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

5,438

Literatures (Medical) associated with Vorinostat

31 Dec 2026·Molecular & Cellular Oncology

Lactylation-related genes signature panel in hepatocellular carcinoma reveals the prognostic and therapeutic optimization

Article

Author: Guo, Shuang ; Xue, Han ; Xu, Xianqun ; Zhao, Ziwu ; Long, Xinghua ; Xiao, Ting

Lactylation-related genes (LRGs) exert a significant influence on tumor progression and are critically involved in modulating responses to immune checkpoint inhibitors (ICIs). Leveraging a curated set of 332 LRGs derived from the literature, we identified prognosis-specific hepatocellular carcinoma (HCC) clusters through consensus clustering analysis. Using an integrated bioinformatics approach, we systematically evaluated differences in immune microenvironment infiltration, somatic mutations, copy number variations, and epigenetic modifications between the two tumor subtypes. We subsequently developed a lactylation-related genes signature (LRGS) and independently validated its prognostic efficacy in the TCGA cohort. Notably, the high-LRGS group exhibited significantly reduced overall survival(OS) and diminished response to ICIs. Conversely, the high-LRGS group demonstrated heightened sensitivity to several antitumor agents, including AZD5153, cediranib, foretinib, and vorinostat, relative to the low-LRGS group. Our findings establish a robust LRGS model, offering a clinically actionable tool for prognostic assessment and precision therapy in HCC.

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·Materials Today Bio

Bipolar CD4-targeted dual-DARPin-55/57 lipid nanoparticle enables efficient CRISPR/Cas-mediated HIV-1 DNA excision and reactivation blockade in latent CD4 T cell lines

Article

Author: Zhu, Yuanjun ; Baloch, Abdul Rasheed ; Sisto, Ian ; Saribas, A Sami ; Chen, Jackson ; Hu, Wenhui ; Yang, Xiaofeng ; Zhang, Danmeng ; Hossain, Reafa ; Yuan, Xinxu ; Li, Qingsheng ; Mandal, Subhra ; Xu, Jian ; Wang, Hong ; Jaijyan, Dabbu

The persistence of HIV-1 latent reservoirs remains the principal barrier to a cure, as viral rebound occurs upon interruption of antiretroviral therapy. CRISPR/Cas genome editing offers a promising strategy to excise proviruses from host genome; however, the absence of a targeted and clinically viable delivery platform has hindered its translational application. Here, we report a chemistry-driven, CD4-targeted lipid nanoparticle (LNP) delivery platform employing a unique bipolar conjugation strategy to decorate dual CD4-targeted Designed Ankyrin Repeat Proteins (DARPins-55 and -57) on LNP (dual-DARPin-LNP). The N- and C-terminally modified DARPin-55/57 was thiolated stepwise, then bipolar maleimide-thiol coupling conjugated the thiolates to the maleimide-functionalized LNP surface. This coupling strategy ensured DARPin proper orientation on the LNP surface for efficient uptake by resting CD4 T cells. This dual-DARPin-LNP system was engineered for selective and efficient co-delivery of spCas9-GFP mRNA (Sp9m) and HIV-1-specific single-guide RNAs (sgRNAs) targeting LTR and Gag (LGsg) into HIV-1 latently infected CD4 T cells. In widely used HIV-1 latency models with defined proviral modifications (J-Lat 10.6 and 2D10 cell lines), dual-DARPin-LNP loaded with Sp9m/LGsg efficiently excised integrated HIV-1 proviral DNA, as confirmed by standard PCR genotyping, absolute digital PCR quantification, confocal microscopy, and flow cytometry. Importantly, proviral excision functionally blocked HIV-1 reactivation following stimulation with latency-reversing agents suberoylanilide hydroxamic acid (SAHA) and TNFα. Together, these findings establish a modular, non-viral, receptor-guided delivery platform for CD4 T cell targeting and provide proof-of-concept for precise HIV-1 DNA excision and reactivation blockade in established latency models. This new strategy represents a step toward next-generation curative interventions against persistent HIV-1 infection.

News (Medical) associated with Vorinostat

20 Mar 2026

·www.biospace.com

Kaida BioPharma Announces Executive Leadership Team to Advance KAD-101, Built for the Ovarian Cancer Gap

FORT LAUDERDALE, Fla., March 19, 2026 (GLOBE NEWSWIRE) -- Kaida BioPharma, a biotechnology company developing targeted therapies for women with hard-to-treat gynecologic cancers, today announced its executive leadership team and board. Each member brings decades of experience in biotechnology, clinical development, regulatory strategy, manufacturing, finance, and commercialization to help advance Kaida’s lead product candidate, KAD-101, toward first-in-human clinical evaluation. The company is developing KAD-101 for recurrent ovarian cancer, where treatment options narrow quickly and durable control remains limited. Kaida BioPharma is building KAD-101, a next-generation prolactin receptor antagonist for the ovarian cancer gap, the space where patients are too often left cycling through treatment with too few durable options. In addition to her role as Chairperson of the Board, Kaida founder Dr. Stella Vnook has been named Acting Chief Executive Officer. Dr. Vnook is an accomplished biotechnology founder, executive, and advisor with extensive experience guiding early-stage therapeutics from scientific foundation through capital formation. At Kaida, she leads corporate strategy, development planning, and investor engagement. “The science brought me here, and the women out there still fighting are why I stay. At Kaida, we are building for the ovarian cancer gap, for patients whose options narrow too fast and whose disease still outpaces available therapies,” said Dr. Vnook. “By targeting the survival signals that keep these tumors alive, we believe KAD-101 has the potential to help overcome treatment resistance and create a new path toward more durable control. More options mean more time, and more time means everything.” Craig Pierson , a founder alongside Dr. Vnook, has been appointed Director at Kaida BioPharma. Mr. Pierson is a life sciences investor and company builder with more than 25 years of experience in investment banking and private capital formation. He provides strategic oversight and financing expertise. George E. Peoples, MD, FACS has been appointed fractional Chief Medical Officer. Dr. Peoples is a surgical oncologist with decades of experience in translational oncology and clinical development. He is also the founder of the Cancer Vaccine Development Program, Cancer Insight, and LumaBridge. At Kaida, he helps guide clinical strategy and IND planning. John Langenheim, PhD has been appointed fractional Chief Scientific Officer. Dr. Langenheim is a molecular biologist with more than 20 years of experience in therapeutic protein design and preclinical development. His scientific leadership has been instrumental in advancing KAD-101 toward clinical evaluation. Pamela Swiggard has been appointed Head of Regulatory Affairs. She is a pharmaceutical executive with prior experience at companies including Pfizer and Endo Pharmaceuticals. She brings deep expertise across the product lifecycle, from development through regulatory approval and commercialization. At Kaida, she leads regulatory strategy and FDA engagement in support of IND readiness for KAD-101. Eric Hacherl, PhD has been appointed Head of Manufacturing. Dr. Hacherl is a senior pharmaceutical operations leader with more than 25 years of experience in biologics manufacturing, cGMP compliance, and process development. At Kaida, he is responsible for helping ensure clinical supply readiness for KAD-101. Mark Booth has been appointed Chief Commercial Officer. Mr. Booth is a seasoned pharmaceutical and biotechnology executive with more than 25 years of experience building commercial strategies for oncology and rare disease products. His background includes leadership across launch planning, brand strategy, access, and market development for important oncology products, including EMEND, EMEND IV, Zolinza, vintafolide, KEYTRUDA, and the avutometinib plus defactinib combination for low grade serous ovarian cancer. At Kaida, he is responsible for commercial strategy for KAD-101, including positioning, market development, and long-term access planning. About Kaida BioPharma Kaida BioPharma is a biopharmaceutical company developing targeted therapies for hormone-driven gynecologic cancers. Its lead candidate, KAD-101, is a prolactin receptor antagonist being developed for recurrent ovarian cancer, where treatment options narrow quickly and many patients still face limited durable control. Kaida is focused on building innovative therapies for patients left in the ovarian cancer gap and in other areas of high unmet need. For more information, visit kaida-biopharma.com . Contact: Michelle Kafka michelle@kafka.com 407-603-5716

Executive ChangeVaccine

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

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
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
Lung Cancer	Phase 3	-	Merck Sharp & Dohme Corp.	30 Jun 2005
Malignant Pleural Mesothelioma	Phase 3	-	Merck Sharp & Dohme Corp.	30 Jun 2005
Estrogen receptor positive breast cancer	Phase 2	United States	Merck Sharp & Dohme Corp.	11 Mar 2021
Metastatic breast cancer	Phase 2	United States	Merck Sharp & Dohme Corp.	11 Mar 2021
Anus Neoplasms	Phase 2	France	Merck Sharp & Dohme Corp.	28 Oct 2020
Penile Neoplasms	Phase 2	France	Merck Sharp & Dohme Corp.	28 Oct 2020
Squamous cell carcinoma of head and neck metastatic	Phase 2	France	Merck Sharp & Dohme Corp.	28 Oct 2020

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	yqesaqlgcv(eqocwzupqf) = frkdibbpfg fwpvosnmfp (csuyqqzfbn )	Positive	01 Mar 2026
NCT03842696 (ASH2025)	Phase 1/2	Hematologic Neoplasms	43	Vorinostat + standard immunosuppression	euokurdnhw(fawgjlxxez) = uddbuahwym pxcnkhdntj (sjucphjbrv ) View more	Positive	06 Dec 2025
NCT03263936 (CTgov)	Phase 1	Refractory acute myeloid leukemia	37	Decitabine (Dose Level 1)	aynqxhcklx = zbmmtagakj rlobllznay (zsewcishwb, vwrprxhbsz - qrujrwbdbu) View more	-	16 Oct 2025
				Decitabine (Dose Level 2)	aynqxhcklx = znfpjkttuo rlobllznay (zsewcishwb, jakleyjizz - pmmngjlbcl) View more
NCT03426891 (CTgov)	Phase 1	Glioblastoma Multiforme	21	Radiotherapy+Vorinostat+Temozolomide+Pembrolizumab (Dose Escalation Phase)	cwmliapzue = muvoxxaovf emcsmkhawy (exhqafmirl, ryjcjyuzkk - ukdziqjcfm) View more	-	06 Oct 2025
				Radiotherapy+Vorinostat+Temozolomide+Pembrolizumab (Radiotherapy and Maintenance Phase)	jrfmpvptko(ywjjltwdfx) = slgdtyazvi bxaczdkzmx (cvydkxlgfw, ckogidhmhz - gtgrfduzdf) View more
NCT02638090 (CTgov)	Phase 1/2	metastatic non-small cell lung cancer	120	Vorinostat+Pembrolizumab (Phase I Dose Escalation)	mfatspkali = xkmezdfdfi plektacbua (zsxkhgnlfo, pqdwizjxbk - arzzyrxniy)	-	04 May 2025
				Pembrolizumab (Arm A: Pembrolizumab)	mzbrngedfk(crjvnwmtoh) = hbccwwprkz xbouvsafoe (vptgzbgmlk, ussunuddjx - usewnnodrb) View more
NCT00503971 (CTgov)	Phase 1/2	Non-Small Cell Lung Cancer	33	Erlotinib+Vorinostat (Vorinostat Plus Erlotinib Phase II)	rdvvavzxow = xwqoapnysx mbeyuhvilm (cvvxvuhkka, erkjcaleeu - wpydgsofae) View more	-	07 Mar 2025
				Erlotinib+Vorinostat (Vorinostat Plus Erlotinib Phase I)	fituydbcxw = ujtmfvvfhx ashlywbind (exgvvrusjh, xmqraljsze - tdekoktqqm)
NCT02619253 (Phase I/IB, CTgov)	Phase 1	Advanced Urothelial Carcinoma \| Renal Cell Carcinoma \| Bladder Cancer	52	vorinostat+pembrolizumab (Expansion Cohort A)	nldfyocgnr = fzpulsigqi dclpgpxjpe (tohblpbfjs, ykqgcfirmg - dtopjlnlki) View more	-	18 Nov 2024
				vorinostat+pembrolizumab (Expansion Cohort B)	nldfyocgnr = uyjnkcrlmv dclpgpxjpe (tohblpbfjs, ikudqswtbb - lnaomahgoh) 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	avsgygugok(qzaghltmmi) = trwumzetot bgajsvcalj (ftklpupllg ) 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+)	aabfwhkugk(tzbhbdfafg) = pstxsmyfuz kjbggbxyyr (ceetrowrjt )
NCT03198559 (CTgov)	Phase 1/2	HIV Infections	5	Vorinostat+Disulfiram	ngxgdathmn = vdltzaxnda frfcmibdlu (smbnvcxrjc, vwusbxcafc - wnwxpiptin) View more	-	09 Feb 2024

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