Refractory Osteosarcoma

GSK plans the therapy’s commercial launch in the US for the second half of 2025. Credit: MAXSHOT.PL/Shutterstock. The US Food and Drug Administration (FDA) has approved GSK’s triazaacenaphthylene antibiotic, Blujepa (gepotidacin), for treating uncomplicated urinary tract infections (uUTIs) in adult females and paediatric patients weighing 40kg and above. The approval is indicated for children aged 12 years and above. It is for uUTIs caused by susceptible micro-organisms such as Escherichia coli, Citrobacter freundii complex, Enterococcus faecalis, Klebsiella pneumonia and Staphylococcus saprophyticus. Blujepa’s approval stems from the positive outcomes of the pivotal Phase III trials, EAGLE-2 and EAGLE-3. These trials showed the therapy’s non-inferiority to nitrofurantoin, a current standard of care for the condition, and even showed statistical superiority in the EAGLE-3 trial. GSK chief scientific officer Tony Wood stated: “The approval of Blujepa is a crucial milestone with uUTIs among the most common infections in women. “We are proud to have developed Blujepa, the first in a new class of oral antibiotics for uUTIs in nearly three decades, and to bring another option to patients given recurrent infections and rising rates of resistance to existing treatments.” In the EAGLE-2 trial, the therapy achieved a therapeutic success rate of 50.6% against 47% for nitrofurantoin. The therapy’s tolerability and safety profile in these trials was found to be consistent with earlier studies. Commercial launch in the US is planned for the second half of this year. Blujepa’s development has been partly financed by federal funds from the US Department of Health and Human Services, the Biomedical Advanced Research and Development Authority, the Administration for Strategic Preparedness and Response, and the Defense Threat Reduction Agency. It works by blocking bacterial DNA replication through a unique binding site, offering a new approach to tackling pathogens and offering balanced blocking of Type II topoisomerase enzymes. GSK achieved breakthrough therapy designation from the FDA for its B7-H3-targeted antibody-drug conjugate, GSK’227, for the treatment of relapsed or refractory osteosarcoma in adults in January 2025.

Endometrial cancer originates in the endometrium, which is the inner lining of the uterus. Credit: megaflopp/Shutterstock. The European Commission (EC) has approved GSK’s Jemperli (dostarlimab) combined with chemotherapy that includes carboplatin and paclitaxel for the first-line treatment of primary advanced or recurrent endometrial cancer in adults. The decision extends the use of the combination in the European Union (EU) for individuals with mismatch repair proficient (MMRp)/microsatellite stable (MSS) tumours. Such tumours account for 75% of individuals with endometrial cancer, and present limited treatment alternatives. The expanded approval is based on the outcomes from Part I of the RUBY Phase III trial – the only study in this setting to demonstrate statistically significant overall survival (OS) benefit across the complete subject population with this cancer type. A 31% decrease in the mortality risk is observed in the trial versus chemotherapy alone. GSK oncology, research and development global head and senior vice-president Hesham Abdullah stated: “For the first time, all patients with primary advanced or recurrent endometrial cancer in the EU have an approved immuno-oncology-based treatment that has shown a statistically significant and clinically meaningful overall survival benefit.” The dual-primary endpoints in Part I were investigator-assessed progression-free survival based on the Response Evaluation Criteria in Solid Tumours v1.1 and OS. At the two-and-a-half-year mark, the probability of survival was 61% for subjects who received the combination, as opposed to 49% for those receiving chemotherapy alone. A median OS improvement of 16.4 months was observed in the combination group compared to the chemotherapy group alone. The tolerability and safety profile for Jemperli combined with carboplatin-paclitaxel was consistent with the known safety profiles of the individual agents. Endometrial cancer originates in the endometrium, which is the inner lining of the uterus. In early January 2025, the company received the US Food and Drug Administration (FDA) breakthrough therapy designation (BTD) for its B7-H3-targeted antibody-drug conjugate, GSK’227, for treating adults with relapsed or refractory osteosarcoma after a minimum of two lines of previous therapy.

一、新药快讯 1、节拍化疗联合内分泌治疗激素受体阳性乳腺癌 2025年1月2日，中山大学肿瘤防治中心王树森教授领衔的一项研究刊登在《临床肿瘤学杂志》，研究结果表明，节拍化疗药物卡培他滨联合芳香化酶抑制剂，能够显著延长激素受体阳性乳腺癌患者的无进展生存期，中位总生存时间也获得了延长，这个研究未那些CDK4/6抑制剂不能耐受的乳腺癌患者提供了新的治疗选择。 2、针对B7-H3的抗体偶联药，获得美国FDA突破性疗法认定 近日，美国食品药品监督管理局FDA授予新型抗体偶联药GSK5764227突破性疗法资格认定，这款抗体偶联药是针对B7-H3这个靶点，在许多癌症中，B7-H3靶点都会显著上升，癌细胞可以借助它们成功躲避免疫系统的剿灭，相应的临床试验证据表明，在11名晚期小细胞肺癌患者中，7人肿瘤大幅缩小或消失，如果加上肿瘤保持稳定的患者，则总体疾病控制率达到81.8%。此外，该药在难治性骨肉瘤患者中展现了充满希望的抗肿瘤活性和可控的安全性。 3、雷替凡利单抗治疗HPV引起的肛管鳞状细胞癌，56%的患者病灶缩小 HPV病毒不仅仅会引起宫颈癌，还会引发其他部位的癌症，比如肛管鳞状细胞癌，雷替凡利单抗是一种靶向PD-1的免疫治疗药物，在一项三期临床试验中，常规化疗卡铂联合紫杉醇和雷替凡利单抗，有56%的患者可评估肿瘤病灶缩小，而使用单独化疗的患者组仅有44%的患者病灶大幅度缩小。此外雷替凡利单抗联合化疗的中位总生存时间为29.2个月，而单独化疗组的中位总生存时间是23个月。 二、新药快讯 1、自身免疫疾病患者，能不能使用免疫治疗？ 最近，一项包括2万5千名患有自身免疫疾病的肿瘤患者使用免疫药物的研究被公布，这些患者包括皮肤癌、肺癌、胃肠道肿瘤和泌尿系统肿瘤，他们接受了PD-1或PD-L1抑制剂治疗，研究的结果表明，如果这些患者能够将自身免疫疾病控制好，那么也可以从免疫治疗药物里获益，而且不会增加风险。但具体临床用药时仍然需要结合病情由主治医生来判断。