NOVARTIS

AI in pharma could boost global operating profits by $254 billion by 2030, revolutionizing drug discovery, clinical trials, and personalized medicine. Key benefits include accelerated drug discovery, personalized care, enhanced clinical trials, optimized manufacturing, and advanced biomarker discovery. Real-world examples from companies like AstraZeneca, GSK, Novartis, and Sanofi highlight AI's transformative impact. Challenges include reproducibility, data quality, privacy, bias, and regulatory compliance, but solutions involve standardized datasets, data-sharing, encryption, diverse data sources, and proactive regulatory engagement.

Ractigen Therapeutics receives FDA orphan drug designation for RAG-18, a saRNA for DMD/Becker muscular dystrophy. Biogen's nusinersen shows higher dose efficacy in SMA. Cartesian Therapeutics initiates Descartes-15 CAR-T phase 1 trial for r/r multiple myeloma. Imugene's azer-cel CAR-T produces complete responses in r/r diffuse large B cell lymphoma. Novartis licenses Voyager Therapeutics' capsid for neurological target. BridgeBio Pharma's BBP-812 receives FDA RMAT designation for Canavan disease. Genprex plans to spin off its diabetes program into NewCo. NCI awards TransCode $2 million for TTX-MC138 RNA therapy in advanced solid tumors.

European biotech hubs like Cambridge, Leiden, Heidelberg, and Paris excel in scientific expertise, infrastructure, entrepreneurship, and financing. Despite strong clusters, Europe lags in translating research into commercial success, unlike the U.S., where Boston/Cambridge and the San Francisco Bay Area dominate. Factors like network, funding, patents, and jobs define successful clusters. German hubs, particularly Heidelberg, Berlin, and Munich, are innovative but face challenges in collaboration and venture funding, trailing behind Paris and London.

E.R. received honoraria from Eli Lilly, Novartis, Seagen; travel support from Gilead, Mundipharma, Novartis, Pfizer, Roche; research funding from Gilead, Menarini, MSD. F.A. consulted/advised AstraZeneca, Daiichi–Sankyo, Lilly, Novartis, Pfizer, Relay Tx, Roche; received travel support from AstraZeneca, GlaxoSmithKline, Lilly, Novartis, Roche; research funding from AstraZeneca, Daiichi, Eli Lilly, Novartis, Pfizer, Roche.

Sanofi and Regeneron plan to resubmit an FDA application for Dupixent in chronic spontaneous urticaria (CSU) by year-end, after a trial showed a 50% reduction in itch and urticaria activity scores. The companies also announced positive results in bullous pemphigoid and plan to seek U.S. approval for this indication. Despite challenges, Dupixent is used by 1 million patients across seven indications, with Japan being the first to approve it for CSU.

Various drugs and their statuses are listed, including IN-001 for anaphylaxis (Fast Track), Finereonone for heart failure (Phase 3), Tirzepatide for heart failure and obesity (Phase 3), TMB-001 for congenital ichthyosis (Phase 3), Bentracimab for ticagrelor reversal (Priority Review), Fabhalta for immunoglobulin A nephropathy (accelerated approval), RLS-0071 for acute graft-vs-host disease (Fast Track and Orphan Drug), CHIKV VLP for chikungunya (Priority Review), Diazoxide choline for Prader-Willi syndrome (Priority Review), Mirdametinib for neurofibromatosis type 1 (Priority Review), Setmelanotide for genetic obesity (Priority Review), AR882 for gout (Fast Track), SPN-830 for Parkinson disease (NDA review), BGB-16673 for chronic lymphocytic leukemia (Fast Track), Cabozantinib for neuroendocrine tumors (NDA review), Linvoseltamab for multiple myeloma (Complete Response Letter), Nivolumab plus ipilimumab for hepatocellular carcinoma (BLA review), Midomafetamine for PTSD (Complete Response Letter), and Tezepelumab-ekko for COPD (Breakthrough Therapy).

Bayer announced positive results from the phase I/II SOHO-01 study on BAY 2927088, an oral TKI for HER2-mutant NSCLC, showing a 72.1% ORR and manageable safety profile. The EU also approved Eylea 8mg for nAMD and DME, extending treatment intervals up to five months. Bayer aims to strengthen its pharma portfolio through label expansions and new drug development, despite recent stock declines.

A report by the Access to Medicine Foundation examines how 20 major pharmaceutical companies are addressing patient access in low- and middle-income countries (LMICs), highlighting progress and areas needing improvement. The report identifies 42 different approaches to measuring patient reach, with 19 companies actively tracking this, but no industry-wide consensus. It calls for clearer, more actionable commitments to ensure lifesaving products reach underserved populations, emphasizing the need for collaboration and refining measurement methods to address health inequities.

Novartis, selling Pluvicto and Lutathera for prostate and digestive tract cancers, plans to expand with new California site and Indianapolis facility to meet growing demand for radiopharmaceuticals, ensuring production resilience.

Recent FDA approvals of three new therapies for Duchenne muscular dystrophy have increased hope but also confusion among families. The lack of long-term data on gene therapy, especially in older and nonambulatory patients, and the potential for future treatment ineligibility due to antibody creation, adds to the complexity of decision-making. Families need better guidance from neurologists on therapy priorities and polypharmacy, as well as support to make informed decisions without judgment.