Inavolisib

点击上方蓝字 关注我们 值此北京大学肿瘤医院建院50周年之际，“复发转移乳腺癌诊疗格局革新论坛”于2026年5月8日在北京成功举办。本次论坛聚焦复发转移乳腺癌的诊疗前沿，汇聚国内顶尖专家学者，通过主旨报告与专题研讨，深入剖析精准治疗、临床转化及多学科协作等核心议题，为行业呈现了一场高水平的学术盛宴。 此外，大会举行了“晚期乳腺癌ctDNA检测共建单位启动仪式”。该项目旨在通过多家单位的联合协作，共同推进液体活检技术在乳腺癌动态监测与疗效评估中的规范化应用，为后续的精准诊疗提供扎实的数据支撑。 一、开幕致辞：共贺半世纪辉煌，擘画学科新篇 论坛伊始，多位院士及专家出席开幕式并致辞。詹启敏院士高度评价乳腺团队与多科室实质性的MDT协作及前沿科研成果，寄语引入“源头活水”，推动合作走深走实。徐兵河院士向论坛致以热烈祝贺。季加孚院士与马少华书记代表医院，对各位专家的到来表示热烈欢迎，并衷心感谢各位乳腺肿瘤领军专家长期以来对乳腺内科发展的支持与帮助。两位领导强调，乳腺内科近年来的快速进步，正是得益于各方鼎力相助；未来将在多中心研究及人才培养方面持续深化协作。吴炅教授代表兄弟单位，回顾了北肿在临床研究及特色学科等领域的引领式发展历程，期待未来继续深化合作，携手共促肿瘤医学事业高质量发展。 左右滑动查看 二、学术报告：聚焦前沿突破，推进诊疗进阶 在主旨汇报和讨论环节中，有多位专家担任主持。 左右滑动查看 中国医学科学院肿瘤医院徐兵河院士分享了乳腺癌重要临床研究进展。乳腺癌治疗格局正经历重大变革：HR阳性/HER2阴性领域，聚焦CDK4/6抑制剂耐药后的治疗，基于PI3K/AKT/ESR1分型的靶向治疗及TROP2 ADC取得突破；三阴性乳腺癌则围绕免疫联合治疗与TROP2 ADC展开。他强调，未来需依托精准分子分型与人工智能技术，迈向“分人群”诊疗及全生命周期管理。此外，徐兵河院士作为Leading PI牵头了全球首款CDK2/4/6抑制剂库莫西利辅助治疗HR阳性/HER2阴性乳腺癌的III期临床研究。该药覆盖HR阳性晚期一线、二线，早期辅助的全周期布局，标志着中国原创新药在乳腺癌领域正从“跟跑”迈向“领跑”的新局面。 复旦大学附属肿瘤医院邵志敏教授分享了依托“复旦分型”构建的三大原创平台成果：在AI指导的新辅助治疗中，HER2阳性患者病理完全缓解率可达80%、三阴性乳腺癌可达70%；针对三阴性乳腺癌的“复旦四分型”实现了晚期精准的“同病异治”；针对CDK4/6抑制剂耐药构建了LINUX平台，以高效团队推动了中国原创精准诊疗的落地。邵志敏教授作还分享了作为Leading PI牵头的双抗ADC药物TQB2102的临床探索。II期研究显示，TQB2102新辅助治疗HER2阳性乳腺癌的总病理完全缓解率最高达76.9%，进一步彰显了中国原创研究的国际影响力。 中山大学孙逸仙纪念医院宋尔卫院士回顾了HER2阳性晚期乳腺癌从单抗、TKI到ADC药物的治疗演进，重点展示了国产ADC药物SHR-A1811（瑞康曲妥珠单抗）在二线治疗中的突破性数据（中位无进展生存期约30个月，客观缓解率近80%），其疗效对标国际标准，且间质性肺病风险显著较低（约2.8%）。他指出，未来治疗范式正从单通路靶向向“生态治疗”拓展，探索双特异性抗体、HER2疫苗及联合策略，以克服耐药并延长生存。 北京大学肿瘤医院李惠平教授基于该院的ctDNA监测数据，系统阐述了针对基因突变的晚期乳腺癌精准治疗策略。她重点介绍了FABULOUS研究（氟唑帕利±阿帕替尼），证实该方案为gBRCA突变患者带来显著生存获益；同时深入剖析了PIK3CA、ESR1及AKT/PTEN通路等关键靶点的最新进展，强调通过动态监测将基础发现转化为临床干预。 北京大学肿瘤医院宋国红教授系统梳理了HR阳性晚期乳腺癌CDK4/6抑制剂耐药后的精准策略：针对PIK3CA突变，推荐伊那利塞+哌柏西利+氟维司群或卡匹色替+氟维司群；针对ESR1突变，优选口服SERD；针对BRCA突变，采用PARP抑制剂联合阿帕替尼。此外，ADC药物（T-DXd、TROP2ADC）在后线及HER2低表达中发挥重要作用，强调ctDNA动态监测下的全程管理。 北京大学肿瘤医院姜晗昉教授指出，HER2阳性晚期乳腺癌已全面进入ADC时代。二线治疗中，A1811（HORIZON01研究）、T-DXd（DB03研究）及国产RC48（C006研究）均显示出优于既往标准治疗的无进展生存获益。此外，ADC在脑转移患者中也展现出良好的颅内控制效果，为难治人群提供了新选择。 复旦大学附属肿瘤医院江一舟教授聚焦三阴性乳腺癌的临床难题，回顾了复旦分型（IM、BLIS、LAR、MES）指导下的精准治疗，使一线治疗中位无进展生存期从5.8个月提升至11.3个月。针对难治性患者，团队从癌症神经科学视角发现：肿瘤分泌NGF吸引感觉神经浸润，后者释放CGRP作用于成纤维细胞，形成胶原屏障，导致免疫阻隔（“冷肿瘤”）。基于此机制，提出“老药新用”策略，即使用偏头痛药物瑞美吉泮（CGRP受体拮抗剂）打破屏障以增敏免疫治疗，为逆转耐药提供了新方向。 在各位专家精彩汇报后，现场围绕“ADC药物耐药机制”“跨瘤种治疗策略借鉴”及“动态监测临床落地路径”等关键议题展开了热烈讨论。与会专家各抒己见、畅所欲言，不仅进一步凝聚了学术共识，更为后续的研究设计和临床实践提供了具体的切入点和着力点。 左右滑动查看 三、结语：传承创新，共赴新程 五秩芳华，是里程碑，更是新起点。此次论坛不仅是北肿50华诞的学术献礼，更是乳腺肿瘤诊疗格局革新的集结号。从院士们的宏观指引到专家们的微观突破，从分子分型的深耕到神经调控的新疆界，中国学者正以扎实的临床研究与原创思维，不断重塑诊疗范式。愿以此为新起点，以仁心为灯，以创新为帆，在战胜乳腺癌的长路上，共赴下一个辉煌的五十年。 （来源：《肿瘤瞭望》编辑部） 声 明 凡署名原创的文章版权属《肿瘤瞭望》所有，欢迎分享、转载。本文仅供医疗卫生专业人士了解最新医药资讯参考使用，不代表本平台观点。该等信息不能以任何方式取代专业的医疗指导，也不应被视为诊疗建议，如果该信息被用于资讯以外的目的，本站及作者不承担相关责任。

Development of a gedatolisib formulation for subcutaneous injection is underway; first patent application submitted to the U.S. Patent and Trademark Office MINNEAPOLIS, May 14, 2026 (GLOBE NEWSWIRE) -- Celcuity Inc. (Nasdaq: CELC), a clinical-stage biotechnology company focused on the development of targeted therapies for the treatment of multiple solid tumor indications, today announced updates to the Phase 3 VIKTORIA-2 clinical trial of gedatolisib as a first-line treatment for patients with hormone receptor positive (“HR+”), human epidermal growth factor receptor 2 negative (“HER2-”) locally advanced or metastatic breast cancer (“ABC”). Following last year’s announcement and subsequent data presentation demonstrating clinically meaningful improvement in progression-free survival (“PFS”) for patients treated with gedatolisib combined with palbociclib and fulvestrant compared to those treated with fulvestrant in the PIK3CA wild-type (“WT”) cohort of the VIKTORIA-1 Phase 3 study, several important elements of the VIKTORIA-2 clinical trial protocol have been amended. First, in addition to evaluating patients in the first-line setting with endocrine-resistant HR+/HER2- ABC, VIKTORIA-2 will now include a separate study to evaluate the safety and efficacy of patients in the first-line setting who have endocrine-sensitive HR+/HER2- ABC. Second, patients will be assigned manually according to their endocrine sensitivity status to either Study 1 (endocrine-resistant) or Study 2 (endocrine-sensitive) and subsequently be randomized to a treatment arm. Third, efficacy analyses for both Study 1 and Study 2 of VIKTORIA-2 will evaluate the entire intent-to-treat population (combined WT and mutant type (“MT”)) enrolled in their respective study. Each study will have independent statistical analysis plans that will include separate primary endpoints. The primary endpoints for the VIKTORIA-2 clinical trial are PFS, per RECIST 1.1 criteria, as assessed by blinded independent central review (“BICR”). Prior to finalizing the amended Phase 3 trial design, Celcuity conducted a Type B meeting with the U.S. Food and Drug Administration (the “FDA”) to obtain their feedback and to gain alignment on these planned amendments. “We are excited to expand the VIKTORIA-2 study to include patients with endocrine-sensitive HR+/HER2- ABC. For patients with endocrine-sensitive HR+/HER2- advanced breast cancer who received gedatolisib in combination with palbociclib and letrozole in our Phase 1b clinical trial, median progression free survival was 48.6 months, median overall survival was 77.3 months, and the objective response rate was 79%,” said Igor Gorbatchevsky, MD, Chief Medical Officer of Celcuity. “These results are very encouraging and compare favorably to published data for the current standard-of-care regimens for these patients.” “Results from the PIK3CA wild-type and mutation cohort of our VIKTORIA-1 study demonstrated the benefit of gedatolisib combination treatment in HR+/HER2- ABC in the second-line setting,” said Brian Sullivan, CEO and co-founder of Celcuity. “Developing the gedatolisib triplet for nearly all patients in the first-line setting, irrespective of their endocrine sensitivity or PIK3CA status, offers the potential to advance the standard of care for the approximately 90,000 women each year who are diagnosed with late-stage HR+/HER2- ABC.” Gedatolisib Subcutaneous Formulation To support its long-term lifecycle development plan, Celcuity also announced that it submitted to the United States Patent and Trademark Office (“USPTO”) its first patent application for a subcutaneous formulation of gedatolisib that would enable a patient to receive gedatolisib as an injection as an alternative to an infusion. Development of the subcutaneous gedatolisib formulation is ongoing with the goal of demonstrating clinical equivalence to the current intravenous formulation of gedatolisib. The subcutaneous formulation is aimed to support potential future indications for gedatolisib regimens that may result in duration of treatment periods greater than several years. “As we initiate pivotal studies for indications that may offer several years of progression-free survival benefit, development of a subcutaneous formulation of gedatolisib would particularly benefit patients who may potentially be receiving treatment with gedatolisib for extended time periods,” said Brian Sullivan, CEO and co-founder of Celcuity. About HR+/HER2- Breast Cancer Breast cancer is the second most common cancer and one of the leading causes of cancer-related deaths worldwide. 1 More than two million breast cancer cases were diagnosed globally in 2022. 1 While survival rates are high for those diagnosed with early breast cancer, only approximately 30% of patients who are diagnosed with or who progress to metastatic disease are expected to live five years after their diagnosis. 2 HR+/HER2- breast cancer is the most common subtype of breast cancer, accounting for approximately 70% of all breast cancers. 2 Three interconnected signaling pathways, estrogen, cyclin D1-CDK4/6, and PI3K/AKT/mTOR (“PAM”), are primary oncogenic drivers of HR+/HER2- ABC. 3 Therapies inhibiting these pathways are approved and used in various combinations for advanced breast cancer. Currently approved inhibitors of the PAM pathway for breast cancer target a single PAM pathway component, such as PI3Kα, AKT, or mTORC1. 4,5,6,7 However, resistance to CDK4/6 inhibitors and current endocrine therapies develops in many patients with advanced disease. 8 Optimizing the inhibition of the PAM pathway is an active area of focus for breast cancer research. About the Phase 3 VIKTORIA-2 Study VIKTORIA-2 is a Phase 3, global, open-label, randomized, clinical trial designed to evaluate the efficacy and safety of gedatolisib plus palbociclib and endocrine therapy as first-line treatment for patients with HR+/HER2- ABC. Patients will be assigned manually according to their endocrine sensitivity status to either Study 1 (endocrine-resistant) or Study 2 (endocrine-sensitive) and subsequently be randomized 1:1 to either investigational treatment or standard-of-care control. Study 1 is expected to enroll approximately 440 patients with treatment-naïve endocrine-resistant ABC whose cancer progressed while receiving or within 12 months of completing adjuvant endocrine therapy. The trial will evaluate the efficacy and safety of gedatolisib combined with palbociclib and fulvestrant (Arm A) compared to ribociclib combined with fulvestrant (Arm B). Study 2 is expected to enroll approximately 740 subjects with treatment-naïve endocrine-sensitive ABC whose cancer relapsed or progressed 12 months or more after completion of adjuvant endocrine therapy, or those with de novo metastatic disease without prior endocrine therapy exposure. The trial will evaluate the efficacy and safety of gedatolisib combined with palbociclib and letrozole (Arm C) compared to ribociclib combined with letrozole (Arm D). The clinical trial primary endpoints for the VIKTORIA-2 clinical trial are PFS, per RECIST 1.1 criteria, as assessed by BICR. This global trial is expected to enroll subjects at up to 200 clinical sites across North America, Europe, and Asia-Pacific. About Gedatolisib Gedatolisib is an investigational, multi-target PAM inhibitor that potently targets all four class I PI3K isoforms, mTORC1, and mTORC2 to induce comprehensive blockade of the PAM pathway. 9,10,11 As a multi-target PAM inhibitor, gedatolisib’s mechanism of action is highly differentiated from currently approved single-target inhibitors of the PAM pathway. 11 Inhibition of only a single PAM component gives tumors an escape mechanism through cross-activation of the uninhibited targets. Gedatolisib’s comprehensive PAM pathway inhibition ensures full suppression of PAM activity by eliminating adaptive resistance cross-activation that occurs with single-target inhibitors. Unlike single-target inhibitors of the PAM pathway, gedatolisib has demonstrated equal potency and comparable cytotoxicity in PIK3CA MT and WT breast tumor cells in nonclinical studies and early clinical data. 11,12 The FDA has accepted and granted Priority Review of Celcuity’s New Drug Application (“NDA”) for gedatolisib in patients with HR+/HER2-/ PIK3CA WT advanced breast cancer and assigned a Prescription Drug User Fee Act (“PDUFA”) goal date of July 17, 2026. About Celcuity Celcuity is a clinical-stage biotechnology company focused on the development of targeted therapies for the treatment of multiple solid tumor indications. Our lead therapeutic candidate is gedatolisib, a kinase inhibitor of the PAM pathway that binds to all class I PI3K isoforms and the mTOR complexes, mTORC1 and mTORC2. By targeting all class I PI3K isoforms and mTORC1/2, gedatolisib induces comprehensive inhibition of the PAM pathway. Its mechanism of action and pharmacokinetic properties are differentiated from other currently approved and investigational therapies that target PI3Kα, AKT, or mTORC1 alone or together. Our Phase 3 clinical trial, VIKTORIA-1, evaluating gedatolisib in combination with fulvestrant with or without palbociclib in patients with HR+/HER2- ABC, has reported detailed results for Study 1, which evaluated patients with PIK3CA WT tumors, and announced topline results for Study 2, which evaluated patients with PIK3CA MT tumors. Our Phase 3 clinical trial, VIKTORIA-2, is ongoing and incorporates two independent studies, Study 1 and Study 2, evaluating two separate cohorts of patients with ABC who are treatment-naive in the advanced setting. Study 1 is evaluating gedatolisib combined with palbociclib and fulvestrant as first-line treatment for patients with endocrine-resistant HR+/HER2- ABC. Study 2 is evaluating gedatolisib combined with palbociclib and letrozole as first-line treatment for patients with endocrine-sensitive HR+/HER2- ABC. A Phase 1b/2 clinical trial, CELC-G-201, evaluating gedatolisib in combination with darolutamide in patients with metastatic castration resistant prostate cancer, is ongoing. More detailed information about Celcuity’s active clinical trials can be found at ClinicalTrials.gov . Celcuity is headquartered in Minneapolis. Further information about Celcuity can be found at . Follow us on LinkedIn and X . Forward-Looking Statements This press release contains statements that constitute "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995 including statements relating to the potential therapeutic benefits of gedatolisib; the size, design and timing of our clinical trials; our interpretation of clinical trial data; the status and timing of the FDA’s review of our NDA for gedatolisib, including the PDUFA goal date assigned by the FDA; the market opportunity for gedatolisib; our expectations regarding the timing of and our ability to obtain FDA approval to commercialize gedatolisib; our strategy, marketing and commercialization plans, including the benefits of strategic decisions regarding studies and trials; other expectations with respect to gedatolisib including future subcutaneous formulations of gedatolisib; our anticipated use of cash; and the strength of our balance sheet. Words such as, but not limited to, “look forward to,” “believe,” “expect,” “anticipate,” “estimate,” “intend,” "confidence," "encouraged," “potential,” “plan,” “targets,” “likely,” “may,” “will,” “would,” “should” and “could,” and similar expressions or words identify forward-looking statements. The forward-looking statements included in this press release are based on management's current expectations and beliefs which are subject to a number of risks, uncertainties and factors, including that our clinical results are based on an ongoing analysis of key efficacy and safety data, and such data may change following a more comprehensive review of the data related to the clinical trial; unforeseen delays in our clinical trials or the FDA’s review of our NDA for gedatolisib; our ability to obtain and maintain regulatory approvals to commercialize gedatolisib, and the market acceptance of gedatolisib; the development of therapies and tools competitive with gedatolisib; and our ability to access capital upon favorable terms. In addition, all forward-looking statements are subject to other risks detailed in our Annual Report on Form 10-K for the year ended December 31, 2025, as such risks may be updated in our subsequent filings with the Securities and Exchange Commission. You are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof. All forward-looking statements are qualified in their entirety by these cautionary statements, and we undertake no obligation to revise or update this press release to reflect events or circumstances after the date hereof. References: Sung H, et al. Global Cancer Statistics 2020: GLOBOCAN Estimates of Incidence and Mortality Worldwide for 36 Cancers in 185 Countries. CA Cancer J Clin. 2021;10.3322/caac.21660. National Cancer Institute. Surveillance, Epidemiology and End Results Program (accessed July 2025). Alves, C. L., & Ditzel, H. J. Drugging the PI3K/AKT/mTOR Pathway in ER+ Breast Cancer. Int J Mol Sci, 2023;24(5),4522. United States Package Insert, US FDA, ITOVEBI United States Package Insert, US FDA, PIQRAY United States Package Insert, US FDA, TRUCAP United States Package Insert, US FDA, AFINITOR Lloyd M R, et al. Mechanisms of Resistance to CDK4/6 Blockade in Advanced Hormone Receptor-positive, HER2-negative Breast Cancer and Emerging Therapeutic Opportunities. Clin Cancer Res. 2022;28(5):821-30 Venkatesan, A. M., et al. Bis(morpholino-1,3,5-triazine) derivatives: potent adenosine 5'-triphosphate competitive phosphatidylinositol-3-kinase/mammalian target of rapamycin inhibitors: discovery of compound 26 (PKI-587), a highly efficacious dual inhibitor. J Med Chem, 2010;53(6), 2636-2645. Mallon, R., et al. Antitumor efficacy of PKI-587, a highly potent dual PI3K/mTOR kinase inhibitor. Clin Cancer Res, 2011;17(10), 3193-3203. Rossetti, S., et al. Gedatolisib shows superior potency and efficacy versus single-node PI3K/AKT/mTOR inhibitors in breast cancer models. NPJ Breast Cancer, 2024;10(1), 40. Layman, R., et al. Gedatolisib in combination with palbociclib and endocrine therapy in women with hormone receptor-positive, HER2-negative advanced breast cancer: results from the dose expansion groups of an open-label, phase 1b study. Lancet Oncol, 2024;25(4), 474-487. (24)00034-2 Contacts: For Investors: Brian Sullivan, bsullivan@celcuity.com Vicky Hahne, vhahne@celcuity.com (763) 392-0123 Jodi Sievers, jsievers@celcuity.com (415) 494-9924 For Media: Sam Brown LLC Laura Morgan, lauramorgan@sambrown.com (951) 333-9110