Alnylam Pharmaceuticals

FDA approves BridgeBio Pharma's Attruby (acoramidis) for transthyretin amyloidosis cardiomyopathy, priced at $244,000 annually. The drug stabilizes misfolded TTR protein, showing survival and hospitalization benefits. Market competition heats up with Pfizer's tafamidis and potential rivals. BridgeBio aims for 30% market share, planning head-to-head trials.

Conference on global health and clinical trials addresses bioethics, regulations, patient recruitment, site selection, real-world data, data integration, outsourcing, vendor management, quality in trial conduct, risk-based monitoring, clinical auditing, financial planning, and innovative technologies. Focuses on networking, case studies, presentations, and round tables.

The Global Clinical Trials Connect 2025 conference will discuss futuristic advancements in clinical trials, focusing on innovative strategies, new technologies, and quality collaborations to address increased complexity. Topics include patient recruitment, site selection, real-world data, and risk-based monitoring, among others. The event targets senior-level professionals from pharmaceutical, bio-tech, and CRO companies, offering networking and case study opportunities.

The Global Clinical Trials Connect 2025 conference will discuss futuristic advancements in clinical trials, focusing on innovative strategies, new technologies, and quality collaborations to address increased complexity. Topics include patient recruitment, site selection, data integration, outsourcing, and risk-based monitoring. The event targets senior-level professionals from pharmaceutical, bio-tech, and CRO companies, offering networking and case study presentations.

Global equities rebounded in 3Q24, with health care stocks advancing in July and August, then falling in September. Biotechnology stocks led the sector, driven by factors like interest in innovative weight-loss drugs, easing supply constraints, increased health care utilization, and the sector's defensive nature. Key contributors included Applied Therapeutics, ARS Pharmaceuticals, and Revance Therapeutics. The fund's performance was aided by favorable stock selection, particularly in biotechnology, while detractors included HilleVax and Guardant Health. The outlook remains positive for biotechnology, supported by strong fundamentals, innovation, and M&A activity, despite risks from inflation and the IRA drug pricing measures.

EMA's COMP issues positive opinion on orphan drug designation for tobevibart and elebsiran in chronic hepatitis delta, based on Phase 2 SOLSTICE trial data. This follows U.S. FDA fast track designation, highlighting potential in treating CHD.