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.

Predominantly affecting children and young adults, osteosarcoma represents the most widespread primary bone cancer. Credit: Anciens Huang/Shutterstock. GSK has gained US Food and Drug Administration (FDA) breakthrough therapy designation (BTD) for its B7-H3-targeted antibody-drug conjugate (ADC), GSK’227, to treat adults with relapsed or refractory osteosarcoma who have progressed on a minimum of two previous lines of therapy. This marks the third regulatory milestone for the therapy. The BTD is backed by data from the randomised, multi-centre Phase II ARTEMIS-002 trial assessing the safety and efficacy of the therapy in subjects with this condition and other unresectable bone and soft tissue sarcomas. Conducted by China-based pharmaceutical company Hansoh Pharma, the trial enrolled more than 60 subjects, along with 42 osteosarcoma subjects. GSK oncology research and development global head and senior vice-president Hesham Abdullah stated: “This latest regulatory designation for GSK’227 exemplifies the potential of our targeted ADC in patients with difficult to treat cancers. “For patients with relapsed or refractory osteosarcoma, there is an urgent unmet medical need with no approved treatment options once the cancer returns, and chemotherapy provides limited benefit in this setting.” GSK acquired exclusive global rights, excluding mainland China Macau, Taiwan and Hong Kong, to the therapy from Hansoh Pharma in 2024. The acquisition allows GSK to clinically develop and commercialise GSK’227 further globally clinically. The company has recently initiated a global Phase I trial as part of the development plan to support the therapy’s registrational pathway. Predominantly affecting children and young adults, osteosarcoma is the most widespread primary bone cancer. Also known as HS-20093, GSK’227 is a fully human anti-B7-H3 monoclonal antibody linked covalently to a topoisomerase inhibitor payload. In November 2024, GSK received Japan’s Ministry of Health, Labour and Welfare’s approval to extend the indication of its respiratory syncytial virus (RSV) vaccine, Arexvy, to include individuals aged 50 to 59 at increased risk.