Clinical Trial News

Infusions of Vertex's Casgevy and bluebird bio's Lyfgenia for sickle cell treatment have begun, marking a breakthrough. Both companies face a slow uptake due to the lengthy treatment process, but interest is high. Vertex aims to activate 75 centers globally, while bluebird expects a consistent revenue stream from Lyfgenia.

FDA’s final guidance on DCTs clarifies HCP task log, inspection requirements, and data variability.

Clinical trials show GLP-1 mimics like liraglutide and semaglutide help children and adolescents lose weight, but long-term effects on growth and puberty are unknown. Experts debate the ethical implications and effectiveness of using BMI as a metric, emphasizing the need for more diverse studies and prolonged monitoring.

KEYNOTE-522 study shows Keytruda plus chemotherapy improves overall survival in high-risk early-stage TNBC, with 5-year OS at 86.6% vs. 81.7% with placebo. Keytruda's OS benefits support its role as perioperative therapy, despite higher immune-related AEs. Keytruda outperforms Roche's Tecentriq in TNBC treatment, though T-cell exhaustion and resistance remain challenges.

BMS-986365, a heterobifunctional ligand-directed degrader of WT AR, exhibits low intrinsic agonism and direct antagonism via ligand-binding domain occupancy, leading to deep AR pathway inhibition and resistance combat. It shows a favorable preclinical profile and interim clinical data, differentiating from Arvinas' AR degraders.

Oncology accelerated approvals often rely on non-comparative trials assessing response rate.

Angela Harmantas, Editor at Proactive, has 15+ years in equity markets, focusing on junior resource stocks. Proactive provides global finance news, spanning hubs like London and New York, covering markets from biotech to crypto. Proactive uses technology to enhance workflows, ensuring human-authored content.

Bubs Australia (ASX:BUB) navigated challenges post-pandemic, shifting focus from China to ASEAN markets and the USA. Despite boardroom turmoil and China sales disappointments, Bubs achieved growth in the USA, becoming a top 6 infant formula seller. The company aims for permanent US market access by October 2025, conducting a clinical trial to support this goal.

Senseonics and Ascensia Diabetes Care received FDA clearance for Eversense 365, the first implantable glucose sensor wearable for a year, doubling the current sensor's wear time. The CGM, implantable under the skin with a removable transmitter, is for adults with Type 1 or Type 2 diabetes and has an integrated CGM designation for automated insulin delivery systems. Senseonics CEO Tim Goodnow anticipates this as a significant catalyst for the company. The sensor requires less frequent calibration and is expected to launch in the fourth quarter, aiming to double sales and user base in 2025.

The shift from NAFLD/NASH to MASLD/MASH reflects a deeper understanding of hepatic conditions linked to metabolic disorders. Clinical trials for MASH are progressing rapidly, with FDA approvals and ongoing Phase 3 trials for drugs like resmetirom and pegozafermin.