Publications & Meeting Presentations

Access Scientific References for Our Pipeline Below

• Revumenib / Menin-KMT2A Inhibitions

  Chapron A, et al. (2024, Nov). “Revumenib Physiologically Based Pharmacokinetic (PBPK) Model for Evaluation of Age Effect and CYP3A4-mediated Drug-Drug Interaction (DDI) in Relapsed /Refractory Acute Leukemias.” Poster presented at the 2024 American Conference on Pharmacometrics (ACoP), Phoenix, AZ.

  Lin Y-W, et al. (2024, Nov). “Population Pharmacokinetics (PopPK) of Revumenib in Patients with Relapsed/Refractory Acute Leukemias.” Poster presented at the 2024 American Conference on Pharmacometrics (ACoP), Phoenix, AZ.

  Papayannidis C, et al. (2024, Sept). “Revumenib Monotherapy in Patients with Relapsed/Refractory KMT2Ar Acute Leukemia: Topline Efficacy and Safety Results from the Pivotal AUGMENT-101 Phase 2 Study.” Oral presented at the Italian Society of Hematology (SIE) National Congress, Milan, Italy

  Lin YW, et al. (2024, Sept). “Pharmacokinetics and Exposure-Response Analyses of Revumenib Support Dosing in a Broad Population of Patients with Relapsed/Refractory Acute Leukemias.” Poster presented at the American College of Clinical Pharmacology annual meeting, Bethesda, MD.

  Issa, G.C., et al. (2024, August). “Menin Inhibition With Revumenib for KMT2A-Rearranged Relapsed or Refractory Acute Leukemia (AUGMENT-101).”Journal of Clinical Oncology.e-pub_JCO.24.00826

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  Zeidner, J., et al. (2024, June). “Phase 1b Study of Azacitidine, Venetoclax and Revumenib in Newly Diagnosed Older Adults with NPM1 Mutated or KMT2A Rearranged AML: Interim Results of Dose Escalation from the Beat AML Consortium.” Oral presented at the 2024 European Hematology Association Annual Meeting, Madrid, Spain.

  Aldoss, I., et al. (2024, June). “Revumenib Monotherapy in Patients with Relapsed/Refractory KMT2Ar Acute Leukemia: Topline Efficacy and Safety Results from the Pivotal AUGMENT-101 Phase 2 Study.” Oral presented at the 2024 European Hematology Association Annual Meeting, Madrid, Spain.

  Shukla, N., et al. (2024, June). “Safety and activity of revumenib in combination with fludarabine/cytarabine (FLA) in patients with relapsed/refractory acute leukemias.” Poster presented at the 2024 European Hematology Association Annual Meeting, Madrid, Spain.

  Aldoss, I., et al. (2024, April). “Revumenib Monotherapy in Patients With Relapsed/Refractory KMT2Ar Acute Leukemia: Efficacy and Safety Results From the AUGMENT-101 Phase 1/2 Study.” Poster presented at the 2024 Academy of Managed Care Pharmacy Annual Meeting, New Orleans, LA.

  Zwaan, C.M. et al. (2024, April). “Pivotal Phase 2 Results of AUGMENT-101 for Revumenib in KMT2Ar Acute Leukemia: Pediatric Experience.” Slides presented at the 2024 American Society of Pediatric Hematology/Oncology (ASPHO) Conference, Seattle, WA.

  Aldoss, I., et al. (2023, December). “Revumenib monotherapy in patients with relapsed/refractory KMT2Ar acute leukemia: topline efficacy and safety results from the pivotal AUGMENT-101 Phase 2 study.” Slides presented at the 2023 American Society of Hematology Annual Meeting, San Diego, CA.

  Aldoss, I., et al. (2023, December). “Revumenib monotherapy in patients with relapsed/refractory KMT2Ar acute leukemia: efficacy and safety results from the AUGMENT-101 phase 1/2 study.” Poster presented at the 2023 American Society of Hematology Annual Meeting, San Diego, CA.

  Zucenka, A., et al. (2023, December). “Revumenib maintenance therapy following revumenib-induced remission and transplant.” Poster presented at the 2023 American Society of Hematology Annual Meeting, San Diego, CA.

  Rubnitz, J., et al. (2023, June) “Revumenib in patients with acute leukemias: compassionate use program experience.” Poster presented at the 2023 European Hematology Association Congress, Frankfurt, Germany.

  Cuglievan, B., et al. (2023, May) “Menin Inhibition in Pediatric KMT2Ar Leukemia: Phase 1 and Expanded Access Experience With Revumenib.” Poster presented at the 2023 American Society of Pediatric Hematology/Oncology Conference, Fort Worth, TX.

  Perner, F., et al. (2023, April). “Characterization of Acquired Resistance Mutations to Menin Inhibitors.” Slides presented at the American Association for Cancer Research Annual Meeting, Orlando, FL.

  Issa, G.C., Aldoss, I., DiPersio, J. et al. (2023, March) “The menin inhibitor revumenib in KMT2A-rearranged or NPM1-mutant leukaemia.” Nature. 615, 920–924.

  Perner, F., Stein, E.M., Wenge, D.V. et al. (2023, March) “MEN1 mutations mediate clinical resistance to menin inhibition”. Nature 615, 913–919.

  Issa, G., et al. (2022, December). “Outcomes After Transplant in Relapsed/Refractory KMT2Ar (MLLr) and mNPM1 (NPM1c) Leukemia Patients Achieving Remissions After Menin Inhibition: Revumenib (SNDX-5613) Ph1 Experience.” Slides presented at the American Society of Hematology Annual Meeting, New Orleans, LA.

  Issa, G., et al. (2022, December). “The Menin Inhibitor Revumenib (SNDX-5613) Leads to Durable Responses in Patients with KMT2A-Rearranged or NPM1 Mutant AML: Updated Results of a Phase 1 Study.” Slides presented at the American Society of Hematology Annual Meeting, New Orleans, LA.

  Stein, E., I. Aldoss, et al. (2021, December). “Safety and Efficacy of Menin Inhibition in Patients with MLL-Rearranged and NPM1 Mutant Acute Leukemia: A Phase 1, First-in-Human Study of SNDX-5613.” Slides presented at the American Society of Hematology Annual Meeting, Atlanta, GA.

  McGeehan G., (2020, April). “A first-in-class Menin-MLL1 antagonist for the treatment of MLL-r and NPM1 mutant leukemias.” Slides presented at the 2020 American Association for Cancer Research Virtual Annual Meeting I.

  Uckelmann, H., S. M. Kim et al. (2020, January) “Therapeutic targeting of preleukemia cells in a mouse model of NPM1 mutant acute myeloid leukemia.” Science. 367(6477):586-590.

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  Krivstov, A., (2019, December) “A Menin-MLL Inhibitor Induces Specific Chromatin Changes and Eradicates Disease in Models of of MLL-Rearranged Leukemia.”Cancer Cell. 36(6), 660–673.

  McGeehan, G., (2019, January) “Targeting the Menin MLL1 Interaction Site as a Treatment for Mixed Lineage Leukemia rearranged (MLL r) and NPM1c+ AML.” Slides presented at Clinical Translation of Epigenetics in Cancer Therapy Conference, Litchfield Park, AZ.

  Armstrong S. (2018). “VTP50469 is a novel, orally available menin-MLL1 inhibitor effective against MLL-rearranged and NPM1c⁺ leukemia.” Slides presented at European School of Hematology Conference, Stockholm, Sweden.

  Armstrong S. (2018, December). “Targeting Epigenetic Mechanisms to Reverse Stem Cell Programs in Cancer.” Slides presented at the American Society of Hematology Annual Meeting, San Diego, CA.

  Uckelmann, H. (2018, December). “MLL-Menin inhibition reverses pre-leukemic progenitor self-renewal induced by NPM1 mutations and prevents AML development.” Slides presented at the American Society of Hematology Annual Meeting, San Diego, CA.

  McGeehan, G. (2018, July). “Targeting the Menin-MLL1 Interaction Site as a Treatment for Mixed Lineage Leukemia-rearranged (MLL-r) and NPM1c+ AML.” Slides presented at the Molecular Therapeutics of Cancer Research Conference, Sundance, UT.

  Krivtsov A. V., J. Y. Gadrey, et al. (2018, April). “VTP50469 is a novel, orally available menin-MLL1 inhibitor effective against MLL-rearranged and NPM1c⁺ leukemia.” Slides presented at the American Association for Cancer Research Annual Meeting, Chicago, IL.

  Lock R. B., K. Evans, et al. (2018, April). “Pediatric Preclinical Testing Consortium evaluation of the menin inhibitor, VTP-50469, against xenograft models of MLL-rearranged infant acute lymphoblastic leukemia.” Poster presented at the American Association for Cancer Research Annual Meeting, Chicago, IL.

• Axatilimab / Targeting CSF-1R

  Galvin J, et al. (2024, Oct). “Evaluation of Clinical Trial Efficacy Endpoints in Chronic Graft-Versus-Host Disease.” Poster presented at the International Chronic Graft-versus-Host Disease (cGVHD) Symposium, Vancouver, BC, Canada.

  Yang Y, et al. (2024, Oct). “Exposure-Response Relationships for Axatilimab, a Humanized Monoclonal Antibody Targeting CSF-1R, in Patients With Chronic Graft-Versus-Host Disease.” Poster presented at the International Chronic Graft-versus-Host Disease (cGVHD) Symposium, Vancouver, BC, Canada.

  Wolff, D., et al. (2024, September). “Axatilimab in Recurrent or Refractory Chronic Graft-Versus-Host Disease.” New England Journal of Medicine. 391(11):1002-1014.

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  Salhotra A., et al. (2024, Sept). “Efficacy and Safety of Axatilimab at 3 Different Doses in Patients with Chronic Graft-Versus-Host Disease (cGVHD) and Related Bronchiolitis Obliterans Syndrome (BOS): Results of a Pivotal Phase 2 Study.” Poster presented at the European Respiratory Society Congress, Vienna, Austria.

  Sica, S., et al. (2024, June). “Axatilimab for Chronic Graft-Versus-Host Disease: Responses in Fibrosis-Dominant Organs in AGAVE-201.” Poster presented at the 2024 European Hematology Association Annual Meeting, Madrid, Spain.

  DeFilipp, Z., et al. (2024, February). “Safety and Efficacy of Axatilimab in Patients With Chronic Graft-Versus-Host Disease (AGAVE-201).” Slides presented at the 2024 Tandem Meeting (American Society for Transplantation and Cellular Therapy [ASTCT]-Center for International Blood and Marrow Transplant Research [CIBMTR]), San Antonio, TX, USA.

  Perez-Simon, J, et al. (2024, April). “Axatilimab for Chronic Graft-Versus-Host Disease: Responses in Fibrosis-Dominant Organs in AGAVE-201.” Slides presented at the 2024 European Society for Blood and Marrow Transplantation (EBMT) Annual Meeting, Glasgow, Scotland.

  White, J., et al. “Axatilimab for chronic graft-versus-host disease: responses in fibrosis-dominant organs in AGAVE-201.” Slides presented at the 2024 Cell Therapy Transplant Canada (CTTC) Annual Conference, Victoria, BC, Canada.

  Wolff, D., et al. (2023, December). “Safety and Efficacy of Axatilimab at 3 Different Doses in Patients with Chronic Graft-Versus-Host Disease (AGAVE-201).” Slides presented at the 2023 American Society of Hematology Annual Meeting, San Diego, CA.  

  Radojcic, V., et al.(2023, May) “Safety, Tolerability, and Efficacy of Axatilimab, a Colony-Stimulating Factor 1 Receptor (CSF-1R) Humanized Antibody, in Advanced Chronic Graft-versus-host Disease-Related Bronchiolitis Obliterans Syndrome.” Slides and poster presented at the American Thoracic Society 2023, Washington, DC

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  • Click here to view the presentation.

  Kitko, C. L., M. Arora, et. al. (2022, December). “Axatilimab for Chronic Graft-Versus-Host Disease After Failure of at Least Two Prior Systemic Therapies: Results of a Phase I/II Study.” J Clin Oncol. DOI: 10.1200/JCO.22.00958

  Lee, S. J., M. Arora, et al. (2021, December). “Safety, Tolerability and Efficacy of Axatilimab, a CSF-1R humanized antibody, for Chronic Graft-versus-Host Disease after 2 or more Lines of Systemic Treatment.” Slides presented at the American Society of Hematology Annual Meeting, Atlanta, GA.

  Arora M., M. Jagasia, et al. (2020, December). “Phase 1 Study of Axatilimab (SNDX-6352), a CSF-1R Humanized Antibody, For Chronic Graft-Versus-Host Disease after 2 or More Lines of Systemic Treatment.” Slides presented at the American Society of Hematology Virtual Annual Meeting.

  Azad N., D. Rasco, et al. (2020, April). “SNDX-6352-0502 – A phase 1, open-label, dose escalation trial to investigate the safety, tolerability, pharmacokinetics and pharmacodynamic activity of axatilimab (SNDX-6352) monotherapy in patients with unresectable, recurrent, locally-advanced, or metastatic solid tumors.” Slides presented at the 2020 American Association for Cancer Research Virtual Annual Meeting I.

  Tolcher A. W., D. Rasco, et al. (2020, April). “SNDX-6352-0502: A phase 1, open-label, dose escalation trial to investigate the safety, tolerability, pharmacokinetics and pharmacodynamic activity of SNDX-6352 in combination with durvalumab in patients with unresectable, recurrent, locally-advanced, or metastatic solid tumors.” Slides presented at the 2020 American Association for Cancer Research Virtual Annual Meeting I.

  Tiessen R., A. Visser, et al. (2017, November). “First in human, single ascending dose study in healthy volunteers of SNDX-6352, a humanized IgG4 monoclonal antibody targeting colony stimulating factor-1 receptor (CSF-1R).” Poster presented at the Society for the Immunotherapy of Cancer Annual Meeting, National Harbor, MD.

  Ordentlich P., P. Vajjah, et. al. (2016, November). “Targeting colony stimulating factor- 1 receptor (CSF-1R) with SNDX-6352, a novel anti-CSF-1R targeted antibody.” Poster presented at the Society for the Immunotherapy of Cancer Annual Meeting, National Harbor, MD.

  Ruffell B., and L. Coussens (2015). “Macrophages and Therapeutic Resistance in Cancer.” Cancer Cell. 27(4):462-72.

• Entinostat

  von Keudell G., D. Sermer, et al. (2020, December) “A Phase II Trial of Pembrolizumab and Entinostat in Relapsed/Refractory Hodgkin Lymphoma.” Slides presented at the American Society for Hematology Virtual Annual Meeting.

  Hellman, M. D., P. A. Janne, et al. (2020, November). “Entinostat plus Pembrolizumab in Patients with Metastatic NSCLC Previously Treated with Anti-PD-(L)1 Therapy”. Clin Cancer Res. DOI: 10.1158/1078-0432.CCR-20-3305.

  Cadoo K., M. Meyers, et al. (2019, June). “ENCORE 603: A phase II randomized study of avelumab plus entinostat versus avelumab plus placebo in patients with advanced and recurrent epithelial ovarian cancer.” Slides presented at the American Society of Clinical Oncology Annual Meeting, Chicago, IL.

  Sullivan R., S. Moschos, et al. (2019, April). “Efficacy and safety of entinostat (ENT) and pembrolizumab (PEMBRO) in patients with melanoma previously treated with anti-PD-1 therapy.” Slides presented at the American Association for Cancer Research Annual Meeting, Atlanta, GA.

  Ordentlich P., L. Wang, et al. (2019, March). “Identification of gene signatures associated with response in a Phase 2 trial of entinostat (ENT) plus pembrolizumab (PEMBRO) in non-small cell lung cancer (NSCLC) patients whose disease has progressed on or after anti-PD-(L)1 therapy.” Slides presented at the American Association for Cancer Research Annual Meeting, Atlanta, GA.

  Nandre R., V. Verma, et al. (2018, November). “Entinostat Increases the Frequency of Tumor-Specific Effector T-cells and Their Functionality is Enhanced by Anti-OX40 Leading to Durable anti-Tumor Effects.” Poster presented at the Society for the Immunotherapy of Cancer Annual Meeting, Washington, DC.

  Hellmann M., P. Janne, et al. (2018, September). “Efficacy/safety of entinostat (ENT) and pembrolizumab (PEMBRO) in NSCLC patients previously treated with anti-PD-(L)1 therapy.” Slides presented at the IASLC 19th World Conference on Lung Cancer, Toronto, Canada.

  Agarwala S. et al. (2018, June) “Efficacy and safety of entinostat (ENT) and pembrolizumab (PEMBRO) in patients with melanoma progressing on or after a PD-1/L1 blocking antibody.” Poster presented at the American Society of Clinical Oncology Annual Meeting, Chicago, IL.

  Azad N. et al. (2018, June). “ENCORE 601: A phase 2 study of entinostat in combination with pembrolizumab in patients with microsatellite stable metastatic colorectal cancer.” Poster presented at the American Society of Clinical Oncology Annual Meeting, Chicago, IL.

  Gandhi L. et al. (2018, June). “Efficacy and safety of entinostat (ENT) and pembrolizumab (PEMBRO) in patients with non-small cell lung cancer (NSCLC) previously treated with anti-PD-(L)1 therapy.” Poster presented at the American Society of Clinical Oncology Annual Meeting, Chicago, IL.

  Tolcher A., M. L. Meyers, et al. (2018, April). “Safety, Efficacy, and Immune Correlates of Alternative Doses and Schedules of Entinostat Combined With Pembrolizumab in Patients With Advanced Solid Tumors – Results From SNDX-275-0141 Phase I Trial.” Slides presented at the American Association for Cancer Research Annual Meeting, Chicago, IL.

  Wang L., D. Chen F., et al. (2018, April). “Enhanced Anti-tumor Activity of the Combination of Entinostat and NKTR-214 in Renal and Colon Cancer Tumor Models.” Poster presented at the American Association for Cancer Research Annual Meeting, Chicago, IL.

  Krieg C., M. Nowicka, et al. (2018) “High-dimensional single-cell analysis predicts response to anti-PD-1 immunotherapy.” Nat Med. 24(2), 144–153.

  Yeruva, S., F. Zhao et al. (2018). “E2112: randomized phase III trial of endocrine therapy plus entinostat/placebo in patients with hormone receptor-positive advanced breast cancer.” NPJ Breast Cancer. 4:1. doi: 10.1038/s41523-017-0053-3. eCollection 2018.

  Pili R., D. Quinn, et at. (2017). “Immunomodulation by Entinostat in Renal Cell Carcinoma Patients Receiving High-Dose Interleukin 2: A Multicenter, Single-Arm, Phase I/II Trial (NCI-CTEP#7870).” Clin Cancer Res. 23(23), 7199-7208

  Johnson M., D. Gabrilovich, et al. (2017, November). “Analysis of biomarkers from a cohort of advanced melanoma patients treated with entinostat (ENT) and pembrolizumab (PEMBRO) previously exposed to immune checkpoint inhibition.” Poster presented at the Society for the Immunotherapy of Cancer Annual Meeting, National Harbor, MD.

  Gandhi L.., M. Johnson, et al. (2017, November). “ENCORE-601: Phase 1b/2 study of entinostat (ENT) in combination with pembrolizumab (PEMBRO) in patients with non-small cell lung cancer.” Poster and slides presented at the Society for the Immunotherapy of Cancer Annual Meeting, National Harbor, MD.

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  Orillion A., A. Hashimoto, et al. (2017). “Entinostat neutralizes myeloid derived suppressor cells and enhances the antitumor effect of PD-1 inhibition in murine models of lung and renal cell carcinoma.” Clin Cancer Res. DOI: 10.1158/1078-0432.CCR-17-0741

  Johnson M. et al. (2017, June). “ENCORE 601: A Phase 2 study of entinostat (ENT) in combination with pembrolizumab (PEMBRO) in patients with melanoma.” Poster presented at the American Society of Clinical Oncology Annual Meeting, Chicago, IL.

  Johnson M., A. Adjei, et al. (2016, November). “Dose-escalation/confirmation results of ENCORE 601, a Phase 1b/2, open-label study of entinostat (ENT) in combination with pembrolizumab (PEMBRO) in patients with non–small cell lung cancer (NSCLC).” Poster presented at the Society for the Immunotherapy of Cancer Annual Meeting, National Harbor, MD.

  Gameiro SR, AS Malamas, et al. (2016).  “Inhibitors of histone deacetylase 1 reverse the immune evasion phenotype to enhance T-cell mediated lysis of prostate and breast carcinoma cells.” Oncotarget. 7(7):7390-402.

  Shen, L., A. Orillion, R. Pili. (2016). “Histone deacetylase inhibitors as immunomodulators in cancer therapeutics.” Epigenomics. 8(3):415-28.
  Merino, VF., N. Nguyen, et al. (2016). “Combined Treatment with Epigenetic, Differentiating, and Chemotherapeutic Agents Cooperatively Targets Tumor-Initiating Cells in Triple-Negative Breast Cancer.” Cancer Res. 76(7):2013-24.

  Tomita, Y., M. Lee, et al. (2016). “The interplay of epigenetic therapy and immunity in locally recurrent or metastatic estrogen receptor-positive breast cancer: Correlative analysis of ENCORE 301, a randomized, placebo controlled phase II trial of exemestane with or without entinostat.” OncoImmunology. DOI: 10.1080/2162402X.2016.1219008

  Schech A, A. Kazi, et al. (2015). “Histone Deacetylase Inhibitor Entinostat Inhibits Tumor-Initiating Cells in Triple-Negative Breast Cancer Cells.” Mol Cancer Ther. 14(8): 1848–1857.

  Park J., S. Thomas, and P. Munster. (2015). “Epigenetic modulation with histone deacetylase inhibitors in combination with immunotherapy”. Epigenomics. 7(4):641-52.

  Connolly R. et al. (2014, December). “E2112: A randomized phase III trial of endocrine therapy plus entinostat or placebo in patients with hormone receptor-positive advanced breast cancer.” Poster presented at the San Antonio Breast Cancer Symposium, San Antonio, TX.

  Kim, K., A. Skora, et al. (2014). “Eradication of metastatic mouse cancers resistant to immune checkpoint blockade by suppression of myeloid-derived cells.” PNAS. 111(32):11774-9.

  Sabnis, G., O. Goloubeva, et al. (2013). “HDAC inhibitor entinostat restores responsiveness of letrozole resistant MCF-7Ca xenografts to AIs through modulation of Her-2.” Mol Cancer Ther. 12(12):2804-16.

  Shah, P., Gau, Y., Sabnis, G. (2013). “Histone deacetylase inhibitor entinostat reverses epithelial to mesenchymal transition of breast cancer cells by reversing the repression of E-cadherin.” Breast Canc Res Treat. 143(1):99-111.

  Yardley, D., R. Ismail-Khan, et al. (2013). “A randomized phase 2, double-blind, placebo-controlled study of exemestane with and without entinostat (SNDX-275) in postmenopausal women with locally recurrent or metastatic estrogen receptor-positive breast cancer progressing on treatment with a non-steroidal aromatase inhibitor.” J Clin Oncol. 31(17):2128-35.

  Youn JI, V. Kumar, et al. (2013). “Epigenetic silencing of retinoblastoma gene regulates pathologic differentiation of myeloid cells in cancer.” Nat. Immunol. 14(3): 211–220.

  Zhu S., C. Denman, et al. (2013). “The narrow-spectrum HDAC inhibitor entinostat enhances NKG2D expression without NK cell toxicity, leading to enhanced recognition of cancer cells.” Pharm. Res. 32(3): 779–92.

  Shen, L., M. Ciesielksi, et al. (2012). “Class I histone deacetylase inhibitor entinostat suppresses regulatory T cells and enhances immunotherapies in renal and prostate cancer models.” PLoSOne. 7(1): e30815.

  Connolly, R., and V. Stearns (2012). “Epigenetics as a therapeutic target in breast cancer.” J Mammary Gland Biol Neoplasia. 17(3-4):191-204.

  Juergens RA, J. Wrangle, et al. (2011). “Combination epigenetic therapy has efficacy in patients with refractory advanced non-small cell lung cancer.” Cancer Discov. 1(7): 598–607.

  Srivastava, R., R. Kurzrock, et al. (2010). “MS-275 sensitizes TRAIL-resistant breast cancer cells, inhibits angiogenesis and metastasis, and reverses epithelial-mesenchymal transition in vivo.” Mol Cancer Ther. 9(12):3254-66.