VISTA-AML- Venetoclax Intensity Selection Trial in AML: A Randomized Phase II Selection Trial of Venetoclax-Based Induction Intensity in Newly Diagnosed Acute Myeloid Leukemia

This randomized, open label, multi arm phase II trial will evaluate the efficacy and safety of venetoclax based induction regimens of varying intensity (VA, VAM, or 2+5+V) versus standard 3+7 in fit patients aged ≥14 years with newly diagnosed AML. The trial is designed to select the optimal regimen as the experimental arm for a subsequent phase III randomized controlled trial.
A total of 320 patients will be enrolled in this study，and segregated into four groups with 80 in each group. Patients who achieve CR/CRi/CRh after using different induction regimens will receive the same consolidation. Allogeneic hematopoietic stem cell transplantation is recommended for patients in the high-risk group or those with persist MRD positivity. After completion of the treatment phase, patients entered the follow-up period.

Start Date30 Sep 2026

Sponsor / Collaborator

Hematology Hospital of Chinese Academy of Medical Sciences

NCT07725705

/ Not yet recruitingPhase 1/2IIT

An Open-label, Multicenter, Phase Ib/II Exploratory Clinical Study of EZH2 Inhibitor SHR2554 in Combination With Liposomal Mitoxantrone for the First-line Treatment of Peripheral T-cell Lymphoma

This is a single-arm, multicenter, Phase Ib/IIa study designed to explore the efficacy and safety of SHR2554 in combination with liposomal mitoxantrone for the treatment of patients with treatment-naive peripheral T-cell lymphoma (PTCL). The study is divided into a Phase Ib safety lead-in phase and a Phase IIa dose expansion phase.

Start Date01 Aug 2026

Sponsor / Collaborator

Hematology Hospital of Chinese Academy of Medical Sciences

ChiCTR2600130735

/ Not yet recruitingPhase 2IIT

A Prospective, Multicenter, Single-Arm Phase II Clinical Study of Mitoxantrone Combined with Cytarabine and Lisaftoclax (MAL Regimen) as Induction Therapy in Newly Diagnosed Fit Adult Patients with Acute Myeloid Leukemia

Start Date01 Apr 2026

Sponsor / Collaborator

The First Affiliated Hospital of Guangzhou University of Traditional Chinese Medicine

100 Clinical Results associated with Mitoxantrone Hydrochloride

100 Translational Medicine associated with Mitoxantrone Hydrochloride

100 Patents (Medical) associated with Mitoxantrone Hydrochloride

5,060

Literatures (Medical) associated with Mitoxantrone Hydrochloride

31 Dec 2026·Virulence

The essential role of heparan sulfate in the entry of PDCoV and other porcine coronaviruses

Article

Author: Zhou, Pei ; Guo, Guanghao ; Lebbink, Robert Jan ; He, Qigai ; Zhu, Hongmei ; Bosch, Berend Jan ; Zhang, Mengjia ; Ma, Ningning ; Zhou, Jiaru ; Li, Wentao ; Wang, Chunyan ; Ghonaim, Ahmed H.

Porcine enteric coronaviruses, including porcine deltacoronavirus (PDCoV), porcine epidemic diarrhea virus (PEDV), swine acute diarrhea syndrome coronavirus (SADS-CoV), and transmissible gastroenteritis coronavirus (TGEV), can cause acute diarrhea, vomiting, dehydration, and high mortality in suckling piglets. Recent studies revealing human PDCoV infections and the potential of SADS-CoV to penetrate human cell lines have heightened apprehensions about the zoonotic transmission risks of these viruses. While heparan sulfate (HS) serves as a receptor in PDCoV binding, the key host genes involved in HS biogenesis and the specific molecular mechanisms underlying this process have not been fully examined. Enzymes involved in HS biosynthesis, including SLC35B2, EXT1, and NDST1, were identified as critical host factors via the use of CRISPR-Cas9 knockout cells. Moreover, inhibition assays using heparin sodium, a competitive HS mimic, demonstrated dose-dependent reductions in PDCoV infection in vitro. Additionally, mitoxantrone, an HS-binding drug, reduced PDCoV infection. Furthermore, HS was confirmed to facilitate the entry of other porcine enteric coronaviruses (SeCoVs), including PEDV, SADS-CoV, and TGEV, underscoring the conserved role of HS in CoV pathogenesis. These insights contribute to the understanding of porcine coronavirus-host interactions and support the development of innovative antiviral interventions.

01 Aug 2026·PHYTOCHEMISTRY

Discovery of spiroindolediketopiperazine alkaloids from Penicillium sp. KYS-32 as dual inhibitors of ABCB1 and ABCG2 to reverse multidrug resistance

Article

Author: Liu, Xiang-Yu ; Zhu, Yu-Lei ; Yue, Jian-Min ; Yu, Jin-Hai ; Li, Ying ; Zhu, Yan-Ping

Chemical investigation of the fungus Penicillium sp. KYS-32 afforded eleven spiroindolediketopiperazine alkaloids (1-11), including seven previously unreported compounds penispiroketins A-G (1-7). Compounds 2, 3, 6, and 7 are characterized by an oxidation at C-12, a structural feature that is exceptionally rare among this chemical family. Their structures were elucidated through a combination of spectroscopic analyses, X-ray crystallography, and ECD calculations. In the first assessment of this chemical class as multidrug resistance (MDR) reversers, compounds 10 and 11 effectively resensitized HCT-15/Taxol cells to both paclitaxel and mitoxantrone. Preliminary structure-activity relationship studies indicated that the N-1 prenyl and C-18 isobutenyl substituents are critical for the observed activity. Subsequent biological studies identified 11 as a dual-target inhibitor that directly binds to and inhibits both ABCB1/P-gp and ABCG2/BCRP transporters. Molecular docking simulations revealed that 11 occupies the substrate-binding pockets of both proteins, forming pivotal hydrogen bonds with Tyr941 in ABCB1 and Thr542 in ABCG2. These findings establish compound 11 as a novel dual inhibitor of ABCB1 and ABCG2, offering a promising chemical scaffold for developing next-generation MDR reversal agents.

01 Aug 2026·PEDIATRIC BLOOD & CANCER

Pharmacokinetics, Efficacy, and Safety Profiles of Mitoxantrone Hydrochloride Liposome Plus Cytarabine in Pediatric Patients With Newly Diagnosed Acute Myeloid Leukemia

Article

Author: Liu, Tianfeng ; Hu, Tianyuan ; Li, Tianyu ; Li, Xiaolan ; Zhu, Xiaofan ; Chen, Xiaojuan ; Zhang, Aoli ; Zhang, Yingchi ; Wang, Shuchun ; Zhang, Ranran ; Guo, Ye ; Liu, Lipeng ; Chen, Yumei ; Qi, Benquan ; Yang, Wenyu

ABSTRACT:

Background:

Mitoxantrone hydrochloride liposome (Lipo‐MIT), an innovative anthracycline nano‐drug, has demonstrated a favorable pharmacokinetics (PK) profile in lymphoma and preliminary efficacy in adult acute myeloid leukemia (AML). This study investigated the PK profile, efficacy, and safety of Lipo‐MIT plus cytarabine in newly diagnosed pediatric AML.

Methods:

In this single‐arm, open‐label trial, eligible patients received Lipo‐MIT and cytarabine every 4 weeks for two cycles. The primary endpoint was the PK characteristics of Lipo‐MIT after the first dose.

Results:

Between July 19, 2023, and November 14, 2023, eight pediatric patients were enrolled. The total and free mitoxantrone concentrations increased rapidly during the absorption phase and then declined gradually to minimal concentrations before the administration of the second dose. The mean elimination half‐life of total mitoxantrone was 60.2 ± 9.4 h, and the ratio of the area under the plasma concentration–time curve from Time 0 to the time of the last quantifiable concentration (AUC 0‐t ) of free mitoxantrone to total mitoxantrone was 1.8% ± 0.6%. The overall response rate and composite complete remission rate were both 85.7% (6/7). Measurable residual disease negativity was achieved in four (66.7%) patients after Cycle 1 and in six (100.0%) after Cycle 2. The 1‐year relapse‐free survival rate was 83.3%. The common Grade ≥3 treatment‐related adverse events were hematological toxicities (100.0%) and soft tissue infection (28.6%). No treatment‐related deaths occurred.

Conclusion:

Lipo‐MIT plus cytarabine showed a favorable PK profile and a manageable safety profile in newly diagnosed pediatric AML, with early evidence of clinical activity in this population.Clinical Trial Registration : Chinese Clinical Trial Registry: ChiCTR2300067964 ( https://www.chictr.org.cn/showproj.html?proj=188738 ).

News (Medical) associated with Mitoxantrone Hydrochloride

09 Dec 2024

·www.prnewswire.com

ADC Therapeutics Announces The Lancet Haematology Publication of Data from Investigator-Initiated Trial Evaluating ZYNLONTA® in Combination with Rituximab to Treat Relapsed/Refractory Follicular Lymphoma

Publication follows oral presentation at the American Society of Hematology (ASH) Annual Meeting Combination in r/r follicular lymphoma shows best ORR of 97% and CR rate of 77% with 12-month PFS of 95% Additional IIT data also presented at ASH on ZYNLONTA monotherapy in marginal zone lymphoma shows ORR of 91% and 70% CR LAUSANNE, Switzerland, Dec. 9, 2024 /PRNewswire/ -- ADC Therapeutics SA (NYSE: ADCT), a commercial-stage global leader and pioneer in the field of antibody drug conjugates (ADCs), today announced updated data from the investigator-initiated Phase 2 clinical trial evaluating ZYNLONTA® (loncastuximab tesirine-lpyl) in combination with rituximab to treat relapsed or refractory (r/r) follicular lymphoma (FL) were published in the December issue of The Lancet Haematology, following an oral presentation of the data at the recent 66th American Society of Hematology (ASH) Annual Meeting and Exposition. "We are excited by the publication of these results in The Lancet Haematology demonstrating ZYNLONTA's robust clinical activity in follicular lymphoma, particularly in patients classified as high-risk POD24 and those with high tumor burden where there remains significant unmet need," said Mohamed Zaki, MD, PhD, Chief Medical Officer of ADC Therapeutics. "In addition, encouraging data from another investigator-initiated trial of ZYNLONTA as a single agent to treat marginal zone lymphoma were also presented at ASH. Collectively, we believe these data underscore ZYNLONTA's promise for patients with indolent B-cell lymphomas and add to a growing body of evidence showing the potential of ZYNLONTA beyond diffuse large B-cell lymphoma." ZYNLONTA in combination with rituximab to treat r/r follicular lymphoma (FL) The investigator-initiated trial conducted at the Sylvester Comprehensive Cancer Center at the University of Miami Miller School of Medicine evaluated the combination in patients with r/r FL treated with ≥1 line of systemic therapy presenting high-disease burden as defined by GELF criteria or POD24 at enrollment. The primary endpoint of the study is complete response rate (CR) by week 12 PET/CT based on Lugano 2014 criteria. The trial enrolled 39 patients, all of which were evaluated for safety and 35 of which were evaluated for efficacy. Patients were a median age of 68 years (range 47 to 89) and the majority received one previous line of therapy (n=26; 67%). R-CHOP was the most common first-line therapy (n=22; 56%) followed by bendamustine with rituximab (n=10; 26%), single-agent rituximab (n=6; 15%) and fludarabine, mitoxantrone and dexamethasone (n=1; 3%). Highlights from the results published in The Lancet Haematology included: Best overall response rate (ORR) of 97.4% (n=38) and CR rate of 76.9% (n=30) After a median follow-up of 15.6 months, the median progression-free survival (PFS) was not reached, and the 12-month PFS was 94.6% The most common treatment-emergent adverse events (TEAEs) were hyperglycemia (n=17; 43.6%) followed by increased alkaline phosphatase (n=16; 41%) and neutropenia, fatigue and increased aspartate aminotransferase and alanine aminotransferase (n=15; 38.5%) The most common grade ≥3 TEAE were lymphopenia (n=8; 20.5%) followed by neutropenia (n=5; 12.9%) No Grade 5 TEAEs occurred. The publication titled, "Loncastuximab tesirine with rituximab in patients with relapsed or refractory follicular lymphoma: a single centre, single arm Phase 2 trial," is now available online and will be published in the December issue of The Lancet Haematology. The results were also presented during a session on indolent B-cell lymphomas at ASH by Juan Pablo Alderuccio, MD, lead investigator and Associate Professor of Medicine and Hematologist at Sylvester. More details on the trial can be found at (identifier: NCT04998669). ZYNLONTA as a single agent to treat r/r marginal zone lymphoma (MZL) Data from an open-label, multi-institutional investigator-initiated trial evaluating the safety and efficacy of ZYNLONTA in 23 adult r/r MZL patients, previously treated with ≥1 line of systemic therapy, were also shared as a poster presentation at ASH by lead investigator, Izidore Lossos, MD, Professor of Medicine and Chief of the Lymphoma Section of the Division of Hematology at the Sylvester Comprehensive Cancer Center, University of Miami. The median age in this study was 65 years (range 45-82). The median number of previous treatments was 2 (range 1 to 4). As of October 15, 2024, 23 patients were evaluable for response. Highlights from the results presented include: ORR of 91% (n=21); 70% CR (n=16). ZYNLONTA led to CR in 7 of 11 patients (64%) with POD24 assessed for response and one patient who progressed after CAR-T. All but 1 CR are currently maintained with the longest follow-up of 27 months from the start of treatment (median duration of CR is 11.5 months). All of the 23 enrolled patients experienced expected adverse events (AE), most commonly grade 1 or 2. Grade 3 and 4 AEs were observed in 15 and 1 (neutropenia) patients, respectively. Local edema was observed in 10 (43.4%) patients. Three patients needed dose reduction and one patient discontinued treatment after cycle 4 because of cholestatic hepatitis. The patient clinically fully recovered with normalization in liver function test abnormalities. More details on this ongoing Phase 2 clinical trial can be found at (identifier: NCT05296070). About ZYNLONTA® (loncastuximab tesirine-lpyl) ZYNLONTA® is a CD19-directed antibody drug conjugate (ADC). Once bound to a CD19-expressing cell, ZYNLONTA is internalized by the cell, where enzymes release a pyrrolobenzodiazepine (PBD) payload. The potent payload binds to DNA minor groove with little distortion, remaining less visible to DNA repair mechanisms. This ultimately results in cell cycle arrest and tumor cell death. The U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) have approved ZYNLONTA (loncastuximab tesirine-lpyl) for the treatment of adult patients with relapsed or refractory (r/r) large B-cell lymphoma after two or more lines of systemic therapy, including diffuse large B-cell lymphoma (DLBCL) not otherwise specified (NOS), DLBCL arising from low-grade lymphoma and also high-grade B-cell lymphoma. The trial included a broad spectrum of heavily pre-treated patients (median three prior lines of therapy) with difficult-to-treat disease, including patients who did not respond to first-line therapy, patients refractory to all prior lines of therapy, patients with double/triple hit genetics and patients who had stem cell transplant and CAR-T therapy prior to their treatment with ZYNLONTA. This indication is approved by the FDA under accelerated approval and in the European Union under conditional approval based on overall response rate and continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial. Please see full prescribing information including important safety information about ZYNLONTA at . ZYNLONTA is also being evaluated as a therapeutic option in combination studies in other B-cell malignancies and earlier lines of therapy. About ADC Therapeutics ADC Therapeutics (NYSE: ADCT) is a commercial-stage global leader and pioneer in the field of antibody drug conjugates (ADCs). The Company is advancing its proprietary ADC technology to transform the treatment paradigm for patients with hematologic malignancies and solid tumors. ADC Therapeutics' CD19-directed ADC ZYNLONTA (loncastuximab tesirine-lpyl) received accelerated approval by the FDA and conditional approval from the European Commission for the treatment of relapsed or refractory diffuse large B-cell lymphoma after two or more lines of systemic therapy. ZYNLONTA is also in development in combination with other agents and in earlier lines of therapy. In addition to ZYNLONTA, ADC Therapeutics has multiple ADCs in ongoing clinical and preclinical development. ADC Therapeutics is based in Lausanne (Biopôle), Switzerland, and has operations in London and New Jersey. For more information, please visit the Company website at adctherapeutics.com and follow us on LinkedIn. ZYNLONTA® is a registered trademark of ADC Therapeutics SA. Forward-Looking Statements This press release contains forward-looking statements within the meaning of the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. In some cases you can identify forward-looking statements by terminology such as "may", "will", "should", "would", "expect", "intend", "plan", "anticipate", "believe", "estimate", "predict", "potential", "seem", "seek", "future", "continue", or "appear" or the negative of these terms or similar expressions, although not all forward-looking statements contain these identifying words. Forward-looking statements are subject to certain risks and uncertainties that can cause actual results to differ materially from those described. Factors that may cause such differences include, but are not limited to: whether the interim results for the investigator-initiated trials led by the University of Miami studying FL and MZL are predictive of future results and the timing and success of the same IIT trials, the potential regulatory and/or compendia strategy and the future opportunity for FL and MZL; the expected cash runway into mid-2026 the Company's ability to grow ZYNLONTA® revenue in the United States; the ability of our partners to commercialize ZYNLONTA® in foreign markets, the timing and amount of future revenue and payments to us from such partnerships and their ability to obtain regulatory approval for ZYNLONTA® in foreign jurisdictions; the timing and results of the Company's or its partners' research and development projects or clinical trials including LOTIS 5 and 7, ADCT 602 as well as early research in certain solid tumors with different targets, linkers and payloads; the timing and outcome of regulatory submissions for the Company's products or product candidates; actions by the FDA or foreign regulatory authorities; projected revenue and expenses; the Company's indebtedness, including Healthcare Royalty Management and Blue Owl and Oaktree facilities, and the restrictions imposed on the Company's activities by such indebtedness, the ability to comply with the terms of the various agreements and repay such indebtedness and the significant cash required to service such indebtedness; and the Company's ability to obtain financial and other resources for its research, development, clinical, and commercial activities. Additional information concerning these and other factors that may cause actual results to differ materially from those anticipated in the forward-looking statements is contained in the "Risk Factors" section of the Company's Annual Report on Form 10-K and in the Company's other periodic and current reports and filings with the U.S. Securities and Exchange Commission. These statements involve known and unknown risks, uncertainties and other factors that may cause actual results, performance, achievements or prospects to be materially different from any future results, performance, achievements or prospects expressed in or implied by such forward-looking statements. The Company cautions investors not to place undue reliance on the forward-looking statements contained in this document. CONTACTS: Investors Marcy Graham ADC Therapeutics [email protected] +1 650-667-6450 Media Nicole Riley ADC Therapeutics [email protected] +1 862-926-9040 SOURCE ADC Therapeutics SA WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

$ADC Therapeutics Announces The Lancet Haematology Publication of Data from Investigator-Initiated Trial Evaluating ZYNLONTA® in Combination with Rituximab to Treat Relapsed/Refractory Follicular Lymphoma$

Clinical ResultPhase 2ASHDrug ApprovalImmunotherapy

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 Mitoxantrone Hydrochloride

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KEGG	Wiki	ATC	Drug Bank
D02166	Mitoxantrone Hydrochloride	L01DB07	DBSALT000121

R&D Status

Approved

10 top approved records.

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Indication	Country/Location	Organization	Date
Locally advanced breast cancer	Australia	Baxter Healthcare Pty Ltd.	24 Jan 2001
Myeloid Leukemia	Australia	Baxter Healthcare Pty Ltd.	24 Jan 2001
Non-Hodgkin Lymphoma	Australia	Baxter Healthcare Pty Ltd.	24 Jan 2001
Cancer Pain	United States	Serono SA	13 Oct 2000
Multiple Sclerosis, Relapsing-Remitting	United States	Serono SA	13 Oct 2000
Castration-Resistant Prostatic Cancer	United States	EMD Serono, Inc.	13 Nov 1996
Acute Myeloid Leukemia	United States	EMD Serono, Inc.	13 Nov 1996
Hepatocellular Carcinoma	Japan	ASKA Pharmaceutical Co., Ltd.	01 Jun 1989
Metastatic breast cancer	United States	EMD Serono, Inc.	23 Dec 1987
Leukemia	Japan	ASKA Pharmaceutical Co., Ltd.	02 Oct 1987
Lymphoma	Japan	ASKA Pharmaceutical Co., Ltd.	02 Oct 1987
Breast Cancer	Japan	ASKA Pharmaceutical Co., Ltd.	02 Oct 1987

Developing

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Locally Advanced Gastric Adenocarcinoma	Phase 3	China	Benxi Economic Development Zone Kangmaisi Pharmaceutical Co., Ltd.	05 Jul 2023
Locally Advanced Gastric Adenocarcinoma	Phase 3	China	Shenyang Pharmaceutical University	05 Jul 2023
stomach adenocarcinoma	Phase 3	China	Benxi Economic Development Zone Kangmaisi Pharmaceutical Co., Ltd.	05 Jul 2023
stomach adenocarcinoma	Phase 3	China	Shenyang Pharmaceutical University	05 Jul 2023
Stomach Cancer	Phase 3	China	Shenzhen China Resources Jiuchuang Pharmaceutical Co., Ltd.	27 Jun 2023
Invasive Mammary Carcinoma	Phase 3	China	Benxi Economic Development Zone Kangmaisi Pharmaceutical Co., Ltd.	11 Dec 2019
Invasive Mammary Carcinoma	Phase 3	China	Shenyang Pharmaceutical University	11 Dec 2019
Thyroid Cancer	Phase 3	China	Shenyang Pharmaceutical University	07 Nov 2017
Thyroid Cancer	Phase 3	China	Benxi Economic Development Zone Kangmaisi Pharmaceutical Co., Ltd.	07 Nov 2017
Indolent Non-Hodgkin Lymphoma	Phase 3	United States	Genentech, Inc.	16 Mar 1998

Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


ChiCTR1800015883 (Pubmed \| Blood)	Phase 3	Acute Myeloid Leukemia	224	Low-dose cytarabine, mitoxantrone or idarubicin, and granulocyte colony-stimulating factor	dwjayxxafn(kigldtimci) = jqmhthdxjc ffpwnjskuw (qzbmsxrveh ) View more	Non-inferior	02 Jul 2026
ChiCTR1800015883 (Pubmed \| Blood)	Phase 3	Acute Myeloid Leukemia	224	Cytarabine, daunomycin, and etoposide	dwjayxxafn(kigldtimci) = jlopxdkxox ffpwnjskuw (qzbmsxrveh ) View more	Non-inferior	02 Jul 2026
ASCO2026	Not Applicable	Acute Myeloid Leukemia	16	HiDAC/mitoxantrone/venetoclax (HMV)	jpgahhjsbl(yzyozhclax) = ezcaxdlqqq qiyrfnxkrk (qdjtyrkzue ) View more	Positive	29 May 2026
NCT02583893 (CTgov)	Phase 2	Adult Acute Myeloblastic Leukemia	39	Etoposide+Sirolimus+Mitoxantrone+Cytarabine	tocrdjzgfz = tqgmnxuvjt nalpvnkrzn (bekbeghqfg, boxamomarm - dbqnmjpied) View more	-	29 May 2026
EHA2026	Not Applicable	Refractory acute myeloid leukemia	59	FLAG-Mitox-Ven	xtmvhsqtbh(wvesuafwxm) = oxvifglpvm zttqnwqmvr (oquzhvdhoi ) View more	Positive	12 May 2026
EHA2026	Not Applicable	Mixed phenotype acute leukemia	16	MIT-LIP combined chemotherapy regimen	dhmjuryqzx(ktbldpaxwu) = The main adverse effects were hematological toxicities. The incidence of agranulocytosis was 100%, the median duration time was 9 days (ranging from 2 to 18 days). The incidences of grade 4, grade 3, and grade 2 thrombocytopenia were 85.3%, 8.8%, and 5.9%, respectively. Only 4 cases (11.8%) of grade 3 transaminase elevation were observed, and no other obvious organ toxicity was observed. gselymfxlq (xgmhytpsyi ) View more	Positive	12 May 2026
EHA2026	Not Applicable	Refractory acute myeloid leukemia	128	High-dose cytarabine+mitoxantrone+venetoclax	edepfuhmoi(mzwebhqvcz) = wfvfkvzzlf vwkrwemynl (zhrvvmrvtg ) View more	Positive	12 May 2026
EHA2026	Not Applicable	Refractory acute myeloid leukemia	128	High-dose cytarabine+mitoxantrone+venetoclax (Allogeneic HCT recipient)	edepfuhmoi(mzwebhqvcz) = erxszlketa vwkrwemynl (zhrvvmrvtg ) View more	Positive	12 May 2026
EHA2026	Not Applicable	Immunoblastic Lymphadenopathy TET2 \| RHOA \| DNMT3A ... View more	9	MINE regimen + targeted agents	vmejhfrwpz(yjwaztwuek) = xsrxcqaenp rbmnwkroen (radwekcbhx ) View more	Positive	12 May 2026
ChiCTR2500104950 (EHA2026)	Phase 2	Refractory acute myeloid leukemia	16	Selinexor + Decitabine + Dose-Adjusted CMG	zxchzgduwk(osleowaafh) = mujzuuqjhf gimenpbceu (xpkxzmevvv ) View more	Positive	12 May 2026
ChiCTR2500104950 (EHA2026)	Phase 2	Refractory acute myeloid leukemia	16	Selinexor + Decitabine + Dose-Adjusted CMG (early salvage therapy (1-2 prior lines))	zxchzgduwk(osleowaafh) = ralrsbmcds gimenpbceu (xpkxzmevvv ) View more	Positive	12 May 2026
ASH2025	Not Applicable	Acute Myeloid Leukemia	105	FLAG-mitoxantrone combined with low-dose venetoclax (7 Days) (newly diagnosed)	notinqsduj(pbfjnkaluq) = The most frequent adverse events (grade ≥3) were hematologic, infectious (pneumonia), and gastrointestinal. uzqqmozzyy (hnmsydkyoc ) View more	Positive	06 Dec 2025
ASH2025	Not Applicable	Acute Myeloid Leukemia	105	FLAG-mitoxantrone combined with low-dose venetoclax (7 Days) (relapsed/refractory)		Positive	06 Dec 2025
NCT01842672 (Pubmed \| EClinicalMedicine)	Phase 1/2	Recurrent Acute Leukemia	40	Clofarabine 35 mg/m^2/day	yzxruuzvek(vawhoruciy) = oqdacwtcom lgeeqgvpca (hujpcsumhv ) View more	Positive	01 May 2025

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