A Phase 1b, Multi-centre, Open-label, Randomized Study to Evaluate the Safety, Tolerability, and Clinical Activity of Combining Paxalisib with Olaparib or Pembrolizumab/Chemotherapy in Patients with Advanced Breast Cancer

开始日期2025-06-05

申办/合作机构

Kazia Therapeutics Ltd.

NCT06208657

/ Recruiting临床1/2期IIT

Optimal Precision TherapIes to CustoMISE Care in Childhood and Adolescent Cancer

A companion platform trial to test novel targeted agents based on the patient's tumor profile.

开始日期2024-07-10

申办/合作机构

Bristol Myers Squibb Co.

[+5]

NCT05183204

/ Suspended临床2期IIT

A Phase 2 Trial of Paxalisib Combined With a Ketogenic Diet and Metformin for Newly Diagnosed and Recurrent Glioblastoma

This study is for patients with newly diagnosed glioblastoma, as well as patients who have recurring glioblastoma. Subjects will be given daily paxalisib and metformin while also maintaining a ketogenic diet.
The purpose of this study is to assess the safety of Paxalisib while maintaining a ketogenic diet (a high fat, low carbohydrate diet) and Metformin (a drug approved by the Food and Drug Administration to treat type 2 diabetes), and to see what effects it has on glioblastoma.

开始日期2022-02-14

申办/合作机构

Weill Medical College of Cornell University

[+1]

100 项与帕沙利西布相关的临床结果

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100 项与帕沙利西布相关的转化医学

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100 项与帕沙利西布相关的专利（医药）

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项与帕沙利西布相关的文献（医药）

2025-11-01·BIOCHEMICAL AND BIOPHYSICAL RESEARCH COMMUNICATIONS

Synthesis and evaluation of pyrimidine derivatives for Glioblastoma Multiforme inhibition

Article

作者: Zheng, Chaonan ; Shao, Dong ; Zhang, Jie ; Hong, Rui ; Zhang, Li ; Yu, Rongrong ; Qu, Yanghui ; Yin, Nengjing ; Ma, Zhen ; Wang, Yuan

Glioblastoma Multiforme (GBM) is a fatal brain tumor with limited treatment options. Here, we report KJ-25, a novel pyrimidine-based dual PI3K/mTOR inhibitor designed to overcome the limitations of current therapies like GDC-0084 (Paxalisib). Through structure-oriented optimization, the efficacy of KJ-25 on human GBM cell lines and primary tumor cells of Hras-shTp53-driven mouse model is higher than that of GDC-0084. In terms of mechanism, KJ-25 exhibits superior inhibition of the PI3K/mTOR signaling pathway and almost completely inhibits phosphorylation of AKT (Ser473) and its downstream effectors at low nanomolar concentrations. In an orthotopic transplantation GBM model, compared with GDC-0084, KJ-25 significantly suppressed tumor growth. These results suggest that KJ-25 is a promising candidate drug for the treatment of GBM, providing a strategy for improving the prognosis of this refractory malignant tumor.

2025-10-01·MOLECULAR CANCER THERAPEUTICS

Depleting the Action of EZH2 through PI3K–mTOR Inhibition to Overcome Metastasis and Immunotherapy Resistance in Triple-Negative Breast Cancer

Article

作者: Anandam, Aravind ; Prasanna, Thiru ; Zhang, Shaoqian ; Miranda, Mariska ; Chaudhary, Sumit ; Hartel, Gunter ; Vandermeide, John ; Tu, Wen Juan ; Proctor, Martina ; Chaudhary, Ramesh Kumar ; Melino, Michelle ; Yip, Desmond ; Bain, Amanda L. ; Srihari, Sriganesh ; Nottage, Michelle ; Malik, Laeeq ; Dahlstrom, Jane E. ; Goh, Sal Lee ; Rao, Sudha ; Bielefeldt-Ohmann, Helle ; Seal, Jonathan T. ; Nallan, Gahyathiri ; Nguyen, Tam Hong ; Ives, Amy ; Eastgate, Melissa ; Ahuja, Taniya ; Sinha, Debottam

Abstract:

Almost half of patients with triple-negative breast cancer develop distant metastases, heralding unfavorable outcomes. Here, we provide novel insights into the contribution of the PI3K–mTOR pathway to the triple-negative breast cancer phenotypes that promote growth, migration, metastasis, and therapy resistance. Specifically, we demonstrate that dual targeting of PI3K and mTOR but not PI3K alone inhibits cancer cell proliferation and migration in vitro. Dual PI3K–mTOR inhibition with paxalisib not only promotes a favorable mesenchymal-to-epithelial phenotype but also inhibits signatures associated with metastasis-initiating cells, including the highly aggressive cancer stem cell phenotype, persister cancer cell phenotype (p65, FOXQ1, NRF2, and NNMT), and a cancer drug resistance signature (ABCB5, SNAIL, and ALDH1). In vivo, paxalisib overcomes immunotherapy resistance to reduce primary tumor burden, circulating tumor cells, and direct and indirect indicators of metastasis with a favorable toxicity profile. Gene expression and spatial analyses show that paxalisib profoundly affects the immune microenvironment in tumors, reducing adaptive immune phenotypes associated with immunotherapy resistance (exhausted T cells and regulatory T cells) and protumor innate immune populations such as mast cells. PI3K–mTOR blockade acts upstream of EZH2, impacting both the classic repressive catalytic p85β–EZH2–H27ME3 and active EZH2–NF-κB pathways. Our data suggest that dual targeting of the PI3K–mTOR pathway disrupts both the catalytic and noncatalytic axes of EZH2 to inhibit metastasis and enhance cancer immune visibility, potentially increasing the utility of immunotherapy in resistant individuals.

2025-08-01·MOLECULAR DIVERSITY

iDCNNPred: an interpretable deep learning model for virtual screening and identification of PI3Ka inhibitors against triple-negative breast cancer

Article

作者: Jaiswal, Ravishankar ; Siddiqi, Mohammad Imran ; Bhati, Girdhar ; Ahmed, Shakil

Triple-negative breast cancer (TNBC) lacks estrogen, progesterone, and HER2 expression, accounting for 15-20% of breast cancer cases. It is challenging due to low therapeutic response, heterogeneity, and aggressiveness. The PI3Ka isoform is a promising therapeutic target, often hyperactivated in TNBC, contributing to uncontrolled growth and cancer cell formation. We have proposed an interpretable deep convolutional neural network prediction (iDCNNPred) system using 2D molecular images to classify bioactivity and identify potential PI3Ka inhibitors. We built Custom-DCNN models and pre-trained models such as AlexNet, SqueezeNet, and VGG19 by using the Bayesian optimization algorithm, and found that our Custom-DCNN model performed better than a pre-trained model with lower complexity and memory usage. All top-performed models were screened with the Maybridge Chemical library to find predictive hit molecules. The screened molecules were further evaluated for protein-ligand interaction with molecular docking and finally 12 promising hits were shortlisted for biological validation using in-vitro PI3K inhibition studies. After biological evaluation, 4 potent molecules with different structural moieties were identified, and these molecules present new starting scaffolds for further improvement in terms of their potency and selectivity as PI3K inhibitors with the help of medicinal chemistry efforts. Furthermore, we also showed the significance of the interpretation and visualization of the model's predictions by the Grad-CAM technique, enhancing the robustness, transparency, and interpretability of the model's predictions. The data and script files and prediction run of models used for this study to reproduce the experiment are available in the GitHub repository at https://github.com/ravishankar1307/iDCNNPred.git .

105

项与帕沙利西布相关的新闻（医药）

2026-01-08

·PRNewswire

Kazia Therapeutics to Participate in J.P. Morgan Healthcare Conference Week; Clinical and Translational Update Anticipated Before Month-End

SYDNEY, Jan. 8, 2026 /PRNewswire/ -- Kazia Therapeutics (NASDAQ: KZIA) today announced that its Chief Executive Officer, John Friend, MD, will be in San Francisco next week to participate in meetings during the annual J.P. Morgan Healthcare Conference week (JPM Week). During the week, Kazia will connect with existing and prospective institutional investors, sell-side analysts, and strategic collaborators, including both established partners and potential new industry counterparties, as part of ongoing investor relations and business development outreach. "We are entering JPM Week with strong strategic and clinical momentum," said Dr. John Friend, M.D., Chief Executive Officer of Kazia Therapeutics. "Following the successful completion of our recent financing, we are well capitalized to execute across multiple near-term catalysts, including an anticipated triple negative breast cancer clinical and biomarker update before the end of the month. At the same time, renewed industry focus on the PI3K/mTOR pathway—particularly in hormone receptor–positive, HER2-negative breast cancer—underscores the growing relevance of approaches that are designed to be differentiated and patient-friendly, such as paxalisib." Anticipated Clinical and Program Updates Kazia expects to provide a clinical and translational update before the end of the month from its ongoing Phase 1b trial evaluating paxalisib in advanced triple-negative breast cancer (TNBC). The update is anticipated to include additional clinical response observations, together with expanded circulating tumor cell (CTC) and CTC cluster biomarker analyses, further evaluating paxalisib's potential impact on metastatic disease biology. As previously reported, paxalisib treatment in advanced TNBC has been associated with rapid and sustained reductions in CTCs and CTC clusters, biomarkers increasingly linked to metastatic potential and adverse clinical outcomes. Notably, prior observations in the patient demonstrated that temporary interruption of paxalisib dosing was accompanied by a rebound increase in CTC clusters, with subsequent re-initiation again suppressing overall CTC count and cluster formation, supporting a mechanistically distinct and pharmacodynamically consistent contribution beyond that of immunotherapy alone. The impending update could meaningfully expand the clinical and biological dataset supporting paxalisib in metastatic breast cancer, providing further context around treatment response, biological consistency, and translational relevance, and may further inform the broader development strategy for paxalisib across breast cancer subtypes. In addition, Kazia plans to provide the first update on its potential first-in-class PD-L1 protein degrader program. This program is designed to leverage a novel and differentiated mechanism of action, reflecting evolving scientific strategies aimed at addressing biological complexity, resistance, and durability challenges in immune-based cancer therapies. About Kazia Therapeutics Limited Kazia Therapeutics Limited is an innovative oncology-focused drug development company, based in Sydney, Australia. Our lead program is paxalisib, a brain-penetrant pan-PI3K/mTOR inhibitor, which is being developed to treat multiple forms of brain cancer, including glioblastoma and brain metastases. A Phase 1b clinical trial is also underway evaluating paxalisib in combination with checkpoint inhibition and chemotherapy for patients with advanced triple-negative breast cancer. EVT801, a selective VEGFR3 inhibitor, is currently in a Phase 1 clinical trial for advanced solid tumors. Kazia is working to rapidly progress these assets through clinical development and toward commercialization. Forward Looking Statements This announcement may contain forward-looking statements, within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, which can generally be identified as such by the use of words such as "may," "will," "estimate," "future," "forward," "anticipate," or other similar words. Any statement describing Kazia's future plans, strategies, intentions, expectations, objectives, goals or prospects, and other statements that are not historical facts, are also forward-looking statements, including, but not limited to, statements regarding: the timing, scope, design, conduct, progress, and results of clinical trials and clinical updates; the anticipated clinical and translational update in Kazia's ongoing Phase 1b trial evaluating paxalisib in TNBC before the end of January; the plan to provide the first update on the PD-L1 protein degrader program; the interpretation, durability, and potential significance of clinical and preclinical results; the continued development, regulatory pathway, and the potential therapeutic benefits of the Company's product candidates, including paxalisib and its PD-L1 protein degrader program; the Company's ability to advance its pipeline, obtain regulatory approvals, pursue and maintain strategic collaborations, and execute on its business, development, and capital strategies; and the Company's future expectations, plans, and prospects. Such statements are based on Kazia's current expectations and projections about future events and future trends affecting its business and are subject to certain risks and uncertainties that could cause actual results to differ materially from those anticipated in the forward-looking statements, including risks and uncertainties: related to market and other conditions, associated with clinical and preclinical trials and product development, including the risk that preliminary or interim data may not reflect final results, related to regulatory approvals, and related to the impact of global economic conditions. These and other risks and uncertainties are described more fully in Kazia's most recent Annual Report on Form 20-F filed with the SEC and in subsequent filings with the SEC. Kazia undertakes no obligation to publicly update any forward-looking statement, whether as a result of new information, future events, or otherwise, except as required under applicable law. Investors should not place undue reliance on these forward-looking statements, which apply only as of the date of this announcement. CONTACT: Kate Hill, +61 437 376 171, [email protected] SOURCE Kazia Therapeutics Limited 21% more press release views with Request a Demo

临床1期免疫疗法

2026-01-07

·PRNewswire

How $594B in Precision Tech is Cracking the Metastatic Cancer Code

Issued on behalf of Oncolytics Biotech Inc. USANewsGroup.com News Commentary VANCOUVER, BC, Jan. 7, 2026 /PRNewswire/ -- The global cancer drugs market is surging toward $594.3 billion by 2035 as high-tech immunotherapies begin to replace traditional chemotherapy[1]. With response rates now exceeding 90% in some previously untreatable blood cancers, these platforms are demonstrating a unique ability to reactivate a patient's own immune system to fight back[2]. This fundamental shift toward "reprogramming" the body's defenses drives the investment case for Oncolytics Biotech Inc. (NASDAQ: ONCY), BriaCell Therapeutics Corp. (NASDAQ: BCTX) (TSX: BCT), Greenwich LifeSciences, Inc. (NASDAQ: GLSI), Citius Oncology (NASDAQ: CTOR), and Kazia Therapeutics Limited (NASDAQ: KZIA). With the metastatic cancer treatment market already valued at $84.66 billion, precision-guided therapies now command a dominant 54.51% market share as they displace toxic legacy regimens[3]. As capital flows into these novel modalities, the industry is entering a new era where metastatic spread is no longer an insurmountable barrier, but a prime target for smart, targeted activation. Clinical-stage biotech company Oncolytics Biotech Inc. (NASDAQ: ONCY) is well on its way in developing pelareorep, an investigational immunotherapy designed to activate the immune system against cancer. Closing out the company's 2025, Oncolytics announced exciting efficacy data showing pelareorep achieved a 33% objective response rate in second-line KRAS-mutant microsatellite-stable metastatic colorectal cancer patients when combined with standard chemotherapy. This triples the historical 6-11% response rate for chemotherapy alone in this difficult-to-treat population, addressing a colorectal cancer market projected to reach $20 billion by 2033. Equally compelling anal cancer results put out in late October showed a 30% response rate in second-line squamous cell anal carcinoma, more than doubling the 13.8% benchmark for the only FDA-approved immunotherapy in this setting. The median duration of response reached 15.5 months compared to 9.5 months for standard care, with two durable complete responses. These results across colorectal and anal cancers reinforce pelareorep's potential as a platform immunotherapy for gastrointestinal tumors. Along the way, this clinical momentum has attracted elite academic validation. Now Oncolytics has expanded its Gastrointestinal Tumor Scientific Advisory Board with three globally recognized experts: Dr. Eileen O'Reilly, Winthrop Rockefeller Endowed Chair of Medical Oncology at Memorial Sloan Kettering Cancer Center ("MSK"); Dr. Neil Segal, Research Director of the Division of Gastrointestinal Oncology at MSK; and Dr. Van Morris, Section Chief for Colorectal Cancer at MD Anderson Cancer Center. "I have a strong belief in the potential of immunotherapies like pelareorep to help patients in the gastrointestinal cancer setting," said Dr. Morris. "This drug candidate has shown very promising findings in early trials for patients with colorectal and anal cancers, which should be further studied as soon as possible." The SAB now comprises six leading oncologists spanning Rutgers Cancer Institute, Northwestern University, Asklepios Tumorzentrum Hamburg, MSK, and MD Anderson. The inaugural three members have served as lead investigators in clinical trials involving pelareorep. When researchers of this caliber attach their names to a development program, they stake their professional reputation on the underlying science. Big pharma business development teams take notice when elite academic oncologists congregate around a platform asset. Oncolytics has also secured FDA alignment on its Phase 3 study design for pelareorep in first-line metastatic pancreatic cancer. This positions the company to launch the only immunotherapy registration trial currently planned for this disease. Pelareorep holds both Fast Track and Orphan Drug designations from the FDA for pancreatic cancer. CEO Jared Kelly and Chief Business Officer Andrew Aromando were both crucial contributors to Ambrx Biopharma's $2 billion acquisition by Johnson & Johnson. This track record demonstrates their proven ability to advance clinical assets through value-creating transactions. CONTINUED… Read this and more news for Oncolytics Biotech at: In other recent industry developments and happenings in the market include: BriaCell Therapeutics Corp. (NASDAQ: BCTX) (TSX: BCT) presented positive data from its Phase 3 Bria-ABC study at the 2025 San Antonio Breast Cancer Symposium, demonstrating that biomarkers may predict clinical responses in metastatic breast cancer patients treated with the Bria-IMT regimen plus checkpoint inhibitors. The interim analysis of 116 patients with a median of 6 prior therapies showed favorable progression-free survival in HR+/HER2- disease (3.7 months) and HER2-Low disease (3.9 months), with patients exhibiting a favorable Neutrophil-to-Lymphocyte Ratio achieving median PFS of 4.4 months versus 2.6 months in those with unfavorable NLR. "We are very excited to see the robust and positive biomarker data, which may enable us to more confidently predict clinical responses in patients treated with the Bria-IMT regimen early in their treatment course," said Kelly E. McCann, MD, PhD, breast medical oncologist at UCLA Health Jonsson Comprehensive Cancer Center and lead investigator at UCLA for the Bria-ABC pivotal Phase 3 study. "These biomarkers could serve as highly valuable tools for clinicians, helping them inform treatment decisions for metastatic breast cancer, a complex disease in which many patients have limited or no remaining treatment options." BriaCell's Phase 2 data demonstrated a median overall survival of 11.3 months in DTH-positive patients versus 4.7 months in DTH-negative patients, with 100% clinical benefit rate in evaluable CNS metastasis patients. The Bria-IMT regimen was well tolerated with no treatment-related discontinuations due to adverse events across all studies. Greenwich LifeSciences, Inc. (NASDAQ: GLSI) provided updates on its Phase III FLAMINGO-01 trial evaluating GLSI-100 to prevent breast cancer recurrences, announcing that preliminary analysis of the fully enrolled 250-patient non-HLA-A*02 arm shows approximately 80% reduction in recurrence rates after the Primary Immunization Series, trending similarly to Phase IIb results where treated patients achieved up to 80% recurrence reduction. The trial has approximately 140 sites actively enrolling, with plans to activate 10 additional sites in 2026, while the company entered discussions with leading sites in the United Kingdom and Canada regarding joining the study pending separate regulatory approvals. "We are looking forward to continuing our financing strategy and implementing the planned Phase III trial derisking modifications, pending regulatory approvals," said Snehal Patel, CEO. "The discussions with clinicians at SABCS 2025 were encouraging, as the study has become more widely recognized by the breast cancer community, leading to patient and investigator driven interest to expand FLAMINGO-01 into the United Kingdom and Canada." Greenwich is managing its $7 million annual burn rate through its ATM facility while pursuing non-dilutive strategic partnerships, with planned trial modifications including increased study size, accelerated enrollment, and use of a commercial drug product designed to derisk the registration pathway. Patent filings for non-HLA-A*02 patients will strengthen intellectual property beyond biologics data exclusivity, potentially doubling the addressable market for GLSI-100. Citius Oncology (NASDAQ: CTOR) recently launched LYMPHIR in the U.S. in December 2025 following FDA approval for adult patients with relapsed or refractory Stage I-III cutaneous T-cell lymphoma after at least one prior systemic therapy, marking the first new systemic treatment option for CTCL patients since 2018. Management estimates the initial market for LYMPHIR exceeds $400 million, with the company having secured distribution agreements with three leading U.S. pharmaceutical wholesalers, NCCN guideline inclusion with Category 2A recommendation, and a permanent HCPCS J-code to support coverage and reimbursement. "2025 was a pivotal year for Citius as we successfully launched LYMPHIR following its FDA approval, marking the first new systemic therapy for cutaneous T-cell lymphoma patients since 2018," said Leonard Mazur, Chairman and CEO of Citius Pharma. "With LYMPHIR commercially available as of December 2025, we are focused on its successful launch and adoption in 2026." Citius Pharma owns approximately 78% of Citius Oncology and reported fiscal 2025 net loss of $39.7 million with no revenues as expected pre-launch, while raising approximately $61 million in gross proceeds through strategic financings. The companies deployed an AI-powered sales and marketing platform and secured international access to LYMPHIR in 19 markets through regional distribution partners via named patient programs. Kazia Therapeutics Limited (NASDAQ: KZIA) presented new data at the 2025 San Antonio Breast Cancer Symposium demonstrating that paxalisib, its brain-penetrant dual PI3K/mTOR inhibitor, disrupted highly aggressive circulating tumor cell clusters and reinvigorated exhausted immune populations across both HER2-positive and triple-negative metastatic breast cancer. In HER2-positive patients, paxalisib reduced single CTCs by 42% and CTC clusters by 78% ex vivo, while the first triple-negative breast cancer patient in the Phase 1b PaxPlus-ABC trial experienced 76% primary tumor volume shrinkage with corresponding reductions in CTCs after just one treatment cycle. "These findings reveal an important biological gap left by existing HER2+ directed therapies," said Prof. Sudha Rao, QIMR Berghofer. "CTC clusters persist even in responding patients, and paxalisib is the first agent we have observed that can directly dismantle this highly aggressive and clinically relevant compartment." The data revealed that when paxalisib was temporarily interrupted in the TNBC patient, CTC clusters rapidly resurged, but resumption of treatment restored suppression, indicating that pembrolizumab alone cannot control these metastatic drivers. Paxalisib's ability to simultaneously disrupt mesenchymal CTC clusters while reversing T-cell exhaustion and epigenetically reprogramming tumors toward less aggressive phenotypes positions the drug as a potentially transformative immunotherapy-enhancing agent across multiple breast cancer subtypes. Source: CONTACT: USA NEWS GROUP [email protected] (604) 265-2873 DISCLAIMER: Nothing in this publication should be considered as personalized financial advice. We are not licensed under securities laws to address your particular financial situation. No communication by our employees to you should be deemed as personalized financial advice. Please consult a licensed financial advisor before making any investment decision. This is a paid advertisement and is neither an offer nor recommendation to buy or sell any security. We hold no investment licenses and are thus neither licensed nor qualified to provide investment advice. The content in this report or email is not provided to any individual with a view toward their individual circumstances. USA News Group is a wholly-owned subsidiary of Market IQ Media Group, Inc. ("MIQ"). MIQ has been paid a fee for Oncolytics Biotech Inc. advertising and digital media from the company directly. There may be 3rd parties who may have shares of Oncolytics Biotech Inc., and may liquidate their shares which could have a negative effect on the price of the stock. This compensation constitutes a conflict of interest as to our ability to remain objective in our communication regarding the profiled company. Because of this conflict, individuals are strongly encouraged to not use this publication as the basis for any investment decision. The owner/operator of MIQ own shares of Oncolytics Biotech Inc. which were purchased in the open market, and reserve the right to buy and sell, and will buy and sell shares of Oncolytics Biotech Inc. at any time without any further notice commencing immediately and ongoing. We also expect further compensation as an ongoing digital media effort to increase visibility for the company, no further notice will be given, but let this disclaimer serve as notice that all material, including this article, which is disseminated by MIQ has been approved by Oncolytics Biotech Inc.; this is a paid advertisement, we currently own shares of Oncolytics Biotech Inc. and will buy and sell shares of the company in the open market, or through private placements, and/or other investment vehicles. While all information is believed to be reliable, it is not guaranteed by us to be accurate. Individuals should assume that all information contained in our newsletter is not trustworthy unless verified by their own independent research. Also, because events and circumstances frequently do not occur as expected, there will likely be differences between the any predictions and actual results. Always consult a licensed investment professional before making any investment decision. Be extremely careful, investing in securities carries a high degree of risk; you may likely lose some or all of the investment. SOURCES CITED: Logo: 21% more press release views with Request a Demo

临床结果临床3期免疫疗法临床1期

2026-01-02

·盛诺一家

2025脑癌治疗大爆发：5大突破让最难治的胶质母细胞瘤迎来新希望！

本文由盛诺一家原创编译，转载需经授权导语 2025年被证明是神经肿瘤学领域具有里程碑意义的一年，多项临床突破和监管进展为一些最具挑战性、侵袭性最强的肿瘤带来了新的治疗希望。尤其是在胶质母细胞瘤这一致死率高、极难治疗的脑肿瘤领域，出现了多项重要进展。这些突破不断推动治疗格局的变化，改善了患者预后，并为脑癌研究和临床实践提供了新的方向。回顾2025年，这5项进展最值得关注。来源：ChatGPT生成AI示意图 1 新药Paxalisib有望获批，成为胶质母细胞瘤常规治疗药物在一次会议之后，美国食品药品监督管理局（FDA）表示，Paxalisib用于胶质母细胞瘤的总生存期数据有望支持其获得常规批准。 Paxalisib是由澳大利亚公司 Kazia Therapeutics Limited研发的一种口服靶向药物，是一种小分子PI3K/AKT/mTOR信号通路抑制剂。这一判断基于2/3期GBM-AGILE临床试验的次要终点总生存期数据。结果显示，在新诊断且MGMT启动子未甲基化的胶质母细胞瘤患者中，Paxalisib可将总生存期延长3.8个月。该药物耐受性良好，未发现新的安全性信号。 Kazia Therapeutics公司已与FDA就一项关键性3期临床研究的设计达成一致，以进一步评估Paxalisib在这一人群中的疗效。 2FDA授予新型胶质母细胞瘤疗法BA-101孤儿药资格 2025年9月，FDA授予BA-101孤儿药资格认定。BA-101是由美国生物制药公司NeuroNOS Limited研发的一种用于治疗胶质母细胞瘤的在研小分子药物。该认定可为其研发提供多项权益，从而鼓励针对这一高度未满足医疗需求疾病的药物开发。 BA-101通过靶向失调的一氧化氮信号通路发挥作用，该通路在胶质母细胞瘤中与肿瘤增殖和治疗耐药密切相关。临床前研究数据显示，BA-101不仅可以抑制癌细胞生长，还能增强当前标准治疗药物替莫唑胺（Temodar）的疗效。 3声动力治疗在胶质母细胞瘤中展现潜力今年早些时候，发表在《神经肿瘤学杂志》上的一项研究显示，声动力治疗（SDT）能够在保留健康脑组织的同时，安全地破坏胶质母细胞瘤细胞。该疗法通过将低强度超声与口服5-ALA（一种光敏感前体药物）相结合，实现非侵入式治疗，患者在单次治疗后未出现不良事件。在另一项针对复发性高级别胶质瘤的1/2期临床试验中，声动力治疗将中位（平均）总生存期显著延长至15.7个月，明显优于历史上6至8个月的生存基准。该疗法能够覆盖整个大脑半球的侵袭性病灶区域，被认为具有突破性潜力。目前，一项针对新诊断胶质母细胞瘤患者的随机对照试验即将启动。 4 Dordaviprone获FDA加速批准，为罕见K27M胶质瘤患者带来新希望 2025年8月，FDA加速批准Dordaviprone（商品名Modeyso），用于治疗成人和儿童K27M突变型弥漫性中线胶质瘤患者。这一批准标志着该侵袭性且罕见脑肿瘤迎来了首个获得FDA批准的全身治疗方案。 Dordaviprone是由美国生物制药公司 Modeyso Therapeutics（现归属爱尔兰Jazz Pharmaceuticals公司）研发的一种口服靶向药物，是一种抑制K27M突变蛋白信号通路的小分子抑制剂。支持性临床试验显示，Dordaviprone的客观缓解率为22%，意味着有22%的患者肿瘤显著缩小或完全消失，耐受性良好，且采用每周一次口服给药。在一次访谈中，美国丹娜法伯癌症研究院的Patrick Wen医生深入解读了这一批准的意义，并指出在评估治疗方案时所面临的挑战和复杂性。尽管该批准为部分患者提供了新的治疗选择，但正在进行中的3期ACTION研究仍然是确认其临床获益的关键。 5新型免疫治疗方案在复发性胶质母细胞瘤中表现亮眼一种不含化疗的新型联合治疗方案，在复发性胶质母细胞瘤中显示出显著潜力。该方案将Anktiva（nogapendekin alfa inbakicept-pmln）、NK细胞疗法（自然杀伤细胞治疗）以及肿瘤电场治疗相结合。 Anktiva是由美国生物制药公司Immunity Bio研发的一种注射用免疫调节药物，是一种重组蛋白IL-15受体激动剂，可激活自然杀伤细胞和T细胞。在一项纳入5名患者的探索性研究中，该方案实现了100%的疾病控制率，并获得了3例客观缓解（肿瘤显著缩小或完全消失），其中包括2例接近完全缓解（肿瘤完全消失）。这一治疗策略通过白介素-15激动剂激活免疫细胞，从而克服肿瘤诱导的免疫抑制。初步数据显示，所有受试者的淋巴细胞减少情况均得到逆转。目前，一项2期临床试验正在招募患者，以进一步评估这一免疫激活联合方案在胶质母细胞瘤中的安全性和疗效。资料来源： https://www.targetedonc.com/view/the-top-brain-cancer-news-of-2025 编者按如果您希望了解更多癌症等重大、复杂疾病的海外前沿研究成果、药物、疗法信息，请扫描下方二维码联系我们，盛诺一家的全球就医规划师可为您提供专业的就医路径规划和全球优质医疗资源快速对接服务，包括预约国外权威专家一对一远程咨询、快速办理医疗签证前往海外权威医院就医等。请在备注中输入口令：01023 盛诺一家只做一件事情，就是为国内患者在全球范围内寻找就医途径。只要人类还有办法，不论是创新的药物还是疗法，不论是在美国哈佛，还是英国、日本著名医院，我们都会快速找到，将信息提供给患者，帮助患者快速和全球专家视频咨询，或直接飞到发达国家进行治疗。很多患者因此重获新生。点点赞点分享点收藏写留言

100 项与帕沙利西布相关的药物交易

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外链

KEGG	Wiki	ATC	Drug Bank
D11869	-	-	DB15186

研发状态

10 条进展最快的记录,

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适应症	最高研发状态	国家/地区	公司	日期
胶质母细胞瘤	临床3期	-	江苏先声药业有限公司	-
IDH1突变胶质瘤	临床3期	美国	Kazia Therapeutics Ltd.	-
复发性胶质母细胞瘤	临床2期	美国	Kazia Therapeutics Ltd.	2022-02-14
非霍奇金淋巴瘤	临床2期	美国	Kazia Therapeutics Ltd.	2021-06-01
难治性中枢神经系统淋巴瘤	临床2期	美国	Kazia Therapeutics Ltd.	2021-06-01
HER2阳性乳腺癌	临床2期	美国	Kazia Therapeutics Ltd.	2019-02-11
多形性胶质母细胞瘤	临床2期	美国	Kazia Therapeutics Ltd.	2018-05-15
脑转移瘤	临床2期	美国	Kazia Therapeutics Ltd.	-
弥漫内生性脑桥神经胶质瘤	临床2期	美国	Kazia Therapeutics Ltd.	-
晚期乳腺癌	临床1期	澳大利亚	Kazia Therapeutics Ltd.	2025-01-30

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临床结果

适应症

分期

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研究	分期	人群特征	评价人数	分组	结果	评价	发布日期


ACTRN12624001340527 (NEWS) 人工标引	临床1期	转移性三阴性乳腺癌	-	Paxalisib+标准化疗+帕博利珠单抗	夢鏇鹹醖壓蓋製顧網鹽(襯鏇廠範繭蓋鏇鑰願築) = 鏇艱網築夢憲顧衊廠壓壓觸糧蓋積鹽簾夢觸襯 (襯鹽壓鬱淵艱窪襯積憲 )	积极	2025-07-17
NCT03522298 (GBM AGILE, CTgov)	临床2期	多形性胶质母细胞瘤 \| 胶质母细胞瘤	30	Paxalisib (Dose-escalating Cohort 1: Paxalisib 60mg)	遞構夢觸齋觸壓鹽構製 = 簾醖壓夢網製繭壓衊餘壓襯齋鑰鹹觸艱齋糧繭 (構蓋鹹選憲選衊簾廠願, 範願獵窪顧醖鬱衊網繭 ~ 製選齋艱鹹醖鑰製蓋範) 更多	-	2025-03-24
NCT03522298 (GBM AGILE, CTgov)	临床2期	多形性胶质母细胞瘤 \| 胶质母细胞瘤	30	Paxalisib (Dose-escalating Cohort 2: Paxalisib 75 mg)	遞構夢觸齋觸壓鹽構製 = 範醖鏇壓構遞憲範獵醖壓襯齋鑰鹹觸艱齋糧繭 (構蓋鹹選憲選衊簾廠願, 廠鬱鑰衊簾憲糧觸襯鬱 ~ 夢製築齋遞獵鏇構蓋簾) 更多	-	2025-03-24
NCT04192981 (CTNI-43, SNO2024)	临床1期	脑转移瘤 PI3K pathway alterations \| PIK3CA mutations \| PTEN deletion	21	Paxalisib 45mg/day	範鹽構廠餘簾簾鬱築遞(憲簾膚糧淵網襯醖繭顧) = 淵衊醖網選築衊積餘範淵積積膚範窪鏇廠鏇夢 (鹽膚築鏇選衊廠淵窪顧 )	积极	2024-11-11
NCT04192981 (CTNI-43, SNO2024)	临床1期		21	Paxalisib 60mg/day	範鹽構廠餘簾簾鬱築遞(憲簾膚糧淵網襯醖繭顧) = 願網窪壓獵淵觸觸襯醖淵積積膚範窪鏇廠鏇夢 (鹽膚築鏇選衊廠淵窪顧 )	积极	2024-11-11
NCT03522298 (GBM AGILE, PRNewswire) 人工标引	临床2/3期	胶质母细胞瘤辅助	313	Paxalisib (newly diagnosed unmethylated (NDU) patients)	襯鏇遞艱壓範鬱顧選膚(糧夢網窪遞鏇齋襯築鬱) = 醖鑰願糧憲觸窪選範艱製願願膚齋襯壓膚膚顧 (壓構顧醖遞製窪衊齋夢 ) 更多	积极	2024-07-10
NCT03522298 (GBM AGILE, PRNewswire) 人工标引	临床2/3期	胶质母细胞瘤辅助	313	Standard of Care (SOC) (newly diagnosed unmethylated (NDU) patients)	襯鏇遞艱壓範鬱顧選膚(糧夢網窪遞鏇齋襯築鬱) = 願糧繭膚憲鬱齋鏇築構製願願膚齋襯壓膚膚顧 (壓構顧醖遞製窪衊齋夢 ) 更多	积极	2024-07-10
NCT05009992 (SNO2023) 人工标引	临床2期	弥漫性中线胶质瘤	68	ONC201+paxalisib	膚窪鏇蓋襯顧獵簾獵憲(選鬱襯艱膚構鏇齋壓襯) = 廠衊膚艱選襯壓艱憲廠構構襯鏇積築顧獵鑰獵 (窪襯鹹鹹鹹廠願顧蓋蓋, lower 11.6) 更多	积极	2023-11-10
NCT04906096 (PRNewswire) 人工标引	临床2期	原发性中枢神经系统淋巴瘤	14	Paxalisib	淵鑰範鹹窪築鑰醖壓夢(觸網網網衊壓衊築壓膚) = preliminarily observed in enrolled patients, including partial responses and stable disease 膚顧夢窪鹽夢衊夢窪醖 (積襯壓鏇襯淵獵艱鏇壓 ) 更多	积极	2023-11-01
NCT03522298 (ESMO2022)	临床2期	胶质母细胞瘤	30	Paxalisib	遞選廠鏇夢蓋窪願襯選(製製夢餘願襯壓獵選夢) = 鹽遞壓鑰製積餘鏇遞齋繭繭構夢築範膚糧夢網 (觸範鹽鹹壓窪憲範窪醖 ) 更多	-	2022-09-09
NCT04192981 (Corporate Publications) 人工标引	临床1期	脑转移瘤 PIK3CA Mutation	12	radiotherapy+paxalisib	築壓襯壓築遞積構蓋艱(餘網憲襯遞築築製壓簾) = Paxalisib 45mg daily in combination with radiotherapy 窪鹹壓繭觸襯鏇願艱網 (獵簾築壓願顧醖鏇憲夢 )	积极	2022-08-08
NCT03522298 (ASCO2022)	临床2期	胶质母细胞瘤	30	Paxalisib	築選製選遞遞獵積鑰鑰(積網遞鹹襯繭膚糧憲蓋) = 膚憲遞艱糧壓膚鑰餘廠餘構構構鬱鏇夢膚淵窪 (襯艱餘窪簾簾襯製膚齋 ) 更多	-	2022-06-02
NCT03522298 (AACR2021)	临床2期	多形性胶质母细胞瘤	30	Paxalisib 60mg (fasted)	淵鹹淵膚鹹鬱鹹鹹鹽鑰(糧淵構醖構壓選艱憲鏇) = 壓鬱淵繭遞觸窪醖鏇遞鬱鏇簾廠艱齋淵淵鹽鬱 (憲艱醖鏇願繭積醖選鑰 ) 更多	-	2021-07-01
NCT03522298 (AACR2021)	临床2期	多形性胶质母细胞瘤	30	Paxalisib 60mg (fed)	淵鹹淵膚鹹鬱鹹鹹鹽鑰(糧淵構醖構壓選艱憲鏇) = 構選憲淵網範顧簾廠夢鬱鏇簾廠艱齋淵淵鹽鬱 (憲艱醖鏇願繭積醖選鑰 ) 更多	-	2021-07-01