Abexinostat

The HDAC inhibitor pipeline remains active, with several next-generation agents advancing in clinical trials. Abexinostat (Xynomic Pharmaceuticals) is in Phase III development for renal cell carcinoma, representing a potential expansion into solid tumors. Quisinostat (Viriom) is in Phase II for uveal melanoma, and Entinostat, partnered between EOC Pharma and Syndax, is being explored in Hodgkin lymphoma. LAS VEGAS, Aug. 20, 2025 /PRNewswire/ -- DelveInsight's HDAC Inhibitors Market Size, Target Population, Competitive Landscape & Market Forecast report includes a comprehensive understanding of current treatment practices, addressable patient population, which includes top indications such as Multiple Myeloma, Peripheral T-cell Lymphoma (PTCL), Cutaneous T-cell Lymphoma (CTCL), Amyotrophic Lateral Sclerosis (ALS), and others. The selected indications are based on approved therapies and ongoing pipeline activity. The report also provides insights into the emerging HDAC inhibitors, market share of individual therapies, and current and forecasted market size from 2020 to 2034, segmented into 7MM. Key Takeaways from the HDAC Inhibitors Market Report As per DelveInsight's analysis, the total market size of HDAC inhibitors in the 7MM is expected to surge significantly by 2034. The report provides the total potential number of patients in the indications, such as Multiple Myeloma, Peripheral T-cell Lymphoma (PTCL), Cutaneous T-cell Lymphoma (CTCL), Amyotrophic Lateral Sclerosis (ALS), and others. Leading HDAC inhibitor companies, such as Xynomic Pharmaceuticals, Viriom, EOC Pharma, Syndax Pharmaceuticals, Jubilant Therapeutics, and others, are developing novel HDAC inhibitors that can be available in the HDAC inhibitors market in the coming years. Some of the key HDAC inhibitors in the pipeline include Abexinostat, Quisinostat, Entinostat, JBI-802, and others. Discover which indication is expected to grab the major HDAC inhibitors market share @ HDAC Inhibitors Market Report HDAC Inhibitors Overview Histone deacetylases (HDACs) remove acetyl groups from both histone and non-histone proteins. Because acetylation of histones is linked to an open chromatin structure and active gene transcription, HDAC activity leads to histone deacetylation and epigenetic suppression of gene expression. Since HDACs influence many pathways involved in cancer development, HDAC inhibitors (HDACis) show clinical effects through several mechanisms, including changes in gene expression of cell cycle and apoptotic proteins, acetylation of other proteins involved in cell growth and survival, effects on angiogenesis, aggresome formation, and DNA repair. There are 18 known HDACs, divided into two major groups and four classes based on their cellular location, homology, enzyme mechanism, sequence of discovery, and histone substrate specificity. The first group includes zinc-dependent HDACs, which all share a similar catalytic core for deacetylating acetyl-lysine residues. These comprise Class I (HDAC1, -2, -3, -8), Class II (IIa: HDAC4, -5, -7, -9; IIb: HDAC6, -10), and Class IV (HDAC11). The second group includes Class III HDACs (sirtuins, SIRT1–7), which rely on NAD+ for their enzymatic activity. HDAC Inhibitors Epidemiology Segmentation The HDAC inhibitors market report proffers epidemiological analysis for the study period 2020–2034 in the 7MM, segmented into: Total Cases of Selected Indications for HDAC Inhibitor Total Eligible Patient Pool of Selected Indications for HDAC Inhibitor Total Treated Cases of Selected Indications for HDAC Inhibitor HDAC Inhibitors Treatment Market Several HDAC inhibitors are currently available on the market. Recent entrants include DUVYZAT (givinostat), while earlier approved drugs include BELEODAQ (belinostat), HIYASTA (tucidinostat), among others. DUVYZAT is a histone deacetylase inhibitor approved for treating Duchenne muscular dystrophy (DMD) in patients aged 6 and above. While it is categorized as an HDAC inhibitor, its exact mechanism of action in DMD is not fully understood. In April 2025, Italfarmaco announced that the CHMP of the European Medicines Agency (EMA) issued a positive opinion on DUVYZAT. The drug is also being evaluated in other indications: Phase III trial in Becker muscular dystrophy (BMD) and Phase II trial in polycythemia vera. HIYASTA (HBI-8000) is an epigenetic immunomodulator approved for several indications, including use as monotherapy for certain subtypes of relapsed or refractory T-cell non-Hodgkin's lymphoma, specifically adult T-cell leukemia-lymphoma (ATLL) and peripheral T-cell lymphoma (PTCL) in Japan. HUYABIO International is conducting a multinational Phase III trial evaluating HIYASTA in combination with nivolumab for previously untreated metastatic or unresectable malignant melanoma. HIYASTA is an oral HDAC Class I inhibitor that induces cell cycle arrest and apoptosis in tumor cells, which underlies its efficacy in lymphoma. In September 2020, the Japanese Ministry of Health, Labour and Welfare (MHLW) granted orphan drug designation (ODD) to HIYASTA for relapsed/refractory ATL, and in December 2015 for PTCL. In June 2021, HUYABIO also received regulatory approval in Japan for HBI-8000 as monotherapy for relapsed/refractory ATL. Key Emerging HDAC Inhibitors and Companies Xynomic Pharmaceuticals (Abexinostat), Viriom (Quisinostat), EOC Pharma/Syndax Pharmaceuticals (Entinostat), Jubilant Therapeutics (JBI-802), and several other companies are currently engaged in the development and production of HDAC inhibitors, which have the potential to significantly impact and enhance the HDAC inhibitors market. Abexinostat, an oral, broad-spectrum HDAC inhibitor originally developed by Pharmacyclics and later licensed by Xynomic Pharmaceuticals in 2017, has demonstrated encouraging activity in both blood cancers and solid tumors. The drug is currently being studied in several Phase I to III clinical trials worldwide. In September 2019, Xynomic announced that the FDA had granted Fast Track Designation (FTD) to abexinostat as a monotherapy for fourth-line treatment of relapsed or refractory follicular lymphoma. The FDA had previously granted FTD for abexinostat in combination with pazopanib for first- or second-line therapy in renal cell carcinoma. Additional Phase II trials are underway in indications such as Diffuse Large B-cell Lymphoma (DLBCL) and Follicular Lymphoma. Quisinostat is another potent, selective oral pan-HDAC inhibitor, initially developed by Janssen Pharmaceuticals. HDAC inhibitors work by inducing cell cycle arrest, promoting differentiation, and triggering apoptosis in various human cancer cell lines. By inhibiting histone deacetylase, they increase acetylation of histone proteins, resulting in a more transcriptionally active chromatin state and altered gene expression involved in proliferation, apoptosis, and differentiation. Multiple preclinical studies and Phase I/II trials have evaluated quisinostat's safety and effectiveness, showing promising anti-tumor effects in several advanced and treatment-resistant cancers — including platinum-resistant ovarian cancer (in combination with paclitaxel and carboplatin), uveal melanoma, and synovial sarcoma. The anticipated launch of these emerging therapies are poised to transform the HDAC inhibitors market landscape in the coming years. As these cutting-edge therapies continue to mature and gain regulatory approval, they are expected to reshape the HDAC inhibitors market landscape, offering new standards of care and unlocking opportunities for medical innovation and economic growth. To know more about HDAC inhibitors clinical trials, visit @ HDAC Inhibitors Treatment Recent Developments in the HDAC Inhibitors Space In May 2025, Amylyx Pharmaceuticals announced positive Week 48 data from the Phase II open-label HELIOS clinical trial of AMX0035 in adults living with Wolfram syndrome. In May 2025, the University of Miami, in collaboration with Viriom, initiated a Phase II clinical trial in the US (NCT06932757) to evaluate an oral therapy for patients with uveal melanoma who have undergone at least one prior line of treatment. In April 2025, Xynomic Pharmaceuticals concluded a Phase I/II clinical trial in the United States (NCT03939182), evaluating a combination oral therapy for patients with Diffuse Large B-cell Lymphoma and Mantle Cell Lymphoma who had received at least one prior line of treatment. Discover more about HDAC inhibitors in development @ HDAC Inhibitors Clinical Trials HDAC Inhibitors Market Dynamics HDAC inhibitors are a class of epigenetic drugs that modify gene expression by altering chromatin structure. Initially developed as anticancer agents, they have shown significant therapeutic potential in various malignancies, especially hematologic cancers like T-cell lymphoma and multiple myeloma. The market initially gained traction with FDA approvals of drugs such as Vorinostat, Romidepsin, and Panobinostat. Their unique mechanism of action targeting epigenetic regulation drew interest as precision medicine and oncology research grew, driving pharmaceutical investment in this segment. The HDAC inhibitors market continues to expand largely due to the rising prevalence of cancer worldwide and the need for more targeted therapies with better safety profiles. Additionally, ongoing clinical trials are exploring HDAC inhibitors in combination with other therapies, such as immune checkpoint inhibitors, proteasome inhibitors, and traditional chemotherapeutics. These combination approaches aim to overcome resistance and enhance efficacy, creating more opportunities for market growth. As more data emerges demonstrating synergistic benefits, partnerships between biotech startups and large pharmaceutical firms are accelerating further development. Despite this positive outlook, the market faces several challenges. Many HDAC inhibitors are associated with toxicity issues such as fatigue, gastrointestinal disturbances, and thrombocytopenia, which limit broader adoption. Competition from newer targeted therapies and immuno-oncology drugs also puts pressure on the HDAC inhibitor segment. Moreover, several compounds failed in late-stage trials due to a lack of efficacy, leading to cautious investor sentiment and prioritization of combination rather than monotherapy approaches. Looking ahead, the market dynamics are expected to be shaped increasingly by precision oncology trends and biomarker-driven patient selection, allowing more effective targeting of responders. If safety profiles can be improved and combination regimens continue to show strong results, HDAC inhibitors will retain a meaningful role in the evolving oncology landscape. Novel formulations, selective HDAC isoform inhibitors, and expansion into non-oncology indications such as neurodegenerative or inflammatory diseases may further diversify the market and drive its next phase of growth. Learn more about the HDAC inhibitors @ HDAC Inhibitors Analysis Scope of the HDAC Inhibitors Market Report HDAC Inhibitors Therapeutic Assessment: HDAC Inhibitors' current marketed and emerging therapies HDAC Inhibitors Market Dynamics: Conjoint Analysis of Emerging HDAC Inhibitor Drugs Competitive Intelligence Analysis: SWOT analysis and Market entry strategies Unmet Needs, KOL's views, Analyst's views, HDAC Inhibitors Market Access and Reimbursement Table of Contents Related Reports Multiple Myeloma Market Multiple Myeloma Market Insights, Epidemiology, and Market Forecast – 2034 report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key multiple myeloma companies, including Sanofi, Karyopharm Therapeutics, Takeda Pharmaceutical, Celgene, Bristol-Myers Squibb, RAPA Therapeutics, Pfizer, Array Biopharma, Cellectar Biosciences, BioLineRx, Celgene, Aduro Biotech, ExCellThera, Janssen Pharmaceutical, Precision BioSciences, Takeda, Glenmark (Ichnos Sciences SA), Poseida Therapeutics, Molecular Partners AG, Chipscreen Biosciences, AbbVie, Genentech (Roche), Janssen Biotech, Nanjing Legend Biotech, Merck Sharp & Dohme Corp., among others. 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Cutaneous T-cell Lymphoma Market Cutaneous T-cell Lymphoma Market Insights, Epidemiology, and Market Forecast – 2034 report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key CTCL companies, including Pfizer, Helsinn Therapeutics, Kyowa Hakko Kirin, Citius Pharmaceuticals, Soligenix, Prescient Therapeutics, Innate Pharma, Bristol-Myers Squibb, BioInvent International AB, among others. About DelveInsight DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports pharma companies by providing comprehensive end-to-end solutions to improve their performance. Get hassle-free access to all the healthcare and pharma market research reports through our subscription-based platform PharmDelve . 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2025年3月24日，比利时生物技术公司Augustine Therapeutics完成7770万欧元（约8480万美元）超额认购的A轮融资，由Novo Holdings和Jeito Capital领投，Asabys Partners、礼来、AdBio Partners、V - Bio Ventures、PMV、VIB、Gemma Frisius Fund、美国腓骨肌萎缩症（CMT）研究基金会和Newton Biocapital等现有投资者跟投。此前公司已于2024年完成1700万欧元A轮首轮融资。本轮资金将用于推进其主要候选药物ACT-100216（外周限制性HDAC6抑制剂）在腓骨肌萎缩症（CMT）的1/2期概念验证临床试验，同时支持另外两个针对神经退行性疾病和心血管代谢疾病的HDAC6抑制剂项目研发。01以改变CMT患者命运为使命的研发团队Augustine成立于2019年，专注于开发神经肌肉、神经退行性疾病和心代谢疾病新疗法，是VIB与比利时鲁汶大学VIB-KU Leuven癌症生物学中心联合孵化的分拆企业。公司名称的灵感源自19世纪法国神经学家让-马丁·夏科（Jean-Martin Charcot）收治的一位特殊患者。这位患者所罹患的遗传性周围神经病变，经后世医学界系统研究后被正式命名为夏科-马里-图斯病（Charcot-Marie-Tooth disease，简称CMT）。作为全球最常见的遗传性神经疾病之一，CMT又称遗传性运动感觉神经病，患病率约为1/2500，多在儿童至青年期发病。其核心病理特征为周围神经轴突或髓鞘的进行性退化，临床表现为肌肉萎缩无力、感觉丧失、剧烈疼痛及足部畸形等并发症。令人遗憾的是，这种严重影响生活质量的疾病至今尚无获批治疗方案。Augestine以该病命名，不仅是对医学先驱探索精神的致敬，更承载着改变患者命运的使命。Augustine拥有经验丰富的生物制药领导者与资深科研团队，并且得到国际领先专家组成的顾问委员会的支持。首席执行官Gerhard Koenig博士深耕神经退行性疾病领域，曾创立Arkuda Therapeutics（治疗包括阿尔茨海默氏病和帕金森病在内的神经退行性疾病），还在拜耳、富达生物科学集团等历任要职。首席医疗官Carl Bjartmar博士专攻神经肌肉，神经病学及肿瘤学适应症的临床开发，支持过多款罕见病新药研发，曾担任赛诺菲旗下Genzyme的高级医学总监。首席科学官Rie Schultz Hansen拥有20余年药物发现与早期药物开发经验，此前曾任Aelin Therapeutics的首席科学官，在基于诱导蛋白聚集的降解剂平台开发（用于中和致病蛋白）中发挥关键作用。科学顾问团队由Ludo Van Den Bosch教授领衔，其突破性研究——“借选择性HDAC6抑制剂解决外周神经轴突变性机制”奠定了公司HDAC6抑制疗法的科学基础，联合创始人Clara campàs-moya与深耕医药创新20年的Bart De Taeye博士等携手，从技术研发到商业化路径为公司提供全方位支持，共同推进靶向HDAC6创新疗法开发落地。02发现HDAC6与运动神经元疾病关联，开发精准靶向小分子抑制剂在运动神经元疾病（如肌萎缩侧索硬化症，ALS）和不同的神经病变（如腓骨肌萎缩症，CMT）的研究过程中，Van Den Bosch教授发现HDAC6在这些疾病的发生发展中扮演着关键角色。HDAC6是一种Ⅱ类组蛋白脱乙酰酶，与其他HDAC不同，它主要位于细胞质中且可使非组蛋白脱乙酰化。通过这种方式，它调节各种细胞过程，包括细胞生长、细胞迁移、细胞内运输以及细胞死亡等。此外，HDAC6还可通过其泛素结合域在泛素化蛋白的清除中发挥作用。在正常生理状态下，微管（microtubule）由α-微管蛋白（α-tubulin）和β-微管蛋白（β-tubulin）聚合而成，是细胞内物质运输的重要通道，维持着轴突运输的正常进行，保证神经细胞的营养供应和信号传导。然而，在CMT、ALS等疾病中，HDAC6异常激活，过度去乙酰化α-微管蛋白，导致微管结构不稳定，完整性和连续性被破坏，进而使轴突运输功能受阻，神经细胞的营养供应和信号传导通路被破坏，最终造成神经细胞变性、功能丧失。基于上述发现，Van Den Bosch教授提出利用选择性HDAC6抑制剂来干预疾病进程。通过抑制HDAC6的活性，能够阻止其对α-微管蛋白的过度去乙酰化，保护微管的乙酰化状态和结构稳定，从而恢复轴突运输功能，促进神经细胞的修复再生，延缓神经损伤，且外周靶向特性可减少对其他组织器官的潜在副作用。HDAC6对微管（microtubule）结构和功能的影响  来自：Augustine官网03围绕HDAC6靶点，聚焦三大疾病领域Augustine的研发管线以HDAC6为核心靶点，构建覆盖周围神经病、中枢神经系统疾病和心脏代谢疾病三大领域的跨领域研发矩阵。Augustine研发管线进程  来自：Augustine官网● 周围神经病领域：CMT项目（AGT-100216）临床前研究显示，HDAC6抑制剂可逆转CMT模型中的疾病进展。同时，对于化疗诱导周围神经病变（Chemotherapy-Induced Peripheral Neuropathy，CIPN），HDAC6抑制剂对CIPN不仅有稳健的治疗作用，还能在不损害化疗抗癌活性的前提下发挥预防作用。针对上述特性，Augustine研发了AGT-100216——一款外周限制性、选择性小分子HDAC6抑制剂，具有独特的“非异羟肟酸（non-hydroxamic acid）、非酰肼（non-hydrazide）”化学结构，口服生物可利用，适用于慢性疾病的长期治疗。目前，Augustine已启动针对CMT的Ⅰ期临床试验。该试验是一项随机、双盲、安慰剂对照的首次人体试验，旨在评估口服AGT-100216在健康成年志愿者中的安全性、耐受性、药代动力学和探索性药效学。● 中枢神经系统疾病领域：神经退行性疾病药物（Central Acting HDAC6i）基于Van Den Bosch教授的研究延伸，HDAC6异常激活在阿尔茨海默病（AD）、肌萎缩侧索硬化症（ALS）等中枢神经退行性疾病中同样扮演关键角色。其过度去乙酰化α-微管蛋白会破坏脑内神经元轴突运输，加剧β-淀粉样蛋白沉积（AD）或运动神经元变性（ALS）。Augustine据此开发针对脑内神经元病理微环境设计的脑渗透型HDAC6抑制剂，旨在穿透血脑屏障（BBB）后选择性抑制中枢HDAC6，恢复神经元物质运输功能，以此突破外周药物无法作用于中枢的局限。目前，该药物处于“先导优化期（lead optimization）”，需通过结构优化提升脑渗透效率、增强靶点选择性，为进入临床前研究（Preclinical）做准备。● 心脏代谢疾病领域：心脏代谢疾病药物（Peripheral HDAC6i）HDAC6在心肌细胞与脂肪细胞中高表达，其异常激活会通过两条路径驱动疾病：促进心肌细胞微管解聚，导致左心室舒张功能障碍（HFpEF）；抑制胰岛素信号通路，加剧脂肪细胞胰岛素抵抗（2型糖尿病）。基于此，Augustine同步开发外周限制性HDAC6抑制剂，聚焦心血管代谢疾病这两大病理环节，通过选择性抑制外周组织靶点，探索改善心血管功能、调节心脏代谢的新路径。目前，该药物处于“先导优化后期（advanced lead optimization）”，后续将推进临床前研究及IND申报，逐步进入临床试验，潜在覆盖心力衰竭等心脏代谢疾病。由此可看出，Augustine以HDAC6为单一靶点深度开发，系统性挖掘神经保护与代谢调节的跨疾病能力，有望为CMT、ALS等难治性疾病提供首个机制驱动的HDAC6靶向疗法，填补临床空白。04改善广谱抑制剂毒副作用，HDAC6靶向治疗赛道加速突破目前，大部分已上市或临床中的HDAC抑制剂为泛HDACs抑制剂，对HDAC亚型并没有区分能力，存在潜在的毒副作用。而HDAC6作为HDAC家族最大的成员，是多种罕见病的潜在靶点，具有巨大潜力，因此众多药企纷纷布局HDAC6抑制剂领域，加速推进研发进程。全球部分HDAC6在研管线盘点国际方面，此前，诺华医药与韩国制药公司钟根堂（CKD）达成13亿美元合作协议，获得HDAC6抑制剂CKD-510除韩国以外的全球独家开发及商业化权利。CKD-510是一款高选择性靶向HDAC6的小分子抑制剂，2020年3月就获得了FDA授予的孤儿药资格认定。除了诺华与CKD的合作外，国内还有多款靶向HDAC6的在研新药备受行业关注。如上海徐诺药业的艾贝司他，其涵盖不同肿瘤适应症，正在推进临床试验，全力推进药物从临床向商业化转化；中国药科大学朱雍&陆涛团队正在开发选择性抑制剂靶向HDAC6降解，用于治疗急性肝损伤。如果您想对接文章中提到的项目，或您的项目想被动脉网报道，或者发布融资新闻，请与我们联系；也可加入动脉网行业社群，结交更多志同道合的好友。近期推荐声明：动脉网所刊载内容之知识产权为动脉网及相关权利人专属所有或持有。未经许可，禁止进行转载、摘编、复制及建立镜像等任何使用。文中如果涉及企业信息和数据，均由受访者向分析师提供并确认。动脉网，未来医疗服务平台