Paxalisib

澳大利亚悉尼，2026年1月27日，Kazia Therapeutics（纳斯达克股票代码：KZIA）今日公布其1b期在研试验的临床最新数据，该试验评估Paxalisib联合帕博利珠单抗及化疗治疗晚期（IV期）转移性三阴性乳腺癌（TNBC）患者的疗效与安全性。 截至目前，已有3例接受Paxalisib联合方案治疗的转移性三阴性乳腺癌患者展现出具有临床意义的应答，其中试验入组患者中2例达到部分缓解（PR），1例在扩展使用项目中接受治疗的患者达到经确认的完全代谢缓解（CR）。 核心临床亮点 1.  1b期试验中可评估的2例患者均达到部分缓解； 2.  1例晚期转移性三阴性乳腺癌患者在扩展使用方案下，接受帕博利珠单抗/化疗联合Paxalisib再治疗后，达到经确认的完全代谢缓解； 3.  出现临床应答的患者均合并内脏病变及多器官转移； 4.  截至本次数据更新，患者的中位治疗时长约为6.1个月，所有患者仍在持续接受治疗； 5.  Paxalisib以每日30mg剂量联合帕博利珠单抗及化疗使用时，整体安全性与耐受性表现良好。 本项正在开展的1b期试验为多中心、开放标签、随机研究，于2025年6月启动，旨在评估Paxalisib联合两种方案治疗包括三阴性乳腺癌在内的晚期乳腺癌患者的安全性、耐受性及初步临床活性，联合方案分别为：帕博利珠单抗联合化疗（当前一线标准治疗方案）、奥拉帕利（用于BRCA突变的晚期乳腺癌患者）。其中，帕博利珠单抗联合方案队列的入组患者需满足PD-L1阳性（综合阳性评分CPS≥10），与转移性三阴性乳腺癌的标准治疗临床实践一致。 患者应答详情 患者1：部分缓解 61岁女性患者，转移性三阴性乳腺癌伴左肺上叶转移，2025年6月起接受每日30mg剂量Paxalisib联合帕博利珠单抗、吉西他滨/卡铂治疗。完成9个治疗周期后，系列影像学检查显示每次评估均可见肿瘤持续缩小，根据iRECIST标准最终判定为部分缓解，该患者目前仍在试验中。 患者2：部分缓解，靶病灶完全消退 47岁女性患者，广泛转移性三阴性乳腺癌伴肺、肝、骨及淋巴结转移，2025年10月起接受每日30mg剂量Paxalisib联合帕博利珠单抗、吉西他滨/卡铂治疗。完成3个治疗周期后，影像学检查显示达到部分缓解，其中肺部靶病灶完全消退，该患者目前仍在试验中。 患者3：经确认的完全代谢缓解 44岁女性转移性三阴性乳腺癌患者，此前接受过帕博利珠单抗/化疗治疗，2025年初疾病进展并出现骨、肺转移。因既往接受过帕博利珠单抗治疗，该患者不符合正式试验入组标准，但在医生指导下，于2025年6月起接受帕博利珠单抗/化疗联合每日30mg剂量Paxalisib的再治疗。2025年11月10日，氟代脱氧葡萄糖正电子发射断层扫描/计算机断层扫描（FDG PET/CT）显示无活动性恶性肿瘤证据，符合完全代谢缓解标准；2026年1月的随访影像学检查进一步确认该完全代谢缓解，患者目前仍在接受Paxalisib联合帕博利珠单抗治疗。 安全性数据 Paxalisib联合帕博利珠单抗及化疗的整体耐受性良好，约75%的不良事件（AE）被评估为与Paxalisib无关或可能性极低。与Paxalisib相关的不良事件均为预期内表现，且以轻至中度为主，与既往研究结果一致。在每日30mg的给药剂量下，仅观察到1例1级高血糖，无需干预；截至目前共报告2例严重不良事件（SAE），均被判定与Paxalisib无关。 试验后续规划 在完成初期研究中心启动阶段（该阶段旨在保障试验方案的严格执行，且前期首批患者的临床信号令人鼓舞）后，公司计划于2026年4月前新增启动2个临床研究中心，并在2026年年中再启动2个中心。Kazia仍预计在2026年底前完成12例三阴性乳腺癌患者的目标入组，并于2027年初公布试验顶线数据。 此外，Kazia正针对更多乳腺癌人群开展Paxalisib的临床评估，包括早期三阴性乳腺癌、激素受体阳性/人表皮生长因子受体2阴性（HR+/HER2-）乳腺癌——PI3K/mTOR通路失调在这类乳腺癌中已被证实为重要发病机制。Paxalisib为每日一次口服给药，该给药方式为患者和临床研究中心带来的额外负担极小，是极具便利性的治疗选择，这也是公司探索将其拓展至更广泛乳腺癌人群的重要考量因素。公司将在相关研究推进过程中及时公布最新数据。 Kazia Therapeutics首席执行官John Friend医学博士表示：“尽管这些结果均来自正在开展的研究，属于初步数据，但观察到的临床应答具有一致性且缓解程度显著——包括多个转移部位的肿瘤消退、1例完全代谢缓解，这些数据都令人备受鼓舞。转移性三阴性乳腺癌患者实现经确认的完全缓解极为罕见，尤其是在标准治疗后疾病进展的患者中。这些早期数据进一步印证了我们的观点：Paxalisib有望显著增强现有免疫治疗方案的临床活性，我们期待在本年度获得更多临床与转化医学层面的研究发现。” 关于Kazia Therapeutics Kazia Therapeutics有限公司（纳斯达克股票代码：KZIA）是一家总部位于澳大利亚悉尼的肿瘤药物研发企业，核心研发项目为Paxalisib——一款具有血脑屏障穿透性的在研PI3K/Akt/mTOR通路抑制剂，目前正针对多种癌症开展临床研发。2016年末，公司从Genentech获得该药物的授权，截至目前其已开展/完成10项相关临床试验。2024年，Paxalisib治疗胶质母细胞瘤的2/3期研究（GBM-Agile）已完成，公司目前正就设计并开展确证性注册临床试验、推动药物标准获批事宜进行沟通。此外，Paxalisib治疗晚期乳腺癌、脑转移瘤、弥漫性中线胶质瘤、原发性中枢神经系统淋巴瘤的临床试验也在进行中，其中多项试验已公布令人鼓舞的中期数据。 2018年2月，Paxalisib获美国食品药品监督管理局（FDA）授予胶质母细胞瘤孤儿药资格；2020年8月，获FDA授予胶质母细胞瘤快速通道资格；2023年7月，其联合放疗治疗携带PI3K通路突变的实体瘤脑转移瘤的适应症，获FDA快速通道资格。2020年8月，Paxalisib获FDA授予弥漫性内因性桥脑胶质瘤罕见儿科疾病资格与孤儿药资格；2022年6月和7月，其又先后获FDA授予非典型畸胎样/横纹肌样瘤的罕见儿科疾病资格与孤儿药资格。 除Paxalisib外，Kazia还在研发EVT801——一款VEGFR3小分子抑制剂，2021年4月公司从Evotec SE获得该药物授权，临床前数据显示其对多种肿瘤具有抑制活性，且与免疫肿瘤学药物存在协同作用。同时，公司还在推进NDL2的研发工作，这是一款潜在同类首创的核PD-L1蛋白降解剂，靶向免疫治疗耐药及肿瘤转移的新发现机制，目前处于临床前研发阶段。

SYDNEY, Jan. 27, 2026 /PRNewswire/ -- Kazia Therapeutics (NASDAQ: KZIA), today provided a clinical update from its ongoing Phase 1b study evaluating paxalisib in combination with pembrolizumab and chemotherapy in patients with late-stage (Stage IV), metastatic triple-negative breast cancer (TNBC). To date, three patients with metastatic TNBC treated with paxalisib-based regimens have demonstrated meaningful clinical responses, including two partial responses (PRs) in trial participants and one confirmed complete metabolic response (CR) in a patient treated under an expanded access program. Clinical Highlights 2 of 2 evaluable patients enrolled in the Phase 1b trial achieved partial responses One advanced metastatic TNBC patient achieved a confirmed complete metabolic response following re-treatment with pembrolizumab/chemotherapy plus paxalisib (under an expanded access protocol) Responses observed in patients with visceral disease and multi-organ metastases Median time on treatment to date is approximately 6.1 months, with all patients continuing on therapy at the time of this update Paxalisib continues to demonstrate a generally favorable safety and tolerability profile when combined with pembrolizumab and chemotherapy at the 30 mg daily dose The ongoing Phase 1b trial is a multi-center, open-label, randomized study initiated in June 2025 designed to evaluate the safety, tolerability, and preliminary clinical activity of paxalisib in patients with advanced breast cancer, including TNBC, in combination with either: Pembrolizumab plus chemotherapy (current standard-of-care first-line regimen) or Olaparib (advanced breast cancer patients with BRCA mutations). Eligibility for the pembrolizumab-containing cohort required PD-L1–positive disease (CPS ≥10), consistent with standard-of-care practice in metastatic TNBC. Patient 1 – Partial Response A 61-year-old female with metastatic TNBC involving the left upper lobe of the lung, initiated treatment with paxalisib (30 mg daily) in combination with pembrolizumab and gemcitabine/carboplatin in June 2025. After nine cycles of therapy, serial imaging demonstrated continued tumor reduction at each assessment, culminating in a partial response by iRECIST criteria. The patient remains active on study. Patient 2 – Partial Response with Complete Resolution of a Target Lesion A 47-year-old female with extensively metastatic TNBC involving the lung, liver, bone, and lymph nodes, initiated treatment with paxalisib (30 mg daily) in combination with pembrolizumab and gemcitabine/carboplatin in October 2025. Following three treatment cycles, imaging demonstrated a partial response, including complete resolution of a target lung lesion and the patient remains active on study. Patient 3 – Confirmed Complete Metabolic Response A 44-year-old female with metastatic TNBC who had previously received pembrolizumab/chemotherapy experienced disease progression involving bone and lung metastases in early 2025. Although ineligible for the formal trial due to prior pembrolizumab exposure, the patient was re-treated beginning in June 2025 with pembrolizumab/chemotherapy plus paxalisib (30 mg daily) under physician supervision. On November 10, 2025, FDG PET/CT imaging demonstrated no evidence of active malignancy, consistent with a complete metabolic response. This complete metabolic response was confirmed on follow-up imaging in January 2026, and the patient remains on paxalisib and pembrolizumab therapy. Paxalisib has been generally well tolerated in combination with pembrolizumab and chemotherapy. Approximately 75% of adverse events (AEs) were assessed as unlikely or unrelated to paxalisib. The paxalisib-related AEs were expected and predominantly mild to moderate, consistent with prior studies. At the 30 mg daily dose, one case of Grade 1 hyperglycemia has been observed, requiring no intervention. Two serious adverse events (SAEs) have been reported to date, both deemed unrelated to paxalisib. Following an initial targeted site activation phase designed to ensure rigorous protocol execution and informed by encouraging early clinical signals from the first patients treated, the Company expects to activate two additional clinical sites by April 2026, with two further sites planned for mid-2026. Kazia continues to anticipate the targeted enrollment of twelve TNBC pts target by the end of 2026 and topline data readout in early 2027 Kazia is also evaluating paxalisib in additional breast cancer populations, including earlier-stage TNBC and hormone receptor–positive, HER2-negative (HR+ / HER2-) breast cancer, where dysregulation of the PI3K/mTOR pathway is well established. Paxalisib's oral, once-daily administration offers a potentially convenient treatment option with minimal incremental burden to patients and clinical sites, an important consideration as the Company explores expansion into broader breast cancer populations. The Company will provide updates as these programs advance. "While these observations represent a preliminary read from ongoing studies, the consistency and depth of responses we are seeing including tumor regression across multiple metastatic sites and a complete metabolic response are highly encouraging," said Dr. John Friend, M.D., Chief Executive Officer of Kazia Therapeutics. "Confirmed complete responses in metastatic triple-negative breast cancer are exceedingly rare, particularly in patients who have already progressed on standard therapies. These early data reinforce our belief that paxalisib has the potential to meaningfully enhance the activity of existing immunotherapy-based regimens, and we look forward to generating additional clinical and translational insights throughout the year." For investor and media, please contact Mike Moyer, Managing Director LifeSci Advisors LLC, mmoyer@lifesciadvisors, +1-617-308-4306. About Kazia Therapeutics Kazia Therapeutics Limited (NASDAQ: KZIA) 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. 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. 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. 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. Preclinical data has shown EVT801 to be active against a broad range of tumor types and has provided evidence of synergy with immuno-oncology agents. 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 development. For more information, please visit or follow us on X @KaziaTx. Forward-Looking Statements 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, the plan to activate additional clinical sites for the Phase 1b study in 2026, the anticipated completion of enrolment in the Phase 1b clinical trial in the fourth quarter of 2026, the anticipated topline data readout in early 2027, the potential of paxalisib's oral, once-daily administration to offer a convenient treatment option with minimal incremental burden to patients and clinical sites, the opportunities of evaluating paxalisib in additional breast cancer populations, and the potential benefits of paxalisib. 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 clinical and preclinical trials and product development, including the risk that interim or early data may not be consistent with final data, risks related to regulatory approvals, risks related to the impact of global economic conditions, and risks related to Kazia's ability to regain and/or maintain compliance with the applicable Nasdaq continued listing requirements and standards. These and other risks and uncertainties are described more fully in Kazia's Annual Report, filed on form 20-F with the SEC, and in subsequent filings with the United States Securities and Exchange Commission. 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. You should not place undue reliance on these forward-looking statements, which apply only as of the date of this announcement. SOURCE Kazia Therapeutics Limited 21% more press release views with Request a Demo