NANT 2021-02: A Randomized Phase 2 Study of 131I-MIBG With Vorinostat VS. 131I-MIBG With Dinutuximab vs. 131I-MIBG With Dinutuximab and Vorinostat for Relapsed or Refractory Neuroblastoma

Patients will then be randomized at study entry to one of three treatment arms. Patients on Arm A will receive a single treatment course with 131I-MIBG with vorinostat. Patients on Arm B will receive a single treatment course with 131I-MIBG and dinutuximab. Patients on Arm C will receive a single treatment course with 131I-MIBG with dinutuximab + vorinostat. After this course of treatment, we will check to see your response and then check to see how you are doing over time. All patients may choose to proceed to a second course of the same treatment if they and their physician feel healthy enough to do so. Approximately 118 patients will be receiving therapy on this trial.

Start Date31 Jul 2026

Sponsor / Collaborator

New Approaches to Neuroblastoma Therapy

[+2]

NCT06995521

/ Not yet recruitingPhase 2IIT

Use of Vorinostat as GVHD Prophylaxis in Children, Adolescents, and Young Adults With Non-Malignant Disorders Undergoing Allogeneic Blood and Marrow Transplantation

This is a single-arm, open label, phase 2 study to determine the safety and efficacy of vorinostat without serotherapy as GVHD prophylaxis when combined with either tacrolimus and methotrexate or post-transplant cyclophosphamide, tacrolimus, and mycophenolate in patients aged 1 to 26 years of age with non-malignant disorders undergoing bone marrow transplant following myeloablative conditioning.

Start Date01 Mar 2026

Sponsor / Collaborator

University of Michigan

NCT06693284

/ RecruitingEarly Phase 1IIT

A Window of Opportunity Trial of Mirdametinib Plus Vorinostat for NF1 Associated, H3K27 Trimethylation Deficient Malignant Peripheral Nerve Sheath Tumor [MPNST]

This is a single center Phase 0 "window of opportunity" trial for the treatment of newly diagnosed, PRC2 deficient, primary malignant peripheral nerve sheath tumors (MPNSTs) with a short course of the combination of mirdametinib and vorinostat prior to the most appropriate standard of care treatment for their specific tumor (typically localized radiation followed by surgical resection). Four to eight patients, 12 years of age or older and meeting the study's biomarker inclusion criteria, would be enrolled onto this trial. After voluntary written consent (assent with parent consent for minors) the patient undergoes MRI and PET imaging of the tumor and a needle biopsy to collect tumor is performed. Patients with histone H3K27 trimethylation deficient MPNST, as confirmed by immunohistochemistry, receive a single 28-day course of mirdametinib and vorinostat at standard oral dosing for each. At day 26, 27, or 28 the patient returns to clinic for a research visit repeating the baseline MRI and PET imaging and the needle biopsy for tumor tissue.
This ends direct study participation. The patient goes on to the most appropriate standard of care treatment for their MPNST. Information about the subsequent standard of care treatment is collected for the purposes of this study.

Start Date20 Nov 2025

Sponsor / Collaborator

University of Minnesota

100 Clinical Results associated with Vorinostat

100 Translational Medicine associated with Vorinostat

100 Patents (Medical) associated with Vorinostat

4,891

Literatures (Medical) associated with Vorinostat

01 May 2026·COLLOIDS AND SURFACES B-BIOINTERFACES

Esterase-activatable dimeric HDAC inhibitor nanotherapeutics for enhanced lymphoma epigenetic therapy

Article

Author: Li, Tongyu ; Qian, Wenbin ; Yi, Ping ; Fan, Shufang ; Zhuang, Wanchuan ; Ouyang, Guifang

BACKGROUND:

Despite advances in lymphoma therapy, significant challenges persist including R-CHOP resistance and CAR-T toxicity. Hydroxamate-based histone deacetylase inhibitors (HDACi) like vorinostat (SAHA) offer epigenetic therapeutic potential but are limited by poor bioavailability and rapid clearance.

METHODS:

To overcome these barriers, we rationally designed an esterase- activatable dimeric prodrug by conjugating two SAHA molecules via a glutaric acid linker (SAHA-cc-SAHA). This prodrug co-assembled with DSPE-PEG₂₀₀₀ into nanoparticles (cc-diSAHA NPs). The system was characterized (DLS/TEM), and its drug release profile was assessed with/without porcine liver esterase (PLE). Antitumor activity was evaluated in EL4/A20 lymphoma cells (apoptosis/cycle assays, etc) and EL4 allograft. Transcriptomic mechanisms were deciphered by RNA-seq.

RESULTS:

The cc-diSAHA NPs were uniform spheres (∼74 nm, PDI = 0.187) with excellent colloidal stability and minimal drug leakage (<4 % in 7 days), while enabling rapid drug release upon esterase stimulation (92.4 % within 7 h with PLE). In vitro, they demonstrated broad-spectrum anti-lymphoma activity, inducing G₀/G₁ arrest and apoptosis, albeit with delayed kinetics versus free formulations, consistent with a sustained-release profile. Transcriptomics revealed multifaceted mechanisms, including potent activation of interferon-mediated immunogenic stress and hematopoietic differentiation, alongside enriched adhesion and redox metabolism pathways. In vivo, intravenous cc-diSAHA NPs suppressed EL4 tumor growth significantly more than oral SAHA (819.36 vs 1594.40 mm³; p < 0.01), without inducing systemic toxicity or organ damage.

CONCLUSION:

This nanoplatform overcomes HDACi delivery barriers by reconciling the stability-activation paradox, providing a therapeutically viable option for lymphoma patients ineligible for standard intensive therapies.

01 Apr 2026·BIOORGANIC & MEDICINAL CHEMISTRY LETTERS

Design, synthesis and biological evaluation of ALK/HDAC dual-targeting agents

Article

Author: Xue, Xia ; Zhang, Yingjie ; Li, Shunda ; Liu, Huiping ; Xue, Xintong ; Fu, Ruiwei ; Gao, Yan ; Kong, Ye

Dual inhibition of ALK and HDACs might be an effective cancer treatment approach. We designed and synthesized novel dual ALK and HDAC inhibitors using molecular hybridization and pharmacophore merging. In enzymatic assays, compound 19b showed dual inhibitory activity against ALK (ALK WT IC₅₀ = 8.0 ± 1.2 nM) and HDACs (HeLa cell nuclear extract IC₅₀ = 1.18 ± 0.22 μM). Notably, 19b exhibited ~5-fold greater inhibition than Staurosporine and approved ALK inhibitor Brigatinib against the ALK G1202R mutant. Additionally, 19b demonstrated strong activity in ALK-related neuroblastoma SK-N-BE2 cells, comparable to controls SAHA, MS275, and Brigatinib. In SK-N-BE2 cells, 19b treatment led to increased apoptosis and G2/M arrest. Docking studies explained the potent dual inhibition by 19b. These findings support the promise of 19b as a dual ALK/HDAC inhibitor for managing neuroblastoma, especially ALK-positive cancer.

01 Mar 2026·BIOORGANIC & MEDICINAL CHEMISTRY LETTERS

Novel 2-mercaptoquinazolinone N-hydroxycinnamamides as histone deacetylase inhibitors: design, synthesis, and anticancer evaluation

Article

Author: Ngan, Nguyen Phuong ; Thao, Le Thi ; Anh, Duong Tien ; Tung, Truong Thanh ; Kim, Ha Young ; Cuong, Bui Quang ; Nam, Nguyen-Hai ; Dung, Do Thi Mai ; Kang, Jong Soon ; Han, Sang-Bae ; Oanh, Dao Thi Kim ; Song, Eun Hye ; Kang, Da Hyeon ; Kim, Hwa Kyung

Histone deacetylases (HDACs) are key therapeutic targets in oncology, and hydroxamic acid derivatives represent one of the most effective inhibitor classes. To explore the structure-activity relationships of this scaffold, a series of 2-mercaptoquinazoline-based hydroxamic acid derivatives (4a-e, 7a-i, and 10a-e) were synthesized and evaluated for HDAC inhibition and anticancer activity, with SAHA serving as a positive control. The 4a-e series emerged as the most potent HDAC inhibitors, with IC₅₀ values of 0.33-0.87 μM, led by 4a (0.33 ± 0.02 μM), though weaker than SAHA (0.06 ± 0.01 μM). Cytotoxicity assays across colorectal (SW620, HCT116), breast (MDA-MB-231), prostate (PC-3), and lung (A549) cancer cell lines demonstrated that 4b (6-CH₃) and 4c (7-CH₃) were the most effective, achieving IC₅₀ values of 0.93-1.34 μM in HCT116 and 1.79-2.08 μM in SW620. In the 7a-i series, compounds 7b (6-CH₃), 7d (6-Cl), and particularly 7f (7-F) demonstrated notable cytotoxicity with IC₅₀ values of 1.14-3.64 μM across SW620, HCT116, and MDA-MB-231 cells, comparable to the most active derivatives in the 4-series; conversely, bulky halogen substituents (7g, 7i) or the unsubstituted analog 7a led to markedly reduced activity (> 4.5 μM). The 10a-e series was significantly less active, and 10e (7-F) showed undesirable toxicity toward normal MRC-5 fibroblasts (IC₅₀ = 0.72 ± 0.02 μM). Mechanistic studies further confirmed that 4c and 7f induced G₂/M arrest and apoptosis in SW620 cells. Collectively, these findings highlight 4a-c and 7f as promising lead compounds, combining submicromolar HDAC inhibition, potent antiproliferative effects, and acceptable selectivity, providing a strong foundation for further development of quinazoline-based HDAC inhibitors. Molecular docking studies supported these results by revealing favorable interactions of the hydroxamate zinc-binding group and the 2-mercaptoquinazoline scaffold within the HDAC active site, consistent with the observed structure-activity relationship (SAR) trends.

News (Medical) associated with Vorinostat

02 Dec 2025

·www.businesswire.com

Unravel Biosciences Announces Approval by Colombian Health Regulatory Authority to Initiate RVL-001 Clinical Studies for Rett Syndrome and Pitt Hopkins Syndrome

BOSTON--(BUSINESS WIRE)--Unravel Biosciences, Inc., (“Unravel”), a clinical-stage therapeutics company established to advance drugs for complex diseases through its Predictable Medicine™ platform, today announced the approval to initiate Unravel’s RVL-001 clinical trials for Rett syndrome (“RTT”) and Pitt Hopkins syndrome (“PTHS”) in Colombia by the Colombian Health Regulatory Agency (“INVIMA”). Today’s announcement is a validation of Unravel's efficient approach towards rapid clinical testing of promising therapeutics for rare and complex disorders, conditions that afflict nearly 10% of the world’s population. Unravel's proprietary data and BioNAV™ discovery engine established dynamic transcriptome network profiles, called “Living Molecular Twins,” to identify RVL-001 as a potentially promising therapeutic drug for RTT and PTHS. RVL-001 targets a novel therapeutic mechanism, which will be clinically evaluated for the first time in these studies. In addition to Unravel’s RVL-001 program for RTT and PTHS, the company has also initiated development of RVL-002, a first-in-class novel molecule for RTT syndrome. INVIMA approval allows both programs to commence recruiting immediately, with first patient dosing anticipated in January 2026 at the Universidad de Antioquia’s Center for Technological Development (“PECET”), a clinical study center of excellence and designated INVIMA clinical trial site in Medellin, Colombia. Both RVL-001 proof of concept studies are placebo-controlled, “n-of-1” trials with a target enrollment of 15 RTT patients and 5 PTHS patients. More information can be found at https://clinicaltrials.gov/study/NCT07150013 and https://clinicaltrials.gov/study/NCT07150026 "We are thrilled to commence both clinical trials as a critical milestone in our patient-driven, platform-developed therapeutics programs,” said Richard Novak, Ph.D., Unravel Co-Founder and CEO. "Today’s announcement is a validation of our efficient approach towards rapid clinical testing of promising therapeutics for rare and complex disorders, conditions that afflict nearly 10% of the world’s population." RTT and PTHS are both rare neurogenetic disorders starting in early childhood that lead to debilitating cognitive, motor and autonomic disability. Despite one approved treatment for RTT, there remains a significant unmet need for novel treatments having meaningful efficacy and acceptable safety and tolerability. There are no known treatments for PTHS. About Unravel Biosciences Unravel Biosciences is a clinical-stage therapeutics company that integrates AI systems biology computation with rapid clinical validation of discovered targets, leading to four clinical trials starting in 2026. Unravel leverages its proprietary BioNAV™ platform and primary transcriptomics data to create Living Molecular Twins of real patients that can be used to predict therapeutic response. This enables target and drug discovery, preclinical screening, patient stratification, and clinical validation to find treatments for complex diseases using a systematic, data-driven approach called Predictable Medicine™. Unravel's platform discovered RVL002, a first-in-class small molecule that targets mitochondrial metabolism and has multiple clinical applications, including neurodegenerative and metabolic disorders, and identified RVL069, a molecule that targets a novel mechanism to treat dystonias. The rareSHIFT™ program provides platform and proprietary datamine access to foundation and biotech partners to accelerate and clinically derisk therapeutics. www.unravel.bio and www.rareshift.org

Clinical Study

22 Oct 2025

·www.businesswire.com

Global Registry Data Show Meaningful Overall Survival Benefit in Patients with Advanced Cutaneous T-cell Lymphoma (CTCL) treated with POTELIGEO (mogamulizumab)

GALASHIELS & MARLOW, & BIRMINGHAM, United Kingdom--(BUSINESS WIRE)--Kyowa Kirin International (KKI), a wholly owned subsidiary of Kyowa Kirin Co., Ltd. (TSE:4151, Kyowa Kirin), a Japan-based global specialty pharmaceutical company, and the University Hospitals Birmingham NHS Foundation Trust, today announced new overall survival (OS) insights from the PROCLIPI (PROgnostic Cutaneous Lymphoma International Prognostic Index) Study aligning with previous findings that suggest mogamulizumab may offer meaningful OS benefit for patients living with mycosis fungoides (MF) and Sézary syndrome (SS), two rare conditions and subtypes of cutaneous T-cell lymphoma (CTCL).2 Key findings2: For patients with advanced-stage MF and SS (n=371), the median overall survival from the onset of treatment was higher for patients treated with mogamulizumab (n=72) at 64 months compared with those not receiving mogamulizumab (n=175) at 54 months (p<0.01). Patients treated with mogamulizumab showed a trend toward improved OS compared to those not receiving mogamulizumab across risk-stratified groups according to the new Cutaneous Lymphoma International Prognostic Index (CLIPI). For a subset of patients with SS (n=96), the median OS is around 6.5 years for patients treated with mogamulizumab (n=46) compared with around 3 years for systemic treatment but not mogamulizumab (n=50) (p<0.01). This research was presented as part of the Scientific Proceedings at this year’s European Organisation for Research and Treatment of Cancer Cutaneous Lymphoma Tumour Group (EORTC-CLTG) Annual Meeting in Athens, Greece.2 The data contributes to a growing body of evidence suggesting potential OS benefits with mogamulizumab, 4,5 highlighting the importance of collaboration across the CTCL community in advancing understanding of survival outcomes and helping to shape the future of patient care. “The PROCLIPI Study demonstrates the power of global collaboration in rare diseases. By bringing together data from across the world, we can generate insights that simply wouldn’t be possible in isolation,” says Professor Julia Scarisbrick, Chief Investigator of the PROCLIPI Study and Honorary Professor of Dermatology, University Hospitals Birmingham NHS Foundation Trust. “We are proud to coordinate this initiative as we’re working to build rigorous scientific evidence while giving patients and their families a better understanding of what long-term survival looks like.” The PROCLIPI Study, now in its tenth year, aims to develop prognostic indices for MF and SS. The study collects comprehensive data, including clinical, haematological, pathological, imaging, treatment with responses, quality of life and survival data.1 Recently, a new prognostic index (CLIPI) for advanced CTCL was published to enable precise patient risk stratification.6 “For those of us in the CTCL community, survival isn’t just about numbers on a chart - it’s about being able to spend more time with our families, plan for the future, and live life with dignity”, says Susan Thornton, CEO, Cutaneous Lymphoma Foundation. “That’s why initiatives like PROCLIPI are important, as the data collected reflects real lived experiences and can help inform future improvements in care for patients.” The independent international PROCLIPI Study aligns with Kyowa Kirin International’s commitment to generating real-world insights that advance the understanding of CTCL and turn these clinical insights into meaningful improvements in access, policy, and patient care. Cutaneous T-cell lymphoma is a rare disease7, and innovation in this environment can be challenging due to the rarity of this disease.8 Yet despite these challenges, mounting evidence continues to emerge4,5 to help strengthen our collective understanding of potential treatment benefits. “These insights into improved overall survival for patients living with CTCL mark an important step forward, providing a stronger clinical evidence base and reinforcing the value of international networks in rare disease research,” says Dr Nick Kronfeld, Head of Medical Affairs, Kyowa Kirin International. “By working alongside scientific and patient communities in CTCL, we can gain a better understanding of real-world outcomes, enabling us to bring life-changing value to patients and families, not just today but over the long term.” Note to editors The OS data from the PROCLIPI Study was presented as an oral presentation during the EORTC CTCL Annual Group Meeting 2025. 2 KKI did not sponsor the PROCLIPI study or OS analysis. The median OS for patients (n=72) receiving mogamulizumab compared to those not receiving mogamulizumab (n=175) was 64 months versus 54 months. 2 Median OS data amongst risk stratifications according to CLIPI: 2,6 Low-risk: 67 months vs. ‘not reached’ for mogamulizumab versus those who had not received mogamulizumab Medium-risk: 64 vs. 48 months for mogamulizumab versus those who had not received mogamulizumab High-risk: 26 vs. 13 months for mogamulizumab and for those who had not received mogamulizumab Low-risk: 67 months vs. ‘not reached’ for mogamulizumab versus those who had not received mogamulizumab Medium-risk: 64 vs. 48 months for mogamulizumab versus those who had not received mogamulizumab High-risk: 26 vs. 13 months for mogamulizumab and for those who had not received mogamulizumab About Kyowa Kirin Kyowa Kirin aims to discover novel medicines with life-changing value. As a Japan-based Global Specialty Pharmaceutical Company, we have invested in drug discovery and biotechnology innovation for more than 70 years and are currently working to engineer the next generation of antibodies and cell and gene therapies with the potential to help patients affected by severe and rare diseases. A shared commitment to our values, to sustainable growth, and to making people smile unites us across our four regions – Japan, Asia Pacific, North America, and EMEA/International. You can learn more about the business of Kyowa Kirin at: https://www.kyowakirin.com About University Hospitals Birmingham NHS Foundation Trust University Hospitals Birmingham NHS Foundation Trust (UHB) is one of the largest teaching hospital trusts in England, serving a regional, national and international population. The Trust provides high-quality acute and specialist care across four main hospital sites, Queen Elizabeth Hospital Birmingham, Heartlands Hospital, Good Hope Hospital, and Solihull Hospital, as well as a range of community services. With over 25,000 staff and approximately 2,700 beds, UHB delivers care to more than 2.2 million patients annually. It is a regional centre for trauma, burns, neurosciences, cancer, and military medicine - proudly hosting the Royal Centre for Defence Medicine. UHB is also a leader in clinical research and innovation, home to the NIHR Birmingham Biomedical Research Centre and the Institute of Translational Medicine. Guided by its 2024-2029 strategy, Building Healthier Lives, UHB is committed to improving health and wellbeing, shaping an equitable and sustainable future, and creating excellent experiences for patients and staff. The Trust’s core values, Kind, Connected, and Bold, underpin its mission to deliver compassionate care, foster collaboration, and drive innovation across all services. For more information, visit www.uhb.nhs.uk. About PROCLIPI PROCLIPI is the PROgnostic Cutaneous Lymphoma International Prognostic Index Study with the main aim to produce a prognostic index in mycosis fungoides (MF) and Sezary syndrome (SS). The study prospectively collects clinical, haematological, pathological, imaging, treatment with responses, quality of life and survival data using careful predefined datasets. Patients are subject to a central review to confirm diagnosis. PROCLIPI opened in 2015 and recruitment has been strong with to date over 2,000 patients recruited from 52 Centres from 19 countries across 6 continents.1 This trial was registered at www.ClinicalTrials.gov NCT02848274.3 About MF and SS MF and SS are two subtypes of CTCL, which is itself a rare form of non-Hodgkin’s lymphoma that presents and persists in the skin.7 CTCL is treatable, but is not generally considered to be curable, and there has been a clear unmet need for novel treatment options. As well as the obvious impact of symptoms upon patients, there can be significant erosions to quality of life for those caring for an individual living with CTCL.9 MF and SS are characterised by localisation of cancerous white blood cells called T lymphocytes (T cells), to the skin.10 These cancerous T cells consistently express a protein called CCR4, which enables them to move from the blood to the skin.11-13 When these cancerous T cells move to the skin, this results in the visible early skin symptoms of red patches or plaques which can resemble psoriasis or eczema in the early stages of the disease.14 Later, for some patients, skin involvement may evolve to include tumours or reddening of the majority of the skin’s surface (erythroderma). MF—the most common CTCL subtype—accounts for approximately 60% of all CTCLs15 and is typically indolent, characterised by skin symptoms including patches or plaques, skin redness and tumours. SS is much rarer, accounting for around 5% of CTCLs,16 and is more aggressive,14 with high levels of blood involvement.16 It can cause severe itching, erythroderma, intense scaling of the skin and hair loss.14 CTCL can take, on average, between 2 and 7 years for individuals to receive a confirmed diagnosis.14 About Poteligeo (mogamulizumab) Mogamulizumab is a first-in-class humanised monoclonal antibody directed against CC-chemokine receptor 4 (CCR4), a protein consistently expressed on cancerous cells seen in both MF and SS. 11-13 Once mogamulizumab binds to CCR4, it increases attraction of immune cells from the immune system to destroy the cancerous cells.17 References 1 Scarisbrick, J. The PROCLIPI international registry, an important tool to evaluate the prognosis of cutaneous T cell lymphomas. Presse Med. 2022 Mar;51(1):104123. doi: 10.1016/j.lpm.2022.104123. 2 Peterknecht E, et al. Real-World Survival with Mogamulizumab versus Brentuximab in Advanced CTCL: Insights from PROCLIPI. Presented at EORTC 2025; Athens, Greece. Abstract #145. 3 ClinicalTrials.Gov. ID Of Prognostic Factors In Mycosis Fungoides/ Sezary Syndrome. #NCT02848274. Available at: https://clinicaltrials.gov/study/NCT02848274?term=%23NCT02848274&rank=1. Last accessed: October 2025. 4 Broccoli, A, et al. Quality and duration of responses with mogamulizumab in cutaneous T-cell lymphomas: Insights into long-lasting outcomes. Br J Haematol. 2025;00:1–5. doi: 10.1111/bjh.70161. 5 Bozonnat, A, et al. Real-life efficacy of immunotherapy for Sézary syndrome: a multicenter observational cohort study. EClinicalMedicine. 2024;79:102979. doi: 10.1016/j.eclinm.2024.102979. 6 Scarisbrick, J, et al. A new prognostic index (CLIPI) for advanced cutaneous lymphoma enables precise patient risk stratification. Blood. 2025;146 (14), 1687–1692. doi: 10.1182/blood.2025029628 7 Kim YH, Bagot M, Pinter-Brown L, et al. Mogamulizumab versus vorinostat in previously treated cutaneous T-cell lymphoma (MAVORIC): an international, open-label, randomised, controlled phase 3 trial. Lancet Oncol. 2018;19(9):1192-1204. 8 Targeted Oncology. Immunotherapy Advances and Challenges in T-Cell Lymphoma. Available at: https://www.targetedonc.com/view/immunotherapy-advances-and-challenges-in-t-cell-lymphoma. Last accessed: October 2025. 9 Williams et al (2020) – Health state utilities associated with caring for an individual with CTCL. Journal of Medical Economics. 2020; 23(10):1142-1150 10 Scarisbrick JJ, Prince M, Vermeer MH, et al. Cutaneous Lymphoma International Consortium Study of Outcome in Advanced Stages of Mycosis Fungoides and Sézary Syndrome: Effect of Specific Prognostic Markers on Survival and Development of a Prognostic Model. J Clin Oncol. 2015;33(32):3766-3773. 11 Ferenczi K, et al. Increased CCR4 expression in cutaneous T cell lymphoma. J Invest Dermatol. 2002;119:1405–10. 12 Yoshie O, et al. Expression of CCR4 in Adult T-Cell Leukemia and Human T-cell Leukemia Virus Type 1-transformed T cells. Blood. 2002;99(5):1505–11. 13 Ishida T, et al. Clinical Significance of CCR4 Expression in Adult T-cell Leukemia/Lymphoma: Its Close Association With Skin Involvement and Unfavorable Outcome. Clin Cancer Res. 2003;9:3625–34. 14 Lymphoma Coalition. Cutaneous lymphoma – a patient’s guide. Available at: https://lymphomacoalition.org/wp-content/uploads/Cutaneous_lymphoma_-_patients_guide_-.pdf. Last accessed: October 2025. 15 Willemze R, et al. The 2018 update of the WHO-EORTC classification for primary cutaneous lymphomas. Blood. 2019;133(16):1703-1714. 16 Trautinger F, et al. European Organisation for Research and Treatment of Cancer consensus recommendations for the treatment of mycosis fungoides/Sézary syndrome - Update 2017. European Journal of Cancer. 2017;77:57–74. 17 Duvic M, et al. Mogamulizumab for the treatment of cutaneous T-cell lymphoma: recent advances and clinical potential. Ther Adv Hematol. 2016;7(3):171–174

Clinical ResultPhase 3Immunotherapy

20 Aug 2025

·www.prnewswire.com

HDAC Inhibitors Market Sees Upward Trajectory Across the 7MM During the Forecast Period (2025-2034) Driven by Innovation in Cancer Therapies | DelveInsight

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. Amyotrophic Lateral Sclerosis Market Amyotrophic Lateral Sclerosis 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 ALS companies, including AB Science, Alector, GSK, Brainstorm Cell Therapeutics, Ionis Pharmaceuticals, Medicinova, Denali Therapeutics, AbbVie, Calico Life Sciences, Clene Nanomedicine Biosciences, Seelos Therapeutics, Prilenia Therapeutics, Rapa Therapeutics, Neurosense Therapeutics, Helixmith, Transposon Therapeutics, Revalesio Corporation, Annexon Biosciences, Corcept Therapeutics, AL-S Pharma, Sanofi, Denali Therapeutics, Orphai Therapeutics, among others. Peripheral T-cell Lymphoma Market Peripheral 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 PTCL companies, including Secura Bio, Shanghai YingLi Pharmaceutical, Dizal Pharmaceuticals, Viracta Therapeutics, Innate Pharma, CStone Pharmaceuticals, Affimed, Otsuka Pharmaceutical, Astex Pharmaceuticals, Autolus, Myeloid Therapeutics, Merck Sharp & Dohme, Bristol Myers Squibb, among others. 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 . Contact Us Shruti Thakur [email protected] +14699457679 Logo: SOURCE DelveInsight Business Research, LLP WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Phase 2Phase 3Fast TrackOrphan DrugImmunotherapy

100 Deals associated with Vorinostat

External Link

KEGG	Wiki	ATC	Drug Bank
D06320	Vorinostat	L01XX38	DB02546

R&D Status

Approved

10 top approved records.

to view more data

Indication	Country/Location	Organization	Date
Cutaneous T-Cell Lymphoma	United States	Merck Sharp & Dohme Corp.	06 Oct 2006

Developing

10 top R&D records.

to view more data

Indication	Highest Phase	Country/Location	Organization	Date
Refractory Multiple Myeloma	Phase 3	-	Merck Sharp & Dohme Corp.	01 Dec 2008
Multiple Myeloma	Phase 3	Spain	Merck & Co., Inc.	27 Nov 2008
Plasma Cell Leukemia	Phase 3	Belgium	-	13 Nov 2008
Recurrent cutaneous T-cell lymphoma	Phase 3	Italy	Merck Sharp & Dohme SA	17 Dec 2007
Advanced Lung Non-Small Cell Carcinoma	Phase 3	-	Merck Sharp & Dohme Corp.	01 May 2007
Non-squamous non-small cell lung cancer	Phase 3	-	Merck Sharp & Dohme Corp.	01 May 2007
metastatic non-small cell lung cancer	Phase 3	Denmark	Merck & Co., Inc.	26 Mar 2007
Non-small cell lung cancer stage IIIB	Phase 3	Denmark	Merck & Co., Inc.	26 Mar 2007
Pleural Effusion	Phase 3	Denmark	Merck & Co., Inc.	26 Mar 2007
Lung Cancer	Phase 3	-	Merck Sharp & Dohme Corp.	30 Jun 2005

Clinical Result

Indication

Phase

Evaluation

View All Results

Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT02553460 (Pubmed \| Lancet Haematol)	Phase 1/2	Acute Lymphoblastic Leukemia KMT2A rearranged	50	BortezomibDexamethasoneVorinostatMitoxantronePegaspargase + BortezomibDexamethasoneVorinostatMitoxantronePegaspargase + BortezomibDexamethasoneVorinostatMitoxantronePegaspargasee + BortezomibDexamethasoneVorinostatMitoxantronePegaspargase + BortezomibDexamethasoneVorinostatMitoxantronePegaspargase	uvtwjiayjc(qlsrlrgeii) = usnyhcpzje huvhlzwwhx (hkyxfkducc )	Positive	01 Feb 2026
NCT03842696 (ASH2025)	Phase 1/2	Hematologic Neoplasms	43	Vorinostat + standard immunosuppression	iaxldulnfz(kydxuwxyny) = lqgclcsdmw ejptjupgob (obwbsweqiq ) View more	Positive	06 Dec 2025
NCT03263936 (CTgov)	Phase 1	Refractory acute myeloid leukemia	37	Decitabine (Dose Level 1)	yvhbkwqfrp = gdijdmklva motxuqhkfx (subodexmmg, iarziaeamf - hwkwmbmdxb) View more	-	16 Oct 2025
				Decitabine (Dose Level 2)	yvhbkwqfrp = boaihnstsu motxuqhkfx (subodexmmg, vztmjvqztt - czdfwdbqsv) View more
NCT03426891 (CTgov)	Phase 1	Glioblastoma Multiforme	21	Radiotherapy+Vorinostat+Temozolomide+Pembrolizumab (Dose Escalation Phase)	zpfgyoojhm = xryryvjcdu oyjxwblpwl (gpuvzrdmsy, fxcqztyqhw - hwcfbbejvn) View more	-	06 Oct 2025
				Radiotherapy+Vorinostat+Temozolomide+Pembrolizumab (Radiotherapy and Maintenance Phase)	xqcjnsubtz(beogdrqbxr) = irbczyhvku vxbhhophjq (knfazhralr, mjeubliyxp - whiodaauml) View more
NCT02638090 (CTgov)	Phase 1/2	metastatic non-small cell lung cancer	120	Vorinostat+Pembrolizumab (Phase I Dose Escalation)	pdpcosybfk = odoweoyhdv qifglsnvjd (nrucdnlody, ptyfckhnri - qlwypxntsf)	-	04 May 2025
				Pembrolizumab (Arm A: Pembrolizumab)	ovubnyatzb(coiyfizfun) = ywhjgewxvq kmybbdchrt (bsjmjcpviy, mwxriozyaa - tbupxtzzvv) View more
NCT00503971 (CTgov)	Phase 1/2	Non-Small Cell Lung Cancer	33	Erlotinib+Vorinostat (Vorinostat Plus Erlotinib Phase II)	affexkjfxc = fnryiutvxo iqoeiruwgu (qvozdoahfs, uvwrtaedfi - coqaqanofu) View more	-	07 Mar 2025
				Erlotinib+Vorinostat (Vorinostat Plus Erlotinib Phase I)	bwghilljbt = qtzxevqrfd fhashmfocw (iwubyaczxv, egblhoefto - bsznrioeql)
NCT02619253 (Phase I/IB, CTgov)	Phase 1	Advanced Urothelial Carcinoma \| Renal Cell Carcinoma \| Bladder Cancer	52	vorinostat+pembrolizumab (Expansion Cohort A)	dimryjzcsk = zusjcdjokm nczqebtlat (mocnjxhyhi, epmrggajpw - prydvhzeqz) View more	-	18 Nov 2024
				vorinostat+pembrolizumab (Expansion Cohort B)	dimryjzcsk = erbahesohg nczqebtlat (mocnjxhyhi, cyhotxbnce - baljycbkaj) View more
NCT03749460 \| NCT02538510 (ASCO2024)	Phase 1/2	Salivary gland cancer recurrent PDL1 expression	45	Pembrolizumab 200 mg IV q3 weeks and vorinostat 400 mg PO 5 days on/2 days off	qdnulcmlby(rybosmnusp) = rjrsmomwjc uqjicrcafh (njnjkzaaov ) View more	Positive	24 May 2024
NCT04357873 (PEVOsq, AACR2024) Manual	Phase 2	Squamous Cell Carcinoma PD-L1 expression \| tumor-infiltrating lymphocytes quantification \| HPV typing ... View more	112	Pembrolizumab anVorinostatVorinostat combination	-	Positive	22 Mar 2024
				Pembrolizumab + Vorinostat (HPV+)	krqoqecakx(ejfowmawuv) = efnpdczwxb lgqqfjspyz (kzuoyipuwd )
NCT03198559 (CTgov)	Phase 1/2	HIV Infections	5	Vorinostat+Disulfiram	sqxzqyxpoe = bjccrmvdbd ruzwgrhxyx (iddyirnspi, hiepxhpnoj - owsdqrcbhz) View more	-	09 Feb 2024

Translational Medicine

Boost your research with our translational medicine data.

view full example data

Deal

Boost your decision using our deal data.

view full example data

Core Patent

Boost your research with our Core Patent data.

view full example data

Clinical Trial

Identify the latest clinical trials across global registries.

view full example data

Approval

Accelerate your research with the latest regulatory approval information.

view full example data

Regulation

Understand key drug designations in just a few clicks with Synapse.

view full example data

AI Agents Built for Biopharma Breakthroughs

Accelerate discovery. Empower decisions. Transform outcomes.

Get started for free today!

Accelerate Strategic R&D decision making with Synapse, PatSnap’s AI-powered Connected Innovation Intelligence Platform Built for Life Sciences Professionals.

Start your data trial now!

Synapse data is also accessible to external entities via APIs or data packages. Empower better decisions with the latest in pharmaceutical intelligence.

Bio

Bio Sequences Search & Analysis

Chemical

Chemical Structures Search & Analysis