A Prospective Multicenter Phase II Study on CD69 Expression as a Predictor of Efficacy for Lisaftoclax Combined with Chemotherapy in Treatment-Naïve Acute Myeloid Leukemia

Start Date01 Feb 2026

Sponsor / Collaborator

The First Affiliated Hospital of Wenzhou Medical College

CTIS2024-512503-39-00

/ RecruitingPhase 4

MRD registry and multicenter, randomized controlled pragmatic trial of standard intensity versus reduced intensity consolidation treatment in MRD-negative patients with AML or CLL (RESOLVE trial).

Start Date19 Dec 2025

Sponsor / Collaborator-

NCT07228273

/ RecruitingPhase 2IIT

A Phase II Randomized Clinical Trial of Venetoclax Combined With FLAG IDA Induction and Consolidation Compared to Standard of Care for Newly Diagnosed Patients With Acute Myeloid Leukemia

This phase II trial compares induction and consolidation therapy with fludarabine, cytarabine, idarubicin, and venetoclax to cytarabine and daunorubicin induction and cytarabine consolidation for the treatment of acute myeloid leukemia (AML). Patients with AML often receive induction and consolidation therapy. Induction therapy is given first to get the patient's AML under control (remission). Consolidation therapy is given after the cancer has disappeared following the initial therapy. Consolidation therapy is used to kill any cancer cells that may be left in the body. Chemotherapy drugs, such as fludarabine, cytarabine, idarubicin, and daunorubicin, work in different ways to stop the growth of cancer cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Venetoclax is in a class of medications called B-cell lymphoma-2 (BCL-2) inhibitors. It may stop the growth of cancer cells by blocking Bcl-2, a protein needed for cancer cell survival. Giving fludarabine, cytarabine, idarubicin, and venetoclax for induction and consolidation therapy may be more effective in treating AML.

Start Date12 Dec 2025

Sponsor / Collaborator

OHSU Knight Cancer Institute

[+1]

100 Clinical Results associated with Idarubicin Hydrochloride

100 Translational Medicine associated with Idarubicin Hydrochloride

100 Patents (Medical) associated with Idarubicin Hydrochloride

3,360

Literatures (Medical) associated with Idarubicin Hydrochloride

01 Feb 2026·POULTRY SCIENCE

Investigating the role of melanocortinergic, glutamatergic and neuropeptide Y systems on hypophagia caused by gastric inhibitory polypeptide (GIP) in broilers

Article

Author: Zarei, Hamed ; Mahdavi, Kimia ; Zendehdel, Morteza ; Gharaie, Maryam Lotfi

Appetite control and the metabolic rate of energy expenditure are under the influence of a broad suite of biochemical messengers, which include peptide-based signals, hormonal agents, and neurotransmitters. This research sought to examine the correlation between melanocortinergic, glutamatergic, and neuropeptide Y (NPY) systems with gastric inhibitory polypeptide (GIP) in the modulation of appetite and consummatory behavior. In experiment 1, broilers were injected with GIP at various doses (3, 6 and 12 nmol) in addition to saline. The experimental design for the second trial involved four distinct ICV treatment groups: a saline control, SHU9119 (a MC3/4 melanocortin receptor antagonist, 0.5 nmol), GIP (12 nmol), and a co-infusion of SHU9119 and GIP. Experiments 3 through 11 followed a protocol analogous to that of experiment 2. The sole modification was the substitution of SHU9119 with specific receptor antagonists, administered via intracerebroventricular (ICV) injection. These antagonists targeted the following receptors and their respective doses: MC4 (HS024, 0.5 nmol), NMDA (MK-801, 15 nmol), AMPA (CNQX, 390 nmol), mGluR1 (AIDA, 2 nmol), mGluR2 (LY341495, 150 nmol), mGluR3 (UBP1112, 2 nmol), NPY1 (BMS193885, 1.25 µg), NPY2 (CYM9484, 1.25 µg), and NPY5 (L-152804, 1.25 µg). Cumulative feed intake was subsequently measured at 30, 60, and 120-minute post-infusion intervals. Based on the results, GIP at doses of 6 and 12 nmol induced a significant reduction in food consumption relative to the control group (P < 0.05). This anorexigenic effect of GIP was significantly attenuated by the co-infusion of either the MC3/MC4 receptor antagonist SHU9119 (P < 0.05) or the NMDA receptor antagonist MK-801 (P < 0.05). The collected data imply that signaling cascades dependent on NMDA and melanocortin (MC3/MC4) receptors potentially mediate the anorectic effect of GIP in neonatal broilers, a process which appears independent of NPY Y1, Y2, and Y5 receptor involvement.

15 Jan 2026·BLOOD

CPX-351 in Down syndrome–associated myeloid leukemia: results and prognostic factors from the phase 3 ML-DS 2018 trial

Article

Author: Schuschel, Konstantin ; Gonçalves-Dias, José ; Klusmann, Jan-Henning ; Diederichs, Antonia ; Thol, Felicitas ; Laszig, Stephanie ; Kerp, Helena ; Kreyenberg, Hermann ; Pawińska-Wąsikowska, Katarzyna ; Issa, Hasan ; Miladinovic, Milica ; Goemans, Bianca F. ; Waack-Buchholz, Katharina ; Reinhardt, Dirk ; Boztug, Heidrun ; Salzmann-Manrique, Emilia ; Scheidegger, Nastassja ; Bremm, Melanie ; Rettinger, Eva ; De Moerloose, Barbara ; Wehner, Sibylle ; Hünecke, Sabine

Abstract:

Myeloid leukemia of Down syndrome (ML-DS) is associated with an excellent prognosis but high treatment-related toxicity and mortality. The Phase 3 Clinical Trial for CPX-351 in ML-DS 2018 aimed to maintain the excellent event-free survival (EFS) achieved in the previous ML-DS 2006 trial while reducing the treatment intensity. Intensity-reduced induction and reinduction therapy with cytarabine and idarubicin with or without etoposide was replaced with CPX-351 (66 U/m2 on 3 days in course 1 and on 2 days in course 2). Risk stratification was based on flow cytometric measurable residual disease (MRD) after first induction. High-risk patients received high-dose cytarabine (3 g/m2 per 12 hour) in consolidation; standard-risk patients received cytarabine at a dose of 1 g/m2 per 12 hour. A total of 35 patients were enrolled until the trial was halted because of an unexpectedly high relapse rate. A per-protocol interim analysis revealed a significantly lower 24-month EFS when compared with the ML-DS 2006 trial (69% vs 90%; P< .001). In contrast with previous studies, most patients who relapsed responded to salvage therapy, leading to a comparable 24-month overall survival of 88% (vs 92%; P = .612). CPX-351 demonstrated a favorable toxicity profile with no treatment-related mortality. Positive MRD by error-corrected GATA1 next-generation sequencing, the presence of trisomy 8 or a complex karyotype were associated with an increased risk for relapse. In conclusion, replacing intensity-reduced induction therapy with CPX-351 in ML-DS led to a significantly lower EFS, highlighting the need for dose optimization to balance the efficacy and toxicity in this sensitive patient population. This trial was registered at https://www.clinicaltrialsregister.eu as EudraCT #2018-002988-25.

01 Jan 2026·BIOSENSORS & BIOELECTRONICS

Idarubicin-labeled electrochemical biosensor for detection of circulating tumor cells in colon cancer patients

Article

Author: Jung, Dong-Keun ; Woo, Kwangsook ; Saputra, Heru Agung ; Lasker, Tahera ; Shim, Yoon-Bo ; Karim, Md Mobarok ; Sahin, Md Ali Zaber ; Park, Deog-Su

A disposable electrochemical biosensor was developed for the early diagnosis of metastatic colon cancer through the detection of circulating tumor cells (CTCs), labeling with the electroactive anticancer drug, idarubicin (IDA) as a redox indicator. The sensing platform was constructed by modifying screen-printed carbon electrodes (SPCEs) layer-by-layer with multi-walled carbon nanotubes and poly-terthiophene benzoate, followed by the covalent immobilization of a colon cancer-specific aptamer (W3). Sequential modification of each sensing layer was confirmed via surface and electrochemical characterization experiments. Biocompatibility of the sensing components was initially validated through cytotoxicity assays, demonstrating high cell viability (92.8 %). Under optimized conditions, the biosensor achieved an excellent detection sensitivity, capable of identifying as few as three CTCs spiked in 1.0 mL whole blood using square-wave voltammetry. The sensor exhibited excellent analytical performance, with high accuracy (≥89.6 %), precision (coefficient of variation ≤5.1 %), anti-interference capability (relative interference effect ≤5 %), reproducibility (relative standard deviation ≤ 3 %), and operational stability for up to 36 days under laboratory-controlled conditions. The feasibility of the proposed sensor for practical application was validated through a preliminary clinical study involving five healthy volunteers and five colon cancer patients. Among them, one patient was identified as being at high risk for metastasis, presenting with 24 CTCs/mL, while the remaining four were classified as moderate-risk cases, with CTC levels ranging from 4 to 9 cells/mL.

News (Medical) associated with Idarubicin Hydrochloride

09 Aug 2024

·www.fda.gov

FDA approves quizartinib for newly diagnosed acute myeloid leukemia

On July 20, 2023, the Food and Drug Administration approved quizartinib (Vanflyta, Daiichi Sankyo, Inc.) with standard cytarabine and anthracycline induction and cytarabine consolidation, and as maintenance monotherapy following consolidation chemotherapy, for the treatment of adult patients with newly diagnosed acute myeloid leukemia (AML) that is FLT3 internal tandem duplication (ITD)-positive, as detected by an FDA-approved test. FDA also approved LeukoStrat CDx FLT3 Mutation Assay as a companion diagnostic for Vanflyta. View full prescribing information for Vanflyta. Efficacy of quizartinib with chemotherapy was evaluated in QuANTUM-First (NCT02668653), a randomized, double-blind, placebo-controlled trial of 539 patients with newly diagnosed FLT3-ITD positive AML. FLT3-ITD status was determined prospectively with a clinical trial assay and verified retrospectively with the companion diagnostic LeukoStrat CDx FLT3 Mutation Assay. Patients were randomized (1:1) to receive quizartinib (n=268) or placebo (n=271) with induction and consolidation therapy and as maintenance monotherapy according to the initial assignment. There was no re-randomization at the initiation of post-consolidation therapy. Patients who proceeded to hematopoietic stem cell transplantation (HSCT) initiated maintenance therapy after HSCT recovery. The main efficacy outcome measure was overall survival (OS), measured from randomization date until death by any cause. The primary analysis was conducted after a minimum follow-up of 24 months after the last patient was randomized. The trial demonstrated a statistically significant improvement in OS for the quizartinib arm [hazard ratio (HR) 0.78; 95% CI: 0.62, 0.98; 2‑sided p=0.0324]. The CR rate in the quizartinib arm was 55% (95% CI: 48.7, 60.9) with a median duration of 38.6 months (95% CI: 21.9, NE), and the CR rate in those receiving placebo was 55% (95% CI: 49.2, 61.4) with a median duration of 12.4 months (95% CI: 8.8, 22.7). Quizartinib is not indicated as maintenance monotherapy following allogeneic HSCT. Improvement in OS with quizartinib has not been demonstrated in this setting. A boxed warning for quizartinib notes QT prolongation, torsades de pointes, and cardiac arrest. Quizartinib is available only through a restricted program under a Risk Evaluation and Mitigation Strategy (REMS), called the Vanflyta REMS. See the prescribing information for the full list of adverse reactions. The recommended quizartinib dose is as follows: Induction: 35.4 mg orally once daily on Days 8-21 of “7 + 3” (cytarabine [100 or 200 mg/m2/day] on Days 1 to 7 plus daunorubicin [60 mg/m2/day] or idarubicin [12 mg/m2/day] on Days 1 to 3) and on Days 8-21 or 6-19 of an optional second induction (“7 + 3” or “5 + 2” [5 days cytarabine plus 2 days daunorubicin or idarubicin], respectively), b) Consolidation: 35.4 mg orally once daily on Days 6-19 of high‑dose cytarabine (1.5 to 3 g/m2 every 12 hours on Days 1, 3 and 5) for up to 4 cycles, and c) Maintenance: 26.5 mg orally once daily on Days 1 to 14 and 53 mg once daily, thereafter, for up to thirty-six 28-day cycles. This review used the Assessment Aid, a voluntary submission from the applicant to facilitate the FDA’s assessment. This application was granted priority review, fast track designation and orphan drug designation. FDA expedited programs are described in the Guidance for Industry: Expedited Programs for Serious Conditions-Drugs and Biologics. Healthcare professionals should report all serious adverse events suspected to be associated with the use of any medicine and device to FDA’s MedWatch Reporting System or by calling 1-800-FDA-1088. For assistance with single-patient INDs for investigational oncology products, healthcare professionals may contact OCE’s Project Facilitate at 240-402-0004 or email OncProjectFacilitate@fda.hhs.gov. For information on the COVID-19 pandemic, see the following resources: FDA: Coronavirus Disease 2019 (COVID-19) NCI: Coronavirus: What People With Cancer Should Know CDC: Coronavirus (COVID-19) Follow the Oncology Center of Excellence on Twitter @FDAOncologyExternal Link Disclaimer.

Clinical ResultOrphan DrugFast TrackPriority ReviewDrug Approval

04 Jun 2024

·www.biospace.com

GlycoMimetics Announces Comprehensive Results from Pivotal Phase 3 Study of Uproleselan in Relapsed/Refractory (R/R) Acute Myeloid Leukemia (AML)

Company exploring path forward for uproleselan in multiple AML settings based on observed efficacy results, including clinically meaningful results in primary refractory AML, and significant unmet patient need Uproleselan demonstrated a clinically meaningful improvement in median overall survival (mOS) for patients with primary refractory AML; mOS was 31.2 months for the uproleselan arm compared to 10.1 months for the placebo arm in this subgroup Adverse events for uproleselan were consistent with known side effect profiles of chemotherapy used in the study Advancing discussions with the National Cancer Institute (NCI) and the Alliance for Clinical Trials in Oncology for Phase 2/3 study of uproleselan with chemotherapy in older adults with frontline AML Conference call and webcast to be hosted today, June 4, 2024, at 8:30 am E.T. ROCKVILLE, Md.--(BUSINESS WIRE)-- GlycoMimetics, Inc. (Nasdaq: GLYC), a late clinical-stage biotechnology company discovering and developing glycobiology-based therapies for cancers and inflammatory diseases, today announced comprehensive results from the company’s pivotal Phase 3 study of uproleselan in R/R AML. “There is a wealth of data across large subsets of this pivotal Phase 3 study that help us understand how prespecified stratification factors such as backbone chemotherapy, disease status, and age impacted survival outcomes for patients,” said Daniel DeAngelo, M.D., Ph.D., Professor of Medicine, Harvard Medical School, Chief, Division of Leukemia, Dana-Farber Cancer Institute, and Principal Investigator of the pivotal Phase 3 study. “In the primary refractory setting, uproleselan’s improvement of mOS and greater duration of remission were particularly compelling, as there is a significant unmet need for new treatment options in this setting that can extend and improve the lives of patients. These results demonstrate uproleselan has the potential to address this unmet need in primary refractory AML.” “As we have analyzed data from this large, well-balanced, and well-executed study alongside medical, statistical, and regulatory experts, it has become clear that uproleselan may offer clinically meaningful patient benefit in multiple settings, including primary refractory AML,” said Harout Semerjian, Chief Executive Officer of GlycoMimetics. “We are committed to addressing unmet needs of AML patients and plan to engage with regulators and NCI to discuss potential paths forward for uproleselan.” Results of Pivotal Phase 3 Study of Uproleselan in R/R AML The randomized, double-blind, placebo-controlled Phase 3 clinical study evaluated uproleselan in combination with MEC (mitoxantrone, etoposide and cytarabine) or FAI (fludarabine, cytarabine and idarubicin) in patients with R/R AML. Patients received either uproleselan or placebo for 8 days over 1 cycle of induction and, if applicable, up to 3 cycles of consolidation. The primary endpoint was overall survival (OS), which was not censored for transplant. Secondary endpoints included incidence of severe oral mucositis, complete remission (CR) rate and CR with partial hematologic recovery (CRh). A total of 388 patients in nine countries were randomized 1:1 between treatment and placebo arms. There were 59 sites that enrolled at least one patient. Median follow up was over three years at the time of primary analysis. Overall Survival Primary Endpoint: mOS in the intent-to-treat (ITT) population (n=388) was 13.0 months for the uproleselan arm, compared to 12.3 months for the placebo arm (hazard ratio [HR] 0.89; 95% confidence interval [CI] 0.69-1.15); this difference is not statistically significant. Disease Status Primary Refractory: mOS for primary refractory patients in the uproleselan arm (n=62) was 31.2 months, compared to 10.1 months (HR 0.58; 95% CI 0.37-0.91) for the placebo arm (n=66). This benefit was irrespective of backbone chemotherapy. Median duration of response (DoR) for complete remission (CR) was not reached for primary refractory patients in the uproleselan arm compared to a median DoR of 12.7 months for the placebo arm. Early Relapse: mOS for early relapse patients in the uproleselan arm (n=28) was 3.7 months, compared to 6.4 months (HR 1.50; 95% CI 0.69-3.27) for the placebo arm (n=22). Late Relapse: mOS for late relapse patients in the uproleselan arm (n=104) was 15.4 months, compared to 18.2 months (HR 1.10; 95% CI 0.77-1.57) for the placebo arm (n=106). Backbone Chemotherapy: FAI: mOS for patients treated with uproleselan plus FAI (n=98) was 30.2 months compared to 12.8 months (HR 0.73; 95% CI 0.50-1.06) for patients treated with FAI alone (n=96) in the ITT population. MEC: mOS for patients treated with uproleselan plus MEC (n=96) was 8.7 months compared to 12.3 months (HR 1.06; 95% CI 0.75-1.51) for patients treated with MEC alone (n=98) in the ITT population. Transplantation Status: For patients who received hematopoietic stem cell transplantation (HSCT) after study treatment, mOS was not reached for patients in the uproleselan arm (n=101). In contrast, for HSCT patients in the placebo arm, mOS for patients receiving FAI (n=53) was 26.3 months and for patients receiving MEC (n=46) was 24.4 months. Secondary Endpoints 7.2% of patients in each arm (n=388) experienced induction emergent severe oral mucositis. 36.1% of patients in the uproleselan arm (n=194) experienced CR at the end of induction (EOI) as determined by an independent endpoint review committee (IERC), compared to 33.5% of patients in the placebo arm (n=194). 46.4% of patients in the uproleselan arm experienced CR/CRh at EOI as determined by IERC, compared to 41.2% of patients in the placebo arm. Post-treatment HSCT rate was 52.1% in the uproleselan arm and 51.0% in the placebo arm. Subsequent AML therapy in non-responders was 40.0% in the uproleselan arm (n=80) and 46.2% in the placebo arm (n=78). Safety Adverse events were consistent with the known safety pro backbone chemotherapy regimens. 35.9% of patients in the uproleselan arm experienced serious treatment-emergent adverse events (TEAEs) compared to 34.2% in the placebo arm. 85.9% of patients in the uproleselan arm experienced grade 3 or higher TEAEs compared to 87.6% in the placebo arm. NCI Phase 2/3 Study of Uproleselan in Frontline AML In addition to the company’s pivotal Phase 3 trial of uproleselan, the National Cancer Institute (NCI) and the Alliance for Clinical Trials in Oncology are conducting an adaptive Phase 2/3 study of uproleselan in adults with newly diagnosed AML who are 60 years or older and fit for intensive chemotherapy. Their randomized, controlled study is evaluating the addition of uproleselan to a standard cytarabine / daunorubicin regimen (7+3) versus chemotherapy alone. The Phase 2 portion of the study completed enrollment of 267 patients in December 2021. The Company is advancing discussions with the NCI and the Alliance for Clinical Trials in Oncology based on the results of the pivotal Phase 3 study of uproleselan in R/R AML. Conference Call Details To access the call by phone, please go to this registration link and you will be provided with dial in details. Participants are encouraged to connect 15 minutes in advance of the scheduled start time. A live webcast of the call and the corresponding slides will be available on the “Investors” tab on the GlycoMimetics website. A webcast replay will be available for 30 days following the call. About AML AML is the most common acute leukemia in adults. A cancer of the bone marrow, nearly 21,000 people in the United States are diagnosed with AML each year. Despite the availability of multiple treatments, disease prognosis is poor, and new treatment options are needed to improve outcomes. Newly diagnosed AML has the lowest 5-year survival rate of all leukemias at 31.7%. The five-year survival rate for people with relapsed/refractory disease is only 10%. About Uproleselan Discovered and developed by GlycoMimetics, uproleselan (yoo’ pro le’se lan) is an investigational, first-in-class E-selectin antagonist. GlycoMimetics has received Breakthrough Therapy and Fast Track designations from the U.S. Food and Drug Administration (FDA) and Breakthrough Therapy designation from the Chinese National Medical Products Administration for uproleselan as a potential treatment for adult AML patients with relapsed or refractory disease. E-selectin is a leukocyte adhesion molecule constitutively expressed on endothelial cells of the vasculature and bone marrow. In AML, there is evidence that E-selectin–ligand interaction between endothelial cells in the protective niche of the Bone Marrow microEnvironment (BME) and leukemic stem cells and blasts promotes leukemic cell survival and hides them from AML therapies. Uproleselan is designed to disrupt E-selectin binding and prevent leukemic myeloid cells using the protective niche of the BME. About GlycoMimetics, Inc. GlycoMimetics is a late clinical-stage biotechnology company discovering and developing glycobiology-based therapies for cancers, including AML, and for inflammatory diseases. The company’s scientific approach is based on an understanding of the role that carbohydrates play in cell recognition. Its specialized chemistry platform is being deployed to discover small molecule drugs, known as glycomimetics, that alter carbohydrate-mediated recognition in diverse disease states, including cancers and inflammation. GlycoMimetics is leveraging its differentiated expertise with this scientific approach in order to advance its pipeline of wholly owned drug candidates. The company’s goal is to develop transformative therapies for diseases with high unmet medical need. GlycoMimetics is headquartered in Rockville, MD in the BioHealth Capital Region. Learn more at . Forward-Looking Statements This press release contains forward-looking statements. These forward-looking statements may include, but are not limited to, statements regarding the conduct of, and timing for analysis and presentation of data from, clinical trials; potential development and regulatory activities; and the potential benefits and impact of uproleselan. Actual results may differ materially from those described in these forward-looking statements. For a further description of the risks associated with these statements, as well as other risks facing GlycoMimetics, please see the risk factors described in the company’s Annual Report on Form 10-K filed with the U.S. Securities and Exchange Commission (SEC) on March 27, 2024, the company’s Quarterly Report on Form 10-Q filed with the SEC on May 9, 2024, and other filings GlycoMimetics makes with the SEC from time to time. Forward-looking statements speak only as of the date of this release, and GlycoMimetics undertakes no obligation to update or revise these statements, except as may be required by law.

Clinical ResultPhase 3Phase 2Fast TrackBreakthrough Therapy

06 May 2024

·www.biospace.com

GlycoMimetics Announces Results of Pivotal Phase 3 Study of Uproleselan in Relapsed/Refractory (R/R) Acute Myeloid Leukemia (AML)

Study of uproleselan combined with chemotherapy did not meet its primary endpoint of overall survival in the intent to treat population Adverse events were consistent with known side effect profiles of chemotherapy used in the study Comprehensive data analysis with medical, statistical, and regulatory experts underway and will be shared as appropriate; company will submit results for presentation at an upcoming medical meeting National Cancer Institute (NCI) Phase 2/3 study in newly diagnosed AML patients remains ongoing Conference call and webcast to be hosted today, May 6, 2024, at 8:30 a.m. ET. ROCKVILLE, Md.--(BUSINESS WIRE)-- GlycoMimetics, Inc. (Nasdaq: GLYC), a late clinical-stage biotechnology company discovering and developing glycobiology-based therapies for cancers and inflammatory diseases, today announced topline results from its Phase 3 global pivotal study of uproleselan in 388 patients with R/R AML. In the study, uproleselan combined with chemotherapy did not achieve a statistically significant improvement in overall survival in the intent to treat population versus chemotherapy alone. Patients treated with uproleselan had a median overall survival of 13 months, compared to 12.3 months in the placebo arm. Adverse events were consistent with known side effect profiles of chemotherapy used in the study. “While the outcome of our Phase 3 study in R/R AML is not what we hoped, we wish to thank the investigators, the participating patients and their families for their dedication to this large, well-controlled randomized study,” said Harout Semerjian, Chief Executive Officer of GlycoMimetics. “We are thoroughly analyzing the data in collaboration with medical, statistical and regulatory experts and are committed to submitting a comprehensive data analysis for presentation at an upcoming medical meeting.” The randomized, double-blind, placebo-controlled Phase 3 clinical study evaluated uproleselan in combination with MEC (mitoxantrone, etoposide and cytarabine) or FAI (fludarabine, cytarabine and idarubicin) in patients with R/R AML. Patients received either uproleselan or placebo for 8 days over 1 cycle of an induction and, if applicable, up to 3 cycles of consolidation. The primary endpoint of the study was overall survival without censoring for transplant. Secondary endpoints included incidence of severe oral mucositis, complete remission rate and remission rate. A total of 388 patients across 70 sites in nine countries were randomized 1:1 between treatment and placebo arms. The NCI and the Alliance for Clinical Trials in Oncology are conducting an adaptive Phase 2/3 study of uproleselan in adults with newly diagnosed AML who are 60 years or older and fit for intensive chemotherapy. The randomized, controlled study is evaluating the addition of uproleselan to a standard cytarabine/daunorubicin regimen (7+3) versus chemotherapy alone. The Phase 2 portion of the study completed enrollment of 267 patients in December 2021. Results of the pre-planned Phase 2 event free survival interim analysis will be reported when available. First Quarter 2024 Preliminary Financial Results Today, the company also disclosed its preliminary financial results for the first quarter of 2024. Cash position: As of March 31, 2024, GlycoMimetics had cash and cash equivalents of $31.3 million, compared to $41.8 million as of December 31, 2023. R&D Expenses: The company’s research and development expenses increased to $6.0 million for the quarter ended March 31, 2024, as compared to $5.4 million for the same period in 2023. These increases were due to raw material acquisition costs for future manufacturing batches. G&A Expenses: The company’s general and administrative expenses decreased to $5.1 million for the quarter ended March 31, 2024, compared to $5.5 million for the same period in 2023. The decrease was due to lower personnel-related and external consulting expenses. Shares Outstanding: Shares of common stock outstanding as of March 31, 2024, were 64,450,835. Conference Call Information The company will host a conference call and webcast today at 8:30 a.m. ET. To access the call by phone, please go to this registration link and you will be provided with dial in details. Participants are encouraged to connect 15 minutes in advance of the scheduled start time. A live webcast of the call will be available on the “Investors” tab on the GlycoMimetics website. A webcast replay will be available for 30 days following the call. Please note this call will replace the previously announced First Quarter 2024 Financial Results call scheduled for May 9, 2024 at 8:30 a.m. ET. About AML AML is the most common acute leukemia in adults. A cancer of the bone marrow, nearly 21,000 people in the United States are diagnosed with AML each year. Despite the availability of multiple treatments, disease prognosis is poor, and new treatment options are needed to improve outcomes. Newly diagnosed AML has the lowest 5-year survival rate of all leukemias at 31.7%. The five-year survival rate for people with relapsed/refractory disease is only 10%. About Uproleselan Discovered and developed by GlycoMimetics, uproleselan (yoo’ pro le’se lan) is an investigational, first-in-class E-selectin antagonist. GlycoMimetics has received Breakthrough Therapy and Fast Track designations from the U.S. Food and Drug Administration (FDA) and Breakthrough Therapy designation from the Chinese National Medical Products Administration for uproleselan as a potential treatment for adult AML patients with relapsed or refractory disease. E-selectin is a leukocyte adhesion molecule constitutively expressed on endothelial cells of the vasculature and bone marrow. In AML, there is evidence that E-selectin–ligand interaction between endothelial cells in the protective niche of the Bone Marrow microEnvironment (BME) and leukemic stem cells and blasts promotes leukemic cell survival and hides them from AML therapies. Uproleselan is designed to disrupt E-selectin binding and prevent leukemic myeloid cells using the protective niche of the BME. About GlycoMimetics, Inc. GlycoMimetics is a late clinical-stage biotechnology company discovering and developing glycobiology-based therapies for cancers, including AML, and for inflammatory diseases. The company’s scientific approach is based on an understanding of the role that carbohydrates play in cell recognition. Its specialized chemistry platform is being deployed to discover small molecule drugs, known as glycomimetics, that alter carbohydrate-mediated recognition in diverse disease states, including cancers and inflammation. GlycoMimetics is leveraging its differentiated expertise with this scientific approach in order to advance its pipeline of wholly owned drug candidates. The company’s goal is to develop transformative therapies for diseases with high unmet medical need. GlycoMimetics is headquartered in Rockville, MD in the BioHealth Capital Region. Learn more at . Forward-Looking Statements This press release contains forward-looking statements. These forward-looking statements may include, but are not limited to, statements regarding the conduct of, and timing for analysis and presentation of data from, clinical trials; potential development and regulatory activities; and the potential benefits and impact of uproleselan. Actual results may differ materially from those described in these forward-looking statements. For a further description of the risks associated with these statements, as well as other risks facing GlycoMimetics, please see the risk factors described in the company’s Annual Report on Form 10-K filed with the U.S. Securities and Exchange Commission (SEC) on March 27, 2024, and other filings GlycoMimetics makes with the SEC from time to time. Forward-looking statements speak only as of the date of this release, and GlycoMimetics undertakes no obligation to update or revise these statements, except as may be required by law. View source version on businesswire.com: Contacts Investor Contact: Argot Partners Leo Vartorella 212-600-1902 Glycomimetics@argotpartners.com Source: GlycoMimetics, Inc. View this news release online at:

Clinical ResultPhase 3Financial StatementBreakthrough Therapy

100 Deals associated with Idarubicin Hydrochloride

External Link

KEGG	Wiki	ATC	Drug Bank
D01747	Idarubicin Hydrochloride	L01DB06	DB01177

R&D Status

10 top approved records.

to view more data

Indication	Country/Location	Organization	Date
acute leukemia	China	Pfizer Pharmaceutical (Wuxi) Co. Ltd.	04 Jan 2005
Acute Lymphoblastic Leukemia	China	Pfizer Pharmaceutical (Wuxi) Co. Ltd.	04 Jan 2005
Acute Myeloid Leukemia	United States	Pfizer Inc.	27 Sep 1990

Clinical Result

Indication

Phase

Evaluation

View All Results

Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


ASH2025	Not Applicable	Acute Myeloid Leukemia	1,770	Daunorubicin	htaibykgfq(xuuxrjypjo): HR = 0.99 (95.0% CI, 0.83 - 1.15), P-Value = 0.96 View more	Negative	06 Dec 2025
				Idarubicin
ASH2025	Not Applicable	Blast Phase Chronic Granulocytic Leukemia	13	CLIA + TKI (frontline)	maxtoymozb(zxikosvmtp) = Eight of 13 patients (62%) experienced at least one adverse event, most commonly infections, febrile neutropenia, or laboratory evidence of renal or hepatic dysfunction. Three patients required hospitalization for infection-related complications. Non-infectious AEs were generally mild and often unrelated to study treatment. No unexpected or treatment-related deaths occurred. rccqpavyfe (bzbwvhsgqj )	Positive	06 Dec 2025
				CLIA + TKI (R/R)
NCT02115295 (ASH2025)	Phase 2	Acute Myeloid Leukemia First line KMT2A rearrangement	22	Cladribine, Idarubicin, Cytarabine (CLIA)	dxkkhebbji(wwbgryjads) = kyxcnqlfrt smqqinsiyr (jjbskijibe ) View more	Positive	06 Dec 2025
				Cladribine, Idarubicin, Cytarabine + Venetoclax (CLIA-Ven)	dxkkhebbji(wwbgryjads) = hhsztytcpa smqqinsiyr (jjbskijibe ) View more
ASH2025	Not Applicable	Refractory acute myeloid leukemia	16	CLIA-venetoclax	nqtniosjtu(fltkezuivx) = ephfippfrn ccsrmyghfp (blbpgbdrmb ) View more	Positive	06 Dec 2025
ASH2025	Phase 2	Acute Myeloid Leukemia	101	Decitabine priming + Low-dose Idarubicin + Cytarabine + G-CSF	rqfpdlimld(zblaymcgyj) = fdawppczmk cxxmhmvyey (jgagprqlxl, 64.6 - 81.9) View more	Positive	06 Dec 2025
ASH2025	Not Applicable	ANKRD26 pathogenic variants	27	Venetoclax combined with cladribine, idarubicin, and cytarabine	obphetcnyb(texkevibzg) = tggugwxhfv cdgskybbdx (igotnimsuo ) View more	Positive	06 Dec 2025
ASH2025	Not Applicable	Acute myeloid leukemia with mutated NPM1 NPM1 mutation	53	FLAI regimen	etoglhvpqp(vqznlnjukw) = gnzchktmlo vyggbowddn (sczdtffrph ) View more	Positive	06 Dec 2025
ASH2025	Not Applicable	Core binding factor acute myeloid leukemia	875	FLAG-IDA	oekdjmfzfd(rxbeqgtxly) = nfferyyhtu kucgdddnjk (ofdpwaqmdt ) View more	Positive	06 Dec 2025
				DAE	oekdjmfzfd(rxbeqgtxly) = vouaupltrc kucgdddnjk (ofdpwaqmdt ) View more
PB2472 (EHA2025)	Not Applicable	Acute Myeloid Leukemia KMT2A-r \| DNMT3A \| TET2 ... View more	-	7+3 chemotherapy	jrxmtwpznd(uvbbahdafy) = ntrvhlqgwk raenlrrcuz (ayrkfwrtyp ) View more	Positive	14 May 2025
GIMEMA LAL1913 (EHA2025)	Phase 2	Adult Lymphoblastic Lymphoma	50	Pegaspargase-based treatment	vanozdqiqv(wtduuoyeiw) = A better 5-year DFS was observed in patients ≤40 years (80%) when compared with those >40 (56%) uymdswtnpc (extadlrnxf ) View more	Positive	14 May 2025
				High cumulative dose (HCD) steroids

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