Request Demo

Last update 09 Dec 2025

Temozolomide

Last update 09 Dec 2025

Overview

R&D Status

Clinical Result

Translational Medicine

Overview

Basic Info

Drug Type

Small molecule drug

Synonyms

3,4-dihydro-3-methyl-4-oxoimidazo(5,1-d)-1,2,3,5-tetrazine-8-carboxamide, 3,4-dihydro-3-methyl-4-oxoimidazo(5,1-d)-as-tetrazine-8-carboxamide, 3-methyl-4-oxo-3,4-dihydroimidazo(5,1-d)(1,2,3,5)tetrazine-8-carboxamide

+ [28]

Target

DNA

Action

inhibitors

Mechanism

DNA inhibitors(DNA inhibitors), DNA alkylating agents

Therapeutic Areas

NeoplasmsNervous System DiseasesSkin and Musculoskeletal Diseases+ [10]

Active Indication

Ewing SarcomaMalignant glioma of brainMalignant melanoma, metastatic+ [81]

Inactive Indication

Acoustic Neuromaacute leukemiaAcute myeloid leukaemia with 11q23 abnormality+ [104]

Originator Organization

Merck Sharp & Dohme Corp.

Active Organization

Novartis AGSun Pharmaceutical Industries (Europe) BV

The Children's Oncology Group Foundation, Inc.

+ [30]

Inactive Organization

Hexal AG

Duke University

Novartis Pharmaceuticals Canada, Inc.

+ [37]

License Organization

Double Bond Pharmaceutical International AB

Institut Gustave Roussy EURL

Belarusian State University

+ [1]

Drug Highest PhaseApproved

First Approval Date

European Union (26 Jan 1999),

AstrocytomaGlioblastoma MultiformeGlioma

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

Structure/Sequence

Molecular FormulaC6H6N6O2

InChIKeyBPEGJWRSRHCHSN-UHFFFAOYSA-N

CAS Registry85622-93-1

1,093

Clinical Trials associated with Temozolomide

NCT04373785

/ Not yet recruitingPhase 1/2

A Multi-Center Pilot/Phase I and Phase II Clinical Trial of NG101m Adjuvant Therapy in Newly Diagnosed Glioblastoma Patients

The purpose of this clinical trial is to evaluate the addition of NG101m adjuvant therapy to standard of care treatment of glioblastoma multiforme. All subjects will receive NG101m capsules along with the standard treatment of temozolomide and radiation.

Start Date01 Jan 2027

Sponsor / Collaborator

NeuGATE Theranostics

NCT01777919

/ Not yet recruitingPhase 2IIT

A PHASE II CLINICAL TRIAL FOR THE EVALUATION OF THE EFFICACY OF DISULFIRAM/COPPER COMBINATION AS AN ADJUVANT AND CONCURRENT CHEMOTHERAPY IN THE TREATMENT OF NEWLY DIAGNOSED GLIOBLASTOMA MULTIFORM

Glioblastoma multiform (GBM) is the most common malignant primary brain tumor in adults. Despite maximal treatment tumor relapse occurs regularly accompanied by unfavourable prognosis. Among other reasons, it is believed that this could be in part due to the existence of the so-called tumor stem cells (TSCs), a cellular subfraction within GBM which escape therapy by being highly resistant to irradiation and chemotherapy and thus constituting the source of tumor recurrence.
GBM, like many other cancers, show a sub-population of aldehyde dehydrogenase (ALDH) overexpressing TSCs. More specifically, ALDH1A1, a cytoplasmatic isoform of ALDH, proved to be a novel stem cell marker in human GBM. In addition, ALDH1A1 has been shown to be a mediator for resistance of GBM to temozolomide (TMZ) and a reliable predictor of clinical outcome; prognosis of patients with a high level of ALDH1A1 expression was poor compared with that of patients with low levels. Consequently, ALDH1A1 may serve as a potential target to improve treatment of human GBM through inhibition of the enzyme.
Disulfiram (DSF) has been used for more than sixty years in the treatment of chronic alcoholism because of the unpleasant symptoms it provokes after ethanol intake. The underlying mechanism is believed to be the accumulation of acetaldehyde in the blood, due to inhibition of the liver ALDHs. Actually, DSF is a strong inhibitor of ALDH1A1 and relatively non-toxic at therapeutic (for chronic alcoholism) doses that can penetrate the blood-brain barrier. In addition, DSF has been shown to be cytotoxic on GBM stem-like cells, inhibiting the growth of TMZ resistant GBM cells and blocking self-renewal by
100% , while it has been identified as an inhibitor of human GBM stem cells in high-throughput chemical screens. Interestingly, a number of these actions were copper-dependent.
In the current Phase II clinical trial, DSF/copper combination will be tested as an adjunctive and concurrent chemotherapy in the treatment of newly diagnosed GBM. According to our hypothesis, initiation of DSF chemotherapy after the resection of the tumor and before the introduction of the standard radio-chemotherapy will inhibit ALDH1A1 of GBM TSCs making them more susceptible to radio-chemotherapy and possibly reducing the recurrence rate of GBM. On the other hand, the addition of copper will probably enhance the cytotoxic effects of DSF possibly through augmentation of its pro-apoptotic and proteasomal inhibitory actions.

Start Date01 Jan 2027

Sponsor / Collaborator

Universität Ulm

[+2]

NCT07120984

/ Not yet recruitingPhase 2

A Phase II Study to Evaluate the Safety and Efficacy of L19TNF With Alkylating Chemotherapy for Patients With Recurrent IDH-mutant Astrocytoma or Oligodendroglioma

The purpose of this study is to explore the safety and efficacy of the antibody-cytokine fusion protein L19TNF alone or in combination with alkylating chemotherapy in patients with recurrent IDH mutant glioma.

Start Date30 Jan 2026

Sponsor / Collaborator

Philogen SpA

100 Clinical Results associated with Temozolomide

100 Translational Medicine associated with Temozolomide

100 Patents (Medical) associated with Temozolomide

13,505

Literatures (Medical) associated with Temozolomide

01 Jan 2026·JOURNAL OF NEURO-ONCOLOGY

Circadian variation in MGMT promoter methylation and expression predicts sensitivity to temozolomide in glioblastoma

Article

Author: Kim, Albert H ; Walch, Olivia J ; Herzog, Erik D ; Butt, Omar H ; Huang, Yitong ; Ruben, Marc D ; Simon, Tatiana ; Rubin, Joshua B ; Gonzalez-Aponte, Maria F ; Leidig, William A

Abstract:

Purpose:

Recent studies show that glioblastoma (GBM) is more sensitive to temozolomide (TMZ) in the morning. In cells, inhibiting O6-Methylguanine-DNA-Methyltransferase ( MGMT ) abolished time-dependent TMZ efficacy, suggesting that circadian regulation of this DNA repair enzyme underlies daily TMZ sensitivity. Here, we tested the hypotheses that MGMT promoter methylation and protein abundance vary with time-of-day in GBM, resulting in daily rhythms in TMZ efficacy.

Methods:

We assessed daily rhythms in MGMT promoter methylation in GBM in vitro and retrospectively analyzed MGMT methylation status in human GBM biopsies collected at different times of day. Next, we measured MGMT and BMAL1 protein abundances in GBM cells collected at four-hour intervals. To understand the therapeutic implications of circadian variations in MGMT, we incorporated its daily rhythms into an in vitro mathematical model capturing interactions between MGMT, TMZ, and GBM DNA.

Results:

We found daily rhythms in MGMT promoter methylation and protein levels in GBM in vitro, and in patient biopsies peaking at midday. Further, MGMT protein levels peaked at CT4, corresponding to the time of maximal TMZ efficacy in vitro. When we incorporated cell-intrinsic circadian rhythms in MGMT protein into a mathematical model for GBM chemotherapy, we found that dosing when daily MGMT levels peaked and began to decline produced maximum DNA damage.

Conclusion:

Our findings suggest that the likelihood of diagnosis of MGMT promoter methylation may vary with time of biopsy in GBM. Furthermore, theoretical modeling predicts that efforts to deliver TMZ after the daily peak of MGMT activity, with exact time being dose-dependent, may significantly enhance its therapeutic efficacy.

Graphical Abstract:

01 Jan 2026·JOURNAL OF NEURO-ONCOLOGY

Investigating glioblastoma chemoresistance: a meta-analysis of microRNA signatures and gene networks

Review

Author: Aftab, Kiran ; Enam, Syed Ather ; Guo, Gao ; Habib, Amyn A ; Mughal, Nouman ; Bajwa, Mohammad Hamza ; Amin, Wajiha ; Sufiyan, Sufiyan

BACKGROUND:

Chemoresistance is a significant issue in glioblastoma (GBM) treatment due to recurrence, poor survival and limited salvage options. Non-invasive biomarkers can identify early chemoresistance. These cohorts may benefit from initiating early chemotherapy or second-line drugs at an earlier timeline.

METHODS:

Meta-analysis of identified miR through systematic review was conducted through data mining and miR-target interaction compilation in validated miR databases. We curated a list of gene targets that underwent pathway enrichment analysis using the EnrichR tool. Network analysis of pathways and protein-protein interactions was conducted using the Metascape bioinformatics tool with several ontology sources.

RESULTS:

Significantly upregulated miR validated in chemoresistant GBM were miR-221, -222, -132, and - 125b. 33 miR are identified in the high expression category. 18 miR were low expression responders in TMZ-resistant GBM. MiR-21 and miR-10b are the most replicated miR for bevacizumab response assessment with a low expression in Avastin-resistant GBM. 5 miR with high expression and 11 miR with low expression were identified; two combined micro-RNA signatures predicted cohorts likely to respond well to bevacizumab therapy. MiRs upregulated in TMZ resistance showed 400 predicted mRNA targets and 411 genes in downregulated miR. For bevacizumab response, upregulated miR yielded 38 genes and 82 in downregulated miR. An integrated, mechanistic model linking these miR to downstream targets and pathways is presented.

CONCLUSIONS:

miR evaluation of GBM chemo-resistance requires insights into molecular subtypes and key signatures associated with TMZ/BVZ treatment failure. Evaluating a compiled signature's efficacy in improving progression-free and overall survival can highlight the impact of miR in GBM response evaluation. Our meta-analysis provides a template for understanding the key pathways and targets involved in miR-regulation of chemo-resistant GBM and potential applications of our signature in clinical practice.

01 Jan 2026·COLLOIDS AND SURFACES B-BIOINTERFACES

Tumor exosome-based drug delivery system targeting ferroptosis and apoptosis for glioblastoma therapy

Article

Author: Han, Bo ; Xu, Yechang ; Ming, Yang ; Peng, Tao ; Tang, Qian ; Liang, Lijun ; Liu, Yingchao ; Chen, Daquan ; Han, Wenhui

Temozolomide (TMZ) is a first-line chemotherapy drug to treat glioblastoma (GBM), but still encounters blood-brain barrier (BBB), and chemotherapy resistance. To address these issues, sensible combination therapy of TMZ with other novel therapeutic techniques is required. Herein, we developed a tumor cell exosome membrane (EM) based drug delivery platform (EMNPs@TMZ) for encapsulating reactive oxygen species (ROS) responsive fucoidan (Fuc) nanoparticles (FNPs). EMNPs@TMZ system can co-deliver TMZ and luteolin (Lut) for the combined targeting apoptosis and ferroptosis. Interestingly, Lut can induce ferroptosis by inhibiting Nrf2 expression, and also inhibit DNA repair through Wnt/β-catenin, promoting TMZ induced cell apoptosis. More importantly, this nanoparticle drug delivery system has tumor dual-targeting effect, which enhances the inhibitory effect on glioma growth, thereby improving the survival rate of mice models, and has no significant side effects during the treatment process, particularly in the orthotopic patient-derived xenograft (PDX) model. This biomimetic nanoparticle provides a promising strategy for ferroptosis combination chemotherapy for GBM therapy.

350

News (Medical) associated with Temozolomide

03 Dec 2025

·www.globenewswire.com

Black Diamond Therapeutics Announces Preliminary Phase 2 Data for Silevertinib in 1L NSCLC and Plans for a Phase 2 Trial of Silevertinib in GBM

Silevertinib delivers robust anti-tumor activity as demonstrated by an ORR of 60% and a CNS response rate of 86% in 43 1L NSCLC patients presenting with 35 different non-classical EGFR mutations; no new safety signals observed to datePFS data for 1L NSCLC patients expected in Q2 2026; Company continues to explore partnership opportunities for pivotal development of silevertinibBased on encouraging CNS activity of silevertinib in multiple trials across NSCLC and GBM, Company plans to initiate a randomized Phase 2 trial of silevertinib in newly diagnosed GBM patients in 1H 2026; initial data anticipated in 2028Cash, cash equivalents, and investments of $135.5 million as of September 30, 2025; expected to be sufficient to fund operations into 2H of 2028Company to host an investor webcast and conference call today at 8:00 AM ET CAMBRIDGE, Mass., Dec. 03, 2025 (GLOBE NEWSWIRE) -- Black Diamond Therapeutics, Inc. (Nasdaq: BDTX), a clinical-stage oncology company developing MasterKey therapies that target families of oncogenic mutations in patients with cancer, today announced topline data from its Phase 2 trial of silevertinib in frontline (1L) non-small cell lung cancer (NSCLC) patients with non-classical epidermal growth factor receptor (EGFR) mutations (NCMs) and outlined plans for a randomized Phase 2 trial of silevertinib in patients with newly diagnosed glioblastoma (ND GBM). “We are pleased to share these initial data in frontline NSCLC patients showing silevertinib’s activity against a broad spectrum of 35 distinct non-classical EGFR mutations,” said Mark Velleca, M.D., Ph.D., President and Chief Executive Officer of Black Diamond Therapeutics. “We are particularly encouraged by the CNS activity of silevertinib in treating NSCLC patients with brain metastases, as published data clearly demonstrate that CNS metastases are a key factor in early disease progression for NCM NSCLC patients treated with second- and third-generation EGFR-TKIs. We also believe that silevertinib is uniquely positioned as a potential treatment for patients with newly diagnosed EGFR-altered GBM, and plan to initiate a randomized Phase 2 trial in the first half of 2026, while PFS data matures in our Phase 2 NSCLC study and we continue our partnering discussions.” Silevertinib Phase 2 1L NSCLC Initial Clinical Results and Program Update 43 frontline NSCLC patients harboring a broad spectrum of 35 distinct non-classical EGFR mutations were enrolled, including 16 patients with brain metastases (7 of whom had measurable CNS target lesions). All patients were enrolled at a 200mg oral daily dose of silevertinib. Efficacy and safety were assessed with a November 3, 2025 data cutoff; median follow-up time as of this date was 7.2 months and the study remains ongoing. Key data highlights include: For the 43 patients enrolled, preliminary efficacy data is as follows: 25 confirmed partial responses, 1 confirmed complete response60% Objective Response Rate (ORR by RECIST 1.1)86% CNS ORR (by RANO-BM)91% disease control rate (DCR) Initial duration of treatment data: 29 patients remain on therapy (5/29 after progression), longest ongoing for >19 months Summary of safety data: No new safety signals observedAdverse events (AEs) experienced by a majority of patients include rash, stomatitis, diarrhea and paronychiaAEs were managed with standard supportive care and dose interruptions/reductions without compromising response depth or durability to date The Company expects to present updated results from the Phase 2 NSCLC trial, including Duration of Response (DOR) and Progression-free Survival (PFS) data in both the recurrent (83 patients) and frontline (43 patients) settings, at a medical meeting in the second quarter of 2026. Black Diamond continues to explore potential partnerships to advance silevertinib into pivotal development. “These highly encouraging data speak to the potential of silevertinib to be the treatment of choice for frontline NSCLC patients with the full spectrum of non-classical EGFR mutations” said Sergey Yurasov, M.D., Ph.D., Black Diamond’s Chief Medical Officer. “We are struck by the compelling CNS response rate, which may translate to prolonged durability of response for patients with CNS metastases. Based on these data, and promising Phase 0/1 and Phase 1 GBM results, we are preparing to initiate a randomized Phase 2 trial of silevertinib in newly diagnosed GBM patients, one of the highest unmet needs in oncology.” GBM Program Update and Phase 2 PlansApproximately 50% of patients with glioblastoma (GBM) present with an oncogenic EGFR alteration that can be targeted by silevertinib; each year approximately 7,000 patients in the U.S. are diagnosed with GBM harboring these EGFR alterations. “Prior attempts to treat EGFR-altered GBM patients have been limited by poor brain penetrance of targeted therapies and/or lack of potency of these therapies on the full spectrum of EGFR alterations” said Elizabeth Buck, Ph.D., Chief Scientific Officer of Black Diamond. “Based on encouraging CNS activity demonstrated by silevertinib across multiple trials, and its preclinical potency on all EGFR alterations found in GBM, we believe that silevertinib has the potential to be the first targeted therapy for these patients.” Black Diamond plans to initiate a randomized Phase 2 trial in newly diagnosed GBM patients in the first half of 2026, with preliminary data expected in 2028. Key trial highlights include: Expected to enroll approximately 150 newly diagnosed patients, randomized to receive TMZ (control arm) or silevertinib + TMZ (experimental arm)Initial focus will be on EGFRvIII-positive patients (approximately 30% of GBM) who are MGMT-negative (unmethylated)Randomization and treatment will begin after patients have had their surgical resection and radiationPrimary endpoint is PFS (RANO by Blinded Independent Committee Review), with an interim analysis; secondary endpoint is overall survival (OS)Trial will be governed by an Independent Data Monitoring Committee (IDMC) Updated Financial Guidance Black Diamond previously reported cash, cash equivalents and investments of approximately $135.5 million as of September 30, 2025, which the Company now believes is sufficient to fund its anticipated operating expenses and capital expenditure requirements into the second half of 2028.Financial guidance assumes Black Diamond funds the Phase 2 trial of silevertinib in ND GBM and a potential partner funds pivotal development in NSCLC. Financial guidance does not assume receipt of potential development milestones from the Company’s partnership with Servier Pharmaceuticals LLC for BDTX-4933 (now S241656). Conference Call Information Black Diamond will host a conference call and webcast on Wednesday, December 3, 2025, at 8:00 AM ET to discuss the preliminary Phase 2 data for silevertinib in 1L NSCLC and plans for a Phase 2 trial of silevertinib in GBM. The webcast may be accessed online here or by visiting the Events page in the Investors section of the Company’s website at www.blackdiamondtherapeutics.com. A replay of the webcast will be available for 30 days on the Investors section of Black Diamond’s website. About Black Diamond Therapeutics Black Diamond Therapeutics is a clinical-stage oncology company developing MasterKey therapies that target families of oncogenic mutations in patients with cancer. The Company’s MasterKey therapies are designed to address a broad spectrum of genetically defined tumors, overcome resistance, minimize wild-type mediated toxicities, and be brain penetrant to treat central nervous system disease. The Company is advancing silevertinib, a brain-penetrant fourth-generation EGFR MasterKey inhibitor targeting EGFR-mutant NSCLC and GBM. For more information, please visit www.blackdiamondtherapeutics.com. From time to time, we may use our website or our LinkedIn profile at www.linkedin.com/company/black-diamond-therapeutics to distribute material information. Our financial and other material information is routinely posted to and accessible on the Investors section of our website, available at www.blackdiamondtherapeutics.com. Investors are encouraged to review the Investors section of our website because we may post material information on that site that is not otherwise disseminated by us. Information that is contained in and can be accessed through our website or our LinkedIn page is not incorporated into, and does not form a part of, this press release. Forward-Looking Statements Statements contained in this press release regarding matters that are not historical facts are “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Such statements include, but are not limited to, statements regarding: the continued development and advancement of silevertinib, including the ongoing Phase 2 clinical trial and the timing of clinical updates for silevertinib in patients with NSCLC and in patients with GBM, enrollment in the investigator sponsored Phase 0/1 clinical trial of silevertinib of newly diagnosed GBM patients with EGFR alterations, the expected timing for regulatory feedback and the disclosure of a potential registrational pathway for silevertinib in NSCLC, the potential of silevertinib to address the unmet medical need for newly diagnosed NSCLC patients with non-classical EGFR mutations and benefit patients with NSCLC across multiple lines of therapy, the potential future development plans for silevertinib in NSCLC and GBM, a potential partnership for silevertinib, and the Company’s expected cash runway. Any forward-looking statements in this press release are based on management’s current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. Risks that contribute to the uncertain nature of the forward-looking statements include those risks and uncertainties set forth in its Annual Report on Form 10-K for the year ended December 31, 2024, filed with the United States Securities and Exchange Commission and in its subsequent filings filed with the United States Securities and Exchange Commission. All forward-looking statements contained in this press release speak only as of the date on which they were made. The Company undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made. ContactFor Investors:investors@bdtx.comFor Media:media@bdtx.com

Phase 2Clinical Result

02 Dec 2025

·www.fiercebiotech.com

Imvax takes brain cancer cell therapy to FDA, betting survival hit will overshadow primary miss

Imvax has told the FDA it plans to file a request to discuss the regulatory pathway for IGV-001.\n Imvax has shrugged off the failure of its phase 2b brain cancer trial to hit its primary endpoint, reporting evidence of improved overall survival (OS) and making a beeline for the FDA.The study compared IGV-001 to placebo in 99 newly diagnosed glioblastoma patients. IGV-001 consists of personalized whole-tumor-derived cells and an antisense oligonucleotide in implantable biodiffusion chambers. The treatment is designed to trigger a tumor-specific immune response to improve outcomes in patients with the highly aggressive brain cancer. All patients received standard of care therapy.Progression-free survival (PFS) was statistically no longer in the IGV-001 arm than in the placebo cohort, causing the study to miss its primary endpoint. Imvax, a 2020 Fierce 15 honoree, buried news of the primary endpoint miss deep in its press release and focused instead on its OS data.Median OS in the IGV-001 cohort was 20.3 months, compared to 14 months in the placebo group. The biotech said the OS improvement of 6.3 months, or 45%, is clinically meaningful, but made no comment on the statistical analysis of the secondary endpoint in its press release. With no patients on IGV-001 experiencing drug-related serious adverse events, Imvax has told the FDA it plans to file a request to discuss the regulatory pathway for the drug candidate. The failure on the primary endpoint is a blemish on the data, but the hit on OS may override the miss on PFS. FDA guidance states OS is usually the preferred endpoint and considered the most reliable measure.Imvax’s 6.3-month improvement in median OS compares favorably to the data that support the current standard of care. Adding the chemotherapy drug temozolomide, which Merck & Co. sells as Temodar, to radiotherapy increased median OS from 12.1 months to 14.6 months. The FDA approved the drug in the indication on the strength of the 2.5-month improvement in 2005.Since then, multiple drugmakers have tried and failed to improve on temozolomide plus radiotherapy. Bevacizumab, which Roche sells as Avastin, improved PFS but failed on OS. Northwest Biotherapeutics reported a significant improvement in OS in patients taking its dendritic cell vaccine DCVax-L. However, independent experts flagged potential concerns with the trial design, and the drug is yet to win approval. With drugmakers failing to move the needle, device company Novocure provided the only progress in the treatment of glioblastoma of the past 20 years. The company linked the tumor treating fields device Optune to a median OS of 20.9 months, compared to 16 months for the control group. Real-world studies have identified barriers to initial and ongoing use of the device.

Clinical ResultVaccineCell TherapyDrug ApprovalPhase 2

02 Dec 2025

·www.prnewswire.com

Insilico Medicine and Atossa Therapeutics Publish AI-Driven Study in Nature Scientific Reports Identifying (Z)-Endoxifen as a Potential Therapeutic Candidate for Glioblastoma

CAMBRIDGE, Mass., Dec. 2, 2025 /PRNewswire/ -- Insilico Medicine ("Insilico"), a global leader in AI-powered drug discovery, and Atossa Therapeutics ("Atossa") (Nasdaq: ATOS), a clinical-stage biopharmaceutical company developing novel treatments for breast cancer and other serious conditions, announce the publication of a joint study evaluating the potential of (Z)-endoxifen for glioblastoma multiforme (GBM). The peer-reviewed article, now published in Nature's Scientific Reports, represents one of the most comprehensive AI-enabled analyses to date exploring whether endoxifen, an active metabolite of tamoxifen with known activity in endocrine-resistant breast cancer, may offer new therapeutic opportunities for one of the deadliest malignant brain tumors in adults. The study aimed to identify new oncology indications with high therapeutic potential for endoxifen, as monotherapy or in combination, by applying Insilico's AI-powered PandaOmics platform across a wide range of cancer types based on its mechanisms of action. Through this systematic evaluation, GBM emerged as a top candidate for further investigation. The publication highlights progress in understanding glioblastoma, an aggressive primary brain tumor with a five-year survival rate of roughly 4%. By applying multi-omics and AI-enabled methods—including transcriptomic integration, computational modeling, single-cell sequencing, survival analysis, and experimental validation—the study offers mechanistic insights that support further exploration of endoxifen's potential role in this setting. (Z)-Endoxifen, already clinically active in hormone-resistant breast cancer and known to act through both estrogen-dependent and estrogen-independent mechanisms, has long been considered a promising molecule for broader oncology applications, but has not been systematically explored in GBM. Using PandaOmics, researchers evaluated more than 900 cancer indications with weighted transcriptomic signatures from endoxifen-treated datasets. PandaOmics combined differential expression, pathway enrichment, protein–protein interaction networks, mechanistic NLP, and disease unmet-need modeling, ultimately ranking GBM among one of the highest-opportunity indications. AI-driven analyses identified more than 1,400 genes shared between GBM tumors and endoxifen-treated cells, revealing strong reversal of biological programs that drive tumor growth and treatment resistance. Endoxifen was predicted to counteract pathways associated with uncontrolled proliferation, inflammation, metabolic dysregulation, and aggressive tumor behavior. Single-cell sequencing further pinpointed key malignant-cell genes linked to poor survival and aggressive subtypes, all of which were downregulated by endoxifen. "This collaboration with Insilico Medicine provides a whole new indication in which we might explore the utility of endoxifen, and potentially significant new opportunities to address an extremely underserved set of cancer patients," said Steven Quay, M.D., Ph.D., CEO of Atossa Therapeutics. "Most of our joint projects have focused on advancing women's health, but this study allowed us to extend our work into glioblastoma, a disease with an urgent and unmet need, and not unlike other highly ranked indications, including but not limited to Duchenne muscular dystrophy (DMD) and multiple gynecologic-related cancers for which (Z)-Endoxifen may have excellent therapeutic outcomes. The results of our collaboration with Insilico highlight how AI-enabled discovery can uncover entirely new opportunities for a well-characterized molecule like endoxifen." Laboratory validation closely matched the computational predictions. In vitro, (Z)-endoxifen significantly suppressed GBM cell proliferation and induced apoptosis, demonstrating greater cytotoxic activity than high-dose temozolomide and showing enhanced effects in combination. In vivo studies confirmed that endoxifen was well tolerated across all doses. Together, the multi-omic analyses and experimental findings highlight endoxifen as a promising therapeutic candidate for GBM and showcase how AI-powered discovery can reveal new opportunities for drug repurposing and cancer treatment innovation. "Atossa is deeply committed to scientific leadership in the prevention and treatment of high risk breast cancers, and Insilico is honored to collaborate with the company to advance genuine innovation and expand indications across different areas of oncology," said Alex Zhavoronkov, Ph.D., Founder and CEO of Insilico Medicine. "This publication represents one of the early fruits of our joint research, and much of our collaborative work remains unpublished. We are hopeful that these efforts will ultimately translate into meaningful therapeutic programs." Harnessing state-of-the-art AI and automation technologies, Insilico has significantly improved the efficiency of preclinical drug development. While traditional early-stage drug discovery typically requires 3 to 6 years, from 2021 to 2024 Insilico nominated 20 preclinical candidates, achieving an average turnaround - from project initiation to preclinical candidate (PCC) nomination - of just 12 to 18 months per program, with only 60 to 200 molecules synthesized and tested in each program. About Insilico Medicine Insilico Medicine, a leading and global AI-driven biotech company, utilizes its proprietary Pharma.AI platform and cutting-stage automated laboratory to accelerate drug discovery and advance innovations in life sciences research. By integrating AI and automation technologies and deep in-house drug discovery capabilities, Insilico is delivering innovative drug solutions for unmet needs including fibrosis, oncology, immunology, pain, and obesity and metabolic disorders. Additionally, Insilico extends the reach of Pharma.AI across diverse industries, such as advanced materials, agriculture, nutritional products and veterinary medicine. For more information, please visit . About Atossa Therapeutics Atossa Therapeutics, Inc. (Nasdaq: ATOS) is a clinical-stage biopharmaceutical company developing innovative therapies for significant unmet needs in breast cancer. Atossa's strategy emphasizes disciplined capital allocation, focusing resources on programs and data packages that can enable future regulatory submissions and potential commercialization. For more information, visit and refer to Atossa's filings with the U.S. Securities and Exchange Commission (SEC). SOURCE Atossa Therapeutics Inc 21% more press release views with Request a Demo

100 Deals associated with Temozolomide

External Link

KEGG	Wiki	ATC	Drug Bank
D06067	Temozolomide	L01AX03	DB00853

R&D Status

Approved

10 top approved records.

to view more data

Indication	Country/Location	Organization	Date
Ewing Sarcoma	Japan	Ohara Pharmaceutical Co., Ltd.	21 Feb 2019
Ewing Sarcoma	Japan	MSD KK (Tokyo)	21 Feb 2019
Malignant glioma of brain	European Union	Teva Pharmaceuticals Europe BV	28 Jan 2010
Malignant glioma of brain	Iceland	Teva Pharmaceuticals Europe BV	28 Jan 2010
Malignant glioma of brain	Liechtenstein	Teva Pharmaceuticals Europe BV	28 Jan 2010
Malignant glioma of brain	Norway	Teva Pharmaceuticals Europe BV	28 Jan 2010
Malignant melanoma, metastatic	Australia	Merck Sharp & Dohme (Australia) Pty Ltd.	13 Nov 2009
Glioblastoma	China	Jiangsu Tasly Diyi Pharmaceutical Co., Ltd.	30 Apr 2004
Astrocytoma	European Union	Merck Sharp & Dohme BV	26 Jan 1999
Astrocytoma	Iceland	Merck Sharp & Dohme BV	26 Jan 1999
Astrocytoma	Liechtenstein	Merck Sharp & Dohme BV	26 Jan 1999
Astrocytoma	Norway	Merck Sharp & Dohme BV	26 Jan 1999
Glioblastoma Multiforme	European Union	Merck Sharp & Dohme BV	26 Jan 1999
Glioblastoma Multiforme	Iceland	Merck Sharp & Dohme BV	26 Jan 1999
Glioblastoma Multiforme	Liechtenstein	Merck Sharp & Dohme BV	26 Jan 1999
Glioblastoma Multiforme	Norway	Merck Sharp & Dohme BV	26 Jan 1999
Glioma	European Union	Merck Sharp & Dohme BV	26 Jan 1999
Glioma	Iceland	Merck Sharp & Dohme BV	26 Jan 1999
Glioma	Liechtenstein	Merck Sharp & Dohme BV	26 Jan 1999
Glioma	Norway	Merck Sharp & Dohme BV	26 Jan 1999

Developing

10 top R&D records.

to view more data

Indication	Highest Phase	Country/Location	Organization	Date
Neuroblastoma	NDA/BLA	European Union	ORPHELIA Pharma SAS	14 Nov 2024
Uterine Leiomyosarcoma	Phase 3	United States	Alliance for Clinical Trials in Oncology	30 Mar 2022
Brain Stem Glioma	Phase 3	United States	National Cancer Institute	26 Jan 2011
Brain Stem Glioma	Phase 3	Canada	National Cancer Institute	26 Jan 2011
Cerebral Astrocytoma, Adult	Phase 3	United States	National Cancer Institute	26 Jan 2011
Cerebral Astrocytoma, Adult	Phase 3	Canada	National Cancer Institute	26 Jan 2011
High grade glioma	Phase 3	United States	National Cancer Institute	26 Jan 2011
High grade glioma	Phase 3	Canada	National Cancer Institute	26 Jan 2011
Pediatric Cerebellar Astrocytoma	Phase 3	United States	National Cancer Institute	26 Jan 2011
Pediatric Cerebellar Astrocytoma	Phase 3	Canada	National Cancer Institute	26 Jan 2011

Clinical Result

Indication

Phase

Evaluation

View All Results

Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT04209790 (NeoGlio, CTgov)	Phase 2	Glioblastoma Multiforme \| Glioblastoma \| High grade glioma	2	Surgery post Radiation and Temozolomide (benzolamide)+Drug Therapy with Temozolomide (benzolamide) (Standard of Care)	xsgucmafhx = otjfhxnjch npqlpmulkt (lbgfkenxdx, bmmkjrzsob - dqqebjqnwq) View more	-	07 Nov 2025
ESMO2025	Not Applicable	Glioma	25	concomitant CHT and TMZ	dglyclpykl(zddofvncxn) = icqgugonjk hypoviqlma (attiqfkqgg, 12.8 - 70.1)	Positive	17 Oct 2025
				concomitant CHT and TMZ (TMB-high)	dglyclpykl(zddofvncxn) = ixudomkgvi hypoviqlma (attiqfkqgg, 14.9 - 67)
ESMO2025	Not Applicable	Endocrine Gland Neoplasms \| Neuroendocrine Tumors	159	Capecitabine + Temozolomide (Pancreatic NETs (PNETs))	ufzksmvhfw(wwtifjspea) = lnuovbxpnc cgbnauardv (dxrluvjozv ) View more	Positive	17 Oct 2025
				Capecitabine + Temozolomide (Gastrointestinal NETs (GINETs))	ufzksmvhfw(wwtifjspea) = taxpojmrqz cgbnauardv (dxrluvjozv ) View more
ESMO2025	Not Applicable	Adrenocortical Carcinoma Second line	45	Gemcitabine-capecitabine (GemCap)	ambystcmqt(ifgevpivrj) = tkccqwuqhw kjexgjqpnu (cshojwdmmt, 7.0 - 18.0) View more	Positive	17 Oct 2025
				Gemcitabine-docetaxel (GemDoc)	ambystcmqt(ifgevpivrj) = zuzsiiuwwo kjexgjqpnu (cshojwdmmt, 7.0 - 18.0) View more
GLILIFE (ESMO2025)	Phase 2	High grade glioma	25	Cannabis sativa extract + Temozolomide	zwbdtszssl(sfbggrqpbi) = The most common adverse events were fatigue (52.0%), nausea (40.0%), and headache (20.0%). eypdioxyxi (werwfcajeb ) View more	Positive	17 Oct 2025
ESMO2025	Not Applicable	Glioblastoma	17	temozolomide	pmidoguejv(kgcgeanrrd) = kevuxicouy cbdxjflicm (ajkuecpgnl, 71.25 - 80) View more	Positive	17 Oct 2025
ESMO2025	Not Applicable	Glioblastoma MGMT promoter methylation	9	Stupp protocol (Pyrosequencing ≥10% methylation but MGMT-STP27 unmethylated)	wyghmzmqfq(tmxvoiyydo) = czbjsljczu gmtxrzhzcf (ncnjzmziiz ) View more	Positive	17 Oct 2025
				Stupp protocol (Pyrosequencing <5% but MGMT-STP27 methylated)	wyghmzmqfq(tmxvoiyydo) = intsjccrsg gmtxrzhzcf (ncnjzmziiz, 5 - 10) View more
NCT03426891 (CTgov)	Phase 1	Glioblastoma Multiforme	21	Radiotherapy+Vorinostat+Temozolomide+Pembrolizumab (Dose Escalation Phase)	yrryxxgapr = rhqhrtzviv byjukvlaya (tvklgxvihh, njfrlwnfun - ykejwfrttg) View more	-	06 Oct 2025
				Radiotherapy+Vorinostat+Temozolomide+Pembrolizumab (Radiotherapy and Maintenance Phase)	erymhcdnmm(atoscqbmpr) = rjkqbdynpo clafweokiz (uuwckyyxpe, yioscxuztr - pnypvqfchg) View more
NCT03137888 (CTgov)	Phase 2	Gliosarcoma \| Glioblastoma	30	Spectroscopic Magnetic Resonance Imaging+Temozolomide	xfwosbwvbg = eetgnuxgpb kimlnrhwxg (twhmmbmngx, rtuikbghgi - yauhjkctsd) View more	-	03 Aug 2025
NCT04574856 (Phase 2 Study of Multiparametric Magnetic Resonance Imaging-Guided High-Dose Response-Adaptive Radiation Therapy With Concurrent Temozolomide in Patients With Newly Diagnosed Glioblastoma, Pubmed \| Int J Radiat Oncol Biol Phys)	Phase 2	Glioblastoma	30	Temozolomide (Patients with newly diagnosed GBM)	ulzdyvivfs(uvqrfdlnzg) = tpnklwegds vfjrhswyfk (fddufqrzvz ) View more	Positive	01 Jul 2025

Translational Medicine

Boost your research with our translational medicine data.

view full example data

Deal

Boost your decision using our deal data.

view full example data

Core Patent

Boost your research with our Core Patent data.

view full example data

Clinical Trial

Identify the latest clinical trials across global registries.

view full example data

Approval

Accelerate your research with the latest regulatory approval information.

view full example data

Regulation

Understand key drug designations in just a few clicks with Synapse.

view full example data

AI Agents Built for Biopharma Breakthroughs

Accelerate discovery. Empower decisions. Transform outcomes.

Get started for free today!

Accelerate Strategic R&D decision making with Synapse, PatSnap’s AI-powered Connected Innovation Intelligence Platform Built for Life Sciences Professionals.

Start your data trial now!

Synapse data is also accessible to external entities via APIs or data packages. Empower better decisions with the latest in pharmaceutical intelligence.

Bio

Bio Sequences Search & Analysis

Chemical

Chemical Structures Search & Analysis