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Last update 27 Dec 2025

Cytarabine

Last update 27 Dec 2025

Overview

R&D Status

Clinical Result

Translational Medicine

Overview

Basic Info

Drug Type

Small molecule drug

Synonyms

1-beta-D-Arabinofuranosylcytosine, 4-Amino-1-beta-D-arabinofuranosyl-2(1H)-pyrimidinone, AC

+ [35]

Target

DNA-directed DNA polymerase

Action

inhibitors

Mechanism

DNA-directed DNA polymerase inhibitors, DNA synthesis inhibitors

Therapeutic Areas

NeoplasmsImmune System DiseasesDigestive System Disorders+ [6]

Active Indication

Blast Phase Chronic Granulocytic LeukemiaLymphomaAcute Lymphoblastic Leukemia+ [17]

Inactive Indication

Acute Erythroblastic LeukemiaAcute Megakaryoblastic LeukemiaAcute Monocytic Leukemia+ [28]

Originator Organization

Teva Pharmaceutical Industries Ltd.

Active Organization

Nippon Shinyaku Co., Ltd.

Libbs Farmacêutica Ltda.

Amgen, Inc.

+ [3]

Inactive Organization

Teva Pharmaceutical Industries Ltd.

Millennium Pharmaceuticals, Inc.Pacira Ltd.

+ [7]

License Organization

Enzon Pharmaceuticals, Inc.

Sigma-Tau Industrie Farmaceutiche Riunite SpA

Drug Highest PhaseApproved

First Approval Date

United States (17 Jun 1969),

Acute Lymphoblastic LeukemiaLeukemiaPhiladelphia chromosome positive chronic myelogenous leukemia

RegulationAccelerated Approval (United States), Orphan Drug (United States), Orphan Drug (Japan), Priority Review (China)

Structure/Sequence

Molecular FormulaC9H13N3O5

InChIKeyUHDGCWIWMRVCDJ-CCXZUQQUSA-N

CAS Registry147-94-4

1,406

Clinical Trials associated with Cytarabine

NCT07227584

/ Not yet recruitingPhase 2IIT

Treatment of Newly Diagnosed Philadelphia Chromosome-Negative Acute Lymphoblastic Leukemia in Adolescents and Young Adults (AYAs)

The goal of this research study is to evaluate a chemotherapy regiment for the treatment of newly diagnosed Philadelphia chromosome-negative acute lymphoblastic leukemia (ALL) in adolescents and young adults (AYAs).
The names of the study drugs involved in this study are:
* blinatumomab (a type of immunotherapy drug)
* cyclophosphamide (a type of chemotherapy drug)
* cytarabine (a type of antineoplastic agent)
* dexamethasone (a type of synthetic glucocorticoid)
* doxorubicin (a type of antineoplastic agent)
* etoposide (a type of antineoplastic agent)
* mercaptopurine (a type of antineoplastic agent)
* methotrexate (a type of chemotherapy drug)
* pegaspargase (a type of antineoplastic agent)
* vincristine (a type of antineoplastic agent)

Start Date01 Apr 2026

Sponsor / Collaborator

Dana-Farber Cancer Institute, Inc.

NCT07072585

/ Not yet recruitingPhase 2/3IIT

A Phase 2/3 Randomized Trial Investigating Daratumumab on a Modified Augmented BFM (aBFM) Backbone in Newly Diagnosed T-Lymphoblastic Leukemia (T-ALL) and T-Lymphoblastic Lymphoma (T-LL)

This phase II/III trial tests the addition of daratumumab to chemotherapy for treating patients with newly-diagnosed T-ALL and T-LL. Daratumumab is in a class of medications called monoclonal antibodies. It binds to a protein called CD38, which is found on some types of immune cells and cancer cells. Daratumumab may block CD38 and help the immune system kill cancer cells. Chemotherapy drugs 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. Giving chemotherapy with daratumumab may kill more cancer cells.

Start Date30 Mar 2026

Sponsor / Collaborator

The Children's Oncology Group Foundation, Inc.

NCT05554419

/ Not yet recruitingPhase 2IIT

Eradicating Measurable Residual Disease in Patients With Acute Myeloid Leukemia (AML) Prior to StEm Cell Transplantation (ERASE): A MyeloMATCH Treatment Trial

This phase II MyeloMATCH treatment trial compares cytarabine versus (vs.) cytarabine and venetoclax vs. liposome-encapsulated daunorubicin-cytarabine and venetoclax vs. azacitidine and venetoclax for treating patients who have residual disease after treatment for acute myeloid leukemia (AML). Cytarabine is in a class of medications called antimetabolites. It works by slowing or stopping the growth of cancer cells in the body. 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. Liposome-encapsulated daunorubicin-cytarabine is a drug formulation that delivers daunorubicin and cytarabine in small spheres called liposomes, which may make the drugs safer or more effective. Azacitidine is a drug that interacts with DNA and leads to the activation of tumor suppressor genes, which are genes that help control cell growth. This study may help the study doctors find out if the different drug combinations are equally effective to the usual approach of cytarabine alone while requiring a shorter duration of treatment. To decide if they are better, the study doctors will be looking to see if the study drugs lead to a higher percentage of patients achieving a deeper remission compared to cytarabine alone.

Start Date01 Mar 2026

Sponsor / Collaborator

National Cancer Institute

100 Clinical Results associated with Cytarabine

100 Translational Medicine associated with Cytarabine

100 Patents (Medical) associated with Cytarabine

22,992

Literatures (Medical) associated with Cytarabine

01 Jan 2026·Neural Regeneration Research

Empowering the NSC-34 cell line as a motor neuron model: Cytosine arabinoside’s action

Article

Author: Vitale, Giuseppe ; Amadio, Susanna ; Liguori, Francesco ; Volonté, Cinzia

JOURNAL/nrgr/04.03/01300535-202601000-00039/figure1/v/2025-03-30T110608Z/r/image-tiff The NSC-34 cell line is a widely recognized motor neuron model and various neuronal differentiation protocols have been exploited. Under previously reported experimental conditions, only part of the cells resemble differentiated neurons; however, they do not exhibit extensive and time-prolonged neuritogenesis, and maintain their duplication capacity in culture. The aim of the present work was to facilitate long-term and more homogeneous neuronal differentiation in motor neuron–like NSC-34 cells. We found that the antimitotic drug cytosine arabinoside promoted robust and persistent neuronal differentiation in the entire cell population. Long and interconnecting neuronal processes with abundant growth cones were homogeneously induced and were durable for up to at least 6 weeks in culture. Moreover, cytosine arabinoside was permissive, dispensable, and mostly irreversible in priming NSC-34 cells for neurite initiation and regeneration after mechanical dislodgement. Finally, the expression of the cell proliferation antigen Ki67 was inhibited by cytosine arabinoside, whereas the expression levels of neuronal growth associated protein 43, vimentin, and motor neuron–specific p75, Islet2, homeobox 9 markers were upregulated, as confirmed by western blot and/or confocal immunofluorescence analysis. Overall, these findings support the use of NSC-34 cells as a motor neuron model for properly investigating neurodegenerative mechanisms and prospectively identifying neuroprotective strategies.

31 Dec 2025·DRUG DELIVERY

Biomaterial-based drug delivery: evaluating the safety profiles of liposomal Vyxeos

Article

Author: Liu, Zhicheng ; Fu, Zhuo ; Liu, Yahui ; Jiao, Yan

Vyxeos, a liposomal combination of cytarabine and daunorubicin, has improved survival outcomes for patients with high-risk acute myeloid leukemia (AML). However, its safety profile in real-world settings requires comprehensive evaluation. This study aims to assess the adverse event profiles associated with Vyxeos using data from the U.S. FDA's Adverse Event Reporting System (FAERS). A retrospective analysis of adverse event reports from the FAERS database was conducted for Vyxeos from January 2017 to June 2024. Reports were analyzed to assess patient demographics, system organ classes (SOCs), and preferred terms (PTs). Signal detection analysis was performed using disproportionality metrics, including Reporting Odds Ratio (ROR), Proportional Reporting Ratio (PRR), Bayesian Confidence Propagation Neural Network (BCPNN), and Multi-Item Gamma Poisson Shrinker (MGPS). A total of 1,036 reports were analyzed. The most frequently reported adverse events were hematologic (37.73%), infectious (28.42%), and cardiac disorders (13.22%). Febrile neutropenia, neutropenic sepsis, and pneumonia fungal were the most commonly reported events, with febrile neutropenia showing a strong association (ROR = 92.18). Males had a higher frequency of infectious events, while females reported more cardiac events. Most adverse events occurred within 30 days of treatment initiation, and 16.92% of reports involved hospitalization, while 18.33% reported death. Vyxeos is associated with significant hematologic, infectious, and cardiac adverse events. Close monitoring, infection prophylaxis, and cardiac assessments are recommended for patients receiving Vyxeos. Further research is needed to validate these findings and explore the mechanisms underlying the observed toxicities.

31 Dec 2025·ANNALS OF MEDICINE

Intermediate-dose cytarabine alone versus combination in consolidation therapy for non-transplant acute myeloid leukemia: a retrospective study

Article

Author: Liu, Yu ; Wang, Zhihui ; Chen, Tairan ; Deng, Xiangping ; He, Qiulian ; Zhou, Lixin ; Zou, Xingli ; Long, Kejie

BACKGROUND:

Cytarabine (Ara-C) is a cornerstone of acute myeloid leukaemia (AML) treatment, particularly in consolidation therapy. Although high-dose cytarabine (HDAC) has been widely adopted for consolidation in AML, intermediate-dose cytarabine (IDAC) is increasingly favoured due to its comparable efficacy and improved tolerability. However, the potential benefit of combining another agent with IDAC during consolidation therapy has yet to be adequately validated.

PATIENTS:

This retrospective study analyzed 204 newly diagnosed adult AML patients who underwent at least two cycles of consolidation therapy with either IDAC monotherapy (N = 84) or IDAC combined with anthracyclines/homoharringtonine (IDAC-plus, N = 120) after complete remission (CR) or complete remission with incomplete count recovery (CRi).

RESULTS:

At a median follow-up of 26.5 months, IDAC-plus significantly improved relapse-free survival (RFS) compared to IDAC monotherapy (HR 0.62, p = 0.019). Although overall survival (OS) was not significantly different, the IDAC-plus group showed a survival advantage (HR, 0.68; p = 0.11). Subgroup analysis identified younger patients (HR = 0.43, p = 0.01), male patients (HR = 0.51, p = 0.03), patients with better performance status (HR = 0.52, p = 0.01), lactate dehydrogenase (LDH) >2 × upper limit of normal (ULN) (HR 0.51, p = 0.01), intermediate-risk disease (HR= 0.51 p = 0.01), measurable residual disease (MRD) positivity at CR/CRi (HR = 0.48, p = 0.03) and patients without hyperleukocytosis (HR = 0.64, p = 0.04), can have a more pronounced benefit from IDAC-plus consolidation chemotherapy. IDAC-plus group had more neutropenia (grade ≥3) (p = 0.032), febrile neutropenia (p = 0.039) and documented infection (p = 0.028), but non-relapse mortality (NRM) was similar between the two groups (p = 0.985).

CONCLUSION:

The findings suggest that IDAC-based combination consolidation chemotherapy reduces the risk of relapse in non-transplant AML compared to IDAC alone, particularly in patients with intermediate-risk disease or MRD positivity at the time of achieving CR/CRi, underscoring the need for intensified regimens in these populations.

315

News (Medical) associated with Cytarabine

17 Dec 2025

·moleculin.com

Moleculin Announces Positive Results from Phase 1 Clinical Trial Evaluating WP1066 for the Treatment of Pediatric Recurrent Malignant Brain Tumors

Emory University physician-sponsored clinical trial conducted at the Aflac Cancer and Blood Disorders Center at Children’s Healthcare of Atlanta Results demonstrated WP1066 induces anti-tumor immune responses and were recently published in the Journal of Clinical Investigation Insight WP1066 found to be safe and effective, warranting a Phase 2 trial HOUSTON, Dec. 17, 2025 (GLOBE NEWSWIRE) — Moleculin Biotech, Inc., (Nasdaq: MBRX) (“Moleculin” or the “Company”), today announced positive results from the Emory University physician-sponsored Phase 1 clinical trial conducted at the Aflac Cancer and Blood Disorders Center at Children’s Healthcare of Atlanta led by pediatric oncologist, Tobey MacDonald, MD, who discovered STAT3 is critical to certain childhood brain tumors and currently serves as Professor of Pediatrics and Director of the Pediatric Neuro-Oncology Program. WP1066 is Moleculin’s flagship Immune/Transcription Modulator designed to stimulate the immune response to tumors by inhibiting the errant activity of regulatory T cells while also inhibiting key oncogenic transcription factors, including p-STAT3 (phosphorylated signal transducer and activator of transcription 3), c-Myc (oncogene driving many aggressive cancers) and HIF-1α (hypoxia-inducible factor 1α). These transcription factors are widely sought targets because of their role in cancer cell survival and proliferation, angiogenesis (coopting vasculature for blood supply), invasion, metastasis, and inflammation associated with tumors. “The results of this first-in-child trial show some encouraging signals of activity in a highly aggressive chemotherapy resistant brain cancer, such as partial tumor response in a diffuse intrinsic pontine glioma (DIPG) patient and clear anti-tumor immune changes,” said Dr. MacDonald. For the Phase 1 trial, 10 children were treated with WP1066 twice daily for 14 days to determine the maximum feasible dose. Compassionate use treatment in three children with high-grade glioma was also evaluated. Results showed there was no significant toxicity, and a maximum feasible dose was determined. Importantly, WP1066 suppressed the expression of STAT3, inhibiting its activity and demonstrating anti-tumor immune responses. While the preclinical efficacy of WP1066 had been previously demonstrated, and its effectiveness had been studied among adults, this trial was the first to test it in children. Dr. MacDonald and his team recruited pediatric patients with high-grade glioma, which include diffuse midline glioma (DMG) and DIPG, and account for most pediatric brain tumor-related deaths. Both DMG and DIPG have an average survival rate of nine to 11 months following diagnosis. Additionally, patients with relapsed medulloblastoma and ependymoma who have no accepted standard therapy following a relapse were also included in the study. Thus, Dr. MacDonald’s research helps fill a critical need to identify new treatment options for patients with these incurable tumors. Mr. Walter Klemp, Chairman and CEO of Moleculin, “We are very encouraged by the positive results from our Phase 1 clinical trial evaluating WP1066 in children with recurrent malignant brain tumors. These early data show that WP1066 has the ability to activate meaningful anti-tumor immune responses, an important proof of mechanism in a patient population with extremely limited treatment options. While still early, these findings reinforce the potential of WP1066 as a novel immunomodulatory approach for some of the most difficult-to-treat pediatric brain cancers. We are deeply grateful to Dr. MacDonald and his team, and the patients and families who participated in this study. We look forward to the continued advancement of WP1066 as a potential therapy that can make a real difference for children facing these devastating diseases.” Results from the study were recently published in the Journal of Clinical Investigation Insight. For more information about the WP1066 Phase 1 clinical trial, visit clinicaltrials.gov and reference identifier NCT04334863. The results of this study are the foundation of the Phase 2 concept Dr. MacDonald’s team would like to pursue in a follow-up trial. The current study was funded by Peach Bowl LegACy Fund and CURE Childhood Cancer. About Moleculin Biotech, Inc. Moleculin Biotech, Inc. is a Phase 3 clinical stage pharmaceutical company advancing a pipeline of therapeutic candidates addressing hard-to-treat tumors and viruses. The Company’s lead program, Annamycin, is a next-generation highly efficacious and well tolerated anthracycline designed to avoid multidrug resistance mechanisms and to lack the cardiotoxicity common with currently prescribed anthracyclines. Annamycin is currently in development for the treatment of relapsed or refractory acute myeloid leukemia (AML) and soft tissue sarcoma (STS) lung metastases. The Company has begun the MIRACLE (Moleculin R/R AML AnnAraC Clinical Evaluation) Trial (MB-108), a pivotal, adaptive design Phase 3 trial evaluating Annamycin in combination with cytarabine, together referred to as AnnAraC (the combination of Annamycin and cytarabine, also referred to as “Ara-C”) and, for the treatment of relapsed or refractory acute myeloid leukemia. Following a successful Phase 1B/2 study (MB-106), with input from the FDA, the Company believes it has substantially de-risked the development pathway towards a potential approval for Annamycin for the treatment of AML. This study remains subject to appropriate future filings with potential additional feedback from the FDA and their foreign equivalents. Additionally, the Company is developing WP1066, an Immune/Transcription Modulator capable of inhibiting p-STAT3 and other oncogenic transcription factors while also stimulating a natural immune response, targeting brain tumors, pancreatic and other cancers. Moleculin also has in its pipeline a portfolio of antimetabolites, including WP1122 for the potential treatment of pathogenic viruses, as well as certain cancer indications. For more information about the Company, please visit www.moleculin.com and connect on X, LinkedIn and Facebook. Forward-Looking Statements Some of the statements in this release are forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, Section 21E of the Securities Exchange Act of 1934 and the Private Securities Litigation Reform Act of 1995, which involve risks and uncertainties. Forward-looking statements in this press release include, without limitation, statements regarding the continued recruitment, treatment, and receipt of the unblinded data for the first 45 subjects of the MIRACLE clinical trial and the timing to complete Part A of the MIRACLE trial with up to 90 patients, each as described above. Moleculin will require significant additional financing, for which the Company has no commitments, in order to conduct its clinical trials as described in this press release, and the milestones described in this press release assume the Company’s ability to secure such financing on a timely basis. Although Moleculin believes that the expectations reflected in such forward-looking statements are reasonable as of the date made, expectations may prove to have been materially different from the results expressed or implied by such forward-looking statements. Moleculin has attempted to identify forward-looking statements by terminology including ‘believes,’ ‘estimates,’ ‘anticipates,’ ‘expects,’ ‘plans,’ ‘projects,’ ‘intends,’ ‘potential,’ ‘may,’ ‘could,’ ‘might,’ ‘will,’ ‘should,’ ‘approximately’ or other words that convey uncertainty of future events or outcomes to identify these forward-looking statements. These statements are only predictions and involve known and unknown risks, uncertainties, and other factors, including those discussed under Item 1A. “Risk Factors” in our most recently filed Form 10-K filed with the Securities and Exchange Commission (SEC) and updated from time to time in our Form 10-Q filings and in our other public filings with the SEC. Any forward-looking statements contained in this release speak only as of its date. We undertake no obligation to update any forward-looking statements contained in this release to reflect events or circumstances occurring after its date or to reflect the occurrence of unanticipated events. Investor Contact:JTC Team, LLCJenene Thomas(908) 824-0775MBRX@jtcir.com

Phase 1Phase 2Phase 3Clinical ResultImmunotherapy

15 Dec 2025

·fibrogen.gcs-web.com

Roxadustat Granted Orphan Drug Designation for the Treatment of Myelodysplastic Syndromes by the U.S. Food and Drug Administration

Company is on track to file the Phase 3 protocol in the fourth quarter of 2025 SAN FRANCISCO, Dec. 15, 2025 (GLOBE NEWSWIRE) -- FibroGen, Inc. (NASDAQ: FGEN) today announced that the Office of Orphan Products Development of the U.S. Food and Drug Administration (FDA) has granted roxadustat Orphan Drug Designation for the treatment of myelodysplastic syndromes (MDS). “The Orphan Drug Designation granted to roxadustat for MDS underscores the significant treatment gap in this indication, and highlights patients’ need for additional convenient treatments that can provide durable response,” said Thane Wettig, Chief Executive Officer of FibroGen. “Roxadustat showed an improvement in transfusion-independence in a subset of patients with high transfusion burden in a post-hoc analysis from the Phase 3 MATTHERHORN trial, which along with its favorable tolerability profile and oral route of administration has the ability to set it apart from current second-line treatments. Our team is finalizing the Phase 3 protocol in this patient population for submission to the FDA in the fourth quarter of 2025.” There are approximately 58,000 patients diagnosed with LR-MDS in the U.S. with 85% of them suffering from anemia. Anemia in patients with MDS is associated with increased risk of cardiovascular complications and the need for blood transfusions. Transfusion-dependent patients suffer higher rates of complications and decreased quality of life. Current first-line treatments lead to transfusion independence in less than 50% of patients and relief is often temporary with limited options for second line and beyond treatments. In a post-hoc analysis from the Phase 3 MATTERHORN trial, roxadustat demonstrated transfusion independence benefits compared to placebo in patients with high transfusion burden. The FDA Orphan Drug Designation is granted to drugs intended for the treatment, diagnosis, or prevention of rare diseases that affect fewer than 200,000 people in the U.S. Benefits of the designation may include exemption from certain FDA fees, financial incentives for qualified clinical development, and seven years of market exclusivity in the U.S. following drug approval. About Myelodysplastic Syndromes Anemia Myelodysplastic syndromes (MDS) are a group of disorders characterized by dysfunctional progenitor blood cells and stem cells, resulting in chronic anemia in most patients. Annual incidence rates of MDS are estimated to be 4.9/100,000 adults in the U.S, of which 77% are considered lower-risk MDS. Approximately 80% of patients with MDS have anemia at the time of diagnosis, and around 60% of patients with MDS will experience severe anemia (hemoglobin <8 g/dL) at some point during the course of their disease. Anemia in patients with MDS is associated with increased risk of cardiovascular complications and the need for blood transfusion. Approximately 50% of patients with MDS require regular red blood cell transfusions. Transfusion-dependent MDS patients suffer higher rates of cardiac events, infections, and iron overload with the related complications. In addition, anemia frequently leads to significant fatigue, cognitive dysfunction, and decreased quality of life. Today, patients are routinely treated with erythropoiesis-stimulating agents (ESAs), luspatercept, imetelstat, or lenalidomide in lower-risk MDS with isolated del(5q), and hypomethylating agents (HMAs) in higher-risk disease. Only 35-40% of patients respond to current treatments and the durability of response is short. Moreover, these treatments are challenging to dose-calibrate and can only be administered via subcutaneous injection or through IV infusion. There remains a high unmet need for the treatment of anemia associated with MDS, and new strategies that provide durable response and the convenience of oral administration are highly desired in managing patients with MDS. About Roxadustat Roxadustat, an oral medication, is the first in a new class of medicines comprising HIF-PH inhibitors that promote erythropoiesis, or red blood cell production, through increased endogenous production of erythropoietin, improved iron absorption and mobilization, and downregulation of hepcidin. Roxadustat is approved in Europe, Japan, and numerous other countries for the treatment of anemia of CKD in adult patients on dialysis (DD) and not on dialysis (NDD). FibroGen has the sole rights to roxadustat in the United States, Canada, Mexico, and in all markets not held by AstraZeneca or licensed to Astellas. Astellas and FibroGen are collaborating on the commercialization of roxadustat for the treatment of anemia in territories including Japan, Europe, Turkey, Russia, and the Commonwealth of Independent States, the Middle East, and South Africa. About FibroGen FibroGen, Inc. is a biopharmaceutical company focused on development of novel therapies at the frontiers of cancer biology and anemia. Roxadustat (爱瑞卓®, EVRENZOTM) is currently approved in Europe, Japan, and numerous other countries for the treatment of anemia in chronic kidney disease (CKD) patients on dialysis and not on dialysis. The Company continues to evaluate the development plan for the Phase 3 trial of roxadustat in anemia associated with lower-risk myelodysplastic syndrome (LR-MDS) in the U.S. FG-3246 (also known as FOR46), a first-in-class antibody-drug conjugate (ADC) targeting CD46, is in Phase 2 development for the treatment of metastatic castration-resistant prostate cancer. This program also includes the development of FG-3180, an associated CD46-targeted PET biomarker. For more information, please visit www.fibrogen.com. Forward-Looking Statements This release contains forward-looking statements regarding FibroGen’s strategy, future plans and prospects, including statements regarding its commercial products and clinical programs and those of it and its collaboration partners Fortis and UCSF. These forward-looking statements include, but are not limited to, statements regarding the efficacy, safety, convenience, and potential clinical or commercial success of FibroGen products and product candidates, statements about regulatory interactions, and statements about FibroGen’s plans and objectives. These forward-looking statements are typically identified by use of terms such as “may,” “will”, “should,” “on track,” “could,” “expect,” “plan,” “anticipate,” “believe,” “estimate,” “predict,” “potential,” “continue” and similar words, although some forward-looking statements are expressed differently. FibroGen’s actual results may differ materially from those indicated in these forward-looking statements due to risks and uncertainties related to the continued progress and timing of its various programs, including the enrollment and results from ongoing and potential future clinical trials, and other matters that are described in FibroGen’s most recent Annual Report on Form 10-K and Quarterly Report on Form 10-Q, each as filed with the Securities and Exchange Commission (SEC), including the risk factors set forth therein. Investors are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date of this release, and FibroGen undertakes no obligation to update any forward-looking statement in this press release, except as required by law. For Investor Inquiries: David DeLucia, CFA Senior Vice President and Chief Financial Officer ir@fibrogen.com Source: FibroGen, Inc.

Phase 2Phase 3Orphan DrugDrug ApprovalClinical Result

10 Dec 2025

·moleculin.com

Moleculin Announces Exercise of Warrants for $6.8 Million Gross Proceeds

HOUSTON, Dec. 10, 2025 (GLOBE NEWSWIRE) — Moleculin Biotech, Inc., (Nasdaq: MBRX) (“Moleculin” or the “Company”), a late-stage pharmaceutical company with a broad portfolio of drug candidates targeting hard-to-treat tumors and viruses, today announced it has entered into agreements with certain holders of its existing warrants for the immediate exercise of certain outstanding warrants to purchase up to an aggregate of 727,969 shares of common stock of the Company originally issued in February 2025 at an exercise price of $6.63 per share and 316,360 shares of common stock of the Company originally issued in August 2025 at an exercise price of $6.3219 per share. The shares of common stock issuable upon exercise of the outstanding warrants are registered pursuant to effective registration statements on Form S-1 (File No. 333-286276) and Form S-3 (File No. 333-290418). The aggregate gross proceeds from the exercise of the existing warrants is expected to total approximately $6.5 million, before deducting financial advisory fees. Roth Capital Partners and Maxim Group LLC are acting as the Company’s financial advisors for this transaction. In consideration for the immediate exercise of the warrants for cash, the Company will issue new unregistered warrants to purchase shares of common stock. The new warrants will be exercisable for an aggregate of up to 2,610,823 shares of common stock, at an exercise price of $6.63 per share and will be exercisable upon shareholder approval and for a term of five years from the date of shareholder approval. The transaction is expected to close on or about December 11, 2025, subject to satisfaction of customary closing conditions. The Company intends to use the net proceeds from the offering for working capital and general corporate purposes. The new warrants described above were offered in a private placement pursuant to an applicable exemption from the registration requirements of the Securities Act of 1933, as amended (the “1933 Act”) and, along with the shares of common stock issuable upon their exercise, have not been registered under the 1933 Act, and may not be offered or sold in the United States absent registration with the Securities and Exchange Commission (“SEC”) or an applicable exemption from such registration requirements. The Company has agreed to file a registration statement with the SEC covering the resale of the shares of common stock issuable upon exercise of the new warrants. This press release shall not constitute an offer to sell or a solicitation of an offer to buy nor shall there be any sale of these securities in any state or jurisdiction in which such offer, solicitation or sale would be unlawful prior to the registration or qualification under the securities laws of any such state or jurisdiction. About Moleculin Biotech, Inc. Moleculin Biotech, Inc. is a Phase 3 clinical stage pharmaceutical company advancing a pipeline of therapeutic candidates addressing hard-to-treat tumors and viruses. The Company’s lead program, Annamycin, is a next-generation highly efficacious and well tolerated anthracycline designed to avoid multidrug resistance mechanisms and to lack the cardiotoxicity common with currently prescribed anthracyclines. Annamycin is currently in development for the treatment of relapsed or refractory acute myeloid leukemia (AML) and soft tissue sarcoma (STS) lung metastases. The Company has begun the MIRACLE (Moleculin R/R AML AnnAraC Clinical Evaluation) Trial (MB-108), a pivotal, adaptive design Phase 3 trial evaluating Annamycin in combination with cytarabine, together referred to as AnnAraC (the combination of Annamycin and cytarabine, also referred to as “Ara-C”) and, for the treatment of relapsed or refractory acute myeloid leukemia. Following a successful Phase 1B/2 study (MB-106), with input from the FDA, the Company believes it has substantially de-risked the development pathway towards a potential approval for Annamycin for the treatment of AML. This study remains subject to appropriate future filings with potential additional feedback from the FDA and their foreign equivalents. Additionally, the Company is developing WP1066, an Immune/Transcription Modulator capable of inhibiting p-STAT3 and other oncogenic transcription factors while also stimulating a natural immune response, targeting brain tumors, pancreatic and other cancers. Moleculin also has in its pipeline a portfolio of antimetabolites, including WP1122 for the potential treatment of pathogenic viruses, as well as certain cancer indications. Forward-Looking Statements Some of the statements in this release are forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, Section 21E of the Securities Exchange Act of 1934 and the Private Securities Litigation Reform Act of 1995, which involve risks and uncertainties. Forward-looking statements in this press release include, without limitation, the closing of the offering and the use of the proceeds thereof. Although Moleculin believes that the expectations reflected in such forward-looking statements are reasonable as of the date made, expectations may prove to have been materially different from the results expressed or implied by such forward-looking statements. Moleculin has attempted to identify forward looking statements by terminology including ‘believes,’ ‘estimates,’ ‘anticipates,’ ‘expects,’ ‘plans,’ ‘projects,’ ‘intends,’ ‘potential,’ ‘may,’ ‘could,’ ‘might,’ ‘will,’ ‘should,’ ‘approximately’ or other words that convey uncertainty of future events or outcomes to identify these forward-looking statements. These statements are only predictions and involve known and unknown risks, uncertainties, and other factors, including those discussed under Item 1A. “Risk Factors” in our most recently filed Form 10-K filed with the Securities and Exchange Commission (SEC) and updated from time to time in our Form 10-Q filings and in our other public filings with the SEC. Any forward-looking statements contained in this release speak only as of its date. We undertake no obligation to update any forward-looking statements contained in this release to reflect events or circumstances occurring after its date or to reflect the occurrence of unanticipated events. Investor Contact:JTC Team, LLCJenene Thomas(908) 824-0775MBRX@jtcir.com

Phase 3

100 Deals associated with Cytarabine

External Link

KEGG	Wiki	ATC	Drug Bank
D00168	Cytarabine	L01BC01	DB00987

R&D Status

Approved

10 top approved records.

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Indication	Country/Location	Organization	Date
Blast Phase Chronic Granulocytic Leukemia	Brazil	Libbs Farmacêutica Ltda.	08 Jan 2007
Lymphoma	Japan	Nippon Shinyaku Co., Ltd.	11 Nov 2002
Meningeal Carcinomatosis	European Union	Pacira Ltd.	11 Jul 2001
Meningeal Carcinomatosis	Iceland	Pacira Ltd.	11 Jul 2001
Meningeal Carcinomatosis	Liechtenstein	Pacira Ltd.	11 Jul 2001
Meningeal Carcinomatosis	Norway	Pacira Ltd.	11 Jul 2001
Acute Myeloid Leukemia	Japan	Nippon Shinyaku Co., Ltd.	18 Jan 2000
Bladder Cancer	Japan	Nippon Shinyaku Co., Ltd.	15 Feb 1984
Bile Duct Neoplasms	Japan	Nippon Shinyaku Co., Ltd.	24 Apr 1973
Breast Cancer	Japan	Nippon Shinyaku Co., Ltd.	24 Apr 1973
Colonic Cancer	Japan	Nippon Shinyaku Co., Ltd.	24 Apr 1973
Gallbladder Neoplasms	Japan	Nippon Shinyaku Co., Ltd.	24 Apr 1973
Liver Cancer	Japan	Nippon Shinyaku Co., Ltd.	24 Apr 1973
Lung Cancer	Japan	Nippon Shinyaku Co., Ltd.	24 Apr 1973
Ovarian Cancer	Japan	Nippon Shinyaku Co., Ltd.	24 Apr 1973
Pancreatic Cancer	Japan	Nippon Shinyaku Co., Ltd.	24 Apr 1973
Rectal Cancer	Japan	Nippon Shinyaku Co., Ltd.	24 Apr 1973
Stomach Cancer	Japan	Nippon Shinyaku Co., Ltd.	24 Apr 1973
Uterine Neoplasms	Japan	Nippon Shinyaku Co., Ltd.	24 Apr 1973
Acute Lymphoblastic Leukemia	United States	Teva Parenteral Medicines, Inc.	17 Jun 1969

Developing

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Philadelphia chromosome positive adult precursor acute lymphoblastic leukemia	Phase 2	United States	Takeda Pharmaceutical Co., Ltd.	01 Jan 2024
Acute Promyelocytic Leukemia	Phase 2	United States	Pfizer Inc.	14 Sep 2018
Myeloid Tumor	Phase 2	United States	Pfizer Inc.	14 Sep 2018
Aggressive-Phase Chronic Myelocytic Leukemia	Phase 2	United States	Amgen, Inc.	29 Nov 2017
Aggressive-Phase Chronic Myelocytic Leukemia	Phase 2	United States	Takeda Pharmaceutical Co., Ltd.	29 Nov 2017
Philadelphia positive acute lymphocytic leukaemia	Phase 2	United States	Takeda Pharmaceutical Co., Ltd.	29 Nov 2017
Philadelphia positive acute lymphocytic leukaemia	Phase 2	United States	Amgen, Inc.	29 Nov 2017
Recurrent Childhood Acute Lymphoblastic Leukemia	Phase 2	United States	Takeda Pharmaceutical Co., Ltd.	29 Nov 2017
Recurrent Childhood Acute Lymphoblastic Leukemia	Phase 2	United States	Amgen, Inc.	29 Nov 2017
Refractory Adult Acute Lymphoblastic Leukemia	Phase 2	United States	Amgen, Inc.	29 Nov 2017

Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


ASH2025	Not Applicable	Acute Myeloid Leukemia	111	cytarabine (High-intensity consolidation (HIC))	sxzodafqmp(dhzcelxwmw) = qxtpxvnyhk eusqybnzqa (gyjqezekcn ) View more	Positive	06 Dec 2025
				cytarabine (Low intensity consolidation (LIC))	sxzodafqmp(dhzcelxwmw) = sfwftpwfmu eusqybnzqa (gyjqezekcn ) View more
ASH2025	Not Applicable	Acute Myeloid Leukemia Consolidation	2,621	High-dose cytarabine (HiDAC)	oqzeuprwuy(jkymcvurgn): HR = 1.04 (95.0% CI, 0.82 - 1.32), P-Value = 0.75 View more	Positive	06 Dec 2025
				Intermediate-dose cytarabine (IDAC)
ASH2025	Not Applicable	Acute Myeloid Leukemia Induction	795	Daunorubicin and Cytarabine (DA)	qjgwjnyztd(akpqegolaw) = asxfhfkeiz ybnpmgupsp (ualujmesyv ) View more	Positive	06 Dec 2025
				Cytarabine, Daunorubicin, and Etoposide (ADE)	qjgwjnyztd(akpqegolaw) = xyazklgbzg ybnpmgupsp (ualujmesyv ) View more
ASH2025	Phase 1/2	-	20	Low-dose cytarabine+blinatumomab	vqjhdissim(tiwpvztdsc) = qmlvenyzsw razeubrvul (blgceukqjp ) View more	Positive	06 Dec 2025
ASH2025	Not Applicable	Acute myeloid leukemia with mutated NPM1 NPM1 mutation	53	FLAI regimen	pcdiwnmgno(npddpufgez) = vsecinifny yboellejec (ogikhpkbzc ) View more	Positive	06 Dec 2025
NCT02115295 (ASH2025)	Phase 2	Acute Myeloid Leukemia First line KMT2A rearrangement	22	Cladribine, Idarubicin, Cytarabine (CLIA)	jbebqrveaz(watnfacvdo) = vbcxenrxos qnmhnvyxws (dswjdldqxu ) View more	Positive	06 Dec 2025
				Cladribine, Idarubicin, Cytarabine + Venetoclax (CLIA-Ven)	jbebqrveaz(watnfacvdo) = aleoaftaao qnmhnvyxws (dswjdldqxu ) View more
ASH2025	Not Applicable	Core binding factor acute myeloid leukemia	875	FLAG-IDA	dxjpzxvxbw(gcfjzziuin) = zqgvgvvsvd cnecxavbio (hlvovlrexj ) View more	Positive	06 Dec 2025
				DAE	dxjpzxvxbw(gcfjzziuin) = mwoumjddep cnecxavbio (hlvovlrexj ) View more
ASH2025	Phase 2	Acute Myeloid Leukemia	101	Decitabine priming + Low-dose Idarubicin + Cytarabine + G-CSF	kzibtyswzy(rxrrphbqxs) = jbhqmlkxiy mflbulbpmn (yclaklwemv, 64.6 - 81.9) View more	Positive	06 Dec 2025
ASH2025	Phase 2	Multiple Myeloma \| Lymphoma First line	65	Etoposide+cytarabine+pegfilgrastim (EAP) regimen	dkgvafclui(pcggmdbpht) = fubvmsxgrt cgbqrbgdtk (drppurvbdj ) View more	Positive	06 Dec 2025
ASH2025	Not Applicable	Lymphoma	37	TEAM regimen (thiotepa, etoposide, cytarabine, and melphalan)	hmbmtciwbi(wolcgpuqck) = xndrpyrtpf lxbyntfkmm (ofvhjjlitn ) View more	Positive	06 Dec 2025

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