Sequential Decitabine in Combination With FLAG-Ida Followed Immediately by Reduced-Intensity Conditioning (RIC) Allogeneic Hematopoietic Cell Transplantation (DEC-FLAG-Ida/RIC) for Adults With Myeloid Malignancies at High Risk of Relapse: A Phase 1/2 Study

This phase I/II trial studies the safety, side effects, and best dose of decitabine in combination with fludarabine, cytarabine, filgrastim, and idarubicin (FLAG-Ida) and total body irradiation (TBI) followed by a donor stem cell transplant in treating adult patients with cancers of blood-forming cells of the bone marrow (myeloid malignancies) that are at high risk of coming back after treatment (relapse). Cancers eligible for this trial are acute myeloid leukemia (AML), myelodysplastic syndrome (MDS), and chronic myelomonocytic leukemia (CMML). Decitabine is in a class of medications called hypomethylation agents. It works by helping the bone marrow produce normal blood cells and by killing abnormal cells in the bone marrow. The FLAG-Ida regimen consists of the following drugs: fludarabine, cytarabine, filgrastim, and idarubicin. These are chemotherapy drugs that 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. Filgrastim is in a class of medications called colony-stimulating factors. It works by helping the body make more neutrophils, a type of white blood cell. Radiation therapy uses high energy x-rays, particles, or radioactive seeds to kill cancer cells and shrink tumors. TBI is radiation therapy to the entire body. Giving chemotherapy and TBI before a donor peripheral blood stem cell (PBSC) transplant helps kill cancer cells in the body and helps make room in the patient's bone marrow for new blood-forming cells (stem cells) to grow. When the healthy stem cells from a donor are infused into a patient, they may help the patient's bone marrow make more healthy cells and platelets. Giving decitabine in combination with FLAG-Ida and TBI before donor PBSC transplant may work better than FLAG-Ida and TBI alone in treating adult patients with myeloid malignancies at high risk of relapse.

Start Date01 Sep 2025

Sponsor / Collaborator

Fred Hutchinson Cancer Research Center

NCT07059975

/ Not yet recruitingEarly Phase 1IIT

UPdated Disease Monitoring And Treatment for Enhanced Outcomes for Pediatric AML: A Pilot Trial

This research study investigates the tolerability of substituting two cycles of chemotherapy into the standard pediatric acute myeloid leukemia (AML) chemotherapy treatment regimen for patients with newly diagnosed AML at intermediate-risk (IR) and high-risk (HR) of relapse. The goal is to achieve similar or better survival with chemotherapy cycles that are intensive but less likely to cause long-term complications. Patients will enroll on this trial at the end of their first induction cycle.
The two cycles to be substituted are:
* "Ida-FLA" (idarubicin+fludarabine/cytarabine) as Induction 2
* "VIA" (venetoclax+idarubicin+cytarabine) as Intensification 1 of the HR treatment regimen, and Intensification 2 of the IR treatment backbone.
Researchers will evaluate side effects and outcomes for up to three years after enrollment.
Participants will also have the opportunity to participate in optional research studies including patient surveys and blood and bone marrow sample testing.

Start Date01 Aug 2025

Sponsor / Collaborator

Baylor College of Medicine

[+1]

NCT07046078

/ Not yet recruitingPhase 2IIT

Pilot Study of FLAG-Ida Followed Immediately by Reduced-Intensity Allogeneic HCT for Adults Age 60 and Older With Newly Diagnosed Adverse-Risk AML or Other High-Grade Myeloid Neoplasm

This phase II trial tests the safety, side effects, and how well combination chemotherapy with fludarabine, high-dose cytarabine, granulocyte colony-stimulating factor (G-CSF), and idarubicin (FLAG-Ida) followed immediately by reduced-intensity total body radiation therapy, called total body irradiation (TBI), and donor hematopoietic cell transplant (HCT) works in treating adults age 60 and older with newly diagnosed adverse-risk acute myeloid leukemia (AML) or other high-grade myeloid cancer. Despite advances in supportive care and the approval of more than 10 new drugs since 2017, the outcomes of older adults with adverse-risk acute myeloid leukemia and other high-grade myeloid cancers remains poor. Most patients are expected to die from their cancer or the consequences of treatment-related side effects. Donor HCT is a very important part of any curative-cancer treatment for these patients. However, while accepted as standard care for decades, this treatment exposes patients to long periods of drug-induced low blood cell counts and the problems associated with low blood counts, like infections and bleeding, which are associated with significant risk of chronic side effects and death. This study will use a different approach to the upfront curative-cancer treatment of older adults with an adverse-risk AML or other high-grade myeloid cancer. This study will use intense chemotherapy followed a few days later by lower-dose TBI and donor HCT. Chemotherapy drugs, such as idarubicin, fludarabine, high-dose cytarabine 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. G-CSF helps the bone marrow make more white blood cells in patients with low white blood cell count due to cancer treatment. This approach allows effective treatment of cancer cells and overall reduction of the period of low blood cells counts. This decreases the risk for problems associated with low blood counts, such as infection and chronic side effects. Decreasing these are important for older adults who undergo HCT. This treatment strategy may improve treatment outcomes by allowing more patients to successfully undergo donor HCT and reduce the risk of low blood cell counts and the problems associated with low blood counts. Giving chemotherapy followed immediately by reduced-intensity TBI and donor HCT may be safe, tolerable and/or effective in treating adults age 60 and older with newly diagnosed adverse-risk AML or other high-grade myeloid cancer.

Start Date01 Jul 2025

Sponsor / Collaborator

Fred Hutchinson Cancer Research Center

100 Clinical Results associated with Idarubicin Hydrochloride

100 Translational Medicine associated with Idarubicin Hydrochloride

100 Patents (Medical) associated with Idarubicin Hydrochloride

4,149

Literatures (Medical) associated with Idarubicin Hydrochloride

31 Dec 2025·OncoImmunology

Potent immune-dependent anticancer effects of the non-cardiotoxic anthracycline aclarubicin

Review

Author: Sauvat, Allan ; Cerrato, Giulia ; Abdellatif, Mahmoud ; Kroemer, Guido

Aclarubicin (also called aclacinomycin A) is an antineoplastic from the anthracycline class that is used in China and Japan but not in Europe nor in the USA. Aclarubicin induces much less DNA damage than the classical anthracyclines doxorubicin, daunorubicin, epirubicin, idarubicin, and the anthracene mitoxantrone, but is equally effective in inhibiting DNA-to-RNA transcription and in eliciting immunogenic stress in malignant cells. Accordingly, aclarubicin lacks the DNA damage-associated cardiotoxicity that is dose-limiting for classical anthracyclines. Conversely, aclarubicin is at least as potent as other anthracyclines in inducing immunogenic cell death (ICD), which is key for the mode of action of efficient chemotherapeutics. This combination of reduced toxicity and equivalent ICD-stimulatory activity may explain why, as compared to other anthracyclines, aclarubicin is particularly efficient against acute myeloid leukemia. As a result, we advocate for clinical studies seeking to replace the anthracyclines used in Western medicine by aclarubicin-like compounds. Such clinical studies should not only embrace hematological malignancies but should also concern solid cancers, including those in which ICD-inducing chemotherapies are followed by immunotherapies targeting the PD-1/PD-L1 interaction.

01 Aug 2025·INTERNATIONAL JOURNAL OF ANTIMICROBIAL AGENTS

The potential of RNA-binding proteins as host-targeting antivirals against RNA viruses

Review

Author: Panagiotopoulos, Antonios-Periklis ; Siafakas, Nikolaos ; Tsakris, Athanasios ; Anastassopoulou, Cleo

RNA-binding proteins (RBPs) are essential regulators of cellular RNA processes, including RNA stability, translation, and post-translational regulation. During viral infections, RBPs are key regulators of the viral cycle due to their interaction with both host and viral RNAs. Herein, we initially explore the roles of specific RBP families, namely heterogeneous nuclear ribonucleoproteins (hnRNPs), DEAD-box helicases, human antigen R (HuR), and the eukaryotic initiation factors of the eIF4F complex, in viral RNA replication, translation, and assembly. Next, we examine the potential of these RBPs as host-targeting antivirals against pandemic-prone RNA viruses that have been gaining momentum in recent years. Targeting RBPs could disrupt cellular homeostasis, leading to unintended effects on host cells; however, RBPs have been successfully targeted mainly in anticancer therapies, showcasing that their modulation can be safely achieved by drug repurposing. By disrupting key viral-RBP interactions or modulating RBP functions, such therapeutic interventions aim to inhibit viral propagation and restore normal host processes. Thus, conceivable benefits of targeting RBPs as alternative antiviral strategies include their broad-spectrum activity and potential for combination therapies with conventional antivirals, reduced or delayed resistance development, and concomitant enhancement of host immune responses. Our discussion also highlights the broader implications of leveraging host-directed therapies in an attempt to overcome viral resistance. Finally, we emphasise the need for continued innovation to refine these strategies for broad-spectrum antiviral applications.

01 Aug 2025·JOURNAL OF PHARMACEUTICAL AND BIOMEDICAL ANALYSIS

Simultaneous analysis of various anticancer drugs by supercritical fluid chromatography-mass spectrometry. Part I: Optimization of chromatographic separation

Article

Author: Guillarme, Davy ; Rudaz, Serge ; Fleury-Souverain, Sandrine ; Bonnabry, Pascal ; Nguyen, Nathalie

This work presents a generic SFC-MS method for the simultaneous analysis of 22 anticancer drugs (fluorouracil, busulfan, cyclophosphamide, cytarabine, dacarbazine, daunorubicin, docetaxel, doxorubicin, epirubicin, etoposide, gemcitabine, idarubicin, ifosfamide, irinotecan, methotrexate, paclitaxel, pemetrexed, raltitrexed, topotecan, treosulfan, vinblastine, vincristine). The separation conditions were optimized by screening nine stationary phases (2-picolylamine, bare hybrid silica, 2-ethylpyridine, fluoro-phenyl, octadecyl, diethylamine, diol, 1-aminoanthracene, zwitterionic modification), evaluating additives effects (2-5 % water, 20-50 mM ammonium formate, 0-1 mM ammonium fluoride), and adjusting the organic modifier composition (methanol, ethanol, isopropanol, acetonitrile). The optimized SFC-MS method successfully analyzed 22 anticancer drugs, along with 5 additional challenging compounds (azacitidine, mitomycin, cisplatin, oxaliplatin, carboplatin), in 12 min, using a diol column (100 × 3 mm, 1.7 µm) and a gradient of 2-100 % methanol containing 2 % water and 50 mM ammonium formate. To overcome overpressure generated by high organic solvent content, a backpressure gradient (110-150 bar) and a flow rate gradient (0.6-1.5 mL/min) were applied. The diol column was selected as the most promising based on five predefined chromatographic criteria. Additives with 5 % water or ammonium fluoride were excluded due to overpressure and signal loss, respectively. Increasing ammonium formate concentration improved peak symmetry by 29 %. For the organic modifier, pure methanol was chosen since ternary mixtures led to system overpressure without improving separation. Comparison with the LC-MS method using real samples confirmed the potential applicability of the SFC method, as the same trace compounds were detected with comparable concentrations. Sensitivity optimization and method validation will be discussed separately in a later paper.

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

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KEGG	Wiki	ATC	Drug Bank
D01747	Idarubicin Hydrochloride	L01DB06	DB01177

R&D Status

10 top approved records.

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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

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


GIMEMA LAL1913 (EHA2025)	Phase 2	Adult Lymphoblastic Lymphoma	50	Pegaspargase-based treatment	hhxbruzahz(mrrkmsihvf) = A better 5-year DFS was observed in patients ≤40 years (80%) when compared with those >40 (56%) gzowxboynv (djtnorafnl ) View more	Positive	14 May 2025
				High cumulative dose (HCD) steroids
PB2472 (EHA2025)	Not Applicable	Acute Myeloid Leukemia KMT2A-r \| DNMT3A \| TET2 ... View more	-	7+3 chemotherapy	vnlcsxylgk(rewifibjqq) = wrhhxolyav doqvpyclka (atlcfhbpak ) View more	Positive	14 May 2025
NCT02115295 (CLIA, Pubmed \| Cancer)	Phase 2	Relapsing acute myeloid leukemia FLT3‐mutated AML	66	Cladribine 5 mg/m2	uhzlltvbit(jcuinbbamq) = ccprkqyiph tabxbilhwc (lktocgioce ) View more	Positive	15 Apr 2025
				Sorafenib 400 mg	xdesjoopru(deqoycbccs) = ajbpnkpnkq aiebbfrfzo (eqxfcbnvtc ) View more
ASH2024	Not Applicable	-	-	Cladribine, Idarubicin, and Cytarabine (CLIA) + Gilteritinib	bvpgxwtylt(xnxrhqmnog) = n = 5 vsxgrwqqpg (daxgulrnlp ) View more	-	08 Dec 2024
ASH2024 Manual	Not Applicable	Acute Myeloid Leukemia First line	32	Idarubicin + Cytarabine + Venetoclax (IAV regimen)	dvzbuwrfwn(nxljtrsrjy) = iguduqyvia jbswgpxngu (eyqcmyhfxc ) View more	Positive	07 Dec 2024
NCT00876031 (CWS-2007-HR, Pubmed \| EClinicalMedicine)	Phase 3	Rhabdomyosarcoma \| Soft Tissue Sarcoma Maintenance	195	Oral maintenance therapy (Trofosfamide, Idarubicin, Etoposide)	fpbhhpeqiw(axexbkybbi) = ymohrpigkz aqaswbazje (pmgslcjpty, 58.1 - 77.2) View more	Negative	01 Dec 2024
				Standard therapy only	fpbhhpeqiw(axexbkybbi) = dtwmryokwd aqaswbazje (pmgslcjpty, 67.6 - 84.6) View more
NCT02185768 (IDASPHERE II, CTgov)	Phase 2	Hepatocellular Carcinoma	46	Dc- Beads 300-500µm+Idarubicin	xjzgrwwhic(xjuqhvxijr) = himnxwtdmp akxsiijdup (yejjnodjoo, omjvksqmwx - oigbvhugwe) View more	-	15 Oct 2024
NCT00801489 (EHA2024) Manual	Phase 2	Core binding factor acute myeloid leukemia Consolidation \| First line \| Induction RUNX1-RUNX1T1 \| CBFB-MYH11 \| FLT3 ... View more	179	FLAG regimen + gemtuzumab ozogamicin or idarubicin	werptbmxfc(yjstwpueut) = fcyirtngwc cjbitjkjly (mumkqtpxjd ) View more	Positive	14 May 2024
				FLAG-GOgemtuzumab ozogamicin	werptbmxfc(yjstwpueut) = kniimpyxme cjbitjkjly (mumkqtpxjd ) View more
EHA2024	Not Applicable	Relapsing acute myeloid leukemia	-	Cladribine 5 mg/m2 IV	tcjcwxqyns(txbcozmtlw) = 8% wcmdlemkkl (blsnvqelzs ) View more	-	14 May 2024
				Cytarabine 1g/m2 IV
NCT02441803 (CTgov)	Phase 2	Acute Myeloid Leukemia	11	Stem Cell Infusion+Clofarabine+Fludarabine+Busulfan+Decitabine+Cytarabine+Idarubicin (Matched Sibling Donor Group)	mxorvpbdgd = pffufwilak ujxwtqykul (bzxxrfmotn, pbjbnllbnk - djxtfecrwr) View more	-	17 Jan 2024
				Stem Cell Infusion+Clofarabine+Fludarabine+Busulfan+Cyclophosphamide+Decitabine+Tacrolimus+mycophenolate mofetil+Cytarabine+Idarubicin (Haploidentical Donor Group)	mxorvpbdgd = qopmsmvqgh ujxwtqykul (bzxxrfmotn, hbtvdputut - agivbhmcty) View more

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