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Last update 25 Mar 2025

Temozolomide

Last update 25 Mar 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

+ [27]

Target

DNA

Action

inhibitors

Mechanism

DNA inhibitors(DNA inhibitors), DNA alkylating agents

Therapeutic Areas

NeoplasmsNervous System DiseasesSkin and Musculoskeletal Diseases+ [9]

Active Indication

Ewing SarcomaMalignant glioma of brainMalignant melanoma, metastatic+ [78]

Inactive Indication

Acoustic NeuromaAcute myeloid leukaemia with 11q23 abnormalityAdrenal Gland Pheochromocytoma+ [100]

Originator Organization

Merck Sharp & Dohme Corp.

Active Organization

Bristol Myers Squibb Co.Sun Pharmaceutical Industries (Europe) BV

Merck Sharp & Dohme Corp.

+ [15]

Inactive Organization

Bayer AG

Celgene Corp.

Enzon Pharmaceuticals, Inc.

+ [16]

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

1,039

Clinical Trials associated with Temozolomide

NCT06428045

/ Not yet recruitingPhase 1IIT

Synergistic Treatment with Antiretrovirals and Laser Interstitial Thermal ThErapy (STARLITE) for Unresectable High-Grade Gliomas: a Phase 1 Study

The purpose of this study is to determine whether newly diagnosed high-grade glioma(s) that cannot be removed surgically change as a result of the study treatment; and to identify and evaluate the potential side effects (good and bad) of the study treatment in patients with newly diagnosed high-grade glioma(s) that cannot be removed surgically.

Start Date01 Jul 2025

Sponsor / Collaborator

University of Miami

[+1]

NCT06202066

/ Not yet recruitingPhase 2IIT

A Phase II Study of Temozolomide and Survivin Long Peptide Vaccine (SurVaxM) in Patients With Progressing Metastatic Neuroendocrine Tumors (NETs)

This phase II trial compares the safety and effect of temozolomide combined with survivin long peptide vaccine (SurVaxM) to temozolomide alone in patients with neuroendocrine tumors (NET) that has spread from where it first started (primary site) to other places in the body (metastatic) and is growing, spreading or getting worse (progressing). Temozolomide is in a class of medications called alkylating agents. It works by damaging the cell's deoxyribonucleic acid and may kill tumor cells and slow down or stop tumor growth. Survivin, a protein, is expressed in 50% of patients that have neuroendocrine tumors and, is associated with poor outcomes. SVN53-67/M57-KLH peptide vaccine (SurVaxM) is a vaccine that has been shown to produce an immune system response against cancer cells that express a survivin and may block the growth of new tumor cells. Giving temozolomide with SurVaxM may kill more tumor cells in patients with progressing metastatic neuroendocrine tumors.

Start Date15 Jun 2025

Sponsor / Collaborator

Roswell Park Comprehensive Cancer Center

NCT06681220

/ Not yet recruitingPhase 2IIT

Biomarker Directed Trial of Temozolomide and PARP Inhibition in Relapsed SCLC

Randomized phase 2, multicenter, biomarker directed clinical trial to assess the efficacy of PARP inhibitor plus Temozolomide (TMZ) in relapsed Small Cell Lung Cancer patients. This study will explore if the biomarkers the investigators test predict sensitivity to the combination of PARP inhibitor plus TMZ and therefore leads to a better treatment response. There are two potential tests of biomarkers that can predict who would benefit from the oral combination of PARP inhibitor with Temozolomide (TMZ), but they have not been evaluated. This study will test for this sensitivity using a biomarker (found in the blood that may be related to how a person reacts to a drug). Participants will be assigned to one of the two groups. Group 1 will be patients that test negative for the biomarker and will receive treatment with Lurbinectedin as per standard of care guidelines. Group 2 will be patients that test positive for the biomarker that will be randomly assigned to either the combination of Niraparib plus Temozolomide (TMZ) or Lurbinectedin.

Start Date01 Jun 2025

Sponsor / Collaborator

VA Office of Research and Development

100 Clinical Results associated with Temozolomide

100 Translational Medicine associated with Temozolomide

100 Patents (Medical) associated with Temozolomide

14,338

Literatures (Medical) associated with Temozolomide

31 Dec 2025·Human Vaccines & Immunotherapeutics

Trends in the immunotherapy for glioblastoma: A two-decade bibliometric analysis

Article

Author: Wang, Hongxin ; Yan, Wei ; Long, Zhi ; Yi, Zhenjie

Glioblastoma is a life-threatening primary malignant brain tumor with an unfavorable prognosis. Contributing factors to its poor outcome include tumor heterogeneity, low mutational burden, and immunosuppression within the tumor microenvironment. Recognizing these challenges, immunotherapeutic strategies have emerged as a promising avenue for glioblastoma treatment. Although several dynamic research and scientific trend have increasingly taken pace in the immunotherapeutic approaches to glioblastoma, systematic bibliometric studies on such trends are few. On this note, this study explores a bibliometric analysis of the research hotspots and trends in glioblastoma immunotherapy. We conducted a search in the Web of Science Core Collection database for articles on glioblastoma immunotherapy published between 2004 and 2024. Using VOSviewer and CiteSpace software, we analyzed collected articles to explore aspects such as country of origin, journal of publication, affiliated institute, authorship, keywords, and citation patterns. As of May 1, 2024, we retrieved 3,729 papers on Glioblastoma Immunotherapy. In the field of glioblastoma immunotherapy, the United States stands out as the leading contributor, with 1,708 publications and a substantial 90,590 citations. Following closely, China has made significant contributions through 926 publications, earning 17,533 citations, while Germany adds to the body of knowledge with 349 publications and 16,355 citations. Furthermore, Authoritative journals in this field include Clinical Cancer Research and Neuro-Oncology. The top five keywords during this period were temozolomide, radiotherapy, dendritic cell, cytotoxic T lymphocyte, and vaccination. Moreover, Hotspots in the field include immune checkpoint inhibitors and chimeric antigen receptor T cell therapy.

01 Dec 2025·CANCER CHEMOTHERAPY AND PHARMACOLOGY

WT161, a selective HDAC6 inhibitor, decreases growth, enhances chemosensitivity, promotes apoptosis, and suppresses motility of melanoma cells

Article

Author: Castro-Gamero, Angel Mauricio ; Oliveira, Leilane Sales ; Aissa, Alexandre Ferro ; de Almeida Lima, Graziela Domingues ; Fonseca, Rafael ; Ionta, Marisa ; de Souza, Carlos Vinicius Expedito ; Oliveira-Silva, João Marcos ; Chiminazo, Carolina Berraut

PURPOSE:

Histone deacetylase 6 (HDAC6) plays a critical role in tumorigenesis and tumor progression, contributing to proliferation, chemoresistance, and cell motility by regulating microtubule architecture. Despite its upregulation in melanoma tissues and cell lines, the specific biological roles of HDAC6 in melanoma are not well understood. This study aims to explore the functional effects and underlying mechanisms of WT161, a selective HDAC6 inhibitor, in melanoma cell lines.

METHODS:

Cell proliferation was assessed using both 2D and 3D cell culture systems, including MTT assays, spheroid growth analyses, and colony formation assays. The interaction between WT161 and the chemotherapeutic agents temozolomide (TMZ) or dacarbazine (DTIC) was evaluated using the Chou-Talalay method. Apoptotic cell death was analyzed through flow cytometry, while migration, adhesion, and invasion assays were conducted to evaluate the motility capacities of melanoma cells. Western blot assays quantified α-tubulin acetylation (Lys40), PARP cleavage, and protein levels of β-catenin and E-cadherin.

RESULTS:

WT161 significantly reduced cell growth in both 2D and 3D cultures, decreased clonogenic capacity, and showed synergistic interactions with TMZ and DTIC. The inhibitor also induced apoptotic cell death and enhanced TMZ-induced apoptosis. Additionally, WT161 reduced cell migration and invasion while increasing cell adhesion. These effects were linked to changes in β-catenin and E-cadherin levels, depending on the specific cell type evaluated.

CONCLUSION:

Our study underscores the pivotal role of HDAC6 in melanoma progression, establishing it as a promising therapeutic target. We provide the first comprehensive evidence of WT161's anti-melanoma effects, setting the stage for further research into HDAC6 inhibitors as a potential strategy for melanoma treatment.

01 Jul 2025·BIOMATERIALS

Anti-neuroinflammatory agent rhein lysinate-based self-assembled injectable hydrogel loaded with ZL006 for promoting post-stroke functional recovery

Article

Author: Jiang, Nan ; Lin, Yuhui ; Song, Jiamei ; Li, Rui ; Wang, Jiayu ; Wang, Ruiqi ; Chen, Xi ; Zhang, Ying ; Gao, Yuhao ; Zeng, Jiaqi

The therapeutic agent-based self-assembled hydrogel is gaining interest for biomedical applications, because it overcomes the poor biodegradability and low therapeutic agent loading of conventional polymer gelator-based hydrogel. Here, we present rhein lysinate (RHL), a therapeutic agent that self-assembles to form a stable hydrogel through the π-π stacking and hydrogen bonding interactions, while also exerting anti-neuroinflammatory effect. As a small molecular hydrogelator, RHL has significantly improved water solubility and enhanced self-assembly and gelation capabilities compared to the natural anthraquinone rhein. The relaxed gel-forming conditions enhance the practical application potential of self-assembled hydrogel of RHL (RHL gel). The RHL gel can be loaded with the bioactive agents such as 5-Fluorouracil, temozolomide, edaravone, and ZL006, mainly based on efficient stacking between aromatic rings in the bioactive agents and anthraquinone rings in the hydrogel network structure. The pre-gelled RHL gel and ZL006-loaded RHL gel (ZL006-RHL gel) exhibit shear-thinning behavior, flowing like a liquid under high shear stress during injection. Once this shear stress is removal within the body, they rapidly recover to the initial solid-like state. When a single dose of ZL006-RHL gel is administrated to stroke cavity in the subacute phase of stroke, RHL gel matrix effectively reduces post-stroke neuroinflammation, creates a favorable environment for ZL006 to enhance neuroplasticity, and confers a sustained and stable action to ZL006, leading to a long-lasting improvement of motor performance. This study may provide a valuable strategy for therapeutic intervention to promote post-stroke functional recovery, for which there are no clinically available drugs.

304

News (Medical) associated with Temozolomide

18 Mar 2025

·www.globenewswire.com

March 2025 Letter to Shareholders

VANCOUVER, Washington, March 18, 2025 (GLOBE NEWSWIRE) -- Dear Shareholders, As envisioned, 2025 is unfolding to be an exciting year for CytoDyn Inc. (“CytoDyn” or the “Company”). On February 24, 2025, the Company announced increased survival rates in patients with metastatic Triple-Negative Breast Cancer (“mTNBC”) who were treated with leronlimab in prior CytoDyn-sponsored studies. The impressive survival observations at 12, 24, and 36 months in patients who previously failed treatment in the metastatic or locally advanced setting indicate leronlimab could play a significant role as a paradigm-shifting therapeutic in oncology. Of particular interest, we identified a subgroup of these patients who remain alive and well today and currently identify as cancer-free. This is only the beginning of the Company’s 2025 oncology story. We are eager to provide updates in the coming months as they are available to share. There is still much work to be done, but I am encouraged by what is on the near horizon. I’d like to offer a word of deep gratitude to the CytoDyn team and the key opinion leaders (“KOLs”) who worked so diligently these last months to bring these data together. I would also like to reiterate my appreciation for you – the shareholders. Our business priorities are (i) getting leronlimab to patients in need; and (ii) generating value for our dedicated shareholders. As a long-time supporter of the company and now CEO, I believe investors deserve clear and direct updates as it relates to milestones, regulatory process, and finances. We will continue to incorporate this principle into our messaging as we move forward, presenting a clear picture of where we stand in the development pipeline and celebrating major milestones together. Looking ahead, we are excited to share more about the clarity forming around the putative mechanism of action of leronlimab in solid tumors, as well continued updates relating to the Colorectal Cancer (“CRC”) trial, as further described below. In terms of the regulatory process, I am confident that our collaborative relationship with the FDA has placed us on a positive trajectory. To accelerate progress in oncology where feasible, we’re establishing an oncology advisory board to ensure we are exploring the fastest and most responsible pathway(s) forward. We will continue to look for opportunities to solicit feedback regarding our development process from both KOLs and the FDA. Maintaining strong relationships and credibility with the FDA and industry partners remains a top priority as we chart our future course. The Company continues to be on track financially and we forecast sufficient cash and drug supply on hand to advance our clinical priorities in 2025. As we approach key milestones and announcements in the coming months, we’ll evaluate opportunities to raise additional funds at optimal times and through methods that best serve the Company and its shareholders. We believe leronlimab has already established the potential for tremendous value in the clinic, and in the coming months we look forward to sharing the basis for that conclusion. In sum, the developments in oncology have set the stage for 2025 to be a benchmark year for CytoDyn. This is no longer a platform drug in search of an indication; we now have compelling data to support a role for leronlimab in solid-tumor oncology and are executing on that vision. With Gratitude, Jacob Lalezari, MDCEO Oncology – March 2025 Update The Company continues to prioritize oncology in 2025, as we believe this indication holds the highest potential and shortest timeline for return on investment in the form of a partnership or drug approval. The exciting survival outcomes announced in February 2025 provide early clinical evidence of leronlimab’s potential impact across the field of solid-tumor oncology. As previously announced, we’ve submitted our findings as an abstract to the European Society for Medical Oncology meeting in Munich, Germany in May 2025. We are eager to share additional insights into the apparent mechanism behind the survival outcomes and will do so once appropriate and in compliance with pre-conference publication and announcement allowances. In the meantime, CytoDyn has initiated a follow-up protocol so we can continue to monitor the surviving patients into the future. The CytoDyn/Syneos study teams have now approved eight clinical sites and counting to participate in CytoDyn’s Phase II study of patients with CRC and refractory disease. These clinical sites will include a mix of both large community practices as well as academic centers which all have well-established track records of superior work and high enrollment. With our clinical trial agreements in place and study initiation visits about to start, we expect screening of patients into the CRC study to commence shortly. As previously mentioned, Dr. Ben Weinberg from Georgetown University and the MedStar Health Alliance will be the lead Principal Investigator for the CRC study. Per the FDA’s request, the first five patients enrolled will receive 350 mg of leronlimab SQ once/week in combination with TAS-102 and bevacizumab. After a preliminary safety review by the Data and Safety Monitoring Board (“DSMB”), subsequent patients will be randomized to 350 or 700 mg of weekly leronlimab along with the same background regimen. The DSMB will perform a second safety review after the first 20 patients have completed at least 1 cycle of therapy and can then recommend restricting further enrollment to a single dose level, if deemed appropriate. For additional information, the CRC study protocol is posted on the NCI Clinical Trials website, and can be viewed here. In concert with the observation of prolonged survival in patients with mTNBC described above, CytoDyn remains focused on expeditiously resuming our clinical development in this indication. Two previously announced preclinical studies in TNBC that will identify treatment strategies to optimize the design of future studies are now underway. A third study has begun to further examine the apparent mechanism behind the observed increase in survival as compared to existing treatment paths. In the meantime, we will continue discussions with KOLs about the possibility of initiating a follow-up study in patients with mTNBC on an abbreviated timeline, based on currently available data. The Company also continues to explore the possible use of leronlimab in the treatment of glioblastoma multiforme (“GBM”). A preclinical study at the Albert Einstein College of Medicine sequencing temozolomide and leronlimab is now underway. CytoDyn is also in discussions with several KOLs in neuro-oncology about the possibility of initiating a pilot study in patients with GBM, also based on currently available data. Inflammation – March 2025 Update As previously announced, CytoDyn applied to the NIH/RECOVER-TLC group for the inclusion of leronlimab in their next round of Long Covid treatment studies. The shifting policy landscape in the United States has created some uncertainty around government-sponsored funding of research, but we have been informed by a member of the RECOVER team that their review process has resumed, and we expect a decision soon. In addition, the protocol for a pilot study of leronlimab in the treatment of patients with mild to moderate Alzheimer’s Disease (“AD”) is now finalized. The study will take place at Cornell Medical Center in New York and will evaluate a neuroradiology endpoint that should provide a clear signal of leronlimab’s potential role in treating AD. The study is fully funded, and our colleagues at Cornell are engaged to move the project forward through Cornell’s institutional review process and FDA submission. A new collaborator, Dr. Tom Carmichael, Professor and Chair of Neurology at the University of California, Los Angeles, has published important preclinical observations demonstrating how a small molecule CCR5 inhibitor can expedite recovery following a cerebrovascular accident (“CVA” or “stroke”). CytoDyn is working with Dr. Carmichael and Dr. Kate Schunke at the University of Hawaii to conduct a preclinical study of stroke in transgenic mice that express human CCR5. We are excited by this initiative, given our view that there is an unmet need for innovative and effective treatment paths for patients in this category, and our belief that the market for therapies to treat stroke and/or traumatic brain injury could grow significantly over the next several decades. Dr. Carmichael will also be advising on the pilot study of AD to be initiated at Cornell Medical Center in New York. As announced via press release on February 6, 2025, the final results from SMC Laboratories (“SMC”) indicated statistically significant reversal of liver fibrosis (p< 0.01) in all 3 studies conducted at SMC. Importantly, the reversal of fibrosis appears to be independent of the mechanism of liver insult, as the effect was seen in both metabolic-dysfunction associated steatohepatitis (“MASH”) and CCL4 models of liver injury. To call attention to a key point of clarification, the final results at SMC did not confirm a significant effect of leronlimab on fat accumulation in the liver in the MASH model. Given this observation, we will pause development efforts related to MASH in the near term. Instead, we are continuing discussions with potential partners who have expressed interest in funding studies of leronlimab in the treatment of patients with organ fibrosis to build on the promising findings listed above. Other – March 2025 Update As previously announced, CytoDyn is partnering with the American Foundation for AIDS Research (“amfAR”) to sponsor an HIV cure study called LATCH (Leronlimab in Allogenic stem cell Transplant to Cure HIV). The clinical teams at Oregon Health and Sciences University and the University of Washington remain confident in the chances of success of their LATCH protocol and we look forward to the launch of this program in 2025. CytoDyn continues to prioritize the publication of our clinical data. The exciting observation of improved survival in patients with mTNBC treated with leronlimab has naturally prompted us to reframe the focus of our oncology manuscripts. Submission of these oncology manuscripts for peer review is a top publication priority. Additional ongoing publication efforts include: The manuscript for our CD02 Phase 3 study in patients with multi-drug-resistant HIV was recently published by the Journal of Acquired Immune Deficiency Syndromes and has been posted to our website.The manuscripts for the CD12 Acute Covid and MASH clinical studies are in final preparation for submission.A manuscript summarizing the results of treatment with leronlimab on liver fibrosis from SMC is in preparation.As previously announced, CytoDyn is preparing a manuscript summarizing the integrated safety data from the almost 1,600 patients who have already received leronlimab. The final draft of that safety summary will be completed shortly. Note Regarding Forward-Looking Statements This letter contains forward-looking statements relating to, among other things, clinical drug development and research strategy. The reader is cautioned not to rely on these statements, which are based on current expectations of future events. For important information about these statements and our Company, including the risks, uncertainties and other factors that could cause actual results to vary materially from the assumptions, expectations and projections expressed in any forward-looking statements, the reader should review our Annual Report on Form 10-K for the fiscal year ended May 31, 2024, including the section captioned “Forward-Looking Statements,” and in Part I, Item 1A, as well as subsequent reports filed with the Securities and Exchange Commission. CytoDyn Inc. does not undertake to update any forward-looking statements as a result of new information or future events or developments other than as required by law. Media Contacts CytoDynRiyaz LalaniGagnier CommunicationsCytoDyn@gagnierfc.com

Phase 2Phase 3

12 Mar 2025

·www.pharmaceutical-technology.com

Orphelia explores avenues for liquid neuroblastoma med after EU CHMP rejection

Orphelia said it will continue to make Kizfizo available to paediatric patients under certain programmes and clinical trials. Credit: Andrii Yalanskyi via Getty Images. Orphelia Pharma is exploring alternative regulatory pathways for Kizfizo (temozolomide) following a negative opinion from the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP). The latest decision follows another negative opinion for Orphelia’s marketing authorisation application (MAA) in November 2024. The CHMP determined that Kizfizo failed to demonstrate sufficient clinical benefit to establish a positive benefit-risk balance. The agency cited modest response rates and the lack of convincing evidence that Kizfizo offered significant advantages over existing therapies for relapsed/refractory neuroblastoma. In response to the initial negative opinion, Orphelia requested a re-examination of the decision in December 2024. However, after a re-evaluation on 27 February 2025, the CHMP upheld its initial stance, stating that the committee’s concerns were not resolved. The company shared this update in a press release today (12 March). Orphelia emphasised that it will continue to make Kizfizo available to paediatric patients under compassionate use or early access programmes as well as to patients participating in ongoing clinical trials. “Considering the important unmet medical need, we are currently exploring all available options to make Kizfizo available to the paediatric patients,” said Laurent Martin, chief pharmaceutical affairs officer at Orphelia Pharma in the 12 March announcement. Neuroblastoma is a type of cancer that forms in certain types of nerve tissue, mostly affecting children under the age of five. Neuroblastoma is the second most common solid tumour in children after brain tumours, according to the UK-based charity The Children & Young People’s Cancer Association (CCLG). Kizfizo was designed as a hybrid medicine similar to MSD’s chemotherapy agent Temodal but is formulated as a liquid for easier administration, as many babies and children cannot take oral tablets. Orphelia presented clinical data from studies enrolling 102 patients up to 21 years old. The Phase I TEMOkids (NCT04610736) and Ped-TMZ studies (NCT04467346) demonstrated safety, pharmacokinetics, and efficacy. The ongoing Phase I/II BEACON2 study is evaluating Kizfizo in combination with chemotherapies irinotecan or topotecan. Originally indicated exclusively for the treatment of glioblastoma in adults in the US, temozolomide has been used off label to treat neuroblastoma patients for years. The drug has been approved for treating glioblastoma in adults and children in Europe since 2010. Kizfizo was granted early access authorisation by the French authorities in March 2022 and has received orphan drug designation from the EMA and the US Food and Drug Administration (FDA). The only approved drug in Orphelia’s portfolio is Kigabeq (vigabatrin), its paediatric formulation of vigabatrin, which has an EU authorisation as a monotherapy for West syndrome and for use as a combination treatment for resistant partial epilepsy.

Orphan DrugPhase 1Drug ApprovalClinical Result

05 Mar 2025

·www.prnewswire.com

Laminar Pharma Announces first open-label Progression-Free Survival data for LAM561 in combination with standard of Care in First-Line Therapy for Newly Diagnosed Glioblastoma MGMT-methylated patients

PALMA DE MALLORCA, Spain, March 5, 2025 /PRNewswire/ -- Laminar Pharma, a leader in the development of innovative cancer therapy based on membrane lipid therapy, announced today optimistic results from its ongoing clinical trial evaluating LAM561 for the treatment of newly diagnosed glioblastoma (ndGBM), the most aggressive form of brain cancer, that has seen limited clinical improvement in the last 20 years. The trial, partially funded with an EU H2020 Grant (ClinGlio), has shown an improvement trend in progression-free survival (PFS) in MGMT-methylated patients receiving LAM561 in combination with chemoradiotherapy (radiotherapy plus temozolomide) as the standard of care (SoC) compared to placebo plus SoC treatment. LAM561 is available for in-license for all territories. Laminar's current fund raising round is still open, for a limited time, to additional investors. The pivotal phase 2b/3 trial, "A randomized, double-blind, placebo-controlled adjuvant trial in newly diagnosed primary glioblastoma subjects to assess the efficacy and safety of LAM561 in combination with radiotherapy and temozolomide standard of care treatment" (NCT04250922), enrolled 144 patients with newly diagnosed glioblastoma. In November 2024, After reaching 66 PFS events the independent data monitoring committee (IDMC) recommended: (1) Continue the trial without modifications until 90 OS events (overall survival), when the final analysis will be carried out, estimated for Q4 2026; (2) that the trial should not be stopped for reasons of safety or futility and (3) opening the study, that is, removing the blind. From that moment, patients, doctors and Laminar as sponsor are able to know if any patient received placebo or LAM561. Although the main primary outcome of the interim analysis (Hazard Ratio of 0.5 for PFS) was not reached for the whole trial population, the unblinding of the trial has allowed Laminar to conduct an ongoing evaluation (non-statistically assessed and with data under monitoring review) considering the per protocol predefined stratification: methylation status of the MGMT promoter and RTOG score (3, 4 or 5), a scale describing the side effects caused by radiation therapy and the higher the RTOG score the worse the clinical position. By revising the data stratified by MGMT promoter methylation status and RTOG, and using data available on 18th February 2025, the median PFS for methylated patients RTOG3 was 56,7 weeks on the LAM561 treatment arm against 19 weeks on the placebo arm (n=9). Moreover, the median PFS for methylated patients RTOG4 was 86,4 weeks on the LAM561 treatment arm against 54,7 weeks on the placebo arm (n=39). With currently available data, methylated patients treated with LAM561 + SoC seem to experience a potentially clinically relevantimprovement in PFS (Hazard-ratio estimation of 0.53) compared to control group (placebo+SoC). PFS was not improved by LAM561 in unmethylated patients. This interim perspective should be taken with caution since the study is still ongoing and providing new information and the final interpretation will only be apparent once the study has completed after reaching 90 OS events; and, although progression of the disease is a relevant clinical event, overall survival is the primary outcome of the trial and conclusions on the final effect of the drug need to wait until the final analysis is performed and data is reviewed by the IDMC. Laminar intends to perform an in-depth independent objective assessment of the findings. The methylation status of the MGMT promoter is highly relevant in the prognosis of glioblastoma [Leske et al., 2023]. MGMT-methylated patients represent 35-50% of the total glioblastoma patients. "We are encouraged by the progression-free survival results from this trial in MGMT-methylated patients, which, if reflected by positive overall survival results, may represent a significant advance in the treatment of glioblastoma, a condition with poor prognosis that affects more than 100,000 people every year", said Dr. Pablo Escribà, CEO of Laminar Pharma. "Glioblastoma remains one of the most challenging cancers to treat, and these findings highlight the potential of LAM561 to improve outcomes for methylated patients, a considerable portion of the total glioblastoma population. Our team is committed to continuing the development of this promising therapy." The combination of LAM561 and SoC was well-tolerated. The safety data in the trial is consistent with the known safety profile of LAM561 studied in previous clinical trials, with no new safety signals observed in the combination arm. Trial Details and Key Findings: The study enrolled 144 patients with newly diagnosed, IDH-wildtype, glioblastoma. MGMT-methylated patients treated with LAM561 and SoC currently show a trend towards longer progression-free survival (86,4 weeks) compared to placebo control SoC group (54,7 weeks, HR 0.53). The study will continue until 90 OS events when final analysis will be conducted, estimated by late 2026. Safety data showed that the treatment was well-tolerated, with adverse events consistent with previous trials and a well-known safety profile. These results built on earlier preclinical and clinical data, which indicated that LAM561 could effectively target, through modification of the membrane lipid, the molecular pathways associated with glioblastoma growth. Further analysis and longer follow-up periods are underway to assess overall survival and other secondary endpoints. About Glioblastoma: Glioblastoma (GBM) is the most common primary malignant brain tumor and accounts for nearly 50 percent of all gliomas and approximately 25 percent of all primary brain and CNS malignant tumors. The incidence of GBM in Europe is currently above 25,000 new cases each year, rising to over 100,000 cases per year worldwide. The prognosis for GBM patients is very poor, with a median survival time of about 14.5 months despite optimum chemo-radiation treatment. About 15% of patients survive two years after diagnosis and ca. 4% survive for five or more years. In this scenario, there is a desperate need for novel treatment alternatives that provide safe and more efficacious clinical outcomes. About LAM561: LAM561 (2-hydroxyoleic acid (2-OHOA); idroxioleic acid, sodium) is a synthetic derivative of oleic acid and the most advanced Laminar's R&D product which is taken orally. This drug alters the composition of the plasma membrane in cancer cells, reducing the activity of membrane-associated signaling proteins that are known to promote tumor growth and affecting tumors in the brain. LAM561 is in the process of completing its last clinical development phase and has shown promising preliminary clinical activity in the treatment of aggressive brain tumors, glioblastoma. About Laminar Pharma: Laminar Pharmaceuticals S.A. (Laminar) is a Spanish biotechnological company created in 2006, committed to translational health research, for the rational design and development of drugs to treat oncological and other pathologies in the form of synthetic fatty acids considered "First-in-class Health Solutions" and based on a novel technology, the Melitherapy (MLT or Membrane Lipid Therapy). Laminar controls all the processes, from the rational design of the molecules to the clinical trials that lead to the launch of a new drug, with constant investment in R&D and high-quality standards. It is currently headquartered in Mallorca (Spain) with a subsidiary in Massachusetts (USA). The development and commercialization of medicines involves a high degree of risk, and only a small number of research and development programs result in the commercialization of a product. This document contains forward-looking statements. These statements do not relate strictly to historical or current facts and may be accompanied by words such as "anticipate," "believe," "conceivably" , "could," "estimate," "expect," "may", "potential," "possible," "will," and other words of similar meaning. The results of clinical trials may not be indicative of the full results or the results of later-stage clinical trials and do not guarantee regulatory approval. Media Contact: Javier Fernández Díaz [email protected] References: Leske, H., Camenisch Gross, U., Hofer, S., Neidert, M. C., Leske, S., Weller, M., ... & Rushing, E. J. (2023). MGMT methylation pattern of long-term and short-term survivors of glioblastoma reveals CpGs of the enhancer region to be of high prognostic value. Acta Neuropathologica Communications, 11(1), 139 SOURCE Laminar Pharmaceuticals, S.A. WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Clinical ResultRadiation Therapy

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	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

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Indication	Highest Phase	Country/Location	Organization	Date
Neuroblastoma	NDA/BLA	European Union	ORPHELIA Pharma SAS	14 Nov 2024
Uterine Leiomyosarcoma	Phase 3	United States	National Cancer Institute	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

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


ESMO_SRC2025	Not Applicable	Glioblastoma IDH wildtype	60	temozolomide (Complete resection (R0))	okrpmeaplt(inuoeisyka) = wjrneiobxy emhlflzojv (oyemaeipkp )	Positive	21 Mar 2025
				temozolomide (Incomplete resection (R1))	okrpmeaplt(inuoeisyka) = nuahzhkoga emhlflzojv (oyemaeipkp )
ESMO_ASIA2024 Manual	Phase 2	Glioblastoma Maintenance	40	Concurrent bevacizumab + standard radiotherapy + temozolomide + adjuvant treatment(temozolomide + bevacizumab + Surufatinib)Surufatinib)	sdildmomvy(hydrmohrtt) = pwxhyqxbjd gxxdkznkem (hjmfcipmfe, 4 - 8) View more	Positive	07 Dec 2024
CNSC-35 (SNO2024)	Not Applicable	Diffuse Intrinsic Pontine Glioma Adjuvant	28	Adjuvant Temozolomide	uzncwblygj(zoopawghld) = bveirjvwiq jazdmpinru (zhwpiqiqdu, 5 - 10.7)	Positive	11 Nov 2024
				No Adjuvant Temozolomide	uzncwblygj(zoopawghld) = hhawpcvjvq jazdmpinru (zhwpiqiqdu, 2.3 - NA)
INB-200 (SNO2024)	Phase 1	Glioblastoma Multiforme Maintenance IDH wild type \| mutant tumors \| MGMT unmethylated	23	Temozolomide (Cohort 1 (1 x 10^7 DRI cells/dose))	xqwdjyqzdv(lqrdftikci) = The most common adverse events were decreased WBC/platelet count dlygrjysen (beritvkccv )	Positive	11 Nov 2024
				Temozolomide (Cohort 2 (1 x 10^7 DRI cells/dose))
RADT-09 (SNO2024)	Not Applicable	IDH1-mutant Glioma IDH wild-type \| TERT promoter mutation	21	Chemoradiotherapy with Temozolomide	sejxtopmhs(cosjdxcxmx) = lciqyyjwih szzukqllnh (wjocmvtmrn ) View more	Positive	11 Nov 2024
				Radiotherapy alone	sejxtopmhs(cosjdxcxmx) = qchxaghuku szzukqllnh (wjocmvtmrn ) View more
QLTI-22 (SNO2024)	Not Applicable	Glioma	13	Patients who received Temozolomide chemotherapy	xghfbpgxgd(nqyopwqdug) = roelfiwrnj osntgszgxw (zrmzxtecsc ) View more	-	11 Nov 2024
phase II clinical study (SNO2024)	Phase 2	Glioblastoma Maintenance MGMT status	69	TTFields therapy concomitant with RT/TMZ	aizpxxeetz(sbeoajdkqs) = gzhnkjbcnv ikntwtcqdh (ivhibndvyn ) View more	Positive	11 Nov 2024
				RT/TMZ alone	aizpxxeetz(sbeoajdkqs) = tdltfcivbn ikntwtcqdh (ivhibndvyn ) View more
INNV-27 (SNO2024)	Not Applicable	Pituitary Neoplasms MGMT methylation status	8	Temozolomide	dzebvjftnk(qukppcptdl) = lujhyocdml caqoaewjku (ticokgkhvs )	Positive	11 Nov 2024
SNO2024	Not Applicable	Glioblastoma First line	-	Temozolomide Chemoradiation	wjhkgkhoyi(pgtkhmrhhs) = nusfnhdxgv logzzcwyat (tkkqazvmqx )	Positive	11 Nov 2024
				Placebo	wjhkgkhoyi(pgtkhmrhhs) = qtqglszrgi logzzcwyat (tkkqazvmqx )
CeTeG/NOA-09 (EANO2024)	Phase 3	Glioblastoma	31	Lomustine-Temozolomide Chemotherapy + Radiotherapy	zsffjbrkte(uxslmyxkvs) = dhvvfvxqzv yphbbiltei (lhdohyrbmc )	Positive	17 Oct 2024

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