Publications & Presentations

Hematologic Malignancies

Latest Publications
 

Imetelstat in intermediate-2 or high-risk myelofibrosis refractory to JAK inhibitor: IMpactMF phase III study design. Mascarenhas et al, Journal of Future Oncology 2022

Imetelstat Induces Leukemia Stem Cell Death in Pediatric Acute Myeloid Leukemia Patient-Derived Xenografts. Barwe et al, Journal of Clinical Medicine 2022

The clinical dilemma of JAK inhibitor failure in myelofibrosis: Predictive characteristics and outcomes. Mascarenhas et al, Cancer 2022

Favorable overall survival with imetelstat in relapsed/refractory myelofibrosis patients compared with real-world data. Kuykendall et al., Annals of Hematology 2021

Efficacy of Imetelstat is Independent of Molecular Subtypes in Heavily Transfused Non-Del(5q) Lower Risk MDS (LR-MDS) Relapsed/Refractory (R/R) to Erythropoiesis Stimulating Agents (ESA). Platzbecker, et al, EHA 2021

Imetelstat Achieves Meaningful and Durable Transfusion Independence in High Transfusion–Burden Patients With Lower-Risk Myelodysplastic Syndromes in a Phase II Study. Steensma, et al., Journal of Clinical Oncology 2021

Imetelstat Demonstrates an Acceptable Safety Profile in Myeloid Malignancies. Mascarenhas, et al, EHA 2021

Randomized, Single-Blind, Multicenter Phase II Study of Two Doses of Imetelstat in Relapsed or Refractory Myelofibrosis. Mascarenhas, et al., Journal of Clinical Oncology 2021

Clinical: MDS
 

Efficacy of Imetelstat is Independent of Molecular Subtypes in Heavily Transfused Non-Del(5q) Lower Risk MDS (LR-MDS) Relapsed/Refractory (R/R) to Erythropoiesis Stimulating Agents (ESA). Platzbecker, et al, EHA 2021

Imetelstat Achieves Meaningful and Durable Transfusion Independence in High Transfusion–Burden Patients With Lower-Risk Myelodysplastic Syndromes in a Phase II Study. Steensma, et al., Journal of Clinical Oncology 2021

Imetelstat Demonstrates an Acceptable Safety Profile in Myeloid Malignancies. Mascarenhas, et al, EHA 2021

Treatment with Imetelstat Provides Durable Transfusion Independence (TI) in Heavily Transfused Non-del(5q) Lower Risk MDS (LR-MDS) Relapsed/Refractory (R/R) to Erythropoiesis Stimulating Agents (ESAs). Platzbecker, et al, ASH 2020

IMerge: A Phase 3 Study to Evaluate Imetelstat in Transfusion-Dependent Subjects with IPSS Low or Intermediate-1 Risk Myelodysplastic Syndromes (MDS) that is Relapsed/Refractory to Erythropoiesis-Stimulating Agent (ESA) Treatment. Platzbecker, et al, ASH 2020

Treatment with Imetelstat Provides Durable Transfusion Independence (TI) in Heavily Transfused Non-Del(5Q) Lower Risk MDS (LR-MDS) Relapsed/Refractory (R/R) to Erythropoiesis Stimulating Agents (ESAs). Platzbecker, et al, EHA 2020

IMerge: A Study to Evaluate Imetelstat (GRN163L) in Transfusion-Dependent Subjects with IPSS Low or Intermediate-1 Risk Myelodysplastic Syndromes (MDS) that is Relapsed/Refractory to Erythropoiesis-Stimulating Agent (ESA) Treatment. Platzbecker, et al, ASH 2019

Treatment with Imetelstat Provides Durable Transfusion Independence in Heavily Transfused Non-del(5q) Lower Risk MDS Relapsed/Refractory to Erythropoiesis-Stimulating Agents. Fenaux, P, et al, EHA 2019

Imetelstat Treatment Leads to Durable Transfusion Independence in RBC Transfusion-Dependent, Non-Del(5q) Lower Risk MDS Relapsed/Refractory to Erythropoiesis-Stimulating Agent Who Are Lenalidomide and HMA Naive. Steemsma D, et al. ASH 2018

Imetelstat in RBC Transfusion-Dependent Lower Risk MDS Relapsed/Refractory to Erythropoiesis-Stimulating Agents (IMerge): Updated Efficacy and Safety. Fenaux P, et al. EHA 2018

Efficacy and Safety of Imetelstat in RBC Transfusion-Dependent (TD) IPSS Low/Int-1 MDS Relapsed/Refractory to Erythropoiesis-Stimulating Agents (ESA) (IMerge). Fenaux P, et al. ASH 2017

Imetelstat Therapy in Refractory Anemia with Ring Sideroblasts with or without Thrombocytosis. Tefferi A, et al. Blood Cancer Journal 2016

Telomerase Inhibitor Imetelstat Therapy in Refractory Anemia with Ring Sideroblasts with or without Thrombocytosis. Tefferi A, et al. ASH 2015

Clinical: MF
 

Imetelstat in intermediate-2 or high-risk myelofibrosis refractory to JAK inhibitor: IMpactMF phase III study design. Mascarenhas et al, Journal of Future Oncology 2022

Favorable overall survival with imetelstat in relapsed/refractory myelofibrosis patients compared with real-world data. Kuykendall et al., Annals of Hematology 2021

Randomized, Single-Blind, Multicenter Phase II Study of Two Doses of Imetelstat in Relapsed or Refractory Myelofibrosis. Mascarenhas, et al., Journal of Clinical Oncology 2021

Potential Disease-Modifying Activity of Imetelstat Demonstrated By Reduction in Cytogenetically Abnormal Clones and Mutation Burden Leads to Clinical Benefits in Relapsed/Refractory Myelofibrosis Patients. Mascarenhas, et al, ASH 2020

Telomerase Activity, Telomere Length and hTERT Expression Correlate with Clinical Outcomes in Higher-Risk Myelofibrosis (MF) Relapsed/Refractory (R/R) to Janus Kinase Inhibitor Treated with Imetelstat. Mascarenhas, et al, ASH 2020

Favorable Overall Survival with Imetelstat Treatment Correlates with Other Clinical Benefits in Intermediate-2 or High-Risk Myelofibrosis Relapsed/Refractory to Janus Kinase Inhibitor. Mascarenhas, et al, ASH 2020

Correlation Analyses of Imetelstat Exposure with Pharmacodynamic Effect, Efficacy and Safety in a Phase 2 Study in Patients with Higher-risk Myelofibrosis Refractory to Janus Kinase Inhibitor Identified an Optimal Dosing Regimen for Phase 3 Study. Mascarenhas, et al, ASH 2020

Imetelstat Treatment Results in Clinical Benefits, Including Improved Overall Survival, in Patients with Higher-Risk Triple Negative Myelofibrosis Relapsed/Refractory to Janus Kinase Inhibitors (JAKi). Kiladjian, et al, ASH 2020

Treatment with Imetelstat Improves Myelofibrosis-Related Symptoms and Other Patient-Reported Outcomes in Patients with Relapsed or Refractory Higher-Risk Myelofibrosis. Kiladjian, et al, ASH 2020

A Randomized Open-Label, Phase 3 Study to Evaluate Imetelstat Versus Best Available Therapy in Patients with Intermediate-2 or High-risk Myelofibrosis (MF) Refractory to Janus Kinase (JAK) Inhibitor. Mascarenhas, et al, ASH 2020

Favorable Overall Survival with Imetelstat Treatment Correlates with Other Clinical Benefits in Intermediate-2 or High-Risk Myelofibrosis Relapsed/Refractory to Janus Kinase Inhibitor. Mascarenhas, et al, EHA 2020

Imetelstat Treatment Results in Clinical Benefits, Including Improved Overall Survival, in Patients with Higher-Risk Triple-Negative Myelofibrosis Relapsed/Refractory to Janus Kinase Inhibitors (JAKi). Kiladjian, et al, EHA 2020

Telomerase Activity, Telomere Length and hTERT Expression Correlate with Clinical Outcomes in Higher-Risk Myelofibrosis (MF) Relapsed/Refractory (R/R) to Janus Kinase Inhibitor Treated with Imetelstat. Mascarenhas, et al, EHA 2020

Favorable Overall Survival of Imetelstat-treated Relapsed/Refractory Myelofibrosis Patients Compared with Closely Matched Real World Data. Kuykendall, A, et al. EHA 2019

Imetelstat Is Effective Treatment for Patients with Intermediate-2 or High-Risk Myelofibrosis Who Have Relapsed on or Are Refractory to Janus Kinase Inhibitor Therapy: Results of a Phase 2 Randomized Study of Two Dose Levels. Mascarenhas J, et al. ASH 2018

A Pilot Study of the Telomerase Inhibitor Imetelstat for Myelofibrosis. Tefferi A, et al. N Engl J Med. 2015

Imetelstat, a Telomerase Inhibitor, Therapy for Myelofibrosis: A Pilot Study. Tefferi A, et al. ASH 2014

Imetelstat, a Telomerase Inhibitor, Induces Morphologic and Molecular Remissions in Myelofibrosis and Reversal of Bone Marrow Fibrosis. Tefferi A, et al. ASH 2013

Non-Clinical
 

Imetelstat Induces Leukemia Stem Cell Death in Pediatric Acute Myeloid Leukemia Patient-Derived Xenografts. Barwe et al, Journal of Clinical Medicine 2022

Imetelstat Inhibits Telomerase and Prevents Propagation of ADAR1-Activated Myeloproliferative Neoplasm and Leukemia Stem Cells. Ma, et al, ASH 2020

Combination Treatment with Imetelstat, a Telomerase Inhibitor, and Ruxolitinib Depletes Myelofibrosis Hematopoietic Stem Cells and Progenitor Cells. Wang, X, et al, ASH 2019

Imetelstat, a Telomerase Inhibitor, Is Capable of Depleting Myelofibrosis Hematopoietic Stem Cells and Progenitor Cells. Hu C, et al. ASH 2017

Telomerase Inhibition Impairs Self-Renewal of b-Catenin Activated Myeloproliferative Neoplasm Progenitors. Ma W, et al. ASH 2017

Integrated Molecular Analysis Identifies Replicative Stress as Sensitizer to Imetelstat Therapy in AML. Bruedigam C, et al. ASH 2017

Telomerase inhibitor imetelstat in combination with the BCL-2 inhibitor venetoclax enhances apoptosis in vitro and increases survival in vivo in acute myeloid leukemia. Rusbuldt JJ, et al. AACR 2017

Imetelstat, a telomerase inhibitor, differentially affects normal and malignant megakaryopoiesis. Mosoyan G, et al. Leukemia 2017 (advance online publication)

The preclinical efficacy of a novel telomerase inhibitor, imetelstat, in AML: A randomized trial in patient-derived xenografts. Bruedigam C, et al. ASH 2016

Telomerase Inhibition with Imetelstat Eradicates β-catenin Activated BC CML Stem Cells. Ma W, et al. ASH 2016

Myelosuppression in Patients Treated With the Telomerase Inhibitor Imetelstat is Not Mediated Through Activation of Toll-Like Receptors. Baerlocher GM, et al. AACR 2016

Impact of Hypomethylating Agents on hTERT Expression and Synergistic Effect in Combination With Imetelstat, a Telomerase Inhibitor, in Acute Myeloid Leukemia Cell Lines. Rusbuldt J, et al. AACR 2016

Telomerase inhibition effectively targets mouse and human AML stem cells and delays relapse following chemotherapy. Bruedigam C, et al. Cell Stem Cell 2014

Imetelstat (GRN163L), a Telomerase Inhibitor Selectively Affects Malignant Megakaryopoiesis in Myeloproliferative Neoplasms (MPN). Iancu-Rubin C, et al. ASH 2014

Effects of Imetelstat on CD34+ Cells of Patients with Myelofibrosis. Wang X, et al. ASH 2014

Imetelstat, A Potent Telomerase Inhibitor, Inhibits the Spontaneous Growth of CFU-Meg In Vitro From Essential Thrombocythemia Patients but Not From Healthy Individuals. Brunold C, et al. ASH 2011

Other Publications and Resources
 

The clinical dilemma of JAK inhibitor failure in myelofibrosis: Predictive characteristics and outcomes. Mascarenhas et al, Cancer 2022

Between a rux and a hard place: evaluating salvage treatment and outcomes in myelofibrosis after ruxolitinib discontinuation. Kuykendall et al, Ann Hematol 2018

Clonal evolution and outcomes in myelofibrosis after ruxolitinib discontinuation. Newberry et al, Blood 2017

Impact of genomic alterations on outcomes in myelofibrosis patients undergoing JAK1/2 inhibitor therapy. Spiegel et al, Blood Advances 2017

Characterization of Disease, Treatment Patterns, and Outcomes of Patients With Myelofibrosis: Analysis of Two United States Commercial Claims Databases. Mehra M, et al. ASH 2016

Telomerase in hematologic malignancies. Bruedigam C, Lane SW. Curr Opin Hematol. 2016

How I treat myelofibrosis. Cervantes F. Blood 2014

Revised response criteria for myelofibrosis: International Working Group-Myeloproliferative Neoplasms Research and Treatment (IWG-MRT) and European LeukemiaNet (ELN) consensus report. Tefferi A, et al. Blood 2013

How we treat lower-risk myelodysplastic syndromes. Fenaux P and Adès L. Blood 2013

DIPSS Plus: A Refined Dynamic International Prognostic Scoring System for Primary Myelofibrosis That Incorporates Prognostic Information From Karyotype, Platelet Count, and Transfusion Status. Gangat N, et al. JCO 2011

Imetelstat (GRN163L) – Telomerase-based cancer therapy. Roeth A, et al. Recent Results in Cancer Research 2010

Telomerase and cancer therapeutics. Harley CB. Nature Reviews Cancer 2008