Paxalisib

SYDNEY 悉尼, ，April 15, 2026 2026年4月15日/PRNewswire/ -- Kazia Therapeutics (NASDAQ: /PRNewswire/ -- Kazia Therapeutics（纳斯达克：KZIA 克齐亚), a clinical-stage oncology company developing differentiated therapies for cancers with high unmet need, today announced the appointment of Dr. Sudha Rao as Chief Scientific Officer (CSO). ），一家临床阶段的肿瘤学公司，致力于开发针对未满足需求高的癌症的差异化疗法，今天宣布任命Sudha Rao博士为首席科学官（CSO）。Dr. Rao is the scientific originator of the epigenetic framework underlying paxalisib and a pioneer in next-generation therapeutic platforms, including PD-L1 protein degradation and SETDB1-targeted chromatin modulation. Her appointment brings deep expertise in translational epigenetics, AI-guided epi-drug discovery capability, liquid and spatial epigenetic clinical biomarker precision medicine platforms, and early clinical development into Kazia's executive leadership as the Company advances its integrated oncology platform strategy.. 拉奥博士是paxalisib背后的表观遗传学框架的科学创始人，同时也是下一代治疗平台的先驱，包括PD-L1蛋白降解和SETDB1靶向染色质调节。她的任命为Kazia公司的执行领导团队带来了深厚的转化表观遗传学专业知识、AI引导的表观药物发现能力、液体和空间表观遗传临床生物标志物精准医学平台以及早期临床开发经验，进一步推动公司综合肿瘤学平台战略的实施。Dr. Rao is a highly accomplished translational scientist and biotech executive with more than 20 years of experience spanning pharmaceutical R&D, biotechnology, and early clinical development. She currently holds a professorial appointment and leads the Gene Regulation and Translational Medicine Laboratory at QIMR Berghofer Medical Research Institute and previously held senior scientific roles at Sanofi/Rhône-Poulenc in the UK, where she contributed to one of the earliest clinical genomics platforms.. 饶博士是一位成就卓著的转化科学家和生物技术主管，拥有超过20年的经验，涵盖制药研发、生物技术和早期临床开发。她目前担任教授职务，并领导QIMR伯格霍夫医学研究所的基因调控与转化医学实验室；此前，她曾在英国赛诺菲/罗纳普朗克公司担任高级科学职位，期间为最早的临床基因组学平台之一做出了贡献。She is the founder and former Chief Scientific Officer of EpiAxis Therapeutics and has advanced first-in-class epigenetic therapeutics from discovery through IND-enabling studies and into early clinical trials, including the first Phase 1b study of an LSD1 inhibitor in metastatic breast cancer. Dr. Rao is lead inventor on 39 international patents and has authored numerous high-impact publications in journals including . 她是EpiAxis Therapeutics的创始人和前首席科学官，曾推动多款首创的表观遗传治疗药物从发现阶段经过IND前研究进入早期临床试验，其中包括首个LSD1抑制剂在转移性乳腺癌中的1b期临床试验。Rao博士是39项国际专利的主要发明人，并在多家高影响力期刊上发表了大量论文。Science 科学, ，Nature 自然, and ，以及Immunity 免疫性. 。At Kazia, Dr. Rao will lead all research and development activities, advancing the Company's pipeline and expanding its next-generation platform capabilities, including: 在Kazia，Rao博士将领导所有研究和开发活动，推动公司研发管线的进展，并扩展其下一代平台能力，包括：Paxalisib, a brain-penetrant dual PI3K/mTOR inhibitor being developed across oncology indications, including advanced breast cancer; Paxalisib，一种正在肿瘤学适应症中开发的可穿透大脑的双重PI3K/mTOR抑制剂，包括晚期乳腺癌；NDL2, a novel PD-L1 protein degrader platform designed to target intracellular and nuclear PD-L1 biology; and NDL2，一个旨在靶向细胞内和核内PD-L1生物学的新型PD-L1蛋白降解平台；以及MSETC, a first-in-class SETDB1-targeted epigenetic program aimed at reversing immune evasion at the chromatin level. MSETC，一个首创的针对SETDB1的表观遗传程序，旨在染色质水平上逆转免疫逃逸。Her scientific work has been central to advancing the concept of PI3K/mTOR inhibition as a driver of epigenetic reprogramming, forming the foundation of Kazia's strategy to move beyond pathway inhibition toward therapeutic control of cancer's regulatory drivers. 她的科学工作对于推动PI3K/mTOR抑制作为表观遗传重编程的驱动因素这一概念至关重要，构成了Kazia战略的基础，即超越通路抑制，转向对癌症调控驱动因素的治疗控制。Dr. John Friend, CEO of Kazia Therapeutics, commented: 'Dr. Rao is a leading translational epigenetics scientist, a breast cancer researcher, and the lead inventor behind the intellectual property linking PI3K/mTOR inhibition to epigenetic regulation. As the architect of much of our platform, including paxalisib, NDL2, and MSETC, her appointment allows us to immediately strengthen execution while advancing a more integrated, platform-driven oncology strategy.'. Kazia Therapeutics 首席执行官约翰·弗兰德博士评论道：“饶博士是一位领先的转化表观遗传学科学家、乳腺癌研究员，也是将 PI3K/mTOR 抑制与表观遗传调控联系起来的知识产权的主要发明者。作为我们平台许多技术的架构师，包括帕克萨利布、NDL2 和 MSETC，她的任命使我们能够立即加强执行能力，同时推进更加整合的、以平台为导向的肿瘤学战略。”'I am excited by the opportunity to advance a next-generation oncology platform at Kazia. PI3K/mTOR is a key driver of tumour growth and a master regulator of epigenetic programs that shape tumour behaviour, the tumour microenvironment (TME), and immune evasion,' stated Dr. Rao. 'We are building a platform focused on precision targeting and precision medicine, integrating spatial and liquid epigenomics and AI-driven drug discovery to accelerate novel therapies. “我为在Kazia推动下一代肿瘤学平台的机会感到兴奋。PI3K/mTOR是肿瘤生长的关键驱动因素，也是塑造肿瘤行为、肿瘤微环境（TME）和免疫逃逸的表观遗传程序的主要调控者，”Rao博士表示。“我们正在构建一个专注于精准靶向和精准医疗的平台，整合空间和液体表观基因组学以及人工智能驱动的药物发现，以加速新型疗法的发展。”This includes paxalisib alongside emerging programs such as the PD-L1 degrader and SETDB1-targeting approaches. We aim to enable patient selection, real-time target engagement, and a scalable pipeline with clear clinical translation.'. 这包括帕克萨利西布以及新兴的项目，如PD-L1降解剂和针对SETDB1的方法。我们的目标是实现患者选择、实时靶点参与以及具有明确临床转化的可扩展管线。Dr. Rao will also play a key role in advancing Kazia's biomarker strategy, external collaborations, scientific publications, and strategic partnerships, while continuing to expand the Company's platform and pipeline. 拉奥博士还将发挥关键作用，推进Kazia的生物标志物战略、外部合作、科学出版物和战略伙伴关系，同时继续扩展公司的平台和产品线。For investor and media, please contact Mike Moyer, Managing Director LifeSci Advisors LLC, 对于投资者和媒体，请联系LifeSci Advisors LLC的董事总经理迈克·莫耶，mmoyer@lifesciadvisors 毫米@生命科学顾问, +1-617-308-4306. ，+1-617-308-4306。About Kazia 关于KaziaKazia Therapeutics Limited (NASDAQ: Kazia Therapeutics Limited (NASDAQ:KZIA 克齐亚) is an oncology-focused drug development company, based in Sydney, Australia. Our lead program is paxalisib, an investigational brain penetrant inhibitor of the PI3K / Akt / mTOR pathway, which is being developed to treat multiple forms of cancer. Licensed from Genentech in late 2016, paxalisib is or has been the subject of ten clinical trials in this disease. 是一家专注于肿瘤学的药物开发公司，总部位于澳大利亚悉尼。我们的主要项目是paxalisib，一种正在研究中的可穿透大脑的PI3K/Akt/mTOR通路抑制剂，正在被开发用于治疗多种癌症。Paxalisib于2016年底从基因泰克获得许可，已经在该疾病中进行了十项临床试验。A completed Phase 2/3 study in glioblastoma (GBM-Agile) was reported in 2024, and discussions are ongoing for designing and executing a pivotal registrational study in pursuit of a standard approval. Other clinical trials involving paxalisib are ongoing in advanced breast cancer, brain metastases, diffuse midline gliomas, and primary central nervous system lymphoma, with several of these trials having reported encouraging interim data. 2024年报道了一项已完成的胶质母细胞瘤（GBM-Agile）二期/三期研究，目前正就设计和执行一项关键注册研究进行讨论，以寻求标准批准。其他涉及帕克萨利西布的临床试验正在晚期乳腺癌、脑转移瘤、弥漫性中线胶质瘤和原发性中枢神经系统淋巴瘤中进行，其中一些试验已报告了令人鼓舞的中期数据。Paxalisib was granted Orphan Drug Designation for glioblastoma by the U.S. Food and Drug Administration (FDA) in February 2018, and Fast Track Designation (FTD) for glioblastoma by the FDA in August 2020. Paxalisib was also granted FTD in July 2023 for the treatment of solid tumor brain metastases harboring PI3K pathway mutations in combination with radiation therapy. 2018年2月，Paxalisib被美国食品药品监督管理局（FDA）授予用于胶质母细胞瘤的孤儿药资格，并于2020年8月获得FDA授予的胶质母细胞瘤快速通道资格（FTD）。2023年7月，Paxalisib还获得了与放射治疗联合用于携带PI3K通路突变的实体瘤脑转移治疗的快速通道资格（FTD）。In addition, paxalisib was granted Rare Pediatric Disease Designation and Orphan Drug Designation by the FDA for diffuse intrinsic pontine glioma in August 2020, and for atypical teratoid / rhabdoid tumors in June 2022 and July 2022, respectively. Kazia is also developing EVT801, a small molecule inhibitor of VEGFR3, which was licensed from Evotec SE in April 2021. 此外，paxalisib于2020年8月获得FDA授予的罕见儿科疾病认定和孤儿药认定，用于治疗弥漫性内生型脑桥胶质瘤，并在2022年6月和2022年7月分别获得用于非典型畸胎样/横纹肌样肿瘤的认定。Kazia还在开发EVT801，这是一种VEGFR3的小分子抑制剂，于2021年4月从Evotec SE获得许可。In addition to its clinical-stage programs, Kazia is advancing NDL2, a potentially first-in-class nuclear PD-L1 protein degrader program targeting a newly identified mechanism of immunotherapy resistance and metastatic progression, currently in preclinical . 除了其临床阶段的项目外，Kazia还在推进NDL2，这是一种潜在的首创核PD-L1蛋白降解剂项目，针对新发现的免疫治疗耐药性和转移进展机制，目前处于临床前阶段。www.kaziatherapeutics.com www.kaziatherapeutics.comor follow us on X @KaziaTx. 或在X上关注我们 @KaziaTx。Forward Looking Statements 前瞻性声明This announcement may contain forward-looking statements, 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 anticipated contributions of Dr. 本公告可能包含前瞻性陈述，通常可以通过使用“可能”、“将”、“估计”、“未来”、“向前”、“预期”等词语或其他类似词语来识别。任何描述Kazia未来计划、战略、意图、期望、目标或前景的陈述，以及其他非历史事实的陈述，也都是前瞻性陈述，包括但不限于关于以下内容的陈述：预计Dr.的贡献。Sudha Rao as Chief Scientific Officer; expectations regarding Dr. Rao's leadership of Kazia's research and development activities; the potential of Kazia's three-platform oncology strategy, including paxalisib, NDL2, and MSETC; expectations regarding the advancement of Kazia's pipeline and platform capabilities; the potential of paxalisib, including in advanced breast cancer and other oncology indications; the potential of NDL2 as a PD-L1 protein degrader platform; the potential of MSETC as a SETDB1-targeted epigenetic program; expectations regarding Kazia's biomarker strategy, AI-driven drug discovery, and precision medicine capabilities; and Kazia's broader pipeline strategy and anticipated benefits of its integrated platform approach. Sudha Rao 担任首席科学官；对Rao博士领导Kazia公司研发活动的期望；Kazia公司三大肿瘤学战略平台的潜力，包括paxalisib、NDL2和MSETC；对Kazia公司研发管线及平台能力进展的期望；paxalisib在晚期乳腺癌及其他肿瘤适应症中的潜力；NDL2作为PD-L1蛋白降解平台的潜力；MSETC作为靶向SETDB1的表观遗传项目的潜力；对Kazia公司生物标志物战略、AI驱动药物发现及精准医学能力的期望；以及Kazia公司更广泛的研发管线战略及其整合平台方法预期带来的益处。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 associated with: the ability to attract and retain key personnel, including Dr. 此类声明基于Kazia对影响其业务的未来事件和未来趋势的当前预期和预测，并受某些风险和不确定性的约束，这些风险和不确定性可能导致实际结果与前瞻性声明中预期的结果有重大差异，包括与以下因素相关的风险和不确定性：吸引和留住关键人员的能力，包括博士。Rao; the development of early-stage therapeutic programs; the risk that preclinical results may not be predictive of clinical resul. 饶；早期治疗计划的开发；临床前结果可能无法预测临床结果的风险。SOURCE Kazia Therapeutics Limited 来源：Kazia Therapeutics Limited21 21% %more press release views with 更多新闻稿浏览量与 Request a Demo 请求演示

Kazia CEO John Friend, M.D., said “SETDB1 represents a compelling emerging target in oncology.”\n Kazia Therapeutics is paying $1.4 million upfront to secure a platform for generating epigenetic drugs from an Australian research institute.Brisbane-based QIMR Berghofer developed the AI-integrated drug discovery engine to create epigenetic therapies that target SETDB1, a group of proteins that silence tumor suppressor genes. By targeting SETDB1, the aim is to restore immune signaling in tumors that have become resistant to immunotherapy, including checkpoint inhibitors.The platform has already produced a lead candidate, dubbed MSETC, which Kazia has touted as a highly selective bicyclic peptide designed to target a novel, disease-associated nuclear SETDB1 complex.Beyond MSETC, QIMR Berghofer has also developed a pipeline of potential follow-ups that Kezia claimed also have “strong selectivity and intracellular targeting capability,” according to an April 14 release. Kazia, which is also based in Australia, has been working on paxalisib, a brain-penetrant inhibitor of the PI3K/Akt/mTOR pathway. Since licensing the asset from Genentech in 2016, Sydney-based Kazia took paxalisib into a phase 2/3 study in glioblastoma, which failed to hit the mark. Despite the setback, Kazia has been mulling a registrational study in this indication while also assessing the drug in other cancers.Announcing today\'s agreement, Kazia CEO John Friend, M.D., said “SETDB1 represents a compelling emerging target in oncology.”“With this acquisition, we are extending our strategy to target how cancer controls its own behavior by addressing immune resistance at the chromatin level, one of the earliest layers of tumor immune regulation, alongside transcriptional reprogramming with paxalisib and targeted protein degradation with our PD-L1 platform,” Friend said. “Together, these programs position Kazia\'s pipeline to address cancer therapy across multiple layers of tumor biology,” the CEO added.