Farydak (panobinostat) / Secura Bio - LARVOL DELTA

Proteogenomic Analysis of HDAC4 and HDAC5 in Uveal Melanoma. (PubMed, Mol Med) - "In drug screening experiments, we identified a high sensitivity of primary UM cell lines to the pan-histone deacetylase (HDAC) inhibitor panobinostat and addressed the question whether two class IIa HDACs, HDAC4 and HDAC5, could serve as potential therapeutic targets in UM...This was particularly evident in the NF-κB pathway, which showed decreased NF-κB p65 phosphorylation in UM cell lines with monosomy in chromosome 3 (M3) genotype and mutations in BRCA1-associated protein 1 (BAP1). In summary, our results suggest that HDAC4/5 inhibitors alone are insufficient for monotherapy in UM cells, but the identification of HDAC4/5-regulated genes involved in cancer progression provides a rationale for exploring combination strategies that include HDAC4/5 inhibition."

Journal • Choroidal Melanomas • Eye Cancer • Melanoma • Ocular Melanoma • Oncology • Solid Tumor • Uveal Melanoma • BAP1 • HDAC4 • HDAC5

Dendritic Cell Modulation Enhances aCD40 Immunotherapy in Multiple Myeloma (IMS 2026) - "While Flt3L effectively expands DCs, it fails to translate into therapeutic benefit in MM, underscoring the limitations of DC expansion alone. In contrast, ICD induction using panobinostat enhances cDC activation and synergizes with α CD40, supporting a strategy that combines DC activation with tumor-intrinsic immunogenic modulation to improve anti-tumor immunity in MM."

IO biomarker • Hematological Malignancies • Immune Modulation • Immunology • Multiple Myeloma • CD40 • FLT3

Functional Precision Medicine Using Ex Vivo Drug Sensitivity Profiling to Guide Therapy Selection in Relapsed/Refractory Multiple Myeloma: Results of a Prospective Clinical Trial (IMS 2026) - P=N/A | "Prior exposure included lenalidomide (100%), bortezomib (97.5%), carfilzomib (85%), daratumumab (82%), pomalidomide (77.5%), and BCMA CAR-T (12.5%). Top-ranked agents by DSS in v1 included bortezomib (median DSS 47.7), carfilzomib (47.3), panobinostat (47.0), and romidepsin (45.4). In v2, leading agents were marizomib ( 46.1), carfilzomib (40.2), ixazomib (37.2), and oprozomib (31.6)...ROC analysis for bortezomib (n=12) and selinexor (n=8) yielded AUCs of 0.471 and 0.462, respectively... High-throughput drug sensitivity testing was feasible in heavily pretreated RRMM and generated actionable results in nearly all patients. DSS-based prediction varied across agents, highlighting the need for drug-specific thresholds and larger validation cohorts. These findings support further development of functional precision medicine as a complementary tool to refine therapeutic decision-making in RRMM"

Late-breaking abstract • Preclinical • Hematological Malignancies • Leukemia • Multiple Myeloma • Plasma Cell Leukemia • Plasmacytoma • SDC1

ADAM17-Driven NKG2D Ligand Shedding Defines a Leukemia-Intrinsic Immune Evasion Program That Predicts Venetoclax Resistance and Reveals MEK and PI3K as Therapeutic Vulnerabilities in AML (SOHO 2026) - "Drug vulnerability mapping identified MEK inhibitors (trametinib, selumetinib), the HDAC inhibitor panobinostat, and PI3K/AKT/mTOR inhibitors (GDC-0941, MK-2206, and INK-128) as selectively active in NK-high AML (all FDR <0.001). An ADAM17-centered NK immune evasion score identifies venetoclax-resistant AML across two independent cohorts and associates with a survival disadvantage equivalent to an 88% increase in hazard per SD of score. This phenotype is consistent with BCL2 independence and engagement of compensatory MAPK and PI3K survival signaling, nominating MEK and PI3K inhibitors as biologically therapeutic alternatives for refractory patients. Prospective biomarker-stratified trials are required before clinical implementation of this scoring approach."

IO biomarker • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Lymphoma • Oncology • ADAM17 • BCL2 • HLA-E • NKG2D • SMAD3 • TGFB1 • TIMP3 • ULBP1 • ULBP2

Panobinostat represses glioma progression by inhibiting macrophage protumor polarization in hypoxic oxidative stress microenvironment (EANO 2026) - "Using multi-omics analysis, this study constructed and validated a novel prognostic model for glioma based on the hypoxia/oxidative stress-related immune microenvironment, identifying tumor-associated macrophages as a critical link between stress microenvironment and immunosuppression. Panobinostat was shown to modulate macrophage phenotypes by targeting this axis, offering a potential therapeutic target and experimental rationale for reversing immunosuppression and developing combination strategies against the glioma microenvironment."

Oxidative stress • Brain Cancer • Glioma • Oncology • Solid Tumor

Comprehensive Analyses of SOX7 Provide Novel Insights on Its Tumor Suppressor Role and Its Target Genes with Therapeutic Implications in Multiple Myeloma. (PubMed, Int J Mol Sci) - "Although SOX7 re-expression did not enhance bortezomib efficacy, treatment with the pan-histone deacetylase inhibitor panobinostat induced G1 arrest, promoted apoptosis, and increased SOX7 expression in MM.1S cells. Collectively, these results indicate that SOX7 functions as a tumor suppressor in MM, and its inactivation promotes cell cycle progression. The anti-myeloma effects of panobinostat in MM.1S cells may be partially mediated through SOX7 induction."

Journal • Hematological Disorders • Hematological Malignancies • Multiple Myeloma • Oncology

Safety and Efficacy of a New High-Dose Chemotherapy Regimen of Panobinostat, Gemcitabine, Busulfan and Melphalan (Pano/GBM) with Autologous Stem Cell Transplant (ASCT) for Patients with High-Risk or Refractory/Relapsed Myeloma – Matched Pair Comparisons with a Concurrent Control Cohort. (TCT-ASTCT-CIBMTR 2023) - P2 | "The benefit in ASCT-1 of study patients was also seen over those controls who received BuMel in PFS (P<0.01) and OS (P<0.01). CONCLUSIONS : Pano/GemBuMel is safe and effective in HR or R/R MM, with better outcomes after ASCT-1 than matched concurrent controls."

Clinical • Bone Marrow Transplantation • Hematological Malignancies • Leukemia • Mucositis • Multiple Myeloma • Oncology • Transplantation • SLC1A5

An intratumoural bioelectronic device for controlled, solid-state chemotherapy in paediatric high-grade glioma (EANO 2026) - "This work therefore investigates whether different drugs can be released from the device, and whether advanced stimulation paradigms can be used to control drug distribution for pHGG.Materials and Methods Devices were loaded with doxorubicin, topotecan, or panobinostat. Varying stimulation parameters improved control of drug release and distribution, opening the possibility for tailoring treatment to individual tumour characteristics. Next steps, including testing device efficacy in pHGG in vitro models followed by safety and survival studies in vivo, will establish whether this device represents a viable, precise delivery strategy for localised chemotherapy."

Brain Cancer • Glioma • High Grade Glioma • Oncology • Solid Tumor

Drug repurposing strategies targeting core pathways in glioblastoma to address heterogeneity (EANO 2026) - "Standard treatment includes surgical resection, radiation, and temozolomide chemotherapy; however, 80-90% of patients experience relapse...Of these, three were prioritized for further investigation based on their targeting of essential cellular pathways, in particular we selected: Homoharringtonine (a protein synthesis inhibitor), Ixazomib citrate (a proteasome inhibitor), and Panobinostat (a histone deacetylase inhibitor)... These findings demonstrate that targeting core cellular pathways in TICs is a feasible and clinically actionable strategy to counteract GBM heterogeneity and therapeutic resistance. This approach offers the potential for broadly effective treatments. Ongoing studies aim to expand the compound panel, investigate resistance mechanisms using single-cell transcriptomic and epigenomic profiling, and develop optimized combination therapies to improve outcomes for patients with GBM."

Heterogeneity • Brain Cancer • Glioblastoma • Oncology • Solid Tumor

BRAINTOX: uremic toxins in chemotherapy response in the brain (EANO 2026) - "Then, U87MG cells were co-treated with p-cresyl sulfate or indoxyl sulfate and ten selected drugs in a drug screening platform (drugs: Temozolamide, Dacomitinib, Selinexor, Panobinostat, Ixazomib, AMG232, Bortezomib, Lazertinib, Temsirolimus, Carmilzomib). The uremic toxins p-cresyl sulfate and indoxyl sulfate were enriched in brain tumor tissue samples of IDH wt patients compared to IDH mut patients, validating the findings of the discovery cohort. IDH wt patients have higher levels of uremic toxins in the circulation compared to patients with IDH mutant adult-type diffuse gliomas and the toxins reach the tumor site. Further experiments shall elucidate the mechanism by which uremic toxins reach the tumor cells and whether they modulate drug responsiveness."

Brain Cancer • Diffuse Glioma • Glioblastoma • Glioma • Oncology • Solid Tumor

Sequential multi-agent therapy eradicates tumours in preclinical models of Diffuse Midline Glioma (EANO 2026) - "Inspired by the curative "Total Therapy" approach in ALL, we hypothesised that a sequentially administered multi-agent regimen could achieve meaningful benefit in DMG by targeting key vulnerabilities: epigenetic/chromatin (CBL0137-Panobinostat, CP), polyamine metabolism (DFMO-AMXT1501, DA), and the NFκB pathway (ACT001). This rationally designed sequential multi-agent "Total Therapy" approach achieves profound tumour regression and long-term survival in aggressive DMG preclinical models. The strategy warrants further optimisation and clinical translation, offering new hope for children with this devastating disease."

Preclinical • Acute Lymphocytic Leukemia • Brain Cancer • Diffuse Midline Glioma • Glioma • Hematological Malignancies • Leukemia • Oncology • Solid Tumor

Dynamics of Tumor Progression in Glioblastoma Patients through Longitudinal Profiling (EANO 2026) - "Analysis of matched primary and recurrent samples from two IDH wildtype glioblastoma patients revealed highly similar drug sensitivity and resistance patterns, including sensitivity to Pexidartinib and Pazopanib and resistance to Panobinostat and Selinexor, suggesting convergent therapeutic response profiles. In a subset of patients, recurrent glioblastomas may undergo convergent adaptation, characterized by shared drug responses and metabolic changes that cannot be fully explained by genomic alterations, highlighting the role of non-genetic mechanisms. Longitudinal profiling can reveal the dynamics of therapy resistance and identify targetable vulnerabilities and will may further support personalized treatment strategies especially for recurrent tumors."

Clinical • Astrocytoma • Brain Cancer • Glioblastoma • Oncology • Solid Tumor

Randomized Treatments for Myelodysplastic Syndrome and Related Disorders: A Comprehensive Component Network Meta-Analysis of Randomized Controlled Trials (SOHO 2026) - "Compared with best supportive care, higher odds of complete remission were observed for azacitidine, azacitidine plus lenalidomide, azacitidine plus vorinostat, panobinostat plus azacitidine, APR-246 plus azacitidine, decitabine, decitabine plus valproic acid, and glasdegib plus cytarabine. This review combines broad trial identification, structured screening, detailed extraction, and treatment level synthesis across a complex therapeutic landscape. Its findings may support future guidelines, whereas the contrast between regimen- and component-level effects leaves key clinical questions for the full review to resolve."

Retrospective data • Hematological Malignancies • Myelodysplastic Syndrome • Oncology

Synergy in Dual Engagement of Extrinsic and Intrinsic Apoptosis Pathways by Bleomycin and Panobinostat in Hepatocellular Carcinoma and Targeting Mcl-1-Dependent Apoptosis Resistance. (PubMed, Biomedicines) - "In summary, the combination of panobinostat and bleomycin overcomes anti-apoptotic defenses in HepG2 cells through synergistic and coordinated disruption of mitochondrial survival checkpoints and dual apoptosis pathway activation. By blocking a compensatory Mcl-1 escape response while simultaneously engaging extrinsic apoptosis signaling, this strategy produces potent synergistic cell death in this defined p53-functional HCC model and represents a promising mechanistic proof of concept that warrants further validation in additional molecularly diverse HCC models before broader translational conclusions can be drawn."

Journal • B Cell Lymphoma • Hematological Malignancies • Hepatocellular Cancer • Leukemia • Lymphoma • Non-Hodgkin’s Lymphoma • Oncology • Solid Tumor • ANXA5 • BCL2L1 • CASP3 • CASP8 • CASP9 • MCL1

CINC424A2X01B Rollover Protocol (clinicaltrials.gov) - P4 | N=279 | Active, not recruiting | Sponsor: Novartis Pharmaceuticals | Trial primary completion date: Sep 2027 ➔ Apr 2032

Trial primary completion date • Acute Graft versus Host Disease • Acute Myelogenous Leukemia • Genetic Disorders • Graft versus Host Disease • Hematological Malignancies • Immunology • Leukemia • Myelofibrosis • Oncology • Polycythemia Vera

Identifying novel Ewing sarcoma therapeutics using a high-throughput chromatin accessibility assay (AACR 2025) - "The set of active compounds included Panobinostat, which is currently in preclinical development for Ewing sarcoma. This pilot screen supports the HT-ATAC-seq assay as a robust approach to identify novel compounds that reverse the chromatin alterations associated with Ewing sarcoma. This approach and assay can be adapted to investigate other cancers or pathologies associated with chromatin state alterations."

Ewing Sarcoma • Oncology • Osteosarcoma • Sarcoma • Solid Tumor • EWSR1 • FLI1

Rational design of soft liposomal drug carriers: Computational Insights into how lipid acyl chain Identity, and hydrophobic drug loading drive liposomal deformability | Poster Board #D16 (ACS-Fall 2026) - "We first looked at how three hydrophobic anticancer drugs, Panobinostat, Ki16425, and Vorinostat, changed the mechanics of DOPC bilayers. The softest mixtures had KA values that were 23% to 30% lower than those of pure DOPC. The stiffness hierarchy among PC lipids (DOPC > SOPC ≥ POPC) is the inverse of the chain order hierarchy (SOPC > POPC > DOPC), showing a disconnect between KA and SC."

Thio-hydroxamic acids as HDAC6 inhibitors | Poster Board #1454 (ACS-Fall 2026) - "Histone deacetylase (HDAC) inhibitors such as Vorinostat, Belinostat, and Panobinostat are well-known for their ability to coordinate with Zn ions in the enzyme active site through a hydroxamic acid functional group, thereby inhibiting HDAC activity. Preliminary findings indicate that electron-donating substituents enhance coordination ability, whereas electron-withdrawing groups diminish it. Additionally, this study examines the crystal structures and metal (Fe, Cu) coordination behavior of Vorinostat (SAHA), HPOB, and HPB to better understand structure activity relationships."

Cost-Effectiveness of Epigenetic Drugs in Oncology: A Systematic Review of Economic Evaluations. (PubMed, Value Health) - "This systematic review offers a comprehensive synthesis of current economic evaluations of epidrugs in oncology. The wide variability in ICERs and methodological approaches underscores the need for more standardized and robust evaluations to clarify the economic value of epigenetic drugs."

HEOR • Journal • Review • Hematological Disorders • Oncology

Rational design of soft liposomal drug carriers: Computational Insights into how lipid acyl chain Identity, and hydrophobic drug loading drive liposomal deformability | Poster Board #D16 (ACS-Fall 2026) - "We first looked at how three hydrophobic anticancer drugs, Panobinostat, Ki16425, and Vorinostat, changed the mechanics of DOPC bilayers. The softest mixtures had KA values that were 23% to 30% lower than those of pure DOPC. The stiffness hierarchy among PC lipids (DOPC > SOPC ≥ POPC) is the inverse of the chain order hierarchy (SOPC > POPC > DOPC), showing a disconnect between KA and SC."

Single-Dose TACE-Like Injection of Sustained-Release Panobinostat/IL-12 Polymeric Microparticles is Effective in Preclinical Liver Cancer Models. (PubMed, Adv Sci (Weinh)) - "A PLGA/PBAE sustained-release microparticle platform was successfully developed for TACE-like co-delivery of panobinostat and IL-12. This combined chemoimmunotherapy strategy shows promise for overcoming immune suppression and improving liver cancer treatment."

Journal • Preclinical • Hepatocellular Cancer • Liver Cancer • Oncology • Solid Tumor • IL12A

Synergistic cell-cycle repression by HDAC/EZH2 co-targeting in H3K27-altered diffuse midline glioma. (PubMed, Mol Ther Oncol) - "Panobinostat and tazemetostat showed robust synergy across patient-derived DMG GSC cultures, suppressing GSC growth, proliferation, and self-renewal while inducing apoptosis and G0/G1 cell-cycle arrest. Together, these findings indicate that dual HDAC/EZH2 inhibition disrupts epigenetically maintained proliferative and stem-like programs through coordinated cell-cycle repression, apoptosis induction, and impaired self-renewal. This work supports HDAC/EZH2 co-targeting as a rational therapeutic backbone for H3K27-altered DMG and its incorporation into future rational combination strategies."

Journal • Brain Cancer • Diffuse Midline Glioma • Gene Therapies • Glioma • Oncology • Solid Tumor

Comprehensive Pan-Cancer Analysis of ABCA1: Insights From Multi-Omics Data and Exploratory Validation in Esophageal Squamous Cell Carcinoma. (PubMed, Cell Biochem Funct) - "In silico drug sensitivity analysis suggested potential associations with dasatinib response and panobinostat resistance, warranting further experimental validation. Collectively, these findings highlight the context-dependent associations of ABCA1 with cancer progression, immune modulation, and genomic integrity, suggesting its potential utility as a prognostic biomarker that warrants further investigation for therapeutic targeting."

Biomarker • Journal • Tumor mutational burden • Esophageal Squamous Cell Carcinoma • Immune Modulation • Immunology • Oncology • Squamous Cell Carcinoma • ABCA1

From design to clinic: Medicinal chemistry and pharmacology of approved epigenetic drugs. (PubMed, Eur J Med Chem) - "This review explores the ten epigenetic drugs that have attained worldwide regulatory approval for human therapy: the DNA methyltransferase inhibitors azacitidine (2004) and decitabine (2006), the histone deacetylase inhibitors vorinostat (2006), romidepsin (2009), belinostat (2014), panobinostat (2015) tucidinostat (2015) and givinostat (2024), and the histone methyltransferase inhibitors tazemetostat (2020) and valemetostat (2022). The history and strategy of their discovery and development, their biological targets and mechanisms of action and their therapeutic use. In addition, current advancements and efforts, as well as future perspectives in the design and clinical approval of new epigenetic drugs are discussed."

Journal • Review • Oncology

A Simulation-Based Assessment of Dosage Regimen Appropriateness for Multiple Myeloma Medicines Reflecting Demographic and Ethnic Differences. (PubMed, Clin Transl Sci) - "Simulated carfilzomib exposure was approximately 12%-15% lower in KP than in TP across two dosing regimens, corresponding to an estimated 4%-9% lower predicted overall response rate based on the published exposure-response data. Conversely, simulated lenalidomide exposure was approximately 25% higher in KP, corresponding to an approximately 1.27-fold higher estimated probability of grade ≥ 3 hematologic adverse events. No meaningful exposure differences were observed for daratumumab, melphalan, or panobinostat. These findings suggest that demographic and regional differences between TP and KP may translate into measurable differences in predicted drug exposure for selected multiple myeloma therapies. Simulation-based approaches integrating real-world demographic data may help prioritize drugs or indications that warrant further pharmacokinetic evaluation or focused bridging studies in Korean or broader East Asian populations."

Journal • Hematological Disorders • Hematological Malignancies • Multiple Myeloma • Oncology