NOVARTIS

In the Phase IIIB APPULSE-PNH study, oral Fabhalta® (iptacopan) improved average hemoglobin levels in adults with paroxysmal nocturnal hemoglobinuria (PNH) switched from anti-C5 therapies.

The US clinical trials market is projected to grow by USD 5 billion from 2024-2028, driven by increased drug trials and technological advancements, though rising costs pose a challenge.

The ARANOTE trial demonstrated that darolutamide plus androgen-deprivation therapy (ADT) without chemotherapy improved radiological progression-free survival by 46% in metastatic hormone-sensitive prostate cancer (mHSPC) patients compared to placebo plus ADT (HR 0.54, 95% CI 0.41-0.71, P<0.0001). The combination showed consistent benefits across subgroups, including high- and low-volume disease, and improved overall survival (OS) rates at 24 months (79.8% vs. 75.5%). Darolutamide also exhibited better tolerability with lower rates of fatigue and adverse event-related discontinuation compared to placebo.

The global precision medicine market was valued at USD 102.17 billion in 2024, expected to reach USD 470.53 billion by 2034, growing at a CAGR of 16.5%. Precision medicine involves personalized treatments based on genetic, environmental, and behavioral profiles. Key technologies include next-generation sequencing and bioinformatics. The U.S. leads with the NIH's 'All of Us Research Program' and collaborations like AstraZeneca's cancer research. AI tools like Tempus and Foundation Medicine enhance genomic analysis. Potential benefits include improved patient privacy, new data tools, and better FDA oversight. North America dominates the market, with Asia-Pacific expected to grow fastest due to strategic collaborations and government standards.

The MANIFEST-2 trial (NCT04603495) showed pelabresib (CPI-0610; Novartis) plus ruxolitinib (Jakafi; Incyte Corp) improved spleen volume, total symptom score (TSS), anemia, and bone marrow fibrosis (BMF) in JAK inhibitor-naïve myelofibrosis (MF) patients compared to ruxolitinib and placebo. At week 24, 65.9% achieved SVR35, and at week 48, 56.5% achieved SVR35 with pelabresib and ruxolitinib, versus 37.5% with placebo and ruxolitinib. The data will be presented at the 66th ASH Annual Meeting and Exposition on December 8, 2024.

The global RNA-targeted small molecules market is projected to grow from USD 2,453.5 million in 2024 to USD 4,311.7 million by 2034, driven by increased R&D investments and the potential of RNA-targeted therapies in treating genetic and chronic diseases.

The RNA-targeted small molecules market is expected to grow from USD 2,453.5 million in 2024 to USD 4,311.7 million by 2034, driven by R&D investments and the potential of RNA-targeted therapies in genetic and chronic diseases. These therapies offer precision and adaptability, reshaping the pharmaceutical landscape with opportunities for innovation and growth.

Micaela F. Bayard, MD, discusses the inclusion of patient voices in rheumatoid arthritis clinical trials at ACR Convergence 2024, emphasizing the need for diversity and representation.

EFPIA highlights EU's roadmap for precision oncology to address rising cancer burden, despite forecasted mortality rate declines. Stakeholder roundtable in Brussels aims to improve personalized cancer treatment. EU initiatives, including AI-powered cancer care and the DigiONE pilot, aim to advance precision medicine. A gold-standard cancer data management system is necessary for innovative precision medicine access, emphasizing standardized data formats, robust privacy, and professional education.

AI in pharma addresses R&D costs, supply chain complexity, and data management by accelerating drug development, automating compliance, managing large datasets, and optimizing clinical trials. Swiss startup Risklick uses AI to streamline clinical trial protocols and integrate historical data. As AI evolves, stakeholders must integrate these innovations to optimize research, enhance data-driven decisions, and meet patient needs.