NOVARTIS

CAR-T cell therapy, a novel immunotherapy combining mAbs' specificity and T cells' cytotoxicity, shows promise in treating advanced B cell malignancies. FDA-approved products like axicabtagen ciloleucel and tisagenlecleucel target CD19, while over 10 BCMA-targeted CAR-T products are in early-phase studies, showing high response rates despite common adverse events like CRS and NTX. Research continues to improve efficacy and manage side effects.

Over 20 drugmakers from Japan, the U.S., and Europe, including Eisai, Amgen, and Novartis, launched a $1 billion+ fund to support startups tackling antibiotic-resistant infections, with WHO and European Investment Bank participation.

Christopher Ma, Brian Feagan, Vipul Jairath, Reena Khanna, Parambir S. Dulai, and William J Sandborn disclose various financial relationships with pharmaceutical companies and research organizations, including grants, personal fees, and other forms of compensation, outside their submitted work. Robert Battat and Parambir S. Dulai report no conflict of interest.

In Shanghai, Novartis AG's 600 scientists aim to develop China-specific medicines, reflecting the industry's shift towards 'Discovered in China.' Despite investments, innovative therapeutics remain scarce. Recent reforms aim to lower drug development hurdles, with China's pharmaceutical sector aspiring to global R&D prominence. Regulatory changes and increased patent protection are expected to accelerate clinical development and drug approvals, fostering a more competitive international stance.

Ribociclib, a new CDK 4/6 inhibitor, showed significant improvement in progression-free survival for hormone receptor-positive breast cancer patients in the MONALEESA trial. Despite side effects like neutropenia and cardiac rhythm changes, its efficacy offers hope. However, its high cost and comparison with similar drugs like palbociclib remain concerns.

The multiple myeloma (MM) market is expected to grow significantly, driven by monoclonal antibodies elotuzumab and daratumumab. MM, a blood cancer, affects bone marrow and blood cell production. Treatments include chemotherapy, biologic therapy, and stem-cell transplant. New drugs like elotuzumab and daratumumab are anticipated to extend relapse intervals and increase treatment accessibility.

A multicenter retrospective analysis evaluated combination systemic therapies in 253 nccRCC patients from 2012-2024. Results showed limited antitumor activity, with differential responses among nccRCC subsets based on regimen type. Optimal nccRCC management remains an unmet need, highlighting the necessity for further research.

LEAP-012 trial showed len + pembro + TACE significantly improved PFS vs placebo + TACE in intermediate-stage HCC patients. OS data were immature, with a trend toward improvement. Safety profiles were consistent with known effects of the treatments. OS will be reassessed in future analyses.

Myelofibrosis (MF) is a severe myeloproliferative neoplasm characterized by dysregulated JAK/STAT signaling, leading to symptoms like splenomegaly and anemia. Ruxolitinib, a JAK1/JAK2 inhibitor, is approved for MF treatment, improving spleen size and symptoms but not reversing bone marrow fibrosis. Ongoing research explores combination therapies and new agents targeting anemia and fibrosis.

CAR-T cell therapy, a groundbreaking immunotherapy, modifies patient T-cells to target and destroy cancer cells, acting as 'living drugs.' With 11 products commercialized, including 7 FDA-approved, the therapy shows promise in treating blood cancers. Challenges include expanding to solid tumors, reducing 'vein-to-vein' time, and improving reimbursement. The industry is advancing through gene-transfer techniques, partnerships, and 'on-off' switches in CAR-T cells to enhance efficacy and safety.