University of Glasgow

Johnson & Johnson (J&J) has announced that its bladder cancer therapy Balversa (erdafitinib) has been accepted by the Scottish Medicines Consortium (SMC) to treat a subset of urothelial cancer (UC) patients. The pan-FGFR3 tyrosine kinase inhibitor can now be used on the NHS in Scotland to treat unresectable or metastatic UC in adults who are harbouring susceptible FGFR3 genetic alterations and have received at least one line of therapy containing a PD-1 or PD-L1 inhibitor. The SMC’s decision comes shortly after the National Institute for Health and Care Excellence recommended that Balversa be used on the NHS in England and Wales for the same indication, and was supported by positive results from the phase 3 THOR trial. J&J’s drug was shown to increase overall survival from 7.8 months to 12.1 months compared to chemotherapy in the second-line setting. It also demonstrated an improvement in progression-free survival compared to chemotherapy, at 5.6 months versus 2.7 months, and had a confirmed objective response rate of 35.3% versus 8.5% for chemotherapy. John Fleming, UK country medical director, J&J Innovative Medicine, said the SMC’s acceptance “means that eligible UC patients from across the UK can now access this first in class treatment option”, adding that the news “marks a significant step forward for the bladder cancer community”. Almost 1,700 people are diagnosed with bladder cancer in Scotland every year and UC, which starts in the innermost lining of the bladder, accounts for more than 90% of cases. As many as 20% of patients with metastatic UC have an FGFR3 alteration, which can drive the growth of cancer cells is associated with a poor prognosis. Taken orally once daily, J&J’s Balversa is designed to stop or slow the growth of FGFR3-expressing cancer cells, and is the only treatment available in the UK for patients with this specific type of cancer. “This is good news for patients with incurable forms of bladder cancer, as it adds an additional line of life-prolonging treatment for some patients,” said Rob Jones, professor of clinical cancer research at the University of Glasgow. He continued: “Moreover, because the treatment is only suitable for patients with specific genetic changes, we can accurately select patients where the treatment is more likely to work. This means we can spare the other patients from the side effects of a treatment which would be ineffective.”

AstraZeneca's AZD1390 Now Enrolling in GBM AGILE, a Registrational-Intent Phase 2/3 Adaptive Platform Trial for Patients with Glioblastoma AZD1390 Arm Will Recruit Patients with Newly Diagnosed Glioblastoma Across Multiple International Trial Sites LARKSPUR, Calif., May 13, 2025 /PRNewswire/ -- The Global Coalition for Adaptive Research (GCAR) today announced the activation of AstraZeneca's AZD1390 in GBM AGILE (Glioblastoma Adaptive Global Innovative Learning Environment – NCT03970447), the world's first global adaptive platform trial for glioblastoma. The AZD1390 arm will be evaluated for the treatment of adult patients with newly diagnosed glioblastoma. Glioblastoma is the most aggressive and common form of primary brain cancer. Treatment options and patient outcomes have seen little progress over the past several decades. Continue Reading GBM AGILE Adaptive Global Innovative Learning Environment AstraZeneca's AZD1390 is entering the GBM AGILE trial as the 7th investigational arm. Since launching in 2019, the GBM AGILE trial has evaluated multiple therapies and has screened over 2,300 patients at trial locations in six countries. GBM AGILE, a seamless phase 2/3 trial, is conducted under a master protocol, allowing multiple therapies or combinations of therapies from different pharmaceutical companies to be evaluated simultaneously against a common control arm. With its innovative design and efficient operational infrastructure, data from GBM AGILE can potentially be used as the foundation for a new drug application (NDA) and registrations to the US FDA and other health authorities. Professor Anthony Chalmers, Chair of Clinical Oncology at the University of Glasgow and Dr. Patrick Wen, Director, Center for Neuro-Oncology at Dana-Farber Cancer Institute, and Professor of Neurology at Harvard Medical School, are serving as arm Principal Investigators for AZD1390's evaluation in GBM AGILE. Dr. Timothy Cloughesy, Director, Neuro-Oncology Program and Distinguished Professor of Neurology at the University of California, Los Angeles, is the Global Principal Investigator for the overall study. "Glioblastoma is an invariably fatal cancer with few effective treatment options. There is an urgent need for better treatment options," said Professor Anthony Chalmers. "We are excited to evaluate AZD1390 in GBM AGILE based on encouraging data from previous studies and the extensive evidence that AZD1390 can increase the effectiveness of radiation therapy. This investigational drug has clear potential to improve outcomes for glioblastoma patients." "GBM AGILE is designed to rapidly identify and confirm effective therapies for patients with glioblastoma through response adaptive randomization," said Dr. Patrick Wen. "With the addition of AZD1390 to GBM AGILE, a global clinical trial, we are able to offer GBM patients access to the latest and most promising investigational therapies." AZD1390 is a brain penetrant ataxia telangiectasia mutant (ATM) kinase inhibitor that blocks ATM-dependent signaling and repair of DNA double strand breaks (DSBs) in the genome. Preclinically, AZD1390 exhibits activity in combination with agents such as radiation therapy that induce DSBs. AZD1390 has also been shown to achieve clinically relevant concentrations in resected glioblastoma tissue and suppress the ability of tumor cells to repair the DNA damage that is induced by radiation. Furthermore, data from a Phase I trial of AZD1390 with radiotherapy showed a manageable toxicity profile and preliminary efficacy for patients with recurrent glioblastoma and was shared in an oral presentation at the American Association for Cancer Research (AACR) Annual Meeting 2024. "At GCAR, we're dedicated to accelerating the development of critically needed therapies for patients facing rare and deadly diseases like glioblastoma," said Dr. Meredith Buxton, CEO and President of GCAR. "We believe master protocols and adaptive platform trials are a powerful way to expedite that progress. We're excited to collaborate with AstraZeneca and look forward to rapidly advancing the development of AZD1390 in GBM AGILE to help address the urgent need for more effective glioblastoma therapies." About Global Coalition for Adaptive Research (GCAR) Global Coalition for Adaptive Research (GCAR) is a 501(c)(3) non-profit corporation uniting physicians, clinical researchers, advocacy and philanthropic organizations, biotech/pharma companies, health authorities, and other key stakeholders in healthcare to expedite the discovery and development of treatments for patients with rare and deadly diseases. As the sponsor of innovative trials, including master protocols and adaptive platform trials, GCAR is dedicated to the advancement of science by modernizing clinical trials that support more efficient, less costly drug development. To learn more about GCAR and its initiatives, visit gcaresearch.org and follow us on social media @GCAResearch. SOURCE Global Coalition for Adaptive Research WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

EnteroBiotix Presents Positive Data in Liver Cirrhosis at EASL 2025 EBX-102 was well tolerated and showed multiple positive trends over placeboFindings support progression to a Phase 2b trial Glasgow, Scotland – 13 May 2025. EnteroBiotix Limited (‘EnteroBiotix)’ a clinical-stage biotechnology company developing best-in-class therapies for gut health, today announced positive results from its Phase 1b clinical trial evaluating EBX-102, the Company’s next-generation oral full-spectrum microbiome product, in patients with liver cirrhosis. The data were presented during an oral session at the European Association for the Study of the Liver (EASL) Congress 2025, held in Amsterdam, the Netherlands from 7–10 May 2025. The IMPuLCE (Intestinal Microbiota Product in Liver Cirrhosis and Encephalopathy) trial evaluated two ascending doses of EBX-102 in 24 patients with stable cirrhosis. Headline data were first announced in November 2024, and key findings presented at EASL included: Safety and tolerability: EBX-102 was well tolerated. Adverse events were mainly mild, self-limiting, and gastrointestinal in natureMicrobial engraftment: Shotgun metagenomic sequencing showed that engraftment of EBX-102 species occurred in both treatment cohorts across all measured timepoints. EBX-102 treatment also led to changes in microbiome-associated metabolic outputs, including short-chain fatty acids and secondary bile acidsClinical and bioanalytical trends: Patients treated with EBX-102 demonstrated decreased venous ammonia and increased stool ammonia concentrations in the high-dose cohort. Reductions in plasma lipopolysaccharide binding protein (LBP) – a marker of bacterial translocation and gut barrier function – were observed in the higher dose cohort. Additionally, Hospital Anxiety and Depression Scale (HADS) scores improved in patients receiving EBX-102, with trends persisting in the higher dose group to the end of follow-up Professor Ewan Forrest, Chief Investigator of the IMPuLCE trial and Honorary Professor, School of Cardiovascular & Metabolic Health at the University of Glasgow, said: “These positive initial results show that EBX-102 was well tolerated in patients with chronic liver disease. The observed EBX-102 species engraftment and positive clinical and bioanalytical trends are highly promising. These findings strongly support further clinical development of EBX-102 in decompensated liver cirrhosis, where it has the potential to become a first-in-class therapy.” Dr. James McIlroy, CEO of EnteroBiotix, added: "These positive Phase 1b results highlight the potential of EBX-102 as a novel therapeutic approach for patients with hepatic diseases, including liver cirrhosis. We look forward to progressing into Phase 2b clinical studies in patients with more advanced cirrhosis.”This presentation of significant results in liver cirrhosis follows EnteroBiotix’s recent presentation of positive results in irritable bowel syndrome with constipation (IBS-C) at Digestive Disease Week 2025. About EnteroBiotixEnteroBiotix is a clinical-stage biotechnology company developing microbiome-based therapeutics for irritable bowel syndrome (IBS) and other gastrointestinal and hepatic indications. Using its proprietary platform technology, EnteroBiotix creates next-generation therapies with differentiated characteristics designed to restore and enhance gut microbiome function. The company has established independent control over the supply chain for its drug formulations, with MHRA licensed manufacturing capabilities, and a donor programme called Number2®. About EBX-102EBX-102 is an encapsulated full-spectrum microbiome therapeutic with differentiated characteristics, such as consistency, high-bacterial diversity, and robust stability. EnteroBiotix controls the supply chain for its products through sophisticated MHRA-licensed manufacturing infrastructure together with its Number 2® brand, which ensures the safety, security, quality, and sufficiency of supply of microbiota obtained from healthy human donors. Media contacts EnteroBiotixDr James McIlroy, CEOinfo@enterobiotix.com ICR HealthcareNamrata Taak, Kris Lamenterobiotix@ICRHealthcare.com