NOVARTIS

Biotech has shifted from single-technology focus to a portfolio model, where a central team manages multiple companies across technologies and diseases. This model, exemplified by BridgeBio and Roivant Sciences, leverages expertise in various areas, raising $6 billion by 2020. It offers diversified investment, simplified governance, and attracts top talent, despite risks of systemic failure and inefficiencies. The model is reshaping biotech R&D, offering alternatives to traditional funding and increasing competition for assets and talent.

Scemblix® (asciminib), approved by the FDA, offers a new treatment for chronic myeloid leukemia (CML) patients resistant or intolerant to at least two tyrosine kinase inhibitors (TKIs). It showed a higher major molecular response rate and lower discontinuation due to side effects compared to Bosulif®. Scemblix is also effective for CML patients with the T315I mutation, marking a significant advancement in CML care.

Mesoblast Limited received FDA Fast Track designation for remestemcel-L to treat COVID-19 induced ARDS, aiming to expedite therapy development. A Phase 3 trial is ongoing, with promising results from a pilot study showing 75% of ventilator-dependent patients discharged after treatment. Mesoblast collaborates with Novartis for remestemcel-L's development.

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.

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.