NOVARTIS

Focke Ziemssen, Michelle Sylvanowicz, Winfried M. Amoaku, Tariq Aslam, Bora Eldem, Robert P. Finger, Richard P. Gale, Laurent Kodjikian, Jean-François Korobelnik, Xiaofeng Lin, Anat Loewenstein, Paul Mitchell, Moira Murphy, David R. Owens, Nick Parker, Ian Pearce, Francisco J. Rodríguez, Jude Stern, S. James Talks, David T. Wong, Tien Yin Wong, and Jane Barratt disclose various financial interests including travel grants, personal fees, advisory board memberships, speaker fees, research sponsorship, and consulting roles with companies such as Allergan, AbbVie, Alimera, Bayer Healthcare, Biogen, Boehringer Ingelheim, Janssen, Novartis, Novo Nordisk, Merck, Regeneron, Roche, Sanofi, and others.

The ZEST trial failed to enroll enough ctDNA-positive patients for niraparib to prevent breast cancer recurrence. Lessons include starting ctDNA testing during treatment and focusing on high-risk patients. Only 40 patients were enrolled, with niraparib showing a median recurrence-free interval of 11.4 months vs. 5.4 for placebo.

US-centric forecasts for pharma drugs are inadequate. Key global markets like Europe, China, and Japan have unique regulatory, pricing, and market access requirements. Ethnic representation in clinical trials and local data are crucial for regulatory approval in these regions, impacting sales forecasts. Broadening forecasting horizons and understanding market-specific barriers are essential for accurate projections.

Chris Bishop discusses how his career in machine learning led to focusing on AI for Science, highlighting pivotal moments like Geoff Hinton's backprop paper. He explains the four prior paradigms of scientific discovery and introduces the fifth paradigm, where AI uses simulation results for training, offering significant speedups. Bishop emphasizes the transformative potential of AI in scientific discovery, particularly in molecular-level research and drug discovery, where AI can accelerate processes from years to months. He also mentions Project Aurora, an AI weather forecasting model 5,000 times faster than traditional methods, showcasing the power of foundation models in diverse applications.

Tasca Therapeutics announced a $52 million Series A financing to advance its drug discovery platform, progress CP-383 into Phase 1/2 clinical studies, and expand its drug candidate pipeline. The company identifies auto-palmitoylated proteins as unique therapeutic targets across various diseases. CP-383, a first-in-class molecule, has shown robust anti-cancer activity. The executive team brings extensive experience in drug discovery and development.

PROTAC technology enables targeted protein degradation, offering a novel approach to cancer therapeutics. With about 90 candidates in development, PROTACs can tackle 'undruggable' targets, potentially improving efficacy and overcoming resistance. The first PROTAC drug approval is expected by 2027, marking a significant milestone. Collaboration and proprietary platforms are accelerating advancements in this field.

The ZEST trial, evaluating niraparib for breast cancer recurrence prevention, was terminated early due to low ctDNA detection post-treatment. Lessons include starting ctDNA testing during treatment and focusing on high-risk patients for future trials.

The Breakthrough Therapies Market was valued at USD 150.6B in 2024, projected to reach USD 287.5B by 2029, growing at a CAGR of 13.80%. The report analyzes BTD's risks, benefits, fast-track drug development, therapy areas, leading companies, and market dynamics.

The global Duchenne muscular dystrophy market was valued at US$2.2 billion in 2023 and is projected to grow at a CAGR of 11.7% to reach US$7.4 billion by 2034, driven by advancements in gene therapy, novel treatment approvals, and increased awareness.

Pfizer's IBRANCE, a CDK4/6 inhibitor, extended progression-free survival by 15 months in metastatic breast cancer patients when combined with anti-HER2 therapies. IBRANCE, initially approved in 2015, is the first treatment of its kind to show benefit in HR+, HER2+ metastatic breast cancer.