Clinical Trial News

The FDA issued a draft guidance for conducting multiregional clinical trials (MRCT) in oncology, aiming to ensure data applicability to U.S. patients and medical practice. The guidance addresses the decreasing proportion of U.S. participants in MRCTs and aims to improve trial planning, design, conduct, and analysis.

Updated data from VERSATILE-002 trial shows Versamune® HPV + pembrolizumab well-tolerated with mOS of 30 months, ORR of 36%, and DCR of 77% in HPV16-positive R/M HNSCC patients. 21% experienced 90-100% tumor shrinkage. Phase 3 trial VERSATILE-003 planned to start this year.

The TACITO trial preliminary results show FMT increased 1-year PFS rate in mRCC patients receiving pembrolizumab + axitinib, meeting primary endpoint with 66.7% PFS in FMT vs. 35% in placebo. FMT also prolonged PFS and OS, with 52% ORR in FMT vs. 28% in placebo, though no complete responders. FMT was well-tolerated, requiring longer follow-up and further investigation.

AstraZeneca's Phase III NIAGARA study shows perioperative Imfinzi regimen significantly reduces risk of disease progression, recurrence, and death by 32% and 25% respectively in muscle-invasive bladder cancer patients, with no new safety signals.

PharmAust seeks European Medicines Agency's orphan medicinal product designation for monepantel (MPL) in treating ALS/MND, following a pre-submission meeting. The designation could offer incentives like protocol assistance and 10 years of market protection. PharmAust already received an orphan drug designation from the FDA in May, providing US incentives.

Boehringer Ingelheim's nerandomilast met primary endpoint in phase 3 trial for idiopathic pulmonary fibrosis (IPF), marking the first successful IPF phase-3 trial in a decade. The oral drug, a preferential inhibitor of phosphodiesterase 4B, received FDA breakthrough therapy designation in 2022. Boehringer plans to submit a new drug application to the FDA and other regulators based on these positive results.

Kissei announced Theramex's first commercial sale of YSELTY®, a GnRH receptor antagonist for uterine fibroids, in Germany. The drug, developed by Kissei and licensed to Theramex, received marketing approval in 2022 and offers a new treatment option for patients unable to take hormonal agents. Kissei is committed to improving patient QOL worldwide.

The HIMALAYA study's 5-year survival analysis shows 19.6% survival with STRIDE (durvalumab plus tremelimumab) vs 9.4% with sorafenib in unresectable hepatocellular carcinoma, with no new safety concerns.

Sanofi received FDA approval for a new manufacturing line to expand production of Beyfortus, a preventive therapy for RSV in infants. Beyfortus is the first long-acting monoclonal antibody approved by the FDA to prevent RSV-related lower respiratory tract disease in newborns and infants up to 24 months old. Sanofi has begun shipping doses to private healthcare providers and the CDC's Vaccines for Children program.

Metformin, a drug used to treat Type 2 diabetes, shows potential to slow aging in monkeys, improving cognitive abilities, reducing brain shrinkage, and restoring youthful cellular states. The drug also reduces chronic inflammation and age-related issues in multiple organs. The study bridges rodent and primate research, suggesting metformin could inform future clinical trials aimed at extending health span.