NOVARTIS

The UK aims to lead in clinical research post-Brexit, focusing on regulatory reform, NHS workforce and data infrastructure, patient engagement, and innovative treatments. Emphasizing AI, digital platforms, and global trials, it seeks to accelerate drug development, improve trial efficiency, and address global health challenges.

The article highlights advancements in covalent drug discovery, focusing on inhibitors targeting KEAP1/NRF2, KRAS(G12C), WRN helicase, and other oncology targets. It discusses novel compounds like BBO-8520, RMC-9805, VVD-214, and PF-07817883, emphasizing their mechanisms, clinical trials, and potential in treating cancers and COVID-19. The piece also covers the evolution of covalent inhibitors beyond oncology, showcasing significant approvals and acquisitions.

Randomized Controlled Trials (RCTs) are the gold standard for clinical trials, but the pharmaceutical industry faces challenges in reusing vast amounts of patient data. External Control Data (ECD) offers a way to avoid duplicating research by complementing RCTs. Challenges include data silos, awareness, patient consent, and regulatory acceptance. ECD can expedite drug development, especially for rare diseases and pediatric populations, by leveraging historical data. The data42 platform by Novartis exemplifies efforts to harness ECD for accelerating research and addressing unmet medical needs.

Novartis' Scemblix (asciminib) approved by EC for Ph+ CML-CP treatment in adults previously treated with ≥2 TKIs, based on phase III ASCEMBL study showing higher response and lower discontinuation rates vs. Bosulif. Scemblix, a STAMP inhibitor, also FDA-approved, strengthens Novartis' oncology portfolio amid strategic focus on core pharma and therapeutic areas.

Scemblix (asciminib) received FDA approval for treating chronic myeloid leukemia (CML) in patients resistant or intolerant to at least two TKIs, showing superior MMR rates and lower discontinuation due to side effects compared to Bosulif. It's also approved for CML patients with the T315I mutation, offering a novel STAMP inhibitor mechanism.

Scemblix® (asciminib) approved in Europe for Ph+ CML-CP, showing a 25.5% MMR rate vs. 13.2% for Bosulif®, with lower discontinuation rates due to adverse reactions. It targets the ABL myristoyl pocket, offering a new option for patients resistant or intolerant to prior TKIs.

Novartis AG announces spin-off of Sandoz unit, aiming to form Europe's largest generic and biosimilar drug company. This decision follows a strategic review initiated in October, addressing shareholder concerns over Sandoz's performance.

Novartis AG announced a positive CHMP opinion recommending marketing authorization for Scemblix (asciminib) for Ph+ CML-CP treatment in adults previously treated with two or more TKIs. The EC will review the opinion, with a decision expected soon. Scemblix, if approved, offers a new CML treatment option targeting the ABL myristoyl pocket. Additionally, the EC approved Cosentyx for new juvenile arthritis indications, based on phase III study data.

Scemblix® (asciminib), with a unique STAMP mechanism, offers a new treatment for CML patients in Europe resistant or intolerant to prior TKIs. The CHMP's positive opinion, based on Phase III ASCEMBL trial data, shows Scemblix nearly doubles MMR rates and significantly lowers discontinuation due to adverse reactions compared to Bosulif®.

FDA approved Novartis' Tafinlar and Mekinist combo for advanced solid tumors with BRAF V600E mutation, following prior therapy progression and no alternatives. Approved for various cancers, including pediatric use, based on trials showing up to 80% response rates in some tumors.