Synribo (omacetaxine mepesuccinate) / Teva, ArchiMed - LARVOL DELTA

Amplified MYCN, GLI2 and mutant TP53 medulloblastoma are vulnerable to the translation inhibitor homoharringtonine (AACR 2026) - "Abstract is embargoed at this time."

Late-breaking abstract • Brain Cancer • Medulloblastoma • Oncology • Solid Tumor • GLI2 • MYCN • TP53

THE EFFECT OF ALLOGENEIC HSCT ON PEDIATRIC NON–DOWN SYNDROME AMKL: RESULTS FROM A MULTICENTER CALD-AMKL-18 TRIAL (EBMT 2026) - P1/2 | " CALD-AMKL-18 is a prospective multicenter trial using a low-dose HAG induction backbone (low-dose of homoharringtonine and cytarabine, routine dose of G-CSF) for newly diagnosed pediatric non-DS AMKL at 9 Chinese centers. CR1 HSCT appeared to improve survival for some adverse genotypes. These data support a risk-adapted HSCT strategy built on a low-toxicity HAG backbone, but also highlight the need for "low-dose chemotherapy (LDC) + X" approaches that integrate targeted agents and MRD-guided HSCT to overcome fusion-driven resistance in pediatric non-DS AMKL."

Clinical • Bone Marrow Transplantation • Developmental Disorders • Genetic Disorders • Hematological Malignancies • Infectious Disease • Leukemia • Pediatrics • CBFA2T3 • CCDC6 • CSF1R • ETV6 • GATA1 • GLIS2 • KDM5A • KMT2A • MLLT10 • MLLT3 • MRTFA • NUP98 • PRKAR1A • RBM15 • RUNX1 • ZEB2

THE EFFECT OF ALLOGENEIC HSCT ON PEDIATRIC NON–DOWN SYNDROME AMKL: RESULTS FROM A MULTICENTER CALD-AMKL-18 TRIAL (EBMT 2026) - P1/2 | " CALD-AMKL-18 is a prospective multicenter trial using a low-dose HAG induction backbone (low-dose of homoharringtonine and cytarabine, routine dose of G-CSF) for newly diagnosed pediatric non-DS AMKL at 9 Chinese centers. CR1 HSCT appeared to improve survival for some adverse genotypes. These data support a risk-adapted HSCT strategy built on a low-toxicity HAG backbone, but also highlight the need for "low-dose chemotherapy (LDC) + X" approaches that integrate targeted agents and MRD-guided HSCT to overcome fusion-driven resistance in pediatric non-DS AMKL."

Clinical • Bone Marrow Transplantation • Developmental Disorders • Genetic Disorders • Hematological Malignancies • Infectious Disease • Leukemia • Pediatrics • CBFA2T3 • CCDC6 • CSF1R • ETV6 • GATA1 • GLIS2 • KDM5A • KMT2A • MLLT10 • MLLT3 • MRTFA • NUP98 • PRKAR1A • RBM15 • RUNX1 • ZEB2

Potent and selective LSD1 inhibitor DC551040 reveals a promising combination therapy for AML with insight into epigenetic dysregulation. (PubMed, Signal Transduct Target Ther) - "Through a search of the Connectivity Map (CMAP) database, we identify homoharringtonine (HHT), an approved anti-leukemia drug, which mimics the anti-transcriptional activation of inflammatory pathways. Subsequent in vitro and in vivo experiments validated the efficacy of combining HHT with DC551040, demonstrating a synergistic antitumor effect and extended survival in MV-4-11 disseminated xenograft model mice. Together, this study not only introduces a novel LSD1 inhibitor but also delves into the molecular mechanisms underlying LSD1 inhibitors, while proposing a promising combination therapy for AML individuals in clinical trials."

Journal • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology • STAT5

NETWORK-BASED PROTEOMIC SIGNATURES REVEAL DIAGNOSTIC BIOMARKERS AND REPURPOSABLE THERAPEUTICS IN PARKINSON'S DISEASE (ADPD 2026) - "Drug perturbation mapping identified SAL-1, fostamatinib, homoharringtonine, and 2-aminopurine as candidates capable of reversing PD-associated proteomic signatures. Our findings demonstrate that proteomics-based models outperform RNA-based and multimodal approaches in classifying PD and tracking disease severity. Our findings demonstrate that proteomics-based models outperform RNA-based and multimodal approaches in classifying PD and tracking disease severity. The identified biomarkers are functionally enriched in extracellular and immune-related processes, offering insight into PD pathophysiology. Network-informed perturbation analysis further nominates candidate therapeutics for experimental validation."

Biomarker • CNS Disorders • Movement Disorders • Parkinson's Disease

Observation of the Therapeutic Effect of Venetoclax Combined with HEA Regimen on Acute Myeloid Leukemia Patients with KMT2A Gene Rearrangement (PubMed, Zhongguo Shi Yan Xue Ye Xue Za Zhi) - "The VHEA regimen can significantly improve the CR rate of AML patients with KMT2A gene rearrangement, with good safety. It may be a better choice for induction remission chemotherapy in newly diagnosed and relapsed/refractory AML patients with KMT2A gene rearrangement."

Journal • Acute Myelogenous Leukemia • Bone Marrow Transplantation • Hematological Malignancies • Infectious Disease • Leukemia • Oncology • Respiratory Diseases • Transplantation • KMT2A

VEN-OM: Safety and Efficacy of Venetoclax With Escalating Doses of Omacetaxine in Patients With Acute Myeloid Leukemia Safety and Efficacy of Venetoclax With Escalating Doses of Omacetaxine in Patients With Acute Myeloid Leukemia (AML) (clinicaltrials.gov) - P1 | N=6 | Completed | Sponsor: University of Illinois at Chicago | Active, not recruiting ➔ Completed

Trial completion • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology

Efficacy and safety of venetoclax-cytarabine-homoharringtonine-based cytoreductive therapy before allogeneic hematopoietic stem cell transplantation in refractory/relapsed acute myeloid leukemia with RUNX1::RUNX1T1: a retrospective study (PubMed, Zhonghua Xue Ye Xue Za Zhi) - "All patients remained disease-free, with no events of measurable residual disease (MRD) positivity by flow cytometry or molecular analysis documented. These findings preliminarily confirm that venetoclax, cytarabine, and homoharringtonine-based cytoreductive therapy is a safe and effective bridging therapy for allo-HSCT in patients with refractory/relapsed AML and RUNX1::RUNX1T1 fusion gene."

Journal • Retrospective data • Acute Myelogenous Leukemia • Bone Marrow Transplantation • Hematological Malignancies • Leukemia • Oncology • Transplantation • RUNX1 • RUNX1T1

Homoharringtonine inhibits growth and migration in non-small cell lung cancer via PDIA4-mediated modulation of autophagy and EMT. (PubMed, Arch Pharm Res) - "Additionally, HHT inhibits NSCLC migration by modulating epithelial-mesenchymal transition (EMT) signaling. These findings highlight PDIA4 as a critical mediator of HHT efficacy against NSCLC, provide novel insights into PDIA4-associated therapy, and support the translational potential of HHT in NSCLC treatment."

Journal • Lung Cancer • Non Small Cell Lung Cancer • Oncology • Solid Tumor • PDIA4

VAH vs VA in Newly Diagnosed Elderly AML (clinicaltrials.gov) - P3 | N=308 | Not yet recruiting | Sponsor: Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine

New P3 trial • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology

Targeting dengue virus infection through a translation-modulator small-molecule screen. (PubMed, Antiviral Res) - "Two clinically relevant inhibitors, homoharringtonine (HHT) and bruceantin (BCT), blocked DENV propagation at low nanomolar doses in cell lines and reduced viral RNA and titers in primary human macrophages, underscoring their translational potential. Similar effects were observed with the related Zika virus (ZIKV) in cell lines. These pharmacological data suggest that translation elongation and eIF4A helicase activity are candidate host dependencies for Orthoflaviviruses, motivating stage-resolved and in vivo evaluation studies."

Journal • Dengue Fever • Infectious Disease • EIF4A1 • EIF4A2 • EIF4E • EIF4G1

New Treatments to Rabies Virus Infections. (PubMed, Antiviral Res) - "Among them, homoharringtonine might be a good candidate, although further clinical trials are needed. Artificial intelligence (AI) assisted drug design against RABV has also been prospected."

Journal • Review • Infectious Disease

Homoharringtonine and Gilteritinib Synergistically Induce Apoptosis and Suppress Viability in FLT3-ITD-Positive AML Cells. (PubMed, Biomedicines) - "The combination enhanced the p53 expression. Our findings elucidate the mechanism underlying this synergistic interaction and underscore the potential of p53 status as a predictive biomarker for identifying patients most likely to benefit from HHT and gilteritinib combination therapy."

Journal • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology • FLT3 • HSPA8

A conserved eIF1A+ luminal cell-centered hypoxic and "cold" tumor microenvironment promotes pan-subtype prostate cancer progression. (PubMed, Cell Rep Med) - P2 | "In luminal cells, EIF1A knockdown and the translation inhibitor homoharringtonine (HHT) both suppress HIF-1α translation and tumor growth, while promoting infiltration of anticancer immune cells including PD-1- T cells and CD163- macrophages...Collectively, this work defines conserved molecular features across PCa subtypes, providing promising insights for clinical management. This study was registered at Clinicaltrials.gov (NCT06834321)."

Biomarker • Journal • Genito-urinary Cancer • Oncology • Prostate Cancer • Solid Tumor • CD163 • EIF1AX • HIF1A • PD-1

Clinical efficacy analysis of seven pediatric patients with Acute myeloid leukemia and the t(16;21)(p11;q22) FUS::ERG fusion gene (PubMed, Zhonghua Yi Xue Yi Chuan Xue Za Zhi) - "The t(16;21)(p11;q22) FUS::ERG fusion gene is rare in pediatric AML and associated with poor prognosis. Allo-HSCT may mitigate the adverse prognostic impact of the FUS::ERG fusion gene and contribute to prolonged survival."

IO biomarker • Journal • Retrospective data • Acute Myelogenous Leukemia • Bone Marrow Transplantation • Hematological Malignancies • Leukemia • Oncology • Pediatrics • Transplantation • ANPEP • CD123 • CD33 • CD34 • CD38 • ERG • FUS • IL3RA • ITGAM • KIT • NCAM1

Olverembatinib (HQP1351) Demonstrates Efficacy Vs. Best Available Therapy (BAT) in Patients (Pts) with Tyrosine Kinase Inhibitor (TKI)-Resistant Chronic Myeloid Leukemia Chronic-Phase (CML-CP) in a Registrational Randomized Phase 2 Study (ASH 2023) - P2 | "Introduction This was a multicenter, randomized, registrational phase 2 study to assess the efficacy and safety of olverembatinib compared with BAT in pts with CML-CP who were resistant and/or intolerant to 3 TKIs (imatinib [I], dasatinib [D], nilotinib [N]) in China...Pts were randomized 2:1 to investigational olverembatinib (40 mg QOD) or the BAT arm, which could be one of the following per investigator choice: TKIs (I, D, or N), interferon (IFN), hydroxyurea (HU), and homoharringtonine (HHT)...Olverembatinib was observed to be better tolerated and more effective than BAT in treating these pts. Internal study (CT.gov) numbers: HQP1351CC203 (NCT04126681)."

Clinical • P2 data • Anemia • Cardiovascular • Chronic Myeloid Leukemia • CNS Disorders • Congestive Heart Failure • Coronary Artery Disease • Dyslipidemia • Heart Failure • Hematological Disorders • Hematological Malignancies • Hypertriglyceridemia • Leukemia • Leukopenia • Myocardial Infarction • Neutropenia • Oncology • Thrombocytopenia • ABL1

Targeting Hippo-YAP/TAZ signaling pathway: an updated review demonstrating the therapeutic potential of key plant derived anticancer compounds. (PubMed, Front Pharmacol) - "The upregulation of YAP/TAZ/TEAD complex has been demonstrated to result in cellular proliferation, transformation, and ultimately, carcinogenesis. This article offers extensive insight into plant derived anticancer compounds mainly apigenin, curcumin, EGCG, resveratrol, homoharringtonine, and ursolic acid of the Hippo pathway, specifically YAP/TAZ, in several cancer therapies. This will enhance the discovery of novel Hippo inhibitors and the optimal therapeutic application of Hippo signaling-related pharmaceuticals in synergistic cancer therapies."

Journal • Review • Oncology

Screening of FDA-approved drugs using a recombinant Cedar virus to improve treatment options for Nipah virus infection. (PubMed, J Gen Virol) - "Using recombinant Cedar virus expressing luciferase (rCedV-Luc) as a CL2 surrogate, we screened a library of 2,703 Food and Drug Administration (FDA)-approved compounds, yielding 5 promising candidates: bortezomib, harringtonine, homoharringtonine, ixazomib citrate and lanatoside C. Compounds demonstrated low half-maximal inhibitory concentration (IC50) values of ≤0.45 µM and high selectivity indexes >6 in mammalian cell lines. Time-of-addition studies suggest that these compounds target a post-entry stage of henipavirus replication. This study demonstrates the utility of rCedV-Luc as a surrogate for the antiviral screening of henipaviruses and identification of promising candidates for further investigation and development as henipavirus antivirals."

FDA event • Journal • Preclinical • Infectious Disease

SRSF2 mutations drive daunorubicin resistance in acute myeloid leukemia via THBS1 stabilization. (PubMed, J Exp Clin Cancer Res) - "SRSF2 mutations promoted DNR resistance through multiple mechanisms, and targeted combination therapy with PDGFB pathway inhibitors may represent a novel strategy to improve therapeutic outcomes in patients with mutations."

Journal • Acute Myelogenous Leukemia • Hematological Disorders • Hematological Malignancies • Leukemia • Oncology • PDGFB • SRSF2 • THBS1

Omacetaxine and venetoclax in relapsed/refractory acute myeloid leukemia or myelodysplastic syndrome with mutant RUNX1. (PubMed, Blood Neoplasia) - P1/2 | "Clinical responses were seen exclusively in patients with MDS, which suggests that dose optimization or combination with cytoreductive agents may be necessary for eliciting clinical activity in AML. This trial was registered at www.ClinicalTrials.gov as #NCT04874194."

Journal • Acute Myelogenous Leukemia • Hematological Malignancies • Infectious Disease • Leukemia • Myelodysplastic Syndrome • Oncology • Transplantation • BCL2L1 • MCL1 • RUNX1

Cladribine Plus Homoharringtonine and Cytarabine Regimen (CHA) for de Novo Acute Myeloid Leukemia (clinicaltrials.gov) - P2 | N=65 | Completed | Sponsor: First Affiliated Hospital of Zhejiang University | Recruiting ➔ Completed | N=30 ➔ 65 | Trial primary completion date: Aug 2025 ➔ Apr 2025

Enrollment change • Trial completion • Trial primary completion date • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology

Venetoclax Combined with Hag Regimen in Newly Diagnosed ETP-ALL: A Prospective, Multicenter, Phase 2 Trial (ASH 2024) - "The V-HAG regimen included venetoclax (100 mg on day 1, 200 mg on day 2, and 400 mg on days 3-14; if the blast cells in the bone marrow (BM) were more than 5% on day 14, the patient continued to receive venetoclax 400 mg until day 28), homoharringtonine (1.4 mg/m², maximum 2 mg daily, intravenously from days 1-10), low-dose cytarabine (10 mg/m² subcutaneously every 12 hours on days 1-14), and G-CSF (200 µg/m² daily on days 1-14 if the WBC <10*109/L). Conclusions : Venetoclax Combined with HAG regimen achieved an 100% CRc rate in newly diagnosed ETP-ALL. This therapy is a promising and well-tolerated regimen in newly diagnosed ETP-ALL patients."

Clinical • P2 data • Acute Lymphocytic Leukemia • Anemia • Basal Cell Carcinoma • Bone Marrow Transplantation • Chronic Obstructive Pulmonary Disease • CNS Disorders • Febrile Neutropenia • Hematological Disorders • Hematological Malignancies • Hepatology • Immunology • Infectious Disease • Leukemia • Melanoma • Neutropenia • Non-melanoma Skin Cancer • Oncology • Respiratory Diseases • Solid Tumor • T Acute Lymphoblastic Leukemia • T-cell Acute Lymphoblastic Lymphoma • Thrombocytopenia • Thyroid Gland Carcinoma • Tuberculosis

Therapeutic Effects of Cephalotaxus harringtonia Leaf Extract on Hepatocellular Carcinoma via Regulation of the Intrinsic Apoptosis Pathway and Cell Cycle. (PubMed, Curr Issues Mol Biol) - "Cephalotaxus harringtonia, traditionally used in East Asian medicine, contains several bioactive constituents, including homoharringtonine (HHT) and quercetin 3-β-D-glucoside (Q3G), which are known for their anticancer properties...Mechanistic analyses revealed that CHLE induced apoptosis through a mitochondria-mediated intrinsic pathway, characterized by increased reactive oxygen species production, mitochondrial membrane depolarization, and BAX upregulation. These findings demonstrate that C. harringtonia leaf extract possesses potent, selective anticancer activity and may serve as a promising natural candidate for the prevention and therapeutic management of liver cancer."

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

Efficacy and safety of sequential therapy with Venetoclax, Azacitidine, and Homoharringtonine in elderly patients with de novo AML: A prospective, single-arm, multicenter, exploratory clinical trial (ChiCTR) - P=N/A | N=40 | Not yet recruiting | Sponsor: the First Affiliated Hospital, Zhejiang University School of Medicine; The People's Hospital Affiliated to Ningbo University

New trial • Acute Myelogenous Leukemia

Discovery of novel and potent harringtonine derivative P2 via systematic structure-activity Optimization: Semi-Synthesis, anti-leukemia activity, and mechanism study. (PubMed, Eur J Med Chem) - "P2 exhibited a ∼10-fold increase in antiproliferative potency against human leukemic cell lines compared to homoharringtonine (HHT)...Our findings provide valuable insights to guide the future structural optimization of harringtonine derivatives. Furthermore, P2 has been identified as a promising anti-leukemic candidate and warrants further development."

Journal • Hematological Disorders • Hematological Malignancies • Leukemia • Oncology • MCL1 • MYC