NOVARTIS

July's Molecules of the Month feature Novartis' molecular glue degrader for sickle cell disease, Pfizer's tinlorafenib for melanoma with brain metastasis, AstraZeneca's AZ-PRMT5i-1 for MTAP-deficient cancers, MTX-531 for dual EGFR/PI3K inhibition, and LifeMine Therapeutics' XC219 discovered via top-down approach. Paxalisib shows promising data in glioblastoma, and Takeda's OX2R-selective agonist offers potential in Alzheimer's disease.

Brazil emerges as a key hub for oncology clinical trials, with Roche leading in trials for drugs like Atezolizumab and Pertuzumab. The country focuses on prevalent cancers, with NSCLC trials dominating. Phase III trials are prevalent, supported by CROs, indicating Brazil's role in global cancer research.

Novartis receives FDA accelerated approval for Fabhalta (iptacopan) to reduce proteinuria in primary IgA nephropathy (IgAN), targeting the alternative complement pathway. Approval based on Phase III APPLAUSE-IgAN study interim analysis showing 44% reduction in proteinuria at 9 months vs. 9% with placebo. Continued approval contingent on eGFR data expected in 2025.

Novartis received FDA accelerated approval for Fabhalta, a complement inhibitor for reducing proteinuria in adults with primary IgAN at risk of rapid progression. Continued approval depends on Phase III study results expected in 2025. IgAN is a rare, progressive kidney disease affecting about 25 per million annually.

FDA approved Novartis' Fabhalta for reducing proteinuria in adults with primary immunoglobulin A nephropathy (IgAN) at risk of rapid progression. Approval based on Phase 3 APPLAUSE-IgAN study showing a 44% proteinuria reduction at nine months. Continued approval may depend on further study results expected in 2025. IgAN is a rare kidney disease affecting about 25 per million annually. Novartis is also developing two other IgAN therapies.

Fabhalta, a first-in-class complement inhibitor, achieved a 44% proteinuria reduction in Phase III APPLAUSE-IgAN study, significantly outperforming placebo. FDA granted accelerated approval for IgAN treatment, highlighting its potential to address a critical unmet need in rare kidney disease care.

D.A.A.V. and C.J.W. hold patents on LAG-3, with potential income from licensing. D.A.A.V. has multiple roles in biotech firms and receives funding from BMS and Novasenta. E.J.W. advises and holds stock in several biotech companies. A.H.S. has patents on the PD-1 pathway, receives research funding, and serves on various advisory boards. A.R.C. consults for Abound Bio.

The global biotechnology market is projected to grow from USD 1.38 trillion in 2023 to USD 4.25 trillion by 2033, at a CAGR of 11.8%. Growth is driven by chronic disease prevalence, personalized medicine, and R&D. North America leads with a 37.79% market share in 2023, followed by Asia Pacific at 23.99%. Key applications include bio-pharmacy, which dominated in 2023, and technologies like tissue engineering and regeneration.

The recurrent glioblastoma market is projected to grow at a CAGR of 5.85% from 2024 to 2034, driven by advancements in targeted therapies, immunotherapies, and combination treatments. Key developments include targeting EGFR and VEGF pathways, CAR T-cell therapy, and integrating tumor-treating fields with traditional therapies. Leading companies like Novartis, Pfizer, and Roche are innovating treatments, with the U.S. leading in patient pool and treatment advancements.