetic immune-modulating antibody conjugate (EIMAC). Its antibody component is a humanized anti-PD-L1 monoclonal antibody, conjugated to a novel histone deacetylase inhibitor (HDACi) payload. Upon binding to cell-surface PD-L1, NW001 is internalized and undergoes intracellular cleavage, releasing the HDACi payload to exert antitumor activity through combined epigenetic and immune modulation. This design aims to enable precise release of the epigenetic modulator payload inside tumor cells and reduce systemic exposure. It has the potential to overcome limitations of current immunotherapies and marks a key milestone in Chipscreen Biosciences’ IO 2.0 strategy. Preclinical studies show that NW001 exhibits marked antitumor activity in models refractory or resistant to PD-1/PD-L1 inhibitors. In responsive models, it also demonstrates greater antitumor activity than that of PD-L1 inhibitors. NW001 shows favorable pharmacokinetic properties and is well tolerated in non-human primates. Through its work on chidamide, the world’s first approved oral, subtype-selective HDAC inhibitor, the company has extensively explored the interplay between epigenetics and tumor immunology, accumulating substantial preclinical and clinical evidence. The combination of chidamide and immune checkpoint inhibitors has met the primary efficacy endpoint in a global Phase 3 study in melanoma. A Phase 3 study in colorectal cancer conducted in China has completed patient enrollment. Built on the clinically validated epigenetic immune-modulating mechanism, NW001 is designed to widen the therapeutic window; it therefore has the potential to offer superior efficacy to that of current immunotherapies, and to overcome treatment limitations in immunologically "cold" tumors and tumors resistant to immunotherapy. About Chipscreen Biosciences Founded in March 2001, Chipscreen Biosciences is a pioneer in innovative drug development based in China. The company is committed to providing affordable medicines with novel mechanisms of action and transformative efficacy that address urgent clinical needs. With its AI-enabled Chemical Genomics Platform (AI-ChemGen), Chipscreen Biosciences supports the full process from basic research to clinical translation. The company has successfully developed two first-in-class medicines: Epidaza® (chidamide) for oncology and Bilessglu® (chiglitazar sodium) for type 2 diabetes. These products are marketed for multiple indications worldwide. Chipscreen Biosciences has built an R&D pipeline spanning from early discovery to late-stage clinical development across oncology, metabolic diseases, autoimmune diseases, and neurodegenerative diseases. Eleven products are currently in clinical development, three of which are undergoing concurrent clinical studies in China and the United States. The company continues to advance a range of differentiated, globally competitive R&D programs. About Chipscreen Newway Biosciences Chengdu Chipscreen Newway Biosciences Co., Ltd. (“Chipscreen Newway Biosciences”) is an associate company of Chipscreen Biosciences, established as part of Chipscreen Biosciences’ strategic expansion into innovative large-molecule drug development. The company aims to address urgent clinical needs by applying new discoveries and technologies at the forefront of life sciences. It has an R&D team with more than 50 members and a 3,000 m² laboratory that integrates small-molecule and large-molecule drug discovery. Chipscreen Newway Biosciences has established world-leading technology platforms for ADC development. These include a platform that combines three approaches to high-throughput antibody discovery based on precision immunization strategies with AI-assisted protein engineering; the DSHL (DAR homogeneous Site-specific Hydrophilic Linker) platform; and an innovative cytotoxic payload platform featuring novel mechanisms of action and dual-payload designs with complementary mechanisms of action. With a focus on immuno-oncology and ADCs (IO + ADC), the company is developing molecules with novel mechanisms of action, including ADCs directed at novel targets, bispecific ADCs and dual-payload ADCs. It is committed to continually advancing differentiated drug candidates to address unmet clinical needs. A number of its innovative products are currently in clinical or preclinical development. Contacts pr@chipscreen.com

ImmunotherapyPhase 3Clinical ResultADCIND

17 Aug 2026

·allsci.com

Pitt, Magee-Womens team turns cancer dormancy into a druggable vulnerability

A preclinical study from the University of Pittsburgh and Magee-Womens Research Institute & Foundation identifies a two-drug strategy for killing quiescent, treatment-resistant cancer cells that can seed relapse after chemotherapy, according to a paper published August 13 in Cancer Gene Therapy. Most cytotoxic regimens preferentially target actively dividing cells, allowing slow-cycling or non-dividing cancer cells to survive treatment and potentially drive recurrence. The problem is particularly relevant in ovarian cancer, where high initial response rates to platinum-based chemotherapy are frequently followed by relapse. The study, led by corresponding author Ronald J. Buckanovich, proposes exploiting a vulnerability created by cancer-cell quiescence rather than waiting for residual cells to resume proliferation. The researchers found that downregulation of the nucleosome remodeling and deacetylase (NuRD) complex — specifically its components MBD3 and CHD4 — promotes entry into a quiescent, drug-tolerant state. Genetic knockdown of either component induced G0 arrest across ovarian cancer models. Histone deacetylase inhibitors (HDACi) phenocopied this effect pharmacologically, altering chromatin accessibility at RHO/RAC GTPase-associated loci, suppressing the MRTFA/SRF pro-proliferative transcriptional axis, and driving cells into reversible G0 arrest. Rather than directly killing these cells, HDAC inhibition helps establish a quiescent state that creates a second vulnerability: increased dependence on proteasome and autophagy activity to maintain protein homeostasis. Combining HDACi with the proteasome inhibitor carfilzomib or the autophagy inhibitors hydroxychloroquine or chloroquine exploited that dependency and produced synergistic cell death. The HDACi-carfilzomib combination produced synergy indices of 25–73 under the highest single agent (HSA) model in vitro. In an ovarian cancer patient-derived xenograft (PDX) mouse model, the HDACi vorinostat (SAHA) plus carfilzomib produced greater tumor growth inhibition than either monotherapy or vehicle control across groups of 8–10 tumors. HDACi-induced quiescence was also demonstrated in triple-negative breast cancer patient-derived organoids and breast and lung cancer cell lines, suggesting the underlying biology may extend beyond ovarian cancer. Activity was additionally evaluated in an ex vivo co-culture model incorporating primary murine hepatocytes to approximate aspects of the liver microenvironment. HDAC inhibitors including vorinostat and belinostat have previously been evaluated as single agents in ovarian cancer with limited efficacy, consistent with the study's finding that HDAC inhibition can induce arrest without killing the cells. There is also clinical precedent for combining HDAC and proteasome inhibition: panobinostat plus bortezomib was previously FDA-approved for multiple myeloma, although panobinostat was subsequently withdrawn from the US market for commercial reasons. The rationale here is distinct, using HDAC inhibition to establish quiescence before exploiting the resulting proteostasis dependency. The approach also differs from cancer dormancy strategies aimed at maintaining residual cells in a dormant state or preventing their reactivation. Here, quiescence itself becomes a targetable vulnerability. The most immediately translatable strategy pairs the HDACi valproic acid (VPA) with chloroquine. Both are repurposed drugs with extensive human clinical experience, potentially shortening the translational path compared with new molecular entities. The authors said they are planning a window-of-opportunity clinical study of the combination in ovarian cancer. The approach remains preclinical, with relatively small in vivo groups, and established safety profiles for the individual drugs do not establish the safety or optimal dosing of the combination in ovarian cancer. No industry partner or regulatory designation has been disclosed. If translated successfully, the strategy would turn a conventional obstacle in cancer treatment into a therapeutic vulnerability: rather than waiting for dormant residual cells to resume proliferation, it seeks to exploit the biological dependencies created by quiescence itself. Access the AllSci platform to explore the science behind the news. Spot something wrong? Report an issue with this article

Gene TherapyClinical Result

15 Jul 2026

·firstwordpharma.com

Biopharma bites: HUYABIO claims late-stage melanoma success, Summit's antibiotic sale and Lyora's debut

HUYABIO eyes frontline melanoma with Phase III winWith its focus on ivonescimab, Summit sells antibiotic to BiossilLyora raises $2.5M for retinal gene therapies HUYABIO eyes frontline melanoma with Phase III winHUYABIO International said its global Phase III trial of HBI-8000 in combination with Bristol Myers Squibb's anti–PD-1 antibody Opdivo (nivolumab) met its primary endpoint in patients with advanced melanoma, possibly putting a first-line indication within reach.In the 404-patient study, those receiving the combo achieved median progression-free survival of 11.7 months, compared to 7.4 months for Opdivo plus placebo, translating to a 58% benefit in favour of the HBI-8000 arm. Overall survival data weren't disclosed; additional analyses are underway and detailed results will be presented at a future medical meeting.HBI-8000 is an oral histone deacetylase (HDAC) inhibitor already approved for certain lymphoma indications in China and Japan, where HUYABIO says it has been used in more than 90,000 patients. "We believe HBI-8000 will become an important addition to the standard of care [in melanoma]," said CEO Mireille Gillings.-Anna BratulicWith its focus on ivonescimab, Summit sells antibiotic to BiossilSummit Therapeutics said Tuesday it has sold the late-stage antibiotic ridinilazole to Biossil for $500,000 upfront. The drugmaker, which is working towards the US approval of PD-1/VEGF bispecific ivonescimab for lung cancer, is also eligible for up to $104.5 million in regulatory and commercial milestones, plus tiered royalties.Prior to the sale, Summit completed the Phase III Ri-CoDIFy study of ridinilazole in patients with a C. difficile infection. The company reported in late 2021 that ridinilazole had a higher observed sustained clinical response rate than vancomycin, but did not meet the study’s primary endpoint for superiority within the established time boundaries. "Recurrent C. difficile infection, selection of antibiotic-resistant genes, and gut dysbiosis are common, burdensome, and in need of new approaches," said Biossil Chief Science Officer Alexander Mosa. "We thank Summit for entrusting us with continued development of ridinilazole, and we will advance this programme with urgency and focus."-Elizabeth EatonLyora raises $2.5M for retinal gene therapiesLyora Therapeutics launched on Tuesday with $2.5 million in pre-seed funding to advance its in-licensed portfolio of preclinical-stage genetic medicines for inherited retinal diseases. Each of the biotech's assets are intended to be "one-and-done" therapies. LYA-101 is designed to treat retinitis pigmentosa caused by pathogenic variants in the PRPF31 gene, and LYA-102 is a CRISPR-based therapy aimed at addressing mutations in exon 13 of the USH2A gene to treat Usher Syndrome Type 2. The startup has an exclusive option to license from Editas Medicine certain rights applicable to USH2A gene editing.Lyora plans to submit an IND for LYA-101 within the next 18 months, with an application for LYA-102 shortly after.-Elizabeth Eaton

Phase 3Clinical ResultDrug ApprovalGene Therapy

100 Deals associated with Chidamide

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External Link

KEGG	Wiki	ATC	Drug Bank
D10993	Chidamide	L01XX	DB06334

R&D Status

Approved

10 top approved records.

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Indication	Country/Location	Organization	Date
Diffuse Large B-Cell Lymphoma	China	Shenzhen Chipscreen Biosciences Co., Ltd.	24 Apr 2024
Adult T-Cell Leukemia-Lymphoma	Japan	Meiji Seika Pharma Co., Ltd.	23 Jun 2021
Hormone receptor positive HER2 negative breast cancer	China	Shenzhen Chipscreen Biosciences Co., Ltd.	20 Nov 2019
Peripheral T-Cell Lymphoma	China	Shenzhen Chipscreen Biosciences Co., Ltd.	23 Dec 2014

Developing

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
pMMR/MSS/MSI-L Colon Cancer	Phase 3	China	Shenzhen Chipscreen Biosciences Co., Ltd.	06 Dec 2024
Unresectable Colorectal Carcinoma	Phase 3	China	Shenzhen Chipscreen Biosciences Co., Ltd.	06 Dec 2024
Metastatic Microsatellite Stable Colorectal Carcinoma	Phase 3	China	Sun Yat-Sen University	01 Mar 2023
MSS/pMMR/MSI-L Colorectal Carcinoma	Phase 3	China	Sun Yat-Sen University	01 Mar 2023
Brain metastases	Phase 3	United States	Huyabio International LLC	12 Aug 2021
Brain metastases	Phase 3	Japan	Huyabio International LLC	12 Aug 2021
Brain metastases	Phase 3	Australia	Huyabio International LLC	12 Aug 2021
Brain metastases	Phase 3	Austria	Huyabio International LLC	12 Aug 2021
Brain metastases	Phase 3	Belgium	Huyabio International LLC	12 Aug 2021
Brain metastases	Phase 3	Brazil	Huyabio International LLC	12 Aug 2021

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Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


ChiCTR2300068737 (ESMO2026)	Phase 2	Locally Advanced Soft Tissue Sarcoma Second line	21	Radiotherapy+chidamide+PD-1 antibody	uukiuvynmn(hjyoecevcu) = pgdjmjofmo gvvezdwntu (buqjyubtwq ) View more	Positive	23 Oct 2026
NCT06951997 (ESMO2026)	Phase 2	Metastatic Pancreatic Cancer First line	34	QL1706 + chidamide + nab-paclitaxel + gemcitabine	ftnfrxwlfx(znonkjytkg) = plyptmdneb poggfbdiwl (sprogqarsp ) View more	Positive	23 Oct 2026
CSCO2026	Not Applicable	Locally Advanced Soft Tissue Sarcoma Second line	21	Radiotherapy + Chidamide + PD-1 Antibody	zyhwjliuff(wdutgpbexa) = 89.3% of adverse events (AEs) were grade 1 or 2, and no grade 4 severe AEs were reported cuqzcnpvsk (lwpugrgzhl ) View more	Positive	11 Sep 2026
NCT06066905 (Pubmed \| Ann Hematol)	Not Applicable	Acute Myeloid Leukemia	43	Chidamide (preemptive group)	bziyldzbkk(rbppdvymqj) = reducing CD4+ T cells (P=0.007) zvkynkxroy (mkztgslewa ) View more	Positive	13 Jul 2026
NCT06066905 (Pubmed \| Ann Hematol)	Not Applicable	Acute Myeloid Leukemia	43	Chidamide (prophylactic group)		Positive	13 Jul 2026
ChiCTR2300077213 (C-ooperate/SCOG-C001, ASCO2026)	Phase 2	Advanced Colorectal Adenocarcinoma Third line	30	Regorafenib 120 mgChidamide 20 mgFruquintinib 5 mg + Regorafenib 120 mgChidamide 20 mgg/kgFruquintinib 5 mg + Regorafenib 120 mgChidamide 20 mg mg or Fruquintinib 5 mg (pMMR/MSS + Advanced Colorectal Cancer)	vkaffgmsmi(mmgfqcpqrb) = mwvnysexyc bdvqiilnev (azenrdeatc, 2.4 - 30.2) View more	Positive	29 May 2026
NCT06979908 (ASCO2026)	Phase 2	Colorectal Cancer Microsatellite stable (MSS)	9	Fruquintinib+sintilimab+chidamide (Microsatellite stable metastatic colorectal cancer)	zhrggebcic(tqjkwfpvie) = rwcdxmpybv rleakqbxrl (gtvrbkmbss ) View more	Positive	29 May 2026
NCT04231448 (Pubmed \| JAMA)	Phase 3	Diffuse Large B-Cell Lymphoma First line	423	Tucidinostat + R-CHOP	fgsqboaaov(vcwwjtmxmj) = cbneekrsky ednsajjyyh (tgxzoifhpw ) View more	Positive	19 May 2026
NCT04231448 (Pubmed \| JAMA)	Phase 3	Diffuse Large B-Cell Lymphoma First line	423	Placebo + R-CHOP	fgsqboaaov(vcwwjtmxmj) = rhtbgsyniu ednsajjyyh (tgxzoifhpw ) View more	Positive	19 May 2026
EHA2026	Not Applicable	Immunoblastic Lymphadenopathy	7	Chidamide plus Azacitidine	euubulkaem(ingyskdksz) = yccbxexjva ixqxbuexco (ywjnzmwwkp ) View more	Positive	12 May 2026
EHA2026	Not Applicable	Immunoblastic Lymphadenopathy TET2 \| RHOA \| DNMT3A ... View more	9	MINE regimen + targeted agents	jvhvlvgykj(zhpkjbmhyc) = vjedptcsho ovplpaozsr (tandjnjfdc ) View more	Positive	12 May 2026
EHA2026	Not Applicable	T-Cell Lymphoma \| Precursor T-Cell Lymphoblastic Leukemia-Lymphoma	85	Chidamide combined with modified BuCy conditioning regimen	bngschqaot(gtydnciwkt) = ymyhxcyvdg occhesvuzn (uxzjvjvbdi ) View more	Positive	12 May 2026
EHA2026	Not Applicable		85	Traditional modified BuCy conditioning regimen	bngschqaot(gtydnciwkt) = alknhygymi occhesvuzn (uxzjvjvbdi ) View more	Positive	12 May 2026